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## Protocol Section ### Identification Module **NCT ID:** NCT00653679 **Brief Title:** Exercise and Muscle Protein Signalling **Official Title:** The Impact of Resistance Versus Endurance Exercise on the Muscle Protein Signalling Response #### Organization Study ID Info **ID:** MEC0001 #### Organization **Class:** OTHER **Full Name:** Maastricht University Medical Center ### Status Module #### Completion Date **Date:** 2009-02 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2015-04-14 **Type:** ESTIMATED **Last Update Submit Date:** 2015-04-13 **Overall Status:** WITHDRAWN #### Primary Completion Date **Date:** 2009-02 **Type:** ESTIMATED #### Start Date **Date:** 2008-09 **Status Verified Date:** 2015-04 #### Study First Post Date **Date:** 2008-04-07 **Type:** ESTIMATED **Study First Submit Date:** 2008-04-01 **Study First Submit QC Date:** 2008-04-04 **Why Stopped:** A paper on the same topic came out just before we wanted to start our study. ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Maastricht University Medical Center #### Responsible Party **Investigator Affiliation:** Maastricht University Medical Center **Investigator Full Name:** Milou Beelen **Investigator Title:** Dr. **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** This study examines the muscle protein signalling response after resistance and endurance exercise. We hypothesize that the signalling response after endurance and resistance exercise will be partly similar. ### Conditions Module **Conditions:** - Muscle Protein Synthesis **Keywords:** - exercise - muscle protein signalling - protein synthesis ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER ##### Masking Info **Masking:** NONE **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** 1 hour of endurance or resistance exercise **Name:** exercise **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** The difference in phosphorylation of different signalling proteins in the cascade towards protein synthesis **Time Frame:** 1 day ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * healthy men * inactive Exclusion Criteria: * enrolled in a endurance and/or resistance training program **Healthy Volunteers:** True **Maximum Age:** 28 Years **Minimum Age:** 18 Years **Sex:** MALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Maastricht **Country:** Netherlands **Facility:** Maastricht University **State:** Limburg **Zip:** 6200 MD #### Overall Officials **Official 1:** **Affiliation:** Maastricht University **Name:** L.J.C van Loon, PhD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02510079 **Brief Title:** Emergent Expanded Access for ahSC Augmentation of Nerve Autografts After Severe Peripheral Nerve Injury. **Official Title:** Emergent Expanded Access to Use Autologous Human Schwann Cell Augmentation of Nerve Autograft Repair in a Single Patient With Severe Peripheral Nerve Injury #### Organization Study ID Info **ID:** 20150501 #### Organization **Class:** OTHER **Full Name:** University of Miami ### Status Module #### Expanded Access Info #### Last Update Post Date **Date:** 2021-11-16 **Type:** ACTUAL **Last Update Submit Date:** 2021-11-15 **Overall Status:** NO_LONGER_AVAILABLE **Status Verified Date:** 2021-11 #### Study First Post Date **Date:** 2015-07-28 **Type:** ESTIMATED **Study First Submit Date:** 2015-07-15 **Study First Submit QC Date:** 2015-07-26 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** The Miami Project to Cure Paralysis #### Lead Sponsor **Class:** OTHER **Name:** W. Dalton Dietrich #### Responsible Party **Investigator Affiliation:** University of Miami **Investigator Full Name:** W. Dalton Dietrich **Investigator Title:** Professor of Neurosurgery **Type:** SPONSOR_INVESTIGATOR ### Description Module **Brief Summary:** The primary purpose of this research study is to determine the safety of injecting ones own Schwann cells to augment sural nerve autografts after a severe, non-lacerating injury to the sciatic nerve has occurred. **Detailed Description:** The secondary purpose of this research project is to evaluate whether transplanted Schwann cells can enhance recovery of sensory and motor function. Emergency expanded access for a single patient was granted, as the patient was an ideal candidate for sural nerve repair augmented by autologous Schwann cells which could be harvested from a relatively small segment of sensory nerve. The cells were expanded using cell culture techniques and placed along with the sural nerve grafts wrapped by an FDA approved collagen matrix (Duragen). This study is being conducted at the Jackson Memorial and the University of Miami health systems. As a research subject the patient will be in this study for a total of 5 years from the date receiving Schwann cell transplant augmentation of sural nerve autografts. ### Conditions Module **Conditions:** - Sciatic Nerve Injury ### Design Module **Study Type:** EXPANDED_ACCESS ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** Schwann cells harvested from the sural nerve will be autologously transplanted along sural nerve autografts wrapped in a collagen matrix (Duragen). **Name:** Autologous human Schwann cells **Type:** BIOLOGICAL ### Eligibility Module **Eligibility Criteria:** Single patient with severe sciatic nerve injury enrolled under emergency request **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Miami **Country:** United States **Facility:** University of Miami **State:** Florida **Zip:** 33136 #### Overall Officials **Official 1:** **Affiliation:** University of Miami **Name:** W. Dalton Dietrich, MD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010523 - Term: Peripheral Nervous System Diseases - ID: D000009468 - Term: Neuromuscular Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000020196 - Term: Trauma, Nervous System ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M29440 - Name: Peripheral Nerve Injuries - Relevance: HIGH - As Found: Peripheral Nerve Injury - ID: M17685 - Name: Wounds and Injuries - Relevance: HIGH - As Found: Injury - ID: M13432 - Name: Peripheral Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M12411 - Name: Neuromuscular Diseases - Relevance: LOW - As Found: Unknown - ID: M22023 - Name: Trauma, Nervous System - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000059348 - Term: Peripheral Nerve Injuries - ID: D000014947 - Term: Wounds and Injuries ### Intervention Browse Module - Browse Branches - Abbrev: Repr - Name: Reproductive Control Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M266279 - Name: Estradiol 17 beta-cypionate - Relevance: LOW - As Found: Unknown - ID: M266280 - Name: Estradiol 3-benzoate - Relevance: LOW - As Found: Unknown - ID: M8108 - Name: Estradiol - Relevance: LOW - As Found: Unknown - ID: M234941 - Name: Polyestradiol phosphate - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02628379 **Brief Title:** Outcomes of FoundationOne Directed Therapy in Cancer of Unknown Primary **Official Title:** A Prospective Observational Trial Evaluating Outcomes of FoundationOne - Directed Matched Targeted Therapy in Patients With Cancer of Unknown Primary (CUP) #### Organization Study ID Info **ID:** RAP-CLT-15-010 #### Organization **Class:** INDUSTRY **Full Name:** Foundation Medicine ### Status Module #### Completion Date **Date:** 2017-03 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-07-10 **Type:** ACTUAL **Last Update Submit Date:** 2018-07-09 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2017-03 **Type:** ACTUAL #### Start Date **Date:** 2015-05 **Status Verified Date:** 2018-07 #### Study First Post Date **Date:** 2015-12-11 **Type:** ESTIMATED **Study First Submit Date:** 2015-12-01 **Study First Submit QC Date:** 2015-12-09 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Rush University Medical Center **Class:** UNKNOWN **Name:** Stormont-Vail Healthcare **Class:** OTHER **Name:** Jefferson Medical College of Thomas Jefferson University **Class:** OTHER **Name:** The Cleveland Clinic **Class:** OTHER **Name:** Sparrow Health System **Class:** UNKNOWN **Name:** Cancer & Hematology Centers of Western Michigan (CHCWM) **Class:** UNKNOWN **Name:** Allegheny Health Network **Class:** OTHER **Name:** St. Luke's Hospital and Health Network, Pennsylvania **Class:** UNKNOWN **Name:** Solano Hematology Oncology **Class:** UNKNOWN **Name:** Horizon Oncology Center **Class:** OTHER **Name:** Comprehensive Blood and Cancer Center **Class:** OTHER **Name:** Lancaster Cancer Center **Class:** UNKNOWN **Name:** Western Maryland Health Center **Class:** UNKNOWN **Name:** Valley Medical Oncology **Class:** UNKNOWN **Name:** Cape Fear Valley Health System **Class:** UNKNOWN **Name:** Tri-County Hematology Oncology **Class:** UNKNOWN **Name:** Hematology & Oncology Associates **Class:** UNKNOWN **Name:** Broome Oncology **Class:** UNKNOWN **Name:** Orchard Healthcare **Class:** UNKNOWN **Name:** Ashland Bellefonte Cancer Center **Class:** UNKNOWN **Name:** Tennessee Cancer Specialists **Class:** UNKNOWN **Name:** North Shore Hematology Oncology **Class:** UNKNOWN **Name:** Good Samaritan **Class:** UNKNOWN **Name:** Zangmeister Cancer Center **Class:** UNKNOWN **Name:** Watson clinic **Class:** UNKNOWN **Name:** Oncology Consultants **Class:** UNKNOWN **Name:** Oncology Hematology Care **Class:** OTHER **Name:** Northern Westchester Hospital **Class:** UNKNOWN **Name:** Peter MacCallum Cancer Center Trials Unit #### Lead Sponsor **Class:** INDUSTRY **Name:** Foundation Medicine #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The goal of the current study is to determine whether Foundation Medicine's next generation sequencing assay, called FoundationOne, will provide information that allows physicians to make treatment decisions using targeted therapies in clinical trials or FDA approved therapies, including "off-label" agents, that result in superior OS compared to historical outcomes for standard CUP therapy. ### Conditions Module **Conditions:** - Neoplasms, Unknown Primary - CUP - Metastatic Disease - Poorly Differentiated Adenocarcinoma - Poorly Differentiated Carcinoma - Squamous Carcinoma Poorly Differentiated **Keywords:** - Genomic Testing - Cancer of Unknown Primary - CUP ### Design Module #### Design Info **Observational Model:** CASE_ONLY **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 125 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients put on targeted therapies matched to specific genomic alterations. **Label:** Class 1, 2, or 3 alterations, with targeted therapy #### Arm Group 2 **Description:** Patients put on therapy determined by tissue of origin testing (e.g., CancerTYPE ID) **Label:** Site-specific therapy determined by tissue of origin testing #### Arm Group 3 **Description:** Patients put on empiric treatment at physician discretion **Label:** Empiric CUP therapy ### Outcomes Module #### Primary Outcomes **Measure:** Proportion of CUP patients who receive matched targeted therapy. **Time Frame:** Baseline visit #### Secondary Outcomes **Measure:** Overall survival (OS) in CUP patients receiving matched targeted therapy based on FoundationOne versus internal control CUP patients not receiving FoundationOne-directed therapy **Time Frame:** Every three months until death, [20 months] ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Patients with a histologically or cytologically confirmed diagnosis of metastatic or advanced unresectable cancer of unknown primary including adenocarcinoma, poorly differentiated adenocarcinoma, poorly differentiated carcinoma, or squamous carcinoma. 2. To be categorized as CUP, the following clinical evaluations must have been performed without identification of an anatomic primary site: medical history, physical examination, chemistry profile, blood counts, serum PSA (men), CT scans of chest/abdomen/pelvis, specific evaluation of symptomatic areas. 3. Sufficient Formalin Fixed Paraffin Embedded tissue from cancer of study will allow previously completed profiling panels other than for treatment assignment; however, FoundationOne profiling is required as part of this study. Adequate tumor tissue must remain, in the estimate of the consenting physician, to confirm genomic alterations in enrolled patients. Previous unknown primary available for FoundationOne testing. (Note: This FoundationOne® profiling is also allowed and is not required to be FoundationOne repeated.) (see Appendix A and Appendix B) 4. First and second line patients enrolling in this study must have an Eastern Cooperative Oncology Group (ECOG) Performance Status score of 0 to 2. Third line patients enrolling in this study must have an ECOG Performance Status score of 0 to 1 (Appendix C). 5. Patients must have measurable or evaluable disease per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 (Appendix D). 6. Age greater than or equal to 18 years. 7. Patients are considered potential candidates for treatment with targeted therapy. 8. Willingness and ability to comply with study and follow-up procedures. 9. Ability to understand the nature of this study and give written informed consent. 10. The presence of other active cancers is not allowed, unless indolent and not requiring therapy. Patients with early stage cancer who have received definitive local treatment and are considered unlikely to recur are eligible. All patients with previously treated in situ carcinoma (i.e., non-invasive) are eligible, as are patients with history of non-melanoma skin cancer. Exclusion Criteria: 1. Patients who have received three or more lines of systemic therapy for cancer of unknown primary. 2. Patients who have previously received matched targeted therapy for the same Class 1 alteration (see Table 1) or the same drug. 3. Patients with treatable CUP syndrome, including the following: * extragonadal germ cell syndrome * neuroendocrine carcinoma * adenocarcinoma isolated to axillary lymph nodes (women) * peritoneal carcinomatosis (women) * squamous cell carcinoma limited to cervical, supraclavicular, or inguinal lymph nodes * single resectable metastasis 4. Previously untreated brain metastases. Patients who have received radiation or surgery for brain metastases are eligible if therapy was completed at least 2 weeks prior to study entry and there is no evidence of central nervous system disease progression, mild neurologic symptoms, and no requirement for chronic corticosteroid therapy. Enzyme-inducing anticonvulsants are contraindicated. 5. Pregnant or lactating. 6. Psychological, familial, sociologic, or geographic conditions that do not permit compliance with the protocol. **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Newly diagnosed and previously treated patients with CUP. ### Contacts Locations Module #### Locations **Location 1:** **City:** Cambridge **Country:** United States **Facility:** Foundation Medicine, Inc **State:** Massachusetts **Zip:** 02141 ### References Module #### References **Citation:** Drilon A, Wang L, Arcila ME, Balasubramanian S, Greenbowe JR, Ross JS, Stephens P, Lipson D, Miller VA, Kris MG, Ladanyi M, Rizvi NA. Broad, Hybrid Capture-Based Next-Generation Sequencing Identifies Actionable Genomic Alterations in Lung Adenocarcinomas Otherwise Negative for Such Alterations by Other Genomic Testing Approaches. Clin Cancer Res. 2015 Aug 15;21(16):3631-9. doi: 10.1158/1078-0432.CCR-14-2683. Epub 2015 Jan 7. **PMID:** 25567908 **Citation:** Ross JS, Cronin M. Whole cancer genome sequencing by next-generation methods. Am J Clin Pathol. 2011 Oct;136(4):527-39. doi: 10.1309/AJCPR1SVT1VHUGXW. **PMID:** 21917674 **Citation:** Frampton GM, Fichtenholtz A, Otto GA, Wang K, Downing SR, He J, Schnall-Levin M, White J, Sanford EM, An P, Sun J, Juhn F, Brennan K, Iwanik K, Maillet A, Buell J, White E, Zhao M, Balasubramanian S, Terzic S, Richards T, Banning V, Garcia L, Mahoney K, Zwirko Z, Donahue A, Beltran H, Mosquera JM, Rubin MA, Dogan S, Hedvat CV, Berger MF, Pusztai L, Lechner M, Boshoff C, Jarosz M, Vietz C, Parker A, Miller VA, Ross JS, Curran J, Cronin MT, Stephens PJ, Lipson D, Yelensky R. Development and validation of a clinical cancer genomic profiling test based on massively parallel DNA sequencing. Nat Biotechnol. 2013 Nov;31(11):1023-31. doi: 10.1038/nbt.2696. Epub 2013 Oct 20. **PMID:** 24142049 **Citation:** Johnson DB, Dahlman KH, Knol J, Gilbert J, Puzanov I, Means-Powell J, Balko JM, Lovly CM, Murphy BA, Goff LW, Abramson VG, Crispens MA, Mayer IA, Berlin JD, Horn L, Keedy VL, Reddy NM, Arteaga CL, Sosman JA, Pao W. Enabling a genetically informed approach to cancer medicine: a retrospective evaluation of the impact of comprehensive tumor profiling using a targeted next-generation sequencing panel. Oncologist. 2014 Jun;19(6):616-22. doi: 10.1634/theoncologist.2014-0011. Epub 2014 May 5. **PMID:** 24797823 **Citation:** Tsimberidou AM, Iskander NG, Hong DS, Wheler JJ, Falchook GS, Fu S, Piha-Paul S, Naing A, Janku F, Luthra R, Ye Y, Wen S, Berry D, Kurzrock R. Personalized medicine in a phase I clinical trials program: the MD Anderson Cancer Center initiative. Clin Cancer Res. 2012 Nov 15;18(22):6373-83. doi: 10.1158/1078-0432.CCR-12-1627. Epub 2012 Sep 10. **PMID:** 22966018 **Citation:** Eisenhauer EA, Therasse P, Bogaerts J, Schwartz LH, Sargent D, Ford R, Dancey J, Arbuck S, Gwyther S, Mooney M, Rubinstein L, Shankar L, Dodd L, Kaplan R, Lacombe D, Verweij J. New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1). Eur J Cancer. 2009 Jan;45(2):228-47. doi: 10.1016/j.ejca.2008.10.026. **PMID:** 19097774 **Citation:** Massard C, Loriot Y, Fizazi K. Carcinomas of an unknown primary origin--diagnosis and treatment. Nat Rev Clin Oncol. 2011 Nov 1;8(12):701-10. doi: 10.1038/nrclinonc.2011.158. **PMID:** 22048624 **Citation:** Pavlidis N, Pentheroudakis G. Cancer of unknown primary site. Lancet. 2012 Apr 14;379(9824):1428-35. doi: 10.1016/S0140-6736(11)61178-1. Epub 2012 Mar 12. **PMID:** 22414598 **Citation:** Stella GM, Senetta R, Cassenti A, Ronco M, Cassoni P. Cancers of unknown primary origin: current perspectives and future therapeutic strategies. J Transl Med. 2012 Jan 24;10:12. doi: 10.1186/1479-5876-10-12. **PMID:** 22272606 **Citation:** Gray SW, Hicks-Courant K, Cronin A, Rollins BJ, Weeks JC. Physicians' attitudes about multiplex tumor genomic testing. J Clin Oncol. 2014 May 1;32(13):1317-23. doi: 10.1200/JCO.2013.52.4298. Epub 2014 Mar 24. **PMID:** 24663044 **Citation:** Varadhachary GR, Raber MN. Carcinoma of unknown primary site. N Engl J Med. 2014 Nov 20;371(21):2040. doi: 10.1056/NEJMc1411384. No abstract available. **PMID:** 25409386 **Citation:** Petrakis D, Pentheroudakis G, Voulgaris E, Pavlidis N. Prognostication in cancer of unknown primary (CUP): development of a prognostic algorithm in 311 cases and review of the literature. Cancer Treat Rev. 2013 Nov;39(7):701-8. doi: 10.1016/j.ctrv.2013.03.001. Epub 2013 Apr 6. **PMID:** 23566573 **Citation:** Hainsworth JD, Rubin MS, Spigel DR, Boccia RV, Raby S, Quinn R, Greco FA. Molecular gene expression profiling to predict the tissue of origin and direct site-specific therapy in patients with carcinoma of unknown primary site: a prospective trial of the Sarah Cannon research institute. J Clin Oncol. 2013 Jan 10;31(2):217-23. doi: 10.1200/JCO.2012.43.3755. Epub 2012 Oct 1. **PMID:** 23032625 **Citation:** Oken MM, Creech RH, Tormey DC, Horton J, Davis TE, McFadden ET, Carbone PP. Toxicity and response criteria of the Eastern Cooperative Oncology Group. Am J Clin Oncol. 1982 Dec;5(6):649-55. No abstract available. **PMID:** 7165009 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000009385 - Term: Neoplastic Processes - ID: D000010335 - Term: Pathologic Processes - ID: D000018307 - Term: Neoplasms, Squamous Cell ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M5534 - Name: Carcinoma - Relevance: HIGH - As Found: Carcinoma - ID: M5550 - Name: Carcinoma, Squamous Cell - Relevance: HIGH - As Found: Squamous Carcinoma - ID: M12307 - Name: Neoplasm Metastasis - Relevance: HIGH - As Found: Metastatic Disease - ID: M3585 - Name: Adenocarcinoma - Relevance: LOW - As Found: Unknown - ID: M12327 - Name: Neoplasms, Unknown Primary - Relevance: HIGH - As Found: Neoplasms, Unknown Primary - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M12330 - Name: Neoplastic Processes - Relevance: LOW - As Found: Unknown - ID: M20451 - Name: Neoplasms, Squamous Cell - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002277 - Term: Carcinoma - ID: D000009362 - Term: Neoplasm Metastasis - ID: D000002294 - Term: Carcinoma, Squamous Cell - ID: D000009382 - Term: Neoplasms, Unknown Primary ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05055479 **Acronym:** BHReICU **Brief Title:** Nosocomial Outbreak of BHRe in an Intensive Care Unit During SARS-CoV-2 Pandemic **Official Title:** Nosocomial Outbreak of Monoclonal VIM Carbapenemase Producing Enterobacter Cloacae in an Intensive Care Unit During SARS-CoV-2 Pandemic #### Organization Study ID Info **ID:** PI2021_843_0187 #### Organization **Class:** OTHER **Full Name:** Centre Hospitalier Universitaire, Amiens ### Status Module #### Completion Date **Date:** 2022-02-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-01-18 **Type:** ACTUAL **Last Update Submit Date:** 2023-01-17 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-01-01 **Type:** ACTUAL #### Start Date **Date:** 2021-08-01 **Type:** ACTUAL **Status Verified Date:** 2023-01 #### Study First Post Date **Date:** 2021-09-24 **Type:** ACTUAL **Study First Submit Date:** 2021-09-15 **Study First Submit QC Date:** 2021-09-21 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Centre Hospitalier Universitaire, Amiens #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Multidrug Resistant (MDR) bacteria have become a major worldwide public health challenge and hospitals are now increasingly faced with management of local outbreaks involving such pathogens. Especially, intensive care units (ICU) provide an ideal background for outbreaks caused by MDR bacteria among which carbapenemase-producing Enterobacteriaceae (CPE) can be found. Among CPE involved in ICU outbreaks, VIM producers have been reported worldwide, and described as especially difficult to control. The COVID-19 pandemic and all of the measures health workers have to implement to fight the spread of SARS-Cov-2 have also impacted the management of such outbreaks. In this retrospective study, the investigators aim to describe the management of an outbreak caused by a VIM-producing Enterobacter cloacae strain during the 2020 COVID-19 pandemic in an ICU, and show the importance of concerted measures and actions implemented at multiple levels to prevent the spread of this MDR strain. **Detailed Description:** Multidrug Resistant (MDR) bacteria have become a major worldwide public health challenge and hospitals are now increasingly faced with management of local outbreaks involving such pathogens. Especially, intensive care units (ICU) provide an ideal background for outbreaks caused by MDR bacteria among which carbapenemase-producing Enterobacteriaceae (CPE) can be found. Among CPE involved in ICU outbreaks, VIM producers have been reported worldwide, and described as especially difficult to control. The COVID-19 pandemic and all of the measures health workers have to implement to fight the spread of SARS-Cov-2 have also impacted the management of such outbreaks. The likely source of an outbreak is not always easy to pinpoint, but some common sources can be cited: an index patient with a history of hospitalization abroad, a contaminated instrument, and/or an environmental reservoir. CPE outbreaks in ICU usually have multifactorial origins, thus needing to combine several actions to be controlled. Infection and prevention control (IPC) measures habitually rely on patients screening, use of contact precautions, staff education, enhanced environmental cleaning, cohorting of patients and staff as well as a proper antimicrobial stewardship. Investigation of these outbreaks and implementation of IPC measures are ensured by various professional categories in the hospital. This implies a tight cooperation and communication between all involved healthcare workers. In this retrospective study, the investigators aim to describe the management of an outbreak caused by a VIM-producing Enterobacter cloacae strain during the 2020 COVID-19 pandemic in an ICU, and show the importance of concerted measures and actions implemented at multiple levels to prevent the spread of this MDR strain. ### Conditions Module **Conditions:** - Enterobacteriaceae Infections - Hospital-Acquired Infection **Keywords:** - outbreak - Enterobacteriaceae Infections - Hospital-Acquired Infection - integrated approach - VIM ### Design Module #### Design Info **Observational Model:** CASE_ONLY **Time Perspective:** RETROSPECTIVE #### Enrollment Info **Count:** 14 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Outcomes Module #### Primary Outcomes **Description:** Management of VIM-producing Enterobacter cloacae strain care in ICU. All types of therapeutics used to manage atrial fibrillation are collected **Measure:** Change in care type of BHRe in ICU **Time Frame:** 1 month ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * patients who have stayed in ICU * patients with positive sample to E. cloacae VIM between March and October 2020 Exclusion Criteria: * patients who didn't agreed to be included **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** patients who have stayed in ICU and with positive sample to E. cloacae VIM between March and October 2020. ### Contacts Locations Module #### Locations **Location 1:** **City:** Amiens **Country:** France **Facility:** CHU Amiens Picardie **State:** Picardie **Zip:** 80054 ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes - ID: D000016905 - Term: Gram-Negative Bacterial Infections - ID: D000001424 - Term: Bacterial Infections - ID: D000001423 - Term: Bacterial Infections and Mycoses ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M10283 - Name: Infections - Relevance: HIGH - As Found: Infection - ID: M6368 - Name: Communicable Diseases - Relevance: HIGH - As Found: Infection - ID: M7918 - Name: Enterobacteriaceae Infections - Relevance: HIGH - As Found: Enterobacteriaceae Infections - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown - ID: M4722 - Name: Bacterial Infections - Relevance: LOW - As Found: Unknown - ID: M19249 - Name: Gram-Negative Bacterial Infections - Relevance: LOW - As Found: Unknown - ID: M12136 - Name: Mycoses - Relevance: LOW - As Found: Unknown - ID: M4721 - Name: Bacterial Infections and Mycoses - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007239 - Term: Infections - ID: D000003141 - Term: Communicable Diseases - ID: D000004756 - Term: Enterobacteriaceae Infections ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04036279 **Acronym:** NANOS **Brief Title:** NANOS Neck Preserving Hip Stem **Official Title:** A Prospective, Multicenter, Clinical Outcome Study of the NANOS Neck Preserving Stem in Patients With Degenerative Hip Joint Disease #### Organization Study ID Info **ID:** R11014-1 #### Organization **Class:** INDUSTRY **Full Name:** Smith & Nephew, Inc. ### Status Module #### Completion Date **Date:** 2023-03 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-06-07 **Type:** ACTUAL **Last Update Submit Date:** 2023-06-05 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2023-03 **Type:** ACTUAL #### Start Date **Date:** 2010-01-21 **Type:** ACTUAL **Status Verified Date:** 2023-06 #### Study First Post Date **Date:** 2019-07-29 **Type:** ACTUAL **Study First Submit Date:** 2019-07-25 **Study First Submit QC Date:** 2019-07-25 **Why Stopped:** This study was originally designed to meet MDR commitments. MDR Approval was secured without this study. Therefore, this study does not fulfil its role as a regulatory body commitment. ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Smith & Nephew Orthopaedics AG #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The objective of this study is to determine the long-term safety and effectiveness of the NANOS neck preserving stem in terms of radiographic and clinical performance as well as short-, mid- and long-term survivorship **Detailed Description:** The goal of this multicenter clinical observation is to validate short-, mid-, and long-term outcome (efficacy and safety) of the NANOS neck preserving hip stem. Effectiveness Measures: * Evaluation of function, range of motion (ROM) and pain as assessed by Harris Hip Score, HOOS Score \[4-6\], UCLA Activity Rating \[7\] * Radiographic changes as defined by radiolucent lines, osteolysis, hypo- and hypertrophy, implant loosening or migration Safety evaluations: * Intra- and peri-operative device-related adverse events (AE) and complications up to discharge * Postoperative AE up to 10 years ### Conditions Module **Conditions:** - Primary Osteoarthritis - Secondary Osteoarthritis - Dysplasia Osteoarthritis - Avascular Femoral Neck Necrosis - Post-Traumatic Femoral Neck Necrosis - Intact Femoral Neck With Good Bone Quality **Keywords:** - NANOS - Hip - Neck Preserving Stem ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 130 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Name:** Total Hip Arthroplasty **Type:** PROCEDURE ### Outcomes Module #### Other Outcomes **Description:** Any reported safety events or complications up to 10 years post study procedure **Measure:** Intra- and peri-operative device-related adverse events (AE) and complications up to discharge **Time Frame:** 10 years **Description:** Any reported safety events or complications up to 10 years post study procedure **Measure:** Postoperative AE up to 10 years **Time Frame:** 10 years **Description:** Harris Hip Score is a joint specific score that consists of 10 items covering domains of pain (1 item, 0-44 points), function (7 items, 0-47 points), functional activities, absence of deformity (1 item, 0 or 4 points), and hip range of motion (2 items, 0-5 points). Scores range from 0 (worst) to 100 (best). HHS scores for each subject will further be categorized as follows: Excellent (90-100); Good (80-89); Fair (70-79); Poor (60-69) and; Very poor (\<60). **Measure:** Evaluation of function, range of motion and pain assessed by Harris Hip Score **Time Frame:** 10 years **Description:** HOOS consists of 5 subscales: Pain, other Symptoms, Function in daily living (ADL), Function in sport and recreation (Sport/Rec) and hip related QOL. The last week is taken into consideration when answering the questions. Standardized answer options are given (5 Likert boxes) and each question gets a score from 0 to 4. A normalized score (100 indicating no symptoms and 0 indicating extreme symptoms) is calculated for each subscale. **Measure:** Evaluation of function, range of motion and pain as assessed by HOOS score **Time Frame:** 10 years **Description:** UCLA Activity Score is a measure of physical activity levels in subjects undergoing total joint arthroplasty. The scale is from 1 - 10 with higher values indicating greater physical function. **Measure:** Evaluation of function, range of motion and pain as assessed by UCLA Activity Rating **Time Frame:** 10 years **Description:** Any radiographic changes up to 10 years post study procedure **Measure:** Radiographic changes as defined by resorption, radiolucent lines, osteolysis, hypo- and hypertrophy, implant migration or loosening **Time Frame:** 10 years #### Primary Outcomes **Description:** Revision for any reason. Implant survivorship at 10 years post study procedure. A revision is a surgical procedure of the study hip where one or more of the study components are removed and replaced with new implants **Measure:** Long term survivorship of Nanos **Time Frame:** 10 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Inclusion criteria: * Patient requires Primary total hip replacement (THR) to the affected side, unilateral or bilateral. Bilateral only if \>3 months after the first THR * Patient agreed to participate in the MCO study by signing the Informed Consent form. * Age of patient at date of surgery 18 to 65 years * Patient is likely to comply with study follow-up requirements Exclusion criteria: * Previously failed endoprosthesis and /or THR components in relevant hip * Patient has proven osteoporosis * Pronounced coxa valga with a femoral neck angle \> 145º * Pronounced coxa vara with a femoral neck angle \< 125º * History of infection in the affected joint; systemic infections * Grossly insufficient femoral or acetabular bone stock in the involved hip where a revision cup is indicated * Spinal disease with neurologic movement disorders * Alcoholism or addictive disorders * ASA score is 3 or 4 * Body mass index (BMI) \> 30 * Patient is pregnant or being pregnant during follow up intervals * Patients understanding of the language is insufficient for understanding the Patient Information and Consent **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Clinic for Orthopedics ### Contacts Locations Module #### Locations **Location 1:** **City:** Marburg **Country:** Germany **Facility:** University Marburg, Orthopedics and Rheumatology **Zip:** 35043 **Location 2:** **City:** Alkmaar **Country:** Netherlands **Facility:** Medisch Centrum Alkmaar **Location 3:** **City:** Piekary Śląskie **Country:** Poland **Facility:** Samodzielny Publiczny Wojewodzki Spital **Zip:** 41-940 #### Overall Officials **Official 1:** **Affiliation:** Phillips University Marburg, Germany **Name:** Turgay Efe, Dr.med **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001168 - Term: Arthritis - ID: D000007592 - Term: Joint Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000012216 - Term: Rheumatic Diseases - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases ### Condition Browse Module - Browse Leaves - ID: M12926 - Name: Osteoarthritis - Relevance: HIGH - As Found: Osteoarthritis - ID: M12307 - Name: Neoplasm Metastasis - Relevance: LOW - As Found: Unknown - ID: M10621 - Name: Joint Diseases - Relevance: LOW - As Found: Unknown - ID: M17912 - Name: Osteoarthritis, Hip - Relevance: LOW - As Found: Unknown - ID: M12284 - Name: Necrosis - Relevance: HIGH - As Found: Necrosis - ID: M4476 - Name: Arthritis - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: M15045 - Name: Rheumatic Diseases - Relevance: LOW - As Found: Unknown - ID: M6323 - Name: Collagen Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010003 - Term: Osteoarthritis - ID: D000009336 - Term: Necrosis ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03742479 **Brief Title:** Restylane Silk Microinjections to Cheeks **Official Title:** A Single Center, Prospective, Open-label, Clinical Trial Using Micro-injections of Transparent Hyaluronic Acid Gel (Restylane® Silk) for Rejuvenation of the Aging Cheek #### Organization Study ID Info **ID:** Silkcheek-2018-01 #### Organization **Class:** OTHER **Full Name:** Goldman, Butterwick, Fitzpatrick and Groff ### Status Module #### Completion Date **Date:** 2020-02 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2019-05-03 **Type:** ACTUAL **Last Update Submit Date:** 2019-05-01 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2019-12 **Type:** ESTIMATED #### Start Date **Date:** 2019-02-26 **Type:** ACTUAL **Status Verified Date:** 2019-05 #### Study First Post Date **Date:** 2018-11-15 **Type:** ACTUAL **Study First Submit Date:** 2018-11-08 **Study First Submit QC Date:** 2018-11-14 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Goldman, Butterwick, Fitzpatrick and Groff #### Responsible Party **Investigator Affiliation:** Dermatology Cosmetic Laser Medical Associates of La Jolla, Inc. **Investigator Full Name:** Isabella Guiha **Investigator Title:** Certified Clinical Research Coordinator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** True **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The objective of this study is to determine the efficacy and safety of Restylane® Silk microinjections when used in a grid-like injection pattern for the correction of fine lines to the cheeks. **Detailed Description:** Silk for correction of mid to low cheek fine lines and wrinkles. Twenty (20) subjects will be enrolled. Each subject will receive Restylane® Silk to the entire cheeks. The defined area will be defined as the following: line extending from the upper margin of the nasal ala to the upper margin of the tragus, from the tragus to 1 cm above the mandibular angle, from 1 cm above the mandibular angle to 1 cm above the pre-jowl sulcus, and from 1 cm above the pre-jowl sulcus to the upper margin of the nasal ala. The injections will be delivered intradermally via multiple 0.02 cc microinjections distributed in a grid array pattern with 1-2 cm between each injection point (see figure 1; injections will extend 1 cm above the illustration). Following completion of injection treatment, manual massage will be applied to the full area to promote even distribution of the product. Each subject will undergo a total of three treatment sessions, one month apart, day 1, week 4, and week 8. The maximum amount of Restylane to be used per treatments session/per cheek is 1.5cc (not to exceed a total of 9 cc) per treated patient. Three-dimensional digital photography utilizing the Vectra 3D System (Canfield) will be utilized to document pre-treatment status, sites of injection, and post-treatment effect. Subjects will be followed up at 4 weeks, 8 weeks, post-treatment day 90, and day 180. Objective measures of efficacy will be me performed pre-treatment, at day 90 and 180. ### Conditions Module **Conditions:** - Wrinkle ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP **Intervention Model Description:** Single Group, Open Label Study ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 20 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Device: Restylane® Silk **Label:** Hyaluronic Acid Microinjection **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Hyaluronic Acid Microinjection **Description:** Injectable Hyaluronic Acid (HA) **Name:** Restylane® Silk **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Change in Investigator five point global aesthetic improvement score from Baseline at all follow up visits through month 6 using Investogator's Global Aesthetic Improvement Scale **Measure:** Investigator five point global aesthetic improvement score **Time Frame:** Baseline to 6 - Months Post Final Treatment **Description:** Change in elasticity from baseline to the last visit measured using a Cutometer **Measure:** Cutometer Measured Skin Elaticity **Time Frame:** Baseline to 6 - Months Post Final Treatment **Description:** Change in transepidermal waterloss from baseline to the last visit measured using a hydrometer. **Measure:** Hydrometer measured Transepidermal Water Loss **Time Frame:** Baseline to 6 - Months Post Final Treatment #### Secondary Outcomes **Description:** At each follow up visit, any adverse effects such as but not limited to bruising, erythema, swelling, pain, tenderness, itching, dysesthesia, and nodularity will be recorded descriptively. **Measure:** Safety - Injection Site Adverse Events **Time Frame:** Baseline to 6 - Months Post Final Treatment ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: b. Symmetrical visible sign of aging involving at least a 9 cm2 area of the mid to low cheeks. c. Must be willing to give and sign a HIPPA form, photo consent and informed consent form. d. Must be willing to comply with study dosing and complete the entire course of the study. e. Female patients will be either of non-childbearing potential defined as: 1. Having no uterus 2. No menses for at least 12 months. Or; (WOCBP) women of childbearing potential must agree to use an effective method of birth control during the course of the study, such as: 1. Oral contraceptive pill, injection, implant, patch, vaginal ring, intrauterine device 2. Intrauterine coil 3. Bilateral tubal ligation 4. Barrier method used with an additional form of contraception (e.g., sponge, spermicide or condom) 5. Abstinence (If practicing abstinence must agree to use barrier method described above (4) if becomes sexually active). 6. Vasectomized partner (must agree to use barrier method described above (4) if becomes sexually active with un-vasectomized). f. Negative urine pregnancy test results Baseline prior to study entry (if applicable) Exclusion Criteria: 1. Pregnant, planning pregnancy during the course of the study or breastfeeding 2. Extremely Severe aging face with extensive photodamage 3. Previous use of any form of soft tissue augmentation in the treatment area within the past 12 months 4. Pre-existing medical or dermatologic condition in the treatment area that may affect the treatment or interpretation of treatment effect (at investigator discretion) 5. Presence of tattoo and/or scar in the treatment area that in the investigators opinion would interfere with study assessments 6. Use of oral/topical retinoids within 1 month of Baseline 7. Previous use of botulinum toxins in the treatment area within the past 6 months 8. Previous surgical procedure in the treatment area within the past 12 months 9. Presence or evidence of any conditions that in the opinion of the investigator might impede the subject's ability to give consent or comply with protocol requirements. 10. Current participation or participation within 30 days prior to the start of this study in a drug or other investigational research study 11. History of non-compliance with clinical research protocols 12. Ablative laser resurfacing to on their face within 12 months 13. Non-ablative laser or light procedures to their face within the past 3 months 14. Known allergy to Restylane® Silk or any of its constituents **Healthy Volunteers:** True **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** SQuigley@CLDerm.com **Name:** Shannon Quigley **Phone:** 858 657 1004 **Phone Ext:** 141 **Role:** CONTACT #### Locations **Location 1:** **City:** San Diego **Contacts:** ***Contact 1:*** - **Email:** KCobb@CLDerm.com - **Name:** Kristi Cobb - **Phone:** 858-657-1004 - **Phone Ext:** 139 - **Role:** CONTACT ***Contact 2:*** - **Email:** IGuiha@CLDerm.com - **Name:** Isabella Guiha - **Phone:** 858 657 1004 - **Phone Ext:** 119 - **Role:** CONTACT ***Contact 3:*** - **Name:** Mitchel P Goldman, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** West Dermatology Research Center **State:** California **Status:** RECRUITING **Zip:** 92121 ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M9878 - Name: Hyaluronic Acid - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05727579 **Acronym:** DESIGN **Brief Title:** DiEtary Sodium Intake Effects on Ertugliflozin-induced Changes in GFR, reNal Oxygenation and Systemic Hemodynamics: the DESIGN Study **Official Title:** DiEtary Sodium Intake Effects on Ertugliflozin-induced Changes in GFR, reNal Oxygenation and Systemic Hemodynamics: the DESIGN Study, a Randomized, Placebo-controlled, Cross-over Study With Ertugliflozin in People With Type 2 Diabetes #### Organization Study ID Info **ID:** DC2022ERTU #### Organization **Class:** OTHER **Full Name:** Amsterdam UMC, location VUmc ### Status Module #### Completion Date **Date:** 2025-03-01 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-05-01 **Type:** ACTUAL **Last Update Submit Date:** 2023-04-28 **Overall Status:** NOT_YET_RECRUITING #### Primary Completion Date **Date:** 2024-09-01 **Type:** ESTIMATED #### Start Date **Date:** 2023-06-01 **Type:** ESTIMATED **Status Verified Date:** 2023-04 #### Study First Post Date **Date:** 2023-02-14 **Type:** ACTUAL **Study First Submit Date:** 2023-01-24 **Study First Submit QC Date:** 2023-02-03 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Merck Sharp & Dohme LLC **Class:** OTHER **Name:** University of Colorado, Denver #### Lead Sponsor **Class:** OTHER **Name:** Amsterdam UMC, location VUmc #### Responsible Party **Investigator Affiliation:** Amsterdam UMC, location VUmc **Investigator Full Name:** D van Raalte **Investigator Title:** Dr. **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Is US Export:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** SGLT2 inhibitors such as ertugliflozin improve blood pressure and kidney outcomes in people living with diabetes through incompletely understood mechanisms, however, not all patients treated with SGLT2 inhibition have improved outcomes. Changes in kidney sodium handling is among the mechanisms by which SGLT2 inhibition may reduce blood pressure and drive beneficial kidney outcomes. This process is heavily dependent on daily sodium intake by patients receiving SGLT2 inhibitor treatment. In this study, the effect of daily sodium intake on SGLT2-inhibitor induced physiological effect is studied, including blood pressure regulation and kidney physiology. ### Conditions Module **Conditions:** - Diabetes Mellitus - Diabetic Kidney Disease - Hypertension ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER **Intervention Model Description:** While the treatment by ertugliflozin or placebo will be blinded, the sodium interventions are open-label. ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Count:** 34 **Type:** ESTIMATED **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Other: Salt-Diet and/or Ertugliflozin **Label:** low-sodium diet; placebo **Type:** OTHER #### Arm Group 2 **Intervention Names:** - Other: Salt-Diet and/or Ertugliflozin **Label:** low-sodium diet; ertugliflozin 15 once daily **Type:** OTHER #### Arm Group 3 **Intervention Names:** - Other: Salt-Diet and/or Ertugliflozin **Label:** high-sodium diet; placebo **Type:** OTHER #### Arm Group 4 **Intervention Names:** - Other: Salt-Diet and/or Ertugliflozin **Label:** High-sodium diet; ertugliflozin 15 mg once daily **Type:** OTHER ### Interventions #### Intervention 1 **Arm Group Labels:** - High-sodium diet; ertugliflozin 15 mg once daily - high-sodium diet; placebo - low-sodium diet; ertugliflozin 15 once daily - low-sodium diet; placebo **Description:** The interventions consist of an determined amount of dietary sodium intake in combination with either Ertugliflozin 15mg once daily or placebo **Name:** Salt-Diet and/or Ertugliflozin **Type:** OTHER ### Outcomes Module #### Other Outcomes **Description:** To investigate the modifying effects of WHO-recommended sodium intake (90 mmol per day) vs. high sodium intake (targeted at 250 mmol per day) on the effect of ertugliflozin 15 mg daily, versus placebo, on GFR. **Measure:** Glomerular filtration rate (GFR) **Time Frame:** 24 weeks **Description:** To investigate the modifying effects of WHO-recommended sodium intake (90 mmol per day) vs. high sodium intake (targeted at 250 mmol per day) on the effect of ertugliflozin 15 mg daily, versus placebo, on hematocrit. **Measure:** Hematocrit **Time Frame:** 24 weeks **Description:** To investigate the modifying effects of WHO-recommended sodium intake (90 mmol per day) vs. high sodium intake (targeted at 250 mmol per day) on the effect of ertugliflozin 15 mg daily, versus placebo, on blood pressure. **Measure:** Blood pressure **Time Frame:** 24 weeks **Description:** To investigate the modifying effects of WHO-recommended sodium intake (90 mmol per day) vs. high sodium intake (targeted at 250 mmol per day) on the effect of ertugliflozin 15 mg daily, versus placebo, on kidney oxygenation. **Measure:** Kidney oxygenation **Time Frame:** 24 weeks **Description:** To investigate the modifying effects of WHO-recommended sodium intake (90 mmol per day) vs. high sodium intake (targeted at 250 mmol per day) on the effect of ertugliflozin 15 mg daily, versus placebo, on effective renal plasma flow. **Measure:** Effective renal plasma flow **Time Frame:** 24 weeks **Description:** To investigate the modifying effects of WHO-recommended sodium intake (90 mmol per day) vs. high sodium intake (targeted at 250 mmol per day) on the effect of ertugliflozin 15 mg daily, versus placebo, on renal vasculare resistance. **Measure:** Renal vasculare resistance **Time Frame:** 24 weeks **Description:** To investigate the modifying effects of WHO-recommended sodium intake (90 mmol per day) vs. high sodium intake (targeted at 250 mmol per day) on the effect of ertugliflozin 15 mg daily, versus placebo, on body anthropometrics **Measure:** Body anthropometrics **Time Frame:** 24 weeks **Description:** To investigate the modifying effects of WHO-recommended sodium intake (90 mmol per day) vs. high sodium intake (targeted at 250 mmol per day) on the effect of ertugliflozin 15 mg daily, versus placebo, on fasting plasma glucose **Measure:** Fasting plasma glucose **Time Frame:** 24 weeks **Description:** To investigate the modifying effects of WHO-recommended sodium intake (90 mmol per day) vs. high sodium intake (targeted at 250 mmol per day) on the effect of ertugliflozin 15 mg daily, versus placebo, on albumin excretion rate **Measure:** Albumin excretion rate **Time Frame:** 24 weeks **Description:** To investigate the modifying effects of WHO-recommended sodium intake (90 mmol per day) vs. high sodium intake (targeted at 250 mmol per day) on the effect of ertugliflozin 15 mg daily, versus placebo, on 24hr urinary glucose excretion **Measure:** Glucose excretion **Time Frame:** 24 weeks **Description:** To investigate the modifying effects of WHO-recommended sodium intake (90 mmol per day) vs. high sodium intake (targeted at 250 mmol per day) on the effect of ertugliflozin 15 mg daily, versus placebo, on biomarkers **Measure:** Biomarkers **Time Frame:** 24 weeks #### Primary Outcomes **Description:** To investigate the modifying effects of WHO-recommended sodium intake (90 mmol per day) vs. high sodium intake (targeted at 250 mmol per day) on the effect of ertugliflozin 15 mg daily, versus placebo, on 24-hour blood pressure in overweight/obese adults with type 2 diabetes **Measure:** To investigate the modifying effect of sodium intake on Ertugliflozin on blood pressure **Time Frame:** 24 weeks #### Secondary Outcomes **Description:** To investigate the efficacy of ertugliflozin 15 mg daily, versus placebo, in overweight/obese adults with type 2 diabetes to reduce the hypertensive effects of a high-sodium diet (250 mmol per day) versus participant's normal diet (170 mmol/per day). **Measure:** To investigate the effect of Ertugliflozin on the hypertensive effects of high dietary sodium intake **Time Frame:** 24 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Adults with previously diagnosed T2DM according to American Diabetes Association (ADA) criteria * HbA1c 6.5-10% * Age 35-80 years of age * Overweight or obese with BMI: \>25 kg/m2 * We will make every effort to enrol participants of all races/ethnicities." * Both sexes (females must be post-menopausal; no menses \>1 year; in case of doubt, Follicle-Stimulating Hormone (FSH) will be determined with cut-off defined as \>31 U/L) * Ability to provide signed and dated, written informed consent prior to any study procedures * Estimated GFR 60-90 ml/min/1.73m2 by CKD-EPI matching the eGFR range of most participants in VERTIS-CV * Sodium intake at baseline \< 200 mmol/day * UACR \< 30 mg/mmol * All participants need to be on a stable dose of Diabetes medication, including Metformin, SU, insulin * All participants need to be on a stable dose of RAS inhibition Exclusion Criteria: History of unstable or rapidly progressing renal disease * Estimated GFR \<60 mL/min/1.73m2 or eGFR \> 90 mL/min/1.73m2 determined by CKD-EPI * UACR \> 30 mg/mmol * Current/chronic use of the following medication: SGLT2 inhibitors, TZD, GLP-1RA, glucocorticoids, immune suppressants, antimicrobial agents, chemotherapeutics, antipsychotics, tricyclic antidepressants (TCAs) and monoamine oxidase inhibitors (MAOIs). Subjects on diuretics will only be excluded when these drugs cannot be stopped for the duration of the study. * Chronic use of non-steroidal anti-inflammatory drugs (NSAIDs) will not be allowed, unless used as incidental medication (1-2 tablets) for non-chronic indications (i.e. sports injury, headache or back ache). However, no such drug can be taken within a timeframe of 2 weeks prior to renal testing * History of diabetic ketoacidosis (DKA) requiring medical intervention (e.g. emergency room visit and/or hospitalization) within 1 month prior to the Screening visit. * Current urinary tract infection and active nephritis * Recent (\<6 months) history of cardiovascular disease, including: * Acute coronary syndrome * Chronic heart failure (New York Heart Association grade II-IV) * Stroke or transient ischemic neurologic disorder * Severe hepatic insufficiency and/or significant abnormal liver function defined as aspartate aminotransferase (AST) \>3x upper limit of normal (ULN) and/or alanine aminotransferase (ALT) \>3x ULN * History of or actual malignancy (except basal cell carcinoma) * History of or actual severe mental disease * Substance abuse (alcohol: defined as \>4 units/day) * Allergy to any of the agents used in the study * Individuals who are investigator site personnel, directly affiliated with the study, or are immediate (spouse, parent, child, or sibling, whether biological or legally adopted) family of investigator site personnel directly affiliated with the study * Inability to understand the study protocol or give informed consent **Maximum Age:** 80 Years **Minimum Age:** 35 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** d.vanraalte@amsterdamumc.nl **Name:** Daniel van Raalte, MD PhD **Phone:** +31204440534 **Role:** CONTACT ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014570 - Term: Urologic Diseases - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000052801 - Term: Male Urogenital Diseases - ID: D000048909 - Term: Diabetes Complications - ID: D000003920 - Term: Diabetes Mellitus - ID: D000004700 - Term: Endocrine System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: All - Name: All Conditions - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M7115 - Name: Diabetes Mellitus - Relevance: LOW - As Found: Unknown - ID: M7119 - Name: Diabetes Mellitus, Type 2 - Relevance: LOW - As Found: Unknown - ID: M10698 - Name: Kidney Diseases - Relevance: HIGH - As Found: Kidney Disease - ID: M10024 - Name: Hypertension - Relevance: LOW - As Found: Unknown - ID: M7123 - Name: Diabetic Nephropathies - Relevance: HIGH - As Found: Diabetic Kidney Disease - ID: M17319 - Name: Urologic Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M26004 - Name: Diabetes Complications - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown - ID: T4202 - Name: Oculocerebral Syndrome With Hypopigmentation - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007674 - Term: Kidney Diseases - ID: D000003928 - Term: Diabetic Nephropathies ### Intervention Browse Module - Ancestors - ID: D000077203 - Term: Sodium-Glucose Transporter 2 Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000007004 - Term: Hypoglycemic Agents - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: Hypo - Name: Hypoglycemic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M263808 - Name: Ertugliflozin - Relevance: HIGH - As Found: Esophagitis - ID: M1691 - Name: Sodium-Glucose Transporter 2 Inhibitors - Relevance: LOW - As Found: Unknown - ID: M10054 - Name: Hypoglycemic Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000570288 - Term: Ertugliflozin ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02352779 **Brief Title:** Omega-3 Fatty Acid in Reducing Cancer-Related Fatigue in Breast Cancer Survivors **Official Title:** Feasibility of Omega-3 Supplementation for Cancer-Related Fatigue #### Organization Study ID Info **ID:** URCC13091 #### Organization **Class:** OTHER **Full Name:** University of Rochester #### Secondary ID Infos **Domain:** CTRP (Clinical Trial Reporting Program) **ID:** NCI-2014-01191 **Type:** REGISTRY **Domain:** University of Rochester NCORP Research Base **ID:** URCC13091 **Type:** OTHER **Domain:** DCP **ID:** URCC-13091 **Type:** OTHER **Domain:** CTEP **ID:** URCC-13091 **Type:** OTHER **ID:** U10CA037420 **Link:** https://reporter.nih.gov/quickSearch/U10CA037420 **Type:** NIH **ID:** UG1CA189961 **Link:** https://reporter.nih.gov/quickSearch/UG1CA189961 **Type:** NIH **ID:** R03CA175599 **Link:** https://reporter.nih.gov/quickSearch/R03CA175599 **Type:** NIH ### Status Module #### Completion Date **Date:** 2016-04 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-08-08 **Type:** ACTUAL **Last Update Submit Date:** 2017-07-05 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2016-02 **Type:** ACTUAL #### Results First Post Date **Date:** 2017-08-08 **Type:** ACTUAL **Results First Submit Date:** 2017-02-03 **Results First Submit QC Date:** 2017-07-05 #### Start Date **Date:** 2015-02 **Status Verified Date:** 2017-07 #### Study First Post Date **Date:** 2015-02-02 **Type:** ESTIMATED **Study First Submit Date:** 2015-01-28 **Study First Submit QC Date:** 2015-01-30 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Cancer Institute (NCI) #### Lead Sponsor **Class:** OTHER **Name:** Gary Morrow #### Responsible Party **Investigator Affiliation:** University of Rochester NCORP Research Base **Investigator Full Name:** Gary Morrow **Investigator Title:** Principal Investigator, URCC NCORP Research Base **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** This randomized pilot clinical trial studies omega-3 fatty acid in reducing cancer-related fatigue in breast cancer survivors. Supplementation with omega-3 fatty acid may help reduce cancer-related fatigue in breast cancer survivors. **Detailed Description:** PRIMARY OBJECTIVE: I. To collect preliminary statistical data (mean changes and standard deviations) on two omega-3 supplementation (omega-3 fatty acid) regimens (1.65 g/day and 3.3 g/day) compared to placebo for reducing cancer-related fatigue (CRF) in fatigued breast cancer survivors. OUTLINE: Patients are randomized to 1 of 3 arms. ARM I: Patients receive low-dose omega-3 fatty acid supplementation orally (PO) twice daily (BID) and placebo PO BID for 6 weeks. ARM II: Patients receive high-dose omega-3 fatty acid supplementation PO BID for 6 weeks. ARM III: Patients receive placebo PO BID for 6 weeks. ### Conditions Module **Conditions:** - Breast Carcinoma - Cancer - Fatigue **Keywords:** - Cancer Survivor ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 108 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients receive low-dose omega-3 fatty acid supplementation PO BID and placebo PO BID for 6 weeks. **Intervention Names:** - Dietary Supplement: Omega-3 Fatty Acid - Other: Placebo - Other: Questionnaire Administration - Other: Laboratory Biomarker Analysis **Label:** Arm I (low-dose omega-3 fatty acid) **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Patients receive high-dose omega-3 fatty acid supplementation PO BID for 6 weeks. **Intervention Names:** - Dietary Supplement: Omega-3 Fatty Acid - Other: Questionnaire Administration - Other: Laboratory Biomarker Analysis **Label:** Arm II (high-dose omega-3 fatty acid) **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Patients receive placebo PO BID for 6 weeks. **Intervention Names:** - Other: Placebo - Other: Questionnaire Administration - Other: Laboratory Biomarker Analysis **Label:** Arm III (placebo) **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Arm I (low-dose omega-3 fatty acid) - Arm II (high-dose omega-3 fatty acid) **Description:** Given PO **Name:** Omega-3 Fatty Acid **Other Names:** - O3FA - Omega-3 Fatty Acids - Omega-3 PUFA **Type:** DIETARY_SUPPLEMENT #### Intervention 2 **Arm Group Labels:** - Arm I (low-dose omega-3 fatty acid) - Arm III (placebo) **Description:** Given PO **Name:** Placebo **Other Names:** - PLCB **Type:** OTHER #### Intervention 3 **Arm Group Labels:** - Arm I (low-dose omega-3 fatty acid) - Arm II (high-dose omega-3 fatty acid) - Arm III (placebo) **Description:** Ancillary studies **Name:** Questionnaire Administration **Type:** OTHER #### Intervention 4 **Arm Group Labels:** - Arm I (low-dose omega-3 fatty acid) - Arm II (high-dose omega-3 fatty acid) - Arm III (placebo) **Description:** Correlative studies **Name:** Laboratory Biomarker Analysis **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** BFI-SF is a 4 item questionnaire to assess the severity of fatigue, ranging from 0 (No Fatigue) to 10 (As bad as you can imagine). MFSI-SF is a 30 item questionnaire to assess the level of fatigue in terms of general fatigue, physical fatigue, emotional fatigue, mental fatigue, and vigor). First four subscales (general, physical, emotional, and mental) are summed and the vigor scale is subtracted to create fatigue total score with a range of -32 (low fatigue) to 96 (high fatigue). **Measure:** Mean Change (6 Weeks - Baseline) and Standard Deviation in Cancer-related Fatigue, Using the Brief Fatigue Inventory-Short Form (BFI-SF) and Multidimensional Fatigue Symptom Inventory-Short Form (MFSI-SF). 81 Subjects Had Both a Baseline and 6 Week Value **Time Frame:** Baseline to 6 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Have a confirmed diagnosis of breast cancer; participants can have had more than one primary cancer diagnosis in the past * Have undergone some type or combination of standard adjuvant treatment (surgery, chemotherapy, radiation therapy) for breast cancer * Have completed all forms of standard adjuvant treatment (surgery, chemotherapy, radiation therapy) for breast cancer between 4 and 36 months prior to enrollment in the study; participants can be currently taking hormones (such as tamoxifen) or monoclonal antibodies (such as Herceptin) * Must have cancer-related fatigue, as indicated by a response of 4 or greater when asked to rate their level of fatigue at its worst in the past week on an 11-point scale anchored by "0" = no fatigue and "10" = as bad as you can imagine * Be able to read English * Able to swallow medication * Give written informed consent Exclusion Criteria: * Have used marine omega-3 supplements at any time within previous 3 months (this includes prescription omega-3 drugs such as Lovaza®) * Be taking anticoagulant medication (does not include aspirin) * Have sensitivity or allergy to fish and/or shellfish * Have sensitivity or allergy to soy and/or soybeans * Have confirmed diagnosis of chronic fatigue syndrome or other diagnosis known to cause severe fatigue **Maximum Age:** 120 Years **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Wichita **Country:** United States **Facility:** Wichita NCORP **State:** Kansas **Zip:** 67214 **Location 2:** **City:** Grand Rapids **Country:** United States **Facility:** Cancer Research Consortium of West Michigan **State:** Michigan **Zip:** 49503 **Location 3:** **City:** Rochester **Country:** United States **Facility:** University of Rochester NCORP Research Base **State:** New York **Zip:** 14642 **Location 4:** **City:** Dayton **Country:** United States **Facility:** Dayton NCORP **State:** Ohio **Zip:** 45420 **Location 5:** **City:** Greenville **Country:** United States **Facility:** Greenville Health System NCORP **State:** South Carolina **Zip:** 29605 **Location 6:** **City:** Marshfield **Country:** United States **Facility:** Wisconsin NCORP **State:** Wisconsin **Zip:** 54449 #### Overall Officials **Official 1:** **Affiliation:** University of Rochester NCORP Research Base **Name:** Luke Peppone **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Kleckner AS, Kleckner IR, Culakova E, Wojtovich AP, Klinedinst NJ, Kerns SL, Hardy SJ, Inglis JE, Padula GDA, Mustian KM, Janelsins MC, Dorsey SG, Saligan LN, Peppone LJ. Exploratory Analysis of Associations Between Whole Blood Mitochondrial Gene Expression and Cancer-Related Fatigue Among Breast Cancer Survivors. Nurs Res. 2022 Sep-Oct 01;71(5):411-417. doi: 10.1097/NNR.0000000000000598. Epub 2022 Apr 13. **PMID:** 35416182 **Citation:** Inglis JE, Kleckner AS, Lin PJ, Gilmore NJ, Culakova E, VanderWoude AC, Mustian KM, Fernandez ID, Dunne RF, Deutsch J, Peppone LJ. Excess Body Weight and Cancer-Related Fatigue, Systemic Inflammation, and Serum Lipids in Breast Cancer Survivors. Nutr Cancer. 2021;73(9):1676-1686. doi: 10.1080/01635581.2020.1807574. Epub 2020 Aug 19. **PMID:** 32812824 #### See Also Links **Label:** Clinical trial summary from the National Cancer Institute's PDQ® database **URL:** https://www.cancer.gov/publications/pdq ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000001941 - Term: Breast Diseases - ID: D000012871 - Term: Skin Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M5220 - Name: Breast Neoplasms - Relevance: HIGH - As Found: Breast Carcinoma - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - ID: M8364 - Name: Fatigue - Relevance: HIGH - As Found: Fatigue - ID: M5218 - Name: Breast Diseases - Relevance: LOW - As Found: Unknown - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001943 - Term: Breast Neoplasms - ID: D000005221 - Term: Fatigue ### Intervention Browse Module - Browse Branches - Abbrev: Ot - Name: Other Dietary Supplements - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: T415 - Name: Omega 3 Fatty Acid - Relevance: HIGH - As Found: Machine ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Arm I (Low-dose Omega-3 Fatty Acid) **Description:** Patients receive low-dose omega-3 fatty acid supplementation PO BID and placebo PO BID for 6 weeks. Omega-3 Fatty Acid: Given PO Placebo: Given PO Questionnaire Administration: Ancillary studies Laboratory Biomarker Analysis: Correlative studies **ID:** EG000 **Other Num at Risk:** 29 **Serious Number At Risk:** 29 **Title:** Arm I (Low-dose Omega-3 Fatty Acid) **Group ID:** EG001 **Title:** Arm II (High-dose Omega-3 Fatty Acid) **Description:** Patients receive high-dose omega-3 fatty acid supplementation PO BID for 6 weeks. Omega-3 Fatty Acid: Given PO Questionnaire Administration: Ancillary studies Laboratory Biomarker Analysis: Correlative studies **ID:** EG001 **Other Num at Risk:** 35 **Serious Number At Risk:** 35 **Title:** Arm II (High-dose Omega-3 Fatty Acid) **Group ID:** EG002 **Title:** Arm III (Placebo) **Description:** Patients receive placebo PO BID for 6 weeks. Placebo: Given PO Questionnaire Administration: Ancillary studies Laboratory Biomarker Analysis: Correlative studies **ID:** EG002 **Other Num Affected:** 1 **Other Num at Risk:** 33 **Serious Number At Risk:** 33 **Title:** Arm III (Placebo) **Frequency Threshold:** 0 #### Other Events **Term:** Pruritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE (Unspecified) ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 29 **Group ID:** BG001 **Value:** 35 **Group ID:** BG002 **Value:** 33 **Group ID:** BG003 **Value:** 97 **Units:** Participants ### Group **ID:** BG000 **Title:** Arm I (Low-dose Omega-3 Fatty Acid) **Description:** Patients receive low-dose omega-3 fatty acid supplementation PO BID and placebo PO BID for 6 weeks. Omega-3 Fatty Acid: Given PO Placebo: Given PO Questionnaire Administration: Ancillary studies Laboratory Biomarker Analysis: Correlative studies ### Group **ID:** BG001 **Title:** Arm II (High-dose Omega-3 Fatty Acid) **Description:** Patients receive high-dose omega-3 fatty acid supplementation PO BID for 6 weeks. Omega-3 Fatty Acid: Given PO Questionnaire Administration: Ancillary studies Laboratory Biomarker Analysis: Correlative studies ### Group **ID:** BG002 **Title:** Arm III (Placebo) **Description:** Patients receive placebo PO BID for 6 weeks. Placebo: Given PO Questionnaire Administration: Ancillary studies Laboratory Biomarker Analysis: Correlative studies ### Group **ID:** BG003 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 11.10 **Value:** 60.62 #### Measurement **Group ID:** BG001 **Spread:** 9.35 **Value:** 60.40 #### Measurement **Group ID:** BG002 **Spread:** 10.94 **Value:** 58.03 #### Measurement **Group ID:** BG003 **Spread:** 10.40 **Value:** 59.66 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 29 #### Measurement **Group ID:** BG001 **Value:** 35 #### Measurement **Group ID:** BG002 **Value:** 33 #### Measurement **Group ID:** BG003 **Value:** 97 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 28 #### Measurement **Group ID:** BG001 **Value:** 35 #### Measurement **Group ID:** BG002 **Value:** 33 #### Measurement **Group ID:** BG003 **Value:** 96 **Category Title:** Not Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 1 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 1 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 4 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 5 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 27 #### Measurement **Group ID:** BG001 **Value:** 31 #### Measurement **Group ID:** BG002 **Value:** 33 #### Measurement **Group ID:** BG003 **Value:** 91 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 6.07 **Value:** 30.11 #### Measurement **Group ID:** BG001 **Spread:** 7.45 **Value:** 32.31 #### Measurement **Group ID:** BG002 **Spread:** 8.17 **Value:** 32.64 #### Measurement **Group ID:** BG003 **Spread:** 7.34 **Value:** 31.76 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 5.81 **Value:** 91.38 #### Measurement **Group ID:** BG001 **Spread:** 6.32 **Value:** 92.00 #### Measurement **Group ID:** BG002 **Spread:** 7.70 **Value:** 89.70 #### Measurement **Group ID:** BG003 **Spread:** 6.69 **Value:** 91.03 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 6 #### Measurement **Group ID:** BG001 **Value:** 5 #### Measurement **Group ID:** BG002 **Value:** 4 #### Measurement **Group ID:** BG003 **Value:** 15 **Category Title:** Graduate Degree #### Measurement **Group ID:** BG000 **Value:** 13 #### Measurement **Group ID:** BG001 **Value:** 18 #### Measurement **Group ID:** BG002 **Value:** 14 #### Measurement **Group ID:** BG003 **Value:** 45 **Category Title:** 2 or 4 year Degree / Some college #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 12 #### Measurement **Group ID:** BG002 **Value:** 14 #### Measurement **Group ID:** BG003 **Value:** 36 **Category Title:** HS / GED Degree #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 1 #### Measurement **Group ID:** BG003 **Value:** 1 **Category Title:** No HS Degree or GED **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 16 #### Measurement **Group ID:** BG001 **Value:** 20 #### Measurement **Group ID:** BG002 **Value:** 25 #### Measurement **Group ID:** BG003 **Value:** 61 **Category Title:** Married #### Measurement **Group ID:** BG000 **Value:** 2 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 3 #### Measurement **Group ID:** BG003 **Value:** 5 **Category Title:** Long Term, Committed SO #### Measurement **Group ID:** BG000 **Value:** 5 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 12 **Category Title:** Divorced #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 4 #### Measurement **Group ID:** BG002 **Value:** 2 #### Measurement **Group ID:** BG003 **Value:** 9 **Category Title:** Single #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 4 #### Measurement **Group ID:** BG002 **Value:** 3 #### Measurement **Group ID:** BG003 **Value:** 10 **Category Title:** Widowed **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 16 #### Measurement **Group ID:** BG001 **Value:** 18 #### Measurement **Group ID:** BG002 **Value:** 13 #### Measurement **Group ID:** BG003 **Value:** 47 **Category Title:** Employment outside the house #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 6 **Category Title:** Self Employed #### Measurement **Group ID:** BG000 **Value:** 6 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 9 #### Measurement **Group ID:** BG003 **Value:** 18 **Category Title:** Home Maker #### Measurement **Group ID:** BG000 **Value:** 4 #### Measurement **Group ID:** BG001 **Value:** 11 #### Measurement **Group ID:** BG002 **Value:** 11 #### Measurement **Group ID:** BG003 **Value:** 26 **Category Title:** Unemployed **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 28 #### Measurement **Group ID:** BG001 **Value:** 33 #### Measurement **Group ID:** BG002 **Value:** 33 #### Measurement **Group ID:** BG003 **Value:** 94 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 2 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 3 **Category Title:** No **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 13 #### Measurement **Group ID:** BG001 **Value:** 19 #### Measurement **Group ID:** BG002 **Value:** 17 #### Measurement **Group ID:** BG003 **Value:** 49 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 16 #### Measurement **Group ID:** BG001 **Value:** 16 #### Measurement **Group ID:** BG002 **Value:** 16 #### Measurement **Group ID:** BG003 **Value:** 48 **Category Title:** No **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 19 #### Measurement **Group ID:** BG001 **Value:** 28 #### Measurement **Group ID:** BG002 **Value:** 25 #### Measurement **Group ID:** BG003 **Value:** 72 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 8 #### Measurement **Group ID:** BG003 **Value:** 25 **Category Title:** No **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 2 #### Measurement **Group ID:** BG001 **Value:** 6 #### Measurement **Group ID:** BG002 **Value:** 9 #### Measurement **Group ID:** BG003 **Value:** 17 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 27 #### Measurement **Group ID:** BG001 **Value:** 29 #### Measurement **Group ID:** BG002 **Value:** 24 #### Measurement **Group ID:** BG003 **Value:** 80 **Category Title:** No **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Ethnicity (NIH/OMB) **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race (NIH/OMB) **Unit of Measure:** Participants ### Measure 5 **Description:** Body Mass Index (BMI) is a person's weight in kilograms divided by the square of height in meters. Values below 18.5 represent "Underweight", 18.5 to 24.9 represent "Normal or Healthy Weight", 25.0 to 29.9 represent "Overweight" and 30.0 and above represent "Obese". **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** BMI **Unit of Measure:** kg/m^2 ### Measure 6 **Description:** The Karnofsky Performance Scale (KPS) Index allows patients to be classified as to their functional impairment. Essentially as used in oncology, the KPS is a measure of time to death. A higher score is favorable. There are 3 general categories which are broken down into 11 specific criteria. Scale range is from 0 (Dead) to 100 (Normal, no complaints, no evidence of disease). The scale increases in units of 10. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Karnofsky Performance Status (KPS) **Unit of Measure:** Karnofsky Performance Scale ### Measure 7 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Education Level **Unit of Measure:** Participants ### Measure 8 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Marital Status **Unit of Measure:** Participants ### Measure 9 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Employment Status (Current) **Unit of Measure:** Participants ### Measure 10 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Previous Treatment - Surgery **Unit of Measure:** Participants ### Measure 11 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Previous Treatment - Chemotherapy **Unit of Measure:** Participants ### Measure 12 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Previous Treatment - Radiation **Unit of Measure:** Participants ### Measure 13 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Previous Treatment - Hormone Therapy **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement ### Point of Contact **Email:** Luke_Peppone@urmc.rochester.edu **Organization:** University of Rochester Medical Center **Phone:** 585-275-7827 **Title:** Luke J. Peppone, PhD, MPH. Assistant Professor ## Results Section - Outcome Measures Module ### Outcome Measure 1 #### Analysis **CI Lower Limit:** **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 0.4843 **Estimate Comment:** **Group Description:** BFI-SF **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.1666 **P-Value Comment:** **Parameter Type:** Mean Difference (Final Values) **Parameter Value:** 0.6763 **Statistical Comment:** Based on a contrast using the three-arm ANCOVA. **Statistical Method:** ANCOVA **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 0.4567 **Estimate Comment:** **Group Description:** BFI-SF **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.1329 **P-Value Comment:** **Parameter Type:** Mean Difference (Final Values) **Parameter Value:** 0.6936 **Statistical Comment:** Based on a contrast using the three-arm ANCOVA. **Statistical Method:** ANCOVA **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 3.8065 **Estimate Comment:** **Group Description:** MFSI-SF **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.9826 **P-Value Comment:** **Parameter Type:** Mean Difference (Final Values) **Parameter Value:** 0.0831 **Statistical Comment:** Based on a contrast using the three-arm ANCOVA. **Statistical Method:** ANCOVA **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 3.5448 **Estimate Comment:** **Group Description:** MFSI-SF **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.4016 **P-Value Comment:** **Parameter Type:** Mean Difference (Final Values) **Parameter Value:** 2.9898 **Statistical Comment:** Based on a contrast using the three-arm ANCOVA. **Statistical Method:** ANCOVA **Tested Non-Inferiority:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** -4.37 - **Spread:** - **Upper Limit:** -2.96 - **Value:** -3.66 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -4.31 - **Spread:** - **Upper Limit:** -3.05 - **Value:** -3.68 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** -3.65 - **Spread:** - **Upper Limit:** -2.33 - **Value:** -2.99 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** -16.55 - **Spread:** - **Upper Limit:** -5.50 - **Value:** -11.03 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -18.82 - **Spread:** - **Upper Limit:** -9.05 - **Value:** -13.93 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** -16.07 - **Spread:** - **Upper Limit:** -5.82 - **Value:** -10.94 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** BFI-SF is a 4 item questionnaire to assess the severity of fatigue, ranging from 0 (No Fatigue) to 10 (As bad as you can imagine). MFSI-SF is a 30 item questionnaire to assess the level of fatigue in terms of general fatigue, physical fatigue, emotional fatigue, mental fatigue, and vigor). First four subscales (general, physical, emotional, and mental) are summed and the vigor scale is subtracted to create fatigue total score with a range of -32 (low fatigue) to 96 (high fatigue). **Dispersion Type:** 95% Confidence Interval **Parameter Type:** LEAST_SQUARES_MEAN **Reporting Status:** POSTED **Time Frame:** Baseline to 6 weeks **Title:** Mean Change (6 Weeks - Baseline) and Standard Deviation in Cancer-related Fatigue, Using the Brief Fatigue Inventory-Short Form (BFI-SF) and Multidimensional Fatigue Symptom Inventory-Short Form (MFSI-SF). 81 Subjects Had Both a Baseline and 6 Week Value **Type:** PRIMARY **Unit of Measure:** units on a scale ##### Group **Description:** Patients receive low-dose omega-3 fatty acid supplementation PO BID and placebo PO BID for 6 weeks. Omega-3 Fatty Acid: Given PO Placebo: Given PO Questionnaire Administration: Ancillary studies Laboratory Biomarker Analysis: Correlative studies **ID:** OG000 **Title:** Arm I (Low-dose Omega-3 Fatty Acid) ##### Group **Description:** Patients receive high-dose omega-3 fatty acid supplementation PO BID for 6 weeks. Omega-3 Fatty Acid: Given PO Questionnaire Administration: Ancillary studies Laboratory Biomarker Analysis: Correlative studies **ID:** OG001 **Title:** Arm II (High-dose Omega-3 Fatty Acid) ##### Group **Description:** Patients receive placebo PO BID for 6 weeks. Placebo: Given PO Questionnaire Administration: Ancillary studies Laboratory Biomarker Analysis: Correlative studies **ID:** OG002 **Title:** Arm III (Placebo) ### Participant Flow Module #### Group **Description:** Patients receive low-dose omega-3 fatty acid supplementation PO BID and placebo PO BID for 6 weeks. Omega-3 Fatty Acid: Given PO Placebo: Given PO Questionnaire Administration: Ancillary studies Laboratory Biomarker Analysis: Correlative studies **ID:** FG000 **Title:** Arm I (Low-dose Omega-3 Fatty Acid) #### Group **Description:** Patients receive high-dose omega-3 fatty acid supplementation PO BID for 6 weeks. Omega-3 Fatty Acid: Given PO Questionnaire Administration: Ancillary studies Laboratory Biomarker Analysis: Correlative studies **ID:** FG001 **Title:** Arm II (High-dose Omega-3 Fatty Acid) #### Group **Description:** Patients receive placebo PO BID for 6 weeks. Placebo: Given PO Questionnaire Administration: Ancillary studies Laboratory Biomarker Analysis: Correlative studies **ID:** FG002 **Title:** Arm III (Placebo) #### Period **Title:** Overall Study ##### Withdraw **Type:** Personal Issues ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ###### Reason **Group ID:** FG002 **Number of Subjects:** 0 ##### Withdraw **Type:** Too Many Forms ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ###### Reason **Group ID:** FG002 **Number of Subjects:** 0 ##### Withdraw **Type:** Feeling Overwhelmed ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ###### Reason **Group ID:** FG002 **Number of Subjects:** 0 ##### Withdraw **Type:** Disliked Intervention ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ###### Reason **Group ID:** FG002 **Number of Subjects:** 0 ##### Withdraw **Type:** Changed Mind ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ###### Reason **Group ID:** FG002 **Number of Subjects:** 0 ##### Withdraw **Type:** Study Related Symptoms ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ###### Reason **Group ID:** FG002 **Number of Subjects:** 0 ##### Withdraw **Type:** Absence of Symptoms ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ###### Reason **Group ID:** FG002 **Number of Subjects:** 0 ##### Withdraw **Type:** Other Medical Reason ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ###### Reason **Group ID:** FG002 **Number of Subjects:** 1 ##### Withdraw **Type:** No Reason ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ###### Reason **Group ID:** FG002 **Number of Subjects:** 1 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 37 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 36 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 35 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 29 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 35 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 33 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 8 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 1 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 2 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT01922479 **Acronym:** PRACTICEASIAHF **Brief Title:** Pilot Study of Ferric Carboxymaltose to Treat Iron Deficiency in Asians With Heart Failure **Official Title:** Pilot RAndomized Controlled Trial of FerrIC CarboxymaltosE in ASIAns With Heart Failure (the PRACTICE-ASIA-HF) Study #### Organization Study ID Info **ID:** 2013/00265 #### Organization **Class:** OTHER **Full Name:** National University Hospital, Singapore #### Secondary ID Infos **Domain:** NUHS IMU Bridging Fund Award 2012 **ID:** IMU/BFA/2012/12 **Type:** OTHER_GRANT ### Status Module #### Completion Date **Date:** 2015-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-04-12 **Type:** ACTUAL **Last Update Submit Date:** 2017-04-11 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2015-09 **Type:** ACTUAL #### Start Date **Date:** 2013-09 **Type:** ACTUAL **Status Verified Date:** 2017-04 #### Study First Post Date **Date:** 2013-08-14 **Type:** ESTIMATED **Study First Submit Date:** 2013-08-11 **Study First Submit QC Date:** 2013-08-11 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** National University Heart Centre, Singapore **Class:** OTHER **Name:** Tan Tock Seng Hospital #### Lead Sponsor **Class:** OTHER **Name:** National University Hospital, Singapore #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Heart failure (HF) is a major global public health issue which also affects Asia. Data from the National Registry of Disease in Singapore shows a 9.4% rise in HF admissions in public hospitals from 2008 to 2009 (4140 to 4530). Anaemia (low blood Haemoglobin level) is a common problem occurring in HF, ranging from 14% to 56% in outpatient registries and clinical trials. Anaemia exacerbates the basic symptoms of HF of dyspnea and exercise intolerance, thereby reducing quality of life (QoL). However, recent approaches aimed at improving and normalizing Haemoglobin have been unsuccessful.Novel approaches are required to address this problem. Iron deficiency (ID) is a well-understood cause of anaemia. ID without overt anaemia may be present in HF patients. A recent study by Jankowska et al published in 2010 of 546 HF patients showed a 37% prevalence of ID, regardless of Haemoglobin level. This was associated with worse outcomes including impaired exercise capacity. The presence of ID indicates a higher likelihood of deteriorating and dying early. A landmark study published in the New England Journal of Medicine (The Ferinject Assessment in Patients with Iron Deficiency and Chronic Heart Failure (FAIR-HF) study) showed that HF patients who were treated with IV iron in the form of Ferric Carboxymaltose (FCM) had better outcomes, including improved exercise capacity, overall function, and quality of life. There is a lack of contemporary data on ID in HF patients in Asia, including data on treatment with this novel IV iron FCM. Hypothesis We hypothesise that treating ID in HF patients in Asia using FCM will improve outcomes including exercise capacity, quality of life, overall functional status, and the need to be hospitalised for complications arising from HF. **Detailed Description:** Heart failure (HF) is a major global public health issue which also affects Asia. Singapore National Registry of Disease data shows a 9.4% rise in public hospital HF admissions from 2008 to 2009 (4140 to 4530). Anaemia (low blood Haemoglobin level) is a common co-morbidity in HF, ranging from 14% to 56% in outpatient registries and clinical trials. Anaemia exacerbates the basic symptoms of HF of dyspnea and exercise intolerance, thereby reducing quality of life (QoL). However, recent approaches aimed at improving and normalizing Haemoglobin have been unsuccessful (STAMINA-HeFT, RED-HF).Novel approaches are required to address this problem. Iron deficiency (ID) is a well-understood cause of anaemia. ID without overt anaemia may be present in HF patients. The study by Jankowska (2010) of 546 systolic HF patients had a 37% prevalence of ID, regardless of Haemoglobin level. This was associated with reduced peak oxygen consumption, high ventilatory response, impaired exercise capacity, and depressive symptoms in HF patients. ID was a strong independent predictor of death, heart transplantation, and poor clinical outcome in chronic HF.The Ferinject Assessment in Patients with Iron Deficiency and Chronic Heart Failure (FAIR-HF) study showed significant improvement in 6MWT, NYHA class, and overall QoL score in HF patients treated with IV iron in the form of Ferric Carboxymaltose (FCM).Unpublished preliminary data from the ongoing Nation-wide Singapore study on Heart Failure (SHOP) indicates that the observed point prevalence of ID is approximately 60% with a significant and direct correlation with exercise performance.To date, no studies exist of FCM in an Asian HF population. We hypothesise that IV Iron repletion therapy using FCM in Asian patients with HF and ID will improve outcomes including exercise capacity (measured by 6MWT), quality of life (measured by the Kansas City Cardiomyopathy Questionnaire (KCCQ amp; VAS), NYHA functional class, and rate of HF hospitalization. Primary Aim To determine the effect of IV iron repletion therapy compared to placebo on exercise capacity change as assessed by the 6MWT at the 4th and 12th week after administration of IV FCM in subjects with recent acutely decompensated heart failure and iron deficiency. Secondary Aims To assess the effect of IV FCM compared with placebo on change in QoL assessments (KCCQ amp; VAS).To assess the effect of IV FCM compared with placebo on change in NYHA Functional Class.To assess the effect of IV FCM compared with placebo on the rate of HF Hospitalization.To assess the safety and tolerability of IV FCM compared to placebo. Hypothesis We hypothesise that IV Iron repletion therapy using FCM in patients with HF and ID will improve outcomes including exercise capacity (measured by 6MWT), quality of life (measured by KCCQ amp; VAS), NYHA functional class, and rate of HF hospitalization. ### Conditions Module **Conditions:** - Heart Failure - Anemia - Iron Deficiency **Keywords:** - Heart Failure - Anemia - Iron Deficiency - Ferric Carboxymaltose - Asian ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 50 **Type:** ACTUAL **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 1000mg intravenous Ferric Carboxymaltose, given as undiluted slow bolus injection over 15 minutes. Allowed to take concomitant oral iron supplements in usual clinical doses as prescribed clinically by attending physicians. **Intervention Names:** - Drug: Ferric Carboxymaltose **Label:** Ferric Carboxymaltose **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** 20mls intravenous Normal Saline (0.9%), given as slow bolus injection over 15 minutes. Allowed to take oral iron supplements in usual clinical doses as prescribed clinically by attending physicians. **Intervention Names:** - Drug: Placebo **Label:** Placebo **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Ferric Carboxymaltose **Description:** 1000mg intravenous Ferric Carboxymaltose, given as undiluted slow bolus injection over 15 minutes. Allowed to take concomitant oral iron supplements in usual clinical doses as prescribed clinically by attending physicians. **Name:** Ferric Carboxymaltose **Other Names:** - FerInject **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo **Description:** 20mls intravenous Normal Saline (0.9%), given as slow bolus injection over 15 minutes. Allowed to take concomitant oral iron supplements in usual clinical doses as prescribed clinically by attending physicians. **Name:** Placebo **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Assess the change in the patient's 6MWT distance over time, from baseline, at 4 weeks, and at 12 weeks. **Measure:** Change in 6MWT distance over time **Time Frame:** 12 weeks #### Secondary Outcomes **Description:** Assess the change in the patient's QoL as assessed by the Kansas City Cardiomyopathy Questionnaire (KCCQ) and the Visual Analogue Scale (VAS) over time, from baseline, at 4 weeks, and at 12 weeks. **Measure:** Change in QoL as assessed by the Kansas City Cardiomyopathy Questionnaire (KCCQ) and the Visual Analogue Scale (VAS). **Time Frame:** 12 weeks **Description:** Assess the change in the patient's NYHA Functional Class over time, from baseline, at 4 weeks, and at 12 weeks. **Measure:** Change in NYHA Functional Class **Time Frame:** 12 weeks **Description:** Assess the change in the patient's Rate of HF Hospitalisation over time, from baseline, at 4 weeks, and at 12 weeks. **Measure:** Rate of HF Hospitalisation **Time Frame:** 12 weeks **Description:** Assess any and all adverse events reported during the study, from baseline to 12 weeks. **Measure:** Summary of any adverse events reported during the study **Time Frame:** 12 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients hospitalized for HF (regardless of LVEF) * Capable of completing the 6MWT * Screening TSAT \<20%, Serum Ferritin \<300 ng/mL and Hb≤14 g/dL * At least 21 years of age * Written informed consent. Exclusion Criteria: * Acute coronary syndrome * Acute valvular heart dysfunction * Known sensitivity to FCM * IV iron therapy and/or blood transfusion in the 4 weeks prior to randomisation * Body weight ≤35 kg * Active bacterial infection * Haemochromatosis or other iron storage disorder * Serious medical condition, emergency condition, uncontrolled systemic disease or any other medical condition that, in the judgment of the Investigator, prohibits the patient from participating or potentially completing the study * Planned participation in any other interventional study or having received trial medication in the context of a clinical trial within the last 4 weeks prior to participating in this trial. **Maximum Age:** 90 Years **Minimum Age:** 21 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Singapore **Country:** Singapore **Facility:** National University Heart Centre, Singapore **Zip:** 119074 **Location 2:** **City:** Singapore **Country:** Singapore **Facility:** Tan Tock Seng Hospital **Zip:** 308433 #### Overall Officials **Official 1:** **Affiliation:** National University Heart Centre, Singapore **Name:** Carolyn SP Lam, MBBS, MRCP (UK) **Role:** STUDY_CHAIR **Official 2:** **Affiliation:** Tan Tock Seng Hospital **Name:** Poh Shuan Daniel Yeo, MBBS, MRCP(UK) **Role:** PRINCIPAL_INVESTIGATOR **Official 3:** **Affiliation:** National University Heart Centre, Singapore **Name:** Tee Joo Yeo, MBBS, MRCP (UK) **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000006331 - Term: Heart Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000000740 - Term: Anemia - ID: D000006402 - Term: Hematologic Diseases - ID: D000019189 - Term: Iron Metabolism Disorders - ID: D000008659 - Term: Metabolic Diseases - ID: D000000747 - Term: Anemia, Hypochromic ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC15 - Name: Blood and Lymph Conditions - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases ### Condition Browse Module - Browse Leaves - ID: M9421 - Name: Heart Failure - Relevance: HIGH - As Found: Heart Failure - ID: M20857 - Name: Anemia, Iron-Deficiency - Relevance: HIGH - As Found: Iron Deficiency - ID: M2781 - Name: Iron Deficiencies - Relevance: HIGH - As Found: Iron Deficiency - ID: M4070 - Name: Anemia - Relevance: LOW - As Found: Unknown - ID: M9419 - Name: Heart Diseases - Relevance: LOW - As Found: Unknown - ID: M9490 - Name: Hematologic Diseases - Relevance: LOW - As Found: Unknown - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: M21177 - Name: Iron Metabolism Disorders - Relevance: LOW - As Found: Unknown - ID: M4077 - Name: Anemia, Hypochromic - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006333 - Term: Heart Failure - ID: D000018798 - Term: Anemia, Iron-Deficiency - ID: D000090463 - Term: Iron Deficiencies ### Intervention Browse Module - Browse Branches - Abbrev: Micro - Name: Micronutrients - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Mi - Name: Mineral ### Intervention Browse Module - Browse Leaves - ID: M10533 - Name: Iron - Relevance: LOW - As Found: Unknown - ID: T341 - Name: Iron Supplement - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02234479 **Brief Title:** MediHoney for Radiation Dermatitis **Official Title:** A Pilot Study: Topical Application of Medihoney for Management of Radiation Dermatitis #### Organization Study ID Info **ID:** HP-00058645 #### Organization **Class:** OTHER **Full Name:** University of Maryland, Baltimore ### Status Module #### Completion Date **Date:** 2014-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-03-18 **Type:** ACTUAL **Last Update Submit Date:** 2020-03-16 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2014-11 **Type:** ACTUAL #### Results First Post Date **Date:** 2016-08-08 **Type:** ESTIMATED **Results First Submit Date:** 2016-02-23 **Results First Submit QC Date:** 2016-06-27 #### Start Date **Date:** 2014-06 **Status Verified Date:** 2020-03 #### Study First Post Date **Date:** 2014-09-09 **Type:** ESTIMATED **Study First Submit Date:** 2014-09-02 **Study First Submit QC Date:** 2014-09-04 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Integra LifeSciences Corporation #### Lead Sponsor **Class:** OTHER **Name:** University of Maryland, Baltimore #### Responsible Party **Investigator Affiliation:** University of Maryland, Baltimore **Investigator Full Name:** Department of Radiation Oncology **Investigator Title:** Principle Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The aim of this study is to compare the effects of Medihoney and Hydrophor on radiation dermatitis reactions in a group of women undergoing radiation therapy for breast cancer. It is hoped that the outcome of this pilot study will provide evidence supporting the use of Medihoney in preventing and treating radiation dermatitis as well as sufficient preliminary data to expand this study to larger, federally funded research (R01) looking at the beneficial aspects of Medihoney across a spectrum of radiation dermatitis and mucositis in several disease settings. **Detailed Description:** Background: Radiation dermatitis, also known as radiodermatitis or radiation skin reaction, is a widely reported side effect of radiation therapy in cancer. The most common radiation therapy-induced side effect is acute skin reaction, which can range from mild erythema to confluent moist desquamation. Almost all patients receiving radiation therapy have a risk of developing radiation dermatitis (Trueman, 2012). When not managed appropriately, radiation dermatitis can affect patients' physical functioning and quality of life. It can also cause pain and discomfort and may result in infection and/or interruption of treatment (Feight et al., 2011). An important role for oncology nurses is in educating, assessing, and monitoring patients for radiation dermatitis. Many nursing interventions are in use, including those based on tradition, physician preference, and published reports. In routine practice, most interventions for radiation dermatitis are institution-specific and not reliably evidence based. At the investigators institution, Hydrophor (Aquaphor), an ointment containing petrolatum, mineral oil, ceresin, and lanolin alcohol is the current standard of care for preventing and treating radiation dermatitis. Some women under treatment for breast cancer report disliking the smell and/or texture of Hydrophor. Rarely reported side effects have included allergic reactions, burning, stinging, and/or redness. Honey is an ancient remedy that has most recently shown promising results in treating burns, oral infections, and promoting surgical wound healing and palliation. The topical application of honey has been reported to be advantageous in radiation mucositis (Biswal et al., 2003). In a continuing effort to identify those treatment options that can make the investigators patients more comfortable and reduce complications from radiation dermatitis, the investigators are interested in conducting a small pilot study comparing the effectiveness of a honey-based treatment (Medihoney) with that of the investigators current standard of care (Hydrophor). ### Conditions Module **Conditions:** - Breast Cancer - Radiation Dermatitis **Keywords:** - Breast Cancer - Radiation Dermatitis ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 34 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Group A (current standard of care): Patients will be instructed (by nurses and with printed study materials) to apply a thin layer of the Hydrophor daily, starting at the onset of radiation therapy (RT) and continuing until 2 weeks after the final RT session or until the RT site is healed (whichever is first). Hydrophor application should include the entire treatment area, including the axillae and shoulder/back area in patients treated with modified radical mastectomy. To avoid possible build-up effects, patients should not apply the Hydrophor within 4 hours of receiving RT. Patients should wash the application area daily with perfume-free soap and tap water. Patients will be asked to refrain from using other topical agents in the irradiated area. **Intervention Names:** - Biological: Hydrophor (Group A) **Label:** Hydrophor (Group A) **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Group B (study target): Patients will be instructed (by nurses and with printed study materials) to apply a thin layer of the Medihoney daily, starting at the onset of RT and continuing until 2 weeks after the final RT session or until the RT site is healed (whichever is first). Medihoney application should include the entire treatment area, including the axillae and shoulder/back area in patients treated with modified radical mastectomy. To avoid possible build-up effects, patients should not apply the Medihoney within 4 hours of receiving RT. Patients should wash the application area daily with perfume-free soap and tap water. Patients will be asked to refrain from using other topical agents in the irradiated area. **Intervention Names:** - Biological: MediHoney (Group B) **Label:** MediHoney (Group B) **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Hydrophor (Group A) **Description:** * Rehydrates dry, chapped or chafed skin * May be used alone as a skin lubricant or protectant **Name:** Hydrophor (Group A) **Type:** BIOLOGICAL #### Intervention 2 **Arm Group Labels:** - MediHoney (Group B) **Description:** It helps the body's natural healing processes in three key ways which have been shown to have healing benefits: * Maintain a balanced environment for healing. * Aids in reducing dermatitis. * Reduce affected area pH.2-3 **Name:** MediHoney (Group B) **Type:** BIOLOGICAL ### Outcomes Module #### Primary Outcomes **Description:** The aim of this study is to compare the effects of Medihoney and Hydrophor on radiation dermatitis reactions in a group of women undergoing radiation therapy for breast cancer. It is hoped that the outcome of this pilot study will provide evidence supporting the use of Medihoney in preventing and treating radiation dermatitis as well as sufficient preliminary data to expand this study to larger, federally funded research (R01) looking at the beneficial aspects of Medihoney across a spectrum of radiation dermatitis and mucositis in several disease settings. **Measure:** Number of Participants Whom Received Medihoney Treatment and Were Analyzed Weekly for Skin Changes While Undergoing Radiation Therapy **Time Frame:** 12 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Breast cancer treated with either lumpectomy or mastectomy (with or without reconstruction). * The patient must be female. * Radiation therapy planned to whole breast/chestwall area (can include lymph node radiation; conventional 3D radiation, IMRT/IGRT, and hypofractionation are all allowed). * Age ≥ 18 years old. Exclusion Criteria: * Previous radiation therapy to chest area that would result in overlapping radiation fields. * Wound care issues. * Patients undergoing concurrent cytotoxic chemotherapy and radiation therapy (concurrent Herceptin and/or tamoxifen/aromatase inhibitors and RT is allowed). * Patients receiving HDR (savi or mammosite) brachytherapy treatments. * Patients with an allergy and/or sensitivity to Hydrophor, honey, and/or Medihoney. * Immunocompromised status. * Age \< 18 years old. **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Baltimore **Country:** United States **Facility:** University of Maryland **State:** Maryland **Zip:** 21201 #### Overall Officials **Official 1:** **Affiliation:** University of Maryland, College Park **Name:** Elizabeth Nichols, MD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012871 - Term: Skin Diseases - ID: D000011832 - Term: Radiation Injuries - ID: D000014947 - Term: Wounds and Injuries ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC26 - Name: Wounds and Injuries ### Condition Browse Module - Browse Leaves - ID: M5220 - Name: Breast Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7067 - Name: Dermatitis - Relevance: HIGH - As Found: Dermatitis - ID: M14701 - Name: Radiodermatitis - Relevance: HIGH - As Found: Radiation Dermatitis - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown - ID: M14679 - Name: Radiation Injuries - Relevance: LOW - As Found: Unknown - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000003872 - Term: Dermatitis - ID: D000011855 - Term: Radiodermatitis ### Intervention Browse Module - Browse Branches - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Ot - Name: Other Dietary Supplements ### Intervention Browse Module - Browse Leaves - ID: M4854 - Name: Benzocaine - Relevance: LOW - As Found: Unknown - ID: M21869 - Name: Protective Agents - Relevance: LOW - As Found: Unknown - ID: T433 - Name: Tannic Acid - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Hydrophor (Group A) **Description:** Group A (current standard of care): Patients will be instructed (by nurses and with printed study materials) to apply a thin layer of the Hydrophor daily, starting at the onset of radiation therapy (RT) and continuing until 2 weeks after the final RT session or until the RT site is healed (whichever is first). Hydrophor application should include the entire treatment area, including the axillae and shoulder/back area in patients treated with modified radical mastectomy. To avoid possible build-up effects, patients should not apply the Hydrophor within 4 hours of receiving RT. Patients should wash the application area daily with perfume-free soap and tap water. Patients will be asked to refrain from using other topical agents in the irradiated area. Hydrophor (Group A): •Rehydrates dry, chapped or chafed skin •May be used alone as a skin lubricant or protectant **ID:** EG000 **Other Num Affected:** 1 **Other Num at Risk:** 16 **Serious Number At Risk:** 16 **Title:** Hydrophor (Group A) **Group ID:** EG001 **Title:** MediHoney (Group B) **Description:** Group B (study target): Patients will be instructed (by nurses and with printed study materials) to apply a thin layer of the Medihoney daily, starting at the onset of RT and continuing until 2 weeks after the final RT session or until the RT site is healed (whichever is first). Medihoney application should include the entire treatment area, including the axillae and shoulder/back area in patients treated with modified radical mastectomy. To avoid possible build-up effects, patients should not apply the Medihoney within 4 hours of receiving RT. Patients should wash the application area daily with perfume-free soap and tap water. Patients will be asked to refrain from using other topical agents in the irradiated area. MediHoney (Group B): It helps the body's natural healing processes in three key ways which have been shown to have healing benefits: * Maintain a balanced environment for healing. * Aids in reducing dermatitis. * Reduce affected area pH.2-3 **ID:** EG001 **Other Num at Risk:** 18 **Serious Number At Risk:** 18 **Title:** MediHoney (Group B) **Frequency Threshold:** 0 #### Other Events **Term:** dermatitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 10.0 **Time Frame:** Adverse event data were collected in a 6 month period. ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 16 **Group ID:** BG001 **Value:** 18 **Group ID:** BG002 **Value:** 34 **Units:** Participants ### Group **ID:** BG000 **Title:** Hydrophor (Group A) **Description:** Group A (current standard of care): Patients will be instructed (by nurses and with printed study materials) to apply a thin layer of the Hydrophor daily, starting at the onset of radiation therapy (RT) and continuing until 2 weeks after the final RT session or until the RT site is healed (whichever is first). Hydrophor application should include the entire treatment area, including the axillae and shoulder/back area in patients treated with modified radical mastectomy. To avoid possible build-up effects, patients should not apply the Hydrophor within 4 hours of receiving RT. Patients should wash the application area daily with perfume-free soap and tap water. Patients will be asked to refrain from using other topical agents in the irradiated area. Hydrophor (Group A): •Rehydrates dry, chapped or chafed skin •May be used alone as a skin lubricant or protectant ### Group **ID:** BG001 **Title:** MediHoney (Group B) **Description:** Group B (study target): Patients will be instructed (by nurses and with printed study materials) to apply a thin layer of the Medihoney daily, starting at the onset of RT and continuing until 2 weeks after the final RT session or until the RT site is healed (whichever is first). Medihoney application should include the entire treatment area, including the axillae and shoulder/back area in patients treated with modified radical mastectomy. To avoid possible build-up effects, patients should not apply the Medihoney within 4 hours of receiving RT. Patients should wash the application area daily with perfume-free soap and tap water. Patients will be asked to refrain from using other topical agents in the irradiated area. MediHoney (Group B): It helps the body's natural healing processes in three key ways which have been shown to have healing benefits: * Maintain a balanced environment for healing. * Aids in reducing dermatitis. * Reduce affected area pH.2-3 ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 16 #### Measurement **Group ID:** BG001 **Value:** 18 #### Measurement **Group ID:** BG002 **Value:** 34 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 18 **Upper Limit:** 75 **Value:** 40 #### Measurement **Group ID:** BG001 **Lower Limit:** 18 **Upper Limit:** 75 **Value:** 40 #### Measurement **Group ID:** BG002 **Lower Limit:** 18 **Upper Limit:** 75 **Value:** 40 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 16 #### Measurement **Group ID:** BG001 **Value:** 18 #### Measurement **Group ID:** BG002 **Value:** 34 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 10 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 13 #### Measurement **Group ID:** BG001 **Value:** 11 #### Measurement **Group ID:** BG002 **Value:** 24 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 16 #### Measurement **Group ID:** BG001 **Value:** 18 #### Measurement **Group ID:** BG002 **Value:** 34 **Class Title:** United States **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Age, Categorical **Unit of Measure:** Participants ### Measure 2 **Dispersion Type:** FULL_RANGE **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race (NIH/OMB) **Unit of Measure:** Participants ### Measure 5 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants ## Results Section - More Information Module ### Certain Agreement **PI Sponsor Employee:** True ### Point of Contact **Email:** enichols1@umm.edu **Organization:** University of Maryland **Phone:** 410-328-2324 **Title:** Elizabeth Nichols, MD ## Results Section - Outcome Measures Module ### Outcome Measure 1 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 15 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 15 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 15 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 15 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** The aim of this study is to compare the effects of Medihoney and Hydrophor on radiation dermatitis reactions in a group of women undergoing radiation therapy for breast cancer. It is hoped that the outcome of this pilot study will provide evidence supporting the use of Medihoney in preventing and treating radiation dermatitis as well as sufficient preliminary data to expand this study to larger, federally funded research (R01) looking at the beneficial aspects of Medihoney across a spectrum of radiation dermatitis and mucositis in several disease settings. **Parameter Type:** NUMBER **Population Description:** Participants were analyzed for skin changes (during weekly visits with physician) during radiation treatment. Only 15 participants from each group completed the study, 1 subject from Group A and 3 subjects from Group B withdrew. **Reporting Status:** POSTED **Time Frame:** 12 months **Title:** Number of Participants Whom Received Medihoney Treatment and Were Analyzed Weekly for Skin Changes While Undergoing Radiation Therapy **Type:** PRIMARY **Unit of Measure:** participants ##### Group **Description:** Group A (current standard of care): Patients will be instructed (by nurses and with printed study materials) to apply a thin layer of the Hydrophor daily, starting at the onset of radiation therapy (RT) and continuing until 2 weeks after the final RT session or until the RT site is healed (whichever is first). Hydrophor application should include the entire treatment area, including the axillae and shoulder/back area in patients treated with modified radical mastectomy. To avoid possible build-up effects, patients should not apply the Hydrophor within 4 hours of receiving RT. Patients should wash the application area daily with perfume-free soap and tap water. Patients will be asked to refrain from using other topical agents in the irradiated area. Hydrophor (Group A): •Rehydrates dry, chapped or chafed skin •May be used alone as a skin lubricant or protectant **ID:** OG000 **Title:** Hydrophor (Group A) ##### Group **Description:** Group B (study target): Patients will be instructed (by nurses and with printed study materials) to apply a thin layer of the Medihoney daily, starting at the onset of RT and continuing until 2 weeks after the final RT session or until the RT site is healed (whichever is first). Medihoney application should include the entire treatment area, including the axillae and shoulder/back area in patients treated with modified radical mastectomy. To avoid possible build-up effects, patients should not apply the Medihoney within 4 hours of receiving RT. Patients should wash the application area daily with perfume-free soap and tap water. Patients will be asked to refrain from using other topical agents in the irradiated area. MediHoney (Group B): It helps the body's natural healing processes in three key ways which have been shown to have healing benefits: * Maintain a balanced environment for healing. * Aids in reducing dermatitis. * Reduce affected area pH.2-3 **ID:** OG001 **Title:** MediHoney (Group B) ### Participant Flow Module #### Group **Description:** Group A (current standard of care): Patients will be instructed (by nurses and with printed study materials) to apply a thin layer of the Hydrophor daily, starting at the onset of radiation therapy (RT) and continuing until 2 weeks after the final RT session or until the RT site is healed (whichever is first). Hydrophor application should include the entire treatment area, including the axillae and shoulder/back area in patients treated with modified radical mastectomy. To avoid possible build-up effects, patients should not apply the Hydrophor within 4 hours of receiving RT. Patients should wash the application area daily with perfume-free soap and tap water. Patients will be asked to refrain from using other topical agents in the irradiated area. Hydrophor (Group A): •Rehydrates dry, chapped or chafed skin •May be used alone as a skin lubricant or protectant **ID:** FG000 **Title:** Hydrophor (Group A) #### Group **Description:** Group B (study target): Patients will be instructed (by nurses and with printed study materials) to apply a thin layer of the Medihoney daily, starting at the onset of RT and continuing until 2 weeks after the final RT session or until the RT site is healed (whichever is first). Medihoney application should include the entire treatment area, including the axillae and shoulder/back area in patients treated with modified radical mastectomy. To avoid possible build-up effects, patients should not apply the Medihoney within 4 hours of receiving RT. Patients should wash the application area daily with perfume-free soap and tap water. Patients will be asked to refrain from using other topical agents in the irradiated area. MediHoney (Group B): It helps the body's natural healing processes in three key ways which have been shown to have healing benefits: * Maintain a balanced environment for healing. * Aids in reducing dermatitis. * Reduce affected area pH.2-3 **ID:** FG001 **Title:** MediHoney (Group B) #### Period **Title:** Overall Study ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 3 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 16 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 18 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 15 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 15 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 1 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 3 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT04943679 **Brief Title:** Anti-PD-1/PD-L1 Antibodies Plus Pegylated Interferon Alfa-2b Treatment in Patients With Advanced-Stage HCC **Official Title:** Phase I/II Study of Anti-PD-1/PD-L1 Antibodies Combined With Pegylated Interferon Alfa-2b in Patients With Advanced-Stage Hepatocellular Carcinoma #### Organization Study ID Info **ID:** B20210618 #### Organization **Class:** OTHER **Full Name:** Shanghai Zhongshan Hospital ### Status Module #### Completion Date **Date:** 2024-12-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2022-03-31 **Type:** ACTUAL **Last Update Submit Date:** 2022-03-28 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-12-31 **Type:** ESTIMATED #### Start Date **Date:** 2021-06-15 **Type:** ACTUAL **Status Verified Date:** 2022-03 #### Study First Post Date **Date:** 2021-06-29 **Type:** ACTUAL **Study First Submit Date:** 2021-06-21 **Study First Submit QC Date:** 2021-06-21 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Shanghai Zhongshan Hospital #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** This study is being done to analyze the safety, tolerability, and efficacy of treatment using combination of Pegylated Interferon Alfa-2b and anti-PD-1/PD-L1 antibodies for patients with advanced hepatocellular carcinoma. **Detailed Description:** This is a study of combination anti-PD-1/PD-L1 antibodies and peginterferon alfa-2b for adult patients (≥18) with advanced hepatocellular carcinoma. Each 21 day dosing period will constitute a cycle. Pegylated Interferon Alfa-2b has been proven to prolong the survival of HCC patients. Pegylated Interferon Alfa-2b is given subcutaneously, weekly during each 21-day cycle, for at least 6 cycles (18 weeks). Treatment may continue until disease progression, intolerable toxicity, or consent withdrawal. Anti-PD-1/PD-L1 antibodies (including pembrolizumeb, nivolumab, sintilimab, toripalimab, camrelizumeb, tislelizumab and atezolizumab etc.) are given intravenously at assigned dose. Treatment may continue until disease progression, intolerable toxicity, or consent withdrawal. This study is aimed to evaluate the safety and efficacy of the combination of Pegylated Interferon Alfa-2b and PD-1/PD-L1 mAb in unresectable late-stage HCC patients. ### Conditions Module **Conditions:** - Hepatocellular Carcinoma **Keywords:** - Hepatocellular Carcinoma - Anti-PD-1 antibody - Interferon Alfa - Anti-PD-L1 antibody ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 15 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Peginterferon alfa-2b: 3 µg/kg every week by subcutaneous injection for up to 2 years; Anti-PD-1/PD-L1 Antibodies: given by intravenous injection at indicated dose for up to 2 years **Intervention Names:** - Drug: Anti-PD-1/PD-L1 - Drug: PEG-Interferon Alfa **Label:** Anti-PD-1/PD-L1 antibodies and Pegylated Interferon Alfa-2b ### Interventions #### Intervention 1 **Arm Group Labels:** - Anti-PD-1/PD-L1 antibodies and Pegylated Interferon Alfa-2b **Description:** Intravenous injection for up to 2 years **Name:** Anti-PD-1/PD-L1 **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Anti-PD-1/PD-L1 antibodies and Pegylated Interferon Alfa-2b **Description:** 3 µg/kg every week by subcutaneous injection for up to 2 years **Name:** PEG-Interferon Alfa **Other Names:** - Pegasys **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Safety will be monitored by addressing and recording all adverse events (AEs), serious adverse events (SAEs) and specific laboratory abnormalities (worst grade). Toxicities will be graded using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0. **Measure:** Incidence of adverse events **Time Frame:** Up to 30 days after last treatment dose #### Secondary Outcomes **Description:** Evaluated by researchers based on the RECIST 1.1 standard **Measure:** Objective response rate(ORR) **Time Frame:** 2 years **Description:** Evaluated by researchers based on the RECIST 1.1 standard **Measure:** Progression free survival(PFS) [ Time Frame: 2 years ] **Time Frame:** 2 years **Description:** Evaluated by researchers based on the RECIST 1.1 standard **Measure:** To the relief time (TOR) **Time Frame:** 2 years **Description:** Evaluated by researchers based on the RECIST 1.1 standard **Measure:** Duration of relief(DOR) **Time Frame:** 2 years **Description:** Evaluated by researchers based on the RECIST 1.1 standard **Measure:** Disease Control Rate (DCR) **Time Frame:** 2 years **Description:** Evaluated by researchers based on the RECIST 1.1 standard **Measure:** 9-month survival rate **Time Frame:** 9-month **Description:** Evaluated by researchers based on the RECIST 1.1 standard **Measure:** 12-month survival rate **Time Frame:** 12-month ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. ≥18 years old, male or female 2. Advanced hepatocellular carcinoma (cannot be removed or metastasized) diagnosed clinically or pathologically, at least one measurable lesion without local treatment, Child-Pugh A ;Barcelona Clinic Liver Cancer(BCLC) staging is stage B or C 3. Eastern Cooperative Oncology Group (ECOG) Performance Status 0-1 4. Patient has given written informed consent. 5. The function of important organs meets the requirements 6. Expected survival ≥12 weeks 7. Non-surgical sterilization or women of childbearing age need to use a medically-accepted contraceptive (such as an intrauterine device, contraceptive or condom) during the study period and within 3 months after the end of the study treatment period. Exclusion Criteria: 1.The patient has any active autoimmune disease or a history of autoimmune disease (such as the following, but not limited to: autoimmune hepatitis, interstitial pneumonia, uveitis, enteritis, hepatitis, pituitary inflammation, vasculitis, nephritis, thyroid Hyperfunction; patients with vitiligo; complete remission of asthma in childhood, can be included without any intervention after adulthood; asthma patients who require bronchodilators for medical intervention cannot be included); 2.The patient is using immunosuppressive agents or systemic hormonal therapy to achieve immunosuppressive purposes (agents amount \> 10 mg / day of prednisone or other therapeutic hormones), and continue to use within 2 weeks before enrollment; 3.Have clinical symptoms or disease that are not well controlled 4.Significant clinically significant bleeding symptoms or a clear bleeding tendency within 3 months prior to randomization 5.Arterial/venous thrombosis in the first 6 months of randomization 6.According to the investigator, the patient has other factors that may affect the results of the study or lead to the termination of the study, such as alcohol abuse, drug abuse, other serious diseases (including mental illness) requiring combined treatment, and serious laboratory abnormalities.，with family or social factors, it will affect the safety of patients. 7.Liver tumor burden greater than 50% of the total liver volume, or patients who have previously undergone liver transplantation;Known for a history of central nervous system metastasis or hepatic encephalopathy;Severe allergic reactions to other monoclonal antibodies; - **Maximum Age:** 90 Years **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Advanced HCC patients who received the combination therapy with Anti-PD-1/PD-L1 Antibodies Plus Pegylated Interferon Alfa-2b Treatment ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** 191784843@qq.com **Name:** Jing Ma, M.D. **Phone:** +86 18721278764 **Role:** CONTACT #### Locations **Location 1:** **City:** Shanghai **Contacts:** ***Contact 1:*** - **Email:** 191784843@qq.com - **Name:** Jin Ma - **Phone:** +86 18721278764 - **Role:** CONTACT **Country:** China **Facility:** Zhongshan Hospital, Fudan University **Status:** RECRUITING **Zip:** 200032 #### Overall Officials **Official 1:** **Affiliation:** Fudan University **Name:** Yang Xu, M.D.&Ph.D **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Ji J, Shi J, Budhu A, Yu Z, Forgues M, Roessler S, Ambs S, Chen Y, Meltzer PS, Croce CM, Qin LX, Man K, Lo CM, Lee J, Ng IO, Fan J, Tang ZY, Sun HC, Wang XW. MicroRNA expression, survival, and response to interferon in liver cancer. N Engl J Med. 2009 Oct 8;361(15):1437-47. doi: 10.1056/NEJMoa0901282. **PMID:** 19812400 **Citation:** Davar D, Wang H, Chauvin JM, Pagliano O, Fourcade JJ, Ka M, Menna C, Rose A, Sander C, Borhani AA, Karunamurthy A, Tarhini AA, Tawbi HA, Zhao Q, Moreno BH, Ebbinghaus S, Ibrahim N, Kirkwood JM, Zarour HM. Phase Ib/II Study of Pembrolizumab and Pegylated-Interferon Alfa-2b in Advanced Melanoma. J Clin Oncol. 2018 Oct 25;36(35):JCO1800632. doi: 10.1200/JCO.18.00632. Online ahead of print. **PMID:** 30359157 **Citation:** Finn RS, Ryoo BY, Merle P, Kudo M, Bouattour M, Lim HY, Breder V, Edeline J, Chao Y, Ogasawara S, Yau T, Garrido M, Chan SL, Knox J, Daniele B, Ebbinghaus SW, Chen E, Siegel AB, Zhu AX, Cheng AL; KEYNOTE-240 investigators. Pembrolizumab As Second-Line Therapy in Patients With Advanced Hepatocellular Carcinoma in KEYNOTE-240: A Randomized, Double-Blind, Phase III Trial. J Clin Oncol. 2020 Jan 20;38(3):193-202. doi: 10.1200/JCO.19.01307. Epub 2019 Dec 2. **PMID:** 31790344 **Citation:** Zhou J, Sun H, Wang Z, Cong W, Wang J, Zeng M, Zhou W, Bie P, Liu L, Wen T, Han G, Wang M, Liu R, Lu L, Ren Z, Chen M, Zeng Z, Liang P, Liang C, Chen M, Yan F, Wang W, Ji Y, Yun J, Cai D, Chen Y, Cheng W, Cheng S, Dai C, Guo W, Hua B, Huang X, Jia W, Li Y, Li Y, Liang J, Liu T, Lv G, Mao Y, Peng T, Ren W, Shi H, Shi G, Tao K, Wang W, Wang X, Wang Z, Xiang B, Xing B, Xu J, Yang J, Yang J, Yang Y, Yang Y, Ye S, Yin Z, Zhang B, Zhang B, Zhang L, Zhang S, Zhang T, Zhao Y, Zheng H, Zhu J, Zhu K, Liu R, Shi Y, Xiao Y, Dai Z, Teng G, Cai J, Wang W, Cai X, Li Q, Shen F, Qin S, Dong J, Fan J. Guidelines for the Diagnosis and Treatment of Hepatocellular Carcinoma (2019 Edition). Liver Cancer. 2020 Dec;9(6):682-720. doi: 10.1159/000509424. Epub 2020 Nov 11. **PMID:** 33442540 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000000230 - Term: Adenocarcinoma - ID: D000008113 - Term: Liver Neoplasms - ID: D000004067 - Term: Digestive System Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000004066 - Term: Digestive System Diseases - ID: D000008107 - Term: Liver Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions - Abbrev: BC06 - Name: Digestive System Diseases ### Condition Browse Module - Browse Leaves - ID: M5534 - Name: Carcinoma - Relevance: HIGH - As Found: Carcinoma - ID: M9613 - Name: Carcinoma, Hepatocellular - Relevance: HIGH - As Found: Hepatocellular Carcinoma - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M3585 - Name: Adenocarcinoma - Relevance: LOW - As Found: Unknown - ID: M11113 - Name: Liver Neoplasms - Relevance: LOW - As Found: Unknown - ID: M8886 - Name: Gastrointestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7256 - Name: Digestive System Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M11107 - Name: Liver Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002277 - Term: Carcinoma - ID: D000006528 - Term: Carcinoma, Hepatocellular ### Intervention Browse Module - Ancestors - ID: D000000970 - Term: Antineoplastic Agents - ID: D000000998 - Term: Antiviral Agents - ID: D000000890 - Term: Anti-Infective Agents - ID: D000007155 - Term: Immunologic Factors - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: ANeo - Name: Antineoplastic Agents ### Intervention Browse Module - Browse Leaves - ID: M4225 - Name: Antibodies - Relevance: LOW - As Found: Unknown - ID: M10184 - Name: Immunoglobulins - Relevance: LOW - As Found: Unknown - ID: M10407 - Name: Interferons - Relevance: HIGH - As Found: Recurrent - ID: M254669 - Name: Peginterferon alfa-2b - Relevance: LOW - As Found: Unknown - ID: M19243 - Name: Interferon-alpha - Relevance: HIGH - As Found: Status - ID: M247369 - Name: Peginterferon alfa-2a - Relevance: LOW - As Found: Unknown - ID: M1685 - Name: Interferon alpha-2 - Relevance: LOW - As Found: Unknown - ID: M4314 - Name: Antiviral Agents - Relevance: LOW - As Found: Unknown - ID: M4214 - Name: Anti-Infective Agents - Relevance: LOW - As Found: Unknown - ID: M10201 - Name: Immunologic Factors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000007372 - Term: Interferons - ID: D000016898 - Term: Interferon-alpha ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01085279 **Brief Title:** Fractional Laser as Treatment Option for Various Pigment Disorders (Fractional-3) **Official Title:** Non-ablative 1,550 nm Fractional Laser Therapy Versus Triple Topical Therapy for the Treatment of Melasma: a Randomized Controlled Split-face Study #### Organization Study ID Info **ID:** fractional-3 #### Organization **Class:** OTHER **Full Name:** Netherlands Institute for Pigment Disorders ### Status Module #### Completion Date **Date:** 2010-02 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2010-03-11 **Type:** ESTIMATED **Last Update Submit Date:** 2010-03-10 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2010-02 **Type:** ACTUAL #### Start Date **Date:** 2009-03 **Status Verified Date:** 2010-03 #### Study First Post Date **Date:** 2010-03-11 **Type:** ESTIMATED **Study First Submit Date:** 2010-03-10 **Study First Submit QC Date:** 2010-03-10 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Netherlands Institute for Pigment Disorders #### Responsible Party **Old Name Title:** Albert Wolkerstorfer, MD PhD **Old Organization:** Netherlands Institute for Pigment Disorders ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to determine whether the use of non-ablative fractional laser is safe and effective in the treatment of melasma. ### Conditions Module **Conditions:** - Pigmentation Disorder **Keywords:** - fractional laser - melasma - topical bleaching ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 29 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** In each patient, one side of the face was treated with non-ablative fractional laser in four-five sessions. Note: this study had a split-face design. In each patient, each side of the face was randomized to receive either non-ablative fractional laser therapy or triple topical therapy. **Intervention Names:** - Device: Fraxel Restore, Solta Medical Inc. (Non-ablative fractional laser) **Label:** Non-ablative fractional laser **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** In each patient, one side of the face was treated with triple topical therapy (Hydroquinone 5%, tretinoin 0.05%, triamcinolone acetonide 0.1%) during 15 weeks. Note: this study had a split-face design. In each patient, each side of the face was randomized to receive either non-ablative fractional laser therapy or triple topical therapy. **Intervention Names:** - Drug: Modified Kligman's formula (Triple topical therapy) **Label:** Triple topical therapy **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Non-ablative fractional laser **Description:** 1,550 nm non-ablative fractional laser Irradiance: 15 mJ/microbeam. Coverage: 14-20%. Number of treatment sessions: 4-5 **Name:** Fraxel Restore, Solta Medical Inc. (Non-ablative fractional laser) **Other Names:** - Fraxel re:store, Solta Medical Inc., Hayward, CA **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - Triple topical therapy **Description:** Hydroquinone 5%, tretinoin 0.05%, triamcinolone acetonide 0.1% once a day during 15 weeks **Name:** Modified Kligman's formula (Triple topical therapy) **Other Names:** - modified Kligman formula **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Improvement of hyperpigmentation was assessed by an independent blinded dermatologist. The results were scored on a scale from zero to six (0: total clearance (100% improvement), 1: almost total clearance (90-99% improvement), 2: distinct clearance (75-89% improvement) 3: moderate clearance (50-74% improvement) 4: mild clearance (25-49% improvement) 5: no change, 6: worsening of hyperpigmentation). **Measure:** Physician's global assessment **Time Frame:** T0, 3 weeks, and 3 and 6 months follow-up #### Secondary Outcomes **Description:** Improvement of hyperpigmentation was assessed by color measurement through reflectance spectroscopy (Microflash 200 d, Datacolor International, Lawrenceville, GA). This instrument, with an aperture of 4 mm, determines color by measuring the intensity of reflected light of particular wavelengths. In this study, the obtained L value, indicating the lightness of the measured area of skin, was used. **Measure:** L-value **Time Frame:** T0, 3 weeks and 3 and 6 months follow-up **Description:** Melanin index was measured using a spectrometer (Derma-Spectrometer, Cortex Technology ApS, Hadsund, Denmark) in order to assess changes in the amount of dermal and epidermal melanin. **Measure:** Melanin index **Time Frame:** T0, 3 weeks and 3, and 6 months follow-up **Description:** Patients were asked to score the improvement of hyperpigmentation on a visual analogue scale (VAS) from 0 to 10 (Patient's Global Assessment, PGA) at all follow-up moments. **Measure:** Patient's global assessment **Time Frame:** 3 weeks, 3 and 6 months follow-up **Description:** Patient's satisfaction was scored on a visual analogue scale (VAS) from 0 to 10. **Measure:** Patient's satisfaction **Time Frame:** 3 weeks, 3 and 6 months follow-up ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Melasma * Subjects attending the outpatient department of the Netherlands Institute for Pigment Disorders * Age at least 18 years * Subject is willing and able to give written informed consent Exclusion Criteria: * use of bleaching creams during the past six weeks * history of keloid * active eczema * suspected hypersensitivity to lidocaine or triple therapy * use of isotretinoin in the past six months * high exposure of the lesion to sunlight or UV light (UVA or UVB). **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Amsterdam **Country:** Netherlands **Facility:** Netherlands Institute for Pigment disorders **Zip:** 1105 AZ #### Overall Officials **Official 1:** **Affiliation:** Netherlands Institute for Pigment Disorders **Name:** Albert Wolkerstorfer, MD PhD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012871 - Term: Skin Diseases - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases ### Condition Browse Module - Browse Leaves - ID: M21089 - Name: Facies - Relevance: LOW - As Found: Unknown - ID: M13754 - Name: Pigmentation Disorders - Relevance: HIGH - As Found: Pigmentation Disorder - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010859 - Term: Pigmentation Disorders ### Intervention Browse Module - Browse Branches - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: Derm - Name: Dermatologic Agents ### Intervention Browse Module - Browse Leaves - ID: M16975 - Name: Triamcinolone Acetonide - Relevance: LOW - As Found: Unknown - ID: M16974 - Name: Triamcinolone - Relevance: LOW - As Found: Unknown - ID: M237966 - Name: Triamcinolone hexacetonide - Relevance: LOW - As Found: Unknown - ID: M209573 - Name: Triamcinolone diacetate - Relevance: LOW - As Found: Unknown - ID: M16965 - Name: Tretinoin - Relevance: LOW - As Found: Unknown - ID: M261781 - Name: Hydroquinone - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05580679 **Brief Title:** Effect of Abdominal Massage on Palliative Care Patients **Official Title:** The Effect of Abdominal Massage on Managing Constipation in Palliative Care Patients #### Organization Study ID Info **ID:** 1919B012101971 #### Organization **Class:** OTHER **Full Name:** Bartın Unıversity ### Status Module #### Completion Date **Date:** 2022-12-30 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-09-21 **Type:** ACTUAL **Last Update Submit Date:** 2023-09-17 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-08-30 **Type:** ACTUAL #### Start Date **Date:** 2022-03-15 **Type:** ACTUAL **Status Verified Date:** 2023-09 #### Study First Post Date **Date:** 2022-10-14 **Type:** ACTUAL **Study First Submit Date:** 2022-10-07 **Study First Submit QC Date:** 2022-10-11 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** The Scientific and Technological Research Council of Turkey #### Lead Sponsor **Class:** OTHER **Name:** Bartın Unıversity #### Responsible Party **Investigator Affiliation:** Bartın Unıversity **Investigator Full Name:** Özge Uçar **Investigator Title:** Research Assistant **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** Constipation is defined as infrequent defecation or difficulty in evacuation. Although the constipation problem is mostly self-managed by the patients, 22% mostly refer to primary health care providers (\>50%), resulting in large expenditures for diagnostic tests and treatments. According to studies conducted in our country, the incidence of constipation varies between 22-40%, while it is stated that the prevalence of constipation in hospitalized patients is 79%. Annual medical costs due to constipation are estimated to exceed 230 million dollars. Pharmacological and non-pharmacological methods are used in the management of constipation, which is one of the most common symptoms in the hospital. The use of non-pharmacological methods is recommended due to the possibility of side effects and cost of pharmacological methods. However, the prevalence of use of non-pharmacological methods in the clinic is low. Nurses need to have knowledge about non-pharmacological methods in order to manage the problem of constipation, which is one of the most common symptoms in the hospital. Abdominal massage, which is included in the Nursing Interventions Classification (NIC), is an application performed with manipulations such as pressure and rubbing on certain points on the body to reduce pain, provide relaxation and relaxation, prevent and reduce nausea, and prevent constipation. **Detailed Description:** Introduction: Constipation is defined as infrequent defecation or difficulty in evacuation. Although the constipation problem is mostly self-managed by the patients, 22% mostly refer to primary health care providers (\>50%), resulting in large expenditures for diagnostic tests and treatments. According to studies conducted in our country, the incidence of constipation varies between 22-40%, while it is stated that the prevalence of constipation in hospitalized patients is 79%. Annual medical costs due to constipation are estimated to exceed 230 million dollars. Pharmacological and non-pharmacological methods are used in the management of constipation, which is one of the most common symptoms in the hospital. The use of non-pharmacological methods is recommended due to the possibility of side effects and cost of pharmacological methods. However, the prevalence of use of non-pharmacological methods in the clinic is low. Nurses need to have knowledge about non-pharmacological methods in order to manage the problem of constipation, which is one of the most common symptoms in the hospital. Abdominal massage, which is included in the Nursing Interventions Classification (NIC), is an application performed with manipulations such as pressure and rubbing on certain points on the body to reduce pain, provide relaxation and relaxation, prevent and reduce nausea, and prevent constipation. Objective: This study was planned to examine the effect of abdominal massage on the management of constipation in palliative care patients. Method: The project will be carried out in a state hospital in Bartın, between 01.09.2021 and 01.09.2022, in the palliative care service, with patients over 18 years of age, without a history of gastrointestinal cancer, history of abdominal surgery or abdominal hernia, and hospitalized for at least 7 days. Palliative care patients hospitalized in Bartın State Hospital in the last 1 year will form the universe. Sample calculation will be made with G power analysis and the participants will be divided into experimental and control groups with block randomization. The patients in the experimental group will be given abdominal massage in the morning and evening every day for 1 week and the defecation status of the patients will be compared. The data will be recorded in the data collection form prepared in line with the literature. The data will be used in the patient identification form, Gastrointestinal Symptom Rating Scale, Constipation Evaluation Scale, Bristol Stool Consistency Scale. The collected data will be evaluated using the SPSS program. Conclusion: With this project, the incidence of constipation in palliative patients will be determined and the knowledge and skills of nursing students on how to apply abdominal massage, which is one of the nursing practices, will increase. With their practice in this study, they will play an active role in the management of constipation in palliative patients, and they will be able to perform this practice in patients in various services throughout their education and professional lives. ### Conditions Module **Conditions:** - Constipation - Abdominal Pain - Palliative Care **Keywords:** - palliative - palliative care - nursing - abdominal massage - constipation ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER **Intervention Model Description:** Randomized Controlled Trials ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 90 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Abdominal massage using for evaluation of constipation. **Intervention Names:** - Other: Abdominal massage **Label:** Abdominal massage group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** No intervention palliative care patient **Label:** Standart care group **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Abdominal massage group **Description:** Application time of the massage: 30 minutes after meals, twice a day, at 10:00 in the morning and at 16:00 in the evening. Application time of the massage: 15 minutes each Materials needed: Gloves, Liquid Vaseline, Paper Towel Massage methods used: Efflorage, Petrissage, Vibration **Name:** Abdominal massage **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** First measurements of both group before intervention using by Bristol Stool Consistency Scale. The Bristol Stool Consistency Scale, developed by a team of gastroenterologists at the University of Bristol, UK; It is used to evaluate stool form and to have information about changes in bowel habits and a pathological formation that may occur in the intestine. This scale was designed to classify an individual's bowel movements in seven distinct categories of stool. According to the Bristol Stool Consistency Scale, Type 1 and Type 2 are constipation, Type 3 and Type 4 are normal stools, and Type 5, Type 6 and Type 7 are diarrhea. It is accepted that there is a direct relationship between stool shape and length of stay in the colon (Lewis and Heaton 1997). **Measure:** First measurements of defecation type. **Time Frame:** 1-7 days **Description:** First measurements of both groups before the intervention using a Gastrointestinal Symptom Rating Scale.The Turkish validity and reliability of the scale, which was developed by Revicki et al. in 1998 to evaluate the symptoms frequently seen in gastrointestinal system disorders, was performed by Turan and Aştı in 2011. GSRS; It is a 15-item Likert-type scale with options starting from "no discomfort" to "very severe discomfort". Based on factor analysis, 15 items of the GSRS have five sub-dimensions: Abdominal Pain, Reflux, Diarrhea, Indigestion and Constipation. In the GSRS, how the individual felt in terms of gastrointestinal problems in the last week is questioned. High scores on the GSRS indicate that the symptoms are more severe (Revicki et al. 1998; Kaya and Turan 2011; Turan and Aştı 2011). **Measure:** First measurements of gastrointestinal symptoms **Time Frame:** 1-7 days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Volunteering * Being over 18 years old * No history of gastrointestinal cancer, history of abdominal surgery or abdominal hernia * At least 7 days of clinical treatment Exclusion Criteria: * Patient information form * Gastrointestinal system rating scale * Constipation assessment scale * Bristol stool consistency scale **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Bartın **Country:** Turkey **Facility:** Özge Uçar **Zip:** 74100 #### Overall Officials **Official 1:** **Affiliation:** Bartin University Health Science Faculty **Name:** Özge Uçar **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Bartin University Health Science Faculty **Name:** Nursena Eren **Role:** STUDY_DIRECTOR **Official 3:** **Affiliation:** Bartin University Health Science Faculty **Name:** Nisa Ün **Role:** STUDY_CHAIR **Official 4:** **Affiliation:** Bartin University Health Science Faculty **Name:** Erdem Altuner **Role:** STUDY_CHAIR **Official 5:** **Affiliation:** Bartin University Health Science Faculty **Name:** İrem Yılmaz **Role:** STUDY_CHAIR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012817 - Term: Signs and Symptoms, Digestive - ID: D000010146 - Term: Pain - ID: D000009461 - Term: Neurologic Manifestations ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases ### Condition Browse Module - Browse Leaves - ID: M6472 - Name: Constipation - Relevance: HIGH - As Found: Constipation - ID: M18311 - Name: Abdominal Pain - Relevance: HIGH - As Found: Abdominal Pain - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown - ID: M15622 - Name: Signs and Symptoms, Digestive - Relevance: LOW - As Found: Unknown - ID: M12404 - Name: Neurologic Manifestations - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000003248 - Term: Constipation - ID: D000015746 - Term: Abdominal Pain ### Intervention Browse Module - Browse Branches - Abbrev: Derm - Name: Dermatologic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M13485 - Name: Petrolatum - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02412579 **Brief Title:** Genetic Profiling of Liver Cancer in Patients Undergoing Liver Transplantation **Official Title:** Circulating MicroRNA Isoforms as Biomarkers in Hepatocellular Carcinoma and Associated Liver Transplantation #### Organization Study ID Info **ID:** 014-121 #### Organization **Class:** OTHER **Full Name:** Baylor Research Institute ### Status Module #### Completion Date **Date:** 2022-03-17 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-07-20 **Type:** ACTUAL **Last Update Submit Date:** 2022-07-19 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-03-17 **Type:** ACTUAL #### Start Date **Date:** 2015-03 **Type:** ACTUAL **Status Verified Date:** 2022-07 #### Study First Post Date **Date:** 2015-04-09 **Type:** ESTIMATED **Study First Submit Date:** 2015-04-06 **Study First Submit QC Date:** 2015-04-06 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Baylor Research Institute #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to determine whether genetic markers unique to liver cancer are present and to assess usefulness as a diagnostic tool. **Detailed Description:** Hepatocellular carcinoma (HCC) is the most common form of liver cancer and a growing trend worldwide. Decision-making regarding treatment for patients is limited by the lack of clinically actionable biomarkers. This is pronounced in the case for determining and monitoring liver transplant recipients. In an effort to improve diagnosis, the use of circulating serum-based microRNA isoforms (isomiRs) can serve as non-invasive, screening biomarkers in HCC patients pre- and post-transplantation of the liver. ### Conditions Module **Conditions:** - Hepatocellular Carcinoma **Keywords:** - HCC - Liver Cancer ### Design Module #### Bio Spec **Description:** Hepatocellular carcinoma tumor tissue Background liver tissue **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 40 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Subjects with hepatocellular carcinoma and cirrhosis. **Intervention Names:** - Other: None - Standard of Care **Label:** Hepatocellular Carcinoma with Cirrhosis #### Arm Group 2 **Description:** Subjects with only cirrhosis. **Intervention Names:** - Other: None - Standard of Care **Label:** Cirrhosis without Hepatocellular Carcinoma ### Interventions #### Intervention 1 **Arm Group Labels:** - Cirrhosis without Hepatocellular Carcinoma - Hepatocellular Carcinoma with Cirrhosis **Name:** None - Standard of Care **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** Using next-generation sequencing methods to detect isomiRs across different HCC cohorts at all stages undergoing liver transplantation. **Time Frame:** One year ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients with the diagnosis of HCC who are on the wait list for liver transplantation * Patients with the diagnosis of HCC who have been admitted to hospital, scheduled to undergo liver transplantation within the following 24 hours. * Age Range: 18 to 75 years old Exclusion Criteria: * Patients with metastatic HCC and patients with the diagnosis of HCC who are not on the wait list for liver transplantation. * Patients older than 75 years old and younger than 18 years old. * Patients who are unable to consent. * Pregnant patients. **Maximum Age:** 75 Years **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Hepatocellular Carcinoma Clinic Pre-Liver Transplant Clinic ### Contacts Locations Module #### Locations **Location 1:** **City:** Dallas **Country:** United States **Facility:** Baylor University Medical Center **State:** Texas **Zip:** 75246 #### Overall Officials **Official 1:** **Affiliation:** Annette C. & Harold C. Simmons Transplant Institute **Name:** Peter Kim, M.D. **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000000230 - Term: Adenocarcinoma - ID: D000008113 - Term: Liver Neoplasms - ID: D000004067 - Term: Digestive System Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000004066 - Term: Digestive System Diseases - ID: D000008107 - Term: Liver Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions - Abbrev: BC06 - Name: Digestive System Diseases ### Condition Browse Module - Browse Leaves - ID: M5534 - Name: Carcinoma - Relevance: HIGH - As Found: Carcinoma - ID: M9613 - Name: Carcinoma, Hepatocellular - Relevance: HIGH - As Found: Hepatocellular Carcinoma - ID: M11113 - Name: Liver Neoplasms - Relevance: LOW - As Found: Unknown - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M3585 - Name: Adenocarcinoma - Relevance: LOW - As Found: Unknown - ID: M8886 - Name: Gastrointestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7256 - Name: Digestive System Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M11107 - Name: Liver Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002277 - Term: Carcinoma - ID: D000006528 - Term: Carcinoma, Hepatocellular ### Intervention Browse Module - Browse Branches - Abbrev: Hemat - Name: Hematinics - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M11110 - Name: Liver Extracts - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05982379 **Brief Title:** Motivation Program for Children With Cancer **Official Title:** Investigation Into the Impact of Technology-Based Motivation Program Applied to the Children Followed With Cancer Diagnosis and Their Families: A Randomized Controlled Study #### Organization Study ID Info **ID:** ZSengul-experimental study #### Organization **Class:** OTHER **Full Name:** Kırıkkale University ### Status Module #### Completion Date **Date:** 2020-08-28 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-08-08 **Type:** ACTUAL **Last Update Submit Date:** 2023-08-01 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-06-01 **Type:** ACTUAL #### Start Date **Date:** 2019-09-02 **Type:** ACTUAL **Status Verified Date:** 2023-08 #### Study First Post Date **Date:** 2023-08-08 **Type:** ACTUAL **Study First Submit Date:** 2023-07-25 **Study First Submit QC Date:** 2023-08-01 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Zeynep Kisecik Şengül #### Responsible Party **Investigator Affiliation:** Kırıkkale University **Investigator Full Name:** Zeynep Kisecik Şengül **Investigator Title:** Assistant Professor **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Purpose: To determine the effectiveness of the technology-based motivation program implemented with children with cancer diagnosis and their primary caregivers. Methods: This randomized controlled trial was completed with 31 children and 31 primary caregivers between the ages of 9 and18, who were being treated for cancer. A 10-week "Technology Based Motivation Program" was given to the intervention group. "State/ Trait Anxiety Inventory for Children", "Paediatric Quality of Life Inventory", "Paediatric Cancer Coping Scale", "State and Trait Anxiety Inventory," and "Process Evaluation Forms" were administered. Chi-square test, Fisher-Exact test, Independent Sample-t test, Man-Whitney U test, Wilcoxon test, Pearson and Spearman correlation coefficients were used for data analysis. **Detailed Description:** Design The study was conducted in two hospitals at Leukemia Polyclinic, Paediatric Haematology and Oncology Polyclinic, and Paediatric Haematology-Oncology Service from September 2019 to August 2020. Setting and Samples In determining the research population, hospital registry information was used, and 119 children followed up with a diagnosis of cancer formed the research population. The sample of the research consisted of 44 children (leukaemia n = 33, solid tumour n = 11) and their primary caregivers meeting the inclusion criteria. Randomization and blinding A stratified randomization method was used in the research. 44 children (leukaemia and solid tumour) who met the inclusion criteria were grouped according to gender (male and female) and age (9-13 years-14-18 years) and eight layers were obtained. Among the identified strata, intervention and control groups were randomized by an independent statistician using the website www.randomizer.org, 22 children were assigned to the intervention, and 22 to the control group. Five children from the intervention group and five children from the control group (unreachable (n=3), ex (n=1), refusal (n=4), Hematopoietic stem cell transplantation (HSCT) performed (n=1), intensive care unit admission (n=1)) could not be included in the research for various reasons and 34 children were included in the research. The purpose and content of the research were explained without informing the children and their primary caregivers that they were in the intervention or control group. At the end of the research, the power analysis performed by using the mean scores of State Anxiety Inventory for Children with G \* Power 3.0.10 program yielded a total of at least 28 samples that were found to be sufficient with 90% power and 5% margin of error (n1 = 14; n2 = 14). Theoretical underpinnings The use of models is important in establishing the theoretical framework of research. In this research, the Transactional Stress and Coping Model of Lazarus and Folkman, which is the most widely used model in studies examining the coping and anxiety levels of children with cancer diagnosis, was used. The practices in the motivation program were created for the coping strategies of "seeking social support, self-control, positive evaluation and accepting responsibility" in the model. Interventions The research consists of four stages. (1) Constructing training modules, (2) Creating a website, (3) Conducting preliminary intervention, and (4) Implementing the training modules with the children and primary caregivers in the intervention group. Constructing training modules Four training modules for children and three training modules for primary caregivers were created by the researchers in line with the literature Content was added to the website after it was approved by 6 experts. Creating the website The website was designed by a professional web design firm. The website can be accessed by computer, tablet and phone. There is an administrator and user panel on the website. Children and primary caregivers logged on the website with different passwords through the user panel and accessed the modules specially prepared for them. Conducting preliminary intervention The preliminary intervention of the technology-based program was conducted with five children aged 9-18 years who were followed up with a diagnosis of leukaemia, and with their primary caregivers. As the child and primary caregivers found the program applicable as a result of the preliminary intervention, no change was made and it was included in the sample. Using the training modules with children and primary caregivers in the intervention group The website and four training modules were used with the children in the intervention group. Nine coaching interviews with each child (once a week) were conducted over WhatsApp. The children were encouraged to do progressive relaxation and breathing exercises at least twice a week, and eight therapeutic stories (once a week) were sent to the child's phone in the form of animation. Three training modules were used with the primary caregivers, and coaching interviews were conducted on WhatsApp twice during the program. Primary caregivers practiced progressive relaxation and breathing exercises at least twice a week. In addition, eight motivation messages were sent to the child and primary caregiver, and 24/7 consultancy was provided. Control Group The routine practices of the children in the control group and their primary caregivers at the hospital continued. In addition, forms other than process evaluation were given to the child and their primary caregivers at the beginning and end of the program. Data gathering After the children and primary caregivers who participated in the research were informed about the research in an empty room or in the room where they were treated during the polyclinic controls, data collection forms were given, the children and primary caregivers in the intervention group were subscribed to the website, and the exercises were explained in practice. It took approximately 60 minutes for the participants to fill out the forms and learn about the program. At the end of the research, the data collection forms (excluding the descriptive data form) were collected through WhatsApp messages. Data collection tools Child and Parent Descriptive Characteristics Form, Spielberger State-Trait Anxiety Inventory, State/ Trait Anxiety Inventory for Children, Paediatric Cancer Coping Scale, Paediatric Quality of Life Inventory, and Process Evaluation Forms were used to collect the data. Ethical considerations Permission for the research was obtained from the University Ethics Committee and Hospitals. Informed written consent was obtained from the children and their primary caregivers, who agreed to participate in the research, after written and verbal explanations were given to the patients to participate in the research. At the end of the study, all interventions were also performed with the control group. ### Conditions Module **Conditions:** - Childhood Cancer **Keywords:** - Cancer - Child - Motivation - Program - Parent - Psychosocial - Technology ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** A randomized controlled trial ##### Masking Info **Masking:** SINGLE **Masking Description:** Among the identified strata, intervention and control groups were randomized by an independent statistician using the website www.randomizer.org. The data of the study were entered by a person other than the researcher. Statistical analysis was performed by a person other than the researcher. **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** OTHER #### Enrollment Info **Count:** 31 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** This study was carried out with two groups. Technology Based Motivation Program were used for the intervention group. **Intervention Names:** - Other: Technology Based Motivation Program **Label:** Intervention **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** No application was made to the control group, standard procedure was followed. **Label:** Control **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Intervention **Description:** A 10-week "Technology Based Motivation Program" was given to the intervention group. The website and four training modules were used with the children in the intervention group. Nine coaching interviews with each child (once a week) were conducted over WhatsApp. The children were encouraged to do progressive relaxation and breathing exercises at least twice a week, and eight therapeutic stories (once a week) were sent to the child's phone in the form of animation. Three training modules were used with the primary caregivers, and coaching interviews were conducted on WhatsApp twice during the program. Primary caregivers practiced progressive relaxation and breathing exercises at least twice a week. In addition, eight motivation messages were sent to the child and primary caregiver, and 24/7 consultancy was provided. **Name:** Technology Based Motivation Program **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** State/ Trait Anxiety Inventory for Children: 20 items for the State Anxiety, 20 items for the Trait Anxiety. The scores on the scale range from 20 to 60, and a high score indicates high anxiety. **Measure:** Anxiety levels of the children **Time Frame:** baseline (Initial interview) and through study completion (10th week) **Description:** Pediatric Quality of Life Inventory: The scale consists of 23 items and is scored from 0 to 100. The higher the score, the higher the quality of life **Measure:** Children's quality of life scores **Time Frame:** baseline (Initial interview) and through study completion (10th week) **Description:** Pediatric Cancer Coping Scale:The scale comprises 33 items related to cognitive coping, problem oriented coping, and defensive coping.The score obtained from the scale ranges from 0 to 99. Higher scores on the scale indicate a high level of coping strategies. **Measure:** Coping scores of the children **Time Frame:** baseline (Initial interview) and through study completion (10th week) **Description:** Spielberger State/ Trait Anxiety Inventory: 20 items of the 40-item scale are used to determine state anxiety and 20 items are used to determine trait anxiety.The scores on the scale range from 20 to 80, and the higher the score, the higher the level of anxiety. **Measure:** Anxiety levels of the primary caregivers **Time Frame:** baseline (Initial interview) and through study completion (10th week) #### Secondary Outcomes **Description:** Process evaluation forms:Six forms were prepared by the researchers. It was used to follow the practices and interviews of children and primary caregivers. 1. Website training follow-up form, 2. Mobile video interview follow-up form, 3. Counseling follow-up form, 4. Children's follow-up form for interventions, 5. Primary caregivers follow-up form for interventions, 6. Satisfaction and suggestion form **Measure:** Application tracking forms **Time Frame:** Every week during the intervention ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: For children: * Those having received cancer treatment (leukaemia, lymphomas and solid tumours), * Those aged 9-18 years, * Those who speak Turkish, * Those who have internet access on their smart phone or computer, * Self and primary caregiver having agreed to participate in the study, and * Those who can continue the research for 9 weeks (Trying progressive relaxation exercises, breathing exercises and imagination at least twice a week). For primary caregivers: * Those who speak Turkish, * Those who are literate, * Those who have internet access on their smart phones and computers, * Those who agree to participate in the research, and * Those who can continue the research for 9 weeks (Trying progressive relaxation exercises at least twice a week). Exclusion Criteria: * Those who do not agree to participate in the research, * Those who have experienced significant life events in the last 6 months (migration, death, divorce etc.), * The presence of another important disease in the family that can increase stress and prevent coping, and * Those who do not have internet access. **Healthy Volunteers:** True **Maximum Age:** 18 Years **Minimum Age:** 9 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Kırıkkale **Country:** Turkey **Facility:** Zeynep Kisecik Şengül #### Overall Officials **Official 1:** **Affiliation:** Kırıkkale University **Name:** Zeynep Kisecik Şengül, RN, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Description:** The confidentiality of the data used in the study will be protected. **IPD Sharing:** NO ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01726179 **Brief Title:** Efficacy of Proximal Caries Infiltration **Official Title:** Efficacy of Resin Infiltration of Proximal Caries Lesions in Primary Molars: Randomized Clinical Trial #### Organization Study ID Info **ID:** ECIPRJ13 #### Organization **Class:** INDUSTRY **Full Name:** DMG Dental Material Gesellschaft mbH ### Status Module #### Completion Date **Date:** 2018-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-04-26 **Type:** ACTUAL **Last Update Submit Date:** 2022-04-19 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2017-12 **Type:** ACTUAL #### Start Date **Date:** 2013-12 **Type:** ACTUAL **Status Verified Date:** 2022-04 #### Study First Post Date **Date:** 2012-11-14 **Type:** ESTIMATED **Study First Submit Date:** 2012-11-01 **Study First Submit QC Date:** 2012-11-09 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Universidade Federal do Rio de Janeiro **Class:** OTHER_GOV **Name:** Rio de Janeiro State Research Supporting Foundation (FAPERJ) #### Lead Sponsor **Class:** INDUSTRY **Name:** DMG Dental Material Gesellschaft mbH #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The goal of the project is to investigate the clinical efficacy of management of early caries lesions by resin infiltration and to further scrutinize the patient stress experiences. **Detailed Description:** The aim of this study is to test the clinical efficacy of caries infiltration to arrest proximal caries lesions in primary molars. It was hypothesized that infiltrated lesions (test lesions) will progress significantly less than non infiltrated lesions (control lesions). Additionally, it was hypothesized that dental anxiety and stress related to caries infiltration will be significantly less than dental anxiety related to a conventional restorative procedure. The study will be a controlled clinical trial with a split mouth design and blind evaluation of the outcome (caries progression). Caries progression will be evaluated annually, 12, 24, and 36 months after treatment. ### Conditions Module **Conditions:** - Dental Health - Caries **Keywords:** - caries infiltration - caries arrest - dental anxiety - dental stress - resin infiltration ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 50 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** This study is a split mouth design. One tooth with a proximal caries lesion is randomized into this arm and another tooth to the arm "Control". Teeth in this arm are treated by the resin infiltration technique using Icon (DMG, Germany) according to manufactures´ instructions. In addition patients and their guardians are instructed to floss once a day and to brush with fluoridated toothpaste twice a day. Digital bitewing radiographs will be taken at baseline and repeated after 12 months. Additionally caries risk will be evaluated. **Intervention Names:** - Device: Resin infiltration **Label:** Resin infiltration **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** This study is a split mouth design. One tooth with a proximal caries lesion is randomized into this arm and another tooth to the arm "Resin infiltration". Teeth randomized into this arm do not recieve any special treatment except general oral nonivasiv treatment (flossing and brushing). Patients and their guardians are instructed to floss once a day and to brush with fluoridated toothpaste twice a day. Digital bitewing radiographs will be taken at baseline and repeated after 12 months. Additionally caries risk will be evaluated. **Intervention Names:** - Device: Control **Label:** Control **Type:** OTHER ### Interventions #### Intervention 1 **Arm Group Labels:** - Resin infiltration **Description:** Proximal caries lesions that are selected for this intervention will be treated with the resin infiltrant Icon according to the manufacturer's instructions. In short, local anesthesia of the gingival papila, application of rubber dam, etching of the lesion surface for 120 s using 15%HCl gel, washing the lesion with water spray for 30 s, drying the lesion with 100% ethanol for 30 s and subsequent air blowing, application of the infiltrant for 180 s using an applicator provided with the kit, removing excess material from the lesion surface by air blowing and flossing, light curing of the infiltrant for 40 s, repeated application for 60 s, light curing for 40 s, polishing, and removal of the rubber dam. **Name:** Resin infiltration **Other Names:** - Icon (DMG, Germany), Approximal resin infiltration kit **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - Control **Description:** Proximal caries lesions do not recieve a placebo treatment, but simply left untreated. **Name:** Control **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Radiographic evaluation of infiltrated and control caries lesions to evaluate caries progression. This is done by pairwise comparison of digital bitewing radiographs by one calibrated, blinded examiner. **Measure:** Caries progression rate **Time Frame:** 1 year #### Secondary Outcomes **Description:** To evaluate dental anxiety and stress reactions in relation to the caries infiltration procedure in comparison with a dental examination appointment and conventional resin restoration appointment. A facial image scale with five faces ranging from very happy (1) to very unhappy (5) will be used. **Measure:** Stress reaction during treatment **Time Frame:** Immediate **Description:** Caries risk is asses based on caries index (Nyvad criteria), proximal plaque index (0 = no visible plaque; 1 = visible plaque) and gingival bleeding index (0 = no bleeding after flossing; 1 = bleeding after flossing), dietary habits, and exposition to fluorides based on the Cariogram model. **Measure:** Caries risk **Time Frame:** Baseline, 6 months, 1 year, 2 years, 3 years **Description:** Radiographic evaluation of infiltrated and control caries lesions to evaluate caries progression in long term. This is done by pairwise comparison of digital bitewing radiographs by one calibrated, blinded examiner. **Measure:** Caries progression rate **Time Frame:** 2 years, 3 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * children with one tooth surface with active caries lesions * two primary molars with proximal lesion detected on the x-ray (scores 2 (E2) or 3 (D1) in Mejàre et al. scoring system with less depth * asigned informed consent. Exclusion Criteria: * children who do not cooperate during dental appointments * primary molars supposed to exfoliate in less than two years * lesions showing obvious cavitation or clear sings of inactivity **Maximum Age:** 9 Years **Minimum Age:** 5 Years **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Locations **Location 1:** **City:** Rio de Janeiro **Country:** Brazil **Facility:** Universidade do Estado do Rio de Janeiro, Faculdade de Odontologia. **Zip:** 20551-031 #### Overall Officials **Official 1:** **Affiliation:** Universidade do Estado do Rio de Janeiro, Faculdade de Odontologia **Name:** Vera M Soviero, Prof., Dr. **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Ammari MM, Jorge RC, Souza IPR, Soviero VM. Efficacy of resin infiltration of proximal caries in primary molars: 1-year follow-up of a split-mouth randomized controlled clinical trial. Clin Oral Investig. 2018 Apr;22(3):1355-1362. doi: 10.1007/s00784-017-2227-7. Epub 2017 Oct 8. **PMID:** 28990122 **Citation:** Jorge RC, Ammari MM, Soviero VM, Souza IPR. Randomized controlled clinical trial of resin infiltration in primary molars: 2 years follow-up. J Dent. 2019 Nov;90:103184. doi: 10.1016/j.jdent.2019.103184. Epub 2019 Aug 26. **PMID:** 31465818 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions - Abbrev: BC14 - Name: Heart and Blood Diseases ### Condition Browse Module - Browse Leaves - ID: M4324 - Name: Anxiety Disorders - Relevance: LOW - As Found: Unknown - ID: M9411 - Name: Heart Arrest - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Browse Branches - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infe - Name: Anti-Infective Agents ### Intervention Browse Module - Browse Leaves - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown - ID: M3777 - Name: Ethanol - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03592979 **Brief Title:** Acute Exposure of Simulated Hypoxia on Heart Rate and Ventilation During Exercise **Official Title:** Acute Exposure to Hypoxia in Precapillary Pulmonary Hypertension: Physiological and Clinical Effects at Rest and During Exercise #### Organization Study ID Info **ID:** 2018-00455_A7 #### Organization **Class:** OTHER **Full Name:** University of Zurich ### Status Module #### Completion Date **Date:** 2019-04-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-01-07 **Type:** ACTUAL **Last Update Submit Date:** 2020-01-06 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-04-01 **Type:** ACTUAL #### Start Date **Date:** 2018-07-01 **Type:** ACTUAL **Status Verified Date:** 2020-01 #### Study First Post Date **Date:** 2018-07-19 **Type:** ACTUAL **Study First Submit Date:** 2018-07-09 **Study First Submit QC Date:** 2018-07-18 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Zurich #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Randomized crossover trial in patients with Pulmonary Hypertension (PAH, CTEPH) to assess the acute response to simulated altitude (FIO2: 15.1% = equivalent to 2500m above sea level) on heart rate and Ventilation changes under exercise. **Detailed Description:** Low altitude baseline measurements will be performed in Zurich (469m asl) including Echocardiography, right heart catheterization, six-minute walk test (6MWT), pulmonary function test, clinical assessment and blood gas analysis at rest and under exercise. Randomly assigned to the order of testing, the participants will be tested under simulated altitude (FiO2: 15.1% with the "AMC Altitrainer") and shamed altitude with the same device. During the exposure to simulated altitude (FiO2: 15.1%) and shamed altitude of 1 hour each, the participants' heart rate and Ventilation will be compared under exercise. ### Conditions Module **Conditions:** - Pulmonary Hypertension **Keywords:** - Simulated altitude - exercise - ventilation - heart rate ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER **Intervention Model Description:** Including a baseline assessment and assessments under simulated altitude and normoxia ##### Masking Info **Masking:** SINGLE **Masking Description:** The allocated gas mixture will not be disclosed to the patient since he will breath trough a facemask during both interventions. **Who Masked:** - PARTICIPANT **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 28 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The participants will be consecutively exposed to shamed hypoxia (FiO2: 20.9%) equivalent to sea level and to simulated altitude (FiO2: 15.1%) equivalent to 2500m above sea level administered by an altitude simulator ("Altitrainer, SMTEC") with a facemask. **Intervention Names:** - Device: Simulated Altitude: (FiO2: 15.1) - Device: Shamed Hypoxia (FiO2: 20.9) **Label:** Order A **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** The participants will be consecutively exposed to hypoxia (FiO2: 15,1%) equivalent to 2500m above sea level and to shamed hypoxia (FiO2: 20.9%) equivalent to sea level administered by an altitude simulated ("Altitrainer, SMTEC") with a facemask. **Intervention Names:** - Device: Simulated Altitude: (FiO2: 15.1) - Device: Shamed Hypoxia (FiO2: 20.9) **Label:** Order B **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Order A - Order B **Description:** Inhalation of deoxygenated air through an altitude simulator ("Altitrainer") for approximately 1 hour given by a facemask. **Name:** Simulated Altitude: (FiO2: 15.1) **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - Order A - Order B **Description:** Inhalation of unmodified air through an altitude simulator ("Altitrainer") for approximately 1 hour given by a facemask. **Name:** Shamed Hypoxia (FiO2: 20.9) **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Change in heart rate under exercise during the exposure to simulated altitude (FiO2: 15,1%) and normoxia. **Measure:** heart rate under exercise **Time Frame:** 1 hour #### Secondary Outcomes **Description:** Change in Ventilation under exercise during the exposure to simulated altitude **Measure:** Ventilation under exercise **Time Frame:** 1 hour ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Informed consent * PH diagnosed according to internation Guidelines: mean pulmonary artery pressure (mPAP) ≥ 25 mmHg along with a pulmonary artery wedge pressure (PAWP) ≤15 mmHg during right heart catheterization at the time of Initial diagnosis * PH class 1 (PAH) or 4 (CTEPH) * Stable condition, on the same medication for \> 4 weeks * Patient live permanently at an altitude \< 1000m asl. Exclusion Criteria: * Resting partial oxygen pressure (PaO2) ≤7.3 kilopascal (kPA) corresponding to the requirement of long-term oxygen therapy \> 16hour daily (nocturnal oxygen therapy alone is allowed) * Severe daytime hypercapnia (pCO2 \> 6.5 kPa) * Susceptibility to high altitude related diseases (AMS, high-altitude pulmonary edema (HAPE), etc.) based on previous experienced discomfort at altitudes. * Exposure to an altitude \>1500m for ≥3 nights during the last 4 weeks before the study participation * Residence \> 1000m above sea level * Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders, neurological or orthopedic problems with Walking disability * Women who are pregnant or breast feeding **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Zurich **Country:** Switzerland **Facility:** Respiratory Clinic, University Hospital of Zurich **Zip:** 8091 #### Overall Officials **Official 1:** **Affiliation:** University of Zurich **Name:** Silvia Ulrich, Prof. **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000012818 - Term: Signs and Symptoms, Respiratory ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10024 - Name: Hypertension - Relevance: HIGH - As Found: Hypertension - ID: M4185 - Name: Hypoxia - Relevance: HIGH - As Found: Hypoxia - ID: M10027 - Name: Hypertension, Pulmonary - Relevance: HIGH - As Found: Pulmonary Hypertension - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M15623 - Name: Signs and Symptoms, Respiratory - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006976 - Term: Hypertension, Pulmonary - ID: D000006973 - Term: Hypertension - ID: D000000860 - Term: Hypoxia ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01031979 **Brief Title:** Prolonged Exposure for Post Traumatic Stress Disorder (PTSD) With/Without Yohimbine **Official Title:** Psychophysiology of Prolonged Exposure for PTSD With/Without Yohimbine #### Organization Study ID Info **ID:** CDA-2-013-09F #### Organization **Class:** FED **Full Name:** VA Office of Research and Development ### Status Module #### Completion Date **Date:** 2015-07-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-02-07 **Type:** ACTUAL **Last Update Submit Date:** 2018-01-10 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2015-04-01 **Type:** ACTUAL #### Results First Post Date **Date:** 2017-02-24 **Type:** ACTUAL **Results First Submit Date:** 2016-11-03 **Results First Submit QC Date:** 2017-01-03 #### Start Date **Date:** 2010-12-01 **Type:** ACTUAL **Status Verified Date:** 2018-01 #### Study First Post Date **Date:** 2009-12-15 **Type:** ESTIMATED **Study First Submit Date:** 2009-12-04 **Study First Submit QC Date:** 2009-12-14 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** FED **Name:** VA Office of Research and Development #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Is US Export:** True **Oversight Has DMC:** False ### Description Module **Brief Summary:** The proposed study has three distinct but related research objectives. The first goal is to measure physiological correlates of successful treatment with Prolonged Exposure (PE) therapy for posttraumatic stress disorder (PTSD) in veterans of the Iraq and Afghanistan wars. Individuals with PTSD often experience elevated heart rates and other objectively measurable signs of anxiety when confronted with safe situations that remind them of past dangerous situations. We will measure physiological responses and compare the outcomes to patient's self reported subjective accounts of symptom improvement on traditional measures of PTSD. Developing a way to measure objective gains in symptoms improvement may help researchers who are studying ways to improve PTSD treatment. The second goal of the study is to investigate if yohimbine, a drug found to promote a specific type of learning, will improve treatment outcomes for veterans in PTSD treatment. The third goal is to investigate if ability to get used to loud startling audio tones correlates to baseline PTSD pathology and treatment outcomes for PE. This goal represents an important step forward in understanding characteristics of heritable traits that are related PTSD. It is significant because such research may one day lead to the development of individual responder policies that will assist patients by individualizing treatment plans based on personal characteristics. **Detailed Description:** The proposed study has three distinct but related research objectives. The first research goal is to measure psychophysiological correlates of treatment gains associated with Prolonged Exposure (PE) therapy for PTSD in veterans of Operations Enduring Freedom and Iraqi Freedom (OEF/OIF). Specifically, heart rate, heart rate variability, skin conductance, and facial electromyography, will be recorded before and after treatment during a three minute anxiety probe specific to the patient's index trauma. These measures will be compared to patient's self reported subjective accounts of symptom improvement on traditional measures of PTSD pathology (Subjective Units of Distress (SUDs), PTSD Checklist-Military Version (PCL), Clinician Administered PTSD Scale (CAPS), and the Beck Depression Inventory (BDI)). This goal is significant for veterans because currently no widely used objective criteria exist to measure treatment progress in PTSD. While the preponderance of existing evidence suggests that no one objective psychophysiological measurement will be a valid correlate for all individuals, even establishing a measurement paradigm that can show mean differences between groups will provide researchers with an objective tool to measure outcomes on clinical trials. The second goal of the study is to investigate if the administration of yohimbine, a drug found to promote the extinction of conditioned fear in animal models, and more recently, in humans with claustrophobia, improves the facilitation of fear extinction in PE. Yohimbine is a safe drug that is already extensively used in human populations. Specifically, this goal will be investigated using a double blind placebo controlled randomized trial design. The hypothesis is that one 21mg oral dose of yohimbine given concurrently with a 40 minute imaginal exposure exercise in PE will lead to a greater reduction in cue-induced anxiety during the following weekly PE session than placebo. This goal is significant because current projections of PTSD in OEF/OIF veterans indicate that the need for psychological services will likely outpace the supply of such services. Accordingly, assisting treatments to be more efficient will likely translate into more veterans receiving much needed mental health services. The 3rd goal is to investigate if ability to habituate to loud, 95db, audio tones correlates to baseline PTSD pathology and treatment outcomes for PE. This goal represents an important step forward in understanding characteristics of trait habituation, fear extinction, and learning in humans, which are all factors related to the successful treatment of PTSD. It is also significant because such research may lead to the development of individual responder policies that will assist veterans by individualizing treatment plans based on personal characteristics. ### Conditions Module **Conditions:** - Post-Traumatic Stress Disorder **Keywords:** - PTSD - Prolonged Exposure - extinction - habituation - psychophysiology ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 26 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients will take one 21.6 mg. dose of yohimbine one hour before first imaginal exposure in PE. **Intervention Names:** - Drug: Yohimbine **Label:** Yohimbime Group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Patients will take a placebo one hour before first imaginal exposure in PE. **Intervention Names:** - Drug: Placebo **Label:** Placebo Group **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Yohimbime Group **Description:** alpha-2 adrenergic receptor antagonist **Name:** Yohimbine **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo Group **Description:** Placebo **Name:** Placebo **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** The primary outcome was trauma-cued heart rate reactivity a week after the drug visit as measured by the PTSD Brief Reactivity (PBR) task. For each patient, a 3-minute trauma script was constructed containing vivid details of the target trauma and used in tandem with a standard neutral script for baseline measurement. Heart rate reactivity for each time point was the beats per minute (BPM) difference between the neutral and trauma scripts represented as a slope. **Measure:** Trauma-Cued Heart Rate Reactivity **Time Frame:** One week after drug visit #### Secondary Outcomes **Description:** The CAPS is a structured interview for diagnosis of PTSD and is widely considered the gold-standard assessment. The CAPS produces a total score ranging from 0-136, with higher scores indicating more severe PTSD symptom severity. A 15-point decrease is considered clinically significant. **Measure:** Change in Clinician Administered PTSD Scale (CAPS) Score **Time Frame:** 0 Weeks, 15 weeks **Description:** The PCL is a 17-item self-report measure of PTSD symptom severity based on the DSM-IV and has adequate psychometric properties. The PCL produces a score range between 17-85, with higher scores indicating more distress related to PTSD symptoms. A 10-point decrease on the PCL is considered clinically significant. **Measure:** Change in Post Traumatic Stress Disorder Checklist (PCL) Score **Time Frame:** 0 weeks, 15 weeks **Description:** The BDI-II is a 21-item self-report measure that assesses depressive behavioral symptoms. It has demonstrated adequate psychometric validity, and external validity and is used widely as the dependent variable in treatment outcomes research. The BDI-II produces score ranges from 0-63, with higher scores indicating more severe depression symptom severity. A 5-point decrease on the BDI-II is considered clinically significant. **Measure:** Change in Becks Depression Inventory (BDI-II) Score **Time Frame:** 0 weeks, 15 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Subjects must be competent to provide informed consent for research participation. * Subjects must be male veterans and post deployed active duty male personnel of OEF/OIF. * Subjects must be between the ages of 18 and 45. * Subjects must meet DSM-IV diagnostic criteria for PTSD on the CAPS. * For subjects taking SSRI's, subjects must be stabilized on the current prescribed dose for a period of at least 14 days prior to the trial and remain at that dose for the remainder of the study. Subjects who change their SSRI status or dosage during the study will continue to receive services via the study resources but data generated will not be used in analyses. Subjects will be eligible for the study if they are willing to titrate off potentially confounding agents prior to yohimbine administration (for a period of five half-lives), given that such titration is also clinically appropriate and deemed to be in the patient's best interests. Exclusion Criteria: * Subjects with a recent (\< 2 month) history of psychiatric hospitalization or suicide attempt. Recent work with veterans with severe mental illness suggests that a 2-month period of stabilization is sufficient to minimize risk and possible relapse (Frueh, 2005). Subjects with an existing diagnosis of schizophrenia or other Axis I serious mental illnesses (SMI, besides PTSD) will be excluded. SMI will include any severe and persistent mental illness. * Subjects with a current diagnosis of drug dependence, due to potential interactions with study measurements and treatments. Alcohol use disorders will be allowed given that subjects can pass exclusion criterion 12 without withdrawal symptoms. * Subjects with any acute illness or fever. Individuals who otherwise meet study criteria will be rescheduled for evaluation for participation. * Subjects with evidence of or a history of clinically significant hematological, endocrine, cardiovascular, hepatic, pulmonary, renal, gastrointestinal, or neurological disease including diabetes, as these conditions may affect physiological/subjective responses. * Subjects with SCID-diagnosed panic disorder, as yohimbine may precipitate panic attacks. * Subjects with an abnormal ECG. * Subjects with a blood pressure of 140/90 or higher, as yohimbine has been shown to elevate blood pressure. * Subjects taking Beta blockers, alpha-adrenergic agents, Beta-agonist inhalers, opiates or opiate antagonists and any psychotropic medications other than SSRI's because these may affect test response. * Subjects who are unwilling or unable to maintain abstinence for three days prior to yohimbine administration from over-the-counter drugs with sympathomimetic properties, e.g., asthma medications, cold medicines with ephedrine, dietary supplements with ephedrine alkaloids, and illegal drugs, e.g., amphetamines, methamphetamine, cocaine, and MNDA as well as alcohol because these may exacerbate the action of yohimbine. * Subjects taking alpha-adrenergic antagonists, e.g. prazosin for hypertension; and beta-adrenergic antagonists, e.g. propranolol. Because they may attenuate effects of yohimbine. Subjects will be eligible for the study if they are willing to titrate off potentially confounding agents prior to yohimbine administration (for a period of five half-lives), given that such titration is also clinically appropriate and deemed to be in the patient's best interests. * Asthmatic subjects and subjects on medications for hypertension, due to criteria 9 and 10. These inclusion/exclusion criteria will allow the majority of veterans treated in the PCT to be study eligible. Accordingly, the sample will be likely generalizable to the population of interest. **Gender Based:** True **Gender Description:** Males only. **Maximum Age:** 45 Years **Minimum Age:** 18 Years **Sex:** MALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Charleston **Country:** United States **Facility:** Ralph H. Johnson VA Medical Center, Charleston, SC **State:** South Carolina **Zip:** 29401-5799 #### Overall Officials **Official 1:** **Affiliation:** Ralph H. Johnson VA Medical Center, Charleston, SC **Name:** Peter W. Tuerk, PhD MA BA **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Keller SM, Tuerk PW. Evidence-based psychotherapy (EBP) non-initiation among veterans offered an EBP for posttraumatic stress disorder. Psychol Serv. 2016 Feb;13(1):42-8. doi: 10.1037/ser0000064. Epub 2015 Dec 14. **PMID:** 26654474 **Citation:** Yuen EK, Gros DF, Price M, Zeigler S, Tuerk PW, Foa EB, Acierno R. Randomized Controlled Trial of Home-Based Telehealth Versus In-Person Prolonged Exposure for Combat-Related PTSD in Veterans: Preliminary Results. J Clin Psychol. 2015 Jun;71(6):500-12. doi: 10.1002/jclp.22168. Epub 2015 Mar 25. **PMID:** 25809565 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000068099 - Term: Trauma and Stressor Related Disorders - ID: D000001523 - Term: Mental Disorders ### Condition Browse Module - Browse Branches - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions - Abbrev: BC26 - Name: Wounds and Injuries ### Condition Browse Module - Browse Leaves - ID: M16103 - Name: Stress Disorders, Post-Traumatic - Relevance: HIGH - As Found: Post Traumatic Stress Disorder - ID: M24916 - Name: Stress Disorders, Traumatic - Relevance: HIGH - As Found: Traumatic Stress Disorder - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown - ID: M222 - Name: Trauma and Stressor Related Disorders - Relevance: LOW - As Found: Unknown - ID: M4815 - Name: Mental Disorders - Relevance: LOW - As Found: Unknown - ID: M14473 - Name: Psychotic Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000040921 - Term: Stress Disorders, Traumatic - ID: D000013313 - Term: Stress Disorders, Post-Traumatic ### Intervention Browse Module - Ancestors - ID: D000009184 - Term: Mydriatics - ID: D000001337 - Term: Autonomic Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000058669 - Term: Adrenergic alpha-2 Receptor Antagonists - ID: D000000317 - Term: Adrenergic alpha-Antagonists - ID: D000018674 - Term: Adrenergic Antagonists - ID: D000018663 - Term: Adrenergic Agents - ID: D000018377 - Term: Neurotransmitter Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000064804 - Term: Urological Agents ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Urol - Name: Urological Agents - Abbrev: HB - Name: Herbal and Botanical ### Intervention Browse Module - Browse Leaves - ID: M20746 - Name: Adrenergic Agents - Relevance: LOW - As Found: Unknown - ID: M20755 - Name: Adrenergic Antagonists - Relevance: LOW - As Found: Unknown - ID: M29195 - Name: Adrenergic alpha-2 Receptor Antagonists - Relevance: LOW - As Found: Unknown - ID: M17752 - Name: Yohimbine - Relevance: HIGH - As Found: Laparoscopic appendectomy - ID: M12139 - Name: Mydriatics - Relevance: LOW - As Found: Unknown - ID: M3669 - Name: Adrenergic alpha-Antagonists - Relevance: LOW - As Found: Unknown - ID: M20504 - Name: Neurotransmitter Agents - Relevance: LOW - As Found: Unknown - ID: T332 - Name: Yohimbe - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000015016 - Term: Yohimbine ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Yohimbine Group **Description:** Patients will take one 21.6 mg. dose of yohimbine one hour before first imaginal exposure in PE. Yohimbine: alpha-2 adrenergic receptor antagonist **ID:** EG000 **Other Num at Risk:** 12 **Serious Number At Risk:** 12 **Title:** Yohimbine Group **Group ID:** EG001 **Title:** Placebo Group **Description:** Patients will take a placebo one hour before first imaginal exposure in PE. Placebo: Placebo **ID:** EG001 **Other Num at Risk:** 12 **Serious Number At Risk:** 12 **Title:** Placebo Group **Frequency Threshold:** 0 ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 12 **Group ID:** BG001 **Value:** 12 **Group ID:** BG002 **Value:** 24 **Units:** Participants ### Group **ID:** BG000 **Title:** Yohimbine Group **Description:** Patients will take one 21.6 mg. dose of yohimbine one hour before first imaginal exposure in PE. Yohimbine: alpha-2 adrenergic receptor antagonist ### Group **ID:** BG001 **Title:** Placebo Group **Description:** Patients will take a placebo one hour before first imaginal exposure in PE. Placebo: Placebo ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 12 #### Measurement **Group ID:** BG001 **Value:** 12 #### Measurement **Group ID:** BG002 **Value:** 24 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 18 **Upper Limit:** 45 **Value:** 34.6 #### Measurement **Group ID:** BG001 **Lower Limit:** 18 **Upper Limit:** 45 **Value:** 29.9 #### Measurement **Group ID:** BG002 **Lower Limit:** 18 **Upper Limit:** 45 **Value:** 32.4 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 12 #### Measurement **Group ID:** BG001 **Value:** 12 #### Measurement **Group ID:** BG002 **Value:** 24 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 12 #### Measurement **Group ID:** BG001 **Value:** 12 #### Measurement **Group ID:** BG002 **Value:** 24 **Class Title:** United States **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Age, Categorical **Unit of Measure:** Participants ### Measure 2 **Dispersion Type:** FULL_RANGE **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants **Population Description:** 14 participants were randomized to the yohimbine group; however only 12 completed the primary outcome assessment at session 4 and were able to be analyzed. ## Results Section - More Information Module ### Certain Agreement **PI Sponsor Employee:** True ### Point of Contact **Email:** peter.tuerk@va.gov **Organization:** Ralph H. Johnson VAMC **Phone:** (843) 789-6188 **Title:** Dr. Peter Tuerk ## Results Section - Outcome Measures Module ### Outcome Measure 1 #### Analysis **CI Lower Limit:** -2.29 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** -0.59 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** Beta **Parameter Value:** -1.44 **Statistical Comment:** **Statistical Method:** mixed effects/hierarchical linear model **Tested Non-Inferiority:** ### Outcome Measure 2 #### Analysis **CI Lower Limit:** **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** .39 **P-Value Comment:** **Parameter Type:** Slope **Parameter Value:** -0.87 **Statistical Comment:** **Statistical Method:** Hierarchical Linear Modeling **Tested Non-Inferiority:** ### Outcome Measure 3 #### Analysis **CI Lower Limit:** **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** .58 **P-Value Comment:** **Parameter Type:** Slope **Parameter Value:** -0.55 **Statistical Comment:** **Statistical Method:** Hierarchical Linear Modeling **Tested Non-Inferiority:** ### Outcome Measure 4 #### Analysis **CI Lower Limit:** -2.71 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** -0.49 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** .03 **P-Value Comment:** **Parameter Type:** Beta **Parameter Value:** -1.60 **Statistical Comment:** **Statistical Method:** Hierarchical Linear Modeling **Tested Non-Inferiority:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.84 - **Upper Limit:** - **Value:** 71.01 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 3.11 - **Upper Limit:** - **Value:** 75.08 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 24.49 - **Upper Limit:** - **Value:** 67.33 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 13.01 - **Upper Limit:** - **Value:** 65.00 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 14.93 - **Upper Limit:** - **Value:** 25.13 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 10.84 - **Upper Limit:** - **Value:** 20.62 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 12.49 - **Upper Limit:** - **Value:** 64.00 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 14.53 - **Upper Limit:** - **Value:** 60.25 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 6.63 - **Upper Limit:** - **Value:** 27.37 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 8.88 - **Upper Limit:** - **Value:** 28.50 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 13.10 - **Upper Limit:** - **Value:** 30.56 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 7.05 - **Upper Limit:** - **Value:** 22.62 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 5.68 - **Upper Limit:** - **Value:** 7.00 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 7.97 - **Upper Limit:** - **Value:** 7.87 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** The primary outcome was trauma-cued heart rate reactivity a week after the drug visit as measured by the PTSD Brief Reactivity (PBR) task. For each patient, a 3-minute trauma script was constructed containing vivid details of the target trauma and used in tandem with a standard neutral script for baseline measurement. Heart rate reactivity for each time point was the beats per minute (BPM) difference between the neutral and trauma scripts represented as a slope. **Dispersion Type:** Standard Error **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** One week after drug visit **Title:** Trauma-Cued Heart Rate Reactivity **Type:** PRIMARY **Unit of Measure:** beats per minute ##### Group **Description:** Patients will take one 21.6 mg. dose of yohimbine one hour before first imaginal exposure in PE. Yohimbine: alpha-2 adrenergic receptor antagonist **ID:** OG000 **Title:** Yohimbine Group ##### Group **Description:** Patients will take a placebo one hour before first imaginal exposure in PE. Placebo: Placebo **ID:** OG001 **Title:** Placebo Group #### Outcome Measure 2 **Description:** The CAPS is a structured interview for diagnosis of PTSD and is widely considered the gold-standard assessment. The CAPS produces a total score ranging from 0-136, with higher scores indicating more severe PTSD symptom severity. A 15-point decrease is considered clinically significant. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 0 Weeks, 15 weeks **Title:** Change in Clinician Administered PTSD Scale (CAPS) Score **Type:** SECONDARY **Unit of Measure:** units on a scale ##### Group **Description:** Patients will take one 21.6 mg. dose of yohimbine one hour before first imaginal exposure in PE. Yohimbine: alpha-2 adrenergic receptor antagonist **ID:** OG000 **Title:** Yohimbine Group ##### Group **Description:** Patients will take a placebo one hour before first imaginal exposure in PE. Placebo: Placebo **ID:** OG001 **Title:** Placebo Group #### Outcome Measure 3 **Description:** The PCL is a 17-item self-report measure of PTSD symptom severity based on the DSM-IV and has adequate psychometric properties. The PCL produces a score range between 17-85, with higher scores indicating more distress related to PTSD symptoms. A 10-point decrease on the PCL is considered clinically significant. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 0 weeks, 15 weeks **Title:** Change in Post Traumatic Stress Disorder Checklist (PCL) Score **Type:** SECONDARY **Unit of Measure:** units on a scale ##### Group **Description:** Patients will take one 21.6 mg. dose of yohimbine one hour before first imaginal exposure in PE. Yohimbine: alpha-2 adrenergic receptor antagonist **ID:** OG000 **Title:** Yohimbine Group ##### Group **Description:** Patients will take a placebo one hour before first imaginal exposure in PE. Placebo: Placebo **ID:** OG001 **Title:** Placebo Group #### Outcome Measure 4 **Description:** The BDI-II is a 21-item self-report measure that assesses depressive behavioral symptoms. It has demonstrated adequate psychometric validity, and external validity and is used widely as the dependent variable in treatment outcomes research. The BDI-II produces score ranges from 0-63, with higher scores indicating more severe depression symptom severity. A 5-point decrease on the BDI-II is considered clinically significant. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 0 weeks, 15 weeks **Title:** Change in Becks Depression Inventory (BDI-II) Score **Type:** SECONDARY **Unit of Measure:** units on a scale ##### Group **Description:** Patients will take one 21.6 mg. dose of yohimbine one hour before first imaginal exposure in PE. Yohimbine: alpha-2 adrenergic receptor antagonist **ID:** OG000 **Title:** Yohimbine Group ##### Group **Description:** Patients will take a placebo one hour before first imaginal exposure in PE. Placebo: Placebo **ID:** OG001 **Title:** Placebo Group ### Participant Flow Module #### Group **Description:** Patients will take one 21.6 mg. dose of yohimbine one hour before first imaginal exposure in PE. Yohimbine: alpha-2 adrenergic receptor antagonist **ID:** FG000 **Title:** Yohimbime Group #### Group **Description:** Patients will take a placebo one hour before first imaginal exposure in PE. Placebo: Placebo **ID:** FG001 **Title:** Placebo Group #### Period **Title:** Overall Study ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 5 ###### Reason **Group ID:** FG001 **Number of Subjects:** 4 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 14 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 12 ##### Milestone **Type:** Completed Primary Outcome Measure ###### Achievement **Group ID:** FG000 **Number of Subjects:** 12 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 12 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 9 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 8 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 5 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 4 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT04749979 **Brief Title:** Freeze All on Oocyte Maturatin by Gonadotropin Agonist Versus Human Chorionic Gonadotroipin **Official Title:** Freeze All on Oocyte Maturatin by Gonadotropin Agonist Versus Human Chorionic Gonadotroipin in IVF Cycles #### Organization Study ID Info **ID:** Oocyte #### Organization **Class:** OTHER **Full Name:** Aljazeera Hospital ### Status Module #### Completion Date **Date:** 2021-07-01 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** NOT_YET_RECRUITING #### Last Update Post Date **Date:** 2021-02-11 **Type:** ACTUAL **Last Update Submit Date:** 2021-02-10 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2021-06-25 **Type:** ESTIMATED #### Start Date **Date:** 2021-02-05 **Type:** ESTIMATED **Status Verified Date:** 2021-02 #### Study First Post Date **Date:** 2021-02-11 **Type:** ACTUAL **Study First Submit Date:** 2021-01-29 **Study First Submit QC Date:** 2021-02-10 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Cairo University #### Lead Sponsor **Class:** OTHER **Name:** Aljazeera Hospital #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** Estradiol valerate in the form of cycloprogenova™ (white tablet only) was started on day 2 or 3 of cycle with oral dose 4 mg divided on 2 doses in addition to Aspocid 75 mg once daily with folic acid 500 mcg once daily. **Detailed Description:** U/S was done on day 9 or 10 of cycle to assess endometrial thickness, then every other day until endometrial thickness of 8 mm or more is reached and when endometrial thickness reaches 8 mm or more FET was planned. Progesterone in the form of prontogest™ 400 mg vaginal suppository twice dTwo to Four Day 5 Embryos was transferred ### Conditions Module **Conditions:** - Infertility ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 100 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Giving women agonist **Intervention Names:** - Drug: Agonist (decapeptyl ) **Label:** giving agonist ( eg. decapeptyl ) **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Giving women HCG **Intervention Names:** - Drug: HCG ( eg. Choriomon ) **Label:** Giving HCG (eg. choriomon ) **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - giving agonist ( eg. decapeptyl ) **Description:** giving agonist **Name:** Agonist (decapeptyl ) **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Giving HCG (eg. choriomon ) **Description:** giving HCG **Name:** HCG ( eg. Choriomon ) **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** how many good quality embryos **Measure:** the number of good quality embryos **Time Frame:** within 5 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * PCOS patients according to Rotterdam criteria * Female age between 20 and 40 years. * Primary or secondary infertility. * Body mass index (BMI) between 18 and 40 kg/m2. Exclusion Criteria: * Ovarian endometriosis. * Ovarian cysts before induction. * Known uncontrolled endocrinal abnormalities (like hypo or hyperthyroidism) **Gender Based:** True **Gender Description:** females candidate for ICSI **Healthy Volunteers:** True **Maximum Age:** 42 Years **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** mahmoudalalfy@ymail.com **Name:** Mahmoud Alalfy **Phone:** 01002611058 **Role:** CONTACT #### Locations **Location 1:** **City:** Giza **Contacts:** ***Contact 1:*** - **Email:** mahmoudalalfy@ymail.com - **Name:** Mahmoud Alalfy, PhD - **Phone:** +201002611058 - **Phone Ext:** +2 - **Role:** CONTACT **Country:** Egypt **Facility:** Aljazeera( Al Gazeera) hospital #### Overall Officials **Official 1:** **Affiliation:** Algezeera hospitaland National Research Centre ,Egypt **Name:** Mahmoud Alalfy, PhD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000091662 - Term: Genital Diseases - ID: D000091642 - Term: Urogenital Diseases ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M10290 - Name: Infertility - Relevance: HIGH - As Found: Infertility - ID: M2876 - Name: Genital Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007246 - Term: Infertility ### Intervention Browse Module - Ancestors - ID: D000008186 - Term: Luteolytic Agents - ID: D000003271 - Term: Contraceptive Agents, Female - ID: D000003270 - Term: Contraceptive Agents - ID: D000012102 - Term: Reproductive Control Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000080066 - Term: Contraceptive Agents, Hormonal - ID: D000018931 - Term: Antineoplastic Agents, Hormonal - ID: D000000970 - Term: Antineoplastic Agents ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: Repr - Name: Reproductive Control Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M19618 - Name: Triptorelin Pamoate - Relevance: HIGH - As Found: Passed - ID: M6494 - Name: Contraceptive Agents - Relevance: LOW - As Found: Unknown - ID: M6495 - Name: Contraceptive Agents, Female - Relevance: LOW - As Found: Unknown - ID: M2116 - Name: Contraceptive Agents, Hormonal - Relevance: LOW - As Found: Unknown - ID: M20966 - Name: Antineoplastic Agents, Hormonal - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000017329 - Term: Triptorelin Pamoate ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02235779 **Brief Title:** Diagnostic Yield of Transbronchial Cryobiopsy in Diagnosis of Interstitial Lung Diseases **Official Title:** DIagnostic Yield of Transbronchial Cryobiopsies in Subjects With Interstitial Lung Disease #### Organization Study ID Info **ID:** CryobiopsieTBB #### Organization **Class:** OTHER **Full Name:** Laval University ### Status Module #### Completion Date **Date:** 2021-12 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2021-03-08 **Type:** ACTUAL **Last Update Submit Date:** 2021-03-04 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2021-12 **Type:** ESTIMATED #### Start Date **Date:** 2014-08 **Status Verified Date:** 2021-03 #### Study First Post Date **Date:** 2014-09-10 **Type:** ESTIMATED **Study First Submit Date:** 2014-08-26 **Study First Submit QC Date:** 2014-09-08 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Laval University #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Evaluation of diagnostic yield, feasibility and safety of transbronchial lung cryobiopsies done via bronchoscopy in the investigation of interstitial lung disease in comparison with videothoracoscopy-assisted surgical lung biopsy. **Detailed Description:** See above ### Conditions Module **Conditions:** - Interstitial Lung Disease **Keywords:** - ILD - Pulmonary fibrosis - Cryobiopsy - Transbronchial ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** DIAGNOSTIC #### Enrollment Info **Count:** 50 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Other: Transbronchial lung cryobiopsy **Label:** Cryobiopsy **Type:** OTHER ### Interventions #### Intervention 1 **Arm Group Labels:** - Cryobiopsy **Name:** Transbronchial lung cryobiopsy **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Number of cryobiopsy specimens from which a specific diagnosis of interstitial lung disease can be ascertained, as assessed by a two different pathologists blinded to the known final diagnosis. **Measure:** Overall diagnostic yield of cryobiopsy specimens in interstitial lung disease **Time Frame:** After biopsy (up to 24 months) #### Secondary Outcomes **Description:** Assessment of complications related to cryoTBB: * pneumothorax * hemorrage * other complications **Measure:** Complication rate of transbronchial cryobiopsy procedure **Time Frame:** Per-procedure and up to 24 hours after **Description:** Diagnostic concordance between cryobiopsy specimen analysis and corresponding surgical lung biopsy done in same subjects (same lung/lobe) **Measure:** Number of diagnostic specimens of cryoTBB in comparison with surgical lung biopsy specimen **Time Frame:** After biopsy (up to 24 months) **Description:** Interobserver agreement rate between two different pathologists blinded to subject history/radiological record and surgical lung biopsy specimen. **Measure:** Inter-observer agreement for cryobiopsy specimens **Time Frame:** After biopsy (up to 24 months) **Description:** Size of TBB cryobiopsy specimen measured by two different pathologists responsible for its interpretation. **Measure:** Size of cryobiopsy specimen **Time Frame:** After biopsy (up to 24months) **Description:** Pathological quality score of TBB cryobiopsy specimens (artefacts, size, etc.) as assessed by two different pathologists responsible for their interpretation. **Measure:** Quality of cyrobiopsy specimens **Time Frame:** After biopsy (up to 24 months) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Volunteer subjects with interstitial lung disease in whom a surgical lung biopsy is planned for diagnostic investigation. Exclusion Criteria: * Aged less than 18 y.o. * Pregnancy * Known coagulatoin disorder or blood dyscrasia * Aaking antiplatelet agents other than aspirin or therapeutic anticoagulants drugs that cannot be safely discontinued to undergo lung biopsy * Arterial oxgen pressure less than 60 mmHg on more than 2 liters per minute oxygen * Known pulmonary hypertension defined as systolic PAP above 40 mmHg on echocardiography * Unable to provide informed consent **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** antoine.delage@criucpq.ulaval.ca **Name:** Antoine Delage, MDCM **Phone:** 418-656-4747 **Role:** CONTACT #### Locations **Location 1:** **City:** Québec **Contacts:** ***Contact 1:*** - **Email:** antoine.delage@criucpq.ulaval.ca - **Name:** Antoine Delage, MDCM - **Phone:** 418-656-4747 - **Role:** CONTACT ***Contact 2:*** - **Name:** Antoine Delage, MDCM - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 3:*** - **Name:** Simon Martel, MD - **Role:** SUB_INVESTIGATOR ***Contact 4:*** - **Name:** Steeve Provencher, MD - **Role:** SUB_INVESTIGATOR ***Contact 5:*** - **Name:** Genevieve Dion, MD - **Role:** SUB_INVESTIGATOR ***Contact 6:*** - **Name:** Philippe Joubert, MD - **Role:** SUB_INVESTIGATOR ***Contact 7:*** - **Name:** Massimo Conti, MD - **Role:** SUB_INVESTIGATOR ***Contact 8:*** - **Name:** Paula Ugalde, MD - **Role:** SUB_INVESTIGATOR **Country:** Canada **Facility:** Institut universitaire de cardiologie et de pneumologie de Québec **Status:** RECRUITING **Zip:** G1V 4G5 #### Overall Officials **Official 1:** **Affiliation:** Institut universitaire de cardiologie et de pneumologie de Québec, University Laval **Name:** Antoine Delage, MDCM **Role:** STUDY_CHAIR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012140 - Term: Respiratory Tract Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases ### Condition Browse Module - Browse Leaves - ID: M8485 - Name: Fibrosis - Relevance: LOW - As Found: Unknown - ID: M11168 - Name: Lung Diseases - Relevance: HIGH - As Found: Lung Disease - ID: M14512 - Name: Pulmonary Fibrosis - Relevance: LOW - As Found: Unknown - ID: M19813 - Name: Lung Diseases, Interstitial - Relevance: HIGH - As Found: Interstitial Lung Disease - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000008171 - Term: Lung Diseases - ID: D000017563 - Term: Lung Diseases, Interstitial ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04254679 **Brief Title:** Pilot Trial: Postoperative Opioid-free Analgesia **Official Title:** Opioid-free Analgesia After Outpatient General Surgery: A Pilot Randomized Controlled Trial #### Organization Study ID Info **ID:** MUHC REB 2020-5965 #### Organization **Class:** OTHER **Full Name:** McGill University Health Centre/Research Institute of the McGill University Health Centre ### Status Module #### Completion Date **Date:** 2020-12-03 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-02-24 **Type:** ACTUAL **Last Update Submit Date:** 2021-02-22 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-09-20 **Type:** ACTUAL #### Start Date **Date:** 2020-01-29 **Type:** ACTUAL **Status Verified Date:** 2021-02 #### Study First Post Date **Date:** 2020-02-05 **Type:** ACTUAL **Study First Submit Date:** 2020-01-30 **Study First Submit QC Date:** 2020-01-31 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Society of American Gastrointestinal and Endoscopic Surgeons #### Lead Sponsor **Class:** OTHER **Name:** McGill University Health Centre/Research Institute of the McGill University Health Centre #### Responsible Party **Investigator Affiliation:** McGill University Health Centre/Research Institute of the McGill University Health Centre **Investigator Full Name:** Julio F Fiore Jr **Investigator Title:** Assitant Professor (McGill) **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Is US Export:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** North America is facing an opioid epidemic fueled by surgeons, who are the second largest subgroup of physicians involved in opioid prescribing. Surgery often serves as the initial event for opioid-naïve patients to obtain a prescription for opioids and spiral into misuse and addiction. From the perspective of perioperative care clinicians, the answer to the opioid crisis may be using opioid-free analgesia. However, the number of comparative studies in this field is limited and existing small trials do not reflect current standards of care in North America. Lack of evidence means that the decision to prescribe opioids after outpatient surgery largely depends on surgeon preference and healthcare culture. Hence, there is an urgent need for a robust randomized controlled trial (RCT) to guide clinical decision-making. The feasibility and optimal design of this RCT should be informed by a pilot trial. The overarching goal of this pilot RCT is to investigate the feasibility of conducting a full-scale RCT to assess the comparative-effectiveness of opioid versus opioid-free analgesia after outpatient general surgery. **Detailed Description:** This study will be a pragmatic, parallel, two-group, assessor-blind, pilot RCT. The investigators aim to recruit 80 adult patients at two tertiary hospitals in Montreal. Eligibility criteria will span outpatient procedures in abdominal and breast surgery. Patients are randomized on a 1:1 ratio to treatment with either opioid (standard care) or without opioid (only non-opioid analgesics). Patients will be followed up for 3 months after surgery; postoperative day (POD) 1 to POD 7 and at 2, 3 and 4 weeks after surgery, and at 3 months. Assessments will include postoperative pain, physical and mental function, adverse drug events, prolonged opioid use and opioid misuse. Feasibility outcomes will include the number of patients screened, consented and randomized, adherence with treatment and completion of follow-up. Data from this pilot study will inform the calculation of sample size requirements for the full-scale RCT. An embedded qualitative study will be conducted to help optimize trial design based on clinicians' and patients' perspective. ### Conditions Module **Conditions:** - Outpatient Surgery - Abdominal Surgery - Breast Surgery **Keywords:** - Opioids - Surgery - Postoperative Period - Postoperative Pain - Analgesia - Pain Management ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** This study will be a parallel, two-group, assessor-blind, pilot randomized trial. ##### Masking Info **Masking:** SINGLE **Masking Description:** Patient-reported outcomes and treatment adherence data will be collected via self-administered electronic questionnaires distributed using REDCap (http://project-redcap.org/) and completed by patients via smartphone, tablet or personal computer. Electronic outcome data will be transmitted directly to the REDCap database and verified by a blinded assessor. Adherence data will be verified by unblinded study staff. Patients who are not computer savvy, have limited access or prefer non-electronic assessment will complete the questionnaires via telephone interviews with a blinded assessor; in this case, data will be recorded in paper forms and subsequently transferred to the REDCap database. Outcome data that are not patient-reported (e.g. postoperative complications, unplanned healthcare utilization, chronic opioid use), will be obtained from medical records by a blinded assessor. **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 81 **Type:** ACTUAL **Phases:** - PHASE2 - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Drug: Opioid analgesics **Label:** Opioid analgesia **Type:** EXPERIMENTAL #### Arm Group 2 **Intervention Names:** - Drug: Non-opioid analgesics **Label:** Opioid-free analgesia **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Opioid analgesia **Description:** Current standard of care in the participating centers Prescription of around-the-clock non-opioid analgesics (acetaminophen and/or NSAIDs/COX-2) and a supply of opioids to be used as a rescue in case of breakthrough pain. The specific round-the-clock analgesia and rescue opioid regimens will be determined by the patient's primary surgeon considering the surgical procedure, comorbidities and patient's preference. **Name:** Opioid analgesics **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Opioid-free analgesia **Description:** Prescription of around-the-clock non-opioid analgesics (acetaminophen alone or combined with NSAIDs/COX-2). The specific non-opioid analgesia regimens will be determined by the patient's primary surgeon considering the surgical procedure, comorbidities and patient's preference. **Name:** Non-opioid analgesics **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** At least 70% of patient undergoing the outpatient general surgery procedures of interest are eligible to be randomized. **Measure:** Feasibility: Rate of eligibility **Time Frame:** 4 months **Description:** At least 90% of the surgeons who agreed to have their patients randomized will comply with the agreement (i.e. not change their minds). **Measure:** Feasibility: Rate of acceptability of the trial by surgeons **Time Frame:** 4 months **Description:** At least 50% of eligible patients agree to participate in the study and are randomized. **Measure:** Feasibility: Rate of recruitment (acceptability of the trial by patients) **Time Frame:** 4 months **Description:** At least 80% of the randomized patients comply with their allocated treatment (i.e. will take their pain medications as prescribed). **Measure:** Feasibility: Rate of treatment compliance **Time Frame:** 4 months **Description:** At least 80% of the patients randomized complete outcome assessment at 30-days after surgery. **Measure:** Feasibility: Rate of follow-up compliance **Time Frame:** 4 months **Description:** Among patients who complete outcome assessments, the proportion of missing data is less than 10% (i.e. non-response to questionnaires or specific questionnaire items). **Measure:** Feasibility: Rate of missing questionnaire data **Time Frame:** 4 months #### Secondary Outcomes **Description:** Measured using the Brief Pain Inventory Short-Form, which addresses pain severity and interference in the last 24 hours. Scores range from 0 to 10; higher scores represent worse pain outcomes. **Measure:** Postoperative pain **Time Frame:** 30 days. **Description:** The time to the first report of stopping the use of pain medication. **Measure:** Time to stopping pain medication **Time Frame:** 30 days. **Description:** Measured using the PROMIS-29 questionnaire, which assesses 7 domains of health (physical function, anxiety, depression, fatigue, sleep disturbance, ability to participate in social roles and activities, and pain interference). Norm-based scores are calculated for each domain, such that a score of 50 represents the mean of the general population (standard deviation=10). Higher scores represent more of the domain being measured. **Measure:** Postoperative health status **Time Frame:** Weeks 1, 2, 3 and 4 after surgery. **Description:** Measured using the Perioperative Opioid-Related Symptom Distress Scale, which records symptom distress due to common adverse effects experienced by patients who receive opioids to relieve postoperative pain. Scores range from 0 to 4; higher scores represent worse symptoms. **Measure:** Opioid side-effects **Time Frame:** Postoperative days 1 to 7, weeks 2, 3 and 4 after surgery **Description:** Measured using the Prescription Opioid Abuse Index, which includes questions regarding excessive dose, frequency of use, need for early refills, feeling high from the medication, taking the medication due to stress and obtaining prescriptions from multiple physicians. Scores range from 0 to 6; an affirmative answer to \>1 question indicates the patient exhibits prescription opioid misuse. **Measure:** Rate of opioid misuse **Time Frame:** 4 weeks **Description:** Classified according to Clavien-Dindo and transformed into the Comprehensive Complication Index. Scores range from 0 (no complication) to 100 (death). **Measure:** Postoperative complications (index) **Time Frame:** 30 days. **Description:** Emergency department visits, hospital readmissions. **Measure:** Rate of unplanned healthcare utilization **Time Frame:** 30 days. **Description:** Obtained from spontaneous patient reporting (Trigger question "Did you have any significant medical problem related or unrelated to your surgery since the last study assessment?"). Events will be coded using the MedDRA coding dictionary, graded by severity according to the Common Terminology Criteria for Adverse Events (CTCAE) and assessed for potential causality using the WHO/UMC system. **Measure:** Rate of adverse drug events **Time Frame:** 30 days. **Description:** Filling of opioid prescriptions up to 3 months after surgery. **Measure:** Rate of prolonged opioid use **Time Frame:** 3 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Adult patients (\>18 yo) undergoing outpatient surgery * Abdominal surgery (i.e. cholecystectomies, hernia repairs, ovarian cystectomies, salpingectomies) * Breast surgery (i.e. lumpectomies, partial and complete mastectomies, axillary node dissections) Exclusion Criteria: All patients * Intraoperative or early postoperative complications (i.e. diagnosed in the Post-Anesthesia Care Unit (PACU)) that require postoperative hospital stay * Contraindications to any of the drugs used in the trial * Difficult to be reached after surgery * Inability to provide written informed consent **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Montreal **Country:** Canada **Facility:** McGill University Health Centre **State:** Quebec **Zip:** H3G 1A4 ### References Module #### References **Citation:** Do U, El-Kefraoui C, Pook M, Balvardi S, Barone N, Nguyen-Powanda P, Lee L, Baldini G, Feldman LS, Fiore JF Jr; McGill Better Opioid Prescribing Collaboration; Alhashemi M, Antoun A, Barkun JS, Brecht KM, Chaudhury PK, Deckelbaum D, Di Lena E, Dumitra S, Elhaj H, Fata P, Fleiszer D, Fried GM, Grushka J, Kaneva P, Khwaja K, Lapointe-Gagner M, McKendy KM, Meguerditchian AN, Meterissian SH, Montgomery H, Rajabiyazdi F, Safa N, Touma N, Tremblay F. Feasibility of Prospectively Comparing Opioid Analgesia With Opioid-Free Analgesia After Outpatient General Surgery: A Pilot Randomized Clinical Trial. JAMA Netw Open. 2022 Jul 1;5(7):e2221430. doi: 10.1001/jamanetworkopen.2022.21430. **PMID:** 35849399 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M13069 - Name: Pain, Postoperative - Relevance: LOW - As Found: Unknown - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Ancestors - ID: D000009294 - Term: Narcotics - ID: D000002492 - Term: Central Nervous System Depressants - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018689 - Term: Sensory System Agents - ID: D000018373 - Term: Peripheral Nervous System Agents ### Intervention Browse Module - Browse Branches - Abbrev: Analg - Name: Analgesics - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Antipy - Name: Antipyretics - Abbrev: CNSDep - Name: Central Nervous System Depressants ### Intervention Browse Module - Browse Leaves - ID: M4032 - Name: Analgesics - Relevance: HIGH - As Found: Buprenorphine - ID: M2340 - Name: Acetaminophen - Relevance: LOW - As Found: Unknown - ID: M4033 - Name: Analgesics, Opioid - Relevance: HIGH - As Found: SVT - ID: M20786 - Name: Analgesics, Non-Narcotic - Relevance: HIGH - As Found: Tcells - ID: M12245 - Name: Narcotics - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000000701 - Term: Analgesics, Opioid - ID: D000000700 - Term: Analgesics - ID: D000018712 - Term: Analgesics, Non-Narcotic ### Misc Info Module #### Submission Tracking - Estimated Results First Submit Date: 2024-03-22 ##### Submission Infos - MCP Release N: Unknown - Release Date: 2024-03-22 - Reset Date: Unknown - Unrelease Date: Unknown - Unrelease Date Unknown: Unknown - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06118879 **Brief Title:** Role of Sensory Deficits on Sensorimotor Control in Athletes With Chronic Ankle Instability **Official Title:** Role of Sensory Deficits and the Effect of Sensory Stimulation on Sensorimotor Control and Neuromuscular Performance in Athletes With Chronic Ankle Instability #### Organization Study ID Info **ID:** NYCU112052AF #### Organization **Class:** OTHER **Full Name:** National Yang Ming Chiao Tung University ### Status Module #### Completion Date **Date:** 2026-07-01 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-11-07 **Type:** ACTUAL **Last Update Submit Date:** 2023-10-30 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2026-06-30 **Type:** ESTIMATED #### Start Date **Date:** 2023-11-01 **Type:** ESTIMATED **Status Verified Date:** 2023-10 #### Study First Post Date **Date:** 2023-11-07 **Type:** ACTUAL **Study First Submit Date:** 2023-10-30 **Study First Submit QC Date:** 2023-10-30 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** National Yang Ming Chiao Tung University #### Responsible Party **Investigator Affiliation:** National Yang Ming Chiao Tung University **Investigator Full Name:** Yi-Fen Shih **Investigator Title:** Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Purpose: Chronic ankle instability (CAI), characterized by instances of repetitive sprains and giving way, is a common chronic dysfunction among athletes. Despite well documented evidence showing impaired peripheral sensory and both central and peripheral motor control, little was known regarding how CAI affects somatosensory cortical activation. Traditionally, management of CAI mostly focused on restoration of motor deficits, with less emphasis on sensory pathway deficits. Only few sensory targeted intervention methods including joint mobilization and massage, have been described. However, methods such as joint mobilization provide not only sensory stimulation but also mechanical alignment correction. In addition, these studies showed functional improvement without exploring mechanisms. Therefore we would like to apply for a three year study grant to firstly investigate the effect of CAI on peripheral and central somatosensation and neuromuscular performance; and secondly to examine the immediate effect of sensory-level electrical stimulation on neurophysiological variables and neuromuscular performance; and lastly to determine the short-term effect of intervention (sensory stimulation with exercise, sham stimulation with exercise, and exercise alone) in athletes with CAI. Research design and methods: This is a cross-sectional exploratory and randomized controlled study. For the first year, we plan to recruit 45 young athletes (20-40 years old) with CAI and 15 matched controls to compare the somatosensory evoked potentials (SEPs), corticomuscular coherence (CMC), α- band event-related desynchronization (ERD), proprioception, two point discrimination threshold of the plantar surfaces, muscle activation and reaching distances of the Y balance test. Activity in the sensorimotor cortex will be recorded using a 64-chanel EEG (SAGA 32/64+ for EEG). Muscle activation is measured using an 8-channel electromyography (EMG) system (Noraxon myo METRICS Portable Lab, Noraxon U.S.A.). For the second year, the 45 young adults with CAI will be randomized into the sensory stimulation (TENS) group, the sham stimulation group, or the control group. The TENS group will receive 40 minutes of sensory electrical stimulation, and the sham group will receive 30 seconds of sensory stimulation at the beginning and end of the treatment. The control group will rest for 40 minutes. The assessment items are identical to those in the first year, and will be carried out before and immediately after the intervention. For the third year, the 45 young adults with CAI will be randomized into the exercise with sensory stimulation (EX-TENS) group, exercise with sham stimulation (EX-ss) group, or exercise alone (EX) group. Participants will receive five sessions of intervention (30 minutes neuromuscular training with or without 40 min stimulation) within two weeks and the assessment will be carried out before and after the intervention. Data analysis: Comparisons of all continuous variables are performed using oneway analysis of variance (ANOVA), and repeated measures ANOVA. The significance level is set at 0.05. Significance. Results of this study provide a better understanding for central somatosensory control mechanisms for chronic ankle instability, and help clinicians and trainers to choose the most appropriate training strategy for people with unstable ankles. We plan to present our work in 3-4 international conferences, and publish 2-3 papers in SCI journals in 5 years. **Detailed Description:** Characteristics of the chronic ankle instability (CAI): Lateral ankle sprain is one of the most common musculoskeletal injuries in the sports clinics. The high prevalence of lateral ankle sprains, combined with a high recurrence rate and persistent post sprain symptoms make lateral ankle sprains and their sequelae a significant health impact and healthcare burden. Although lateral ankle sprains are generally considered benign injuries, it is estimated that 40% of individuals with lateral ankle sprains will subsequently develop chronic ankle instability within the first year after a lateral ankle sprain. Chronic ankle instability is characterized by repetitive sprains, perceived instability, and giving way, which can be classified into two categories: mechanical and functional ankle instability. Mechanical instability of the ankle is a result of loss of mechanical structural constraints (i.e., ligaments); whereas functional instability refers to instability related to altered postural control and reduced functional capacity due to impaired sensorimotor control including altered muscle recruitment around the ankle and impairment of the proprioceptive system etc.. In addition to interfering with sporting performance, and CAI has been found to be associated with lower quality of life and increased likelihood (68%-75%) of developing ankle arthritis. Hertel reported that individuals with ankle instability had higher risk of recurrent ankle sprains, and the treatment for symptoms of CAI could be time-consuming and costly. Numerous impairments have been reported associated with CAI, ranging from loss of joint mobility and muscle strength, compromised proprioception and sensation, poor postural control and neuromuscular performance, and altered movement patterns. However, the mechanisms and management strategies are still not clearly understood in many aspects. And thus, a great variety of intervention has been proposed with inconsistent results reported. Conceptual model for the development of CAI: The theory linking poor neuromuscular control and CAI has been discussed for more than half a century. It was believed that after the ankle sprain, some of the joint receptors and peripheral nerve fibers around the ankle joint were affected, resulting in impaired reflex responses and functional stability of the ankle. More researchers later reported that CAI not only presented with sensory deficits, but also deficits in motor control, and these changes in sensorimotor system would eventually lead to loss of function. In 2019, Hertel et al. (2019) revised the current model for the development of CAI. It is proposed that the initial ankle injury would influence the neural system, disturbing the balance and stability between the sensation, perception and motor function. These disturbances would lead to self-adjustment of the sensorimotor system to attempt to adapt to the altered condition, and consequently might result in long term changes in neural network, and expose individuals to persistent symptoms of instability. This newly revised model integrates both peripheral and central sensorimotor systems to explain the development of CAI; but it clearly shows that the central sensorimotor processing in individuals with CAI is less explored, particularly for the impact of CAI on the somatosensory cortex. Individuals with CAI have poorer neuromuscular performance: The effect of CAI on the neuromuscular control and posture stability has been extensively studied. The research topics of interest included altered reflexes and neuromuscular inhibition, muscle activation patterns (timing, recruitment order, and amplitude) and joint mechanics of the lower extremity segments during sudden inversion perturbation or jump landing, and posture control characteristics during balance testing tasks. It is generally agreed that people with CAI had lower Hmax/Mmax ratios (H reflex/M reflex ratios) of the soleus and peroneus longus muscle than those un-injured controls, indicating that neural excitability of the spinal level in those with CAI was depressed; this population also presented with altered muscle activation of the peroneus, tibialis anterior and soleus muscles, and changed balance control strategies. Recent publications of systematic review and meta-analysis by Hoch and McKeon (2014) and by Thompson et al. (2018) showed that there was a strong evidence to support dynamic balance, peroneal reaction, and eversion strength deficits contributing to CAI. Because of the strong evidence showing motor impairment in CAI, the dominant intervention strategy for this population has been motor performance driven so far. Deficits in somatosensation in people with CAI Proprioceptive deficits in CAI have been the main focus of research for decades. Thompsons et al. (2018) indicated that there was moderate evidence supporting both active and passive proprioception deficits in CAI as compared with controls. According to the latest systematic review and meta-analysis by Xue et al. (2021), CAI-affected ankles had poorer ankle kinesthesia in both inversion and plantarflexion when compared with the contralateral side; and individuals with CAI showed kinesthesia defects and active joint reposition deficits in the frontal plane movement (both inversion and eversion) when compared to the healthy controls. Not until recent years that more attention has been drawn to deficits in cutaneous sensation. Hoch et al. (2012) found that the plantar surface was less sensitive to vibratory tactile stimulation as compared to non-CAI people at the head of the first metatarsal, the base of the fifth metatarsal, and the heel, along the innervation of the branches of the sural and tibial nerves. Burcal and Wikstrom found similar results that people with CAI displayed higher Semmes Weinstein monofilament thresholds at the head of the first metatarsal, base of the fifth metatarsal, and sinus tarsi31. The possible involvement of impaired cutaneous sensation in CAI have led to the recent approach of sensory-targeted intervention in this population. Deficits in central sensorimotor control in CAI: As described in the previous paragraphs that the classic theory behind the ankle instability has been based on damages of joint receptors and peripheral nerve fibers. However, emerging data has shown that proprioceptive sensory deficits were not enough to explain the neuromuscular deficits in people with CAI, and that more research is needed to determine how CAI influences central sensorimotor control. Previous studies have shown that in individuals with chronic musculoskeletal dysfunction such as shoulder impingement and low back pain, the integration of corticospinal pathways may be affected, presented as higher active motor threshold, longer cortical silent period, reduced cortical mapping area and shifted center of gravity of the cortical mapping area. Possible impairment of the corticospinal control in CAI has recently been examined in a few studies. While Pietrosimone and Gribble (2012), Shih et al. (2017), and Terada et al. (2022) reported that muscles around the ankle joint had impaired corticospinal excitability (reduced motor-evoked potential of the peroneus longus and soleus muscles), the two systemic reviews with meta analysis did not support these findings and showed that people with or without CAI had similar supraspinal neural excitability. These inconsistencies might be related to the differences in study design, such as testing positions and tasks. The role of altered central motor system excitability in CAI has yet to reach agreement; and little was known about the effect of CAI on the somatosensory cortex. Common outcome measures to evaluate somatosensory cortical activation use electroencephalography (EEG) to detect electrophysiological signals of excitatory and inhibitory action potentials in various areas of the cortex. Previous studies reported abnormalities of P27 potentials and somatosensory-evoked potentials (SEPs) in people with anterior cruciate ligament (ACL) deficits, indicating somatosensory cortex neuroplasticity following ACL injuries. Only one study has evaluated the somatosensory cortical activation in people with ankle instability, and found no difference in somatosensory cortical activity (event-related desynchronization (ERD) of the upper alpha frequency) between healthy controls and individuals with unstable ankles. With strong evidence showing connections between central sensorimotor deficits and chronic musculoskeletal problems such as low back pain and ACL deficits, we believe that future research is required to understand the role of sensory deficits, particularly the central somatosensory activation in people with CAI and to provide further explanation for the neurophysiological mechanisms for ankle instability. Restoration of motor performance has been the popular intervention for CAI: From the above review, it is obvious that problems behind chronic ankle instability relate to both sensory and motor systems. However, the majorities of intervention strategies have focused on restoring the defects in motor pathways, including muscle strengthening, neuromuscular training, posture and balance control training. Although many of the studies found positive effects of these motor restoration interventions, the synthesis of evidence only supported partially (limited to moderate level) for the effectiveness of neuromuscular or balance training on muscle performance, postural stability or self-perceived improvement. Emerging evidence showing effectiveness of sensory-orientated intervention for CAI: With more understanding on the involvement of sensory deficits in CAI, some researchers started to describe intervention methods using primarily sensory inputs, such as planar massage, and joint mobilization. Joint mobilization was found to improve dynamic posture performance including landing kinematics, reaching distances of the Y balance test, ankle mobility, and self-perceived dysfunction questionnaires. Although joint mobilization was proposed to have the effect of "resetting the sensorimotor control", the spinal-reflex and corticospinal excitability of the soleus and peroneus longus were not altered after one session of joint mobilization. In addition, joint mobilization not only provides sensory stimulation but also offers alignment correction for people with CAI. The effectiveness of joint mobilization could have come from improvement in ankle mobility rather than sensory intervention. Several studies have investigated the effect of pure sensory inputs, the plantar massage, in people with CAI and found that plantar massage effectively improved static postural control. However, these studies only measured perceived dysfunction, or functional performance. No neurophysiological mechanism of sensory intervention in CAI has been explored before. Electrical stimulation as a form of treatment for CAI: Traditionally, neuromuscular stimulation has been used as a form of treatment for various conditions, from peripheral nerve injuries, conditions involving arthrogenic inhibition such as ACL insufficiency or patellofemoral pain, to neurological conditions including stroke or spinal cord injury. A great body of evidence has shown that neuromuscular stimulation is effective in restoring motor functions in these conditions. Another approach of electrical stimulation is to use sensory level of stimulation in attempt to improve sensorimotor control. The rationale is that somatosensory stimulation would enhance cortical plasticity in people post stroke. Pan et al. recruited 12 patients post stroke and assessed the effect of 40 minutes of sensory electrical stimulation, combined with hand rehabilitation program. The changes in sensorimotor control were evaluated using corticomuscular coherence (CMC). After 4 weeks of treatment, the CMC value of the electrical stimulation group is higher than that of the control group, indicating that the cerebral motor cortex and the corresponding muscles have a greater and physiologically meaningful functional connection. Although people with CAI has been found to have benefited from sensory intervention such as plantar massage, no neurophysiological mechanism has been studied. In addition, application of sensory transcutaneous electrical stimulation (TENS) is a relatively risk-free and easy-to-implement modality for rehabilitation. However, no study has investigated the effect and treatment mechanisms of TENS in people with CAI. Research gap and contribution of the study: Current research evidence supported that people with chronic ankle instability have deficits in peripheral sensorimotor systems and central motor impairments, resulting in defects in sensorimotor control and functional performance. However, little was known about how CAI affects the somatosensory cortical activation. The effects of strength training, neuromuscular control training, posture/balance training has been widely investigated for CAI, and positive effects on neuromuscular control, corticospinal excitability, and functional performance have been found for this group of people. Recently, sensory intervention for CAI has been proposed and its treatment effect on static and dynamic posture control as well as functions were reported. Based on the effectiveness of sensory intervention and posture adaptation strategies shown in individuals with CAI, it is reasonable to speculate that central somatosensory processing might have a role in altered neuromuscular performance and recovery (sensory intervention effect) of CAI. However, these neurophysiological mechanisms have never been studied before. To address the above issues, we would like to apply for a three-year grant to firstly investigate the effect of CAI on somatosensory cortical activation, corticomuscular coherence, proprioception, cutaneous sensation of the plantar surfaces, and neuromuscular performance. These variables will be compared between 45 young active adults with CAI and 15 controls for the first year study. Secondly, those with CAI will be randomized into three groups, the sensory stimulation group, the sham stimulation group, and the control group. The participants will receive 40 minutes of sensory level of electrical stimulation or sham stimulation or nothing, and the immediate effect will be examined to understand the effect of sensory-level stimulation on neurophysiological mechanisms and neuromuscular performance. Afterwards, the intervention (sensory stimulation with exercise, sham stimulation with exercise, and exercise alone) will be carried out for 5 sessions in two weeks, and the short term intervention effect of additional sensory stimulation will be assessed. The results of this study will provide a better understanding for sensorimotor control in CAI, and for the effect of sensory stimulation intervention in the population with CAI. The data from this investigation will provide clinicians, trainers, and researchers an excellent basis for the management and injury prevention of CAI among athletes, and for further research in this field of study. ### Conditions Module **Conditions:** - Chronic Ankle Instability ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Count:** 150 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Device: Transcutaneous electrical stimulation **Label:** Sensory level stimulation with exercise **Type:** EXPERIMENTAL #### Arm Group 2 **Intervention Names:** - Device: Transcutaneous electrical stimulation **Label:** Sham stimulation with exercise **Type:** SHAM_COMPARATOR #### Arm Group 3 **Intervention Names:** - Device: Transcutaneous electrical stimulation **Label:** Exercsie alone **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Exercsie alone - Sensory level stimulation with exercise - Sham stimulation with exercise **Description:** sensory level stimulation, one-leg standing and Y-balance training **Name:** Transcutaneous electrical stimulation **Other Names:** - Neuromuscular training exercise **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** somatosesory evoked potentials and corticomuscular coherence **Measure:** Cortical activation **Time Frame:** 5 minutes #### Secondary Outcomes **Description:** ankle inversion and plantarflexion joint position sense **Measure:** Proprioception **Time Frame:** 10 minutes **Description:** Cutaneous sensation of the plantar surfaces **Measure:** Two-point discrimination **Time Frame:** 10 minutes **Description:** ankle dorsiflexion range of motion **Measure:** Range of motion **Time Frame:** 5 minutes ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * between 18 and 40 years old * having at least one ankle sprain experience in the past year with sequelae; or having repeated ankle sprain experience in the past year, * having a "soft foot" sensation within three months * score ≤ 27 on the Cumberland Ankle Instability Tool (CAIT) Exclusion Criteria: * having a history of lower extremity fracture or surgery, or a history of lower extremity trauma in the past three months * having experiences of sprained ankles within the previous six weeks * having pathological joint laxity (positive results on talar tilt test or drawer forward test **Healthy Volunteers:** True **Maximum Age:** 40 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Taipei **Contacts:** ***Contact 1:*** - **Email:** yfshih@ym.edu.tw - **Name:** Yi-Fen Shih, PhD - **Phone:** +886-2-2826-7340 - **Role:** CONTACT **Country:** Taiwan **Facility:** National Yang Ming Chiao Tung University **Status:** RECRUITING **Zip:** 11221 ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ### References Module #### References **Citation:** Kim KM, Ingersoll CD, Hertel J. Altered postural modulation of Hoffmann reflex in the soleus and fibularis longus associated with chronic ankle instability. J Electromyogr Kinesiol. 2012 Dec;22(6):997-1002. doi: 10.1016/j.jelekin.2012.06.002. Epub 2012 Jul 13. **PMID:** 22795679 **Citation:** Kim KM, Kim JS, Cruz-Diaz D, Ryu S, Kang M, Taube W. Changes in Spinal and Corticospinal Excitability in Patients with Chronic Ankle Instability: A Systematic Review with Meta-Analysis. J Clin Med. 2019 Jul 16;8(7):1037. doi: 10.3390/jcm8071037. **PMID:** 31315231 **Citation:** Suttmiller AMB, McCann RS. Neural excitability of lower extremity musculature in individuals with and without chronic ankle instability: A systematic review and meta-analysis. J Electromyogr Kinesiol. 2020 Aug;53:102436. doi: 10.1016/j.jelekin.2020.102436. Epub 2020 Jun 1. **PMID:** 32505988 **Citation:** Wikstrom EA, McKeon PO. Predicting balance improvements following STARS treatments in chronic ankle instability participants. J Sci Med Sport. 2017 Apr;20(4):356-361. doi: 10.1016/j.jsams.2016.09.003. Epub 2016 Sep 20. **PMID:** 27840034 **Citation:** Shih YF, Yu HT, Chen WY, Liao KK, Lin HC, Yang YR. The effect of additional joint mobilization on neuromuscular performance in individuals with functional ankle instability. Phys Ther Sport. 2018 Mar;30:22-28. doi: 10.1016/j.ptsp.2017.12.001. Epub 2017 Dec 20. **PMID:** 29310055 **Citation:** McKeon PO, Wikstrom EA. Sensory-Targeted Ankle Rehabilitation Strategies for Chronic Ankle Instability. Med Sci Sports Exerc. 2016 May;48(5):776-84. doi: 10.1249/MSS.0000000000000859. **PMID:** 26717498 **Citation:** Pan LH, Yang WW, Kao CL, Tsai MW, Wei SH, Fregni F, Chen VC, Chou LW. Effects of 8-week sensory electrical stimulation combined with motor training on EEG-EMG coherence and motor function in individuals with stroke. Sci Rep. 2018 Jun 15;8(1):9217. doi: 10.1038/s41598-018-27553-4. **PMID:** 29907780 **Citation:** Kim KM, Croy T, Hertel J, Saliba S. Effects of neuromuscular electrical stimulation after anterior cruciate ligament reconstruction on quadriceps strength, function, and patient-oriented outcomes: a systematic review. J Orthop Sports Phys Ther. 2010 Jul;40(7):383-91. doi: 10.2519/jospt.2010.3184. **PMID:** 20592480 **Citation:** Xue X, Ma T, Li Q, Song Y, Hua Y. Chronic ankle instability is associated with proprioception deficits: A systematic review and meta-analysis. J Sport Health Sci. 2021 Mar;10(2):182-191. doi: 10.1016/j.jshs.2020.09.014. Epub 2020 Oct 2. **PMID:** 33017672 **Citation:** Needle AR, Lepley AS, Grooms DR. Central Nervous System Adaptation After Ligamentous Injury: a Summary of Theories, Evidence, and Clinical Interpretation. Sports Med. 2017 Jul;47(7):1271-1288. doi: 10.1007/s40279-016-0666-y. **PMID:** 28005191 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: Rare - Name: Rare Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: HIGH - As Found: Chronic ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04886479 **Brief Title:** Comparing Silicone Hydrogel Contact Lens Wearers to Hydrogel Contact Lens Wearers and Non-lens Wearers **Official Title:** Comparing Habitual Daily Disposable Silicone Hydrogel (DDSH) Contact Lens Wearers to Habitual Daily Disposable Hydrogel (DDH) Contact Lens Wearers and Non-lens Wearers #### Organization Study ID Info **ID:** EX-MKTG-89 #### Organization **Class:** INDUSTRY **Full Name:** Coopervision, Inc. ### Status Module #### Completion Date **Date:** 2022-10-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-01-30 **Type:** ACTUAL **Last Update Submit Date:** 2024-01-03 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-10-07 **Type:** ACTUAL #### Results First Post Date **Date:** 2024-01-30 **Type:** ACTUAL **Results First Submit Date:** 2023-10-05 **Results First Submit QC Date:** 2024-01-03 #### Start Date **Date:** 2019-03-06 **Type:** ACTUAL **Status Verified Date:** 2023-11 #### Study First Post Date **Date:** 2021-05-14 **Type:** ACTUAL **Study First Submit Date:** 2021-05-10 **Study First Submit QC Date:** 2021-05-10 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Coopervision, Inc. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** True **Is FDA Regulated Drug:** False **Is US Export:** True **Oversight Has DMC:** True ### Description Module **Brief Summary:** This study was a multi-site, prospective, randomized, non-masked, unilateral, non-dispensing study. **Detailed Description:** The purpose of this study was to to compare physiological response baseline data between 3 groups - habitual wearers of Daily Disposable Silicone Hydrogel (DDSH) contact lenses, habitual wearers of Daily Disposable Hydrogel (DDH) contact lenses, and non-lens wearers. The study then compared physiological response data following wear of a low Dk HEMA contact lens between habitual wearers of DDSH and habitual wearers of DDH. ### Conditions Module **Conditions:** - Myopia ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 22 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants wore Test Lens for 3 hours **Intervention Names:** - Device: Test Lens **Label:** Daily Disposable Silicone Hydrogel Contact Lens Wearers **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants wore Test Lens for 3 hours **Intervention Names:** - Device: Test Lens **Label:** Daily Disposable Hydrogel Contact Lens Wearers **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Participants did not receive Test Lens **Intervention Names:** - Other: Control **Label:** Non-lens Wearers **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Non-lens Wearers **Description:** No lens **Name:** Control **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Daily Disposable Hydrogel Contact Lens Wearers - Daily Disposable Silicone Hydrogel Contact Lens Wearers **Description:** Low Dk HEMA contact lens worn for 3 hours **Name:** Test Lens **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Objective grading (0-4, severity increases in 0.1 steps; 0=no redness, 4=severe redness) of limbal redness after 6-8 hours of habitual device wear. For each participant, measurements were taken for both eyes (OD - right eye, OS - left eye) at two locations (T - temporal, N - nasal). **Measure:** Limbal Redness - DDSH, DDH, Non-lens Groups **Time Frame:** 6-8 hours of habitual wear **Description:** Objective grading (0-4, severity increases in 0.1 steps; 0=no redness, 4=severe redness) of bulbar redness after 6-8 hours of habitual device wear. For each participant, measurements were taken for both eyes (OD - right eye, OS - left eye) at two locations (T - temporal, N - nasal). **Measure:** Bulbar Redness DDSH, DDH, Non-lens Groups **Time Frame:** 6-8 hours of habitual wear **Description:** Number of participants with limbal vessel ingrowth longer than 0.50 mm at any of the 4 corneal quadrants (superior, inferior, nasal, temporal) after 6-8 hours of device wear. **Measure:** Limbal Vessel Ingrowth - DDSH, DDH, Non-lens Groups **Time Frame:** 6-8 hours of habitual wear **Description:** Objective grading (0-4, severity increases in 0.1 steps; 0=no redness, 4=severe redness) of limbal redness by location (T - temporal, N - nasal) and time (t). Measurements were taken at baseline and over a 3 hour period of closed-eye Test Lens wear. **Measure:** Limbal Redness - DDSH, DDH Groups **Time Frame:** Immediately upon eye opening (t=baseline) and lens removal (t=0), 1 hour (t=1), and 3 hours (t=3) post lens removal **Description:** Objective grading (0-4, severity increases in 0.1 steps; 0=no redness, 4=severe redness) of bulbar redness by location (T - temporal, N - nasal) and time (t). Measurements were taken at baseline and over a 3 hour period of closed-eye Test Lens wear. **Measure:** Bulbar Redness - DDSH, DDH Groups **Time Frame:** Immediately upon eye opening (baseline) and lens removal (t=0), 1 hour (t=1), and 3 hours (t=3) post lens removal #### Secondary Outcomes **Description:** Difference in central corneal swelling (μm, Visante OCT) at baseline (before lens insertion) vs. measurements taken over a 3 hour period following Test Lens removal. Derived by the formula: corneal swelling % = (measured corneal thickness - baseline corneal thickness) x 100 /baseline corneal thickness. **Measure:** Central Corneal Swelling (%) - DDSH, DDH Groups **Time Frame:** Immediately upon eye opening and lens removal (t=0), 0.5 hours (t=0.5), 1 hour (t=1), 2 hours (t=2), and 3 hours (t=3) post lens removal ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Is between 17 and 60 years of age inclusive (age matching required between all 3 study groups), and has full legal capacity to volunteer; 2. Has had a self-reported oculo-visual examination in the last two years. 3. Has read and signed an information consent letter; 4. Is willing and able to follow instructions and maintain the appointment schedule; 5. No previous history of overnight contact lens wear or PMMA (polymethylmethacrylate) lens wear. 6. Auto-refraction vertexed spherical equivalent between +4.00 and -8.00 (prescription matching required between groups 1 \& 2). This criterion is non-applicable to participants in group 3. 7. Is found to be in one of the study groups matched by age (±5 years), ethnicity (Asian vs. non-Asian) and CL Rx (±2.00 D) according using the following criteria. GROUP 1: For the past (minimum) 6 months has ONLY worn spherical DDSH lenses AND prior to wearing this lens they only wore SH material lenses in the past (minimum) 3 total years. GROUP 2: For the past (minimum) 6 months has ONLY worn spherical DDH lenses AND prior to wearing this lens they only wore H material lenses in the past (minimum) 3 total years. 8. For groups 1 \& 2, currently and for at least the previous 6 months has had habitual daily disposable wear schedule of at least 8 hrs a day, 5 days a week in only one material category - either silicone hydrogel or hydrogel material. This criterion is not-applicable to participants in group 3. 9. Is willing to wear the study contact lens in the randomized eye for 3 hours of eye closure on the second study day. This criterion is not-applicable to participants in group 3. 10. Has clear and healthy corneas and anterior eye and no active ocular disease; 11. Can achieve monocular HCVA of logMAR 0.10 or better in each eye with subjective refraction or pinhole. 12. Can achieve acceptable fit and comfort in the randomized eye with the study lens. This criterion will be confirmed at the baseline visit (V1). This criterion is not-applicable to participants in group 3. 13. Has a wearable pair of spectacles. Exclusion Criteria: 1. Is participating in any concurrent clinical trial; 2. Is unable/unwilling to provide permission for the study site to seek CL history from their eye care practitioner 3. Has any known active ocular disease and/or infection; 4. Has a systemic condition that in the opinion of the investigator may affect a study measure or interfere with contact lens wear; this may include, but not be limited to, diabetes, hyperthyroidism, recurrent herpes simplex/zoster, Sjogren's syndromes, xerophthalmia, acne rosacea, Stevens-Johnson syndromes, and systemic connective tissue disorders e.g. rheumatoid arthritis. 5. Is using any systemic or topical medications that in the opinion of the investigator may affect a study measure; 6. Has known sensitivity to fluorescein dye, topical anesthetic, or products to be used in the study; 7. Appears to have any active ocular pathology, ocular anomaly or severe insufficiency of lacrimal secretion (severe dry eye) that would affect the wearing of contact lenses; 8. Appears to have any signs of corneal inflammation or previous infection or corneal opacity/scar; 9. Is pregnant, lactating or planning a pregnancy at the time of enrolment (by verbal confirmation at the screening visit), due to potential ocular physiological changes, such as changes in the corneal shape and cell types; 10. Is aphakic; 11. Has undergone refractive error surgery, or has a history of any ocular surgery or injury. 12. Is a toric or multifocal contact lens wearer. **Healthy Volunteers:** True **Maximum Age:** 60 Years **Minimum Age:** 17 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Waterloo **Country:** Canada **Facility:** CORE, University of Waterloo **Location 2:** **City:** Manchester **Country:** United Kingdom **Facility:** Eurolens Research #### Overall Officials **Official 1:** **Affiliation:** Centre for Ocular Research & Education **Name:** Lyndon Jones, PhD FCOptom **Role:** PRINCIPAL_INVESTIGATOR ## Document Section ### Large Document Module #### Large Docs - Date: 2020-11-28 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 445821 - Type Abbrev: Prot_SAP - Upload Date: 2023-10-05T16:56 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012030 - Term: Refractive Errors - ID: D000005128 - Term: Eye Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC11 - Name: Eye Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M12168 - Name: Myopia - Relevance: HIGH - As Found: Myopia - ID: M14872 - Name: Refractive Errors - Relevance: LOW - As Found: Unknown - ID: M8271 - Name: Eye Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009216 - Term: Myopia ### Misc Info Module #### Submission Tracking ##### First MCP Info ###### Post Date - Date: 2023-11-01 - Type: ACTUAL - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** DDSH Lens Wearers **Deaths Num At Risk:** 10 **Description:** Participants that wore DDSH Lens in Period 1 and Test Lens in Period 2 **ID:** EG000 **Other Num at Risk:** 10 **Serious Number At Risk:** 10 **Title:** DDSH Lens Wearers **Group ID:** EG001 **Title:** DDH Lens Wearers **Deaths Num At Risk:** 3 **Description:** Participants that wore DDH Lens in Period 1 and Test Lens in Period 2 **ID:** EG001 **Other Num at Risk:** 3 **Serious Number At Risk:** 3 **Title:** DDH Lens Wearers **Group ID:** EG002 **Title:** Non-lens Wearers **Deaths Num At Risk:** 9 **Description:** Participants that did not wear DDSH or DDH Lenses in Period 1 **ID:** EG002 **Other Num Affected:** 1 **Other Num at Risk:** 9 **Serious Number At Risk:** 9 **Title:** Non-lens Wearers **Frequency Threshold:** 0 #### Other Events **Term:** Lightheadedness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Transitional feeling of light-headedness during confocal microscopy. Unrelated to study, as neither DDH Lens nor DDSH Lens were worn at time of event. **Organ System:** Eye disorders **Source Vocabulary:** **Time Frame:** Through study exit, up to 30 days from the first visit. ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 10 **Group ID:** BG001 **Value:** 3 **Group ID:** BG002 **Value:** 9 **Group ID:** BG003 **Value:** 22 **Units:** Participants ### Group **ID:** BG000 **Title:** DDSH Lens Wearers **Description:** Participants who habitually wear DDSH lenses ### Group **ID:** BG001 **Title:** DDH Lens Wearers **Description:** Participants who habitually wear DDH lenses ### Group **ID:** BG002 **Title:** Non-lens Wearers **Description:** Participants that do not wear either DDSH or DDH Lens. ### Group **ID:** BG003 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 13.2 **Value:** 32.7 #### Measurement **Group ID:** BG001 **Spread:** 7.5 **Value:** 30.0 #### Measurement **Group ID:** BG002 **Spread:** 13.2 **Value:** 31.7 #### Measurement **Group ID:** BG003 **Spread:** 12.2 **Value:** 31.9 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 5 #### Measurement **Group ID:** BG001 **Value:** 2 #### Measurement **Group ID:** BG002 **Value:** 6 #### Measurement **Group ID:** BG003 **Value:** 13 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 5 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 3 #### Measurement **Group ID:** BG003 **Value:** 9 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 9 #### Measurement **Group ID:** BG003 **Value:** 22 **Class Title:** Non-Asian #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 **Class Title:** Asian **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Description:** Ethnicity was measured due to possible differences in physiological response to corneal hypoxia in Asian and non-Asian participants. All subjects in this study were of non-Asian ethnicity, due to difficulty in finding and recruiting Asian candidates at the study site. **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race/Ethnicity, Customized **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement **Restrictive Agreement:** True ### Point of Contact **Email:** jvega2@coopervision.com **Organization:** CooperVision Inc. **Phone:** +19256213761 **Title:** Director Global Clinical Affairs ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.24 - **Spread:** - **Upper Limit:** 0.58 - **Value:** 0.41 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.05 - **Spread:** - **Upper Limit:** 0.68 - **Value:** 0.37 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 0.26 - **Spread:** - **Upper Limit:** 0.63 - **Value:** 0.44 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.25 - **Spread:** - **Upper Limit:** 0.73 - **Value:** 0.49 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -0.04 - **Spread:** - **Upper Limit:** 0.84 - **Value:** 0.40 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 0.19 - **Spread:** - **Upper Limit:** 0.70 - **Value:** 0.44 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.35 - **Spread:** - **Upper Limit:** 0.73 - **Value:** 0.54 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -0.05 - **Spread:** - **Upper Limit:** 0.65 - **Value:** 0.30 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 0.25 - **Spread:** - **Upper Limit:** 0.66 - **Value:** 0.46 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.18 - **Spread:** - **Upper Limit:** 0.46 - **Value:** 0.32 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.12 - **Spread:** - **Upper Limit:** 0.62 - **Value:** 0.37 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 0.27 - **Spread:** - **Upper Limit:** 0.56 - **Value:** 0.41 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.55 - **Spread:** - **Upper Limit:** 0.97 - **Value:** 0.76 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.28 - **Spread:** - **Upper Limit:** 1.05 - **Value:** 0.67 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 0.56 - **Spread:** - **Upper Limit:** 1.01 - **Value:** 0.79 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.59 - **Spread:** - **Upper Limit:** 1.13 - **Value:** 0.86 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.33 - **Spread:** - **Upper Limit:** 1.33 - **Value:** 0.83 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 0.42 - **Spread:** - **Upper Limit:** 1.00 - **Value:** 0.71 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.63 - **Spread:** - **Upper Limit:** 1.23 - **Value:** 0.93 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.18 - **Spread:** - **Upper Limit:** 1.28 - **Value:** 0.73 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 0.48 - **Spread:** - **Upper Limit:** 1.12 - **Value:** 0.80 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.60 - **Spread:** - **Upper Limit:** 1.12 - **Value:** 0.86 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.13 - **Spread:** - **Upper Limit:** 1.07 - **Value:** 0.60 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 0.40 - **Spread:** - **Upper Limit:** 0.94 - **Value:** 0.67 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.28 - **Spread:** - **Upper Limit:** 0.68 - **Value:** 0.48 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -0.04 - **Spread:** - **Upper Limit:** 0.71 - **Value:** 0.33 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.50 - **Spread:** - **Upper Limit:** 0.90 - **Value:** 0.70 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.07 - **Spread:** - **Upper Limit:** 0.79 - **Value:** 0.43 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.36 - **Spread:** - **Upper Limit:** 0.86 - **Value:** 0.61 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -0.03 - **Spread:** - **Upper Limit:** 0.90 - **Value:** 0.43 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.45 - **Spread:** - **Upper Limit:** 0.97 - **Value:** 0.71 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -0.17 - **Spread:** - **Upper Limit:** 0.77 - **Value:** 0.30 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.16 - **Spread:** - **Upper Limit:** 0.56 - **Value:** 0.36 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.13 - **Spread:** - **Upper Limit:** 0.87 - **Value:** 0.50 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.44 - **Spread:** - **Upper Limit:** 1.00 - **Value:** 0.72 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.11 - **Spread:** - **Upper Limit:** 1.15 - **Value:** 0.63 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.33 - **Spread:** - **Upper Limit:** 0.81 - **Value:** 0.57 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.13 - **Spread:** - **Upper Limit:** 1.01 - **Value:** 0.57 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.34 - **Spread:** - **Upper Limit:** 0.92 - **Value:** 0.63 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -0.10 - **Spread:** - **Upper Limit:** 0.97 - **Value:** 0.43 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.55 - **Spread:** - **Upper Limit:** 1.19 - **Value:** 0.87 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.02 - **Spread:** - **Upper Limit:** 1.18 - **Value:** 0.60 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.73 - **Spread:** - **Upper Limit:** 1.27 - **Value:** 1.00 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.14 - **Spread:** - **Upper Limit:** 1.12 - **Value:** 0.63 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.51 - **Spread:** - **Upper Limit:** 1.27 - **Value:** 0.89 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -0.12 - **Spread:** - **Upper Limit:** 1.25 - **Value:** 0.57 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.69 - **Spread:** - **Upper Limit:** 1.37 - **Value:** 1.03 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -0.03 - **Spread:** - **Upper Limit:** 1.23 - **Value:** 0.60 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.47 - **Spread:** - **Upper Limit:** 1.09 - **Value:** 0.78 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.23 - **Spread:** - **Upper Limit:** 1.37 - **Value:** 0.80 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.87 - **Spread:** - **Upper Limit:** 1.41 - **Value:** 1.14 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.37 - **Spread:** - **Upper Limit:** 1.36 - **Value:** 0.87 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.73 - **Spread:** - **Upper Limit:** 1.27 - **Value:** 1.00 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.24 - **Spread:** - **Upper Limit:** 1.23 - **Value:** 0.73 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.81 - **Spread:** - **Upper Limit:** 1.61 - **Value:** 1.21 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.03 - **Spread:** - **Upper Limit:** 1.50 - **Value:** 0.77 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 7.28 - **Spread:** - **Upper Limit:** 12.07 - **Value:** 9.67 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 4.97 - **Spread:** - **Upper Limit:** 13.71 - **Value:** 9.34 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 5.61 - **Spread:** - **Upper Limit:** 9.75 - **Value:** 7.68 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 3.03 - **Spread:** - **Upper Limit:** 10.60 - **Value:** 6.82 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 3.57 - **Spread:** - **Upper Limit:** 6.76 - **Value:** 5.17 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 1.35 - **Spread:** - **Upper Limit:** 7.17 - **Value:** 4.26 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.20 - **Spread:** - **Upper Limit:** 1.77 - **Value:** 0.98 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -0.99 - **Spread:** - **Upper Limit:** 1.88 - **Value:** 0.45 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** -1.15 - **Spread:** - **Upper Limit:** 0.32 - **Value:** -0.41 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -1.80 - **Spread:** - **Upper Limit:** 0.89 - **Value:** -0.45 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Objective grading (0-4, severity increases in 0.1 steps; 0=no redness, 4=severe redness) of limbal redness after 6-8 hours of habitual device wear. For each participant, measurements were taken for both eyes (OD - right eye, OS - left eye) at two locations (T - temporal, N - nasal). **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 6-8 hours of habitual wear **Title:** Limbal Redness - DDSH, DDH, Non-lens Groups **Type:** PRIMARY **Type Units Analyzed:** Eyes (single) **Unit of Measure:** score on a scale ##### Group **Description:** Participants wore habitual DDSH Lens in both eyes **ID:** OG000 **Title:** DDSH Lens Wearers ##### Group **Description:** Participants wore habitual DDH Lens in both eyes **ID:** OG001 **Title:** DDH Lens Wearers ##### Group **Description:** Participants did not wear either DDSH or DDH Lens **ID:** OG002 **Title:** Non-lens Wearers #### Outcome Measure 2 **Description:** Objective grading (0-4, severity increases in 0.1 steps; 0=no redness, 4=severe redness) of bulbar redness after 6-8 hours of habitual device wear. For each participant, measurements were taken for both eyes (OD - right eye, OS - left eye) at two locations (T - temporal, N - nasal). **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 6-8 hours of habitual wear **Title:** Bulbar Redness DDSH, DDH, Non-lens Groups **Type:** PRIMARY **Type Units Analyzed:** Eyes (single) **Unit of Measure:** score on a scale ##### Group **Description:** Participants wore habitual DDSH Lens in both eyes **ID:** OG000 **Title:** DDSH Lens Wearers ##### Group **Description:** Participants wore habitual DDH Lens in both eyes **ID:** OG001 **Title:** DDH Lens Wearers ##### Group **Description:** Participants did not wear DDSH or DDH Lens **ID:** OG002 **Title:** Non-lens Wearers #### Outcome Measure 3 **Description:** Number of participants with limbal vessel ingrowth longer than 0.50 mm at any of the 4 corneal quadrants (superior, inferior, nasal, temporal) after 6-8 hours of device wear. **Parameter Type:** COUNT_OF_PARTICIPANTS **Reporting Status:** POSTED **Time Frame:** 6-8 hours of habitual wear **Title:** Limbal Vessel Ingrowth - DDSH, DDH, Non-lens Groups **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Participants wore habitual DDSH Lens in both eyes **ID:** OG000 **Title:** DDSH Lens Wearers ##### Group **Description:** Participants wore habitual DDH Lens in both eyes **ID:** OG001 **Title:** DDH Lens Wearers ##### Group **Description:** Participants did not wear either DDSH or DDH Lens **ID:** OG002 **Title:** Non-lens Wearers #### Outcome Measure 4 **Description:** Objective grading (0-4, severity increases in 0.1 steps; 0=no redness, 4=severe redness) of limbal redness by location (T - temporal, N - nasal) and time (t). Measurements were taken at baseline and over a 3 hour period of closed-eye Test Lens wear. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** Immediately upon eye opening (t=baseline) and lens removal (t=0), 1 hour (t=1), and 3 hours (t=3) post lens removal **Title:** Limbal Redness - DDSH, DDH Groups **Type:** PRIMARY **Type Units Analyzed:** Eyes (single) **Unit of Measure:** score on a scale ##### Group **Description:** Habitual DDSH lens wearers wore the Test Lens in one randomized eye that was kept closed and patched for 3 hours. The contralateral eye remained open. **ID:** OG000 **Title:** DDSH Lens Wearers ##### Group **Description:** Habitual DDH lens wearers wore the Test Lens in one randomized eye that was kept closed and patched for 3 hours. The contralateral eye remained open. **ID:** OG001 **Title:** DDH Lens Wearers #### Outcome Measure 5 **Description:** Objective grading (0-4, severity increases in 0.1 steps; 0=no redness, 4=severe redness) of bulbar redness by location (T - temporal, N - nasal) and time (t). Measurements were taken at baseline and over a 3 hour period of closed-eye Test Lens wear. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** Immediately upon eye opening (baseline) and lens removal (t=0), 1 hour (t=1), and 3 hours (t=3) post lens removal **Title:** Bulbar Redness - DDSH, DDH Groups **Type:** PRIMARY **Type Units Analyzed:** Eyes (single) **Unit of Measure:** score on a scale ##### Group **Description:** Habitual DDSH lens wearers wore the Test Lens in one randomized eye that was kept closed and patched for 3 hours. The contralateral eye remained open. **ID:** OG000 **Title:** DDSH Lens Wearers ##### Group **Description:** Habitual DDH lens wearers wore the Test Lens in one randomized eye that was kept closed and patched for 3 hours. The contralateral eye remained open. **ID:** OG001 **Title:** DDH Lens Wearers #### Outcome Measure 6 **Description:** Difference in central corneal swelling (μm, Visante OCT) at baseline (before lens insertion) vs. measurements taken over a 3 hour period following Test Lens removal. Derived by the formula: corneal swelling % = (measured corneal thickness - baseline corneal thickness) x 100 /baseline corneal thickness. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** Immediately upon eye opening and lens removal (t=0), 0.5 hours (t=0.5), 1 hour (t=1), 2 hours (t=2), and 3 hours (t=3) post lens removal **Title:** Central Corneal Swelling (%) - DDSH, DDH Groups **Type:** SECONDARY **Type Units Analyzed:** Eyes (single) **Unit of Measure:** Percent change in corneal swelling (%) ##### Group **Description:** Habitual DDSH lens wearers wore the Test Lens in one randomized eye that was kept closed and patched for 3 hours. The contralateral eye remained open. **ID:** OG000 **Title:** DDSH Lens Wearers ##### Group **Description:** Habitual DDH lens wearers wore the Test Lens in one randomized eye that was kept closed and patched for 3 hours. The contralateral eye remained open. **ID:** OG001 **Title:** DDH Lens Wearers ### Participant Flow Module #### Group **Description:** Participants wore habitual DDSH Lens in both eyes for 6-8 hours (Period 1, observational). Afterwards, participants wore the Test Lens in one randomized eye that was kept closed and patched for 3 hours (Period 2, interventional). **ID:** FG000 **Title:** DDSH Lens Wearers #### Group **Description:** Participants wore habitual DDH Lens in both eyes for 6-8 hours (Period 1, observational). Afterwards, participants wore the Test Lens in one randomized eye that was kept closed and patched for 3 hours (Period 2, interventional). **ID:** FG001 **Title:** DDH Lens Wearers #### Group **Description:** Participants had no previous history of contact lens wear. Participants wore habitual spectacles, if any, for 6-8 hours (Period 1, observational). They did not receive the Test Lens and were not involved in Period 2. **ID:** FG002 **Title:** Non-lens Wearers #### Period **Title:** Period 1: Habitual Device, 6-8 Hours ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 10 **Number of Units:** 20 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 3 **Number of Units:** 6 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 9 **Number of Units:** 18 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 10 **Number of Units:** 20 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 3 **Number of Units:** 6 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 9 **Number of Units:** 18 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 **Number of Units:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 **Number of Units:** 0 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 0 **Number of Units:** 0 #### Period **Title:** Period 2: Test Lens, 3 Hours ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 10 **Number of Units:** 10 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 3 **Number of Units:** 3 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 0 **Number of Units:** 0 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 10 **Number of Units:** 10 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 3 **Number of Units:** 3 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 0 **Number of Units:** 0 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 **Number of Units:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 **Number of Units:** 0 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 0 **Number of Units:** 0 **Pre-Assignment Details:** Twenty-six participants were screened, with 3 of these not meeting all eligibility criteria and were considered screen failures. One participant withdrew for reasons unrelated to the study. These participants were not included in the total number of participants enrolled. **Type Units Analyzed:** Eyes [single] **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT03895879 **Acronym:** TODORA **Brief Title:** Use of Tocilizumab Drug Levels to Optimize Treatment in RA **Official Title:** Concentration-guided Dose Reduction Versus Standard Dosing in Tocilizumab-treated Rheumatoid Arthritis Patients: a Randomised, Multicenter, Non-inferiority Trial (TODORA) #### Organization Study ID Info **ID:** TODORA #### Organization **Class:** OTHER **Full Name:** Reade Rheumatology Research Institute #### Secondary ID Infos **ID:** 2018-004605-57 **Type:** EUDRACT_NUMBER **Domain:** Medical Ethical Committee VUmc, Amsterdam **ID:** NL68462.029.19 **Type:** OTHER ### Status Module #### Completion Date **Date:** 2024-12 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-04-18 **Type:** ACTUAL **Last Update Submit Date:** 2024-04-17 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-12 **Type:** ESTIMATED #### Start Date **Date:** 2020-03-01 **Type:** ACTUAL **Status Verified Date:** 2024-04 #### Study First Post Date **Date:** 2019-03-29 **Type:** ACTUAL **Study First Submit Date:** 2019-03-26 **Study First Submit QC Date:** 2019-03-28 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** ZonMw: The Netherlands Organisation for Health Research and Development #### Lead Sponsor **Class:** OTHER **Name:** Reade Rheumatology Research Institute #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Is US Export:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** Tocilizumab concentrations above 1 mg/L are likely to be sufficient for normalizing C-reactive protein (CRP) production in patients with rheumatoid arthritis (RA). In practice, however, a large variability in the concentrations of tocilizumab is found, and a large proportion of patients treated with tocilizumab subcutaneously (sc) have concentrations far above 1 mg/L. These patients can probably lower their doses without losing clinical response. A 52 weeks non-inferiority, multicenter, randomized controlled study will be performed to investigate whether patients with RA with serum trough concentrations of tocilizumab higher than 15 mg/L can increase their dosing interval to every two weeks without losing clinical response. Patients with relatively high trough concentrations will be randomly assigned to continuation of the standard dose or to increase dosing interval to every two weeks. The main objective is to investigate the difference in mean time weighted Disease Activity Score in 28 joints, including erythrocyte sedimentation rate (DAS28-ESR) between the two groups after 28 weeks. It is expected that patients with relatively high trough concentrations can safely increase their dosing interval without losing response. **Detailed Description:** Tocilizumab is a humanized monoclonal antibody targeting the IL-6 receptor (IL-6R). It has proven to be effective in reducing inflammation and symptoms in rheumatoid arthritis (RA). The registered standard dose of tocilizumab subcutaneously (sc) is 162 mg weekly for every patient. All patients diagnosed with RA and treated with tocilizumab sc receive the same dose, so treatment with expensive biologicals is currently based on a 'one size fits all' approach. Because of the large inter-individual variability in the pharmacokinetics of tocilizumab this standard dose results in a wide range of serum concentrations. In the search to optimize the dose for individual patients it was demonstrated that serum levels of 1 mg/L of tocilizumab are adequate to block the IL-6 receptor systemically, as indicated by a reduction in CRP levels in patients with these low trough concentrations. Therefore, a substantial proportion of patients is likely to be overexposed to tocilizumab. This overtreatment is a waste of health care resources and might be associated with an increased risk of adverse events, mainly infections. We believe that overexposure can be reduced effectively by making use of the drug concentrations found in the serum of individual patients. Our hypothesis is therefore that reducing the dose in the setting of therapeutic drug monitoring (TDM) does not affect clinical disease activity and safety, while it will reduce costs. Based on previous studies we believe that a concentration around 5 mg/L is sufficient to reach the maximal treatment effect. Therefore tapering strategy was developed aiming for serum concentrations around 5 mg/L. Monte Carlo modelling was performed to determine the cut-off concentration for interval prolongation to be used in this study. Simulations were performed and it was found that patients with trough concentrations above 15 mg/L can safely prolong their dosing interval, as this will result in levels around 5 mg/L in the majority of patients. This study is a 52 weeks randomised, multicenter, non-inferiority trial in rheumatoid arthritis patients treated with subcutaneous tocilizumab 162 mg weekly for at least the previous 6 months. After informed consent is obtained during the baseline visit, blood will be drawn to measure drug trough concentrations. Patients with a tocilizumab concentration above 15 mg/L will be randomly assigned to dose reduction by increasing their dosing-interval from once every week to once every two weeks, or to continuation of their tocilizumab dose (standard dose). After randomization, patients are followed for a period of 52 weeks. Data regarding disease status and functioning will be collected during the baseline visit, and 12, 28, 40, and 52 weeks thereafter. Blood will also be drawn from the patients during these visits. All patients with concentrations below 15 mg/L during the first study visit will not be randomized and all continue standard treatment. Only one follow-up visit, after 52 weeks, will be performed in this group of patients. Patients can also choose to participate in a sub-study where the finger prick developed by Sanquin (Amsterdam) will be validated to measure tocilizumab drug levels. This part of the study will comprise performing three finger pricks. These finger pricks will be performed during the visit at week 12 with the help of a nurse, and at home during the two weeks after this visit. ### Conditions Module **Conditions:** - Rheumatoid Arthritis **Keywords:** - Rheumatoid Arthritis - Tocilizumab - Therapeutic Drug Monitoring - Drug level - Dose reduction ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** SEQUENTIAL **Intervention Model Description:** Patients with tocilizumab trough concentrations above 15 mg/L will be randomly assigned to dose reduction by increasing their dosing interval from once every week to once every two weeks, or to continuation of the standard dose. All patients with concentrations below 15 mg/L during the first study visit will not be randomized and all continue standard treatment. ##### Masking Info **Masking:** SINGLE **Masking Description:** During every study visit the joints of all patients will be examined for pain and swelling by a blinded nurse or physician. The number of painful and swollen joints will be used to calculate the DAS28 score, the primary outcome of the study. **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 98 **Type:** ESTIMATED **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Tocilizumab administered every 2 weeks **Intervention Names:** - Drug: Tocilizumab **Label:** Intervention **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Tocilizumab administered every week **Intervention Names:** - Drug: Tocilizumab **Label:** Control **Type:** ACTIVE_COMPARATOR #### Arm Group 3 **Description:** Tocilizumab administered every week **Intervention Names:** - Drug: Tocilizumab **Label:** Standard dose (screening < 15 mg/L) **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Intervention **Description:** Tocilizumab sc (162 mg) once every 2 weeks **Name:** Tocilizumab **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Control - Standard dose (screening < 15 mg/L) **Description:** Tocilizumab sc (162 mg) once every week **Name:** Tocilizumab **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** The difference in mean time weighted DAS28 after 28 weeks between patients undergoing concentration-guided dose reduction or standard dosing. **Measure:** DAS28-ESR **Time Frame:** 28 weeks #### Secondary Outcomes **Description:** The difference in mean time weighted DAS28 after 52 weeks between patients undergoing concentration-guided dose reduction or standard dosing. **Measure:** DAS28-ESR **Time Frame:** 52 weeks **Description:** The difference in CDAI-score after 28 and 52 weeks between the patients undergoing concentration-guided dose reduction or standard dosing. **Measure:** Clinical Disease Activity Index (CDAI) **Time Frame:** 28 and 52 weeks **Description:** The difference in SDAI-score after 28 and 52 weeks between the patients undergoing concentration-guided dose reduction or standard dosing. **Measure:** Simple Disease Activity Index (SDAI) **Time Frame:** 28 and 52 weeks **Description:** The difference in HAQ-score after 28 and 52 weeks between the patients undergoing concentration-guided dose reduction or standard dosing. **Measure:** Health Assessment Questionnaire (HAQ) **Time Frame:** 28 and 52 weeks **Description:** The difference in direct medical costs of TDM compared to the standard treatment regimen. **Measure:** Direct medical costs of TDM **Time Frame:** 52 weeks **Description:** The difference in number of flares at 28 and 52 weeks between patients undergoing concentration-guided dose reduction or standard dosing. **Measure:** Number of flares **Time Frame:** 28 and 52 weeks **Description:** The difference in number and severity of adverse events at 28 and 52 weeks between patients undergoing concentration-guided dose reduction or standard dosing. **Measure:** Number and severity of adverse events **Time Frame:** 28 and 52 weeks **Description:** The difference in drug levels in the intervention group between week 0 and 52. **Measure:** Drug level **Time Frame:** 52 weeks **Description:** A questionnaire will be used to evaluate the perspective of patients towards therapeutic drug monitoring. **Measure:** Patient perspective towards therapeutic drug monitoring **Time Frame:** 52 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Rheumatoid arthritis according to the American College of Rheumatology (ACR) 1987 or 2010 criteria; * Current use of subcutaneous tocilizumab 162 mg weekly, for at least the previous 6 months; * The treating rheumatologist is convinced of the benefit of tocilizumab continuation; * Written informed consent. Exclusion Criteria: * A scheduled surgery in the next 52 weeks or other pre-planned reasons for treatment discontinuation; * Changes in the treatment with glucocorticoids and DMARDs such as methotrexate in the past three months. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** f.hooijberg@reade.nl **Name:** Femke Hooijberg, MD **Phone:** 0031 20 2421633 **Role:** CONTACT **Contact 2:** **Email:** s.atiqi@reade.nl **Name:** Sadaf Atiqi, MD **Phone:** 0031 20 2421641 **Role:** CONTACT #### Locations **Location 1:** **City:** Amsterdam **Contacts:** ***Contact 1:*** - **Email:** f.hooijberg@reade.nl - **Name:** Femke Hooijberg, MD - **Phone:** 0031 20 2421633 - **Role:** CONTACT **Country:** Netherlands **Facility:** Reade Rheumatology Research Institute **Status:** RECRUITING **Zip:** 1056 AB #### Overall Officials **Official 1:** **Affiliation:** Reade Rheumatology Research Institute **Name:** Gertjan Wolbink, MD, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Access Criteria:** Researchers with demonstrable interest in autoimmunity, biologicals, or TDM can contact the investigators of the trial if they are interested in gaining access to the data. Depending on their research objectives the data will be shared. **Description:** To avoid duplication of research, the data gathered in this study will be shared once all desirable data analysis have been performed and the results are published. **Info Types:** - STUDY_PROTOCOL - SAP **IPD Sharing:** YES **Time Frame:** Six months after the final publication from the study is published the data will be shared. ### References Module #### References **Citation:** Kneepkens EL, van den Oever I, Plasencia CH, Pascual-Salcedo D, de Vries A, Hart M, Nurmohamed MT, Balsa A, Rispens T, Wolbink G. Serum tocilizumab trough concentration can be used to monitor systemic IL-6 receptor blockade in patients with rheumatoid arthritis: a prospective observational cohort study. Scand J Rheumatol. 2017 Mar;46(2):87-94. doi: 10.1080/03009742.2016.1183039. Epub 2016 Jul 20. **PMID:** 27440258 **Citation:** l'Ami MJ, Krieckaert CL, Nurmohamed MT, van Vollenhoven RF, Rispens T, Boers M, Wolbink GJ. Successful reduction of overexposure in patients with rheumatoid arthritis with high serum adalimumab concentrations: an open-label, non-inferiority, randomised clinical trial. Ann Rheum Dis. 2018 Apr;77(4):484-487. doi: 10.1136/annrheumdis-2017-211781. Epub 2017 Sep 22. **PMID:** 28939629 **Citation:** Frey N, Grange S, Woodworth T. Population pharmacokinetic analysis of tocilizumab in patients with rheumatoid arthritis. J Clin Pharmacol. 2010 Jul;50(7):754-66. doi: 10.1177/0091270009350623. Epub 2010 Jan 23. **PMID:** 20097931 **Citation:** Bastida C, Ruiz-Esquide V, Pascal M, de Vries Schultink AHM, Yague J, Sanmarti R, Huitema ADR, Soy D. Fixed dosing of intravenous tocilizumab in rheumatoid arthritis. Results from a population pharmacokinetic analysis. Br J Clin Pharmacol. 2018 Apr;84(4):716-725. doi: 10.1111/bcp.13500. Epub 2018 Feb 7. **PMID:** 29314183 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007592 - Term: Joint Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000012216 - Term: Rheumatic Diseases - ID: D000003240 - Term: Connective Tissue Diseases - ID: D000001327 - Term: Autoimmune Diseases - ID: D000007154 - Term: Immune System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC20 - Name: Immune System Diseases ### Condition Browse Module - Browse Leaves - ID: M4476 - Name: Arthritis - Relevance: HIGH - As Found: Arthritis - ID: M4480 - Name: Arthritis, Rheumatoid - Relevance: HIGH - As Found: Rheumatoid Arthritis - ID: M10621 - Name: Joint Diseases - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: M15045 - Name: Rheumatic Diseases - Relevance: LOW - As Found: Unknown - ID: M6323 - Name: Collagen Diseases - Relevance: LOW - As Found: Unknown - ID: M6464 - Name: Connective Tissue Diseases - Relevance: LOW - As Found: Unknown - ID: M4629 - Name: Autoimmune Diseases - Relevance: LOW - As Found: Unknown - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001168 - Term: Arthritis - ID: D000001172 - Term: Arthritis, Rheumatoid ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00088179 **Brief Title:** Pexelizumab in Patients Undergoing Coronary Artery Bypass Grafting With Cardiopulmonary Bypass (PRIMO-CABG II) **Official Title:** A Multicenter, Randomized, Double-Blind, Parallel-group, Placebo-Controlled Study of Pexelizumab in Patients Undergoing Coronary Artery Bypass Grafting With Cardiopulmonary Bypass (PRIMO-CABG II) #### Organization Study ID Info **ID:** 2003141 #### Organization **Class:** INDUSTRY **Full Name:** Alexion Pharmaceuticals, Inc. ### Status Module #### Completion Date **Date:** 2005-10 **Type:** ACTUAL #### Disp First Post Date **Date:** 2017-02-16 **Type:** ACTUAL **Disp First Submit Date:** 2017-02-15 **Disp First Submit QC Date:** 2017-02-15 #### Last Update Post Date **Date:** 2018-02-22 **Type:** ACTUAL **Last Update Submit Date:** 2018-02-20 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2005-10 **Type:** ACTUAL #### Start Date **Date:** 2004-07 **Type:** ACTUAL **Status Verified Date:** 2018-02 #### Study First Post Date **Date:** 2004-07-23 **Type:** ESTIMATED **Study First Submit Date:** 2004-07-21 **Study First Submit QC Date:** 2004-07-22 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Procter and Gamble #### Lead Sponsor **Class:** INDUSTRY **Name:** Alexion Pharmaceuticals, Inc. ### Description Module **Brief Summary:** During a heart bypass procedure, a substance called "complement" is activated by the body. This "complement activation" causes an inflammatory response that can lead to side affects such as chest pain, heart attacks, stroke, heart failure, or death. The purpose of this study is to find out if the study drug (pexelizumab), that blocks "complement activation," can reduce such side effects and be given safely to patients requiring the bypass procedure with the use of the heart-lung machine. ### Conditions Module **Conditions:** - Coronary Artery Disease **Keywords:** - CABG - CPB ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 4000 **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Name:** pexelizumab in conjunction with CABG **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Reduction in mortality. **Measure:** Reduction in MI incidence. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * be at least 18 years of age; * have 2 or more of the following risk factors: • diabetes mellitus; • repeat CABG; • the need for urgent intervention, defined according to the ACC/AHA guidelines as being patients who are required to stay in the hospital due to medical factors, but may be scheduled and operated on within a normal scheduling routine, excluding patients who have had an MI within 48 hours of CABG; • female; • history of a neurologic event (cerebrovascular accident, transient ischemic attack or carotid endarterectomy); • history of congestive heart failure (NYHA CHF Class III or IV); • history of 2 MIs, or an MI that occurred greater than 48 hours but less than 4 weeks prior to CABG; * provide Informed Consent. Exclusion Criteria: * requires salvage intervention as defined by the ACC/AHA guidelines 10 as being ongoing cardiopulmonary resuscitation on the way to the operating room; * has current cardiogenic shock, acute left ventricular rupture, acute septal rupture or acute papillary muscle rupture; * has any active bacterial or other infection which is clinically significant, in the opinion of the Investigator (e.g. evaluate the evidence based on WBC, temperature, cultures etc. as appropriate for the patient); * has a known or suspected hereditary complement deficiency; * has participated in any other investigational drug study or was exposed to an investigational agent or device within 30 days of randomization; * is receiving, or is planning to receive, any other investigational drug or device, or will participate in any other research study within 30 days of randomization; * is pregnant or breast-feeding. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Birmingham **Country:** United States **Facility:** Brookwood Medical Center **State:** Alabama **Zip:** 35209 **Location 2:** **City:** Birmingham **Country:** United States **Facility:** Baptist Medical Center Princeton **State:** Alabama **Zip:** 35211 **Location 3:** **City:** Birmingham **Country:** United States **Facility:** Baptist Health Systems Montclair **State:** Alabama **Zip:** 35213 **Location 4:** **City:** Birmingham **Country:** United States **Facility:** Medical Center East Hospital **State:** Alabama **Zip:** 35235 **Location 5:** **City:** Birmingham **Country:** United States **Facility:** University of Alabama at Birmingham Hospital **State:** Alabama **Zip:** 35249-6810 **Location 6:** **City:** Fairhope **Country:** United States **Facility:** Thomas Hospital **State:** Alabama **Zip:** 36532 **Location 7:** **City:** Huntsville **Country:** United States **Facility:** Huntsville Hospital **State:** Alabama **Zip:** 35801 **Location 8:** **City:** Mobile **Country:** United States **Facility:** Mobile Infirmary Medical Center **State:** Alabama **Zip:** 36607 **Location 9:** **City:** Phoenix **Country:** United States **Facility:** Banner Good Samaritan Medical Center **State:** Arizona **Zip:** 85006 **Location 10:** **City:** Tucson **Country:** United States **Facility:** Arizona Health Sciences Center **State:** Arizona **Zip:** 85724 **Location 11:** **City:** Tucson **Country:** United States **Facility:** University Medical Center **State:** Arizona **Zip:** 85724 **Location 12:** **City:** Tucson **Country:** United States **Facility:** Southern Arizona Veterans Affairs Health Care System **State:** Arizona **Zip:** 85732 **Location 13:** **City:** Fort Smith **Country:** United States **Facility:** Sparks Regional Medical Center **State:** Arkansas **Zip:** 72901 **Location 14:** **City:** Little Rock **Country:** United States **Facility:** Baptist Medical Center **State:** Arkansas **Zip:** 72205 **Location 15:** **City:** Bakersfield **Country:** United States **Facility:** Bakersfield Heart Hospital **State:** California **Zip:** 93301 **Location 16:** **City:** Daly City **Country:** United States **Facility:** Seaton Medical Center **State:** California **Zip:** 94015 **Location 17:** **City:** Escondido **Country:** United States **Facility:** Palomar Medical Center **State:** California **Zip:** 92025 **Location 18:** **City:** Laguna Hills **Country:** United States **Facility:** Saddleback Memorial Medical Center **State:** California **Zip:** 92653 **Location 19:** **City:** Los Angeles **Country:** United States **Facility:** Los Angeles County-University of Southern California Medical Center **State:** California **Zip:** 90033 **Location 20:** **City:** Los Angeles **Country:** United States **Facility:** University of Southern California University Hospital **State:** California **Zip:** 90033 **Location 21:** **City:** Los Angeles **Country:** United States **Facility:** St. Vincent Medical Center **State:** California **Zip:** 90057 **Location 22:** **City:** Los Angeles **Country:** United States **Facility:** Veterans Administration Greater Los Angeles Healthcare Sysytem **State:** California **Zip:** 90073 **Location 23:** **City:** Oceanside **Country:** United States **Facility:** Tri City Medical Center **State:** California **Zip:** 92056 **Location 24:** **City:** Orange **Country:** United States **Facility:** University of California Irvine Medical Center **State:** California **Zip:** 92868 **Location 25:** **City:** Palo Alto **Country:** United States **Facility:** Veterans Administration Palo Alto Healthcare System **State:** California **Zip:** 94304 **Location 26:** **City:** Pasadena **Country:** United States **Facility:** Huntington Memorial Hospital **State:** California **Zip:** 91105 **Location 27:** **City:** Sacramento **Country:** United States **Facility:** Mercy General Hospital **State:** California **Zip:** 95819 **Location 28:** **City:** San Diego **Country:** United States **Facility:** Sharp Memorial Hospital **State:** California **Zip:** 92123 **Location 29:** **City:** San Francisco **Country:** United States **Facility:** Kaiser Permanente Medical Center **State:** California **Zip:** 94115 **Location 30:** **City:** Denver **Country:** United States **Facility:** Porter Adventist Hospital **State:** Colorado **Zip:** 80210 **Location 31:** **City:** Denver **Country:** United States **Facility:** Presbyterian St.Luke's Medical Center **State:** Colorado **Zip:** 80218 **Location 32:** **City:** Denver **Country:** United States **Facility:** University of Colorado Health Sciences Center **State:** Colorado **Zip:** 80262 **Location 33:** **City:** Bridgeport **Country:** United States **Facility:** St. Vincent's Medical Center **State:** Connecticut **Zip:** 06606 **Location 34:** **City:** New Haven **Country:** United States **Facility:** Hospital of Saint Raphael **State:** Connecticut **Zip:** 06511 **Location 35:** **City:** Newark **Country:** United States **Facility:** Christiana Hospital **State:** Delaware **Zip:** 19718 **Location 36:** **City:** Atlantis **Country:** United States **Facility:** John F. Kennedy Medical Center **State:** Florida **Zip:** 33462 **Location 37:** **City:** Clearwater **Country:** United States **Facility:** Morton Plant Hospital **State:** Florida **Zip:** 33756 **Location 38:** **City:** Daytona Beach **Country:** United States **Facility:** Halifax Medical Center **State:** Florida **Zip:** 32114 **Location 39:** **City:** Fort Lauderdale **Country:** United States **Facility:** North Ridge Medical Center **State:** Florida **Zip:** 33334 **Location 40:** **City:** Gainesville **Country:** United States **Facility:** Shands Hospital at the University of Florida **State:** Florida **Zip:** 32610 **Location 41:** **City:** Hudson **Country:** United States **Facility:** Regional Medical Center Bayonet Point **State:** Florida **Zip:** 34667 **Location 42:** **City:** Jacksonville **Country:** United States **Facility:** Baptist Medical Center **State:** Florida **Zip:** 32207 **Location 43:** **City:** Jacksonville **Country:** United States **Facility:** Memorial Hospital **State:** Florida **Zip:** 32216 **Location 44:** **City:** Lakeland **Country:** United States **Facility:** Lakeland Regional Medical Center **State:** Florida **Zip:** 33805 **Location 45:** **City:** Melbourne **Country:** United States **Facility:** Health First Holmes Regional Medical Center **State:** Florida **Zip:** 32901 **Location 46:** **City:** Orlando **Country:** United States **Facility:** Orlando Regional Medical Center **State:** Florida **Zip:** 32801 **Location 47:** **City:** Orlando **Country:** United States **Facility:** Florida Hospital **State:** Florida **Zip:** 32803 **Location 48:** **City:** Pensacola **Country:** United States **Facility:** Sacred Heart Hospital **State:** Florida **Zip:** 32504 **Location 49:** **City:** Rockledge **Country:** United States **Facility:** Wuesthoff Hospital **State:** Florida **Zip:** 32955 **Location 50:** **City:** Saint Petersburg **Country:** United States **Facility:** Northside Hospital and Heart Institute **State:** Florida **Zip:** 33701 **Location 51:** **City:** Sarasota **Country:** United States **Facility:** Sarasota Memorial Hospital **State:** Florida **Zip:** 34239 **Location 52:** **City:** Tampa **Country:** United States **Facility:** University Community Hospital **State:** Florida **Zip:** 33613 **Location 53:** **City:** Atlanta **Country:** United States **Facility:** Piedmont Hospital **State:** Georgia **Zip:** 30309 **Location 54:** **City:** Atlanta **Country:** United States **Facility:** Emory University Hospital **State:** Georgia **Zip:** 30322 **Location 55:** **City:** Decatur **Country:** United States **Facility:** Atlanta Veteran's Administration (VA) Medical Center **State:** Georgia **Zip:** 30033 **Location 56:** **City:** Macon **Country:** United States **Facility:** Medical Center of Central Georgia **State:** Georgia **Zip:** 31208 **Location 57:** **City:** Savannah **Country:** United States **Facility:** St. Joseph's/Candler Health System **State:** Georgia **Zip:** 31404 **Location 58:** **City:** Savannah **Country:** United States **Facility:** St. Joseph's/Candler Health System **State:** Georgia **Zip:** 31419 **Location 59:** **City:** Honolulu **Country:** United States **Facility:** Kaiser Permanente Moanalua Medical Center **State:** Hawaii **Zip:** 96819 **Location 60:** **City:** Peoria **Country:** United States **Facility:** OSF Saint Francis Medical Center **State:** Illinois **Zip:** 61637 **Location 61:** **City:** Springfield **Country:** United States **Facility:** Memorial Medical Center **State:** Illinois **Zip:** 62702 **Location 62:** **City:** Springfield **Country:** United States **Facility:** St. John's Hospital **State:** Illinois **Zip:** 62769 **Location 63:** **City:** Fort Wayne **Country:** United States **Facility:** Indiana Ohio Heart **State:** Indiana **Zip:** 46804 **Location 64:** **City:** Indianapolis **Country:** United States **Facility:** Clarian Health Partners, Inc. Methodist Hospital **State:** Indiana **Zip:** 46202 **Location 65:** **City:** Indianapolis **Country:** United States **Facility:** The Indiana Heart Hospital **State:** Indiana **Zip:** 46250 **Location 66:** **City:** Indianapolis **Country:** United States **Facility:** The Heart Center of Indiana **State:** Indiana **Zip:** 46290 **Location 67:** **City:** Des Moines **Country:** United States **Facility:** Iowa Heart Center **State:** Iowa **Zip:** 50314 **Location 68:** **City:** Olathe **Country:** United States **Facility:** Olathe Medical Center **State:** Kansas **Zip:** 66061 **Location 69:** **City:** Wichita **Country:** United States **Facility:** Galichia Heart Hospital **State:** Kansas **Zip:** 67202 **Location 70:** **City:** Lexington **Country:** United States **Facility:** Central Baptist Hospital **State:** Kentucky **Zip:** 40503 **Location 71:** **City:** Lexington **Country:** United States **Facility:** Veterans Affairs Medical Center **State:** Kentucky **Zip:** 40506 **Location 72:** **City:** Lexington **Country:** United States **Facility:** University of Kentucky **State:** Kentucky **Zip:** 40536 **Location 73:** **City:** Louisville **Country:** United States **Facility:** Jewish Hospital **State:** Kentucky **Zip:** 40202 **Location 74:** **City:** Louisville **Country:** United States **Facility:** Norton Hospital **State:** Kentucky **Zip:** 40202 **Location 75:** **City:** Louisville **Country:** United States **Facility:** University of Louisville Hospital **State:** Kentucky **Zip:** 40202 **Location 76:** **City:** Houma **Country:** United States **Facility:** Terrebonne General Medical Center **State:** Louisiana **Zip:** 70360 **Location 77:** **City:** Lacombe **Country:** United States **Facility:** Louisiana Heart Hospital **State:** Louisiana **Zip:** 70445 **Location 78:** **City:** New Orleans **Country:** United States **Facility:** Tulane University Hospital and Clinic **State:** Louisiana **Zip:** 70112 **Location 79:** **City:** New Orleans **Country:** United States **Facility:** Oschner Clinic Foundation **State:** Louisiana **Zip:** 70121 **Location 80:** **City:** New Orleans **Country:** United States **Facility:** Pendleton Memorial Methodist Hospital **State:** Louisiana **Zip:** 70127 **Location 81:** **City:** New Orleans **Country:** United States **Facility:** Touro Infirmary **State:** Louisiana **Location 82:** **City:** Slidell **Country:** United States **Facility:** Slidell Memorial Hospital **State:** Louisiana **Zip:** 70458 **Location 83:** **City:** Portland **Country:** United States **Facility:** Maine Medical Center **State:** Maine **Zip:** 04102 **Location 84:** **City:** Baltimore **Country:** United States **Facility:** Sinai Hospital of Baltimore **State:** Maryland **Zip:** 21215 **Location 85:** **City:** Baltimore **Country:** United States **Facility:** Union Memorial Hospital **State:** Maryland **Zip:** 21218 **Location 86:** **City:** Salisbury **Country:** United States **Facility:** Peninusla Medical Center **State:** Maryland **Zip:** 21801 **Location 87:** **City:** Towson **Country:** United States **Facility:** St. Joseph Medical Center **State:** Maryland **Zip:** 21204 **Location 88:** **City:** Boston **Country:** United States **Facility:** Massachusetts General Hospital **State:** Massachusetts **Zip:** 02114 **Location 89:** **City:** Boston **Country:** United States **Facility:** Beth Israel Deaconess Medical Center **State:** Massachusetts **Zip:** 02215 **Location 90:** **City:** Hyannis **Country:** United States **Facility:** Cape Cod Hospital **State:** Massachusetts **Zip:** 02601 **Location 91:** **City:** Springfield **Country:** United States **Facility:** Baystate Medical Center **State:** Massachusetts **Zip:** 01199 **Location 92:** **City:** West Roxbury **Country:** United States **Facility:** Veterans Administration (VA) Boston Healthcare System **State:** Massachusetts **Zip:** 02132 **Location 93:** **City:** Detroit **Country:** United States **Facility:** Henry Ford Health System **State:** Michigan **Zip:** 48202 **Location 94:** **City:** Flint **Country:** United States **Facility:** McLaren Regional Medical Center **State:** Michigan **Zip:** 48532 **Location 95:** **City:** Grand Rapids **Country:** United States **Facility:** Spectrum Health Hospitals **State:** Michigan **Zip:** 49503 **Location 96:** **City:** Kalamazoo **Country:** United States **Facility:** Borgess Medical Center **State:** Michigan **Zip:** 49048 **Location 97:** **City:** Lansing **Country:** United States **Facility:** Ingham Regional Medical Center **State:** Michigan **Zip:** 48910 **Location 98:** **City:** Muskegon **Country:** United States **Facility:** West Shore Cardiology Research Department **State:** Michigan **Zip:** 46444 **Location 99:** **City:** Royal Oak **Country:** United States **Facility:** William Beaumont Hospital **State:** Michigan **Zip:** 48073 **Location 100:** **City:** Saginaw **Country:** United States **Facility:** Covenant Medical Center **State:** Michigan **Zip:** 48601 **Location 101:** **City:** Rochester **Country:** United States **Facility:** Mayo Clinic College of Medicine **State:** Minnesota **Zip:** 55905 **Location 102:** **City:** Kansas City **Country:** United States **Facility:** St. Luke's Health System **State:** Missouri **Zip:** 64111 **Location 103:** **City:** Saint Louis **Country:** United States **Facility:** Saint Louis University Hospital **State:** Missouri **Zip:** 63110 **Location 104:** **City:** Saint Louis **Country:** United States **Facility:** Saint Louis University **State:** Missouri **Zip:** 63110 **Location 105:** **City:** Saint Louis **Country:** United States **Facility:** Des Peres Hospital **State:** Missouri **Zip:** 63122 **Location 106:** **City:** Lincoln **Country:** United States **Facility:** Nebraska Heart Hospital **State:** Nebraska **Zip:** 68506 **Location 107:** **City:** Camden **Country:** United States **Facility:** Cooper Hospital University Medical Center **State:** New Jersey **Zip:** 08035 **Location 108:** **City:** Camden **Country:** United States **Facility:** Cooper Health System/University Medical Center **State:** New Jersey **Zip:** 08103 **Location 109:** **City:** Camden **Country:** United States **Facility:** Our Lady Of Lourdes Medical Center **State:** New Jersey **Zip:** 08103 **Location 110:** **City:** Hackensack **Country:** United States **Facility:** Hackensack University Medical Center **State:** New Jersey **Zip:** 07601 **Location 111:** **City:** Morristown **Country:** United States **Facility:** Morristown Memorial Hospital **State:** New Jersey **Zip:** 07962-1956 **Location 112:** **City:** Bronx **Country:** United States **Facility:** Montefiore Medical Center **State:** New York **Zip:** 10461 **Location 113:** **City:** Brooklyn **Country:** United States **Facility:** Maimondes Medical Center **State:** New York **Zip:** 11219 **Location 114:** **City:** Johnson City **Country:** United States **Facility:** United Health Services Hospital **State:** New York **Zip:** 13790 **Location 115:** **City:** New York **Country:** United States **Facility:** New York Presbyterian Hospital **State:** New York **Zip:** 10021 **Location 116:** **City:** New York **Country:** United States **Facility:** Mount Sinai Hospital **State:** New York **Zip:** 10029 **Location 117:** **City:** Poughkeepsie **Country:** United States **Facility:** Vassar Brothers Medical Center **State:** New York **Zip:** 12601 **Location 118:** **City:** Rochester **Country:** United States **Facility:** Rochester General Hospital **State:** New York **Zip:** 14621 **Location 119:** **City:** Rochester **Country:** United States **Facility:** University of Rochester Medical Center **State:** New York **Zip:** 14642 **Location 120:** **City:** Valhalla **Country:** United States **Facility:** Westchester Medical Center **State:** New York **Zip:** 10595 **Location 121:** **City:** Chapel Hill **Country:** United States **Facility:** University of North Carolina at Chapel Hill Hospitals **State:** North Carolina **Zip:** 27599-7075 **Location 122:** **City:** Durham **Country:** United States **Facility:** Duke University Medical Center **State:** North Carolina **Zip:** 27710 **Location 123:** **City:** Greensboro **Country:** United States **Facility:** Moses H. Cone Memorial Hospital **State:** North Carolina **Zip:** 27401 **Location 124:** **City:** Greenville **Country:** United States **Facility:** Pitt County Memorial Hospital **State:** North Carolina **Zip:** 27858 **Location 125:** **City:** Bismarck **Country:** United States **Facility:** Medcenter One **State:** North Dakota **Zip:** 58501 **Location 126:** **City:** Akron **Country:** United States **Facility:** Akron General Medical Center **State:** Ohio **Zip:** 44307 **Location 127:** **City:** Cincinnati **Country:** United States **Facility:** The Christ Hospital **State:** Ohio **Zip:** 45219 **Location 128:** **City:** Cincinnati **Country:** United States **Facility:** Good Samaritan Hospital **State:** Ohio **Zip:** 45220 **Location 129:** **City:** Cincinnati **Country:** United States **Facility:** Bethesda North Hospital **State:** Ohio **Zip:** 45242 **Location 130:** **City:** Cincinnati **Country:** United States **Facility:** University of Cincinnati Medical Center **State:** Ohio **Zip:** 45267-0542 **Location 131:** **City:** Cleveland **Country:** United States **Facility:** University Hospitals Health System **State:** Ohio **Zip:** 44106-5011 **Location 132:** **City:** Cleveland **Country:** United States **Facility:** The Cleveland Clinic Foundation **State:** Ohio **Zip:** 44195 **Location 133:** **City:** Columbus **Country:** United States **Facility:** Riverside Methodist Hospital **State:** Ohio **Zip:** 43214 **Location 134:** **City:** Dayton **Country:** United States **Facility:** The Dayton Heart Hospital **State:** Ohio **Zip:** 45408 **Location 135:** **City:** Toledo **Country:** United States **Facility:** The Toledo Hospital **State:** Ohio **Zip:** 43606 **Location 136:** **City:** Zanesville **Country:** United States **Facility:** Good Samaritan Hospital **State:** Ohio **Zip:** 43701 **Location 137:** **City:** Oklahoma City **Country:** United States **Facility:** Oklahoma University Medical Center **State:** Oklahoma **Zip:** 73104 **Location 138:** **City:** Oklahoma City **Country:** United States **Facility:** INTEGRIS Baptist Medical Center **State:** Oklahoma **Zip:** 73112 **Location 139:** **City:** Oklahoma City **Country:** United States **Facility:** Oklahoma Heart Hospital **State:** Oklahoma **Zip:** 73120 **Location 140:** **City:** Bend **Country:** United States **Facility:** St. Charles Medical Center **State:** Oregon **Zip:** 97701 **Location 141:** **City:** Portland **Country:** United States **Facility:** Legacy Good Samaritan Hospital **State:** Oregon **Zip:** 97210 **Location 142:** **City:** Doylestown **Country:** United States **Facility:** Doylestown Hospital **State:** Pennsylvania **Zip:** 18901 **Location 143:** **City:** Langhorne **Country:** United States **Facility:** St. Mary Medical Center **State:** Pennsylvania **Zip:** 19067 **Location 144:** **City:** Philadelphia **Country:** United States **Facility:** Hahnemann University Hospital **State:** Pennsylvania **Zip:** 19102 **Location 145:** **City:** Philadelphia **Country:** United States **Facility:** Presbyterian Medical Center **State:** Pennsylvania **Zip:** 19104 **Location 146:** **City:** Philadelphia **Country:** United States **Facility:** Temple University Hospital **State:** Pennsylvania **Zip:** 19140 **Location 147:** **City:** Pittsburgh **Country:** United States **Facility:** Allegheny General Hospital **State:** Pennsylvania **Zip:** 15212 **Location 148:** **City:** Pittsburgh **Country:** United States **Facility:** University of Pittsburgh Medical Center **State:** Pennsylvania **Zip:** 15213 **Location 149:** **City:** Upland **Country:** United States **Facility:** Crozer Chester Medical Center **State:** Pennsylvania **Zip:** 19013 **Location 150:** **City:** West Chester **Country:** United States **Facility:** The Chester County Hospital **State:** Pennsylvania **Zip:** 19380 **Location 151:** **City:** Charleston **Country:** United States **Facility:** Roper St. Francis Healthcare **State:** South Carolina **Zip:** 29401 **Location 152:** **City:** Rapid City **Country:** United States **Facility:** Rapid City Regional Hospital **State:** South Dakota **Zip:** 57701 **Location 153:** **City:** Chattanooga **Country:** United States **Facility:** The Chattanooga Heart Institute **State:** Tennessee **Zip:** 37404 **Location 154:** **City:** Jackson **Country:** United States **Facility:** Jackson-Madison County General Hospital **State:** Tennessee **Zip:** 38301 **Location 155:** **City:** Memphis **Country:** United States **Facility:** Baptist Memorial Hospital **State:** Tennessee **Zip:** 38120 **Location 156:** **City:** Nashville **Country:** United States **Facility:** Baptist Hospital **State:** Tennessee **Zip:** 37203 **Location 157:** **City:** Nashville **Country:** United States **Facility:** Vanderbilt University Medical Center **State:** Tennessee **Zip:** 37232-8815 **Location 158:** **City:** Oak Ridge **Country:** United States **Facility:** Methodist Medical Center **State:** Tennessee **Zip:** 37830 **Location 159:** **City:** Austin **Country:** United States **Facility:** Heart Hospital of Austin **State:** Texas **Zip:** 78756 **Location 160:** **City:** Dallas **Country:** United States **Facility:** Medical City Dallas Hospital **State:** Texas **Zip:** 75230 **Location 161:** **City:** Houston **Country:** United States **Facility:** Christus Saint Joseph Hospital **State:** Texas **Zip:** 77002 **Location 162:** **City:** Houston **Country:** United States **Facility:** Michael E. DeBakey Veterans Affairs Medical Center **State:** Texas **Zip:** 77030-4298 **Location 163:** **City:** Houston **Country:** United States **Facility:** Memorial Hermann Hospital **State:** Texas **Zip:** 77030 **Location 164:** **City:** Houston **Country:** United States **Facility:** Methodist Hospital **State:** Texas **Zip:** 77030 **Location 165:** **City:** Houston **Country:** United States **Facility:** St. Luke's Episcopal Hospital - Texas Heart **State:** Texas **Zip:** 77030 **Location 166:** **City:** Houston **Country:** United States **Facility:** The Methodist Hospital **State:** Texas **Zip:** 77030 **Location 167:** **City:** Lubbock **Country:** United States **Facility:** Covenant Medical Center **State:** Texas **Zip:** 79410 **Location 168:** **City:** San Antonio **Country:** United States **Facility:** TexSan Heart Hospital **State:** Texas **Zip:** 78201 **Location 169:** **City:** San Antonio **Country:** United States **Facility:** Northeast Baptist Hospital **State:** Texas **Zip:** 78217 **Location 170:** **City:** Salt Lake City **Country:** United States **Facility:** University of Utah Hospital **State:** Utah **Zip:** 84132 **Location 171:** **City:** Arlington **Country:** United States **Facility:** Virginia Hospital Center **State:** Virginia **Zip:** 22205 **Location 172:** **City:** Charlottesville **Country:** United States **Facility:** University of Virginia Health System **State:** Virginia **Zip:** 22908 **Location 173:** **City:** Falls Church **Country:** United States **Facility:** Inova Fairfax Hospital **State:** Virginia **Zip:** 22042-3300 **Location 174:** **City:** Mechanicsville **Country:** United States **Facility:** Memorial Regional Medical Center **State:** Virginia **Zip:** 23116 **Location 175:** **City:** Norfolk **Country:** United States **Facility:** Sentara Norfolk General Hospital **State:** Virginia **Zip:** 23507 **Location 176:** **City:** Richmond **Country:** United States **Facility:** CJW Medical Center **State:** Virginia **Zip:** 23225 **Location 177:** **City:** Richmond **Country:** United States **Facility:** Henrico Doctors' Hospital **State:** Virginia **Zip:** 23229 **Location 178:** **City:** Seattle **Country:** United States **Facility:** University of Washington Medical Center **State:** Washington **Zip:** 98195-6310 **Location 179:** **City:** Tacoma **Country:** United States **Facility:** Saint Joseph Medical Center **State:** Washington **Zip:** 98405 **Location 180:** **City:** Charleston **Country:** United States **Facility:** Charleston Area Medical Center **State:** West Virginia **Zip:** 25304 **Location 181:** **City:** La Crosse **Country:** United States **Facility:** Gundersen Lutheran Medical Center **State:** Wisconsin **Zip:** 54601 **Location 182:** **City:** Milwaukee **Country:** United States **Facility:** St. Luke's Medical Center **State:** Wisconsin **Zip:** 53215 **Location 183:** **City:** Besancon **Country:** France **Facility:** Hopital Minjoz **Zip:** 25030 **Location 184:** **City:** Dijon **Country:** France **Facility:** Hopital du Bocage **Zip:** 21031 **Location 185:** **City:** Grenoble **Country:** France **Facility:** Hopital Albert Michallon **Zip:** 38043 **Location 186:** **City:** Paris **Country:** France **Facility:** Hopital Europeen Georges Pompidou **Zip:** 75908 **Location 187:** **City:** Erlangen **Country:** Germany **Facility:** Zentrum fuer Herzchirurgie der Frederick-Alexander-Universitaet **Zip:** 91054 **Location 188:** **City:** Leiden **Country:** Netherlands **Facility:** Leiden University Medical Center **Zip:** 2333 #### Overall Officials **Official 1:** **Affiliation:** Alexion Pharmaceuticals, Inc. **Name:** Peter X Adams, MD **Role:** STUDY_DIRECTOR **Official 2:** **Affiliation:** University of Washington **Name:** Edward Verrier, MD **Role:** STUDY_CHAIR ### References Module #### References **Citation:** Smith PK, Shernan SK, Chen JC, Carrier M, Verrier ED, Adams PX, Todaro TG, Muhlbaier LH, Levy JH; PRIMO-CABG II Investigators. Effects of C5 complement inhibitor pexelizumab on outcome in high-risk coronary artery bypass grafting: combined results from the PRIMO-CABG I and II trials. J Thorac Cardiovasc Surg. 2011 Jul;142(1):89-98. doi: 10.1016/j.jtcvs.2010.08.035. Epub 2010 Sep 28. **PMID:** 20880552 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000003327 - Term: Coronary Disease - ID: D000017202 - Term: Myocardial Ischemia - ID: D000006331 - Term: Heart Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000001161 - Term: Arteriosclerosis - ID: D000001157 - Term: Arterial Occlusive Diseases - ID: D000014652 - Term: Vascular Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M6549 - Name: Coronary Disease - Relevance: LOW - As Found: Unknown - ID: M6546 - Name: Coronary Artery Disease - Relevance: HIGH - As Found: Coronary Artery Disease - ID: M19506 - Name: Myocardial Ischemia - Relevance: LOW - As Found: Unknown - ID: M10543 - Name: Ischemia - Relevance: LOW - As Found: Unknown - ID: M9419 - Name: Heart Diseases - Relevance: LOW - As Found: Unknown - ID: M4469 - Name: Arteriosclerosis - Relevance: LOW - As Found: Unknown - ID: M4465 - Name: Arterial Occlusive Diseases - Relevance: LOW - As Found: Unknown - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000003324 - Term: Coronary Artery Disease ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01291979 **Brief Title:** The Effect of Intravenous Lidocaine on Pain After Tonsillectomy **Official Title:** Intravenous Lidocaine for Effective Pain Relief After Tonsillectomy: a Prospective, Randomized, Double-blind, Placebo-controlled Study #### Organization Study ID Info **ID:** ChungAngUH1 #### Organization **Class:** OTHER **Full Name:** Chung-Ang University Hosptial, Chung-Ang University College of Medicine ### Status Module #### Completion Date **Date:** 2012-01 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** NOT_YET_RECRUITING #### Last Update Post Date **Date:** 2011-02-09 **Type:** ESTIMATED **Last Update Submit Date:** 2011-02-08 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2012-01 **Type:** ESTIMATED #### Start Date **Date:** 2011-02 **Status Verified Date:** 2011-02 #### Study First Post Date **Date:** 2011-02-09 **Type:** ESTIMATED **Study First Submit Date:** 2011-02-08 **Study First Submit QC Date:** 2011-02-08 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Chung-Ang University Hosptial, Chung-Ang University College of Medicine #### Responsible Party **Old Name Title:** Kyung Soo Kim **Old Organization:** Chung-Ang University Hospital ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** This prospective randomized study aims to evaluate the effectiveness of intravenous lidocaine injection on the relief of pain in patients undergoing tonsillectomy. A total of 62 patients will be randomized into one of two groups (group C or group I) based on Excel number generation. Patients in group C will receive received normal saline intravenous injection, and patients in group I will receive an intravenous bolus injection of 1.5 mg/kg lidocaine followed by a continuous lidocaine infusion of 2 mg/kg/hr. Visual analogue scale pain scores, fentanyl consumption and the frequency at which patients pushed the button (FPB) of a patient-controlled analgesia system will be recorded at 4, 12, 24, 48 hours postoperatively. ### Conditions Module **Conditions:** - Postoperative Pain **Keywords:** - Pain - Preemptive - Lidocaine - Intravenous - Tonsillectomy ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 62 **Type:** ESTIMATED **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients in Group I (intravenous lidocaine injection group) received an intravenous bolus injection of 1.5 mg/kg lidocaine followed by a continuous lidocaine infusion of 2 mg/kg/hr. **Intervention Names:** - Drug: Intravenous lidocaine injection **Label:** Intravenous lidocaine injection group **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Patients in Group C (placebo control group) received normal saline intravenous injection **Intervention Names:** - Drug: Intravenous normal saline injection **Label:** Placebo control group **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Intravenous lidocaine injection group **Description:** Patients in Group I (intravenous lidocaine injection group) received an intravenous bolus injection of 1.5 mg/kg lidocaine followed by a continuous lidocaine infusion of 2 mg/kg/hr. **Name:** Intravenous lidocaine injection **Other Names:** - IV lidocaine **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo control group **Description:** The patients in Group C (placebo control group) received normal saline intravenous injection **Name:** Intravenous normal saline injection **Other Names:** - IV saline **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Patients will be assessed for pain using a visual analogue pain scale (VAS). It is usually a horizontal line, 100 mm in length, anchored by word descriptors at each end; left side end which represents 0 is 'No pain', right side end with 100 is 'Very severe pain'. The patient marks on the line the point that they feel represents their perception of their current state. The VAS score is determined by measuring in millimeters from the left hand end of the line to the point that the patient marks. To check the severity of pain VAS will be measured at post op 4hour. **Measure:** Visual analogue scale 4hour **Time Frame:** post op 4hour #### Secondary Outcomes **Description:** Patients will be assessed for pain using a visual analogue pain scale (VAS). It is usually a horizontal line, 100 mm in length, anchored by word descriptors at each end; left side end which represents 0 is 'No pain', right side end with 100 is 'Very severe pain'. The patient marks on the line the point that they feel represents their perception of their current state. The VAS score is determined by measuring in millimeters from the left hand end of the line to the point that the patient marks. To check the severity of pain VAS will be measured at post op 12 hour. **Measure:** visual analogue scale 12 hour **Time Frame:** Post op 12 hour **Description:** Patients will be assessed for pain using a visual analogue pain scale (VAS). It is usually a horizontal line, 100 mm in length, anchored by word descriptors at each end; left side end which represents 0 is 'No pain', right side end with 100 is 'Very severe pain'. The patient marks on the line the point that they feel represents their perception of their current state. The VAS score is determined by measuring in millimeters from the left hand end of the line to the point that the patient marks. To check the severity of pain VAS will be measured at post op 12 hour. **Measure:** visual analogue scale 24hour **Time Frame:** Post op 12 hour **Description:** Patients will be assessed for pain using a visual analogue pain scale (VAS). It is usually a horizontal line, 100 mm in length, anchored by word descriptors at each end; left side end which represents 0 is 'No pain', right side end with 100 is 'Very severe pain'. The patient marks on the line the point that they feel represents their perception of their current state. The VAS score is determined by measuring in millimeters from the left hand end of the line to the point that the patient marks. To check the severity of pain VAS will be measured at post op 48 hour. **Measure:** Visual analogue scale 48hour **Time Frame:** Post Op 48hour **Description:** The patients will be taught to push the button of the PCA system, which delivered a bolus of drug, each time pain occurred. In the case of persistent pain greater than a visual analogue scale (VAS) pain score of 30 mm, an additional 50 μg of fentanyl will be injected intra-venously by nursing staff until the pain was relieved to a level less than a VAS pain score of 30 mm. And sum of delivery from PCA system and additional opioid fom immediately after operation to post op 4hour will be measured. **Measure:** Opioid consumption 4hour **Time Frame:** Post Op 4hour **Description:** The patients will be taught to push the button of the PCA system, which delivered a bolus of drug, each time pain occurred. In the case of persistent pain greater than a visual analogue scale (VAS) pain score of 30 mm, an additional 50 μg of fentanyl will be injected intra-venously by nursing staff until the pain was relieved to a level less than a VAS pain score of 30 mm. And sum of delivery from PCA system and additional opioid from 12 hour to post op 24 hour will be measured. **Measure:** Opioid consumption 24hour **Time Frame:** Post op 24 hour **Description:** The patients will be taught to push the button of the PCA system, which delivered a bolus of drug, each time pain occurred. In the case of persistent pain greater than a visual analogue scale (VAS) pain score of 30 mm, an additional 50 μg of fentanyl will be injected intra-venously by nursing staff until the pain was relieved to a level less than a VAS pain score of 30 mm. And sum of delivery from PCA system and additional opioid from post op 4hour to post op 12 hour will be measured. **Measure:** Opioid consumption 12 hour **Time Frame:** Post Op 12 hour **Description:** The patients will be taught to push the button of the PCA system, which delivered a bolus of drug, each time pain occurred. In the case of persistent pain greater than a visual analogue scale (VAS) pain score of 30 mm, an additional 50 μg of fentanyl will be injected intra-venously by nursing staff until the pain was relieved to a level less than a VAS pain score of 30 mm. And sum of delivery from PCA system and additional opioid from post op 24 hour to post op 48 hour will be measured **Measure:** Opioid consumption 48hour **Time Frame:** Post Op 48 hour **Description:** The patients will be taught to push the button of the PCA system, which delivered a bolus of drug, each time pain occurred. And total frequency of patient to push the button of the PCA machine (FPB) fom immediately after operation to post op 4 hour will be measured. **Measure:** FPB 4 hour **Time Frame:** Post Op 4 hour **Description:** The patients will be taught to push the button of the PCA system, which delivered a bolus of drug, each time pain occurred. And frequency of patient to push the button of the PCA machine (FPB) from post op 4 hour to post op 12 hour will be measured. **Measure:** FPB 12 hour **Time Frame:** post op 12 hour **Description:** The patients will be taught to push the button of the PCA system, which delivered a bolus of drug, each time pain occurred. And frequency of patient to push the button of the PCA machine (FPB) fom post op 12 hour to post op 24 hour will be measured. **Measure:** FPB 24 hour **Time Frame:** Post Op 24 hour **Description:** The patients will be taught to push the button of the PCA system, which delivered a bolus of drug, each time pain occurred. And frequency of patient to push the button of the PCA machine (FPB) fom post op 24 hour to post op 48 hour will be measured. **Measure:** FPB 48 hour **Time Frame:** Post Op 48 hour ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Tonsillectomy Exclusion Criteria: * mental change * allergy to local anesthetics **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** roman00@naver.com **Name:** Hyun Kang, Ph.D. **Phone:** 82-2-6299-2571 **Role:** CONTACT #### Locations **Location 1:** **City:** Seoul **Contacts:** ***Contact 1:*** - **Email:** ksdeok@cau.ac.kr - **Name:** SeongDeok Kim, M.D. & Ph.D. - **Phone:** +82-2-6299-2571 - **Role:** CONTACT ***Contact 2:*** - **Name:** Hyun Kang, Ph.D. - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 3:*** - **Name:** Kyung Soo Kim, Ph.D. - **Role:** SUB_INVESTIGATOR **Country:** Korea, Republic of **Facility:** ChungAng University **Zip:** 156-755 #### Overall Officials **Official 1:** **Affiliation:** Chung-Ang University Hosptial, Chung-Ang University College of Medicine **Name:** Hyun Kang, Ph.D. **Role:** STUDY_CHAIR **Official 2:** **Affiliation:** Chung-Ang University Hosptial, Chung-Ang University College of Medicine **Name:** KyungSoo Kim, Ph.D. **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000011183 - Term: Postoperative Complications - ID: D000010335 - Term: Pathologic Processes - ID: D000010146 - Term: Pain - ID: D000009461 - Term: Neurologic Manifestations ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases ### Condition Browse Module - Browse Leaves - ID: M13069 - Name: Pain, Postoperative - Relevance: HIGH - As Found: Postoperative Pain - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown - ID: M14065 - Name: Postoperative Complications - Relevance: LOW - As Found: Unknown - ID: M12404 - Name: Neurologic Manifestations - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010149 - Term: Pain, Postoperative ### Intervention Browse Module - Ancestors - ID: D000000779 - Term: Anesthetics, Local - ID: D000000777 - Term: Anesthetics - ID: D000002492 - Term: Central Nervous System Depressants - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018689 - Term: Sensory System Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000000889 - Term: Anti-Arrhythmia Agents - ID: D000061567 - Term: Voltage-Gated Sodium Channel Blockers - ID: D000026941 - Term: Sodium Channel Blockers - ID: D000049990 - Term: Membrane Transport Modulators - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: AnArAg - Name: Anti-Arrhythmia Agents - Abbrev: ChanBlk - Name: Channel Blockers - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M11014 - Name: Lidocaine - Relevance: HIGH - As Found: Solution - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown - ID: M4109 - Name: Anesthetics, Local - Relevance: LOW - As Found: Unknown - ID: M4213 - Name: Anti-Arrhythmia Agents - Relevance: LOW - As Found: Unknown - ID: M23177 - Name: Sodium Channel Blockers - Relevance: LOW - As Found: Unknown - ID: M30025 - Name: Diuretics, Potassium Sparing - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000008012 - Term: Lidocaine ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02485379 **Acronym:** MRI-FIRST01 **Brief Title:** Assessment of Prostate MRI Before Prostate Biopsies **Official Title:** Improvement in the Detection of Aggressive Prostate Cancer by Targeted Biopsies Using Multiparametric MRI Findings #### Organization Study ID Info **ID:** 69HCL14_0440 #### Organization **Class:** OTHER **Full Name:** Hospices Civils de Lyon ### Status Module #### Completion Date **Date:** 2016-11 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-07-24 **Type:** ACTUAL **Last Update Submit Date:** 2017-07-20 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2016-11 **Type:** ACTUAL #### Start Date **Date:** 2015-07 **Status Verified Date:** 2017-07 #### Study First Post Date **Date:** 2015-06-30 **Type:** ESTIMATED **Study First Submit Date:** 2015-06-24 **Study First Submit QC Date:** 2015-06-25 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Hospices Civils de Lyon #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Background: Prostate cancer is difficult to detect using ultrasound. As a result, in case of suspicion of prostate cancer based on digital rectal examination (DRE) or Prostate Specific Antigen (PSA) level, it is currently recommended to perform "blinded" systematically distributed biopsies with 10-18 samples obtained from predefined locations in the gland. These so-called systematic biopsies (SB) may lead to improper patient management by (i) missing clinically significant cancer, especially in the anterior half of the gland that tends to be undersampled, (ii) inducing chance detection of clinically insignificant cancer foci that may result in overtreatments, (iii) undersampling the tumor foci and thus underestimating their volume and aggressiveness. Multiparametric Magnetic Resonance Imaging (mp-MRI) has yielded promising results in detecting aggressive (Gleason ≥7) prostate cancers. Several monocenter studies showed that targeted biopsies (TB) based on mp-MRI findings could detect significantly more aggressive cancers, reduce the diagnosis of clinically insignificant cancers, and better evaluate the aggressiveness of detected cancers than SB. However, these monocenter studies only provide low-level evidence and three recent independent reviews of literature concluded that there was a need for a robust multicenter trial evaluating the diagnostic yield of TB as compared to SB. This is particularly important since many academic and private centers in France already perform mp-MRI before prostate biopsy in daily routine. Therefore the risk is that this approach becomes the norm without being properly evaluated and it is crucial and urgent to perform a controlled multicentric study to provide high-level evidence as to whether mp-MRI should or should not be obtained before prostate biopsy. One controlled multicentric study has been published recently in which SB and TB had been obtained by two different operators in 95 patients. TB yielded a significantly higher detection rate for all prostate cancers (69% vs 59%, p=0.033) and for clinically significant cancers (67% vs 52%, p=0.0011). However, this study was limited by the fact that patients with negative mp-MRI were not included. Research hypotheses: There is currently no robust multicenter trial comparing prostate TB based on mp-MRI findings versus the current standard of care (SB). We propose a multicentre prospective trial comparing the results of SB and TB performed in the same patients by two independent operators. Our hypothesis is that TB detects aggressive (Gleason ≥7) cancers in a significantly higher percentage of patients than SB. Main objective: To compare the percentage of patients with "clinically significant cancer" (using definition A, i.e. cancer with Gleason score ≥7) detected by SB versus TB. ### Conditions Module **Conditions:** - Prostate Cancer **Keywords:** - Prostate cancer - Multiparametric MRI - Prostate biopsy - Targeted biopsy - Comparative study ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** DIAGNOSTIC #### Enrollment Info **Count:** 275 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Systematic biopsies (SB) and targeted biopsies (TB) are performed in the same patients by two independent operators. In patients without abnormalities on mp-MRI, no targeted biopsies will be carried out and the detection of "clinically significant cancer" will be considered as negative for the TB strategy. **Intervention Names:** - Procedure: Prostate biopsy **Label:** Prostate biopsy **Type:** OTHER ### Interventions #### Intervention 1 **Arm Group Labels:** - Prostate biopsy **Description:** Systematic biopsies (SB) and targeted biopsies (TB) are performed in the same patients by two independent operators. In patients without abnormalities on mp-MRI, no targeted biopsies will be carried out and the detection of "clinically significant cancer" will be considered as negative for the TB strategy. **Name:** Prostate biopsy **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Measure:** Detection of "clinically significant cancer" (using definition A, i.e. Gleason ≥7 cancers) in at least one core of SB or TB. **Time Frame:** Between 1 and 4 months after the enrollment #### Secondary Outcomes **Measure:** To compare the percentage of patients with "clinically significant cancer" (using definition B, i.e. any Gleason ≥7 cancer or Gleason 6 cancer with at least one sample with ≥6 mm of cancer) detected by SB and TB. **Time Frame:** Between 1 and 4 months after the enrollment **Measure:** To compare the percentage of patients with "clinically significant cancer" (using definition C, i.e. any Gleason ≥7 (4+3) cancer) **Time Frame:** Between 1 and 4 months after the enrollment **Measure:** To compare the percentage of patients with "clinically insignificant cancer" (defined as a Gleason ≤6 cancer with ≤2 positive samples and <3 mm of cancer on the positive samples) detected by SB and TB. **Time Frame:** Between 1 and 4 months after the enrollment **Description:** The different subgroups are : * Patients with clinical stage T1c (i.e. normal DRE) versus T2a-T2c, * Patients with a PSA level \<10 ng/mL versus 10-20 ng/mL, * Patients with a prostate volume ≤50 cc versus \>50 cc, * Patients in whom TB have been performed with ultrasound/MRI fusion versus cognitive guidance **Measure:** To compare the percentage of patients with Gleason ≥7 cancer detected by SB and TB in different subgroups **Time Frame:** Between 1 and 4 months after the enrollment **Measure:** To compare the percentage of patients detected by TB and by SB+TB with "clinically significant cancer" (using definitions A, B and C) and "clinically insignificant" cancer. **Time Frame:** Between 1 and 4 months after the enrollment **Measure:** To compare the percentage of patients with "clinically significant cancer" (using definitions A, B and C) detected by the 2 optional US-guided biopsies and by the regular 12 systematic biopsies. **Time Frame:** Between 1 and 4 months after the enrollment **Measure:** To evaluate the percentage of patients with overall cancer and with "clinically significant cancer" (using definitions A, B and C) on SB and who had a negative MRI **Time Frame:** Between 1 and 4 months after the enrollment **Measure:** To evaluate the percentage of patients with overall cancer and "clinically significant cancer" (using definitions A, B and C) on SB and who had a positive MRI in the sextant(s) that were positive on SB. **Time Frame:** Between 1 and 4 months after the enrollment **Measure:** To evaluate the percentage of patients with discordant results (Gleason score, maximum length of invasion) between the local pathological analysis and between the central pathological review. **Time Frame:** Between 1 and 4 months after the enrollment ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patient referred for prostate mp-MRI before a first set of prostate biopsies, with a planned time interval of less than 3 months between MRI and biopsies * Age ≤75 years * PSA level ≤20 ng/mL * Clinical stage ≤T2c * Patient insured under the French social security system or beneficiary of an equivalent regime Exclusion Criteria: * Contraindication to transrectal biopsy * Contraindication to MRI * History of hip prosthesis * History of androgen deprivation therapy * Patients with history of prostate cancer diagnosed on TURP * Patients with history of pelvic radiation therapy (whatever the reason) * Patient deprived of freedom following a court or administrative order * Patient under guardianship or under legal guardianship **Maximum Age:** 75 Years **Minimum Age:** 18 Years **Sex:** MALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Bordeaux **Country:** France **Facility:** Groupe Hospitalier Pellegrin - CHU de Bordeaux **Zip:** 33076 **Location 2:** **City:** Grenoble **Country:** France **Facility:** Hôpital Michallon - CHU de Grenoble **Zip:** 38043 **Location 3:** **City:** Lille **Country:** France **Facility:** Hôpital Huriez - CHU de Lille **Zip:** 59037 **Location 4:** **City:** Lille **Country:** France **Facility:** Hôpital Privé La Louvière **Zip:** 59042 **Location 5:** **City:** Lille **Country:** France **Facility:** CLIMAL (Centre Libéral Imagerie Médicale Agglomération Lille) **Zip:** 59700 **Location 6:** **City:** Lyon Cedex 03 **Country:** France **Facility:** Hôpital Edouard Herriot **Zip:** 69437 **Location 7:** **City:** Lyon **Country:** France **Facility:** Centre Hospitalier St Joseph St Luc **Zip:** 69365 **Location 8:** **City:** Marseille **Country:** France **Facility:** Institut Paoli Calmettes **Zip:** 13273 **Location 9:** **City:** Nantes **Country:** France **Facility:** Clinique Jules Verne **Zip:** 44300 **Location 10:** **City:** Paris **Country:** France **Facility:** Hopital Pitie Salpetriere **Zip:** 75013 **Location 11:** **City:** Paris **Country:** France **Facility:** Hôpital Cochin **Zip:** 75014 **Location 12:** **City:** Paris **Country:** France **Facility:** Hôpital Européen Georges Pompidou **Zip:** 75015 **Location 13:** **City:** Paris **Country:** France **Facility:** Hôpital Necker **Zip:** 75015 **Location 14:** **City:** Pierre Bénite **Country:** France **Facility:** Centre Hospitalier Lyon Sud **Zip:** 69495 **Location 15:** **City:** Saint-Etienne **Country:** France **Facility:** CHU de Saint-Etienne **Zip:** 42055 **Location 16:** **City:** Saint-Priest-en-Jarez **Country:** France **Facility:** IRMAS **Zip:** 42270 **Location 17:** **City:** St Herblain **Country:** France **Facility:** Clinique Urologique Nantes Atlantis **Zip:** 44815 **Location 18:** **City:** Strasbourg **Country:** France **Facility:** Nouvel Hopital Civil - CHU de Strasbourg **Zip:** 67091 **Location 19:** **City:** Toulouse **Country:** France **Facility:** Institut Universitaire du Cancer de Toulouse (IUCT) Oncopole - CHU de Toulouse **Zip:** 31059 **Location 20:** **City:** Vandoeuvre-les-Nancy **Country:** France **Facility:** CHU Nancy Brabois **Zip:** 54511 ### References Module #### References **Citation:** Rouviere O, Puech P, Renard-Penna R, Claudon M, Roy C, Mege-Lechevallier F, Decaussin-Petrucci M, Dubreuil-Chambardel M, Magaud L, Remontet L, Ruffion A, Colombel M, Crouzet S, Schott AM, Lemaitre L, Rabilloud M, Grenier N; MRI-FIRST Investigators. Use of prostate systematic and targeted biopsy on the basis of multiparametric MRI in biopsy-naive patients (MRI-FIRST): a prospective, multicentre, paired diagnostic study. Lancet Oncol. 2019 Jan;20(1):100-109. doi: 10.1016/S1470-2045(18)30569-2. Epub 2018 Nov 21. **PMID:** 30470502 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000005834 - Term: Genital Neoplasms, Male - ID: D000014565 - Term: Urogenital Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000005832 - Term: Genital Diseases, Male - ID: D000091662 - Term: Genital Diseases - ID: D000091642 - Term: Urogenital Diseases - ID: D000011469 - Term: Prostatic Diseases - ID: D000052801 - Term: Male Urogenital Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions - Abbrev: BXM - Name: Behaviors and Mental Disorders ### Condition Browse Module - Browse Leaves - ID: M14335 - Name: Prostatic Neoplasms - Relevance: HIGH - As Found: Prostate Cancer - ID: M3724 - Name: Aggression - Relevance: LOW - As Found: Unknown - ID: M8946 - Name: Genital Neoplasms, Male - Relevance: LOW - As Found: Unknown - ID: M17315 - Name: Urogenital Neoplasms - Relevance: LOW - As Found: Unknown - ID: M2876 - Name: Genital Diseases - Relevance: LOW - As Found: Unknown - ID: M8944 - Name: Genital Diseases, Male - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14333 - Name: Prostatic Diseases - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000011471 - Term: Prostatic Neoplasms ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04392479 **Acronym:** TWICE-IRI **Brief Title:** TWICE-IRI: Optimization of Second-line Therapy With Aflibercept, Irinotecan (Day 1 or Day 1,3), 5-Fluorouracile and Folinic Acid in Patients With Metastatic Colorectal Cancer. A Randomized Phase III Study. **Official Title:** TWICE-IRI: Optimization of Second-line Therapy With Aflibercept, Irinotecan (Day 1 or Day 1,3), 5-Fluorouracile and Folinic Acid in Patients With Metastatic Colorectal Cancer. A Randomized Phase III Study. #### Organization Study ID Info **ID:** FBH-002 #### Organization **Class:** OTHER **Full Name:** Hôpital Franco-Britannique-Fondation Cognacq-Jay ### Status Module #### Completion Date **Date:** 2024-06 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-10-23 **Type:** ACTUAL **Last Update Submit Date:** 2023-10-19 **Overall Status:** ACTIVE_NOT_RECRUITING #### Primary Completion Date **Date:** 2023-06-15 **Type:** ACTUAL #### Start Date **Date:** 2020-09-02 **Type:** ACTUAL **Status Verified Date:** 2023-10 #### Study First Post Date **Date:** 2020-05-18 **Type:** ACTUAL **Study First Submit Date:** 2020-05-13 **Study First Submit QC Date:** 2020-05-13 ### Sponsor Collaborators Module #### Collaborators **Class:** UNKNOWN **Name:** Fondation ARCAD #### Lead Sponsor **Class:** OTHER **Name:** Hôpital Franco-Britannique-Fondation Cognacq-Jay #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** Optimization of second-line therapy with aflibercept, irinotecan (day1 or day 1,3), 5fluorouracile and folinic acid in patients with metastatic colorectal cancer. A randomized Phase III study. **Detailed Description:** Background - Rationale Aflibercept The addition of aflibercept to the standard FOLFIRI regimen as second-line therapy was evaluated in a large phase III study (EFC10262-VELOUR). This combination significantly improved both PFS (4.7 to 6.9 months, HR=0.76; P=\<0.001) and OS (12.1 to 13.5 months, HR=0.82; P=0.003). In the evaluable population (86.5%), the tumor response rate was also improved when adding aflibercept (ORR=19.8% \[16.4-23.2\]) to the FOLFIRI regimen (ORR=11.1% \[8.5-13.8\]). Irinotecan The combination of aflibercept with FOLFIRI3, an optimized irinotecan-based regimen, was evaluated in 65 patients in a French multicentric retrospective cohort. (Carola C et al, WJCO 2018) In the cohort of irinotecan-naïve patients (n=30), the objective response rate was 43.3%, and the disease control rate 76.7%. Median PFS and OS were 11.3 months (95% CI 6.1-29.0) and 17.0 months (95% CI 13.0-17.3). The most common (\>5%) grade 3-4 adverse events were diarrhea (37.9%), neutropenia (14.3%), stomatitis and anemia (10.4%), hypertension (6.7%). In the cohort of patients previously treated with irinotecan (n=35), the objective response rate was 34.3%, and the disease control rate 60.0%. Median PFS and OS were 5.7 months (95% CI 3.9-10.4) and 14.3 months (95% CI 12.8-19.5). Table. FOLFIRI-aflibercept vs. FOLFIRI3-aflibercept: a cross-trial comparison FOLFIRI-aflibercept (VELOUR) FOLFIRI3-aflibercept (Irinotecan-naïve) N = 612 N = 30 Efficacy RR, % 19.3 vs 43.3 PFS, months 6.9 vs 11.3 OS, months 13.5 vs 17.0 Grade 3-4 AEs, % Any 83.4 vs 56.7 Neutropenia 36.7 vs 14.3 Diarrhea 19.3 vs 37.9 Mucositis 13.8 vs 10.4 Hypertension 19.3 vs 6.9 Discontinuation, % Progression 49.8 vs 36.7 Adverse event 26.6 vs 46.7 Study Objectives Primary: •To compare once (day 1) or twice (day 1, day 3) administration of irinotecan in combination with 5-fluorouracile and aflibercept as second-line therapy in patients with metastatic colorectal cancer in terms of overall response rate (ORR) Secondary: * To evaluate disease control rate (DRC) and Early Response Rate (ERR) * To evaluate progression-free survival (PFS) and overall survival (OS), * to evaluate the conversion rate to locoregional therapy, surgery of metastasis, the quality of resection (R0, R1, R2) and the pathological response in patients with resected metastatic disease, * To assess the impact on health-related quality of life (HRQOL) using the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-C30 (QLQ-C30), * To assess Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) on using the National Cancer Institute Common Toxicity Criteria (NCI-CTC version 5.0). Exploratory: * To assess the impact on selected circulating biomarkers (angiogenic and exosomal) * To assess the impact on selected tumor biomarkers ### Conditions Module **Conditions:** - Cancer Colorectal - Metastasis **Keywords:** - Aflibercept ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 202 **Type:** ESTIMATED **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** * Aflibercept (D1) H0: 4mg/kg IV infusion over 60min (+ 2-minute window), * Folinic acid (D1) H+1: 400mg/m² IV infusion over 120min (+ 2-minute window), * Irinotecan (D1) H+1: 180mg/m² IV infusion over 60min (+ 2-minute window), * 5-fluorouracile (D1) H+3: 400mg/m² IV infusion over 15min (+ 2-minute window), * 5-fluorouracile (D1 to D3): H+3.5: 2400mg/m² IV infusion over 46 hours (+ 1hour window) * H+49.5: End of treatment administration **Intervention Names:** - Drug: Aflibercept-FOLFIRI **Label:** Aflibercept-FOLFIRI (arm 1) **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** * Aflibercept (D1) H0: 4mg/kg IV infusion over 60min (+ 2-minute window), * Folinic acid (D1) H+1: 400mg/m² IV infusion over 120min (+ 2-minute window), * Irinotecan (D1 and D3) H+1 and H+49: 75mg/m² IV infusion over 60min (+ 2-minute window) on cycles 1 and 2, then 90mg/m² at cycle 3 and furthers in absence of AEs grade ≥2, * 5-fluorouracile (D1 to D3) H+3: 2400mg/m² IV infusion over 46 hours (+ 1hour window) * H+50: End of treatment administration **Intervention Names:** - Drug: Aflibercept-mFOLFIRI3 **Label:** Aflibercept-mFOLFIRI3 (arm 2) **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Aflibercept-FOLFIRI (arm 1) **Description:** Aflibercept-FOLFIRI **Name:** Aflibercept-FOLFIRI **Other Names:** - ZALTRAP-FOLFIRI **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Aflibercept-mFOLFIRI3 (arm 2) **Description:** Aflibercept-mFOLFIRI3 **Name:** Aflibercept-mFOLFIRI3 **Other Names:** - ZALTRAP- mFOLFIRI3 **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Tumor measurements will be obtained using CT-scans (or MRIs) of the thorax, abdomen, and pelvis at baseline then every 8 weeks (+/- one week) according to RECIST v1.1. At the investigator's discretion, tumor assessments may be repeated at any time if progressive disease is suspected. It is preferred that the scans for a patient are taken with the same technique (CT or MRI) throughout the study. **Measure:** Overall response rate (ORR). **Time Frame:** 2 months #### Secondary Outcomes **Description:** The disease control rate is the sum of patients with tumor response (either CR or PR) or stabilization (SD) as best response. **Measure:** Disease control rate (DCR) **Time Frame:** 2 months **Description:** The early response rate will be evaluated at the first tumor evaluation at 8 weeks (+/- 1 week). **Measure:** Early response rate **Time Frame:** 2 months **Description:** PFS is defined as the time interval from randomization to the date of first documented disease progression or death from any cause, whichever occurs first. Alive patients without progression will be censored at the last tumor assessment, either during study treatment period or during follow-up period. **Measure:** Progression-free survival (PFS) **Time Frame:** 2 months **Description:** OS is defined as the time interval from randomization to the date of death from any cause. Alive patients will be censored at the last date known to be alive, either during study treatment period or during follow-up period. Survival status will be assessed by telephone, ordinary visits, hospital records or other means found suitable until death or the end of the study, whichever occurs first. Information regarding post-study anti-cancer therapies will be collected if new treatment is initiated. **Measure:** Overall survival (OS) **Time Frame:** time interval from randomization to the date of death from any cause. Assessed up to 13 months after the beginning of the study **Description:** Salvage surgery rate (%) corresponding to the number of patients amenable to surgery of metastasis during study treatment. The number of patients with R0 or R1 resection will be evaluated. **Measure:** Salvage surgery rate **Time Frame:** 2 months **Description:** The pathological tumor response will be assessed in patients having primary and/or metastasis resection using Tumor Regression Grade (TRG), modified TRG and Blazer grade. (Rubbia-Brandt L et al, Ann Oncol 2007 ; Blazer 3rd DG et al, J Clin Oncol 2008) **Measure:** Pathological response rate **Time Frame:** 2 months **Description:** frequency of AEs and SAEs per patient and per cycle of treatment using NCI CTCAE v5.0. **Measure:** Tolerance **Time Frame:** 2 weeks **Description:** The time until definitive HRQoL score deterioration (TUDD), (Bonnetain F et al, Eur J Cancer 2010) The survival without HRQoL deterioration-free survival (QFS). HRQoL will be assessed with EORTC QLQ-C30 questionnaire **Measure:** HRQoL **Time Frame:** 2 months **Description:** Blood : (PlGF, VEGF-A, VEGF-B, VEGF-C, VEGF-D, sVEGFR1, sVEGFR2, sVEGFR3, Ang2, sTie2, Syndecan) .Exosomal biomarkers assessments (CEA, HSP70, VEGFR-2) Tumor: Analyses will be performed according to the knowledge on potential markers at that time **Measure:** Exploratory biomarkers **Time Frame:** 2 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Provision of signed and dated informed consent and stated willingness to comply with all study procedures and availability for the duration of the study, Signed, written Informed Consent Form (ICF), 2. Willing and able to comply with the protocol, 3. Age 18-75 years, 4. Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0-1, 5. Life expectancy ≥ 3 months, 6. Histologically proven carcinoma of colon and/or rectum, 7. Confirmed unresectable metastatic disease, 8. At least one measurable and/or evaluable tumor metastasis on CT-scan or MRI per RECIST criteria version 1.1, 9. Prior oxaliplatin-based first-line therapy for metastatic disease (the use of prior bevacizumab or anti-EGFR mabs is allowed but not mandatory) - Less than 6 months from completion of any prior oxaliplatin-based adjuvant therapy can be considered as first-line therapy. Prior use of irinotecan in combination with oxaliplatin and 5FU as first-line therapy is allowed if the interval between the last administration of irinotecan and disease progression is at least 6 months (ie, irinotecan-free interval ≥6 months). 10. Negative urine and/or serum pregnancy test within 7 days before inclusion if female subject is of childbearing potential, 11. Clinical laboratory parameters adequate as follows: * Serum total bilirubin level ≤ 1.5 x upper normal limit (UNL), * Neutrophil count ≥ 1.5x109/L, * Platelet count ≥ 100x109/L, * Hemoglobin ≥ 9 g/dL, * Serum creatinine level ≤ 150µM, * Serum albumin ≥ 25 g/L, * Calcium ≥ 1 x ULN * Alkaline phosphatase (ALP) \< 3 x ULN, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \< 3 x ULN (in the case of liver metastases, \<5 x ULN), * Proteinuria \<2+ (dipstick urinalysis) or ≤1g/24hour, 12. For women of childbearing potential and for men, agreement to use an effective contraceptive method from the time of screening throughout the study until 6 months after administration of the last dose of any study medication. Highly effective contraceptive method consist of prior sterilization, inter-uterine device, intrauterine hormone-releasing system, oral or injectable contraceptives barrier methods, and/or true sexual abstinence), 13. Affiliation to French health care system. Exclusion Criteria: 1. History of arterial thrombotic event in the last 6 months (eg., myocardial infarction, cerebrovascular accident or transient ischemic attack), 2. Uncontrolled hypertension (defined as systolic blood pressure \> 140 mmHg and/or diastolic blood pressure \> 90 mmHg despite optimal medical therapy), or history of hypertensive crisis, or hypertensive encephalopathy, 3. Prior use of aflibercept, 4. Adverse events from prior anticancer therapy grade ≥2 (National Cancer Institute Common Terminology Criteria for Adverse Events \[NCI-CTCAE\] version 5.0), except for neuropathy and alopecia, 5. Bowel obstruction, inflammatory bowel disease 6. Known DPD deficiency. If not known for the patient, testing for DPD should be done during the screening period (patients with uracilemia ≥16ng/mL are not eligible), 7. Known UGT1A1 deficiency (eg, Gilbert syndrome, Crigler-Najjar syndrome). If not known for the patient, genetic testing for UGT1A1 should be done during the screening period for patients with hyperbilirubinemia (ie, total bilirubin level \>1xULN), 8. Active infection requiring intravenous antibiotics at the start of study treatment, 9. Known active infection with human immunodeficiency virus (HIV), hepatitis B (HBV), or hepatitis C (HCV), 10. Known allergy or hypersensitivity to the active substance or ingredients of any study drug, 11. Women currently pregnant or breastfeeding, 12. Inability to comply with study and follow-up procedures as judged by the Investigator, 13. Concomitant unplanned antitumor therapy (e.g. chemotherapy, molecular targeted therapy, immunotherapy) 14. Concomitant use of Saint John Wort herb (millepertuis), Yellow Fever vaccine, Live Attenuated Vaccines (LAV) and phenytoine 15. Treatment with any other investigational medicinal product within 28 days or 5 investigational agent half-lives (whichever is longer) prior to the start of study treatment, 16. Any other disease, active, uncontrolled bacterial, viral or fungal infection requiring systemic therapy, metabolic dysfunction, physical examination finding or clinical laboratory finding that leads to reasonable suspicion of a disease or condition that contraindicates the use of study drugs that may affect the interpretation of the results, or that may render the subject at high risk for treatment complications. 17. Previous or concurrent malignancy, except for adequately treated basal or squamous cell skin cancer, in situ cervical cancer, or other cancer for which the patient has been disease-free for three years prior to study entry, 18. Surgical procedure (including open biopsy, surgical resection, wound revision, or any other major surgery involving entry into a body cavity) or significant traumatic injury within 28 days prior to start of study treatment, or anticipation of need for major surgical procedure during the course of the study. 19. Minor surgical procedure including placement of a vascular access device, within 2 days of start of study treatment, 20. History of abdominal fistula, gastrointestinal (GI) perforation, intra-abdominal abscess or active GI bleeding within 6 months prior to start study treatment. 21. Clinically significant active cardiac disease (including NYHA class III or IV congestive heart failure) 22. Venous thromboembolic event (including pulmonary embolism) grade 3 or 4 within 6 months prior to start study treatment. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Levallois-Perret **Country:** France **Facility:** Franco-British Hospital - GCS IHFB Cognacq-Jay **Zip:** 92300 #### Overall Officials **Official 1:** **Affiliation:** Franco-British Hospital GCS IHFB Cognacq-Jay **Name:** Benoist CHIBAUDEL, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Bray F, Ferlay J, Soerjomataram I, Siegel RL, Torre LA, Jemal A. Global cancer statistics 2018: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2018 Nov;68(6):394-424. doi: 10.3322/caac.21492. Epub 2018 Sep 12. Erratum In: CA Cancer J Clin. 2020 Jul;70(4):313. **PMID:** 30207593 **Citation:** Tournigand C, Andre T, Achille E, Lledo G, Flesh M, Mery-Mignard D, Quinaux E, Couteau C, Buyse M, Ganem G, Landi B, Colin P, Louvet C, de Gramont A. FOLFIRI followed by FOLFOX6 or the reverse sequence in advanced colorectal cancer: a randomized GERCOR study. J Clin Oncol. 2004 Jan 15;22(2):229-37. doi: 10.1200/JCO.2004.05.113. Epub 2003 Dec 2. **PMID:** 14657227 **Citation:** Jolivet J. Role of leucovorin dosing and administration schedule. Eur J Cancer. 1995 Jul-Aug;31A(7-8):1311-5. doi: 10.1016/0959-8049(95)00140-e. **PMID:** 7577042 **Citation:** Van Cutsem E, Tabernero J, Lakomy R, Prenen H, Prausova J, Macarulla T, Ruff P, van Hazel GA, Moiseyenko V, Ferry D, McKendrick J, Polikoff J, Tellier A, Castan R, Allegra C. Addition of aflibercept to fluorouracil, leucovorin, and irinotecan improves survival in a phase III randomized trial in patients with metastatic colorectal cancer previously treated with an oxaliplatin-based regimen. J Clin Oncol. 2012 Oct 1;30(28):3499-506. doi: 10.1200/JCO.2012.42.8201. Epub 2012 Sep 4. **PMID:** 22949147 **Citation:** Peeters M, Price TJ, Cervantes A, Sobrero AF, Ducreux M, Hotko Y, Andre T, Chan E, Lordick F, Punt CJ, Strickland AH, Wilson G, Ciuleanu TE, Roman L, Van Cutsem E, Tzekova V, Collins S, Oliner KS, Rong A, Gansert J. Randomized phase III study of panitumumab with fluorouracil, leucovorin, and irinotecan (FOLFIRI) compared with FOLFIRI alone as second-line treatment in patients with metastatic colorectal cancer. J Clin Oncol. 2010 Nov 1;28(31):4706-13. doi: 10.1200/JCO.2009.27.6055. Epub 2010 Oct 4. **PMID:** 20921462 **Citation:** Tabernero J, Yoshino T, Cohn AL, Obermannova R, Bodoky G, Garcia-Carbonero R, Ciuleanu TE, Portnoy DC, Van Cutsem E, Grothey A, Prausova J, Garcia-Alfonso P, Yamazaki K, Clingan PR, Lonardi S, Kim TW, Simms L, Chang SC, Nasroulah F; RAISE Study Investigators. Ramucirumab versus placebo in combination with second-line FOLFIRI in patients with metastatic colorectal carcinoma that progressed during or after first-line therapy with bevacizumab, oxaliplatin, and a fluoropyrimidine (RAISE): a randomised, double-blind, multicentre, phase 3 study. Lancet Oncol. 2015 May;16(5):499-508. doi: 10.1016/S1470-2045(15)70127-0. Epub 2015 Apr 12. Erratum In: Lancet Oncol. 2015 Jun;16(6):e262. **PMID:** 25877855 **Citation:** Guichard S, Cussac D, Hennebelle I, Bugat R, Canal P. Sequence-dependent activity of the irinotecan-5FU combination in human colon-cancer model HT-29 in vitro and in vivo. Int J Cancer. 1997 Nov 27;73(5):729-34. doi: 10.1002/(sici)1097-0215(19971127)73:53.0.co;2-#. **PMID:** 9398054 **Citation:** Mullany S, Svingen PA, Kaufmann SH, Erlichman C. Effect of adding the topoisomerase I poison 7-ethyl-10-hydroxycamptothecin (SN-38) to 5-fluorouracil and folinic acid in HCT-8 cells: elevated dTTP pools and enhanced cytotoxicity. Cancer Chemother Pharmacol. 1998;42(5):391-9. doi: 10.1007/s002800050835. **PMID:** 9771954 **Citation:** Mans DR, Grivicich I, Peters GJ, Schwartsmann G. Sequence-dependent growth inhibition and DNA damage formation by the irinotecan-5-fluorouracil combination in human colon carcinoma cell lines. Eur J Cancer. 1999 Dec;35(13):1851-61. doi: 10.1016/s0959-8049(99)00222-1. **PMID:** 10674003 **Citation:** Falcone A, Di Paolo A, Masi G, Allegrini G, Danesi R, Lencioni M, Pfanner E, Comis S, Del Tacca M, Conte P. Sequence effect of irinotecan and fluorouracil treatment on pharmacokinetics and toxicity in chemotherapy-naive metastatic colorectal cancer patients. J Clin Oncol. 2001 Aug 1;19(15):3456-62. doi: 10.1200/JCO.2001.19.15.3456. **PMID:** 11481350 **Citation:** Mabro M, Louvet C, Andre T, Carola E, Gilles-Amar V, Artru P, Krulik M, de Gramont A; GERCOR. Bimonthly leucovorin, infusion 5-fluorouracil, hydroxyurea, and irinotecan (FOLFIRI-2) for pretreated metastatic colorectal cancer. Am J Clin Oncol. 2003 Jun;26(3):254-8. doi: 10.1097/01.COC.0000020581.59835.7A. **PMID:** 12796595 **Citation:** Mabro M, Artru P, Andre T, Flesch M, Maindrault-Goebel F, Landi B, Lledo G, Plantade A, Louvet C, de Gramont A. A phase II study of FOLFIRI-3 (double infusion of irinotecan combined with LV5FU) after FOLFOX in advanced colorectal cancer patients. Br J Cancer. 2006 May 8;94(9):1287-92. doi: 10.1038/sj.bjc.6603095. **PMID:** 16622455 **Citation:** Bidard FC, Tournigand C, Andre T, Mabro M, Figer A, Cervantes A, Lledo G, Bengrine-Lefevre L, Maindrault-Goebel F, Louvet C, de Gramont A. Efficacy of FOLFIRI-3 (irinotecan D1,D3 combined with LV5-FU) or other irinotecan-based regimens in oxaliplatin-pretreated metastatic colorectal cancer in the GERCOR OPTIMOX1 study. Ann Oncol. 2009 Jun;20(6):1042-7. doi: 10.1093/annonc/mdn730. Epub 2009 Jan 19. **PMID:** 19153116 **Citation:** Chibaudel B, Maindrault-Goebel F, Bachet JB, Louvet C, Khalil A, Dupuis O, Hammel P, Garcia ML, Bennamoun M, Brusquant D, Tournigand C, Andre T, Arbaud C, Larsen AK, Wang YW, Yeh CG, Bonnetain F, de Gramont A. PEPCOL: a GERCOR randomized phase II study of nanoliposomal irinotecan PEP02 (MM-398) or irinotecan with leucovorin/5-fluorouracil as second-line therapy in metastatic colorectal cancer. Cancer Med. 2016 Apr;5(4):676-83. doi: 10.1002/cam4.635. Epub 2016 Jan 24. **PMID:** 26806397 **Citation:** Carola C, Ghiringhelli F, Kim S, Andre T, Barlet J, Bengrine-Lefevre L, Marijon H, Garcia-Larnicol ML, Borg C, Dainese L, Steuer N, Richa H, Benetkiewicz M, Larsen AK, de Gramont A, Chibaudel B. FOLFIRI3-aflibercept in previously treated patients with metastatic colorectal cancer. World J Clin Oncol. 2018 Sep 14;9(5):110-118. doi: 10.5306/wjco.v9.i5.110. **PMID:** 30254966 **Citation:** Ghiringhelli F, Vincent J, Beltjens F, Bengrine L, Ladoire S. Fluorouracil, leucovorin and irinotecan associated with aflibercept can induce microscopic colitis in metastatic colorectal cancer patients. Invest New Drugs. 2015 Dec;33(6):1263-6. doi: 10.1007/s10637-015-0295-6. Epub 2015 Oct 22. **PMID:** 26490656 **Citation:** Rubbia-Brandt L, Giostra E, Brezault C, Roth AD, Andres A, Audard V, Sartoretti P, Dousset B, Majno PE, Soubrane O, Chaussade S, Mentha G, Terris B. Importance of histological tumor response assessment in predicting the outcome in patients with colorectal liver metastases treated with neo-adjuvant chemotherapy followed by liver surgery. Ann Oncol. 2007 Feb;18(2):299-304. doi: 10.1093/annonc/mdl386. Epub 2006 Oct 23. **PMID:** 17060484 **Citation:** Blazer DG 3rd, Kishi Y, Maru DM, Kopetz S, Chun YS, Overman MJ, Fogelman D, Eng C, Chang DZ, Wang H, Zorzi D, Ribero D, Ellis LM, Glover KY, Wolff RA, Curley SA, Abdalla EK, Vauthey JN. Pathologic response to preoperative chemotherapy: a new outcome end point after resection of hepatic colorectal metastases. J Clin Oncol. 2008 Nov 20;26(33):5344-51. doi: 10.1200/JCO.2008.17.5299. Epub 2008 Oct 20. **PMID:** 18936472 **Citation:** Bonnetain F, Dahan L, Maillard E, Ychou M, Mitry E, Hammel P, Legoux JL, Rougier P, Bedenne L, Seitz JF. Time until definitive quality of life score deterioration as a means of longitudinal analysis for treatment trials in patients with metastatic pancreatic adenocarcinoma. Eur J Cancer. 2010 Oct;46(15):2753-62. doi: 10.1016/j.ejca.2010.07.023. Epub 2010 Aug 17. **PMID:** 20724140 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007414 - Term: Intestinal Neoplasms - ID: D000005770 - Term: Gastrointestinal Neoplasms - ID: D000004067 - Term: Digestive System Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000004066 - Term: Digestive System Diseases - ID: D000005767 - Term: Gastrointestinal Diseases - ID: D000003108 - Term: Colonic Diseases - ID: D000007410 - Term: Intestinal Diseases - ID: D000012002 - Term: Rectal Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC06 - Name: Digestive System Diseases ### Condition Browse Module - Browse Leaves - ID: M12307 - Name: Neoplasm Metastasis - Relevance: LOW - As Found: Unknown - ID: M17890 - Name: Colorectal Neoplasms - Relevance: HIGH - As Found: Colorectal Cancer - ID: M10448 - Name: Intestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M8886 - Name: Gastrointestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7256 - Name: Digestive System Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M6336 - Name: Colonic Diseases - Relevance: LOW - As Found: Unknown - ID: M10444 - Name: Intestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M14844 - Name: Rectal Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000015179 - Term: Colorectal Neoplasms ### Intervention Browse Module - Ancestors - ID: D000020533 - Term: Angiogenesis Inhibitors - ID: D000043924 - Term: Angiogenesis Modulating Agents - ID: D000006133 - Term: Growth Substances - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000006131 - Term: Growth Inhibitors - ID: D000000970 - Term: Antineoplastic Agents ### Intervention Browse Module - Browse Branches - Abbrev: Micro - Name: Micronutrients - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: Hemat - Name: Hematinics - Abbrev: Vi - Name: Vitamins ### Intervention Browse Module - Browse Leaves - ID: M6191 - Name: Leucovorin - Relevance: LOW - As Found: Unknown - ID: M29233 - Name: Levoleucovorin - Relevance: LOW - As Found: Unknown - ID: M257727 - Name: Aflibercept - Relevance: HIGH - As Found: Magnetic Resonance Imaging - ID: M1671 - Name: Irinotecan - Relevance: LOW - As Found: Unknown - ID: M8618 - Name: Folic Acid - Relevance: LOW - As Found: Unknown - ID: M22318 - Name: Angiogenesis Inhibitors - Relevance: LOW - As Found: Unknown - ID: M9231 - Name: Growth Inhibitors - Relevance: LOW - As Found: Unknown - ID: T447 - Name: Folinic Acid - Relevance: LOW - As Found: Unknown - ID: T446 - Name: Folic Acid - Relevance: LOW - As Found: Unknown - ID: T448 - Name: Folate - Relevance: LOW - As Found: Unknown - ID: T475 - Name: Vitamin B9 - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000533178 - Term: Aflibercept ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05994079 **Brief Title:** Effect Of High-Intensity Focused Ultrasound On Abdominal Skin Laxity Post Sleeve Gastrectomy **Official Title:** Effect Of High-Intensity Focused Ultrasound On Abdominal Skin Laxity Post Sleeve Gastrectomy #### Organization Study ID Info **ID:** 012/003743 #### Organization **Class:** OTHER **Full Name:** Cairo University ### Status Module #### Completion Date **Date:** 2022-12-20 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-08-16 **Type:** ACTUAL **Last Update Submit Date:** 2023-08-15 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-11-20 **Type:** ACTUAL #### Start Date **Date:** 2022-06-20 **Type:** ACTUAL **Status Verified Date:** 2023-08 #### Study First Post Date **Date:** 2023-08-16 **Type:** ACTUAL **Study First Submit Date:** 2023-08-08 **Study First Submit QC Date:** 2023-08-15 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Cairo University #### Responsible Party **Investigator Affiliation:** Cairo University **Investigator Full Name:** Rania Hassan Kamel Abdelkader El-Hawary **Investigator Title:** Principal Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of the study is to evaluate the therapeutic effect of High intensity focused ultrasound in treatment of abdominal skin laxity post sleeve gastrectomy. **Detailed Description:** Lax skin is a common consequence of bariatric weight loss, and it may require one or more plastic interventions. This study carried out to investigate the effectiveness of High-intensity focused ultrasound (HIFU) in treatment of abdominal skin laxity post sleeve gastrectomy. ### Conditions Module **Conditions:** - Skin Laxity - Gastrostomy ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** OTHER #### Enrollment Info **Count:** 86 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** This group includes 43 patients who have abdominal skin laxity post sleeve gastrectomy and who will receive high intensity focused ultrasound. patients will receive one session; time of session is 40 minutes. **Intervention Names:** - Device: High intensity focused ultrasound **Label:** high intensity focused ultrasound group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** This group includes 43 patients who have abdominal skin laxity post sleeve gastrectomy and who will receive medical topical firming creams. **Intervention Names:** - Other: topical firming creams **Label:** medical topical firming creams group **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - high intensity focused ultrasound group **Description:** Parameters: Transducer frequency 7-2-mhz 1.5-9.0-mm focal depth. The pulse duration for each individual exposure ranged from 25 to 40 milliseconds. Energy per ultrasound pulse ranged from 1.0 to 1.5 J **Name:** High intensity focused ultrasound **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - medical topical firming creams group **Description:** medical topical firming creams **Name:** topical firming creams **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** By Investigator Global Aesthetic Improvement Scale (IGAIS). * The investigator will take clinical photographs using consistent patient positioning, camera settings (Canon EOS 600D, high-resolution setting, 5760 × 3840 pixels, Canon Inc., Tokyo, Japan), and room lighting. * Baseline and post-treatment photographs will randomly displayed, and evaluated by Investigator Global Aesthetic Improvement Scale (IGAIS) scores will be determined using side-by-side comparisons of pretreatment and post 4 weeks of treatment. * The scale ranges from 0 to 3 as 0 = No change, 1 = Mild improvement, 2 = Moderate improvement, 3 = Significant improvement. **Measure:** Assessment othe change in skin laxity **Time Frame:** at baseline and after 4 weeks of intervention **Description:** Assessment of abdominal skin laxity by the modified tissue tonometer : Mark multiple points to be measured. Include an equivalent normal skin point(s). Apply the MTT baseplate directly to the tissue through the holes in the template. Hold MTT vertically, with bubble of spirit level centered. Ensure no extra downward pressure is applied while resting the device on the tissue. Record the depression of the plunger in millimeters after 6 seconds. The patient must be able to remain immobile when the MTT is in contact. Repeat each point three times with at least 2 minutes' "rest" between attempts. Recalibrate the device to zero on a solid surface between each set of measures. To determine longitudinal change in measures, comparison of averaged score for individual points is appropriate and/or analysis of the change of individual sagging points difference from normal skin control point(s). **Measure:** Assessment the change in abdominal skin laxity **Time Frame:** at baseline and after 4 weeks of intervention ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: The subject selection will be according to the following criteria: * Age range between 30-45 years. * Female patients will participate in the study. * All patients will have post sleeve gastrectomy abdominal skin laxity mild to moderate degree according to skin laxity scale Randomized images were evaluated by three clinical specialists for the degree of skin laxity (0-3 scale; 0 = no laxity, 1 = mild, 2 = moderate, 3 = severe). * All patients enrolled to the study will have their informed consent. Exclusion Criteria: The potential participants will be excluded if they meet one of the following criteria: * Age more than 45 years or less than 30 years. * Patients have acute viral diseases, acute tuberculosis, mental disorders * Older people with more extensive photo-aging, severe skin laxity. * Open skin lesions at the target area. * Severe or cystic acne. * Metallic implants in the treatment area. * Genetic disease * Skin infectious diseases. * Sociological diseases. * Pregnant. **Healthy Volunteers:** True **Maximum Age:** 45 Years **Minimum Age:** 30 Years **Sex:** FEMALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Dokki **Country:** Egypt **Facility:** Outpatient clinic faculty of physical therapy cairo university #### Overall Officials **Official 1:** **Affiliation:** Cairo University **Name:** Rania Elhawary **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012873 - Term: Skin Diseases, Genetic - ID: D000030342 - Term: Genetic Diseases, Inborn - ID: D000003240 - Term: Connective Tissue Diseases - ID: D000012871 - Term: Skin Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M6692 - Name: Cutis Laxa - Relevance: HIGH - As Found: Skin Laxity - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown - ID: M15676 - Name: Skin Diseases, Genetic - Relevance: LOW - As Found: Unknown - ID: M23686 - Name: Genetic Diseases, Inborn - Relevance: LOW - As Found: Unknown - ID: M6464 - Name: Connective Tissue Diseases - Relevance: LOW - As Found: Unknown - ID: T1692 - Name: Cutis Laxa - Relevance: HIGH - As Found: Skin Laxity ### Condition Browse Module - Meshes - ID: D000003483 - Term: Cutis Laxa ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05831579 **Brief Title:** Palliative Spatially Fractionated (GRID) Radiotherapy Using Intensity Modulated Proton Therapy **Official Title:** A Phase I Trial of Palliative Spatially Fractionated (GRID) Radiotherapy Using Intensity Modulated Proton Therapy #### Organization Study ID Info **ID:** 202304011 #### Organization **Class:** OTHER **Full Name:** Washington University School of Medicine ### Status Module #### Completion Date **Date:** 2026-05-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-04-29 **Type:** ACTUAL **Last Update Submit Date:** 2024-04-25 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2026-05-31 **Type:** ESTIMATED #### Start Date **Date:** 2023-05-12 **Type:** ACTUAL **Status Verified Date:** 2024-04 #### Study First Post Date **Date:** 2023-04-26 **Type:** ACTUAL **Study First Submit Date:** 2023-04-13 **Study First Submit QC Date:** 2023-04-25 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Washington University School of Medicine #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** True **Is FDA Regulated Drug:** False **Is US Export:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** Spatially fractionated radiotherapy (SFRT or GRID) addresses some limitations of traditional stereotactic body radiation therapy by relying on beam collimation to create high-dose "peaks" and intervening low-dose "valleys" throughout the target volume. Standard palliative radiotherapy regimens provide limited durability of response, and there are challenges with delivery to large tumors or in previously irradiated fields. In this study, Proton GRID radiotherapy will be used to deliver three-fraction palliative radiotherapy to patients with tumors needing palliative radiation. The safety and efficacy of this approach will be assessed. It is hypothesized that GRID is highly effective, immunogenic, and associated with low rates of toxicity. ### Conditions Module **Conditions:** - Unresectable Solid Tumor - Metastatic Cancer **Keywords:** - proton - palliative radiotherapy - spatially fractionated radiotherapy - GRID - stereotactic body radiotherapy (SBRT) - stereotactic ablative radiotherapy (SABR) - cancer ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** SEQUENTIAL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 20 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Radiotherapy will consist of 20 Gy proton GRID radiotherapy x 3 fractions. **Intervention Names:** - Radiation: Proton GRID Radiotherapy **Label:** Cohort A: Reirradiation of Treatment Fields **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Radiotherapy will consist of 20 Gy proton GRID radiotherapy x 3 fractions. **Intervention Names:** - Radiation: Proton GRID Radiotherapy **Label:** Cohort B: De Novo Radiation Treatment Fields **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Cohort A: Reirradiation of Treatment Fields - Cohort B: De Novo Radiation Treatment Fields **Description:** The proton GRID radiotherapy prescription dose is 20 Gy x 3 fractions to the tumor, with an integrated dose of 6 Gy x 3 fractions to the PTV. Treatment to multiple lesions within the PTV is allowed (ex. a dominant lesion plus satellites). Multiple proton GRID radiotherapy plans may be delivered on the same day or different days, but they cannot overlap. **Name:** Proton GRID Radiotherapy **Type:** RADIATION ### Outcomes Module #### Primary Outcomes **Description:** -Grade per CTCAE v5.0. **Measure:** Rate of treatment-related acute toxicity **Time Frame:** From start of treatment through 90 days **Description:** -Grade per CTCAE v5.0. **Measure:** Rate of treatment-related late toxicity **Time Frame:** From day 91 through 12 months #### Secondary Outcomes **Description:** PRO-CTCAE is a standardized inventory to collected patient reported symptomatic adverse events in clinical trials. **Measure:** Change in PRO-CTCAE (General Inventory and Disease Specific Inventory Assessment) **Time Frame:** Baseline, at last day of radiotherapy (day 3), within 2 weeks after completion of radiotherapy (day 14), 30 days, 90 days, 180 days, and 360 days **Description:** PROMIS Global Health is a 10-item patient-reported questionnaire that assesses health related quality of life compared with normal values for the general population with the response options presented as a 5-point (as well as a single 11-point) rating scale. The question that uses an 11-point scale uses a response scale of 0-10 that is recoded to 5 categories (0 = 1; 1-3 = 2; 4-6 = 3; 7-9 = 4; 10 = 5). The results of the questions are used to calculate two summary scores: a Global Physical Health Score and a Global Mental Health score. These scores are then standardized to the general population, using the "T-Score". The average "T-Score" for the United States population is 50 points, with a standard deviation of 10 points. Higher scores are indicative of a healthier patient. **Measure:** Change in PROMIS Global Health **Time Frame:** Baseline, at last day of radiotherapy (day 3), within 2 weeks after completion of radiotherapy (day 14), 30 days, 90 days, 180 days, and 360 days **Measure:** Rate of target lesion local control **Time Frame:** 3 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Histologically or cytologically confirmed cancer diagnosis. * Planning to undergo palliative radiotherapy to unresectable or metastatic target lesion ≥ 4.5 cm in any dimension as measured with radiographic imaging or with calipers by clinical exam. * Cohort A: 10 patients with lesions that have been previously irradiated. * Cohort B: 10 patients with lesions that have not been previously irradiated. * ECOG performance status ≤ 3 * At least 18 years of age. * Radiotherapy is known to be teratogenic. For this reason, women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of the study, and 6 months after completion of the study * Ability to understand and willingness to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable). Exclusion Criteria: * Patients with tumors in need of urgent surgical intervention, such as life-threatening bleeding or those at high risk for pathologic fracture and amenable to surgical intervention. * Patients with a superficial target lesion ≤ 1 cm deep to skin surface who initially had a superficial lesion irradiated, if the target lesion was in the area of the prior irradiation. * Currently receiving any cytotoxic cancer therapy regimens or VEGF inhibitors that will overlap with the proton GRID administration. * Cytotoxic chemotherapy and VEGF inhibitors prior to radiotherapy or planned after radiotherapy delivery are allowed at the discretion of the treating radiation oncologist. This includes continuing a treatment plan which was initiated prior to the start of radiotherapy. A 2-week washout is recommended, but not required. * Pregnant. Women of childbearing potential must have a negative pregnancy test within 20 days of study entry. * Patients with HIV are eligible unless their CD4+ T-cell counts are \< 350 cells/mcL or they have a history of AIDS-defining opportunistic infection within the 12 months prior to registration. Concurrent treatment with effective ART according to DHHS treatment guidelines is recommended. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** apicella@wustl.edu **Name:** Anthony Apicelli, M.D. **Phone:** 314-362-8610 **Role:** CONTACT #### Locations **Location 1:** **City:** Saint Louis **Contacts:** ***Contact 1:*** - **Email:** apicella@wustl.edu - **Name:** Anthony Apicelli, M.D. - **Phone:** 314-362-8610 - **Role:** CONTACT ***Contact 2:*** - **Name:** Anthony Apicelli, M.D. - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 3:*** - **Name:** Clifford Robinson, M.D. - **Role:** SUB_INVESTIGATOR ***Contact 4:*** - **Name:** Stephanie Perkins, M.D. - **Role:** SUB_INVESTIGATOR ***Contact 5:*** - **Name:** Justin Barnes, M.D. - **Role:** SUB_INVESTIGATOR ***Contact 6:*** - **Name:** Nels Knutson, Ph.D. - **Role:** SUB_INVESTIGATOR ***Contact 7:*** - **Name:** Yi Huang, M.S. - **Role:** SUB_INVESTIGATOR ***Contact 8:*** - **Name:** Michael Prusator, Ph.D. - **Role:** SUB_INVESTIGATOR ***Contact 9:*** - **Name:** Tianyu Zhao, Ph.D. - **Role:** SUB_INVESTIGATOR ***Contact 10:*** - **Name:** Xiandong Zhao, Ph.D. - **Role:** SUB_INVESTIGATOR **Country:** United States **Facility:** Washington University School of Medicine **State:** Missouri **Status:** RECRUITING **Zip:** 63110 #### Overall Officials **Official 1:** **Affiliation:** Washington University School of Medicine **Name:** Anthony Apicelli, M.D. **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Access Criteria:** Researchers who provide a methodologically sound proposal may be granted data access. Proposals should be directed to apicellia@wustl.edu. To gain access, data requestors will need to sign a data access agreement. **Description:** Available data will include de-identified individual participant data collected during the trial. **Info Types:** - STUDY_PROTOCOL - SAP **IPD Sharing:** YES **Time Frame:** Beginning 3 months after publication. No end date ### References Module #### See Also Links **Label:** Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine **URL:** http://www.siteman.wustl.edu ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009385 - Term: Neoplastic Processes - ID: D000009369 - Term: Neoplasms - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M12307 - Name: Neoplasm Metastasis - Relevance: HIGH - As Found: Metastatic Cancer - ID: M12330 - Name: Neoplastic Processes - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009362 - Term: Neoplasm Metastasis ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03057379 **Brief Title:** HPV IIS Reminder/Recall- New York State **Official Title:** State Immunization Information Systems to Improve HPV Vaccination Rates #### Organization Study ID Info **ID:** 16-002040 #### Organization **Class:** OTHER **Full Name:** University of California, Los Angeles ### Status Module #### Completion Date **Date:** 2019-01-17 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-04-28 **Type:** ACTUAL **Last Update Submit Date:** 2020-04-24 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-01-17 **Type:** ACTUAL #### Start Date **Date:** 2017-02-27 **Type:** ACTUAL **Status Verified Date:** 2020-04 #### Study First Post Date **Date:** 2017-02-20 **Type:** ACTUAL **Study First Submit Date:** 2017-02-15 **Study First Submit QC Date:** 2017-02-15 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Cancer Institute (NCI) **Class:** OTHER **Name:** University of Colorado, Denver #### Lead Sponsor **Class:** OTHER **Name:** University of California, Los Angeles #### Responsible Party **Investigator Affiliation:** University of California, Los Angeles **Investigator Full Name:** Peter G Szilagyi, MD MPH **Investigator Title:** Professor of Pediatrics **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The overarching goal of this study is to evaluate the effectiveness, cost-effectiveness and sustainability of utilizing statewide Immunization Information Systems (IIS) to conduct centralized reminder/recall (R/R) to improve Human Papiloma Virus (HPV) vaccination rates among adolescents ages 11-17. **Detailed Description:** The overarching goal of this study is to evaluate the effectiveness, cost-effectiveness and sustainability of utilizing statewide Immunization Information Systems (IIS) to conduct centralized reminder/recall (R/R) to improve HPV vaccination rates among adolescents ages 11-17. The investigators will extend previous research on effectiveness of centralized R/R to a new population--adolescents due for HPV vaccine-- and test the use of centralized R/R as a cancer-prevention strategy. Investigators will assess the effect of centralized R/R in two states--one with and one without mandated reporting of vaccinations to IISs, and disseminate IIS R/R to other states. Investigators will implement, evaluate (using the RE-AIM framework 31-36), and disseminate a collaborative, IIS-based centralized HPV vaccine R/R model in which partnerships of public health systems and primary care practices in two states (NY, CO) collaborate to remind parents about HPV vaccination. Specific Aims and hypotheses: Aim #1: Adapt IIS messages and delivery systems (e.g., algorithms) previously developed for centralized R/R for other vaccines to fit HPV vaccine IIS R/R. Aim #2: Assess the impact and cost-effectiveness of centralized IIS-based (IIS-C) autodialer (phone) R/R in increasing vaccine rates \[initial dose (HPV#1) and a complete series (HPV#3)\] among teens. Conduct a pragmatic trial, to assess the impact and cost effectiveness of centralized IIS-based (IIS-C) autodialer (phone) R/R in increasing initiation and completion rates for the HPV vaccine series in adolescents ages 11-17 years. The investigators will use a within-practice design, randomizing patients within randomly selected primary care practices to IIS-C R/R (1, 2, or 3 reminders per dose) compared to usual care (0 reminders from this study). The investigators will apply the RE-AIM framework to evaluate the reach, effectiveness/cost effectiveness, adoption, and implementation of IIS-C R/R. Hypothesis 2a: IIS-C R/R will result in higher HPV vaccination rates than usual care. Hypothesis 2b: IIS-C R/R will result in higher HPV vaccination rates than usual care in key subgroups (males and females, younger and older teens, urban//rural teens). Hypothesis 2c: IIS-C R/R will be more cost-effective (cost/vaccine received) than usual care. Aim #3: Disseminate IIS-C R/R across NY and CO and pilot in four IISs: (a) Develop an IIS-C HPV R/R toolkit, (b) Use a technical advisory group, (c) Initiate IIS-C R/R in four other IISs \[Yr. 4\]. By the end of the study investigators will have a feasible, sustainable, cost-effective model for HPV vaccine reminders that can be used nationally to prevent cervical cancer and other HPV-related cancers. ### Conditions Module **Conditions:** - Reminder Systems **Keywords:** - HPV vaccine - Immunization registry - Reminder recall ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Participants will be randomized to one of four arms to receive 0,1,2,or 3 reminders for overdue HPV vaccine doses. ##### Masking Info **Masking:** DOUBLE **Who Masked:** - CARE_PROVIDER - OUTCOMES_ASSESSOR **Primary Purpose:** HEALTH_SERVICES_RESEARCH #### Enrollment Info **Count:** 30616 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Usual source of care **Label:** Control arm **Type:** NO_INTERVENTION #### Arm Group 2 **Description:** Sending up to one recall notice per dose of HPV vaccine needed **Intervention Names:** - Other: Vaccine Reminder Recall **Label:** 1 R/R per Dose **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Sending up to two recall notice per dose of HPV vaccine needed **Intervention Names:** - Other: Vaccine Reminder Recall **Label:** 2 R/R per dose **Type:** EXPERIMENTAL #### Arm Group 4 **Description:** Sending up to three recall notice per dose of HPV vaccine needed **Intervention Names:** - Other: Vaccine Reminder Recall **Label:** 3 R/R per dose **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - 1 R/R per Dose - 2 R/R per dose - 3 R/R per dose **Description:** The investigators will be sending recall notices via phone call to 11-17 year olds who are eligible but lacking HPV vaccine doses recorded in the New York State Immunization Information System (NYSIIS). The investigators will be testing the effectiveness and cost effectiveness of up to 3 notices per dose with usual care (no intervention) **Name:** Vaccine Reminder Recall **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Did the adolescent initiate the HPV vaccine series? The investigators are allowing up to 5 months after the initial recall notice for the adolescent to make an appointment and receive their dose. After that time frame, the investigators will not attribute vaccination to the recall. Receipt will be documented by providers within the state immunization registry via electronic medical record (EMR) direct transfer or manual entry. **Measure:** Initiation of the HPV vaccine series (receipt and documentation of initial dose of vaccine series within immunization registry) **Time Frame:** 5 months from initial recall notices for the first dose **Description:** Did those eligible to complete the HPV vaccine series within the time frame of the study do so? The investigators are allowing up to 5 months after the first recall notice of the last dose (could be dose 2 or dose 3, depending on the age at which they received their first dose) for adolescents to complete their vaccination series. After that time frame, the investigators will not attribute vaccination to the recall. Receipt will be documented by providers within the state immunization registry via EMR direct transfer or manual entry. **Measure:** Completion of the HPV vaccine series (receipt and documentation of final dose of vaccine series within immunization registry) **Time Frame:** 5 months from first reminder received for last dose #### Secondary Outcomes **Description:** Is 1, 2, or 3 reminders per arm most effective and cost effective for initiation. Only those who need an initial dose at baseline will be eligible for this outcome. We will compare the arms (receipt of zero, 1, 2 or 3 reminders) to see if there are any differences between the arms in terms of receipt of the 1st dose of HPV vaccine **Measure:** Differences between arms - initiation **Time Frame:** 5 months **Description:** Is 1, 2, or 3 reminders per arm most effective and cost effective for completion. Because the second or third dose of the series is dependent on when the adolescent receives their initial dose, the investigators will be following eligible adolescents for 20 months to allow time for those who did not respond to the recall for the initial dose right away to be able to complete the series within the time frame. However, receipt of the completion dose will only be attributed to the recall if it was received within 5 months of the 1st recall for the completion dose. Receipt will be documented by providers within the state immunization registry via EMR direct transfer or manual entry. **Measure:** Differences between arms - completion **Time Frame:** 20 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 11 through 17 years of age * patient of a participating practice (practices randomly selected) * is either due for an HPV dose at baseline, or * has initiated but not yet completed the HPV series at baseline Exclusion Criteria: * has completed the HPV vaccination series **Maximum Age:** 17 Years **Minimum Age:** 11 Years **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Locations **Location 1:** **City:** Los Angeles **Country:** United States **Facility:** University of California LA **State:** California **Zip:** 99095 ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## Derived Section ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M17360 - Name: Vaccines - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04177979 **Acronym:** SRMC **Brief Title:** Efficacy of Near-Assisted Learning (NAL) in Improving Students' Objective Structured Clinical Examination (OSCE) Grades **Official Title:** Efficacy of Near-Assisted Learning (NAL) in Improving Students' OSCE Grades: A Single-Blinded RCT #### Organization Study ID Info **ID:** SRMCOSCE #### Organization **Class:** OTHER **Full Name:** Sulaiman AlRajhi Colleges ### Status Module #### Completion Date **Date:** 2019-04-30 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2019-11-26 **Type:** ACTUAL **Last Update Submit Date:** 2019-11-23 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-04-30 **Type:** ACTUAL #### Start Date **Date:** 2019-04-06 **Type:** ACTUAL **Status Verified Date:** 2019-11 #### Study First Post Date **Date:** 2019-11-26 **Type:** ACTUAL **Study First Submit Date:** 2019-11-16 **Study First Submit QC Date:** 2019-11-23 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Sulaiman AlRajhi Colleges #### Responsible Party **Investigator Affiliation:** Sulaiman AlRajhi Colleges **Investigator Full Name:** Abdulaziz Alothman **Investigator Title:** Head of medical education department **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** This study aims to evaluate the impact of near-assisted learning (NAL) on first-year medical students' objective structured clinical examination (OSCE) grades in a problem-based learning (PBL) environment. **Detailed Description:** In this randomized controlled trial, 1st year medical students were recruited in the study along with selected highly-performing, trained peer-instructors. The participants were randomly allocated to two groups, an intervention group supervised by the instructors and the control group that had to practice independently. They would attend OSCE sessions and had their performance evaluated, later on, in an exam. In addition to comparing their performance with each other, scores of the previous batch were considered in the comparison. ### Conditions Module **Conditions:** - Cost - Problem-based Learning - Peer Group **Keywords:** - Near-assisted learning - Cost - Education environment - OSCE - Problem-based learning ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Randomized single blinded trial ##### Masking Info **Masking:** SINGLE **Masking Description:** The OSCE assessors were blinded **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** OTHER #### Enrollment Info **Count:** 48 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants in this arm were supervised by the trained peer-instructors. **Intervention Names:** - Behavioral: Near-assisted learning through trained peers **Label:** Near assisted learning group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants in this arm were practicing independently. They were not supervised by any instructors. **Label:** Self directed learning group **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Near assisted learning group **Description:** Students randomly allocated to this group were overseen during sessions by trained peer instructors. **Name:** Near-assisted learning through trained peers **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** After the sessions, the study sample participated in the final osce exam. Their scores were compared. The minimum value was 0 while the maximum value was 100. The higher the score the better students had performed **Measure:** objective structured clinical examination (OSCE) Score **Time Frame:** Less than a month ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 1st year medical students. * Medical students of Sulaiman Alrajhi Colleges. * Males. Exclusion Criteria: * Students of other academic levels. * 1st year medical students of other universities. * Females. **Gender Based:** True **Gender Description:** Only males were recruited, due to sociocultural restrictions in Saudi Arabia **Healthy Volunteers:** True **Sex:** MALE **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Al Bukairiyah **Country:** Saudi Arabia **Facility:** Sulaiman AlRajhi Colleges **State:** Qassim **Zip:** 51941, PO Box 777 ### IPD Sharing Statement Module **Description:** Might be shared through the university webpage. **IPD Sharing:** NO ### References Module #### References **Citation:** Tolsgaard MG, Gustafsson A, Rasmussen MB, Hoiby P, Muller CG, Ringsted C. Student teachers can be as good as associate professors in teaching clinical skills. Med Teach. 2007 Sep;29(6):553-7. doi: 10.1080/01421590701682550. **PMID:** 17978968 **Citation:** Shah I, Mahboob U, Shah S. Effectiveness Of Horizontal Peer-Assisted Learning In Physical Examination Performance. J Ayub Med Coll Abbottabad. 2017 Oct-Dec;29(4):559-565. **PMID:** 29330977 **Citation:** Blank WA, Blankenfeld H, Vogelmann R, Linde K, Schneider A. Can near-peer medical students effectively teach a new curriculum in physical examination? BMC Med Educ. 2013 Dec 11;13:165. doi: 10.1186/1472-6920-13-165. **PMID:** 24325639 **Citation:** Riaz I. Peer assisted versus expert assisted learning: a comparison of effectiveness in terms of academic scores. J Coll Physicians Surg Pak. 2014 Nov;24(11):825-9. **PMID:** 25404441 **Citation:** Haist SA, Wilson JF, Brigham NL, Fosson SE, Blue AV. Comparing fourth-year medical students with faculty in the teaching of physical examination skills to first-year students. Acad Med. 1998 Feb;73(2):198-200. doi: 10.1097/00001888-199802000-00020. **PMID:** 9484194 **Citation:** Field M, Burke JM, McAllister D, Lloyd DM. Peer-assisted learning: a novel approach to clinical skills learning for medical students. Med Educ. 2007 Apr;41(4):411-8. doi: 10.1111/j.1365-2929.2007.02713.x. **PMID:** 17430287 **Citation:** Tai JH, Canny BJ, Haines TP, Molloy EK. The role of peer-assisted learning in building evaluative judgement: opportunities in clinical medical education. Adv Health Sci Educ Theory Pract. 2016 Aug;21(3):659-76. doi: 10.1007/s10459-015-9659-0. Epub 2015 Dec 12. **PMID:** 26662035 **Citation:** Liew SC, Sow CF, Sidhu J, Nadarajah VD. The near-peer tutoring programme: embracing the 'doctors-to-teach' philosophy--a comparison of the effects of participation between the senior and junior near-peer tutors. Med Educ Online. 2015 Sep 8;20:27959. doi: 10.3402/meo.v20.27959. eCollection 2015. **PMID:** 26356229 **Citation:** Tai J, Molloy E, Haines T, Canny B. Same-level peer-assisted learning in medical clinical placements: a narrative systematic review. Med Educ. 2016 Apr;50(4):469-84. doi: 10.1111/medu.12898. **PMID:** 26995485 **Citation:** Khaw C, Raw L. The outcomes and acceptability of near-peer teaching among medical students in clinical skills. Int J Med Educ. 2016 Jun 12;7:188-94. doi: 10.5116/ijme.5749.7b8b. **PMID:** 27295403 **Citation:** Cole JD, Ruble MJ, Donnelly J, Groves B. Peer-assisted Learning: Clinical Skills Training for Pharmacy Students. Am J Pharm Educ. 2018 Aug;82(6):6511. doi: 10.5688/ajpe6511. **PMID:** 30181672 **Citation:** Burke J, Fayaz S, Graham K, Matthew R, Field M. Peer-assisted learning in the acquisition of clinical skills: a supplementary approach to musculoskeletal system training. Med Teach. 2007 Sep;29(6):577-82. doi: 10.1080/01421590701469867. **PMID:** 17978969 **Citation:** Silbert BI, Lake FR. Peer-assisted learning in teaching clinical examination to junior medical students. Med Teach. 2012;34(5):392-7. doi: 10.3109/0142159X.2012.668240. Epub 2012 Apr 3. **PMID:** 22471912 #### See Also Links **Label:** A non-pubmed indexed paper named: Improving Medical Student Confidence in Clinical Skills through a Peer-Facilitated Workshop **URL:** http://www.semanticscholar.org/paper/Improving-Medical-Student-Confidence-in-Clinical-a-Flynn-Williams/b5b6f1171bbd7c3e5d33ebb9644eb75aaaa5f8d1 ## Document Section ### Large Document Module #### Large Docs - Date: 2019-10-04 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 142527 - Type Abbrev: Prot_SAP - Upload Date: 2019-11-10T13:34 ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02130479 **Brief Title:** Testing a Promising Treatment for Youth Substance Abuse in a Community Setting **Official Title:** Testing a Promising Treatment for Youth Substance Abuse in a Community Setting #### Organization Study ID Info **ID:** R01DA034064-01A1 **Link:** https://reporter.nih.gov/quickSearch/R01DA034064-01A1 **Type:** NIH #### Organization **Class:** OTHER **Full Name:** Medical University of South Carolina ### Status Module #### Completion Date **Date:** 2018-06 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-07-06 **Type:** ACTUAL **Last Update Submit Date:** 2018-07-04 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-06 **Type:** ACTUAL #### Start Date **Date:** 2014-04 **Status Verified Date:** 2018-07 #### Study First Post Date **Date:** 2014-05-05 **Type:** ESTIMATED **Study First Submit Date:** 2014-05-01 **Study First Submit QC Date:** 2014-05-01 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Medical University of South Carolina #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** This study aims to address a serious public health problem (i.e., substance abusing adolescents) by testing the effectiveness of a promising substance abuse treatment implemented in a community-based treatment setting (CM-FAM, a family-based contingency management intervention) in comparison to usual treatment services. **Detailed Description:** The overriding purpose of the randomized trial is to examine the effectiveness of a promising and efficient outpatient treatment of adolescent substance abuse delivered in a community-based treatment setting. Although several evidence-based treatments of adolescent substance abuse are emerging, none have experienced widespread adoption in community settings. Thus, as noted by the Institute of Medicine (1998) more than a decade ago and reiterated more recently, a considerable science-service gap exists in regards to treatment of substance abuse in adolescents and adults. For the proposed study, 204 adolescents meeting diagnostic criteria for substance abuse or dependence will be randomized to either the Contingency Management-Family Engagement (CM-FAM) or Treatment as Usual (TAU) conditions. A multimethod, multirespondent approach will be used to track clinical outcomes at 3, 6, 9, 12, and 18 months post recruitment. Clinical level outcomes pertain to youth substance use, criminal behavior, mental health functioning, and key mediators of serious antisocial behavior in adolescents (e.g., self-control, parental supervision, association with deviant peers). In addition, the incremental cost of CM-FAM will be determined for use in cost effectiveness analyses. Aim 1: Over an 18-month post-recruitment follow-up, determine the relative effectiveness of CM-FAM vs. TAU in reducing adolescent participants' substance use, criminal activity (including incarceration), and mental health symptoms; and evaluate the cost effectiveness of CM-FAM in achieving these outcomes. Aim 2: Examine possible moderators and mediators of intervention effectiveness. Moderator variables will include youth demographic and clinical (e.g., co-occurring disorders) characteristics. Mediator variables will include measures of self-control, parenting, and association with deviant peers - constructs targeted by CM-FAM. ### Conditions Module **Conditions:** - Substance Abuse - Delinquency - Mental Health ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 101 **Type:** ACTUAL **Phases:** - PHASE1 - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The Contingency Management-Family Engagement or CM-FAM model integrates behavioral (e.g., drug testing linked with consequences) and cognitive behavioral (e.g., functional analyses of drug use, self-management and drug refusal skills training) strategies based on the Community Reinforcement Approach with effective family engagement strategies used in Multisystemic Therapy. **Intervention Names:** - Behavioral: Contingency Management-Family Engagement **Label:** Contingency Management-Family Engagement **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Standard community-based substance abuse treatment services. **Intervention Names:** - Behavioral: Treatment as Usual **Label:** Treatment as Usual **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Contingency Management-Family Engagement **Name:** Contingency Management-Family Engagement **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - Treatment as Usual **Name:** Treatment as Usual **Type:** BEHAVIORAL ### Outcomes Module #### Other Outcomes **Measure:** Incremental costs associated with CM-FAM as measured by the Drug Abuse Treatment Cost Analysis Program **Time Frame:** 6, 18, and 30 months following initiation of CM-FAM delivery **Measure:** Change in youths' treatment duration and rates of completion as measured by clinic records **Time Frame:** 6, 18, and 30 months following initiation of CM-FAM delivery **Measure:** Change in therapists' perceptions of the functioning of their treatment facility as measured by the Organizational Readiness for Change Scale **Time Frame:** Therapist baseline, 6, 12, 18, 24, & 30 months post-baseline #### Primary Outcomes **Measure:** Change in youths' substance abuse frequency as measured by Urine Drug Screens and the Global Appraisal of Individual Needs Scale **Time Frame:** Youth baseline, 3, 6, 9, 12, & 18 months post-baseline **Measure:** Change in youths' delinquent behavior as measured by Juvenile Justice Archival Records and the Self-Report Delinquency Scale **Time Frame:** Youth baseline, 3, 6, 9, 12, & 18 months post-baseline **Measure:** Change in youths' mental health functioning as measured by the Brief Problem Checklist **Time Frame:** Youth baseline, 3, 6, 9, 12, & 18 months post-baseline #### Secondary Outcomes **Measure:** Change in youths' peer relations as measured by the Peer Delinquency and Drug Activities Scales, the Conventional Activities of Peers Scale, and the Bad Friends Scale from the Pittsburgh Youth Study **Time Frame:** Youth baseline, 3, 6, 9, 12, & 18 months post-baseline **Measure:** Change in family functioning as measured by the Caregiver Supervision Scale, the Discipline Scale, and the Communication Form from the Pittsburgh Youth Study **Time Frame:** Youth baseline, 3, 6, 9, 12, & 18 months post-baseline **Measure:** Change in youths' self-control as measured by the Good Behavioral Self-Control and the Poor Behavioral Regulation Scales **Time Frame:** Youth baseline, 3, 6, 9, 12, & 18 months post-baseline ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age of 12-17 years * Meeting criteria for substance use or abuse. **Healthy Volunteers:** True **Maximum Age:** 17 Years **Minimum Age:** 12 Years **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Locations **Location 1:** **City:** Florence **Country:** United States **Facility:** Circle Park Behavioral Health Services **State:** South Carolina **Zip:** 29501 #### Overall Officials **Official 1:** **Affiliation:** Medical University of South Carolina **Name:** Scott Henggeler, Ph.D. **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000064419 - Term: Chemically-Induced Disorders - ID: D000001523 - Term: Mental Disorders ### Condition Browse Module - Browse Branches - Abbrev: BC25 - Name: Substance Related Disorders - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M21837 - Name: Substance-Related Disorders - Relevance: HIGH - As Found: Substance Abuse - ID: M30302 - Name: Chemically-Induced Disorders - Relevance: LOW - As Found: Unknown - ID: M4815 - Name: Mental Disorders - Relevance: LOW - As Found: Unknown - ID: M14473 - Name: Psychotic Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000019966 - Term: Substance-Related Disorders ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06151379 **Brief Title:** Muscle Parameters and Pathological Response in Breast Cancer Patients **Official Title:** The Relation Between the Initial Value of the Skeletal Mass Index and Pathological Response #### Organization Study ID Info **ID:** AcıbademAH #### Organization **Class:** OTHER **Full Name:** Acıbadem Atunizade Hospital ### Status Module #### Completion Date **Date:** 2023-10-20 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-11-30 **Type:** ACTUAL **Last Update Submit Date:** 2023-11-21 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2023-09-30 **Type:** ACTUAL #### Start Date **Date:** 2023-03-01 **Type:** ACTUAL **Status Verified Date:** 2023-11 #### Study First Post Date **Date:** 2023-11-30 **Type:** ACTUAL **Study First Submit Date:** 2023-11-04 **Study First Submit QC Date:** 2023-11-21 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Acıbadem Atunizade Hospital #### Responsible Party **Investigator Affiliation:** Acıbadem Atunizade Hospital **Investigator Full Name:** Ebru Yilmaz **Investigator Title:** Principal Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The investigators aimed to accurately measure muscle mass and predict pCR to NAC and its effect on clinical outcomes. **Detailed Description:** In patients with operable breast cancer (BC) receiving neoadjuvant chemotherapy (NAC), the pathological response rate was found to be related to body composition. The success of complete pathologic response (pCR) is a known prognostic factor in BC patients treated with NAC. The investigators aimed to accurately measure muscle mass and predict pCR to NAC and its effect on clinical outcomes. ### Conditions Module **Conditions:** - Neoadjuvant Chemotherapy **Keywords:** - operable breast cancer - skeletal mass index - neoadjuvant chemotherapy - pathologic response - L3 vertebra ### Design Module #### Design Info **Observational Model:** CASE_ONLY **Time Perspective:** RETROSPECTIVE #### Enrollment Info **Count:** 69 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patient with complete pathological response **Intervention Names:** - Other: Neoadjuvant therapy **Label:** Pathologic complete response (pCR) #### Arm Group 2 **Description:** Patient with partial pathological response **Intervention Names:** - Other: Neoadjuvant therapy **Label:** Pathologic incomplete response (non-pCR) ### Interventions #### Intervention 1 **Arm Group Labels:** - Pathologic complete response (pCR) - Pathologic incomplete response (non-pCR) **Description:** To measure muscle mass and predict pCR to NAC and its effect on clinical outcomes. **Name:** Neoadjuvant therapy **Type:** OTHER ### Outcomes Module #### Other Outcomes **Description:** Residual cancer burden (RCB) is estimated from routine pathologic sections of the primary breast tumor site and the regional lymph nodes after the completion of neoadjuvant therapy. **Measure:** Residual cancer burden **Time Frame:** Through study completion, an average of 1 year. #### Primary Outcomes **Description:** skeletal muscle area (cm2)/height (m2) et L3 level **Measure:** Sarcopenic index **Time Frame:** Through study completion, an average of 1 year. #### Secondary Outcomes **Description:** weight (kg)/height (m2) **Measure:** Body mass index **Time Frame:** Through study completion, an average of 1 year. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Early and locally advanced operated breast cancer patients receiving neoadjuvant chemotherapy. Exclusion Criteria: * Patients who did not receive neoadjuvant chemotherapy, had metastatic breast cancer and whose radiological images were not available. **Maximum Age:** 72 Years **Minimum Age:** 29 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Early and locally advanced operated breast cancer patients receiving neoadjuvant chemotherapy. ### Contacts Locations Module #### Locations **Location 1:** **City:** Istanbul **Country:** Turkey **Facility:** AcıbademAH **Zip:** 34782 #### Overall Officials **Official 1:** **Affiliation:** Acıbadem Atunizade Hospital **Name:** Aysun Isiklar, M.D. **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M5220 - Name: Breast Neoplasms - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01649479 **Acronym:** MENT-APL-O **Brief Title:** Comparative Prevalence of Psychiatric Manifestations in Purely Obstetrical Antiphospholipid Syndrome **Official Title:** Comparative Prevalence of Psychiatric Manifestations in Purely Obstetrical Antiphospholipid Syndrome #### Organization Study ID Info **ID:** PHRC-I/2012/FC-01 #### Organization **Class:** OTHER **Full Name:** Centre Hospitalier Universitaire de Nīmes #### Secondary ID Infos **Domain:** RCB number **ID:** 2012-A00705-38 **Type:** OTHER ### Status Module #### Completion Date **Date:** 2013-09 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2015-03-25 **Type:** ESTIMATED **Last Update Submit Date:** 2015-03-24 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2013-09 **Type:** ACTUAL #### Start Date **Date:** 2013-04 **Status Verified Date:** 2015-03 #### Study First Post Date **Date:** 2012-07-25 **Type:** ESTIMATED **Study First Submit Date:** 2012-07-23 **Study First Submit QC Date:** 2012-07-24 **Why Stopped:** Impossible to include patients at a correct rate; patients don't want to come back so they refuse participation. ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Centre Hospitalier Universitaire de Nīmes #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The main objective of this study is to estimate the lifetime prevalence of major psychiatric disorders (axis I DSM-IV; Diagnostic and Statistical Manual of Mental Disorders, version IV) in a large sample of patients with developed clinical signs of pure obstetrical antiphospholipid syndrome (suspected APS). **Detailed Description:** The secondary objectives of this study are: A. To compare the lifetime prevalence of these major disorders between groups; B. To assess the association of different, targeted, qualitative biomarkers with clinical symptomatology; C. To assess the association between the presence of "transitory APS" and the presence of psychiatric disorders; D. Estimate and compare the current prevalence (= the day of assessment) of major psychiatric disorders in the sample of patients who developed clinical signs of obstetrical APS; E. Estimate the current prevalence (= the day of assessment) and intensity of major depressive episodes (MDE) in the sample of patients; F. Compare the prevalence of current MDE and the intensity of depressive symptoms present between groups; G. Estimate and compare the (lifetime and current) prevalence by category of psychiatric disorders (psychotic, anxiety, mood, etc..) in the APS group with that in the thrombophilic group and the remaining group; H. To study the average age of onset of psychiatric disorders and clinical manifestations of APS in the sample of patients who developed clinical signs of obstetrical APS; I. Compare the mean ages between groups; J. Compare the mean age at onset of psychiatric disorders with the average age of the first clinical manifestation of the disease in the group of women with APS. ### Conditions Module **Conditions:** - Antiphospholipid Syndrome **Keywords:** - Obstetrical antiphospholipid syndrome - Thrombophilia ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Count:** 20 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The patients included in this study are women actively addressed to the participating departments because of clinical symptoms corresponding to suspected obstetrical anti-phospholipid syndrome. Bloodwork later confirms that these patients have APS. All patients included in this study will have the following interventions: * antiphospholipid antibody tests * thrombophilia bloodwork * psychiatric evaluation **Intervention Names:** - Biological: Antiphospholipid antibody tests - Biological: Thrombophilia bloodwork - Other: Psychiatric evaluation **Label:** Suspected Obstetrical APS; confirmed APS **Type:** OTHER #### Arm Group 2 **Description:** The patients included in this study are women actively addressed to the participating departments because of clinical symptoms corresponding to suspected obstetrical anti-phospholipid syndrome. Bloodwork later confirms that these patients are thrombophilic. All patients included in this study will have the following interventions: * antiphospholipid antibody tests * thrombophilia bloodwork * psychiatric evaluation **Intervention Names:** - Biological: Antiphospholipid antibody tests - Biological: Thrombophilia bloodwork - Other: Psychiatric evaluation **Label:** Sus. Obst. APS, confirmed thrombophilia **Type:** OTHER #### Arm Group 3 **Description:** The patients included in this study are women actively addressed to the participating departments because of clinical symptoms corresponding to suspected obstetrical anti-phospholipid syndrome. Bloodwork cannot confirm APS, nor thrombophilia. All patients included in this study will have the following interventions: * antiphospholipid antibody tests * thrombophilia bloodwork * psychiatric evaluation **Intervention Names:** - Biological: Antiphospholipid antibody tests - Biological: Thrombophilia bloodwork - Other: Psychiatric evaluation **Label:** Suspected Obstectrical APS; unconfirmed **Type:** OTHER ### Interventions #### Intervention 1 **Arm Group Labels:** - Sus. Obst. APS, confirmed thrombophilia - Suspected Obstectrical APS; unconfirmed - Suspected Obstetrical APS; confirmed APS **Description:** Each patient will be tested for antiphospholipid antibodies. **Name:** Antiphospholipid antibody tests **Type:** BIOLOGICAL #### Intervention 2 **Arm Group Labels:** - Sus. Obst. APS, confirmed thrombophilia - Suspected Obstectrical APS; unconfirmed - Suspected Obstetrical APS; confirmed APS **Description:** Bloodwork will be drawn up for: * antithrombin, protein C, protein S * Factor V Leiden polymorphisms (F5 1691A) * prothrombin 20210A gene polymorphism (F2 20210A) * JAK2 617F Mutation * Homocysteine * Factor VIII **Name:** Thrombophilia bloodwork **Type:** BIOLOGICAL #### Intervention 3 **Arm Group Labels:** - Sus. Obst. APS, confirmed thrombophilia - Suspected Obstectrical APS; unconfirmed - Suspected Obstetrical APS; confirmed APS **Description:** During this consultation, the Mini International Neuropsychiatric Interview will be used to screen for psychiatric symptoms. Should the latter be detected, a further consult with a psychiatrist or a psychologist will be organized; this second consult will include the Mood Disorder Questionnaire (MDQ), the Beck Depression Inventory (BDI), the Inventory for Depressive Symptomatology - Clinician (IDS-C) and the Structured Clinical Interview for Disorders (SCID, DSM-IV). **Name:** Psychiatric evaluation **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** The Mini International Neuropsychiatric Interview (MINI 6) will be used to determined the presence/absence of (lifetime) psychiatric symptoms. **Measure:** presence/absence of (lifetime) psychiatric symptoms **Time Frame:** baseline (transversal); Day 0 #### Secondary Outcomes **Description:** The Mini International Neuropsychiatric Interview (MINI 6) will be used to determined the presence/absence of (current) psychiatric symptoms. **Measure:** presence/absence of (current) psychiatric symptoms **Time Frame:** baseline (transversal); Day 0 **Description:** Structured Clinical Interview for Disorders (SCID-1) score for patients with a positive MINI evaluation. **Measure:** SCID-1 score **Time Frame:** baseline (transversal); Day 0 or up to Day 15 **Description:** Mood Disorder Questionnaire score **Measure:** MDQ score **Time Frame:** baseline (transversal); Day 0 **Description:** The Beck Depression Inventory (BDI) score for currently depressed patients only. **Measure:** BDI score **Time Frame:** baseline (transversal); Day 0 or up to Day 15 **Description:** Inventory of Depressive Symptomatology (IDS-C) for currently depressed patients. **Measure:** IDS-C score **Time Frame:** baseline (transversal); Day 0 or up Day 15 **Measure:** presence/absence of lupus anticoagulant **Time Frame:** baseline (transversal); Day 0 **Measure:** presence/absence of anticardiolipid antibodies **Time Frame:** baseline (transversal); Day 0 **Measure:** presence/absence of anti-beta2-glycoprotein 1 antibodies **Time Frame:** baseline (transversal); Day 0 **Measure:** deficit in antithrombin: yes/no **Time Frame:** baseline (transversal); Day 0 **Measure:** Deficit in protein C: yes/no **Time Frame:** baseline (transversal); Day 0 **Measure:** Deficit in protein S: yes/no **Time Frame:** baseline (transversal); Day 0 **Description:** Excess of coagulation factor VIII? **Measure:** Excess of FVIII: yes/no **Time Frame:** baseline (transversal); Day 0 **Measure:** Excess of homocystein? yes/no **Time Frame:** baseline (transversal); Day 0 **Description:** F5 1691A: allele 1691A for the factor V leiden gene **Measure:** presence/absence of allele F5 1691A **Time Frame:** baseline (transversal); Day 0 **Description:** F2 20210A: allele 20210A for the prothrombin gene **Measure:** presence/absence of allele F2 20210A **Time Frame:** baseline (transversal); Day 0 **Description:** JAK2 617F: 617f mutation at the jak2 gene **Measure:** presence/absence of allele JAK2 617F **Time Frame:** baseline (transversal); Day 0 **Description:** in years **Measure:** Age at beginning of psychiatric symptoms **Time Frame:** baseline (transversal); Day 0 **Description:** in years **Measure:** Age at beginning of APL or thrombophilia symptoms **Time Frame:** baseline (transversal); Day 0 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * The patient must have given his/her informed and signed consent * The patient must be insured or beneficiary of a health insurance plan * Not postmenopausal * Able to understand the nature, purpose and methodology of the study and agreed to cooperate in clinical and biological assessments * Available for 12 weeks of follow-up * Isolated obstetric morbidity, defined by at least one of the following criteria: * at least three consecutive episodes of unexplained, early, embryonic miscarriage, which occurred before the 10th week of pregnancy, with normal maternal anatomic and hormonal assessment, normal karyotypes for both biological parents; * at least one unexplained fetal death, defined as occurring after the 10th week of pregnancy, involving a morphologically normal fetus as documented by ultrasound examination or direct examination of the conceptus; * at least one premature birth of a morphologically normal fetus before the 34th week of pregnancy, because of: (1) pre-eclampsia, severe or not, according to the American College of Obstetrics and Gynecology, ACOG, 2002; (2)documented placental insufficiency, defined by the following parameters: (2a) abnormal or non-reassuring fetal monitoring exam, in general a non-reactive absence-of-fetal-stress test (fetal monitoring), suggesting fetal hypoxemia; (2b) a Doppler examination of uterine arteries suggesting fetal hypoxemia, ie the absence of end-diastolic flow in the umbilical arteries; (2c) oligohydramnios, that is to say, an amniotic flow index \<5 cm; (2d) indexed birth weight for gestational age and sex below the 10th percentile. * Patient willing to accept psychological and medical care over the long term Exclusion Criteria: * The patient is participating in another study * The patient is in an exclusion period determined by a previous study * The patient is under judicial protection, under tutorship or curatorship * The patient refuses to sign the consent * It is impossible to correctly inform the patient * The patient is pregnant, parturient or breastfeeding * Systemic vascular morbidity, defined by the following criteria: (1) Any personal history of venous thromboembolism, defined by the occurrence of deep phlebitis and / or a pulmonary embolism, diagnosed by means of objective exploration ; (2)Any personal history of superficial venous thrombosis; (3) Any personal history of clinical, symptomatic relapses of arterial insufficiency - the latter may be cerebro vascular in nature (transient ischemic attack, stroke, etc..), coronary in nature (angina, myocardial infarction, etc..) or otherwise (claudication mesenteric, etc.), and objectively diagnosed. * Systemic inflammatory disease: any history of systemic disease, lupus erythematosus or other connective, rheumatoid arthritis * Any history of neoplastic disease * Chronic antithrombotic treatment taken before the occurrence of obstetrical complications * Any chronic immunosuppressive therapy or immunomodulatory therapy (eg corticosteroids, hydroxochloroquine or intravenous immunoglobulins) * Fetal loss can be explained by infectious, metabolic (including rates of fasting blood glucose\> 7 mmol / L), anatomical or hormonal factors * History of infection with hepatitis B, hepatitis C or HIV * Taking antipsychotic treatment potentially implicated in biological autoimmune abnormalities **Maximum Age:** 45 Years **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Marseille Cedex 20 **Country:** France **Facility:** APHM - Hôpital Nord **Zip:** 13915 **Location 2:** **City:** Marseille Cedex 5 **Country:** France **Facility:** APHM - Hôpital de la Conception **Zip:** 13385 **Location 3:** **City:** Marseille cedex 5 **Country:** France **Facility:** APHM - Hôpital La Timone Adultes **Zip:** 13385 **Location 4:** **City:** Montpellier **Country:** France **Facility:** CHU de Montpellier - Hôpital Saint-Eloi **Zip:** 34295 **Location 5:** **City:** Nîmes Cedex 09 **Country:** France **Facility:** CHU de Nîmes - Hôpital Universitaire Carémeau **Zip:** 30029 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004194 - Term: Disease - ID: D000010335 - Term: Pathologic Processes - ID: D000001327 - Term: Autoimmune Diseases - ID: D000007154 - Term: Immune System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: BC15 - Name: Blood and Lymph Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M16355 - Name: Syndrome - Relevance: HIGH - As Found: Syndrome - ID: M19097 - Name: Antiphospholipid Syndrome - Relevance: HIGH - As Found: Antiphospholipid Syndrome - ID: M21737 - Name: Thrombophilia - Relevance: LOW - As Found: Unknown - ID: M4629 - Name: Autoimmune Diseases - Relevance: LOW - As Found: Unknown - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown - ID: T438 - Name: Antiphospholipid Syndrome - Relevance: HIGH - As Found: Antiphospholipid Syndrome ### Condition Browse Module - Meshes - ID: D000016736 - Term: Antiphospholipid Syndrome - ID: D000013577 - Term: Syndrome ### Intervention Browse Module - Ancestors - ID: D000007155 - Term: Immunologic Factors - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Coag - Name: Coagulants - Abbrev: AnCoag - Name: Anticoagulants - Abbrev: FiAg - Name: Fibrinolytic Agents ### Intervention Browse Module - Browse Leaves - ID: M4225 - Name: Antibodies - Relevance: HIGH - As Found: Program - ID: M10184 - Name: Immunoglobulins - Relevance: LOW - As Found: Unknown - ID: M16676 - Name: Thrombin - Relevance: LOW - As Found: Unknown - ID: M4307 - Name: Antithrombins - Relevance: LOW - As Found: Unknown - ID: M4306 - Name: Antithrombin III - Relevance: LOW - As Found: Unknown - ID: M8312 - Name: Factor VIII - Relevance: LOW - As Found: Unknown - ID: M19461 - Name: Antibodies, Antiphospholipid - Relevance: HIGH - As Found: 1.25 mg/ml - ID: M14349 - Name: Protein C - Relevance: LOW - As Found: Unknown - ID: M19587 - Name: Protein S - Relevance: LOW - As Found: Unknown - ID: M10201 - Name: Immunologic Factors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000000906 - Term: Antibodies - ID: D000017152 - Term: Antibodies, Antiphospholipid ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00097279 **Acronym:** ACTION **Brief Title:** Comparison of Biphasic Insulin Aspart 70/30 With Anti-Diabetic Drugs in Subjects With Type 2 Diabetes **Official Title:** A Multicenter, Open-Label, Randomised Trial to Compare the Efficacy and Safety of NovoLog Mix 70/30 BID in Combination With Metformin and Pioglitazone to Metformin and Pioglitazone Alone in Insulin Naïve Subjects With Type 2 Diabetes #### Organization Study ID Info **ID:** BIASP-2186 #### Organization **Class:** INDUSTRY **Full Name:** Novo Nordisk A/S ### Status Module #### Completion Date **Date:** 2005-08 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-01-11 **Type:** ESTIMATED **Last Update Submit Date:** 2017-01-10 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2005-08 **Type:** ACTUAL #### Start Date **Date:** 2004-08 **Status Verified Date:** 2017-01 #### Study First Post Date **Date:** 2004-11-22 **Type:** ESTIMATED **Study First Submit Date:** 2004-11-19 **Study First Submit QC Date:** 2004-11-19 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Novo Nordisk A/S #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** This trial is conducted in the United States of America (USA). The purpose of this study is to test whether biphasic insulin aspart 70/30 is a safe and at least as effective alternative in combination with two oral anti-diabetics compared to the two oral anti-diabetics alone for the control of blood glucose. ### Conditions Module **Conditions:** - Diabetes - Diabetes Mellitus, Type 2 ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 230 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Name:** biphasic insulin aspart **Type:** DRUG #### Intervention 2 **Name:** pioglitazone **Type:** DRUG #### Intervention 3 **Name:** metformin **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** HbA1c **Time Frame:** After 24 weeks #### Secondary Outcomes **Measure:** Safety variables **Measure:** Other glycemic variables **Measure:** 8-point plasma glucose profiles ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Subjects with type 2 diabetes mellitus **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Birmingham **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Alabama **Zip:** 35235 **Location 2:** **City:** Glendale **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Arizona **Zip:** 85306 **Location 3:** **City:** Phoenix **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Arizona **Zip:** 85006 **Location 4:** **City:** Searcy **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Arkansas **Zip:** 72143 **Location 5:** **City:** Burlingame **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** California **Zip:** 94010 **Location 6:** **City:** Chula Vista **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** California **Zip:** 91911 **Location 7:** **City:** Fullerton **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** California **Zip:** 92835 **Location 8:** **City:** Los Angeles **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** California **Zip:** 90057 **Location 9:** **City:** Redwood City **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** California **Zip:** 94062 **Location 10:** **City:** Denver **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Colorado **Zip:** 80262 **Location 11:** **City:** New Britain **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Connecticut **Zip:** 06050 **Location 12:** **City:** Newark **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Delaware **Zip:** 19713 **Location 13:** **City:** Aventura **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Florida **Zip:** 33180 **Location 14:** **City:** Fort Myers **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Florida **Zip:** 33907 **Location 15:** **City:** Hollywood **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Florida **Zip:** 33021 **Location 16:** **City:** Lake Mary **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Florida **Zip:** 32746 **Location 17:** **City:** Melbourne **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Florida **Zip:** 32901 **Location 18:** **City:** Ocala **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Florida **Zip:** 34471 **Location 19:** **City:** Pembroke Pines **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Florida **Zip:** 33024 **Location 20:** **City:** Tallahassee **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Florida **Zip:** 32308 **Location 21:** **City:** West Palm Beach **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Florida **Zip:** 33401 **Location 22:** **City:** Atlanta **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Georgia **Zip:** 30339 **Location 23:** **City:** Columbus **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Georgia **Zip:** 31904 **Location 24:** **City:** Savannah **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Georgia **Zip:** 31406 **Location 25:** **City:** Boise **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Idaho **Zip:** 83702 **Location 26:** **City:** Chicago **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Illinois **Zip:** 60607 **Location 27:** **City:** Topeka **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Kansas **Zip:** 66606 **Location 28:** **City:** Wichita **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Kansas **Zip:** 67205 **Location 29:** **City:** Baton Rouge **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Louisiana **Zip:** 70808 **Location 30:** **City:** Easton **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Maryland **Zip:** 21601 **Location 31:** **City:** Danvers **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Massachusetts **Zip:** 01923 **Location 32:** **City:** Salisbury **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Massachusetts **Zip:** 01952 **Location 33:** **City:** Waltham **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Massachusetts **Zip:** 02453 **Location 34:** **City:** Detroit **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Michigan **Zip:** 48235 **Location 35:** **City:** Grand Rapids **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Michigan **Zip:** 49506 **Location 36:** **City:** Livonia **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Michigan **Zip:** 48152 **Location 37:** **City:** St. Cloud **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Minnesota **Zip:** 56303 **Location 38:** **City:** Columbia **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Missouri **Zip:** 65212 **Location 39:** **City:** Las Vegas **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Nevada **Zip:** 89030 **Location 40:** **City:** Reno **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Nevada **Zip:** 89502-0111 **Location 41:** **City:** Hampton **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** New Hampshire **Zip:** 03842 **Location 42:** **City:** Berlin **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** New Jersey **Zip:** 08009 **Location 43:** **City:** Turnersville **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** New Jersey **Zip:** 08012 **Location 44:** **City:** East Syracuse **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** New York **Zip:** 13057 **Location 45:** **City:** New York **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** New York **Zip:** 10003 **Location 46:** **City:** New York **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** New York **Zip:** 10023 **Location 47:** **City:** Greenville **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** North Carolina **Zip:** 27834 **Location 48:** **City:** Columbus **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Ohio **Zip:** 43203 **Location 49:** **City:** Dayton **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Ohio **Zip:** 45406 **Location 50:** **City:** Kettering **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Ohio **Zip:** 45429 **Location 51:** **City:** Medford **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Oregon **Zip:** 97501 **Location 52:** **City:** Melrose Park **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Pennsylvania **Zip:** 19027 **Location 53:** **City:** Philadelphia **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Pennsylvania **Zip:** 19107 **Location 54:** **City:** Greer **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** South Carolina **Zip:** 29651 **Location 55:** **City:** Chattanooga **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Tennessee **Zip:** 37404 **Location 56:** **City:** Arlington **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Texas **Zip:** 76014 **Location 57:** **City:** Dallas **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Texas **Zip:** 75231 **Location 58:** **City:** Dallas **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Texas **Zip:** 75235 **Location 59:** **City:** Dallas **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Texas **Zip:** 75390-8858 **Location 60:** **City:** Irving **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Texas **Zip:** 75061 **Location 61:** **City:** Midland **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Texas **Zip:** 79707 **Location 62:** **City:** San Antonio **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Texas **Zip:** 78229 **Location 63:** **City:** Ogden **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Utah **Zip:** 84403 **Location 64:** **City:** Newport News **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Virginia **Zip:** 23606 **Location 65:** **City:** Norfolk **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Virginia **Zip:** 23507 **Location 66:** **City:** Richmond **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Virginia **Zip:** 23249 **Location 67:** **City:** Suffolk **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Virginia **Zip:** 23434 **Location 68:** **City:** Warrenton **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Virginia **Zip:** 20186 **Location 69:** **City:** Kirkland **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Washington **Zip:** 98034 **Location 70:** **City:** Olympia **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Washington **Zip:** 98502 **Location 71:** **City:** Renton **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Washington **Zip:** 98057 **Location 72:** **City:** Tacoma **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Washington **Zip:** 98405 **Location 73:** **City:** Milwaukee **Country:** United States **Facility:** Novo Nordisk Investigational Site **State:** Wisconsin **Zip:** 53209 #### Overall Officials **Official 1:** **Affiliation:** Novo Nordisk A/S **Name:** Global Clinical Registry (GCR, 1452) **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** Raskin P, Matfin G, Schwartz SL, Chaykin L, Chu PL, Braceras R, Wynne A. Addition of biphasic insulin aspart 30 to optimized metformin and pioglitazone treatment of type 2 diabetes mellitus: The ACTION Study (Achieving Control Through Insulin plus Oral ageNts). Diabetes Obes Metab. 2009 Jan;11(1):27-32. doi: 10.1111/j.1463-1326.2007.00796.x. Epub 2007 Oct 17. **PMID:** 17941873 #### See Also Links **Label:** Clinical Trials at Novo Nordisk **URL:** http://novonordisk-trials.com ## Derived Section ### Condition Browse Module - Ancestors - ID: D000044882 - Term: Glucose Metabolism Disorders - ID: D000008659 - Term: Metabolic Diseases - ID: D000004700 - Term: Endocrine System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M7115 - Name: Diabetes Mellitus - Relevance: HIGH - As Found: Diabetes - ID: M7119 - Name: Diabetes Mellitus, Type 2 - Relevance: HIGH - As Found: Diabetes Mellitus, Type 2 - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: M25403 - Name: Glucose Metabolism Disorders - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000003920 - Term: Diabetes Mellitus - ID: D000003924 - Term: Diabetes Mellitus, Type 2 ### Intervention Browse Module - Ancestors - ID: D000007004 - Term: Hypoglycemic Agents - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: Hypo - Name: Hypoglycemic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M11667 - Name: Metformin - Relevance: HIGH - As Found: Assessment - ID: M10365 - Name: Insulin - Relevance: LOW - As Found: Unknown - ID: M10054 - Name: Hypoglycemic Agents - Relevance: LOW - As Found: Unknown - ID: M173166 - Name: Insulin, Globin Zinc - Relevance: LOW - As Found: Unknown - ID: M1693 - Name: Pioglitazone - Relevance: HIGH - As Found: Non-invasive - ID: M29801 - Name: Insulin Aspart - Relevance: HIGH - As Found: Childhood - ID: M29799 - Name: Biphasic Insulins - Relevance: HIGH - As Found: Importance ### Intervention Browse Module - Meshes - ID: D000008687 - Term: Metformin - ID: D000077205 - Term: Pioglitazone - ID: D000061267 - Term: Insulin Aspart - ID: D000061265 - Term: Biphasic Insulins ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05955079 **Acronym:** ctDNA-endo **Brief Title:** Circulating Tumor DNA Study in Patients With Endometrial Cancer **Official Title:** Prospective Validation of the Association Between Circulating Tumor DNA Detection and Risk of Metastatic Relapse in Patients With Localized Endometrial Cancer #### Organization Study ID Info **ID:** CRC22003 #### Organization **Class:** OTHER **Full Name:** Assistance Publique - Hôpitaux de Paris #### Secondary ID Infos **Domain:** AP-HP **ID:** 20200812151455 **Type:** OTHER ### Status Module #### Completion Date **Date:** 2026-01 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-07-21 **Type:** ACTUAL **Last Update Submit Date:** 2023-07-12 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-10 **Type:** ESTIMATED #### Start Date **Date:** 2021-01-01 **Type:** ACTUAL **Status Verified Date:** 2023-05 #### Study First Post Date **Date:** 2023-07-21 **Type:** ACTUAL **Study First Submit Date:** 2023-07-12 **Study First Submit QC Date:** 2023-07-12 ### Sponsor Collaborators Module #### Collaborators **Class:** UNKNOWN **Name:** CARPEM, Institut du Cancer Paris **Class:** UNKNOWN **Name:** Centre de recherche des Cordeliers **Class:** UNKNOWN **Name:** METHYS DX #### Lead Sponsor **Class:** OTHER **Name:** Assistance Publique - Hôpitaux de Paris #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The objective of this study is to identify a population at risk of early recurrence after oncologic resection surgery of a primary uterine tumor based on the detection of ctDNA **Detailed Description:** Despite early management, the risk of recurrence in non-metastatic endometrial cancer (FIGO I-III) is approximately 10-20%. The challenge is to identify the most high-risk cases for relapse in order to adapt surgical and medical management. The development of digital PCR methods in nano-droplets, detecting circulating tumor DNA (ctDNA) with high sensitivity, could help to better specify the prognosis of patients with localized endometrial cancer and to identify a population with residual disease, the source of this ctDNA. The investigators established a universal methylation signature in the laboratory based on analysis of endometrial cancer-specific DNA methylation using in silico analysis of public data from the Cancer Genome Atlas, validated in an independent cohort, with 99% sensitivity and 98% specificity. A prospective biological cohort was established between the gynecology and medical oncology departments and the Cochin Hospital biological resources center (CARPEM-OncoCentre collection). This is a prospective monocentric biological collection study. The aim of this study is to evaluate the prognostic impact of pre- and post-operative ctDNA detection in stage I-III endometrial cancer. ### Conditions Module **Conditions:** - Endometrial Cancer **Keywords:** - Endometrial Cancer - Digital-PCR - Circulating Tumor DNA - minimal residual disease - liquid biopsy ### Design Module #### Bio Spec **Description:** Whole blood, three samples (before, after surgery and before chemotherapy) **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 130 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients over 18 years old with a biopsy-proven endometrial cancer, at FIGO stage I to IV, and amenable and undergoing surgical treatment **Intervention Names:** - Other: Whole blood **Label:** Endometrial Cancer ### Interventions #### Intervention 1 **Arm Group Labels:** - Endometrial Cancer **Description:** 3 samples (before, after surgery and before chemotherapy) **Name:** Whole blood **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** Recurrence-free survival **Time Frame:** 1 year #### Secondary Outcomes **Measure:** Recurrence-free survival **Time Frame:** 3 years **Description:** Frequency of ctDNA detection based on established prognostic parameters: histological type (endometrioid, non-endometrioid), grade (low grade, high grade), stage (localized to the uterus stages I-II or stage III with nodal involvement), lymphovascular invasion (present/absent), and molecular group (low risk: POLE, intermediate risk: MSI/NSMP, high risk: TP53). **Measure:** Frequency of ctDNA detection based on established prognostic parameters **Time Frame:** 3 years **Description:** Frequency of ctDNA detection in other prognostic groups according to the 2021 ESGO-ESTRO-ESP classification **Measure:** Frequency of ctDNA detection in other prognostic groups **Time Frame:** 3 years **Description:** Frequency of ctDNA detection based on the recurrence profile : anatomical (locoregional versus distant; abdominal versus extra-abdominal; visceral or nodal) or dynamic (aggressive recurrence (progression-free survival post recurrence \<6 months) or non-aggressive (\>6 months)) **Measure:** Frequency of ctDNA detection based on the recurrence profile **Time Frame:** 3 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Female patients over 18 years of age who are potentially eligible for inclusion in the OncoCentre collection (registered as a patient at APHP, without legal protection measures, affiliated with a social security system) * Patients diagnosed with histologically documented endometrial cancer on an endometrial biopsy * Surgical intervention performed at Hopital Cochin Exclusion Criteria: * Failure to sign the OncoCentre consent form * Refusal of OncoCentre consent * Patient not eligible for upfront curative surgical treatment **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Women undergoing clinically-indicated hysterectomy for endometrial cancer at the University Paris Cité hospital Cochin, who are age 18 years and older ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** guillaume.beinse@aphp.fr **Name:** Guillaume BEINSE, MD, PhD **Phone:** +33 1 58 41 14 54 **Role:** CONTACT **Contact 2:** **Email:** laetitia.peaudecerf@aphp.fr **Name:** Laetitia PEAUDECERF **Phone:** +33 1 58 41 12 13 **Role:** CONTACT #### Locations **Location 1:** **City:** Paris **Contacts:** ***Contact 1:*** - **Email:** guillaume.beinse@aphp.fr - **Name:** BEINSE Guillaume, MD, PhD - **Phone:** +33 1 58 41 14 54 - **Role:** CONTACT ***Contact 2:*** - **Email:** antoine.gaudetchardonnet@aphp.fr - **Name:** GAUDET CHARDONNET Antoine, MD - **Phone:** +33 1 58 41 36 61 - **Role:** CONTACT **Country:** France **Facility:** Hôpital Cochin **Status:** RECRUITING **Zip:** 75014 #### Overall Officials **Official 1:** **Affiliation:** Assistance Publique - Hôpitaux de Paris **Name:** Bruno BORGHESE, MD, PhD **Role:** STUDY_DIRECTOR **Official 2:** **Affiliation:** Assistance Publique - Hôpitaux de Paris **Name:** Jérôme ALEXANDRE, MD, PhD **Role:** STUDY_CHAIR ### References Module #### References **Citation:** Beinse G, Borghese B, Metairie M, Just PA, Poulet G, Garinet S, Parfait B, Didelot A, Bourreau C, Agueeff N, Lavolle A, Terris B, Chapron C, Goldwasser F, Leroy K, Blons H, Laurent-Puig P, Taly V, Alexandre J. Highly Specific Droplet-Digital PCR Detection of Universally Methylated Circulating Tumor DNA in Endometrial Carcinoma. Clin Chem. 2022 Jun 1;68(6):782-793. doi: 10.1093/clinchem/hvac020. **PMID:** 35323926 **Citation:** Lu KH, Broaddus RR. Endometrial Cancer. N Engl J Med. 2020 Nov 19;383(21):2053-2064. doi: 10.1056/NEJMra1514010. No abstract available. **PMID:** 33207095 **Citation:** Morice P, Leary A, Creutzberg C, Abu-Rustum N, Darai E. Endometrial cancer. Lancet. 2016 Mar 12;387(10023):1094-1108. doi: 10.1016/S0140-6736(15)00130-0. Epub 2015 Sep 6. **PMID:** 26354523 **Citation:** Cancer Genome Atlas Research Network; Kandoth C, Schultz N, Cherniack AD, Akbani R, Liu Y, Shen H, Robertson AG, Pashtan I, Shen R, Benz CC, Yau C, Laird PW, Ding L, Zhang W, Mills GB, Kucherlapati R, Mardis ER, Levine DA. Integrated genomic characterization of endometrial carcinoma. Nature. 2013 May 2;497(7447):67-73. doi: 10.1038/nature12113. Erratum In: Nature. 2013 Aug 8;500(7461):242. **PMID:** 23636398 **Citation:** Leon-Castillo A, de Boer SM, Powell ME, Mileshkin LR, Mackay HJ, Leary A, Nijman HW, Singh N, Pollock PM, Bessette P, Fyles A, Haie-Meder C, Smit VTHBM, Edmondson RJ, Putter H, Kitchener HC, Crosbie EJ, de Bruyn M, Nout RA, Horeweg N, Creutzberg CL, Bosse T; TransPORTEC consortium. Molecular Classification of the PORTEC-3 Trial for High-Risk Endometrial Cancer: Impact on Prognosis and Benefit From Adjuvant Therapy. J Clin Oncol. 2020 Oct 10;38(29):3388-3397. doi: 10.1200/JCO.20.00549. Epub 2020 Aug 4. **PMID:** 32749941 **Citation:** Kommoss S, McConechy MK, Kommoss F, Leung S, Bunz A, Magrill J, Britton H, Kommoss F, Grevenkamp F, Karnezis A, Yang W, Lum A, Kramer B, Taran F, Staebler A, Lax S, Brucker SY, Huntsman DG, Gilks CB, McAlpine JN, Talhouk A. Final validation of the ProMisE molecular classifier for endometrial carcinoma in a large population-based case series. Ann Oncol. 2018 May 1;29(5):1180-1188. doi: 10.1093/annonc/mdy058. **PMID:** 29432521 **Citation:** Colombo N, Creutzberg C, Amant F, Bosse T, Gonzalez-Martin A, Ledermann J, Marth C, Nout R, Querleu D, Mirza MR, Sessa C; ESMO-ESGO-ESTRO Endometrial Consensus Conference Working Group. ESMO-ESGO-ESTRO Consensus Conference on Endometrial Cancer: diagnosis, treatment and follow-up. Ann Oncol. 2016 Jan;27(1):16-41. doi: 10.1093/annonc/mdv484. Epub 2015 Dec 2. Erratum In: Ann Oncol. 2017 Jul 1;28(suppl_4):iv167-iv168. **PMID:** 26634381 **Citation:** de Boer SM, Powell ME, Mileshkin L, Katsaros D, Bessette P, Haie-Meder C, Ottevanger PB, Ledermann JA, Khaw P, Colombo A, Fyles A, Baron MH, Jurgenliemk-Schulz IM, Kitchener HC, Nijman HW, Wilson G, Brooks S, Carinelli S, Provencher D, Hanzen C, Lutgens LCHW, Smit VTHBM, Singh N, Do V, D'Amico R, Nout RA, Feeney A, Verhoeven-Adema KW, Putter H, Creutzberg CL; PORTEC study group. Adjuvant chemoradiotherapy versus radiotherapy alone for women with high-risk endometrial cancer (PORTEC-3): final results of an international, open-label, multicentre, randomised, phase 3 trial. Lancet Oncol. 2018 Mar;19(3):295-309. doi: 10.1016/S1470-2045(18)30079-2. Epub 2018 Feb 12. Erratum In: Lancet Oncol. 2018 Apr;19(4):e184. **PMID:** 29449189 **Citation:** Corcoran RB, Chabner BA. Application of Cell-free DNA Analysis to Cancer Treatment. N Engl J Med. 2018 Nov 1;379(18):1754-1765. doi: 10.1056/NEJMra1706174. No abstract available. **PMID:** 30380390 **Citation:** Phallen J, Sausen M, Adleff V, Leal A, Hruban C, White J, Anagnostou V, Fiksel J, Cristiano S, Papp E, Speir S, Reinert T, Orntoft MW, Woodward BD, Murphy D, Parpart-Li S, Riley D, Nesselbush M, Sengamalay N, Georgiadis A, Li QK, Madsen MR, Mortensen FV, Huiskens J, Punt C, van Grieken N, Fijneman R, Meijer G, Husain H, Scharpf RB, Diaz LA Jr, Jones S, Angiuoli S, Orntoft T, Nielsen HJ, Andersen CL, Velculescu VE. Direct detection of early-stage cancers using circulating tumor DNA. Sci Transl Med. 2017 Aug 16;9(403):eaan2415. doi: 10.1126/scitranslmed.aan2415. **PMID:** 28814544 **Citation:** Bolivar AM, Luthra R, Mehrotra M, Chen W, Barkoh BA, Hu P, Zhang W, Broaddus RR. Targeted next-generation sequencing of endometrial cancer and matched circulating tumor DNA: identification of plasma-based, tumor-associated mutations in early stage patients. Mod Pathol. 2019 Mar;32(3):405-414. doi: 10.1038/s41379-018-0158-8. Epub 2018 Oct 12. **PMID:** 30315273 **Citation:** Shintani D, Hihara T, Ogasawara A, Sato S, Yabuno A, Tai K, Fujiwara K, Watanabe K, Hasegawa K. Tumor-related mutations in cell-free DNA in pre-operative plasma as a prognostic indicator of recurrence in endometrial cancer. Int J Gynecol Cancer. 2020 Sep;30(9):1340-1346. doi: 10.1136/ijgc-2019-001053. Epub 2020 Jul 21. **PMID:** 32699017 **Citation:** Taieb J, Taly V, Henriques J, Bourreau C, Mineur L, Bennouna J, Desrame J, Louvet C, Lepere C, Mabro M, Egreteau J, Bouche O, Mulot C, Hormigos K, Chaba K, Mazard T, de Gramont A, Vernerey D, Andre T, Laurent-Puig P. Prognostic Value and Relation with Adjuvant Treatment Duration of ctDNA in Stage III Colon Cancer: a Post Hoc Analysis of the PRODIGE-GERCOR IDEA-France Trial. Clin Cancer Res. 2021 Oct 15;27(20):5638-5646. doi: 10.1158/1078-0432.CCR-21-0271. Epub 2021 Jun 3. **PMID:** 34083233 **Citation:** Shen H, Laird PW. Interplay between the cancer genome and epigenome. Cell. 2013 Mar 28;153(1):38-55. doi: 10.1016/j.cell.2013.03.008. **PMID:** 23540689 **Citation:** Garrigou S, Perkins G, Garlan F, Normand C, Didelot A, Le Corre D, Peyvandi S, Mulot C, Niarra R, Aucouturier P, Chatellier G, Nizard P, Perez-Toralla K, Zonta E, Charpy C, Pujals A, Barau C, Bouche O, Emile JF, Pezet D, Bibeau F, Hutchison JB, Link DR, Zaanan A, Laurent-Puig P, Sobhani I, Taly V. A Study of Hypermethylated Circulating Tumor DNA as a Universal Colorectal Cancer Biomarker. Clin Chem. 2016 Aug;62(8):1129-39. doi: 10.1373/clinchem.2015.253609. Epub 2016 Jun 1. **PMID:** 27251038 **Citation:** Kint S, De Spiegelaere W, De Kesel J, Vandekerckhove L, Van Criekinge W. Evaluation of bisulfite kits for DNA methylation profiling in terms of DNA fragmentation and DNA recovery using digital PCR. PLoS One. 2018 Jun 14;13(6):e0199091. doi: 10.1371/journal.pone.0199091. eCollection 2018. **PMID:** 29902267 #### See Also Links **Label:** Related Info **URL:** https://carpem.fr/activites/les-plateformes/biobanques/projets-de-recherche-collection-oncocentre/ ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014594 - Term: Uterine Neoplasms - ID: D000005833 - Term: Genital Neoplasms, Female - ID: D000014565 - Term: Urogenital Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000014591 - Term: Uterine Diseases - ID: D000005831 - Term: Genital Diseases, Female - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000091662 - Term: Genital Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions ### Condition Browse Module - Browse Leaves - ID: M20497 - Name: Neoplasm, Residual - Relevance: LOW - As Found: Unknown - ID: M19235 - Name: Endometrial Neoplasms - Relevance: HIGH - As Found: Endometrial Cancer - ID: M17342 - Name: Uterine Neoplasms - Relevance: LOW - As Found: Unknown - ID: M8945 - Name: Genital Neoplasms, Female - Relevance: LOW - As Found: Unknown - ID: M17315 - Name: Urogenital Neoplasms - Relevance: LOW - As Found: Unknown - ID: M17339 - Name: Uterine Diseases - Relevance: LOW - As Found: Unknown - ID: M8943 - Name: Genital Diseases, Female - Relevance: LOW - As Found: Unknown - ID: M2876 - Name: Genital Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000016889 - Term: Endometrial Neoplasms ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06257979 **Acronym:** POCICO **Brief Title:** Unexpected Positive Cultures in Rotator Cuff Revision Surgery **Official Title:** Unexpected Positive Cultures in Rotator Cuff Revision Surgery: Significance and Influence on Outcomes and Tendon Healing #### Organization Study ID Info **ID:** 2023-A02042-43 #### Organization **Class:** OTHER **Full Name:** Elsan ### Status Module #### Completion Date **Date:** 2028-03-15 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-03-05 **Type:** ACTUAL **Last Update Submit Date:** 2024-03-04 **Overall Status:** NOT_YET_RECRUITING #### Primary Completion Date **Date:** 2028-02-15 **Type:** ESTIMATED #### Start Date **Date:** 2024-04 **Type:** ESTIMATED **Status Verified Date:** 2024-03 #### Study First Post Date **Date:** 2024-02-14 **Type:** ACTUAL **Study First Submit Date:** 2024-01-05 **Study First Submit QC Date:** 2024-02-05 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Elsan #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** In the context of rotator cuff re-intervention, the impact of Unexpected Positive Cultures (UPC) is not documented, and their management has not been studied, particularly regarding indications for antibiotic therapy, which is currently not a consensus. A prospective interventional study will be implemented to compare the results of non-randomized patient samples, whether positive or negative, taken during rotator cuff re-intervention. The objective is to assess whether these samples do not affect clinical outcomes and tendon healing rates. **Detailed Description:** Rotator cuff injuries are a common pathology with a 30% prevalence in the general population. If rotator cuff repair fails, patients often experience persistent pain, and bacteria can be detected during the revision period in approximately 30% of cases, even in the absence of septic symptoms. This type of infection, referred to as low-level infection, occurs in around 5% of cases and is frequently implicated as a contributing factor to poor outcomes of the intervention. Bacteriological samples are routinely collected during revisions, especially for prostheses. The appropriate course of action in cases where these samples test positive, yet there are no signs of infection, remains to be clarified. Notably, administering antibiotics in response to unexpected positive cultures (UPC) during the revision period for total shoulder prostheses does not appear to significantly impact results or infection rates. In the context of rotator cuff re-interventions, the impact of UPC has not been well-documented. The management of UPC, particularly regarding antibiotic therapy, has not been thoroughly studied and is not currently recommended. Further research is needed to establish guidelines for managing positive cultures in the absence of infection symptoms during rotator cuff re-interventions. ### Conditions Module **Conditions:** - Rotator Cuff Repair **Keywords:** - Unexpected Positive Cultures (UPC) - Revision cuff repair surgery (RCR) ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** The study procedure involves a re-intervention of the rotator cuff, which is conducted for the patients in accordance with standard practice. The patient undergoes local and/or general anesthesia, along with intravenous or oral antibiotic prophylaxis. During the intervention, the surgeon collects the sutures placed during the first intervention, the product of tendon debridement, and the subacromial bursa (cleaning product) used during the re-intervention. These elements usually discarded, are collected for the bacteriological analysis. Antibiotic treatment cannot be prescribed immediately and during the first month following this re-intervention (time for biofilm formation). As per the protocol, even a positive sample should not lead to the initiation of systematic antibiotic therapy. In the case of identification of a pathogenic microorganism, antibiotic therapy will be prescribed, leading to discontinuation of the study. ##### Masking Info **Masking:** SINGLE **Masking Description:** Following the analysis of the bacteriological samples, the investigator will read the bacteriological results. These will be entered into the database. During subsequent visits, the investigator will not provide this information to the patient who will thus remain as a single blind. **Who Masked:** - PARTICIPANT **Primary Purpose:** OTHER #### Enrollment Info **Count:** 132 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Operated patients with positive results for their pre-operative microbiological samples. **Intervention Names:** - Other: This study is with minimal risk and minimal constraints due to the addition of questionnaires **Label:** Experimental group **Type:** OTHER #### Arm Group 2 **Description:** Operated patients with negative results for their pre-operative microbiological samples. **Intervention Names:** - Other: This study is with minimal risk and minimal constraints due to the addition of questionnaires **Label:** Control group **Type:** OTHER ### Interventions #### Intervention 1 **Arm Group Labels:** - Control group - Experimental group **Description:** In this study, the specific procedures compared to routine care are : * Considering the addition of questionnaires, this study will be prospective. Due to the use of bacteriological samples, it is considered as a minimal-risk research. * The research procedure involves a bacteriological analysis of operative elements (sutures placed during the first intervention, the product of tendon debridement, and the subacromial bursa (cleaning product)) used during the re-intervention. These usually discarded operative samples are collected for bacteriological analysis. * Antibiotic treatment cannot be prescribed immediately after the surgery and during the first month following this re-intervention (time for biofilm formation). As per the protocol, even a positive sample should not lead to the initiation of systematic antibiotic therapy. In the case of identification of a pathogenic microorganism, antibiotic therapy will be prescribed, leading to discontinuation of the study. **Name:** This study is with minimal risk and minimal constraints due to the addition of questionnaires **Other Names:** - Microbiological analysis of samples collected from operated patients: in the presence of UPC, it does not influence the postoperative course - No antibiotics are prescribed during the first month after the operation. **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Compare the functional recovery (Yes/No), at one year after iterative repair of non-healing rotator cuff between patients with unexpected positive cultures and patients with negative results using the Constant Score. **Measure:** Compare the functional recovery of the shoulder at one year after iterative repair of non-healing rotator cuff between patients with unexpected positive cultures and patients with negative results. **Time Frame:** at Month 1, Month 3, Month 6 and 1 Year after the re-intervention **Description:** Compare the 2 groups on tendon healing (Yes/No), after iterative repair of non-healing of the rotator cuff using the Constant Score. **Measure:** Compare the 2 groups on tendon healing, after iterative repair of non-healing of the rotator cuff **Time Frame:** at Month 1, Month 3, Month 6 and 1 Year after the re-intervention **Description:** Compare the 2 groups on the occurrence of post-operative complications (Yes/No), after iterative repair of non-healing of the rotator cuff and list them **Measure:** Compare the 2 groups on the occurrence of post-operative complications, after iterative repair of non-healing of the rotator cuff **Time Frame:** up to one year after the re-intervention **Description:** Compare the 2 groups on the patient's other functional scores such as SSV and ASES questionnaries **Measure:** Compare the 2 groups on the patient's other functional scores **Time Frame:** at Month 1, Month 3, Month 6 and 1 Year after the re-intervention **Description:** List of pathogenic germs found in the positive samples **Measure:** Describe the pathogenic germs found in the positive cultures **Time Frame:** at Month 1 after the re-intervention #### Secondary Outcomes **Description:** The quality of the repair was assessed using an MRI and the Sugaya classification (French version): Type 1: Normal thickness and echostructure of the rotator cuff. Type 2: Normal thickness and heterogeneous echostructure. Type 3: Thinning without discontinuity. Type 4: Moderate discontinuity. Type 5: Frank discontinuity. **Measure:** Sugaya score measured from MRI to assess the influence of positive cultures on tendon healing **Time Frame:** at one year after the re-intervention **Description:** List of of complications during the first post-operative year **Measure:** Occurrence of complications during the first post-operative year **Time Frame:** up to one year after the re-intervention **Description:** ASES score can be viewed as a 100-point scale that evaluates two dimensions of shoulder function: pain and performance in activities of daily living. Each of the two domains make up for 50 of the 100 points. **Measure:** Functional scores at one year: American Shoulder and Elbow Surgeons Score (ASES) **Time Frame:** at one year after the re-intervention **Description:** SSV score is defined as the subjective evaluation by the patient of shoulder function, expressed as a percentage of a normal shoulder. This score ranges from 0 to 100%. **Measure:** Functional scores at one year: Subjective Shoulder Value (SSV) **Time Frame:** at one year after the re-intervention **Description:** List of pathogenic bacteria detected in the group of patients with positive results **Measure:** Pathogenic bacteria detected in the group of patients with positive results **Time Frame:** at Month 1 after the re-intervention ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patient, male or female, aged 18 or over * Patient requiring re- intervention for Rotator Ruff Revision Surgery for non-healing of the same tendons after a first intervention (delay \<2 years) * Patient requiring arthroscopy * Patient having had realized a MRI of the shoulder in the 6 previous months * Affiliate participant or beneficiary of a social security scheme * Participant having been informed and not having objected to the use of their data Exclusion Criteria: * Patient with at least one clinical sign of infection (fever, redness) * Patient with a new distant rupture on a healed cuff (\> 3 years) * Patient with a history of shoulder surgery other than initial Rotator Ruff Surgery * Patient having received antibiotic treatment in the 2 weeks preceding inclusion * Participant in another research * Participant in a period of exclusion from another research still in progress at the time of inclusion * Protected participant: an adult under guardianship, curatorship or other legal protection, deprived of liberty by judicial or administrative decision * Pregnant, breastfeeding or parturient woman * Participant hospitalized without consent **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** zakaria@elsan.care **Name:** Albatoul ZAKARIA, PhD **Phone:** 0033 6 49 77 17 52 **Role:** CONTACT #### Locations **Location 1:** **City:** Beaumont **Contacts:** ***Contact 1:*** - **Email:** pierremetais@mac.com - **Name:** METAIS Pierre, MD - **Role:** CONTACT **Country:** France **Facility:** Hôpital privé La Châtaigneraie ELSAN **Zip:** 63110 **Location 2:** **City:** Caen **Contacts:** ***Contact 1:*** - **Email:** fdordain@gmail.com - **Name:** Franck DORDAIN, MD - **Role:** CONTACT **Country:** France **Facility:** Hôpital privé Saint-Martin **Zip:** 14050 **Location 3:** **City:** Essey-lès-Nancy **Contacts:** ***Contact 1:*** - **Name:** Adrien JACQUOT, MD - **Role:** CONTACT **Country:** France **Facility:** Clinique Louis Pasteur **Zip:** 54270 **Location 4:** **City:** Lyon **Contacts:** ***Contact 1:*** - **Email:** docteurcollotte@gmail.com - **Name:** Philippe COLLOTTE, MD - **Role:** CONTACT **Country:** France **Facility:** Santy-Lyon-Ramsay **Zip:** 69008 **Location 5:** **City:** Muret **Contacts:** ***Contact 1:*** - **Email:** benjamin.adamczewski@gmail.com - **Name:** Benjamin ADAMCZEWSKI, MD - **Role:** CONTACT **Country:** France **Facility:** Clinique d'occitanie ELSAN **Zip:** 31600 **Location 6:** **City:** Nice **Contacts:** ***Contact 1:*** - **Email:** dr.chelli@icr-nice.com - **Name:** Mikaël CHELLI, MD - **Role:** CONTACT **Country:** France **Facility:** ICR-Kantys **Zip:** 06000 **Location 7:** **City:** Saint-Herblain **Contacts:** ***Contact 1:*** - **Email:** yves.bouju@gmail.com - **Name:** Yves BOUJU, MD - **Role:** CONTACT **Country:** France **Facility:** Santé Atlantique ELSAN **Zip:** 44800 **Location 8:** **City:** Strasbourg **Contacts:** ***Contact 1:*** - **Email:** antoni@epaule-coude-orangerie.fr - **Name:** Maxime Antony, MD - **Phone:** 0033 6 50 35 21 12 - **Role:** CONTACT **Country:** France **Facility:** Clinique de l'Orangerie #### Overall Officials **Official 1:** **Affiliation:** Clinique de l'Orangerie-Strasbourg **Name:** Maxime ANTONI, MD **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Zhao J, Luo M, Pan J, Liang G, Feng W, Zeng L, Yang W, Liu J. Risk factors affecting rotator cuff retear after arthroscopic repair: a meta-analysis and systematic review. J Shoulder Elbow Surg. 2021 Nov;30(11):2660-2670. doi: 10.1016/j.jse.2021.05.010. Epub 2021 Jun 2. **PMID:** 34089878 **Citation:** Azar M, Van der Meijden O, Pireau N, Chelli M, Gonzalez JF, Boileau P. Arthroscopic revision cuff repair: do tendons have a second chance to heal? J Shoulder Elbow Surg. 2022 Dec;31(12):2521-2531. doi: 10.1016/j.jse.2022.04.024. Epub 2022 Jun 6. **PMID:** 35671929 **Citation:** Bonnevialle N, Dauzeres F, Toulemonde J, Elia F, Laffosse JM, Mansat P. Periprosthetic shoulder infection: an overview. EFORT Open Rev. 2017 Apr 27;2(4):104-109. doi: 10.1302/2058-5241.2.160023. eCollection 2017 Apr. **PMID:** 28507783 **Citation:** Neufeld ME, Lanting BA, Shehata M, Naudie DDR, McCalden RW, Teeter MG, Vasarhelyi EM. The Prevalence and Outcomes of Unexpected Positive Intraoperative Cultures in Presumed Aseptic Revision Knee Arthroplasty. J Arthroplasty. 2022 Nov;37(11):2262-2271. doi: 10.1016/j.arth.2022.05.036. Epub 2022 May 19. **PMID:** 35598759 **Citation:** Falstie-Jensen T, Lange J, Daugaard H, Sorensen AKB, Ovesen J, Soballe K; ROSA Study Group. Unexpected positive cultures after revision shoulder arthroplasty: does it affect outcome? J Shoulder Elbow Surg. 2021 Jun;30(6):1299-1308. doi: 10.1016/j.jse.2020.12.014. Epub 2021 Feb 3. **PMID:** 33548396 **Citation:** Hodakowski AJ, Cohn MR, Mehta N, Menendez ME, McCormick JR, Garrigues GE. An evidence-based approach to managing unexpected positive cultures in shoulder arthroplasty. J Shoulder Elbow Surg. 2022 Oct;31(10):2176-2186. doi: 10.1016/j.jse.2022.03.019. Epub 2022 May 2. **PMID:** 35513254 **Citation:** Constant CR, Murley AH. A clinical method of functional assessment of the shoulder. Clin Orthop Relat Res. 1987 Jan;(214):160-4. **PMID:** 3791738 **Citation:** Sugaya H, Maeda K, Matsuki K, Moriishi J. Functional and structural outcome after arthroscopic full-thickness rotator cuff repair: single-row versus dual-row fixation. Arthroscopy. 2005 Nov;21(11):1307-16. doi: 10.1016/j.arthro.2005.08.011. **PMID:** 16325080 **Citation:** Angst F, Goldhahn J, Drerup S, Aeschlimann A, Schwyzer HK, Simmen BR. Responsiveness of six outcome assessment instruments in total shoulder arthroplasty. Arthritis Rheum. 2008 Mar 15;59(3):391-8. doi: 10.1002/art.23318. **PMID:** 18311752 **Citation:** Gilbart MK, Gerber C. Comparison of the subjective shoulder value and the Constant score. J Shoulder Elbow Surg. 2007 Nov-Dec;16(6):717-21. doi: 10.1016/j.jse.2007.02.123. **PMID:** 18061114 ## Derived Section ### Intervention Browse Module - Ancestors - ID: D000000890 - Term: Anti-Infective Agents ### Intervention Browse Module - Browse Branches - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M4222 - Name: Anti-Bacterial Agents - Relevance: HIGH - As Found: Initial - ID: M4224 - Name: Antibiotics, Antitubercular - Relevance: LOW - As Found: Unknown - ID: M4214 - Name: Anti-Infective Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000000900 - Term: Anti-Bacterial Agents ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02025179 **Brief Title:** 12 Month Extension Study of the Effect of Teriparatide on Bone in People With Chronic Spinal Cord Injury (SCI) **Official Title:** 12 Month Open-Label Extension Study of the Effect of Teriparatide on Bone in People With Chronic SCI #### Organization Study ID Info **ID:** A1195 #### Organization **Class:** OTHER **Full Name:** Northwestern University ### Status Module #### Completion Date **Date:** 2015-08-14 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-05-10 **Type:** ACTUAL **Last Update Submit Date:** 2023-05-08 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2015-08-14 **Type:** ACTUAL #### Results First Post Date **Date:** 2018-09-25 **Type:** ACTUAL **Results First Submit Date:** 2018-05-10 **Results First Submit QC Date:** 2018-08-28 #### Start Date **Date:** 2013-01 **Type:** ACTUAL **Status Verified Date:** 2018-08 #### Study First Post Date **Date:** 2013-12-31 **Type:** ESTIMATED **Study First Submit Date:** 2013-12-21 **Study First Submit QC Date:** 2013-12-27 ### Sponsor Collaborators Module #### Collaborators **Class:** FED **Name:** United States Department of Defense #### Lead Sponsor **Class:** OTHER **Name:** Northwestern University #### Responsible Party **Investigator Affiliation:** Northwestern University **Investigator Full Name:** Thomas J. Schnitzer **Investigator Title:** Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to determine if a second year of exposure to teriparatide in both subjects that received a year of teriparatide or teriparatide-placebo will result in a greater increase in bone mass density (BMD) compared to that seen in a single year's treatment. This study will also investigate 1) if a second year of teriparatide therapy will increase bone strength in people with chronic spinal cord injury (SCI) who previously received a year of teriparatide or teriparatide-placebo, 2) the number of participants with adverse events from teriparatide, and 3) the effects of teriparatide on serum markers of bone metabolism. **Detailed Description:** This extension study will only enroll individuals who have completed one year of treatment in the parent protocol "Effect of Teriparatide, Vibration and the Combination on Bone Mass and Bone Architecture in Chronic Spinal Cord Injury (NCT01225055)". This study will allow those individuals who received Teriparatide (20ug/day) and vibration (10 min/day) during the parent study to be treated for a second year and will allow those individuals who received teriparatide-placebo to have the opportunity to have a full year of teriparatide therapy. This study will determine if two years of exposure to teriparatide will result in a greater increase in Bone Mass Density (BMD) than that seen after a single year's treatment. Subjects will be evaluated at 6 and 12 months after initiating treatment with measurement of bone density as well as bone markers of formation and resorption. ### Conditions Module **Conditions:** - Spinal Cord Injury - Bone Loss - Osteoporosis **Keywords:** - Teriparatide - Osteoporosis - Spinal Cord Injury - Bone Diseases - Metabolic Bone Diseases - Musculoskeletal Diseases - Spinal Cord Diseases - Central Nervous System Diseases - Nervous System Diseases - Nervous System - Wounds and Injuries - Bone Density Conservation Agents - Physiological Effects of Drugs - Pharmacologic Actions - Vibration ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 25 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months **Intervention Names:** - Drug: Teriparatide - Device: Vibration **Label:** Teriparatide and vibration **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Teriparatide and vibration **Description:** Teriparatide 20 ug daily Sub-Q over 12 months **Name:** Teriparatide **Other Names:** - Forteo **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Teriparatide and vibration **Description:** Vibration 10 min/day for 12 months **Name:** Vibration **Other Names:** - Soloflex Whole Body Vibration platform **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** The mean percent change in BMD of the total hip after 24 months of treatment. Arms/Groups (3) below are divided into the Arms/Groups these subjects were in the parent protocol (NCT01225055) for the 12 month duration of this previous study. These subjects were then enrolled in this 12 month extension study (NCT02025179) and all received Teriparatide and vibration for 12 months as described in the protocol section. The "Baseline" refers to the baseline at the parent protocol (NCT01225055). The "24 months" refers to period after the completion of the 12 month parent protocol (NCT01225055) and this 12 month extension study (NCT02025179). **Measure:** Bone Mineral Density (BMD) of the Total Hip as Assessed by Dual-energy X-ray Absorptiometry (DXA) **Time Frame:** Baseline to 24 months #### Secondary Outcomes **Description:** The mean percent change in C-terminal telopeptide from baseline after 24 months of treatment. Arms/Groups (3) below are divided into the Arms/Groups these subjects were in the parent protocol (NCT01225055) for the 12 month duration of this previous study. These subjects were then enrolled in this 12 month extension study (NCT02025179) and all received Teriparatide and vibration for 12 months as described in the protocol section. The "Baseline" refers to the baseline at the parent protocol (NCT01225055). The "24 months" refers to period after the completion of the 12 month parent protocol (NCT01225055) and this 12 month extension study (NCT02025179). **Measure:** C-terminal Telopeptide **Time Frame:** Baseline to 24 Months **Description:** The mean percent change in BMD at the lumbar spine from baseline after 24 months of treatment. Arms/Groups (3) below are divided into the Arms/Groups these subjects were in the parent protocol (NCT01225055) for the 12 month duration of this previous study. These subjects were then enrolled in this 12 month extension study (NCT02025179) and all received Teriparatide and vibration for 12 months as described in the protocol section. The "Baseline" refers to the baseline at the parent protocol (NCT01225055). The "24 months" refers to period after the completion of the 12 month parent protocol (NCT01225055) and this 12 month extension study (NCT02025179). **Measure:** Bone Mineral Density (BMD) by DXA at the Lumbar Spine **Time Frame:** Baseline to 24 Months **Description:** The mean percent change in BMD of the femoral neck after 24 months of treatment. Arms/Groups (3) below are divided into the Arms/Groups these subjects were in the parent protocol (NCT01225055) for the 12 month duration of this previous study. These subjects were then enrolled in this 12 month extension study (NCT02025179) and all received Teriparatide and vibration for 12 months as described in the protocol section. The "Baseline" refers to the baseline at the parent protocol (NCT01225055). The "24 months" refers to period after the completion of the 12 month parent protocol (NCT01225055) and this 12 month extension study (NCT02025179). **Measure:** Bone Mineral Density (BMD) by DXA at Femoral Neck **Time Frame:** Baseline to 24 Months **Description:** The mean percent change in Amino-terminal of type 1 collagen from baseline after 24 months of treatment. Arms/Groups (3) below are divided into the Arms/Groups these subjects were in the parent protocol (NCT01225055) for the 12 month duration of this previous study. These subjects were then enrolled in this 12 month extension study (NCT02025179) and all received Teriparatide and vibration for 12 months as described in the protocol section. The "Baseline" refers to the baseline at the parent protocol (NCT01225055). The "24 months" refers to period after the completion of the 12 month parent protocol (NCT01225055) and this 12 month extension study (NCT02025179). **Measure:** Amino-terminal Propeptide of Type 1 Collagen **Time Frame:** Baseline to 24 Months **Description:** The mean percent change in Bone-specific alkaline phosphatase from baseline after 24 months of treatment. Arms/Groups (3) below are divided into the Arms/Groups these subjects were in the parent protocol (NCT01225055) for the 12 month duration of this previous study. These subjects were then enrolled in this 12 month extension study (NCT02025179) and all received Teriparatide and vibration for 12 months as described in the protocol section. The "Baseline" refers to the baseline at the parent protocol (NCT01225055). The "24 months" refers to period after the completion of the 12 month parent protocol (NCT01225055) and this 12 month extension study (NCT02025179). **Measure:** Bone-specific Alkaline Phosphatase **Time Frame:** Baseline to 24 Months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Enrollment in, and completion of one year of treatment, in protocol "Effect of Teriparatide, Vibration and the Combination on Bone Mass and Bone Architecture in Chronic Spinal Cord Injury". * Adherence rate for teriparatide use of ≥60%. * Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for 90 days following completion of therapy. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. * A female of child-bearing potential is any woman (regardless of sexual orientation, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria: * Has not undergone a hysterectomy or bilateral oophorectomy; or * Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months). * Ability to understand and the willingness to sign a written informed consent. Exclusion Criteria: * Individuals who could not tolerate teriparatide treatment. * Individuals who will not be able to return for all study visits. * Patients may not be receiving any other investigational agents. * Other medical conditions that in the opinion of the investigator would preclude the subject from completing the study **Minimum Age:** 21 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Chicago **Country:** United States **Facility:** Northwestern University Feinberg School of Medicine **State:** Illinois **Zip:** 60611 #### Overall Officials **Official 1:** **Affiliation:** Northwestern University **Name:** Thomas J Schnitzer, MD, PhD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000013118 - Term: Spinal Cord Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000020196 - Term: Trauma, Nervous System - ID: D000001851 - Term: Bone Diseases, Metabolic - ID: D000001847 - Term: Bone Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000008659 - Term: Metabolic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: All - Name: All Conditions - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M17685 - Name: Wounds and Injuries - Relevance: HIGH - As Found: Injury - ID: M12947 - Name: Osteoporosis - Relevance: HIGH - As Found: Osteoporosis - ID: M5126 - Name: Bone Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: M15916 - Name: Spinal Cord Injuries - Relevance: HIGH - As Found: Spinal Cord Injury - ID: M5130 - Name: Bone Diseases, Metabolic - Relevance: LOW - As Found: Unknown - ID: M15915 - Name: Spinal Cord Diseases - Relevance: LOW - As Found: Unknown - ID: M22023 - Name: Trauma, Nervous System - Relevance: LOW - As Found: Unknown - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010024 - Term: Osteoporosis - ID: D000013119 - Term: Spinal Cord Injuries - ID: D000014947 - Term: Wounds and Injuries ### Intervention Browse Module - Ancestors - ID: D000077264 - Term: Calcium-Regulating Hormones and Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000050071 - Term: Bone Density Conservation Agents ### Intervention Browse Module - Browse Branches - Abbrev: BDCA - Name: Bone Density Conservation Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M21349 - Name: Teriparatide - Relevance: HIGH - As Found: Same as - ID: M9789 - Name: Hormones - Relevance: LOW - As Found: Unknown - ID: M5381 - Name: Calcium - Relevance: LOW - As Found: Unknown - ID: M5398 - Name: Calcium, Dietary - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000019379 - Term: Teriparatide ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** One subject in "Teriparatide and vibration" group was lost to follow-up. Arms/Groups (3) below are divided into the Arms/Groups these subjects were in the parent protocol (NCT01225055) for the 12 month duration of this previous study. These subjects were then enrolled in this 12 month extension study (NCT02025179) and all received Teriparatide and vibration for 12 months as described in the protocol section. #### Event Groups **Group ID:** EG000 **Title:** Teriparatide **Deaths Num At Risk:** 8 **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide alone with sham vibration Teriparatide: 20 ug daily over 12 months **ID:** EG000 **Other Num Affected:** 5 **Other Num at Risk:** 8 **Serious Number At Risk:** 8 **Title:** Teriparatide **Group ID:** EG001 **Title:** Vibration **Deaths Num At Risk:** 9 **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Vibration alone with placebo-teriparatide vibration: 10 min/day for 12 months **ID:** EG001 **Other Num Affected:** 7 **Other Num at Risk:** 9 **Serious Number Affected:** 1 **Serious Number At Risk:** 9 **Title:** Vibration **Group ID:** EG002 **Title:** Teriparatide and Vibration **Deaths Num At Risk:** 7 **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide with vibration applied in conjunction Teriparatide: 20 ug daily over 12 months vibration: 10 min/day for 12 months **ID:** EG002 **Other Num Affected:** 5 **Other Num at Risk:** 7 **Serious Number Affected:** 2 **Serious Number At Risk:** 7 **Title:** Teriparatide and Vibration **Frequency Threshold:** 0 #### Other Events **Term:** Abdominal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Acne **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** **Term:** Allergic Reaction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Immune system disorders **Source Vocabulary:** **Term:** Allergies **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Immune system disorders **Source Vocabulary:** **Term:** Burn **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** **Term:** Cold/Flu **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** **Term:** Constipation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Cut **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Cut Tip of Left Thumb **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** **Term:** Dry scalp **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** **Term:** Fall **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** **Term:** Fatigue **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** **Term:** Food poisoning **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** **Term:** Hypertension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** **Term:** Hyponatremia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** **Term:** Nephrolithiasis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** **Term:** Lower extremity fracture **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** **Term:** Musculoskeletal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** **Term:** Pinched nerve **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** **Term:** Pressure sore **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** **Term:** Rash **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** **Term:** Toe wound **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** **Term:** Urinary tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** #### Serious Events **Term:** Urinary Tract Infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 8 **Group ID:** EG001 **Num At Risk:** 9 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 7 **Num Events:** 2 **Term:** Breast Cancer **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) ##### Stats **Group ID:** EG000 **Num At Risk:** 8 **Group ID:** EG001 **Num At Risk:** 9 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 7 **Num Events:** 1 **Term:** Dehydration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 8 **Group ID:** EG001 **Num At Risk:** 9 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 7 **Num Events:** 1 **Term:** Paresthesia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 8 **Group ID:** EG001 **Num At Risk:** 9 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 7 **Num Events:** 1 **Term:** Kidney Stone **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 8 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 9 **Num Events:** 1 **Group ID:** EG002 **Num At Risk:** 7 **Time Frame:** 12 months ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 8 **Group ID:** BG001 **Value:** 9 **Group ID:** BG002 **Value:** 8 **Group ID:** BG003 **Value:** 25 **Units:** Participants ### Group **ID:** BG000 **Title:** Teriparatide **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide alone with sham vibration Teriparatide: 20 ug daily over 12 months ### Group **ID:** BG001 **Title:** Vibration **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Vibration alone with placebo-teriparatide vibration: 10 min/day for 12 months ### Group **ID:** BG002 **Title:** Teriparatide and Vibration **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide with vibration applied in conjunction Teriparatide: 20 ug daily over 12 months vibration: 10 min/day for 12 months ### Group **ID:** BG003 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 21.1 **Upper Limit:** 65.3 **Value:** 42.2 #### Measurement **Group ID:** BG001 **Lower Limit:** 22.2 **Upper Limit:** 62.7 **Value:** 38.0 #### Measurement **Group ID:** BG002 **Lower Limit:** 22.8 **Upper Limit:** 64.9 **Value:** 41.7 #### Measurement **Group ID:** BG003 **Lower Limit:** 21.1 **Upper Limit:** 65.3 **Value:** 40.5 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 2 #### Measurement **Group ID:** BG001 **Value:** 2 #### Measurement **Group ID:** BG002 **Value:** 3 #### Measurement **Group ID:** BG003 **Value:** 7 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 6 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 5 #### Measurement **Group ID:** BG003 **Value:** 18 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 2 #### Measurement **Group ID:** BG001 **Value:** 2 #### Measurement **Group ID:** BG002 **Value:** 1 #### Measurement **Group ID:** BG003 **Value:** 5 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 6 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 7 #### Measurement **Group ID:** BG003 **Value:** 20 **Category Title:** Not Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 1 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 1 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 5 #### Measurement **Group ID:** BG002 **Value:** 5 #### Measurement **Group ID:** BG003 **Value:** 13 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 5 #### Measurement **Group ID:** BG001 **Value:** 2 #### Measurement **Group ID:** BG002 **Value:** 3 #### Measurement **Group ID:** BG003 **Value:** 10 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 4 #### Measurement **Group ID:** BG001 **Value:** 8 #### Measurement **Group ID:** BG002 **Value:** 7 #### Measurement **Group ID:** BG003 **Value:** 19 **Category Title:** ASIA A (Complete Injury) #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 1 #### Measurement **Group ID:** BG003 **Value:** 2 **Category Title:** ASIA B (Sensory Incomplete Injury) #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 4 **Category Title:** ASIA C (Motor Incomplete Injury) #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 **Category Title:** ASIA D (Motor Incomplete Injury) **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** FULL_RANGE **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Ethnicity (NIH/OMB) **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race (NIH/OMB) **Unit of Measure:** Participants ### Measure 5 **Description:** ASIA impairment scale describes a person's functional impairment as a result of their spinal cord injury. A = Complete. No motor or sensory function in the lowest sacral spine segment (S4-5). B = Sensory Incomplete. Sensory function below the neurological level of injury (NLI) and in S4-S5, no motor function below NLI. C = Motor Incomplete. Motor function is preserved below NLI and more than half of the key muscle groups below NLI have a muscle grade \< 3. D = Motor Incomplete. Motor function is preserved below NLI and at least half of the key muscle groups below NLI have a muscle grade 3. **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** American Spinal Injury Association (ASIA) Impairment Scale **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement ### Point of Contact **Email:** tjs@northwestern.edu **Organization:** Northwestern University **Phone:** 312-503-2315 **Title:** Dr. Thomas Schnitzer ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 3.37 - **Spread:** - **Upper Limit:** 10.1 - **Value:** 6.73 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -3.50 - **Spread:** - **Upper Limit:** 2.82 - **Value:** -0.34 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 0.37 - **Spread:** - **Upper Limit:** 8.11 - **Value:** 4.24 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** -69.0 - **Spread:** - **Upper Limit:** 29.5 - **Value:** -19.8 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -61.2 - **Spread:** - **Upper Limit:** 31.6 - **Value:** -14.8 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** -54.8 - **Spread:** - **Upper Limit:** 50.5 - **Value:** -2.12 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 11.0 - **Spread:** - **Upper Limit:** 17.8 - **Value:** 14.4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 3.87 - **Spread:** - **Upper Limit:** 10.2 - **Value:** 7.05 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 5.08 - **Spread:** - **Upper Limit:** 12.9 - **Value:** 8.98 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** -2.81 - **Spread:** - **Upper Limit:** 8.26 - **Value:** 2.73 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -5.28 - **Spread:** - **Upper Limit:** 5.16 - **Value:** -0.06 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** -1.20 - **Spread:** - **Upper Limit:** 10.6 - **Value:** 4.71 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 73 - **Spread:** - **Upper Limit:** 132 - **Value:** 102 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 76.6 - **Spread:** - **Upper Limit:** 132 - **Value:** 104 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 26.6 - **Spread:** - **Upper Limit:** 89.5 - **Value:** 58 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 9.04 - **Spread:** - **Upper Limit:** 14.0 - **Value:** 11.5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 10.3 - **Spread:** - **Upper Limit:** 14.9 - **Value:** 12.6 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 8.30 - **Spread:** - **Upper Limit:** 13.6 - **Value:** 10.9 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** The mean percent change in BMD of the total hip after 24 months of treatment. Arms/Groups (3) below are divided into the Arms/Groups these subjects were in the parent protocol (NCT01225055) for the 12 month duration of this previous study. These subjects were then enrolled in this 12 month extension study (NCT02025179) and all received Teriparatide and vibration for 12 months as described in the protocol section. The "Baseline" refers to the baseline at the parent protocol (NCT01225055). The "24 months" refers to period after the completion of the 12 month parent protocol (NCT01225055) and this 12 month extension study (NCT02025179). **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** One subject in "Teriparatide and vibration" group was lost to follow-up **Reporting Status:** POSTED **Time Frame:** Baseline to 24 months **Title:** Bone Mineral Density (BMD) of the Total Hip as Assessed by Dual-energy X-ray Absorptiometry (DXA) **Type:** PRIMARY **Unit of Measure:** Percent change ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide alone with sham vibration Teriparatide: 20 ug daily over 12 months **ID:** OG000 **Title:** Teriparatide ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Vibration alone with placebo-teriparatide vibration: 10 min/day for 12 months **ID:** OG001 **Title:** Vibration ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide with vibration applied in conjunction Teriparatide: 20 ug daily over 12 months vibration: 10 min/day for 12 months **ID:** OG002 **Title:** Teriparatide and Vibration #### Outcome Measure 2 **Description:** The mean percent change in C-terminal telopeptide from baseline after 24 months of treatment. Arms/Groups (3) below are divided into the Arms/Groups these subjects were in the parent protocol (NCT01225055) for the 12 month duration of this previous study. These subjects were then enrolled in this 12 month extension study (NCT02025179) and all received Teriparatide and vibration for 12 months as described in the protocol section. The "Baseline" refers to the baseline at the parent protocol (NCT01225055). The "24 months" refers to period after the completion of the 12 month parent protocol (NCT01225055) and this 12 month extension study (NCT02025179). **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** One subject in "Teriparatide and vibration" group was lost to follow-up **Reporting Status:** POSTED **Time Frame:** Baseline to 24 Months **Title:** C-terminal Telopeptide **Type:** SECONDARY **Unit of Measure:** Percent change ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide alone with sham vibration Teriparatide: 20 ug daily over 12 months **ID:** OG000 **Title:** Teriparatide ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Vibration alone with placebo-teriparatide vibration: 10 min/day for 12 months **ID:** OG001 **Title:** Vibration ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide with vibration applied in conjunction Teriparatide: 20 ug daily over 12 months vibration: 10 min/day for 12 months **ID:** OG002 **Title:** Teriparatide and Vibration #### Outcome Measure 3 **Description:** The mean percent change in BMD at the lumbar spine from baseline after 24 months of treatment. Arms/Groups (3) below are divided into the Arms/Groups these subjects were in the parent protocol (NCT01225055) for the 12 month duration of this previous study. These subjects were then enrolled in this 12 month extension study (NCT02025179) and all received Teriparatide and vibration for 12 months as described in the protocol section. The "Baseline" refers to the baseline at the parent protocol (NCT01225055). The "24 months" refers to period after the completion of the 12 month parent protocol (NCT01225055) and this 12 month extension study (NCT02025179). **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** One subject in "Teriparatide and vibration" group was lost to follow-up. Another subject in "Teriparatide and vibration" spine could not be analyzed due to a baclofen pump. **Reporting Status:** POSTED **Time Frame:** Baseline to 24 Months **Title:** Bone Mineral Density (BMD) by DXA at the Lumbar Spine **Type:** SECONDARY **Unit of Measure:** Percent change ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide alone with sham vibration Teriparatide: 20 ug daily over 12 months **ID:** OG000 **Title:** Teriparatide ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Vibration alone with placebo-teriparatide vibration: 10 min/day for 12 months **ID:** OG001 **Title:** Vibration ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide with vibration applied in conjunction Teriparatide: 20 ug daily over 12 months vibration: 10 min/day for 12 months **ID:** OG002 **Title:** Teriparatide and Vibration #### Outcome Measure 4 **Description:** The mean percent change in BMD of the femoral neck after 24 months of treatment. Arms/Groups (3) below are divided into the Arms/Groups these subjects were in the parent protocol (NCT01225055) for the 12 month duration of this previous study. These subjects were then enrolled in this 12 month extension study (NCT02025179) and all received Teriparatide and vibration for 12 months as described in the protocol section. The "Baseline" refers to the baseline at the parent protocol (NCT01225055). The "24 months" refers to period after the completion of the 12 month parent protocol (NCT01225055) and this 12 month extension study (NCT02025179). **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** One subject in "Teriparatide and vibration" group was lost to follow-up **Reporting Status:** POSTED **Time Frame:** Baseline to 24 Months **Title:** Bone Mineral Density (BMD) by DXA at Femoral Neck **Type:** SECONDARY **Unit of Measure:** Percent change ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide alone with sham vibration Teriparatide: 20 ug daily over 12 months **ID:** OG000 **Title:** Teriparatide ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Vibration alone with placebo-teriparatide vibration: 10 min/day for 12 months **ID:** OG001 **Title:** Vibration ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide with vibration applied in conjunction Teriparatide: 20 ug daily over 12 months vibration: 10 min/day for 12 months **ID:** OG002 **Title:** Teriparatide and Vibration #### Outcome Measure 5 **Description:** The mean percent change in Amino-terminal of type 1 collagen from baseline after 24 months of treatment. Arms/Groups (3) below are divided into the Arms/Groups these subjects were in the parent protocol (NCT01225055) for the 12 month duration of this previous study. These subjects were then enrolled in this 12 month extension study (NCT02025179) and all received Teriparatide and vibration for 12 months as described in the protocol section. The "Baseline" refers to the baseline at the parent protocol (NCT01225055). The "24 months" refers to period after the completion of the 12 month parent protocol (NCT01225055) and this 12 month extension study (NCT02025179). **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** One subject in "Teriparatide and vibration" group was lost to follow-up **Reporting Status:** POSTED **Time Frame:** Baseline to 24 Months **Title:** Amino-terminal Propeptide of Type 1 Collagen **Type:** SECONDARY **Unit of Measure:** Percent change ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide alone with sham vibration Teriparatide: 20 ug daily over 12 months **ID:** OG000 **Title:** Teriparatide ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Vibration alone with placebo-teriparatide vibration: 10 min/day for 12 months **ID:** OG001 **Title:** Vibration ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide with vibration applied in conjunction Teriparatide: 20 ug daily over 12 months vibration: 10 min/day for 12 months **ID:** OG002 **Title:** Teriparatide and Vibration #### Outcome Measure 6 **Description:** The mean percent change in Bone-specific alkaline phosphatase from baseline after 24 months of treatment. Arms/Groups (3) below are divided into the Arms/Groups these subjects were in the parent protocol (NCT01225055) for the 12 month duration of this previous study. These subjects were then enrolled in this 12 month extension study (NCT02025179) and all received Teriparatide and vibration for 12 months as described in the protocol section. The "Baseline" refers to the baseline at the parent protocol (NCT01225055). The "24 months" refers to period after the completion of the 12 month parent protocol (NCT01225055) and this 12 month extension study (NCT02025179). **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** One subject in "Teriparatide and vibration" group was lost to follow-up **Reporting Status:** POSTED **Time Frame:** Baseline to 24 Months **Title:** Bone-specific Alkaline Phosphatase **Type:** SECONDARY **Unit of Measure:** Percent change ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide alone with sham vibration Teriparatide: 20 ug daily over 12 months **ID:** OG000 **Title:** Teriparatide ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Vibration alone with placebo-teriparatide vibration: 10 min/day for 12 months **ID:** OG001 **Title:** Vibration ##### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide with vibration applied in conjunction Teriparatide: 20 ug daily over 12 months vibration: 10 min/day for 12 months **ID:** OG002 **Title:** Teriparatide and Vibration ### Participant Flow Module #### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide alone with sham vibration Teriparatide: 20 ug daily over 12 months **ID:** FG000 **Title:** Teriparatide #### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Vibration alone with placebo-teriparatide vibration: 10 min/day for 12 months **ID:** FG001 **Title:** Vibration #### Group **Description:** Experimental: Teriparatide and vibration Drug: Teriparatide (Forteo) 20 ug daily Sub-Q over 12 months Device: Vibration 10 min/day for 12 months Completed in previously enrolled study (STU00033380), Teriparatide with vibration applied in conjunction Teriparatide: 20 ug daily over 12 months vibration: 10 min/day for 12 months **ID:** FG002 **Title:** Teriparatide and Vibration #### Period **Title:** Overall Study ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ###### Reason **Group ID:** FG002 **Number of Subjects:** 1 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 8 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 9 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 8 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 8 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 9 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 7 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 1 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT01202279 **Acronym:** Wait and See **Brief Title:** Safety and Efficacy Study of Guaifenesin and Pseudoephedrine for Symptomatic Therapy to Treat Acute RTI **Official Title:** A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multicenter Study of the Safety and Efficacy of Mucinex D for Symptomatic Therapy in Patients With Acute Upper Respiratory Tract Who Seek Treatment #### Organization Study ID Info **ID:** 2009-MUCD-001 #### Organization **Class:** INDUSTRY **Full Name:** Reckitt Benckiser LLC ### Status Module #### Completion Date **Date:** 2010-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-12-30 **Type:** ACTUAL **Last Update Submit Date:** 2020-12-03 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2010-04 **Type:** ACTUAL #### Results First Post Date **Date:** 2012-11-04 **Type:** ESTIMATED **Results First Submit Date:** 2011-04-04 **Results First Submit QC Date:** 2012-10-04 #### Start Date **Date:** 2009-10 **Status Verified Date:** 2020-12 #### Study First Post Date **Date:** 2010-09-15 **Type:** ESTIMATED **Study First Submit Date:** 2010-09-13 **Study First Submit QC Date:** 2010-09-13 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Reckitt Benckiser Inc. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to determine if treatment with Mucinex D lowers the use of antibiotics in the treatment of upper respiratory infection when compared to placebo ### Conditions Module **Conditions:** - Acute Upper Respiratory Track Infection **Keywords:** - Acute Upper Respiratory Track Infection Who Seek Treatment ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 1179 **Type:** ACTUAL **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Mucinex D (1200 mg guaifenesin and 120 mg pseudoephedrine HCl) extended release bilayer tablet twice a day (bid) with a full glass of water for 7 days **Intervention Names:** - Drug: Guaifenesin **Label:** Mucinex D **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Placebo given bid with a full glass of water for 7 days **Intervention Names:** - Device: Placebo **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Mucinex D **Description:** 1200 mg guaifenesin and 120 mg pseudoephedrine HCl bid for 7 days **Name:** Guaifenesin **Other Names:** - Mucinex D - Mucinex - Pseudoephedrine **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo **Description:** Placebo bid for 7 days **Name:** Placebo **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Number of patients who received an antibiotic **Measure:** Antibiotic Sparing **Time Frame:** Day 7 **Description:** WURSS-21 is made up of 21 questions with a scoring from 0 = no symptom to 7 = severe symptom. With a minimum score of 0 to a maximum score of 147. **Measure:** Change From Baseline in Total Symptom Score of the Wisconsin Upper Respiratory Symptom Survey - 21 (WURSS-21). **Time Frame:** Baseline and 7 Days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * patients aged 18 to 75 yrs of age presenting at a clinical site with symptoms diagnostic for an acute upper respiratory tract infection within the last 5 days Exclusion Criteria: * patients who had chronic, recurring respiratory signs and symptoms due to conditions such as chronic allergic rhinitis, chronic sinusitis or chronic bronchitis which in the investigator's opinion, confounded interpretation of symptom ratings for the acute upper respiratory infection. Also excluded were patients with a fever greater than 101 degrees F. **Maximum Age:** 75 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Mesa **Country:** United States **Facility:** Clinical Research Advantage, Inc. **State:** Arizona **Zip:** 85203 **Location 2:** **City:** Carmichael **Country:** United States **Facility:** Med Center **State:** California **Zip:** 95608 **Location 3:** **City:** Fresno **Country:** United States **Facility:** Research Center of Fresno, 3636 N. First Street, Suite 141 **State:** California **Zip:** 93726 **Location 4:** **City:** San Luis Obispo **Country:** United States **Facility:** Coastal Medical Research Group, Inc., 47 Santa Rosa Street **State:** California **Zip:** 93405 **Location 5:** **City:** New London **Country:** United States **Facility:** Coastal Connecticut Research, LLC, 342 Montauk Avenue **State:** Connecticut **Zip:** 06320 **Location 6:** **City:** Newark **Country:** United States **Facility:** Glasgow Family Practice **State:** Delaware **Zip:** 19702 **Location 7:** **City:** Morton **Country:** United States **Facility:** Koch Family Medicine, 81A E. Queenwood Road **State:** Illinois **Zip:** 61550 **Location 8:** **City:** Erlanger **Country:** United States **Facility:** Sterling Research Group, Ltd., 650 Sprucewood Lane **State:** Kentucky **Zip:** 41018 **Location 9:** **City:** Reisterstown **Country:** United States **Facility:** Clinical Associates Research, 750 Main Street, Suite 310 **State:** Maryland **Zip:** 21136 **Location 10:** **City:** Taylor **Country:** United States **Facility:** Park Place Family Practice & Internal Medicine **State:** Michigan **Zip:** 48180 **Location 11:** **City:** Bloomfield **Country:** United States **Facility:** Immedicenter **State:** New Jersey **Zip:** 07003 **Location 12:** **City:** High Point **Country:** United States **Facility:** Peters Medical Research **State:** North Carolina **Zip:** 27262 **Location 13:** **City:** Salisbury **Country:** United States **Facility:** (Piedmont Medical Research Assoc., Inc. d/b/a), Crescent Medical Research **State:** North Carolina **Zip:** 28144 **Location 14:** **City:** Columbus **Country:** United States **Facility:** Parsons Avenue Medical Clinic **State:** Ohio **Zip:** 43207 **Location 15:** **City:** Enid **Country:** United States **Facility:** Legacy Clinical Research, LLC, 1204 W. Willow Road, Suite B **State:** Oklahoma **Zip:** 73703 **Location 16:** **City:** Ashland **Country:** United States **Facility:** Integrated Medical Research, PC **State:** Oregon **Zip:** 97520 **Location 17:** **City:** Harleysville **Country:** United States **Facility:** Harleysville Medical Associates, 176 Main Street **State:** Pennsylvania **Zip:** 19438 **Location 18:** **City:** Johnstown **Country:** United States **Facility:** Kastelic MD & Associates, 322 Warren Street, Suite 300 **State:** Pennsylvania **Zip:** 15905 **Location 19:** **City:** Penndel **Country:** United States **Facility:** Durham Physicians, P.C. **State:** Pennsylvania **Zip:** 19047 **Location 20:** **City:** Mount Pleasant **Country:** United States **Facility:** Palmetto Medical Research, 180 Wingo Way, Suite 203 **State:** South Carolina **Zip:** 29464 **Location 21:** **City:** Bristol **Country:** United States **Facility:** TriCities Medical Research **State:** Tennessee **Zip:** 37620 **Location 22:** **City:** Plano **Country:** United States **Facility:** Village Health Partners **State:** Texas **Zip:** 75024 **Location 23:** **City:** Virginia Beach **Country:** United States **Facility:** Independence Family Medicine, 813 Independence Blvd., Suite A **State:** Virginia **Zip:** 23455 **Location 24:** **City:** Winchester **Country:** United States **Facility:** Amherst Family Practice, 1867 Amherst Street **State:** Virginia **Zip:** 22601 ### References Module #### References **Citation:** Septimus EJ, Albrecht HH, Solomon G, Shea T, Guenin EP. Extended-Release Guaifenesin/Pseudoephedrine Hydrochloride for Symptom Relief in Support of a Wait-and-See Approach for the Treatment of Acute Upper Respiratory Tract Infections: A Randomized, Double-Blind, Placebo-Controlled Study. Curr Ther Res Clin Exp. 2017 Apr 28;84:54-61. doi: 10.1016/j.curtheres.2017.04.004. eCollection 2017. **PMID:** 28761581 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10283 - Name: Infections - Relevance: HIGH - As Found: Infection - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007239 - Term: Infections ### Intervention Browse Module - Ancestors - ID: D000001993 - Term: Bronchodilator Agents - ID: D000001337 - Term: Autonomic Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018927 - Term: Anti-Asthmatic Agents - ID: D000019141 - Term: Respiratory System Agents - ID: D000014663 - Term: Nasal Decongestants - ID: D000014662 - Term: Vasoconstrictor Agents - ID: D000013566 - Term: Sympathomimetics - ID: D000018663 - Term: Adrenergic Agents - ID: D000018377 - Term: Neurotransmitter Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000005100 - Term: Expectorants - ID: D000000316 - Term: Adrenergic alpha-Agonists - ID: D000000322 - Term: Adrenergic Agonists - ID: D000001067 - Term: Appetite Depressants - ID: D000019440 - Term: Anti-Obesity Agents ### Intervention Browse Module - Browse Branches - Abbrev: VaCoAg - Name: Vasoconstrictor Agents - Abbrev: CNSSti - Name: Central Nervous System Stimulants - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Resp - Name: Respiratory System Agents - Abbrev: AnObAg - Name: Anti-Obesity Agents - Abbrev: AAll - Name: Anti-Allergic Agents - Abbrev: Derm - Name: Dermatologic Agents ### Intervention Browse Module - Browse Leaves - ID: M7966 - Name: Ephedrine - Relevance: LOW - As Found: Unknown - ID: M27586 - Name: Pseudoephedrine - Relevance: HIGH - As Found: LAR - ID: M13570 - Name: Phenylpropanolamine - Relevance: HIGH - As Found: Graph - ID: M9238 - Name: Guaifenesin - Relevance: HIGH - As Found: Graph - ID: M186319 - Name: Chlorpheniramine, phenylpropanolamine drug combination - Relevance: HIGH - As Found: Graph - ID: M5985 - Name: Chlorpheniramine - Relevance: LOW - As Found: Unknown - ID: M5269 - Name: Bronchodilator Agents - Relevance: LOW - As Found: Unknown - ID: M20963 - Name: Anti-Asthmatic Agents - Relevance: LOW - As Found: Unknown - ID: M21137 - Name: Respiratory System Agents - Relevance: LOW - As Found: Unknown - ID: M17410 - Name: Nasal Decongestants - Relevance: LOW - As Found: Unknown - ID: M17409 - Name: Vasoconstrictor Agents - Relevance: LOW - As Found: Unknown - ID: M16345 - Name: Sympathomimetics - Relevance: LOW - As Found: Unknown - ID: M20746 - Name: Adrenergic Agents - Relevance: LOW - As Found: Unknown - ID: M20504 - Name: Neurotransmitter Agents - Relevance: LOW - As Found: Unknown - ID: M8243 - Name: Expectorants - Relevance: LOW - As Found: Unknown - ID: M3668 - Name: Adrenergic alpha-Agonists - Relevance: LOW - As Found: Unknown - ID: M3673 - Name: Adrenergic Agonists - Relevance: LOW - As Found: Unknown - ID: M4379 - Name: Appetite Depressants - Relevance: LOW - As Found: Unknown - ID: M21396 - Name: Anti-Obesity Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000010665 - Term: Phenylpropanolamine - ID: D000054199 - Term: Pseudoephedrine - ID: D000006140 - Term: Guaifenesin - ID: C000083542 - Term: Chlorpheniramine, phenylpropanolamine drug combination ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Mucinex D **Description:** Mucinex D (1200 mg GGE and 120 mg pseudoephedrine HCl extended release bilayer tablet bid with a full glass of water for 7 days **ID:** EG000 **Other Num Affected:** 37 **Other Num at Risk:** 593 **Serious Number At Risk:** 593 **Title:** Mucinex D **Group ID:** EG001 **Title:** Placebo **Description:** Placebo given bid with a full glass of water for 7 days **ID:** EG001 **Other Num Affected:** 21 **Other Num at Risk:** 591 **Serious Number At Risk:** 591 **Title:** Placebo **Frequency Threshold:** 0 #### Other Events **Term:** Nervousness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA (12.0) **Term:** Insomnia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA (12.0) **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (12.0) **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (12.0) **Time Frame:** Upon signing of the informed consent, the patient was requested to report all AEs experienced throughout the duration of their participation in this study. AEs were followed until resolution, stability or until 30 days after last dose of study medication. ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 591 **Group ID:** BG001 **Value:** 588 **Group ID:** BG002 **Value:** 1179 **Units:** Participants ### Group **ID:** BG000 **Title:** Mucinex D **Description:** Mucinex D (1200 mg GGE and 120 mg pseudoephedrine HCl extended release bilayer tablet bid with a full glass of water for 7 days ### Group **ID:** BG001 **Title:** Placebo **Description:** Placebo given bid with a full glass of water for 7 days ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 575 #### Measurement **Group ID:** BG001 **Value:** 575 #### Measurement **Group ID:** BG002 **Value:** 1150 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 16 #### Measurement **Group ID:** BG001 **Value:** 13 #### Measurement **Group ID:** BG002 **Value:** 29 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 13.7 **Value:** 37.4 #### Measurement **Group ID:** BG001 **Spread:** 13.7 **Value:** 38.8 #### Measurement **Group ID:** BG002 **Spread:** 13.7 **Value:** 38.1 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 395 #### Measurement **Group ID:** BG001 **Value:** 404 #### Measurement **Group ID:** BG002 **Value:** 799 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 196 #### Measurement **Group ID:** BG001 **Value:** 184 #### Measurement **Group ID:** BG002 **Value:** 380 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 591 #### Measurement **Group ID:** BG001 **Value:** 588 #### Measurement **Group ID:** BG002 **Value:** 1179 **Class Title:** United States **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Age, Categorical **Unit of Measure:** Participants ### Measure 2 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants ## Results Section - More Information Module ### Certain Agreement **Restriction Type:** LTE60 **Restrictive Agreement:** True ### Point of Contact **Email:** ClinicalRequests@rb.com **Organization:** Reckitt Benckiser **Phone:** 855.966.5483 **Title:** Kim Clark ## Results Section - Outcome Measures Module ### Outcome Measure 1 #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** 95 **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** 0.025 **P-Value Comment:** **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** Fisher Exact **Tested Non-Inferiority:** ### Outcome Measure 2 #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** 95 **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** 0.022 **P-Value Comment:** **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** ANCOVA **Tested Non-Inferiority:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 89 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 121 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 7.71 - **Upper Limit:** - **Value:** -14.2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 7.99 - **Upper Limit:** - **Value:** -13.4 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Number of patients who received an antibiotic **Parameter Type:** NUMBER **Population Description:** Per Protocol Population using Fishers Exact Test. **Reporting Status:** POSTED **Time Frame:** Day 7 **Title:** Antibiotic Sparing **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Mucinex D (1200 mg GGE and 120 mg pseudoephedrine HCl extended release bilayer tablet bid with a full glass of water for 7 days **ID:** OG000 **Title:** Mucinex D ##### Group **Description:** Placebo given bid with a full glass of water for 7 days **ID:** OG001 **Title:** Placebo #### Outcome Measure 2 **Description:** WURSS-21 is made up of 21 questions with a scoring from 0 = no symptom to 7 = severe symptom. With a minimum score of 0 to a maximum score of 147. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** Baseline and 7 Days **Title:** Change From Baseline in Total Symptom Score of the Wisconsin Upper Respiratory Symptom Survey - 21 (WURSS-21). **Type:** PRIMARY **Unit of Measure:** units on a scale ##### Group **Description:** Mucinex D (1200 mg GGE and 120 mg pseudoephedrine HCl extended release bilayer tablet bid with a full glass of water for 7 days **ID:** OG000 **Title:** Mucinex D ##### Group **Description:** Placebo given bid with a full glass of water for 7 days **ID:** OG001 **Title:** Placebo ### Participant Flow Module #### Group **Description:** Mucinex D (1200 mg GGE and 120 mg pseudoephedrine HCl extended release bilayer tablet bid with a full glass of water for 7 days **ID:** FG000 **Title:** Mucinex D #### Group **Description:** Placebo given bid with a full glass of water for 7 days **ID:** FG001 **Title:** Placebo #### Period **Title:** Overall Study ##### Withdraw **Type:** Adverse Event ###### Reason **Group ID:** FG000 **Number of Subjects:** 8 ###### Reason **Group ID:** FG001 **Number of Subjects:** 6 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 13 ###### Reason **Group ID:** FG001 **Number of Subjects:** 10 ##### Withdraw **Type:** Physician Decision ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 10 ###### Reason **Group ID:** FG001 **Number of Subjects:** 7 ##### Withdraw **Type:** Protocol Violation ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ##### Withdraw **Type:** study compliance ###### Reason **Group ID:** FG000 **Number of Subjects:** 5 ###### Reason **Group ID:** FG001 **Number of Subjects:** 2 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 591 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 588 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 554 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 561 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 37 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 27 **Recruitment Details:** Recruitment took place October 2009 through April 2010. Advertising was not allowed for this study. Patients were seen by a Health Care Provider(HCP) and must have been seeking treatment for symptoms diagnostic for an acute upper respiratory tract infection. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT02460679 **Acronym:** EPI-589 **Brief Title:** Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS) **Official Title:** A Phase 2A Safety and Biomarker Study of EPI-589 in Subjects With Amyotrophic Lateral Sclerosis #### Organization Study ID Info **ID:** EPI589-15-001 #### Organization **Class:** INDUSTRY **Full Name:** PTC Therapeutics ### Status Module #### Completion Date **Date:** 2018-02-23 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-10-14 **Type:** ACTUAL **Last Update Submit Date:** 2020-09-23 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-02-23 **Type:** ACTUAL #### Results First Post Date **Date:** 2020-09-23 **Type:** ACTUAL **Results First Submit Date:** 2020-08-26 **Results First Submit QC Date:** 2020-08-31 #### Start Date **Date:** 2016-01-14 **Type:** ACTUAL **Status Verified Date:** 2020-09 #### Study First Post Date **Date:** 2015-06-02 **Type:** ESTIMATED **Study First Submit Date:** 2015-05-21 **Study First Submit QC Date:** 2015-05-29 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** PTC Therapeutics #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** This is an open label study with 30-day run in phase to establish baseline parameters, 90-day treatment phase, and a 90-day withdrawal phase to determine long-term effects, duration of treatment response, and potential effects of EPI-589 therapy on known trajectory. ### Conditions Module **Conditions:** - Amyotrophic Lateral Sclerosis ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 19 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants will receive EPI-589 500 milligrams (mg) (2 tablets of 250 mg each) twice daily (BID) for 3 months, unless discontinued for safety or tolerability issues. **Intervention Names:** - Drug: EPI-589 **Label:** EPI-589 **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - EPI-589 **Description:** An immediate release film-coated table at a 250 mg dosage strength will be administered per dose and schedule specified in arm. **Name:** EPI-589 **Other Names:** - (R)-troloxamide quinone **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. **Measure:** Number of Participants With Drug-Related Serious Adverse Events (SAEs) **Time Frame:** Baseline (Day 0) to Month 6 #### Secondary Outcomes **Measure:** Area Under the Plasma Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12) **Time Frame:** Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3 **Measure:** Maximum Observed Plasma Concentration (Cmax) **Time Frame:** Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3 **Description:** ALSFRS-R is a quickly administered (5 minutes) ordinal rating scale that assesses participant's capability and independence in 12 functional activities. All 12 activities, 6 bulbar-respiratory functions, 3 upper extremity functions (writing, cutting food, and dressing), 2 lower-extremity functions (walking and climbing), and 1 other function (turning in bed), are relevant in ALS. Each activity was recorded to the closest approximation from a list of 5 choices, scored 0-4, with the total score ranging from 48 (normal function) to 0 (unable to attempt the task). **Measure:** Change From Baseline in ALSFRS-R Total Score at Month 6 **Time Frame:** Baseline, Month 6 **Description:** Vital capacity is the maximum amount of air a participant can expel from the lungs after a maximum inhalation. **Measure:** Change From Baseline in Vital Capacity at Month 6 **Time Frame:** Baseline, Month 6 **Description:** MIP is a measure of the strength of inspiratory muscles, primarily the diaphragm, and allows for the assessment of ventilatory failure, restrictive lung disease, and respiratory muscle strength. **Measure:** Change From Baseline in MIP at Month 6 **Time Frame:** Baseline, Month 6 **Description:** Respiratory rate is the rate at which breathing occurs. **Measure:** Change From Baseline in Respiratory Rate at Month 6 **Time Frame:** Baseline, Month 6 **Description:** The heart rate measures the number of times the heart beats per minute. **Measure:** Change From Baseline in Heart Rate at Month 6 **Time Frame:** Baseline, Month 6 **Description:** SpO2, also known as oxygen saturation, is a measure of the amount of oxygen-carrying hemoglobin in the blood relative to the amount of hemoglobin not carrying oxygen. **Measure:** Change From Baseline in SpO2 at Month 6 **Time Frame:** Baseline, Month 6 **Description:** ETCO2 is the amount of CO2 in exhaled air, which assesses ventilation. **Measure:** Change From Baseline in ETCO2 at Month 6 **Time Frame:** Baseline, Month 6 **Description:** The failure to thrive as measured by body weight was defined as weight loss of more than 5% from baseline **Measure:** Failure to Thrive: Number of Participants With Weight Loss of More Than 5 Percent (%) From Baseline at Month 6 **Time Frame:** Baseline, Month 6 **Description:** Participants were observed and timed swallowing water as well as solid foods in accordance with a standardized protocol. **Measure:** Change From Baseline in Average Solid Swallowing Time and Water Swallowing Time at Month 6 **Time Frame:** Baseline, Month 6 **Description:** The strength of designated muscle groups was measured using handheld dynamometry. **Measure:** Change From Baseline in Muscle Function at Month 6, as Assessed by Handheld Dynamometry Parameters (Grip Strength, Shoulder Flexion, Knee Extension, Hip Flexion, Elbow Flexion, Elbow Extension, and Ankle Dorsi Flexion) **Time Frame:** Baseline, Month 6 **Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. **Measure:** Change From Baseline in Number of Words Participant Read at Month 6 **Time Frame:** Baseline, Month 6 **Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. **Measure:** Change From Baseline in Time Spent in Reading at Month 6 **Time Frame:** Baseline, Month 6 **Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. **Measure:** Change From Baseline in Number of Words Per Minute Read at Month 6 **Time Frame:** Baseline, Month 6 **Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. **Measure:** Number of Participants With Normal Loudness, Normal Nasality, and Normal Intelligibility **Time Frame:** Month 6 **Description:** Glutathione lowest limit of quantification (LLOQ) = 0.089 micromoles (uM) and upper limit of quantification (ULOQ) = 13.916 uM in plasma. **Measure:** Level of Disease-Related Biomarker (Glutathione) in Plasma **Time Frame:** Baseline up to Month 6 **Description:** Glutathione LLOQ = 0.002 uM and ULOQ = 0.35 uM in CSF. **Measure:** Level of Disease-Related Biomarker (Glutathione) in Cerebrospinal Fluid (CSF) **Time Frame:** Baseline up to Month 3 **Description:** Glutathione LLOQ = 0.01 uM, and ULOQ = 1.39 uM in urine. **Measure:** Level of Disease-Related Biomarker (Glutathione) in Urine **Time Frame:** Baseline up to Month 6 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Diagnosis of possible, probable, laboratory supported probable, or definite ALS by E1 Escorial Criteria * Forced vital capacity (FVC) ≥ 70% of predicted * Weakness onset within 3 years * Agreement to use contraception if within reproductive years * Willingness and ability to comply with study procedures * Stable regimen of dietary supplements and /or riluzole for at least 30 days prior to enrollment * Abstention from use of other investigative or non-approved drugs * Participants must be able to swallow 0.375 \* 0.700 inch tablets Exclusion Criteria: * Allergy to EPI-589 * Use of ventilation * Participation in other intervention studies * Diagnosis of any other neurologic disease * Malignancy within the past 2 years * History of stroke * History of brain surgery * Hepatic insufficiency with liver function tests (LFTs) greater than 3 times upper limit of normal (ULN) * Renal insufficiency requiring dialysis * End stage cardiac failure * Participation in a trial of a device, drug, or other therapy for ALS within 3 months of screening or during the trial **Maximum Age:** 70 Years **Minimum Age:** 21 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Los Angeles **Country:** United States **Facility:** Cedar's Sinai **State:** California **Zip:** 90048 **Location 2:** **City:** San Francisco **Country:** United States **Facility:** California Pacific Medical Center **State:** California **Zip:** 94115 **Location 3:** **City:** Portland **Country:** United States **Facility:** Providence Brain and Spine Institute ALS Center **State:** Oregon **Zip:** 97213 #### Overall Officials **Official 1:** **Affiliation:** PTC Therapeutics **Name:** Matthew B Klein, MD, FACS **Role:** STUDY_DIRECTOR ## Document Section ### Large Document Module #### Large Docs - Date: 2017-02-17 - Filename: Prot_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: False - Label: Study Protocol - Size: 586189 - Type Abbrev: Prot - Upload Date: 2020-08-31T16:28 - Date: 2018-03-20 - Filename: SAP_002.pdf - Has ICF: False - Has Protocol: False - Has SAP: True - Label: Statistical Analysis Plan - Size: 650327 - Type Abbrev: SAP - Upload Date: 2020-09-23T08:11 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010335 - Term: Pathologic Processes - ID: D000019636 - Term: Neurodegenerative Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000009468 - Term: Neuromuscular Diseases - ID: D000013118 - Term: Spinal Cord Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000057177 - Term: TDP-43 Proteinopathies - ID: D000057165 - Term: Proteostasis Deficiencies - ID: D000008659 - Term: Metabolic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M15415 - Name: Sclerosis - Relevance: HIGH - As Found: Sclerosis - ID: M18879 - Name: Motor Neuron Disease - Relevance: HIGH - As Found: Lateral Sclerosis - ID: M4024 - Name: Amyotrophic Lateral Sclerosis - Relevance: HIGH - As Found: Amyotrophic Lateral Sclerosis - ID: M21558 - Name: Neurodegenerative Diseases - Relevance: LOW - As Found: Unknown - ID: M12411 - Name: Neuromuscular Diseases - Relevance: LOW - As Found: Unknown - ID: M15915 - Name: Spinal Cord Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M28759 - Name: TDP-43 Proteinopathies - Relevance: LOW - As Found: Unknown - ID: M28747 - Name: Proteostasis Deficiencies - Relevance: LOW - As Found: Unknown - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: T349 - Name: Amyotrophic Lateral Sclerosis - Relevance: HIGH - As Found: Amyotrophic Lateral Sclerosis - ID: T4699 - Name: Primary Lateral Sclerosis - Relevance: HIGH - As Found: Lateral Sclerosis ### Condition Browse Module - Meshes - ID: D000016472 - Term: Motor Neuron Disease - ID: D000000690 - Term: Amyotrophic Lateral Sclerosis - ID: D000012598 - Term: Sclerosis ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** Safety population included any participant who received at least 1 dose of EPI-589. #### Event Groups **Group ID:** EG000 **Title:** EPI-589 **Deaths Num At Risk:** 19 **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** EG000 **Other Num Affected:** 15 **Other Num at Risk:** 19 **Serious Number At Risk:** 19 **Title:** EPI-589 **Frequency Threshold:** 0 #### Other Events **Term:** Aortic valve incompetence **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Vertigo **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Ear and labyrinth disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Eye pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Vision blurred **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Abdominal pain lower **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Abdominal pain upper **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Constipation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Diarrhoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Salivary hypersecretion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Vomiting **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Asthenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Chest pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Chills **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Fatigue **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Hypersensitivity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Immune system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Seasonal allergy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Immune system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Clostridium difficile infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 **Term:** Nasopharyngitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 **Term:** Urinary tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 **Term:** Contusion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 15.0 **Term:** Fall **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 15.0 **Term:** Post lumbar puncture syndrome **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 15.0 **Term:** Upper limb fracture **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 15.0 **Term:** Alanine aminotransferase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 15.0 **Term:** Aspartate aminotransferase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 15.0 **Term:** Blood pressure increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 15.0 **Term:** Blood testosterone decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 15.0 **Term:** Decreased appetite **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Back pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Muscle spasms **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Muscular weakness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Musculoskeletal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Pain in extremity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Benign hepatic neoplasm **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 15.0 **Term:** Aura **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Balance disorder **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Dysarthria **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Hypoaesthesia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Hypokinesia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Paraesthesia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Tension headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Depression **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Disorientation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Dyspnoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Nasal congestion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Oropharyngeal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Productive cough **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Acne **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 15.0 **Time Frame:** Baseline (Day 0) to Month 6 ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 19 **Units:** Participants ### Group **ID:** BG000 **Title:** EPI-589 **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. ### Measure #### Measurement **Group ID:** BG000 **Spread:** 8.25 **Value:** 55.9 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 5 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 14 **Category Title:** Male #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 2 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 17 **Category Title:** Not Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Unknown or Not Reported #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 17 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Unknown or Not Reported #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 6.19 **Value:** 37.4 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 18.14 **Value:** 97.7 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 34.53 **Value:** 68.2 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 3.32 **Value:** 15.1 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** Sitting #### Measurement **Group ID:** BG000 **Spread:** 3.71 **Value:** 14.7 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 13 **Class Title:** After 5 minutes supine #### Measurement **Group ID:** BG000 **Spread:** 3.28 **Value:** 14.7 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 15 **Class Title:** After 10 minutes supine ### Measure #### Measurement **Group ID:** BG000 **Spread:** 10.99 **Value:** 68.9 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** Sitting #### Measurement **Group ID:** BG000 **Spread:** 13.46 **Value:** 63.8 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 13 **Class Title:** After 5 minutes supine #### Measurement **Group ID:** BG000 **Spread:** 12.30 **Value:** 65.3 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 15 **Class Title:** After 10 minutes supine ### Measure #### Measurement **Group ID:** BG000 **Spread:** 1.65 **Value:** 96.9 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** Sitting #### Measurement **Group ID:** BG000 **Spread:** 2.79 **Value:** 95.4 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 13 **Class Title:** After 5 minutes supine #### Measurement **Group ID:** BG000 **Spread:** 2.70 **Value:** 95.1 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 15 **Class Title:** After 10 minutes supine ### Measure #### Measurement **Group ID:** BG000 **Spread:** 16.8444 **Value:** 24.833 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** Sitting #### Measurement **Group ID:** BG000 **Spread:** 15.9007 **Value:** 30.964 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 13 **Class Title:** After 5 minutes supine #### Measurement **Group ID:** BG000 **Spread:** 14.5895 **Value:** 31.186 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 15 **Class Title:** After 10 minutes supine ### Measure #### Measurement **Group ID:** BG000 **Spread:** 5.465 **Value:** 12.46 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 3.698 **Value:** 4.68 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 20.630 **Value:** 26.41 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 17 **Class Title:** Grip - Left #### Measurement **Group ID:** BG000 **Spread:** 18.667 **Value:** 26.86 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 18 **Class Title:** Grip - Right #### Measurement **Group ID:** BG000 **Spread:** 8.218 **Value:** 13.94 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 17 **Class Title:** Shoulder Flexion - Left #### Measurement **Group ID:** BG000 **Spread:** 9.850 **Value:** 14.42 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 17 **Class Title:** Shoulder Flexion - Right #### Measurement **Group ID:** BG000 **Spread:** 12.796 **Value:** 22.11 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** Knee Extension - Left #### Measurement **Group ID:** BG000 **Spread:** 13.224 **Value:** 22.13 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** Knee Extension - Right #### Measurement **Group ID:** BG000 **Spread:** 11.832 **Value:** 17.32 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** Knee Flexion - Left #### Measurement **Group ID:** BG000 **Spread:** 12.987 **Value:** 18.02 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** Knee Flexion - Right #### Measurement **Group ID:** BG000 **Spread:** 16.229 **Value:** 20.13 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** Hip Flexion - Left #### Measurement **Group ID:** BG000 **Spread:** 12.738 **Value:** 20.82 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** Hip Flexion - Right #### Measurement **Group ID:** BG000 **Spread:** 9.074 **Value:** 17.16 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 17 **Class Title:** Elbow Flexion - Left #### Measurement **Group ID:** BG000 **Spread:** 10.508 **Value:** 16.52 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 17 **Class Title:** Elbow Flexion - Right #### Measurement **Group ID:** BG000 **Spread:** 10.514 **Value:** 17.51 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 17 **Class Title:** Elbow Extension - Left #### Measurement **Group ID:** BG000 **Spread:** 10.482 **Value:** 15.36 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 18 **Class Title:** Elbow Extension - Right #### Measurement **Group ID:** BG000 **Spread:** 12.188 **Value:** 16.47 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 18 **Class Title:** Ankle Dorsi Flexion - Left #### Measurement **Group ID:** BG000 **Spread:** 10.898 **Value:** 13.04 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 18 **Class Title:** Ankle Dorsi Flexion - Right ### Measure #### Measurement **Group ID:** BG000 **Spread:** 0.45 **Value:** 100.3 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 23.51 **Value:** 48.5 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 45.4527 **Value:** 143.529 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 19 **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Ethnicity (NIH/OMB) **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race (NIH/OMB) **Unit of Measure:** Participants ### Measure 5 **Description:** ALSFRS-R is a quickly administered (5 minutes) ordinal rating scale that assesses participant's capability and independence in 12 functional activities. All 12 activities, 6 bulbar-respiratory functions, 3 upper extremity functions (writing, cutting food, and dressing), 2 lower-extremity functions (walking and climbing), and 1 other function (turning in bed), are relevant in amyotrophic lateral sclerosis (ALS). Each activity was recorded to the closest approximation from a list of 5 choices, scored 0-4, with the total score ranging from 48 (normal function) to 0 (unable to attempt the task). **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Score **Unit of Measure:** units on a scale ### Measure 6 **Description:** Vital capacity is the maximum amount of air a participant can expel from the lungs after a maximum inhalation. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Vital Capacity **Unit of Measure:** percentage of capacity ### Measure 7 **Description:** MIP is a measure of the strength of inspiratory muscles, primarily the diaphragm, and allows for the assessment of ventilatory failure, restrictive lung disease, and respiratory muscle strength. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Maximum Inspiratory Pressure (MIP) **Unit of Measure:** centimetres of water at (cm H2O) ### Measure 8 **Description:** Respiratory rate is the rate at which breathing occurs. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Population Description:** 'Number analyzed' signifies participants evaluable for specified categories. **Title:** Respiratory Rate **Unit of Measure:** breaths/minute ### Measure 9 **Description:** The heart rate measures the number of times the heart beats per minute. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Population Description:** 'Number analyzed' signifies participants evaluable for specified categories. **Title:** Heart Rate **Unit of Measure:** beats/minute ### Measure 10 **Description:** SpO2, also known as oxygen saturation, is a measure of the amount of oxygen-carrying hemoglobin in the blood relative to the amount of hemoglobin not carrying oxygen. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Population Description:** 'Number analyzed' signifies participants evaluable for specified categories. **Title:** Oxygen Saturation (SpO2) **Unit of Measure:** percentage of hemoglobin ### Measure 11 **Description:** ETCO2 is the amount of CO2 in exhaled air, which assesses ventilation. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Population Description:** 'Number analyzed' signifies participants evaluable for specified categories. **Title:** End-Tidal Carbon Dioxide (ETCO2) **Unit of Measure:** percentage of CO2 ### Measure 12 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Average Solid Swallowing Time **Unit of Measure:** seconds ### Measure 13 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Average Water Swallowing Time **Unit of Measure:** seconds ### Measure 14 **Description:** Handheld dynamometry parameters included grip strength, shoulder flexion, knee extension, hip flexion, elbow flexion, elbow extension, and ankle dorsi flexion. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Population Description:** 'Number analyzed' signifies participants evaluable for specified categories. **Title:** Muscle Function, as Assessed by Handheld Dynamometry Parameters **Unit of Measure:** kilograms ### Measure 15 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Number of Words Participant Read **Unit of Measure:** Words ### Measure 16 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Time Spent in Reading **Unit of Measure:** seconds ### Measure 17 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Number of Words Per Minute Read **Unit of Measure:** words/minute **Population Description:** Efficacy intent-to-treat (EITT) population or safety population included any participant who received at least 1 dose of EPI-589. ## Results Section - More Information Module ### Certain Agreement **Other Details:** The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo. **Restriction Type:** OTHER **Restrictive Agreement:** True ### Point of Contact **Email:** medinfo@ptcbio.com **Organization:** PTC Therapeutics, Inc. **Phone:** 1-866-562-4620 **Title:** Patient Advocacy ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 ### Outcome Measure 7 ### Outcome Measure 8 ### Outcome Measure 9 ### Outcome Measure 10 ### Outcome Measure 11 ### Outcome Measure 12 ### Outcome Measure 13 ### Outcome Measure 14 ### Outcome Measure 15 ### Outcome Measure 16 ### Outcome Measure 17 ### Outcome Measure 18 ### Outcome Measure 19 ### Outcome Measure 20 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 721.1 - **Upper Limit:** - **Value:** 7762.7 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 401.1 - **Upper Limit:** - **Value:** 6878.8 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 9 **Units:** Participants #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 930.3 - **Upper Limit:** - **Value:** 5179.0 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 642.2 - **Upper Limit:** - **Value:** 4167.8 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 9 **Units:** Participants #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 4.03 - **Upper Limit:** - **Value:** -4.1 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 9.36 - **Upper Limit:** - **Value:** -3.1 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 24.83 - **Upper Limit:** - **Value:** 2.4 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.45 - **Upper Limit:** - **Value:** 0.3 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 14 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 5.57 - **Upper Limit:** - **Value:** -0.6 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 11 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 4.32 - **Upper Limit:** - **Value:** 0.0 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 13 **Units:** Participants #### Outcome Measure 8 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 10.74 - **Upper Limit:** - **Value:** 5.2 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 14 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 7.49 - **Upper Limit:** - **Value:** 7.1 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 11 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 8.39 - **Upper Limit:** - **Value:** 7.2 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 13 **Units:** Participants #### Outcome Measure 9 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.71 - **Upper Limit:** - **Value:** -1.0 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 14 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 4.64 - **Upper Limit:** - **Value:** -0.8 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 11 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.55 - **Upper Limit:** - **Value:** -0.1 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 13 **Units:** Participants #### Outcome Measure 10 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 8.2343 - **Upper Limit:** - **Value:** 2.384 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 14 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 5.0262 - **Upper Limit:** - **Value:** 0.605 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 11 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 9.6591 - **Upper Limit:** - **Value:** 7.112 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 13 **Units:** Participants #### Outcome Measure 11 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 **Title:** #### Outcome Measure 12 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.718 - **Upper Limit:** - **Value:** 4.80 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 14 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 20.950 - **Upper Limit:** - **Value:** 6.65 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 13 **Units:** Participants #### Outcome Measure 13 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 15.330 - **Upper Limit:** - **Value:** -9.69 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 13 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 7.207 - **Upper Limit:** - **Value:** -7.28 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 13 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 6.922 - **Upper Limit:** - **Value:** -4.53 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 12 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 8.915 - **Upper Limit:** - **Value:** -5.43 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 12 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 10.254 - **Upper Limit:** - **Value:** -5.42 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 14 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.505 - **Upper Limit:** - **Value:** -5.05 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 14 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 8.237 - **Upper Limit:** - **Value:** -4.63 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 14 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 9.170 - **Upper Limit:** - **Value:** -4.01 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 14 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 12.579 - **Upper Limit:** - **Value:** -6.84 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 14 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 9.627 - **Upper Limit:** - **Value:** -5.60 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 14 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 8.377 - **Upper Limit:** - **Value:** -4.84 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 13 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 8.186 - **Upper Limit:** - **Value:** -3.28 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 13 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 10.511 - **Upper Limit:** - **Value:** -5.77 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 13 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 9.048 - **Upper Limit:** - **Value:** -4.28 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 13 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 7.609 - **Upper Limit:** - **Value:** -6.12 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 13 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 7.529 - **Upper Limit:** - **Value:** -4.89 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 13 **Units:** Participants #### Outcome Measure 14 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.00 - **Upper Limit:** - **Value:** 0.0 **Title:** #### Outcome Measure 15 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.78 - **Upper Limit:** - **Value:** 5.5 **Title:** #### Outcome Measure 16 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 26.7792 - **Upper Limit:** - **Value:** -147.535 **Title:** #### Outcome Measure 17 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 10 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 6 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 6 **Title:** #### Outcome Measure 18 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.41 - **Spread:** - **Upper Limit:** 2.15 - **Value:** 1.34 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.83 - **Spread:** - **Upper Limit:** 11.74 - **Value:** 2.73 **Title:** #### Outcome Measure 19 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.04 - **Spread:** - **Upper Limit:** 0.31 - **Value:** 0.13 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.06 - **Spread:** - **Upper Limit:** 0.31 - **Value:** 0.13 **Title:** #### Outcome Measure 20 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.03 - **Spread:** - **Upper Limit:** 0.46 - **Value:** 0.12 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.02 - **Spread:** - **Upper Limit:** 0.82 - **Value:** 0.17 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Safety population included any participant who received at least 1 dose of EPI-589. **Reporting Status:** POSTED **Time Frame:** Baseline (Day 0) to Month 6 **Title:** Number of Participants With Drug-Related Serious Adverse Events (SAEs) **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 2 **Dispersion Type:** Standard Error **Parameter Type:** MEAN **Population Description:** EITT population or safety population included any participant who received at least 1 dose of EPI-589. Here, 'Number analyzed' signifies participants evaluable at specified timepoints. **Reporting Status:** POSTED **Time Frame:** Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3 **Title:** Area Under the Plasma Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12) **Type:** SECONDARY **Unit of Measure:** hours*nanograms/milliliter ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 3 **Dispersion Type:** Standard Error **Parameter Type:** MEAN **Population Description:** EITT population or safety population included any participant who received at least 1 dose of EPI-589. Here, 'Number analyzed' signifies participants evaluable at specified timepoints. **Reporting Status:** POSTED **Time Frame:** Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3 **Title:** Maximum Observed Plasma Concentration (Cmax) **Type:** SECONDARY **Unit of Measure:** ng/mL ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 4 **Description:** ALSFRS-R is a quickly administered (5 minutes) ordinal rating scale that assesses participant's capability and independence in 12 functional activities. All 12 activities, 6 bulbar-respiratory functions, 3 upper extremity functions (writing, cutting food, and dressing), 2 lower-extremity functions (walking and climbing), and 1 other function (turning in bed), are relevant in ALS. Each activity was recorded to the closest approximation from a list of 5 choices, scored 0-4, with the total score ranging from 48 (normal function) to 0 (unable to attempt the task). **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. **Reporting Status:** POSTED **Time Frame:** Baseline, Month 6 **Title:** Change From Baseline in ALSFRS-R Total Score at Month 6 **Type:** SECONDARY **Unit of Measure:** units on a scale ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 5 **Description:** Vital capacity is the maximum amount of air a participant can expel from the lungs after a maximum inhalation. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. **Reporting Status:** POSTED **Time Frame:** Baseline, Month 6 **Title:** Change From Baseline in Vital Capacity at Month 6 **Type:** SECONDARY **Unit of Measure:** percentage of capacity ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 6 **Description:** MIP is a measure of the strength of inspiratory muscles, primarily the diaphragm, and allows for the assessment of ventilatory failure, restrictive lung disease, and respiratory muscle strength. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. **Reporting Status:** POSTED **Time Frame:** Baseline, Month 6 **Title:** Change From Baseline in MIP at Month 6 **Type:** SECONDARY **Unit of Measure:** cm H2O ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 7 **Description:** Respiratory rate is the rate at which breathing occurs. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories. **Reporting Status:** POSTED **Time Frame:** Baseline, Month 6 **Title:** Change From Baseline in Respiratory Rate at Month 6 **Type:** SECONDARY **Unit of Measure:** breaths/minute ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 8 **Description:** The heart rate measures the number of times the heart beats per minute. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories. **Reporting Status:** POSTED **Time Frame:** Baseline, Month 6 **Title:** Change From Baseline in Heart Rate at Month 6 **Type:** SECONDARY **Unit of Measure:** beats/minute ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 9 **Description:** SpO2, also known as oxygen saturation, is a measure of the amount of oxygen-carrying hemoglobin in the blood relative to the amount of hemoglobin not carrying oxygen. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories. **Reporting Status:** POSTED **Time Frame:** Baseline, Month 6 **Title:** Change From Baseline in SpO2 at Month 6 **Type:** SECONDARY **Unit of Measure:** percentage of hemoglobin ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 10 **Description:** ETCO2 is the amount of CO2 in exhaled air, which assesses ventilation. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories. **Reporting Status:** POSTED **Time Frame:** Baseline, Month 6 **Title:** Change From Baseline in ETCO2 at Month 6 **Type:** SECONDARY **Unit of Measure:** percentage of CO2 ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 11 **Description:** The failure to thrive as measured by body weight was defined as weight loss of more than 5% from baseline **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. **Reporting Status:** POSTED **Time Frame:** Baseline, Month 6 **Title:** Failure to Thrive: Number of Participants With Weight Loss of More Than 5 Percent (%) From Baseline at Month 6 **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 12 **Description:** Participants were observed and timed swallowing water as well as solid foods in accordance with a standardized protocol. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Number analyzed' signifies participants evaluable for specified categories. **Reporting Status:** POSTED **Time Frame:** Baseline, Month 6 **Title:** Change From Baseline in Average Solid Swallowing Time and Water Swallowing Time at Month 6 **Type:** SECONDARY **Unit of Measure:** seconds ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 13 **Description:** The strength of designated muscle groups was measured using handheld dynamometry. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories. **Reporting Status:** POSTED **Time Frame:** Baseline, Month 6 **Title:** Change From Baseline in Muscle Function at Month 6, as Assessed by Handheld Dynamometry Parameters (Grip Strength, Shoulder Flexion, Knee Extension, Hip Flexion, Elbow Flexion, Elbow Extension, and Ankle Dorsi Flexion) **Type:** SECONDARY **Unit of Measure:** kilograms ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 14 **Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. **Reporting Status:** POSTED **Time Frame:** Baseline, Month 6 **Title:** Change From Baseline in Number of Words Participant Read at Month 6 **Type:** SECONDARY **Unit of Measure:** words ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 15 **Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. **Reporting Status:** POSTED **Time Frame:** Baseline, Month 6 **Title:** Change From Baseline in Time Spent in Reading at Month 6 **Type:** SECONDARY **Unit of Measure:** seconds ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 16 **Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. **Reporting Status:** POSTED **Time Frame:** Baseline, Month 6 **Title:** Change From Baseline in Number of Words Per Minute Read at Month 6 **Type:** SECONDARY **Unit of Measure:** words/minute ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 17 **Description:** The participant performed tasks in which speech was assessed through a perceptual assessment of overall intelligibility, vocal quality, and other factors by a speech language pathologist. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. **Reporting Status:** POSTED **Time Frame:** Month 6 **Title:** Number of Participants With Normal Loudness, Normal Nasality, and Normal Intelligibility **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 18 **Description:** Glutathione lowest limit of quantification (LLOQ) = 0.089 micromoles (uM) and upper limit of quantification (ULOQ) = 13.916 uM in plasma. **Dispersion Type:** Full Range **Parameter Type:** MEAN **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants with both baseline and Month 6 glutathione in plasma data. **Reporting Status:** POSTED **Time Frame:** Baseline up to Month 6 **Title:** Level of Disease-Related Biomarker (Glutathione) in Plasma **Type:** SECONDARY **Unit of Measure:** µM ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 19 **Description:** Glutathione LLOQ = 0.002 uM and ULOQ = 0.35 uM in CSF. **Dispersion Type:** Full Range **Parameter Type:** MEAN **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants with both baseline and Month 3 glutathione in CSF data. **Reporting Status:** POSTED **Time Frame:** Baseline up to Month 3 **Title:** Level of Disease-Related Biomarker (Glutathione) in Cerebrospinal Fluid (CSF) **Type:** SECONDARY **Unit of Measure:** µM ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 #### Outcome Measure 20 **Description:** Glutathione LLOQ = 0.01 uM, and ULOQ = 1.39 uM in urine. **Dispersion Type:** Full Range **Parameter Type:** MEAN **Population Description:** EITT population included any participant who received at least 1 dose of EPI-589. Here, 'Overall number of participants analyzed' signifies participants with both baseline and Month 6 glutathione in urine data. **Reporting Status:** POSTED **Time Frame:** Baseline up to Month 6 **Title:** Level of Disease-Related Biomarker (Glutathione) in Urine **Type:** SECONDARY **Unit of Measure:** µM ##### Group **Description:** Participants received EPI-589 500 mg (2 tablets of 250 mg each) BID for 3 months, unless discontinued for safety or tolerability issues. **ID:** OG000 **Title:** EPI-589 ### Participant Flow Module #### Group **Description:** Participants received EPI-589 500 milligrams (mg) (2 tablets of 250 mg each) twice daily (BID) for 3 months, unless discontinued for safety or tolerability issues. **ID:** FG000 **Title:** EPI-589 #### Period **Title:** Overall Study ##### Withdraw **Type:** Investigator Decision ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 4 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 19 ##### Milestone **Type:** Received at Least 1 Dose of Study Drug ###### Achievement **Group ID:** FG000 **Number of Subjects:** 19 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 14 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 5 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT02454179 **Acronym:** KEYNOTE147 **Brief Title:** Study of the Combination of Acalabrutinib (ACP-196) and Pembrolizumab in Advanced Head and Neck Squamous Cell Carcinoma **Official Title:** A Phase 2 Proof-of-Concept Study of the Combination of ACP-196 and Pembrolizumab in Subjects With Advanced Head and Neck Squamous Cell Carcinoma #### Organization Study ID Info **ID:** ACE-ST-006 #### Organization **Class:** INDUSTRY **Full Name:** Acerta Pharma BV ### Status Module #### Completion Date **Date:** 2018-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2019-09-12 **Type:** ACTUAL **Last Update Submit Date:** 2019-08-22 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-05 **Type:** ACTUAL #### Results First Post Date **Date:** 2019-09-12 **Type:** ACTUAL **Results First Submit Date:** 2019-07-19 **Results First Submit QC Date:** 2019-08-22 #### Start Date **Date:** 2015-05 **Type:** ACTUAL **Status Verified Date:** 2019-08 #### Study First Post Date **Date:** 2015-05-27 **Type:** ESTIMATED **Study First Submit Date:** 2015-05-22 **Study First Submit QC Date:** 2015-05-26 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Merck Sharp & Dohme LLC #### Lead Sponsor **Class:** INDUSTRY **Name:** Acerta Pharma BV #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Study of the combination of acalabrutinib (ACP-196) and pembrolizumab in subjects with advanced head and neck squamous cell carcinoma. ### Conditions Module **Conditions:** - Squamous Cell Carcinoma of the Head and Neck ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 78 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** pembrolizumab **Intervention Names:** - Drug: pembrolizumab **Label:** Arm 1 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** acalabrutinib in combination with pembrolizumab **Intervention Names:** - Drug: pembrolizumab - Drug: Acalabrutinib **Label:** Arm 2 **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Arm 1 - Arm 2 **Name:** pembrolizumab **Other Names:** - Keytruda **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Arm 2 **Name:** Acalabrutinib **Other Names:** - ACP-196 **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. **Measure:** Number of Participants With Overall Response **Time Frame:** 52 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Men and women ≥ 18 years of age * Histologically or cytologically confirmed recurrent, metastatic or unresectable HNSCC of the oral cavity, oropharynx, hypopharynx, and larynx that that has either progressed during or after platinum-based chemotherapy administered for metastatic disease or has recurred during or within 6 months after the completion of platinum-based neoadjuvant or adjuvant therapy * Presence of radiographically measurable disease as defined by RECIST 1.1 * ECOG performance status of 0 or 1 Exclusion Criteria: * Prior malignancy (other than HNSCC), except for adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, or other cancer from which the subject has been disease free for ≥ 2 years or which will not limit survival to \< 2 years * Known central nervous system metastases and/or carcinomatous meningitis * A life-threatening illness, medical condition (including psychiatric conditions) or organ system dysfunction which, in the investigator's opinion, could compromise the subject's safety * Significant cardiovascular disease such as uncontrolled or symptomatic arrhythmias, congestive heart failure, or myocardial infarction within 6 months of starting study drug **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Portland **Country:** United States **Facility:** Oregon Health and Science University **State:** Oregon #### Overall Officials **Official 1:** **Affiliation:** 1-888-292-9613; acertamc@dlss.com **Name:** Acerta Clinical Trials **Role:** STUDY_DIRECTOR ## Document Section ### Large Document Module #### Large Docs - Date: 2018-01-30 - Filename: SAP_000.pdf - Has ICF: False - Has Protocol: False - Has SAP: True - Label: Statistical Analysis Plan - Size: 2133372 - Type Abbrev: SAP - Upload Date: 2019-08-21T16:53 - Date: 2016-01-15 - Filename: Prot_001.pdf - Has ICF: False - Has Protocol: True - Has SAP: False - Label: Study Protocol - Size: 4061910 - Type Abbrev: Prot - Upload Date: 2019-08-22T11:23 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000018307 - Term: Neoplasms, Squamous Cell - ID: D000006258 - Term: Head and Neck Neoplasms - ID: D000009371 - Term: Neoplasms by Site ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M5534 - Name: Carcinoma - Relevance: HIGH - As Found: Carcinoma - ID: M5550 - Name: Carcinoma, Squamous Cell - Relevance: HIGH - As Found: Squamous Cell Carcinoma - ID: M1689 - Name: Squamous Cell Carcinoma of Head and Neck - Relevance: HIGH - As Found: Squamous Cell Carcinoma of the Head and Neck - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M20451 - Name: Neoplasms, Squamous Cell - Relevance: LOW - As Found: Unknown - ID: M9348 - Name: Head and Neck Neoplasms - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002277 - Term: Carcinoma - ID: D000002294 - Term: Carcinoma, Squamous Cell - ID: D000077195 - Term: Squamous Cell Carcinoma of Head and Neck ### Intervention Browse Module - Ancestors - ID: D000074322 - Term: Antineoplastic Agents, Immunological - ID: D000000970 - Term: Antineoplastic Agents - ID: D000082082 - Term: Immune Checkpoint Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000092004 - Term: Tyrosine Kinase Inhibitors - ID: D000047428 - Term: Protein Kinase Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: AA - Name: Amino Acids ### Intervention Browse Module - Browse Leaves - ID: M42185 - Name: Acalabrutinib - Relevance: HIGH - As Found: Discontinued - ID: M349416 - Name: Pembrolizumab - Relevance: HIGH - As Found: Blind - ID: M1346 - Name: Antineoplastic Agents, Immunological - Relevance: LOW - As Found: Unknown - ID: M2342 - Name: Immune Checkpoint Inhibitors - Relevance: LOW - As Found: Unknown - ID: M2889 - Name: Tyrosine Kinase Inhibitors - Relevance: LOW - As Found: Unknown - ID: M25820 - Name: Protein Kinase Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: T22 - Name: Tyrosine - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000582435 - Term: Pembrolizumab - ID: C000604908 - Term: Acalabrutinib ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Arm 1 - Pembrolizumab **Deaths Num Affected:** 26 **Deaths Num At Risk:** 39 **Description:** Pembrolizumab Monotherapy **ID:** EG000 **Other Num Affected:** 39 **Other Num at Risk:** 39 **Serious Number Affected:** 12 **Serious Number At Risk:** 39 **Title:** Arm 1 - Pembrolizumab **Group ID:** EG001 **Title:** Arm 2 - Acalabrutinib + Pembrolizumab **Deaths Num Affected:** 23 **Deaths Num At Risk:** 37 **Description:** Acalabrutinib in combination with Pembrolizumab **ID:** EG001 **Other Num Affected:** 37 **Other Num at Risk:** 37 **Serious Number Affected:** 25 **Serious Number At Risk:** 37 **Title:** Arm 2 - Acalabrutinib + Pembrolizumab **Frequency Threshold:** 5 #### Other Events **Term:** Mucosal Inflammation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** General Disorders and administration site conditions. **Organ System:** General disorders **Source Vocabulary:** **Term:** Oedema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** General Disorders and administration site conditions. **Organ System:** General disorders **Source Vocabulary:** **Term:** Seasonal Allergy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Immune system disorders **Source Vocabulary:** **Term:** Pneumonia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** **Term:** Upper Respiratory Tract Infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** **Term:** Candida Infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** **Term:** Cellulitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** **Term:** Urinary Tract Infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** **Term:** Aptyalism **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Dry Mouth **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Fatigue **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** General Disorders and administration site conditions. **Organ System:** General disorders **Source Vocabulary:** **Term:** Oedema Peripheral **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** General Disorders and administration site conditions. **Organ System:** General disorders **Source Vocabulary:** **Term:** Pyrexia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** General Disorders and administration site conditions. **Organ System:** General disorders **Source Vocabulary:** **Term:** Asthenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** General Disorders and administration site conditions. **Organ System:** General disorders **Source Vocabulary:** **Term:** Non-Cardiac Chest Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** General Disorders and administration site conditions. **Organ System:** General disorders **Source Vocabulary:** **Term:** Chills **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** General Disorders and administration site conditions. **Organ System:** General disorders **Source Vocabulary:** **Term:** Vomiting **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Dysphagia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Gastrooesophageal Reflux Disease **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Abdominal Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Dyspepsia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Stomatitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Oral Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Mouth Haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Anaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** **Term:** Tachycardia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** **Term:** Sinus Tachycardia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** **Term:** Hypothyroidism **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Endocrine disorders **Source Vocabulary:** **Term:** Vision Blurred **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Diarrhea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Constipation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** White Blood Cell Count Decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** **Term:** Decreased Appetite **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** **Term:** Dehydration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** **Term:** Hypokalaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** **Term:** Hypomagnesaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** **Term:** Hyperglycaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** **Term:** Hyponatraemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** **Term:** Hypocalcaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** **Term:** Oral Candidiasis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** **Term:** Fall **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** **Term:** Contusion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** **Term:** Weight Decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** **Term:** Blood Creatinine Increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** **Term:** Alanine Aminotransferase Increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** **Term:** Aspartate Aminotransferase Increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** **Term:** Platelet Count Decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** **Term:** Musculoskeletel Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** **Term:** Muscular Weakness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** **Term:** Musculoskeletal Chest Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** **Term:** Pain in extremity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** **Term:** Pain in Jaw **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** **Term:** Myalgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** **Term:** Infected Neoplasm **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** **Term:** Speech Disorder **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** **Term:** Syncope **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** **Term:** Insomnia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** **Term:** Anxiety **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** **Term:** Depression **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** **Term:** Eating Disorder **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** **Term:** Acute Kidney Injury **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** **Term:** Nocturia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** **Term:** Dyspnoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** **Term:** Cough **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** **Term:** Productive Cough **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** **Term:** Hypoxia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** **Term:** Wheezing **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** **Term:** Oropharyngeal Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** **Term:** Aspiration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** **Term:** Haemoptysis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** **Term:** Chronic Obstructive Pulmonary Disease **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** **Term:** Pneumonia Aspiration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** **Term:** Rash **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** **Term:** Pruritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** **Term:** Rash Maculo-Papular **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** **Term:** Swelling Face **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** **Term:** Decubitus Ulcer **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** **Term:** Hypotension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** **Term:** Hypertension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** **Term:** Hypercalcaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** **Term:** Hyperuricaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** **Term:** Hypoalbuminaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** **Term:** Arthralgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** **Term:** Back Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** **Term:** Muscle Spasms **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** **Term:** Neck Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** **Term:** Hyperkalaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** #### Serious Events **Term:** Neutropenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Cardiac Failure Congestive **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Atrial Flutter **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Cardio-Respiratory Arrest **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Pulseless Electrical Activity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Dysphagia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Mouth Heamorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Oral Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Vomiting **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Pyrexia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** General disorders and administration site conditions. **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 37 **Term:** Chest Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** General disorders and administration site conditions. **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Urosepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 37 **Term:** Clostridia Sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Clostridium Difficile Infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Epiglottistis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Influenza **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Osteomyelitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Spinal Compression Fracture **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Stoma Site Haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Decreased Appetite **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Dehydration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Hyponatraemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Malnutrition **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Back Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Tumour Heaorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Tumour Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Cerebrovascular Accident **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Hypoxic-Ischaemic Encephalopathy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Syncope **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Confusional State **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Delusion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Acute Kidney Injury **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 37 **Term:** Dyspnoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 4 **Num At Risk:** 37 **Term:** Respiratory Failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 37 **Term:** Acute Respiratory Failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Hypnoxia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Atelectasis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Chronic Obstructive Pulmonary Disease **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Hypercapnia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Obstructive Airways Disorder **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Pleural Effusion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Pulmonary Embolism **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Rash **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Aneurysm **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Embolism **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Hypotension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Orthrostatic Hypotension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Multiple Organ Dysfunction Syndrome **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** General Disorders and Administration Site Conditions **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 39 **Group ID:** EG001 **Num At Risk:** 37 **Term:** Oedema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** General Disorders and Administration Site Conditions **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Oedema Peripheral **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** General Disorders and Administration Site Conditions **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Autoimmune Hepatitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 37 **Term:** Pneumonia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 37 **Term:** Sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations ##### Stats **Group ID:** EG000 **Num At Risk:** 39 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 37 **Time Frame:** Safety Analysis tracked from 0 day to 2 years. ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 39 **Group ID:** BG001 **Value:** 37 **Group ID:** BG002 **Value:** 76 **Units:** Participants ### Group **ID:** BG000 **Title:** Mono Therapy Arm - Pembrolizumab **Description:** Pembrolizumab 200mg administered as an intravenous (IV) infusion every 3 weeks (Q3W). ### Group **ID:** BG001 **Title:** Combo Therapy Arm - Acalabrutinib + Pembrolizumab **Description:** Acalabrutinib 100mg PO BID plus Pembrolizumab 200mg administered as an intravenous (IV) infusion every 3 weeks (Q3W). ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 10.45 **Value:** 62.1 #### Measurement **Group ID:** BG001 **Spread:** 11.12 **Value:** 61.4 #### Measurement **Group ID:** BG002 **Spread:** 10.71 **Value:** 61.8 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 5 #### Measurement **Group ID:** BG001 **Value:** 2 #### Measurement **Group ID:** BG002 **Value:** 7 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 34 #### Measurement **Group ID:** BG001 **Value:** 35 #### Measurement **Group ID:** BG002 **Value:** 69 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 4 #### Measurement **Group ID:** BG001 **Value:** 2 #### Measurement **Group ID:** BG002 **Value:** 6 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 35 #### Measurement **Group ID:** BG001 **Value:** 35 #### Measurement **Group ID:** BG002 **Value:** 70 **Category Title:** Not Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 2 #### Measurement **Group ID:** BG001 **Value:** 2 #### Measurement **Group ID:** BG002 **Value:** 4 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 6 #### Measurement **Group ID:** BG002 **Value:** 6 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 37 #### Measurement **Group ID:** BG001 **Value:** 29 #### Measurement **Group ID:** BG002 **Value:** 66 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 39 #### Measurement **Group ID:** BG001 **Value:** 37 #### Measurement **Group ID:** BG002 **Value:** 76 **Class Title:** United States **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** Years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Ethnicity (NIH/OMB) **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race (NIH/OMB) **Unit of Measure:** Participants ### Measure 5 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement ### Point of Contact **Email:** acertamc@dlss.com **Organization:** Acerta Pharma, LLC **Phone:** 1-888-292-9613 **Title:** Priti Patel, MD, Executive Director - Head of Clinical Development ## Results Section - Outcome Measures Module ### Outcome Measure 1 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 7 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** The study participants had to have a minimum of one scan (for tumor assessment) after randomization/treatment in order to be considered analyzed. And some of the participants didn't make it to Wk 7 of treatment, hence reduction in the number of subjects analyzed. **Reporting Status:** POSTED **Time Frame:** 52 weeks **Title:** Number of Participants With Overall Response **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Pembrolizumab 200mg administered as an intravenous (IV) infusion every 3 weeks (Q3W). **ID:** OG000 **Title:** Mono Therapy Arm - Pembrolizumab ##### Group **Description:** Acalabrutinib 100mg PO BID plus Pembrolizumab 200mg administered as an intravenous (IV) infusion every 3 weeks (Q3W). **ID:** OG001 **Title:** Combo Therapy Arm - Acalabrutinib and Pembrolizumab ### Participant Flow Module #### Group **Description:** Pembrolizumab 200mg administered as an intravenous (IV) infusion every 3 weeks (Q3W). **ID:** FG000 **Title:** Mono Therapy of Pembrolizumab Arm 1 #### Group **Description:** Acalabrutinib 100mg PO BID plus Pembrolizumab 200mg administered as an intravenous (IV) infusion every 3 weeks (Q3W). **ID:** FG001 **Title:** Combo Therapy Acalabrutinib + Pembrolizumab Arm 2 #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 39 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 39 ##### Milestone **Type:** Enrolled ###### Achievement **Group ID:** FG000 **Number of Subjects:** 39 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 39 ##### Milestone **Type:** Received Study Medication ###### Achievement **Group ID:** FG000 **Number of Subjects:** 39 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 37 ##### Milestone **Type:** Discontinue Study ###### Achievement **Group ID:** FG000 **Number of Subjects:** 39 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 39 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 39 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 39 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT03985579 **Brief Title:** Influence of Kinesiology Taping Application on Severity of Signs and Symptoms in Breast Engorgement. **Official Title:** Influence of Kinesiology Taping Application on Severity of Signs and Symptoms in Breast Engorgement at Early Lactation Stage. #### Organization Study ID Info **ID:** 24/2019 #### Organization **Class:** OTHER **Full Name:** Żelazna Medical Centre, LLC ### Status Module #### Completion Date **Date:** 2020-12-01 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2020-08-06 **Type:** ACTUAL **Last Update Submit Date:** 2020-08-05 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2020-09-01 **Type:** ESTIMATED #### Start Date **Date:** 2019-07-01 **Type:** ACTUAL **Status Verified Date:** 2020-08 #### Study First Post Date **Date:** 2019-06-13 **Type:** ACTUAL **Study First Submit Date:** 2019-06-05 **Study First Submit QC Date:** 2019-06-12 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Żelazna Medical Centre, LLC #### Responsible Party **Investigator Affiliation:** Żelazna Medical Centre, LLC **Investigator Full Name:** Anna Jakóbik **Investigator Title:** Physiotherapist **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The aim of the research is to asses the efficacy of kinesiology taping application in breast engorgement in early postpartum stage. Kinesiology application will be performed by women's health physiotherapist. Patient's perceived improvement and milk outflow following application will be assessed. ### Conditions Module **Conditions:** - Lactation Disorders ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - OUTCOMES_ASSESSOR **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 60 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Application of elastic cotton strip with an acrylic adhesive that is used with the intent of treating pain and disability from athletic injuries and a variety of other physical disorders. **Intervention Names:** - Other: Kinesiology taping application **Label:** Standard care and Kinesiology taping Group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Assistance in baby feeding, manual of device-assisted (lactator) milk expression, cold compress, ibuprofen, gentle breast massage, breathing and shoulder girdle exercise. **Label:** Standard care Group **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Standard care and Kinesiology taping Group **Description:** Application of elastic cotton strip with an acrylic adhesive that is used with the intent of treating pain and disability from athletic injuries and a variety of other physical disorders **Name:** Kinesiology taping application **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Pain assesment by Visual Analogue Scale (VAS scale), range 0-10, higher numbers represents worse outcome. **Measure:** Visual Analogue Scale (VAS) change from baseline till 24 hours after intervention **Time Frame:** assessment at the baseline 24 hours after intervention **Description:** Assesment of milk outflow based on infant body mass increase **Measure:** Human milk outflow change from baseline till 24 hours after intervention **Time Frame:** assessment at the baseline and 24 hours after intervention **Description:** assesment of breast engorgement, range 1-6, higher values represent worse outcomes **Measure:** 6 point self-rated breast engorgement scale **Time Frame:** assessment at the baseline **Description:** assessment of improvement of signs and symptoms of breast engorgement range 1-5, higher values represent worse outcomes **Measure:** 5 point perceived improvement scale **Time Frame:** assessment 24 hours after intervention **Description:** assessment of mother's attitude to breastfeeding **Measure:** The Breastfeeding Self-Efficacy Scale **Time Frame:** assessment at the baseline #### Secondary Outcomes **Description:** Pain assesment by Visual Analogue Scale (VAS scale), range from 0-10, higher numbers represents worse outcome. **Measure:** Visual Analogue Scale (VAS) change from baseline till 3 hours after intervention **Time Frame:** assessment at the baseline 3 hours after intervention **Description:** Assesment of milk outflow based on infant body mass increase **Measure:** Human milk outflow change from baseline till 3 hours after intervention **Time Frame:** assessment at the baseline and 3 hours after intervention **Description:** assessment of improvement of signs and symptoms of breast engorgement, range 1-5, higher values represent worse outcomes **Measure:** 5 point perceived improvement scale **Time Frame:** assessment 3 hours after intervention ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * cesarian section or vaginal delivery * delivery between 37 and 41 gestational week * signs and symptoms of breast engorgement (significant increase of breast between 2nd and 10th day postpartum, pain, obstructed milk outflow) * VAS 4 and more * volume of milk outflow less than 20ml * 6 point self-rated engorgement scale between 3 and 6 Exclusion Criteria: * age less than 18 and more than 45 * patients after extensive surgical breast intervention or having breast implants * allergy to kinesiology taping **Maximum Age:** 45 Years **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** a.jakobik@szpitalzelazna.pl **Name:** Anna Jakóbik, MSc **Phone:** +48 501145348 **Role:** CONTACT #### Locations **Location 1:** **City:** Warsaw **Contacts:** ***Contact 1:*** - **Name:** Anna Jakóbik, PT, MSc - **Role:** CONTACT **Country:** Poland **Facility:** Żelazna Medical Centre, St. Sophia Hospital **Status:** RECRUITING **Zip:** 01-004 #### Overall Officials **Official 1:** **Affiliation:** Centre of Postgraduate Medical Education **Name:** Dorota Sys, PhD **Role:** STUDY_CHAIR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Zakarija-Grkovic I, Stewart F. Treatments for breast engorgement during lactation. Cochrane Database Syst Rev. 2020 Sep 18;9(9):CD006946. doi: 10.1002/14651858.CD006946.pub4. **PMID:** 32944940 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000011644 - Term: Puerperal Disorders - ID: D000011248 - Term: Pregnancy Complications - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000001941 - Term: Breast Diseases - ID: D000012871 - Term: Skin Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases ### Condition Browse Module - Browse Leaves - ID: M9991 - Name: Hyperemia - Relevance: LOW - As Found: Unknown - ID: M10794 - Name: Lactation Disorders - Relevance: HIGH - As Found: Lactation Disorders - ID: M14499 - Name: Puerperal Disorders - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M5218 - Name: Breast Diseases - Relevance: LOW - As Found: Unknown - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007775 - Term: Lactation Disorders ### Intervention Browse Module - Browse Branches - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: ARhu - Name: Antirheumatic Agents - Abbrev: Analg - Name: Analgesics - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M10102 - Name: Ibuprofen - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00693979 **Brief Title:** A Prospective Evaluation in a Randomized Trial of TAXUS in the Treatment of De Novo Coronary Artey Lesions **Official Title:** A Prospective Evaluation in a Randomized Trial of the Safety and Efficacy of the Use of the TAXUS® ElementTM Paclitaxel-Eluting Coronary Stent System for the Treatment of De Novo Coronary Artery Lesions #### Organization Study ID Info **ID:** Taxus Perseus #### Organization **Class:** OTHER **Full Name:** National University Hospital, Singapore ### Status Module #### Expanded Access Info **Last Known Status:** NOT_YET_RECRUITING #### Last Update Post Date **Date:** 2008-06-09 **Type:** ESTIMATED **Last Update Submit Date:** 2008-06-05 **Overall Status:** UNKNOWN **Status Verified Date:** 2008-06 #### Study First Post Date **Date:** 2008-06-09 **Type:** ESTIMATED **Study First Submit Date:** 2008-05-28 **Study First Submit QC Date:** 2008-06-05 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Boston Scientific Corporation #### Lead Sponsor **Class:** OTHER **Name:** National University Hospital, Singapore ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** This study is to compare the safety and performance of two stents coated with the same drug (the TAXUS Element Paclitaxel-Eluting Coronary Stent System and the TAXUS Express2 Paclitaxel-Eluting Coronary Stent System). **Detailed Description:** Approximately 1264 subjects at a maximum of 100 clinical sites will be part of this study. This study will recruit approximately 30 subjects from NHC \& NUH (12 from NUH) over a period of 6 to 9 months recruitment period. Paclitaxel is the active ingredient in Taxol®, a drug originally developed to treat cancer. The addition of the paclitaxel coating to the stent could improve the performance of the stent by preventing the re-narrowing of the treated coronary artery. This re-narrowing is called restenosis. Restenosis can occur after balloon angioplasty or the placement of a stent and is the result of too much cell growth at the treatment site in the coronary artery. The TAXUS Element stent is an investigational device. The term investigational means that the stent is not currently approved for commercial use by the FDA or other regulatory agencies worldwide. The TAXUS Express 2 stent is also a coronary stent made with the same drug coating as the Element stent. It is approved for commercial use by the FDA and other regulatory agencies worldwide, with the exception of the 4.0 mm size, which is not yet approved by the FDA. However, the 4.0 mm size was studied for safety and performance in a clinical trial and the results of that trial have been submitted to FDA to support approval. FDA has agreed that the 4.0 mm size may be used in this trial. Paclitaxel was selected as the drug to coat the stent because it is known to prevent the uncontrolled cell growth that contributes to the narrowing of artery, by interfering with the ability of cells to divide and multiply. Because of these properties, it has been used as a drug for the treatment of various types of cancer. Cancer patients are given paclitaxel as a solution into the vein. For this study, paclitaxel will be administered locally to the wall of coronary artery as a coating on the stent. Cancer patients receive a dose of paclitaxel approximately 1100 - 1400 times greater than the dose used in the coating of the stent. It is highly unlikely that levels of paclitaxel in the blood would be measurable or have effects anywhere beyond the heart. ### Conditions Module **Conditions:** - Coronary Heart Disease ### Design Module **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** Paclitaxel was selected as the drug to coat the stent because it is known to prevent the uncontrolled cell growth that contributes to the narrowing of artery, by interfering with the ability of cells to divide and multiply. Because of these properties, it has been used as a drug for the treatment of various types of cancer. Cancer patients are given paclitaxel as a solution into the vein. For this study, paclitaxel will be administered locally to the wall of coronary artery as a coating on the stent. Cancer patients receive a dose of paclitaxel approximately 1100 - 1400 times greater than the dose used in the coating of the stent. It is highly unlikely that levels of paclitaxel in the blood would be measurable or have effects anywhere beyond the heart. **Name:** TAXUS Element Stent or TAXUS Express Stent **Other Names:** - Paclitaxel in Taxol - 4.0 mm TAXUS Element stent **Type:** DEVICE ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Clinical Inclusion Criteria * Subject is ≥ 18 years old and ≤ 80 years old * Eligible for percutaneous coronary intervention (PCI) * Documented stable angina pectoris (Canadian Cardiovascular Society \[CCS\] Classification 1, 2, 3, or 4) or unstable angina pectoris with documented ischemia (Braunwald Class IB-C, IIB-C, or IIIB-C), or documented silent ischemia * Acceptable candidate for coronary artery bypass grafting (CABG) * Left ventricular ejection fraction (LVEF) is \> 30% * Subject (or legal guardian) understands the study requirements and the treatment procedures and provides written Informed Consent before any study-specific tests or procedures are performed * Subject willing to comply with all specified follow-up evaluations Angiographic Inclusion Criteria (visual estimate) * Target lesion located in native coronary artery * Target lesion must be de novo * Target lesion diameter stenosis \> 50% * Reference vessel diameter (RVD) \> 2.75mm to \< 4.0mm * Cumulative target lesion length \< 28 mm (area to be treated must be completely coverable by one study stent) * Target lesion is successfully pre-dilated. Subjects are enrolled after successful balloon catheter pre-dilation of the target lesion and assignment to treatment arm by IVRS. * One non-target lesion may be treated in a non-target vessel * Non-target lesion in non-target vessel must be treated with a commercially available TAXUS stent if use of drug-eluting stent required. * Treatment must be deemed a clinical angiographic success, without requiring use of unplanned additional stent(s). * Treatment must be completed prior to treatment of target lesion. Exclusion Criteria: Clinical Exclusion Criteria * Contraindication to ASA, or to both clopidogrel and ticlopidine * Known hypersensitivity to paclitaxel * Known allergy to stainless steel * Known allergy to platinum * Previous treatment of the target vessel with any anti-restenotic drug-coated or drug-eluting coronary stent * Previous treatment of the target vessel with a bare metal stent (BMS) within 9 months of the index procedure * Previous treatment of any non-target vessel with any anti-restenotic drug-coated or drug-eluting coronary stent within 9 months of the index procedure * Previous treatment with intravascular brachytherapy in the target vessel * Planned PCI or CABG post-index procedure * Planned or actual target vessel treatment with an unapproved device, directional or rotational coronary atherectomy, laser, cutting balloon or transluminal extraction catheter immediately prior to stent placement * Myocardial infarction (MI) within 72 hours prior to the index procedure as defined per protocol definition (see Appendix A) * Cerebrovascular accident (CVA) within the past 6 months * Cardiogenic shock characterized by systolic pressure \< 80mm Hg and/or central filling pressure \> 20 mm Hg, or cardiac index \< 1.8 liters/minute/m2 or intra-aortic balloon pump or intravenous inotropes are needed to maintain a systolic pressure \> 80 mm Hg and a cardiac index \> 1.8 liters/minute/m2 * Acute or chronic renal dysfunction (creatinine \> 2.0 mg/dl or 177 μmol/l) * Any prior true anaphylactic reaction to contrast agents; defined as known anaphylactoid or other non-anaphylactic allergic reactions to contrast agents that cannot be adequately pre-medicated prior to the index procedure * Leukopenia (leukocyte count \< 3.5 x 109/liter) * Thrombocytopenia (platelet count \< 100,000/mm3) * Thrombocytosis (\> 750,000/mm3) * Active peptic ulcer or active gastrointestinal (GI) bleeding * Current treatment, or past treatment within 12 months of the index procedure, with paclitaxel or other chemotherapeutic agent(s) * Anticipated treatment with paclitaxel or oral rapamycin during any period in the 9 months after the index procedure * Male or female with known intention to procreate within 9 months after the index procedure * Positive pregnancy test within 7 days before the index procedure, or lactating * Life expectancy of less than 24 months due to other medical conditions * Co-morbid condition(s) that could limit the subject's ability to comply with study follow-up requirements or impact the scientific integrity of the study * Currently participating in another investigational drug or device study Angiographic Exclusion Criteria (visual estimate) * Target lesion in left main artery, whether protected or unprotected * Target lesion is a chronic total occlusion (TIMI flow \< 1) * Target lesion is restenotic * Target lesion is located in a saphenous vein graft or mammary artery graft * Target lesion is accessed via saphenous vein graft or mammary artery graft * Target lesion is \< 5mm from bare metal stent (BMS) * Target lesion is \< 5mm from ostium * Target lesion is \< 5 mm from a side branch vessel ≥ 1.5 mm in diameter * Untreated lesions with ≥ 50% diameter stenosis or thought to impair flow remaining in target vessel * Target lesion and/or target vessel proximal to the target lesion is moderately or severely calcified * Target lesion and/or target vessel proximal to the target lesion is severely tortuous * Target lesion is located within or distal to a \> 60° bend in the vessel * Target lesion with angiographic presence of probable or definite thrombus * Unprotected left main coronary artery disease * Protected left main coronary artery disease with target lesion in LAD or LCx(subject may be enrolled if only lesion is target lesion in RCA) **Maximum Age:** 80 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000014652 - Term: Vascular Diseases - ID: D000001161 - Term: Arteriosclerosis - ID: D000001157 - Term: Arterial Occlusive Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M9419 - Name: Heart Diseases - Relevance: HIGH - As Found: Heart Disease - ID: M6549 - Name: Coronary Disease - Relevance: HIGH - As Found: Coronary Heart Disease - ID: M6546 - Name: Coronary Artery Disease - Relevance: HIGH - As Found: Coronary Heart Disease - ID: M19506 - Name: Myocardial Ischemia - Relevance: HIGH - As Found: Coronary Heart Disease - ID: M10543 - Name: Ischemia - Relevance: LOW - As Found: Unknown - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown - ID: M4469 - Name: Arteriosclerosis - Relevance: LOW - As Found: Unknown - ID: M4465 - Name: Arterial Occlusive Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006331 - Term: Heart Diseases - ID: D000003327 - Term: Coronary Disease - ID: D000003324 - Term: Coronary Artery Disease - ID: D000017202 - Term: Myocardial Ischemia ### Intervention Browse Module - Ancestors - ID: D000000972 - Term: Antineoplastic Agents, Phytogenic - ID: D000000970 - Term: Antineoplastic Agents - ID: D000050257 - Term: Tubulin Modulators - ID: D000050256 - Term: Antimitotic Agents - ID: D000050258 - Term: Mitosis Modulators - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: PhSol - Name: Pharmaceutical Solutions ### Intervention Browse Module - Browse Leaves - ID: M19537 - Name: Paclitaxel - Relevance: HIGH - As Found: Surgery - ID: M231 - Name: Albumin-Bound Paclitaxel - Relevance: LOW - As Found: Unknown - ID: M21860 - Name: Pharmaceutical Solutions - Relevance: LOW - As Found: Unknown - ID: M26197 - Name: Tubulin Modulators - Relevance: LOW - As Found: Unknown - ID: M26196 - Name: Antimitotic Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000017239 - Term: Paclitaxel ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05940779 **Brief Title:** Relationship Between the Risk of Falls and Frailty, and the Effect of a Physical Exercise Program on These Conditions in the Elderly: a Randomized Crossover Clinical Trial. **Official Title:** Relationship Between the Risk of Falls and Frailty, and the Effect of a Physical Exercise Program on These Conditions in the Elderly: a Randomized Crossover Clinical Trial. #### Organization Study ID Info **ID:** H1504602336419 #### Organization **Class:** OTHER **Full Name:** University of Valencia ### Status Module #### Completion Date **Date:** 2024-09-30 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-07-11 **Type:** ACTUAL **Last Update Submit Date:** 2023-07-03 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-09-30 **Type:** ESTIMATED #### Start Date **Date:** 2021-09-22 **Type:** ACTUAL **Status Verified Date:** 2023-05 #### Study First Post Date **Date:** 2023-07-11 **Type:** ACTUAL **Study First Submit Date:** 2023-05-31 **Study First Submit QC Date:** 2023-07-03 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Valencia #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** The goal of this randomized crossover clinical trial is to examine the effectiveness of a new therapeutic exercise program in elderly patients with risk of falls and physical fragility. The main questions it aims to answer are: * If the therapeutic exercise program proposed is a successful treatment for this kind of patients * If patients at risk of falls also suffer from pre-frailty or frailty, and if they can be treated together with the proposed therapy. Participants will follow a program consisting of exercises to correct posture, gain strength and contribute to greater balance. Researchers will compare the therapy and control groups to see if the program increases the percentage of muscle mass of participants, their mobility, balance, quality of life and if they reduce their Fried´s frailty criteria, fear of falling and falls compared to their usual physical activity. ### Conditions Module **Conditions:** - Frail Elderly Syndrome - Fall ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER ##### Masking Info **Masking:** DOUBLE **Who Masked:** - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 40 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Other: Therapeutic exercise program **Label:** Exercise group **Type:** EXPERIMENTAL #### Arm Group 2 **Label:** Control group **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Exercise group **Description:** The intervention is a therapeutic program composed of self-resisted strength exercises, balance training and exercises to improve posture. **Name:** Therapeutic exercise program **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** Timed Up and Go (TUG) **Time Frame:** Assessed before the first intervention (baseline). **Measure:** Timed Up and Go (TUG) **Time Frame:** Assessed after the first intervention (3 months later). **Measure:** Timed Up and Go (TUG) **Time Frame:** Assessed after the second intervention (3 months after the previous assessement). **Measure:** Timed Up and Go (TUG) **Time Frame:** Assessed after 3 months from the previous assessement. **Description:** The Fried frailty criteria are five: unintentional weight loss, muscular weakness, low resistance or tiredness, slow gait and low level of physical activity. **Measure:** Fried frailty criteria **Time Frame:** Assessed before the first intervention (baseline). **Description:** The Fried frailty criteria are five: unintentional weight loss, muscular weakness, low resistance or tiredness, slow gait and low level of physical activity. **Measure:** Fried frailty criteria **Time Frame:** Assessed after the first intervention (3 months later). **Description:** The Fried frailty criteria are five: unintentional weight loss, muscular weakness, low resistance or tiredness, slow gait and low level of physical activity. **Measure:** Fried frailty criteria **Time Frame:** Assessed after the second intervention (3 months after the previous assessement). **Description:** The Fried frailty criteria are five: unintentional weight loss, muscular weakness, low resistance or tiredness, slow gait and low level of physical activity. **Measure:** Fried frailty criteria **Time Frame:** Assessed after 3 months from the previous assessement. #### Secondary Outcomes **Description:** The number of falls in the last year will be asked. **Measure:** Falls and their consequences **Time Frame:** Assessed before the first intervention (baseline). **Description:** A follow-up made with phone calls. **Measure:** Falls and their consequences **Time Frame:** Every two weeks from date of the first assessement until the date of the study completion, an average of 1 year. **Description:** From 0 to 56. It shows a better balance when the score is higher. **Measure:** Berg Balance Scale (BBS) **Time Frame:** Assessed before the first intervention (baseline). **Description:** From 0 to 56. It shows a better balance when the score is higher. **Measure:** Berg Balance Scale (BBS) **Time Frame:** Assessed after the first intervention (3 months later). **Description:** From 0 to 56. It shows a better balance when the score is higher. **Measure:** Berg Balance Scale (BBS) **Time Frame:** Assessed after the second intervention (3 months after the previous assessement). **Description:** From 0 to 56. It shows a better balance when the score is higher. **Measure:** Berg Balance Scale (BBS) **Time Frame:** Assessed after 3 months from the previous assessement. **Description:** From 16 to 64. Higher scores show more concern of falling. **Measure:** Falls Efficacy Scale-International (FES-I) **Time Frame:** Assessed before the first intervention (baseline). **Description:** From 16 to 64. Higher scores show more concern of falling. **Measure:** Falls Efficacy Scale-International (FES-I) **Time Frame:** Assessed after the first intervention (3 months later). **Description:** From 16 to 64. Higher scores show more concern of falling. **Measure:** Falls Efficacy Scale-International (FES-I) **Time Frame:** Assessed after the second intervention (3 months after the previous assessement). **Description:** From 16 to 64. Higher scores show more concern of falling. **Measure:** Falls Efficacy Scale-International (FES-I) **Time Frame:** Assessed after 3 months from the previous assessement. **Description:** It has two parts: The first one consists of questions about quality of life with scores from 0 to 1, higher if the quality of life of the patient is higher too. The second one is a visual analogic scale (VAS) from 0 (worst quality of life) to 100 (bes quality of life). **Measure:** European Quality of Life-5 Dimensions (EuroQol-5D) **Time Frame:** Assessed before the first intervention (baseline). **Description:** It has two parts: The first one consists of questions about quality of life with scores from 0 to 1, higher if the quality of life of the patient is higher too. The second one is a visual analogic scale (VAS) from 0 (worst quality of life) to 100 (bes quality of life). **Measure:** European Quality of Life-5 Dimensions (EuroQol-5D) **Time Frame:** Assessed after the first intervention (3 months later). **Description:** It has two parts: The first one consists of questions about quality of life with scores from 0 to 1, higher if the quality of life of the patient is higher too. The second one is a visual analogic scale (VAS) from 0 (worst quality of life) to 100 (bes quality of life). **Measure:** European Quality of Life-5 Dimensions (EuroQol-5D) **Time Frame:** Assessed after the second intervention (3 months after the previous assessement). **Description:** It has two parts: The first one consists of questions about quality of life with scores from 0 to 1, higher if the quality of life of the patient is higher too. The second one is a visual analogic scale (VAS) from 0 (worst quality of life) to 100 (bes quality of life). **Measure:** European Quality of Life-5 Dimensions (EuroQol-5D) **Time Frame:** Assessed after 3 months from the previous assessement. **Description:** Assessed with and impedance scale. **Measure:** Muscle mass **Time Frame:** Assessed before the first intervention (baseline). **Description:** Assessed with and impedance scale. **Measure:** Muscle mass **Time Frame:** Assessed after the first intervention (3 months later). **Description:** Assessed with and impedance scale. **Measure:** Muscle mass **Time Frame:** Assessed after the second intervention (3 months after the previous assessement). **Description:** Assessed with and impedance scale. **Measure:** Muscle mass **Time Frame:** Assessed after 3 months from the previous assessement. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Community dwelling older adults. * Independent for ambulation. * With a cognitive state that do not prevent understand the researcher indications. * Not performing regular physical exercise (only basic activities of daily living, but no other type of intervention). Exclusion Criteria: * Contraindication of physical exercise. * Evident impaired cognitive state. * Vestibular or central nervous system affection. * Can´t speak and understand spanish correctly. **Healthy Volunteers:** True **Minimum Age:** 65 Years **Sex:** ALL **Standard Ages:** - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** bequelte@gmail.com **Name:** Belén Quel Tejón **Phone:** 675760137 **Role:** CONTACT **Contact 2:** **Email:** Jose.Maria.Blasco@uv.es **Name:** José María Blasco Igual **Phone:** 963983855 **Role:** CONTACT #### Locations **Location 1:** **City:** Valencia **Contacts:** ***Contact 1:*** - **Email:** Jose.Maria.Blasco@uv.es - **Name:** José María Blasco Igual - **Phone:** 963983855 - **Role:** CONTACT **Country:** Spain **Facility:** University of Valencia **Status:** RECRUITING **Zip:** 46010 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M1175 - Name: Frailty - Relevance: HIGH - As Found: Frailty ### Condition Browse Module - Meshes - ID: D000073496 - Term: Frailty ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03262779 **Brief Title:** Ipilimumab and Nivolumab in Patients With Anti-PD-1-axis Therapy-resistant Advanced Non-small Cell Lung Cancer. **Official Title:** A Phase II Study of Combination Immunotherapy With Ipilimumab and Nivolumab in Patients With Advanced Non-small Cell Lung Cancer Resistant to Anti-PD-1-axis Therapy #### Organization Study ID Info **ID:** 2000020343 #### Organization **Class:** OTHER **Full Name:** Yale University ### Status Module #### Completion Date **Date:** 2022-01-14 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-09-23 **Type:** ACTUAL **Last Update Submit Date:** 2022-09-21 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-01-14 **Type:** ACTUAL #### Start Date **Date:** 2017-07-20 **Type:** ACTUAL **Status Verified Date:** 2022-09 #### Study First Post Date **Date:** 2017-08-25 **Type:** ACTUAL **Study First Submit Date:** 2017-08-23 **Study First Submit QC Date:** 2017-08-23 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Bristol-Myers Squibb #### Lead Sponsor **Class:** OTHER **Name:** Yale University #### Responsible Party **Investigator Affiliation:** Yale University **Investigator Full Name:** Scott Gettinger, MD **Investigator Title:** Assistant Professor of Medicine **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** True ### Description Module **Brief Summary:** The investigators propose a trial to evaluate if the addition of ipilimumab to nivolumab after primary or acquired resistance to anti- programmed death 1 (PD-1) axis therapy can lead to objective radiographic tumor regression. **Detailed Description:** Approximately 20 percent of unselected patients with advanced non-small cell lung cancer (NSCLC) and progression during or after standard first line chemotherapy will experience tumor response to nivolumab. Treatment options for patients who are not responsive to programmed death 1 (PD-1) axis inhibitor therapy are limited, and the mechanisms of primary resistance are poorly understood. The combination of nivolumab and ipilimumab is currently FDA approved for the treatment of advanced melanoma based on superiority to either agent alone5. The results of a phase I study evaluating combination therapy with nivolumab and ipilimumab in patients with advanced NSCLC (NCT01454102) were presented at the annual American Society of Clinical Oncology (ASCO) meeting in 20166. Dosing of nivolumab 3 mg/kg every 2 weeks and ipilimumab 1 mg/kg every 6 weeks yielded an objective response rate (ORR) by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 of 39%, with one-year survival rate of 69% and grade 3-4 treatment-related adverse event rate of 33%. These results prompted an ongoing phase III study comparing this regimen to standard first line chemotherapy, nivolumab monotherapy or combination therapy with chemotherapy and nivolumab for patients with advanced NSCLC (NCT02477826). The investigators propose a trial to evaluate if the addition of ipilimumab to nivolumab after primary resistance to anti-PD-1 axis therapy can lead to objective radiographic tumor regression. It is hypothesized that ipilimumab will enable more effective immune priming in some patients, resulting in the trafficking of tumor-specific cytotoxic T cells to the tumor, as well as depletion of tumor-permissive T regulatory cells. With concurrent nivolumab, PD-1 inhibition in the tumor will enable effective anti-tumor attack by tumor-specific T cells. Serial tumor biopsies and blood collections will allow interrogation of changes in the tumor microenvironment (and periphery) that support this hypothesis. The investigators will primarily enroll patients who have experienced progression of NSCLC after anti-PD-1- axis therapy without initial response to such therapy ('primary resistance'). A smaller cohort of patients with acquired resistance to anti-PD-1 axis therapy (i.e. progression after initial response) will additionally be accrued. The study record was updated to add individual arms for those with primary resistance and acquired resistance. The intent of the study is not to compare these treatment arms. ### Conditions Module **Conditions:** - Carcinoma, Non-Small-Cell Lung ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** SINGLE_GROUP **Intervention Model Description:** This a phase II study employing a Simon two-stage design. If there is at least one response or prolonged stability (\> 24 weeks) using immune related Response Criteria (irRC) appreciated in the first 10 patients who initiate trial therapy, a total of 40 patients will be enrolled. An additional 10 patients with acquired resistance to anti-PD-1 axis therapy will be enrolled in an exploratory cohort receiving the same therapy. ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 20 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Combination therapy with nivolumab 3 mg/kg administered intravenously (IV) every 2 weeks, and ipilimumab 1 mg/kg administered IV every 6 weeks in patients with primary resistance. **Intervention Names:** - Biological: combination nivolumab and ipilimumab **Label:** combination nivolumab and ipilimumab - primary **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Combination therapy with nivolumab 3 mg/kg administered intravenously (IV) every 2 weeks, and ipilimumab 1 mg/kg administered IV every 6 weeks in patients with acquired resistance. **Intervention Names:** - Biological: combination nivolumab and ipilimumab **Label:** combination nivolumab and ipilimumab - acquired **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - combination nivolumab and ipilimumab - acquired - combination nivolumab and ipilimumab - primary **Description:** Combination therapy with nivolumab 3 mg/kg administered intravenously (IV) every 2 weeks, with ipilimumab 1 mg/kg administered IV every 6 weeks. **Name:** combination nivolumab and ipilimumab **Type:** BIOLOGICAL ### Outcomes Module #### Primary Outcomes **Description:** Objective response is defined as a complete or partial response, as determined by investigator assessment using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 confirmed by repeat assessment ≥4 weeks after initial documentation. **Measure:** Objective response rate using RECIST v1.1 to nivolumab and ipilimumab when administered in combination to patients with pre-treated advanced NSCLC who have experienced primary resistance to anti-PD-1 axis therapy as their last line of systemic therapy. **Time Frame:** Tumor response assessment will occur every 9 weeks after initiating trial therapy for the first 24 weeks, and thereafter every 12 weeks until disease progression or up to 4 years. #### Secondary Outcomes **Description:** Progression free survival is defined as the time from the first day of nivolumab treatment until progression of disease using RECIST v1.1 and immune related Response Criteria (irRC) **Measure:** Progression-free survival with nivolumab and ipilimumab when administered in combination to patients with pre- treated advanced NSCLC who have experienced primary resistance to anti-PD-1 axis therapy as their last line of systemic therapy **Time Frame:** Until disease progression, unacceptable toxicity, or study termination, up to four years. **Description:** Overall survival is defined as the time from the first day of treatment until death from any cause. If a patient has not experienced death, OS will be censored at the day of last follow-up. **Measure:** Overall survival (OS) with nivolumab and ipilimumab when administered in combination to patients with pre- treated advanced NSCLC who have experienced PRIMARY resistance to anti-PD-1 axis therapy as their last line of systemic therapy. **Time Frame:** Until death or day of last follow-up, up to four years from enrollment. **Description:** Objective response is defined as a complete or partial response, as determined by investigator assessment using irRC and confirmed by repeat assessment ≥4 weeks after initial documentation. **Measure:** Objective response rate using irRC to nivolumab and ipilimumab when administered in combination to patients with pre-treated advanced NSCLC who have experienced primary resistance to anti-PD-1 axis therapy as their last line of systemic therapy. **Time Frame:** Tumor response assessment will occur every 9 weeks after initiating trial therapy for the first 24 weeks, and thereafter every 12 weeks, up to four years. **Description:** Objective response is defined as a complete or partial response, as determined by investigator assessment using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 and immune-related Response Criteria and confirmed by repeat assessment ≥4 weeks after initial documentation. **Measure:** Objective response rate in in advanced non-small cell lung cancer (NSCLC) with ACQUIRED resistance to anti-programmed death (PD)-1 axis therapy as their last line of systemic therapy. **Time Frame:** Tumor response assessment will occur every 9 weeks after initiating trial therapy for the first 24 weeks, and thereafter every 12 weeks, up to four years. **Description:** Progression free survival is defined as the time from the first day of nivolumab treatment until progression of disease using RECIST v1.1 and immune related Response Criteria **Measure:** Progression free survival by RECIST v1.1 with nivolumab and ipilimumab in patients with pre-treated advanced NSCLC who have experienced acquired resistance to anti-PD-1 axis therapy as their last line of systemic therapy. **Time Frame:** Until disease progression, unacceptable toxicity, or study termination, up to four years from enrollment. **Description:** Overall survival is defined as the time from the first day of treatment until death from any cause. If a patient has not experienced death, OS will be censored at the day of last follow-up. **Measure:** Overall survival with nivolumab and ipilimumab in patients with pre-treated advanced NSCLC who have experienced ACQUIRED resistance to anti-PD-1 axis therapy as their last line of systemic therapy. **Time Frame:** Until death or day of last follow-up (up to four years from study enrollment). **Description:** Determined based on adverse events which will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events v4.0. **Measure:** Safety of nivolumab and ipilimumab when administered in combination in patients with pre-treated advanced NSCLC who have experienced PRIMARY or ACQUIRED resistance to anti-PD-1 axis therapy as their last line of systemic therapy. **Time Frame:** From date of first treatment until 100 days from discontinuation of treatment. **Measure:** Feasibility of sequential biopsies (performed or not performed) in patients with pre-treated advanced NSCLC who have experienced PRIMARY or ACQUIRED resistance to anti-PD-1 axis therapy as their last line of systemic therapy, **Time Frame:** Tumor biopsies will be performed just prior to initiating trial therapy, and 9 to 10 weeks after receiving first dose of trial therapy. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria A. Signed Informed Consent B. Ability to comply with the protocol C. Age ≥18 years D. Histologically or cytologically documented, locally advanced or metastatic (i.e., Stage IIIB not eligible for definitive chemoradiotherapy, Stage IV, or recurrent) NSCLC (per the American Joint Committee /AJCC staging system) E. ECOG performance status of 0 to 2 F. Measurable disease, as defined by RECIST v1.1. Previously irradiated lesions can be counted as target lesions if clearly progressing after radiation. G. Chemotherapy-naive and treated patients will be eligible, with no limit on number of prior therapies. Patients with NSCLC known to harbor an ALK rearrangement, or EGFR mutation known to be sensitive to FDA-approved tyrosine kinase inhibitors (TKI), are only eligible after experiencing disease progression (during or after treatment) or intolerance to an FDA approved EGFR TKI or ALK TKI, respectively. 1. Patients with TKI-treated EGFR mutant NSCLC harboring the secondary EGFR T790M tumor must have received prior osimertinib 2. Patients with crizotinib-treated ALK rearranged NSCLC must have received a next generation ALK inhibitor (e.g. ceritinib, alectinib or brigatinib) H. Prior palliative radiotherapy must have been completed at least 2 weeks before the first dose of study drug. I. Anti- PD-1 Axis therapy (anti-PD-1 or anti-PD-L1, e.g. nivolumab, pembrolizumab, atezolizumab, durvalumab, avelumab) must be the most recent systemic anti-tumor treatment received in all patients, with documented progressive disease. Last administration of anti-PD-1 axis therapy must have been at least 3 weeks before the first dose of study drug. a. Patients to be enrolled to the primary cohort (primary resistance) must have had progressive disease or stable disease less than 24 weeks as the best clinical response to anti-PD-1-axis monotherapy b. Patients to be enrolled to the exploratory cohort (acquired resistance) must have had stable disease for at least 24 weeks, partial response, or complete response as the best clinical response to anti-PD-1-axis monotherapy, with subsequent progression of disease J. At least one tumor amenable to incisional, excisional, core or forceps (transbronchial) biopsy. Patients must be willing to undergo tumor biopsies before starting trial therapy, and 9 to 10 weeks after initiation of therapy. a. If the initial biopsy will be excisional, the excised tumor cannot be counted as a target lesion and there must be another lesion amenable to incisional, excisional, core or forceps biopsy. In this scenario, the second biopsy can only be excisional if the lesion to be excised is not a target lesion. b. Cytology tumor specimens (e.g. from fine-needle biopsies, or drainage of pleural/ pericardial or ascites fluid) are not acceptable. Biopsies of bone lesions that do not have a soft tissue component are also not acceptable (i.e. decalcified tumor samples are not acceptable). K. For female patients of childbearing potential and male patients with partners of childbearing potential, agreement (by patient and/or partner) to use a highly effective form(s) of contraception (i.e., one that results in a low failure rate \[\<1% per year\] when used consistently and correctly) and to continue its use for 6 months after the last dose of trial therapy. Highly effective contraception is one with a failure rate of \<0.1%. Birth control pills on their own do not achieve that rate. 1. Women of childbearing potential must have a negative pregnancy test (serum or urine) within 72 hours of the start of study drug administration 2. Women who have recently given birth must no longer be breastfeeding L. Adequate hematologic and end-organ function, defined by the following laboratory results obtained within 14 days prior to the first study treatment: - Neutrophils ≥1500 cells/μL (without granulocyte colony-stimulating factor support within 2 weeks prior to Cycle 1, Day 1) * Platelets ≥75,000/μL (transfusion to achieve this level is not permitted within 2 weeks of the first study drug administration) * Hemoglobin ≥9.0 g/dL * Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤3 x institutional upper limit of normal (ULN) with the following exceptions: Patients with documented liver metastases: AST and/or ALT≤5 x ULN * Serum bilirubin ≤1.5 x ULN (Patients with known Gilbert disease who have serum bilirubin level ≤3 x ULN may be enrolled) * Serum creatinine ≤1.5 x ULN or creatinine clearance ≥50 mL/min Exclusion Criteria A. Has an active autoimmune disease requiring systemic treatment within the past 3 months or a documented history of clinically severe autoimmune disease, or a syndrome that requires systemic steroids or immunosuppressive agents. Subjects with vitiligo or resolved childhood asthma/atopy would be an exception to this rule. Subjects who require intermittent use of inhaled steroids or local steroid injections would not be excluded from the study. Subjects with hypothyroidism stable on hormone replacement, or psoriasis not requiring systemic therapy (within the past 3 years) will not be excluded from the study. B. Interstitial lung disease that is symptomatic or may interfere with the detection or management of suspected drug-related pulmonary toxicity C. Subjects must not have a history of life-threatening toxicity related to prior anti-PD-1 axis therapy a. Subjects with history of anti-PD-1 axis therapy toxicities that are unlikely to recur with standard countermeasures (e.g., hormone replacement after adrenal crisis) are eligible. D. Prior treatment with anti-CTLA-4 therapeutic antibodies E. Symptomatic or untreated CNS metastases. Patients with a history of treated asymptomatic CNS metastases are eligible, provided they meet all of the following criteria: 1. No evidence of interim progression between the completion of CNS-directed therapy and the start of trial therapy. 2. No ongoing requirement for dexamethasone as therapy for CNS disease; anticonvulsants at a stable dose are allowed. 3. Completed stereotactic radiosurgery at least 1 week prior to Cycle 1, Day 1 or wholebrain radiation at least 2 weeks prior to Cycle 1, Day 1 F. History of leptomeningeal carcinomatosis G. Prior palliative radiotherapy outside the CNS within 2 weeks of the first dose of study drug. H. Treatment with systemic immunosuppressive medications (including but not limited to, dexamethasone at doses \> 2 mg daily (or equivalent dose of other corticosteroids), cyclophosphamide, tacrolimus, sirolimus, azathioprine, methotrexate, thalidomide, and antitumor necrosis factor \[anti-TNF\] agents) within 2 weeks prior to initiating trial therapy (Inhaled or topically applied steroids, and acute and chronic standard-dose NSAIDs are permitted. Replacement steroids are also permitted). I. Subjects must not have received vaccines containing live virus for prevention of infectious diseases within 12 weeks prior to the first dose of study drug. a. The use of inactivated seasonal influenza vaccines (eg, Fluzone®) will be permitted on study without restriction. J. Any approved systemic anti-cancer therapy, within 3 weeks prior to initiation of study treatment; the following exception is allowed: * TKIs approved for treatment of NSCLC discontinued \> 7 days prior to Cycle 1, Day 1. The baseline scan must be obtained after discontinuation of prior TKIs. K. Treatment with any other investigational agent or participation in another clinical trial with therapeutic intent within 28 days prior to enrollment; the following exceptions are allowed: * Unapproved/experimental TKIs discontinued 14 days prior to Cycle 1, Day 1 L. Known infection with HIV, HBV or HCV. Patients with prior exposure to hepatitis, but no evidence of active or chronic infection, may be eligible. * Subjects with positive hepatitis C antibody and negative quantitative hepatitis C by polymerase chain reaction are eligible. M. Active systemic infection requiring systemic antibiotic treatment within 72 hours prior to first dose of study treatment N. Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, or psychiatric illness/social situations that would limit compliance with study requirements O. Major surgery or traumatic injury within 4 weeks of starting study drug P. Women who are pregnant or lactating. Q. Any underlying medical condition that in the Principal Investigator's opinion will make the administration of study drug hazardous to the patient or would obscure the interpretation of adverse events. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** New Haven **Country:** United States **Facility:** Yale Cancer Center **State:** Connecticut **Zip:** 06510 #### Overall Officials **Official 1:** **Affiliation:** Yale University **Name:** Scott Gettinger, M.D. **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002283 - Term: Carcinoma, Bronchogenic - ID: D000001984 - Term: Bronchial Neoplasms - ID: D000008175 - Term: Lung Neoplasms - ID: D000012142 - Term: Respiratory Tract Neoplasms - ID: D000013899 - Term: Thoracic Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases ### Condition Browse Module - Browse Leaves - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - ID: M11172 - Name: Lung Neoplasms - Relevance: LOW - As Found: Unknown - ID: M5546 - Name: Carcinoma, Non-Small-Cell Lung - Relevance: HIGH - As Found: Carcinoma, Non-Small-Cell Lung - ID: M5540 - Name: Carcinoma, Bronchogenic - Relevance: LOW - As Found: Unknown - ID: M5260 - Name: Bronchial Neoplasms - Relevance: LOW - As Found: Unknown - ID: M14979 - Name: Respiratory Tract Neoplasms - Relevance: LOW - As Found: Unknown - ID: M16658 - Name: Thoracic Neoplasms - Relevance: LOW - As Found: Unknown - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002289 - Term: Carcinoma, Non-Small-Cell Lung ### Intervention Browse Module - Ancestors - ID: D000074322 - Term: Antineoplastic Agents, Immunological - ID: D000000970 - Term: Antineoplastic Agents - ID: D000082082 - Term: Immune Checkpoint Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: ANeo - Name: Antineoplastic Agents ### Intervention Browse Module - Browse Leaves - ID: M2853 - Name: Immunomodulating Agents - Relevance: LOW - As Found: Unknown - ID: M1854 - Name: Nivolumab - Relevance: HIGH - As Found: Prospective - ID: M1348 - Name: Ipilimumab - Relevance: HIGH - As Found: While - ID: M1346 - Name: Antineoplastic Agents, Immunological - Relevance: LOW - As Found: Unknown - ID: M2342 - Name: Immune Checkpoint Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000077594 - Term: Nivolumab - ID: D000074324 - Term: Ipilimumab ### Misc Info Module #### Submission Tracking - Estimated Results First Submit Date: 2023-04-14 ##### Submission Infos - MCP Release N: 7 - Release Date: 2023-04-14 - Reset Date: 2023-05-09 - Unrelease Date: Unknown - Unrelease Date Unknown: Unknown - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00617279 **Acronym:** PRODIGY **Brief Title:** GORE PROPATEN Vascular Graft vs. Disadvantaged Autologous Vein Graft **Official Title:** Comparison of Primary Patency Between GORE PROPATEN Vascular Graft and Disadvantaged Autologous Vein Graft For Below-Knee Arterial Bypass (PRODIGY Study) #### Organization Study ID Info **ID:** PPT 07-05 #### Organization **Class:** INDUSTRY **Full Name:** W.L.Gore & Associates ### Status Module #### Completion Date **Date:** 2010-02 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2012-01-12 **Type:** ESTIMATED **Last Update Submit Date:** 2011-12-08 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2010-02 **Type:** ACTUAL #### Results First Post Date **Date:** 2012-01-12 **Type:** ESTIMATED **Results First Submit Date:** 2011-02-07 **Results First Submit QC Date:** 2011-12-08 #### Start Date **Date:** 2007-12 **Status Verified Date:** 2011-12 #### Study First Post Date **Date:** 2008-02-18 **Type:** ESTIMATED **Study First Submit Date:** 2007-12-18 **Study First Submit QC Date:** 2008-02-05 **Why Stopped:** Study terminated due to low enrollment ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** W.L.Gore & Associates #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The primary patency of the GORE PROPATEN Vascular Graft is equivalent to or better than disadvantaged autologous vein graft in an infragenicular peripheral bypass application at 12 months. **Detailed Description:** Primary patency is defined as hemodynamic evidence of blood flow through an open graft that has maintained uninterrupted patency and has not previously undergone a revision to restore blood flow. A disadvantaged autologous vein is defined as meeting at least one of the two following criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator; (2) Usable ipsilateral or contralateral autologous greater saphenous vein that meets any of the following criteria: (a) Diameter of less than or equal to 3.0 mm; (b) Inadequate length requiring splicing; (c) Poor quality vein that is either sclerotic or phlebitic. ### Conditions Module **Conditions:** - Peripheral Arterial Occlusive Disease ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 31 **Type:** ACTUAL **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Device: GORE PROPATEN Vascular Graft **Label:** GORE PROPATEN Vascular Graft: **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Intervention Names:** - Procedure: Disadvantaged Autologous Vein Graft **Label:** Disadvantaged Autologous Vein Graft **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - GORE PROPATEN Vascular Graft: **Description:** Arterial Occlusion Bypass **Name:** GORE PROPATEN Vascular Graft **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - Disadvantaged Autologous Vein Graft **Description:** Arterial Occlusion Bypass **Name:** Disadvantaged Autologous Vein Graft **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** Primary patency is defined as hemodynamic evidence of blood flow through an open graft that has maintained uninterrupted patency and has not previously undergone a revision to restore blood flow. **Measure:** Number of Patients With Primary Patency at 12 Months Post-procedure **Time Frame:** 12 months **Description:** The number of Major Adverse Event occurrences through one month post-procedure. A major adverse event requires significant therapy, including unplanned increase in the level of care, permanent sequelae, hospitalization, or death. This outcome measure presents the number of Major Adverse Event occurrences (i.e. - one patient could have multiple occurrences), and differs from the Serious Adverse Events reporting measure, which presents the number of patients that have been affected by a Serious Adverse Event. **Measure:** Major Adverse Event Occurrences Through One Month Post-procedure **Time Frame:** one month post-index procedure #### Secondary Outcomes **Description:** Primary patency is defined as hemodynamic evidence of blood flow through an open graft that has maintained uninterrupted patency and has not previously undergone a revision to restore blood flow. **Measure:** Number of Patients With Primary Patency at One Month Post-procedure **Time Frame:** One month **Description:** Primary patency is defined as hemodynamic evidence of blood flow through an open graft that has maintained uninterrupted patency and has not previously undergone a revision to restore blood flow. **Measure:** Number of Patients With Primary Patency at 6 Months Post-procedure **Time Frame:** 6 months **Description:** Assisted primary patency is defined as hemodynamic evidence of blood flow through an open graft that has previously undergone revision(s) within the graft to restore blood flow prior to occlusion. The number of patients analyzed at 1 month post-procedure does not equal the number of patients originally enrolled into the study. This is due to the fact that a number of enrolled patients did not have their 1 month follow-up visit (or failed to attend their 1 month visit) prior to the termination of the study. **Measure:** Number of Patients With Assisted Primary Patency at One Month Post-procedure **Time Frame:** One month **Description:** Assisted primary patency is defined as hemodynamic evidence of blood flow through an open graft that has previously undergone revision(s) within the graft to restore blood flow prior to occlusion. **Measure:** Number of Patients With Assisted Primary Patency at 6 Months Post-procedure **Time Frame:** 6 months **Description:** Assisted primary patency is defined as hemodynamic evidence of blood flow through an open graft that has previously undergone revision(s) within the graft to restore blood flow prior to occlusion. **Measure:** Number of Patients With Assisted Primary Patency at 12 Months Post-procedure **Time Frame:** 12 months **Description:** Secondary patency is defined as hemodynamic evidence of blood flow through an open graft that has previously undergone revision(s) to restore blood flow after occlusion. **Measure:** Number of Patients With Secondary Patency at One Month **Time Frame:** One month **Description:** Secondary patency is defined as hemodynamic evidence of blood flow through an open graft that has previously undergone revision(s) to restore blood flow after occlusion. **Measure:** Number of Patients With Secondary Patency at 6 Months **Time Frame:** 6 months **Description:** Secondary patency is defined as hemodynamic evidence of blood flow through an open graft that has previously undergone revision(s) to restore blood flow after occlusion. **Measure:** Number of Patients With Secondary Patency at 12 Months **Time Frame:** 12 months **Description:** Limb salvage is defined as relief from symptoms sufficient to prevent major amputation. An amputation is considered to be major when there is surgical removal of a portion of the study leg that would preclude standing and walking without a prosthesis. **Measure:** Number of Patients With Limb Salvage (no Major Amputations) at One Month Post-procedure **Time Frame:** One month **Description:** Limb salvage is defined as relief from symptoms sufficient to prevent major amputation. An amputation is considered to be major when there is surgical removal of a portion of the study leg that would preclude standing and walking without a prosthesis. **Measure:** Number of Patients With Limb Salvage (no Major Amputations) at 6 Months Post-procedure **Time Frame:** 6 months **Description:** Limb salvage is defined as relief from symptoms sufficient to prevent major amputation. An amputation is considered to be major when there is surgical removal of a portion of the study leg that would preclude standing and walking without a prosthesis. **Measure:** Number of Patients With Limb Salvage (no Major Amputations) at 12 Months Post-procedure **Time Frame:** 12 months **Description:** A major adverse event requires significant therapy, including unplanned increase in the level of care, permanent sequelae, hospitalization, or death. **Measure:** Patients Experiencing Major Adverse Events Through 6 Months Post-procedure **Time Frame:** 6 months **Description:** A major adverse event requires significant therapy, including unplanned increase in the level of care, permanent sequelae, hospitalization, or death. **Measure:** Patients Experiencing Major Adverse Events Through 12 Months Post-procedure **Time Frame:** 12 months **Measure:** Number of Patients Surviving at One Month **Time Frame:** One month **Measure:** Number of Patients Surviving at 6 Months **Time Frame:** 6 months **Measure:** Number of Patients Surviving at 12 Months **Time Frame:** 12 months **Description:** Wound/graft infection was not specifically defined in the protocol and was left to the Investigator's standard of care. **Measure:** Number of Patients With Wound/Graft Infection Through One Month Post-procedure **Time Frame:** One month **Description:** Wound/graft infection was not specifically defined in the protocol and was left to the Investigator's standard of care. **Measure:** Number of Patients With Wound/Graft Infection Through 6 Months **Time Frame:** 6 months **Description:** Wound/graft infection was not specifically defined in the protocol and was left to the Investigator's standard of care. **Measure:** Number of Patients With Wound/Graft Infection Through 12 Months **Time Frame:** 12 months **Description:** Delayed wound healing was not specifically defined in the protocol and was left to the Investigator's standard of care. **Measure:** Number of Patients With Delayed Wound Healing Through One Month Post-procedure **Time Frame:** One month **Description:** Delayed wound healing was not specifically defined in the protocol and was left to the Investigator's standard of care. **Measure:** Number of Patients With Delayed Wound Healing Through 6 Months Post-procedure **Time Frame:** 6 months **Description:** Delayed wound healing was not specifically defined in the protocol and was left to the Investigator's standard of care. **Measure:** Number of Patients With Delayed Wound Healing Through 12 Months Post-procedure **Time Frame:** 12 months **Description:** The SF-36v2 Health Survey asks 36 questions to measure health and well-being from the patient's point of view. The responses to these questions can be presented as physical component summary and mental component summary scores. An increase in score from baseline indicates an improvement in the patient's condition and a decrease in score from baseline indicates a decline in the patient's condition. The subscale and total score ranges from 0 to 100 but is normalized so that a score of 50 is the population mean, with a standard deviation of 10. **Measure:** Change in Quality of Life as Evaluated by the SF-36v2® Health Survey From Baseline Through One Month Post-procedure **Time Frame:** One month ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Patient requires below-knee bypass secondary to severe claudication, rest pain or tissue loss due to peripheral arterial occlusive disease. 2. Patient meets the disadvantaged autologous vein criteria described in Section 4.1. 3. Patient has a post-operative life expectancy greater than one year. 4. Patient is at least 21 years of age. 5. Patient is able to comply with all study requirements and be available for follow-up visits at 1, 6, 12, 24 and 36-months post-procedure. 6. Patient is willing and able to provide written, informed consent. Exclusion Criteria: 1. Patient has had a previous instance of Heparin Induced Thrombocytopenia type 2 (HIT-2) or has known hypersensitivity to heparin. 2. Patient requires an infragenicular bypass for reasons other than peripheral arterial occlusive disease. 3. Patient requires sequential extremity revascularizations or other procedures that require use of additional vascular grafts. 4. Patient is in need of, or is scheduled for, a cardiac surgical procedure or a different vascular surgical procedure within 30 days of study procedure. However, inflow procedures that facilitate the bypass are permitted, as long as they are not performed at the same sitting as the bypass procedure. 5. Patient has been previously randomized for this study. 6. Patient has active infection in the region of graft placement. **Minimum Age:** 21 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Birmingham **Country:** United States **Facility:** University Of Alabama Medical Center **State:** Alabama **Zip:** 35294 **Location 2:** **City:** Washington **Country:** United States **Facility:** Georgetown University Hospital **State:** District of Columbia **Zip:** 20007 **Location 3:** **City:** Atlanta **Country:** United States **Facility:** Emory University School of Medicine **State:** Georgia **Zip:** 30322 **Location 4:** **City:** Macon **Country:** United States **Facility:** Medical Center of Central Georgia **State:** Georgia **Zip:** 31201 **Location 5:** **City:** Chicago **Country:** United States **Facility:** University of Chicago **State:** Illinois **Zip:** 60637 **Location 6:** **City:** Baton Rouge **Country:** United States **Facility:** Vascular Specialty Associates **State:** Louisiana **Zip:** 70809 **Location 7:** **City:** Baltimore **Country:** United States **Facility:** University of Maryland Medical Center **State:** Maryland **Zip:** 21201 **Location 8:** **City:** Boston **Country:** United States **Facility:** Brigham and Women's Hospital **State:** Massachusetts **Zip:** 02115 **Location 9:** **City:** Albany **Country:** United States **Facility:** Albany Medical Center **State:** New York **Zip:** 12208 **Location 10:** **City:** Bronx **Country:** United States **Facility:** Montifiore Medical Center **State:** New York **Zip:** 10467 **Location 11:** **City:** Stony Brook **Country:** United States **Facility:** Stonybrook University Medical Center **State:** New York **Zip:** 11794 **Location 12:** **City:** Cleveland **Country:** United States **Facility:** Cleveland Clinic **State:** Ohio **Zip:** 44195 **Location 13:** **City:** Greenville **Country:** United States **Facility:** Greenville Hospital System **State:** South Carolina **Zip:** 29605 **Location 14:** **City:** Knoxville **Country:** United States **Facility:** University of Tennessee medical Center **State:** Tennessee **Zip:** 37920 **Location 15:** **City:** Houston **Country:** United States **Facility:** The Methodist Hospital System **State:** Texas **Zip:** 77030 **Location 16:** **City:** San Antonio **Country:** United States **Facility:** Peripheral Vascular Associates **State:** Texas **Zip:** 78205 **Location 17:** **City:** Norfolk **Country:** United States **Facility:** Sentara Norfolk Hospital **State:** Virginia **Zip:** 23507 #### Overall Officials **Official 1:** **Affiliation:** Georgetown University Hospital **Name:** Richard F. Neville, MD **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** W.L.Gore & Associates **Name:** Jennifer Recknor, Ph.D. **Role:** STUDY_DIRECTOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M4465 - Name: Arterial Occlusive Diseases - Relevance: HIGH - As Found: Arterial Occlusive Disease - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001157 - Term: Arterial Occlusive Diseases ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** GORE PROPATEN Vascular Graft **Description:** GORE PROPATEN Vascular Graft **ID:** EG000 **Other Num Affected:** 4 **Other Num at Risk:** 14 **Serious Number Affected:** 4 **Serious Number At Risk:** 14 **Title:** GORE PROPATEN Vascular Graft **Group ID:** EG001 **Title:** Disadvantaged Autologous Vein Graft **Description:** Disadvantaged Autologous Vein Graft **ID:** EG001 **Other Num Affected:** 3 **Other Num at Risk:** 17 **Serious Number Affected:** 2 **Serious Number At Risk:** 17 **Title:** Disadvantaged Autologous Vein Graft **Frequency Threshold:** 5 #### Other Events **Term:** Cellulitis of leg **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MEDra Version 13.1 **Term:** Claudication **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MEDra Version 13.1 **Term:** Edema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MEDra Version 13.1 **Term:** Incision site erythema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MEDra Version 13.1 **Term:** Incision site hematoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MEDra Version 13.1 **Term:** Intraoperative bleeding **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MEDra Version 13.1 **Term:** Myocardial infarction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MEDra Version 13.1 **Term:** Post procedural swelling **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MEDra Version 13.1 **Term:** Sepsis NOS **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MEDra Version 13.1 **Term:** Wound dehiscence **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MEDra Version 13.1 **Term:** Wound healing delayed **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MEDra Version 13.1 #### Serious Events **Term:** Below knee amputation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures **Source Vocabulary:** MedDRA Version 13.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 14 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 17 **Term:** Cerebral infarction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA Version 13.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 14 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 17 **Term:** Esophageal tear **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA Version 13.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 14 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 17 **Term:** Foot amputation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures **Source Vocabulary:** MedDRA Version 13.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 14 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 17 **Term:** Graft infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA Version 13.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 14 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 17 **Term:** Myocardial infarction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA Version 13.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 14 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 17 **Term:** Postoperative wound infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA Version 13.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 14 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 17 **Num Events:** 2 **Term:** Transmetatarsal amputation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures **Source Vocabulary:** MedDRA Version 13.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 14 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 17 **Num Events:** 1 **Term:** Vascular graft occlusion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA Version 13.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 14 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 17 ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 14 **Group ID:** BG001 **Value:** 17 **Group ID:** BG002 **Value:** 31 **Units:** Participants ### Group **ID:** BG000 **Title:** GORE PROPATEN Vascular Graft: **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. ### Group **ID:** BG001 **Title:** Disadvantaged Autologous Vein Graft **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 7 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 14 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 7 #### Measurement **Group ID:** BG001 **Value:** 10 #### Measurement **Group ID:** BG002 **Value:** 17 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 10 **Value:** 65 #### Measurement **Group ID:** BG001 **Spread:** 8 **Value:** 68 #### Measurement **Group ID:** BG002 **Spread:** 9 **Value:** 67 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 7 #### Measurement **Group ID:** BG001 **Value:** 5 #### Measurement **Group ID:** BG002 **Value:** 12 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 7 #### Measurement **Group ID:** BG001 **Value:** 12 #### Measurement **Group ID:** BG002 **Value:** 19 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 14 #### Measurement **Group ID:** BG001 **Value:** 17 #### Measurement **Group ID:** BG002 **Value:** 31 **Class Title:** United States **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Age, Categorical **Unit of Measure:** Participants ### Measure 2 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age Continuous **Unit of Measure:** years ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants ## Results Section - More Information Module ### Certain Agreement **Restriction Type:** LTE60 **Restrictive Agreement:** True ### Limitations and Caveats **Description:** The study was terminated prior to completion of enrollment. The primary patency and secondary endpoints were not calculated due to the paucity of data. ### Point of Contact **Email:** cbadorek@wlgore.com **Organization:** W. L. Gore & Associates, Inc. **Phone:** 928.864.3486 **Title:** Chad Badorek, Clinical Study Manager ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 ### Outcome Measure 7 ### Outcome Measure 8 ### Outcome Measure 9 ### Outcome Measure 10 ### Outcome Measure 11 ### Outcome Measure 12 ### Outcome Measure 13 ### Outcome Measure 14 ### Outcome Measure 15 ### Outcome Measure 16 ### Outcome Measure 17 ### Outcome Measure 18 ### Outcome Measure 19 ### Outcome Measure 20 ### Outcome Measure 21 ### Outcome Measure 22 ### Outcome Measure 23 ### Outcome Measure 24 ### Outcome Measure 25 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 8 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 8 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** #### Outcome Measure 8 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 8 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** #### Outcome Measure 9 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** #### Outcome Measure 10 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** #### Outcome Measure 11 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 13 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 17 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 12 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 13 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 17 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 13 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 13 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 17 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 14 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 11 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 15 **Title:** #### Outcome Measure 15 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 10 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 15 **Title:** #### Outcome Measure 16 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 14 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 17 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 17 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 13 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 16 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** #### Outcome Measure 18 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 13 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 15 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 **Title:** #### Outcome Measure 19 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 12 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 16 **Title:** #### Outcome Measure 20 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 12 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 16 **Title:** #### Outcome Measure 21 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 12 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 16 **Title:** #### Outcome Measure 22 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 14 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 16 **Title:** #### Outcome Measure 23 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 13 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 16 **Title:** #### Outcome Measure 24 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 13 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 16 **Title:** #### Outcome Measure 25 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 6.61 - **Upper Limit:** - **Value:** 2.9 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 8.77 - **Upper Limit:** - **Value:** 2.2 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 8.67 - **Upper Limit:** - **Value:** -3.6 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 10.44 - **Upper Limit:** - **Value:** 4.4 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Primary patency is defined as hemodynamic evidence of blood flow through an open graft that has maintained uninterrupted patency and has not previously undergone a revision to restore blood flow. **Parameter Type:** NUMBER **Population Description:** The number of patients analyzed at 12 months post-procedure does not equal the number of patients originally enrolled into the study. This is due to the fact that many enrolled patients did not have their 12 month follow-up visit prior to the termination of the study. **Reporting Status:** POSTED **Time Frame:** 12 months **Title:** Number of Patients With Primary Patency at 12 Months Post-procedure **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 2 **Description:** Primary patency is defined as hemodynamic evidence of blood flow through an open graft that has maintained uninterrupted patency and has not previously undergone a revision to restore blood flow. **Parameter Type:** NUMBER **Population Description:** The number of patients analyzed at 1 month post-procedure does not equal the number of patients originally enrolled into the study. This is due to the fact that a number of enrolled patients did not have their 1 month follow-up visit (or failed to attend their 1 month visit) prior to the termination of the study. **Reporting Status:** POSTED **Time Frame:** One month **Title:** Number of Patients With Primary Patency at One Month Post-procedure **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 3 **Description:** The number of Major Adverse Event occurrences through one month post-procedure. A major adverse event requires significant therapy, including unplanned increase in the level of care, permanent sequelae, hospitalization, or death. This outcome measure presents the number of Major Adverse Event occurrences (i.e. - one patient could have multiple occurrences), and differs from the Serious Adverse Events reporting measure, which presents the number of patients that have been affected by a Serious Adverse Event. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** one month post-index procedure **Title:** Major Adverse Event Occurrences Through One Month Post-procedure **Type:** PRIMARY **Unit of Measure:** Events ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 4 **Description:** Primary patency is defined as hemodynamic evidence of blood flow through an open graft that has maintained uninterrupted patency and has not previously undergone a revision to restore blood flow. **Parameter Type:** NUMBER **Population Description:** The number of patients analyzed at 6 months post-procedure does not equal the number of patients originally enrolled into the study. This is due to the fact that a number of enrolled patients did not have their 6 month follow-up visit (or failed to attend their 6 month visit) prior to the termination of the study. **Reporting Status:** POSTED **Time Frame:** 6 months **Title:** Number of Patients With Primary Patency at 6 Months Post-procedure **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 5 **Description:** Assisted primary patency is defined as hemodynamic evidence of blood flow through an open graft that has previously undergone revision(s) within the graft to restore blood flow prior to occlusion. The number of patients analyzed at 1 month post-procedure does not equal the number of patients originally enrolled into the study. This is due to the fact that a number of enrolled patients did not have their 1 month follow-up visit (or failed to attend their 1 month visit) prior to the termination of the study. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** One month **Title:** Number of Patients With Assisted Primary Patency at One Month Post-procedure **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 6 **Description:** Assisted primary patency is defined as hemodynamic evidence of blood flow through an open graft that has previously undergone revision(s) within the graft to restore blood flow prior to occlusion. **Parameter Type:** NUMBER **Population Description:** The number of patients analyzed at 6 months post-procedure does not equal the number of patients originally enrolled into the study. This is due to the fact that a number of enrolled patients did not have their 6 month follow-up visit (or failed to attend their 6 month visit) prior to the termination of the study. **Reporting Status:** POSTED **Time Frame:** 6 months **Title:** Number of Patients With Assisted Primary Patency at 6 Months Post-procedure **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 7 **Description:** Assisted primary patency is defined as hemodynamic evidence of blood flow through an open graft that has previously undergone revision(s) within the graft to restore blood flow prior to occlusion. **Parameter Type:** NUMBER **Population Description:** The number of patients analyzed at 12 months post-procedure does not equal the number of patients originally enrolled into the study. This is due to the fact that a number of enrolled patients did not have their 12 month follow-up visit (or failed to attend their 12 month visit) prior to the termination of the study. **Reporting Status:** POSTED **Time Frame:** 12 months **Title:** Number of Patients With Assisted Primary Patency at 12 Months Post-procedure **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 8 **Description:** Secondary patency is defined as hemodynamic evidence of blood flow through an open graft that has previously undergone revision(s) to restore blood flow after occlusion. **Parameter Type:** NUMBER **Population Description:** The number of patients analyzed at 1 month post-procedure does not equal the number of patients originally enrolled into the study. This is due to the fact that a number of enrolled patients did not have their 1 month follow-up visit (or failed to attend their 1 month visit) prior to the termination of the study. **Reporting Status:** POSTED **Time Frame:** One month **Title:** Number of Patients With Secondary Patency at One Month **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 9 **Description:** Secondary patency is defined as hemodynamic evidence of blood flow through an open graft that has previously undergone revision(s) to restore blood flow after occlusion. **Parameter Type:** NUMBER **Population Description:** The number of patients analyzed at 6 months post-procedure does not equal the number of patients originally enrolled into the study. This is due to the fact that a number of enrolled patients did not have their 6 month follow-up visit (or failed to attend their 6 month visit) prior to the termination of the study. **Reporting Status:** POSTED **Time Frame:** 6 months **Title:** Number of Patients With Secondary Patency at 6 Months **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 10 **Description:** Secondary patency is defined as hemodynamic evidence of blood flow through an open graft that has previously undergone revision(s) to restore blood flow after occlusion. **Parameter Type:** NUMBER **Population Description:** The number of patients analyzed at 12 months post-procedure does not equal the number of patients originally enrolled into the study. This is due to the fact that a number of enrolled patients did not have their 12 month follow-up visit (or failed to attend their 12 month visit) prior to the termination of the study. **Reporting Status:** POSTED **Time Frame:** 12 months **Title:** Number of Patients With Secondary Patency at 12 Months **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 11 **Description:** Limb salvage is defined as relief from symptoms sufficient to prevent major amputation. An amputation is considered to be major when there is surgical removal of a portion of the study leg that would preclude standing and walking without a prosthesis. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** One month **Title:** Number of Patients With Limb Salvage (no Major Amputations) at One Month Post-procedure **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 12 **Description:** Limb salvage is defined as relief from symptoms sufficient to prevent major amputation. An amputation is considered to be major when there is surgical removal of a portion of the study leg that would preclude standing and walking without a prosthesis. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 6 months **Title:** Number of Patients With Limb Salvage (no Major Amputations) at 6 Months Post-procedure **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 13 **Description:** Limb salvage is defined as relief from symptoms sufficient to prevent major amputation. An amputation is considered to be major when there is surgical removal of a portion of the study leg that would preclude standing and walking without a prosthesis. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 12 months **Title:** Number of Patients With Limb Salvage (no Major Amputations) at 12 Months Post-procedure **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 14 **Description:** A major adverse event requires significant therapy, including unplanned increase in the level of care, permanent sequelae, hospitalization, or death. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 6 months **Title:** Patients Experiencing Major Adverse Events Through 6 Months Post-procedure **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 15 **Description:** A major adverse event requires significant therapy, including unplanned increase in the level of care, permanent sequelae, hospitalization, or death. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 12 months **Title:** Patients Experiencing Major Adverse Events Through 12 Months Post-procedure **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 16 **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** One month **Title:** Number of Patients Surviving at One Month **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 17 **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 6 months **Title:** Number of Patients Surviving at 6 Months **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 18 **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 12 months **Title:** Number of Patients Surviving at 12 Months **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 19 **Description:** Wound/graft infection was not specifically defined in the protocol and was left to the Investigator's standard of care. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** One month **Title:** Number of Patients With Wound/Graft Infection Through One Month Post-procedure **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 20 **Description:** Wound/graft infection was not specifically defined in the protocol and was left to the Investigator's standard of care. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 6 months **Title:** Number of Patients With Wound/Graft Infection Through 6 Months **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 21 **Description:** Wound/graft infection was not specifically defined in the protocol and was left to the Investigator's standard of care. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 12 months **Title:** Number of Patients With Wound/Graft Infection Through 12 Months **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 22 **Description:** Delayed wound healing was not specifically defined in the protocol and was left to the Investigator's standard of care. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** One month **Title:** Number of Patients With Delayed Wound Healing Through One Month Post-procedure **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 23 **Description:** Delayed wound healing was not specifically defined in the protocol and was left to the Investigator's standard of care. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 6 months **Title:** Number of Patients With Delayed Wound Healing Through 6 Months Post-procedure **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 24 **Description:** Delayed wound healing was not specifically defined in the protocol and was left to the Investigator's standard of care. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 12 months **Title:** Number of Patients With Delayed Wound Healing Through 12 Months Post-procedure **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft #### Outcome Measure 25 **Description:** The SF-36v2 Health Survey asks 36 questions to measure health and well-being from the patient's point of view. The responses to these questions can be presented as physical component summary and mental component summary scores. An increase in score from baseline indicates an improvement in the patient's condition and a decrease in score from baseline indicates a decline in the patient's condition. The subscale and total score ranges from 0 to 100 but is normalized so that a score of 50 is the population mean, with a standard deviation of 10. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** The number of patients analyzed at 1 month does not equal the number of patients originally enrolled into the study, due to the fact that a number of enrolled patients did not have (or failed to attend) their 1 month follow-up visit prior to the termination of the study. **Reporting Status:** POSTED **Time Frame:** One month **Title:** Change in Quality of Life as Evaluated by the SF-36v2® Health Survey From Baseline Through One Month Post-procedure **Type:** SECONDARY **Unit of Measure:** scores on a scale ##### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** OG000 **Title:** GORE PROPATEN Vascular Graft: ##### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** OG001 **Title:** Disadvantaged Autologous Vein Graft ### Participant Flow Module #### Group **Description:** Patients receiving the GORE PROPATEN Vascular Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. **ID:** FG000 **Title:** GORE PROPATEN Vascular Graft: #### Group **Description:** Patients receiving Disadvantaged Autologous Vein Graft in a below-knee bypass due to severe claudication (pain, tension, and weakness in the legs), rest pain or tissue loss due to peripheral arterial occlusive disease. 'Disadvantaged Autologous Vein Graft' was defined in the study as meeting one of two criteria: (1) Any autologous vein(s) other than greater saphenous vein deemed usable by the Investigator (and/or) (2) Usable ipsilateral or contralateral autologous greater saphenous vein that is either less than or equal to 3.0 mm in diameter, of inadequate length (requires vein splicing), or of poor quality vein (sclerotic or phlebitic). **ID:** FG001 **Title:** Disadvantaged Autologous Vein Graft #### Period **Title:** Overall Study ##### Withdraw **Type:** Study terminated by Sponsor ###### Reason **Group ID:** FG000 **Number of Subjects:** 10 ###### Reason **Group ID:** FG001 **Number of Subjects:** 15 ##### Withdraw **Type:** Graft abandonment ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ##### Withdraw **Type:** Death ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 2 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 14 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 17 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 14 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 17 **Recruitment Details:** Patients requiring below-knee bypass secondary to severe claudication, rest pain or tissue loss due to peripheral arterial occlusive disease were recruited. Recruitment commenced December 2007 and was terminated by the Sponsor January 2010 due to slow enrollment. Investigative site locations included university hospitals and other medical clinics. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT00277979 **Brief Title:** 3D Echocardiography Managing Infantile Pompe's Disease **Official Title:** Utilization of 3D Echocardiography in the Management of Infantile Pompe's Disease: Two Case Reports #### Organization Study ID Info **ID:** 05-058 #### Organization **Class:** OTHER **Full Name:** Children's Healthcare of Atlanta ### Status Module #### Completion Date **Date:** 2007-02 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2012-03-16 **Type:** ESTIMATED **Last Update Submit Date:** 2012-03-14 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2007-02 **Type:** ACTUAL #### Start Date **Date:** 2005-01 **Status Verified Date:** 2007-12 #### Study First Post Date **Date:** 2006-01-18 **Type:** ESTIMATED **Study First Submit Date:** 2006-01-13 **Study First Submit QC Date:** 2006-01-13 **Why Stopped:** project withdrawn ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Children's Healthcare of Atlanta #### Responsible Party **Old Name Title:** Chairman **Old Organization:** Children's Healthcare of Atlanta Institutional Review Board ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Pompe's disease, also known as glycogen storage disease type II, is a genetic disorder due to deficiency of acid glucosidase (GAA), which results in lysosomal glycogen storage in various tissues. Very low levels of GAA usually present in infancy, lead to a progressive cardiac and skeletal muscle disorder and death before age 1 year. Most infants develop massive hypertrophic cardiomyopathy which progresses to dilated cardiomyopathy and cardiorespiratory arrest. 3D echocardiography can be a simple, non-invasive method of following cardiac disease progression in infantile Pompe's disease. **Detailed Description:** We report the utilization of real-time 3D echocardiography and new in-line software to calculate cardiac mass, volume and function in two cases of infantile Pompe's Disease. Furthermore, the multi-dimension cine loop showed the severity of cavity obliteration during the cardiac cycle. We present 2 cases seen at our institution between January - February, 2005 in infants ages 1 and 5 months, both with cardiac signs as initial presentation. The younger infant had cardiomegaly since birth and was hospitalized briefly for respiratory distress at 1 month. The older infant, diagnosed at 4 months with hypertrophic cardiomyopathy, was hospitalized after cardiac arrest one month later. Both children had various tests done during the course of diagnosis and treatment, including GAA enzyme assay, cardiac catheterization, endomyocardial biopsy, developmental assessment, genetic evaluation, EKG, CXR and echocardiography. ### Conditions Module **Conditions:** - Congenital Disorders **Keywords:** - pediatric - cardiac - Infantile Pompe's Disease - 3D Echocardiography ### Design Module #### Design Info **Observational Model:** CASE_ONLY **Time Perspective:** RETROSPECTIVE #### Enrollment Info **Count:** 2 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Infant seen at Children's Healthcare of Atlanta, Egleston January - February, 2005 Infantile Pompe's Disease Exclusion Criteria: * Those who do not meet inclusion criteria **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT **Study Population:** Subject with Infantile Pompe's Disease seen at Children's between Jan. and February, 2005. ### Contacts Locations Module #### Overall Officials **Official 1:** **Affiliation:** Centers for Disease Control and Prevention **Name:** Tiffany J Riehle, MD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020140 - Term: Lysosomal Storage Diseases, Nervous System - ID: D000020739 - Term: Brain Diseases, Metabolic, Inborn - ID: D000001928 - Term: Brain Diseases, Metabolic - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000008661 - Term: Metabolism, Inborn Errors - ID: D000030342 - Term: Genetic Diseases, Inborn - ID: D000006008 - Term: Glycogen Storage Disease - ID: D000002239 - Term: Carbohydrate Metabolism, Inborn Errors - ID: D000016464 - Term: Lysosomal Storage Diseases - ID: D000008659 - Term: Metabolic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M9115 - Name: Glycogen Storage Disease Type II - Relevance: HIGH - As Found: Pompe Disease - ID: M9114 - Name: Glycogen Storage Disease - Relevance: LOW - As Found: Unknown - ID: M18871 - Name: Lysosomal Storage Diseases - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5205 - Name: Brain Diseases, Metabolic - Relevance: LOW - As Found: Unknown - ID: M22498 - Name: Brain Diseases, Metabolic, Inborn - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M11641 - Name: Metabolism, Inborn Errors - Relevance: LOW - As Found: Unknown - ID: M23686 - Name: Genetic Diseases, Inborn - Relevance: LOW - As Found: Unknown - ID: M5498 - Name: Carbohydrate Metabolism, Inborn Errors - Relevance: LOW - As Found: Unknown - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: T2562 - Name: Glycogen Storage Disease Type 2 - Relevance: HIGH - As Found: Pompe Disease ### Condition Browse Module - Meshes - ID: D000006009 - Term: Glycogen Storage Disease Type II ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03158779 **Brief Title:** Evaluation of SBRT for Patients With Locally Advanced Unresectable Pancreatic Cancer **Official Title:** Phase II Trial Evaluating Stereotactic Body Radiation Therapy (SBRT) After Induction Chemotherapy for Patients With Locally Advanced Unresectable Pancreatic Cancer #### Organization Study ID Info **ID:** 1736 #### Organization **Class:** OTHER **Full Name:** Istituto Clinico Humanitas ### Status Module #### Completion Date **Date:** 2022-07-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-09-15 **Type:** ACTUAL **Last Update Submit Date:** 2022-09-14 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-07-01 **Type:** ACTUAL #### Start Date **Date:** 2017-05-10 **Type:** ACTUAL **Status Verified Date:** 2022-09 #### Study First Post Date **Date:** 2017-05-18 **Type:** ACTUAL **Study First Submit Date:** 2017-05-04 **Study First Submit QC Date:** 2017-05-16 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Istituto Clinico Humanitas #### Responsible Party **Investigator Affiliation:** Istituto Clinico Humanitas **Investigator Full Name:** Michele Tedeschi **Investigator Title:** MD **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** The aim of this phase II study is to assess the efficacy and safety of sequentially integrated treatment of FOLFIRINOX or Gemcitabine-Abraxane and SBRT in patients with unresectable pancreatic cancer. **Detailed Description:** The aim of this prospective mono-institutional phase II study is to assess the efficacy and safety of sequentially integrated treatment of FOLFIRINOX or Gemcitabine-Abraxane and SBRT with a total dose of 54 Gy in 6 fractions of 9 Gy /fractions in patients with locally unresectable pancreatic cancer. Primary endpoint is to evaluate overall survival (OS); the overall survival time will be calculated from the start of chemotherapy to death. Secondary end points are to evaluate acute and late toxicities, freedom from local progression (FFLP) and progression free-survival (PFS). Acute and late toxicities will be scored according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) v3.0. Local progression will be defined according to RECIST criteria. Time to toxicity and time to local or distant progression will be defined from the start of chemotherapy. Technical success will be defined as the ability to implant at least 2 fiducials in the tumor area. Migration will be defined as a change in inter-fiducial distance. Clinical success will be defined as the ability to guide the application of SBRT by using the fiducials. Any adverse event will be recorded (acute pancreatitis, clinically relevant upper GI bleeding requiring blood transfusion, abscesses in the area of the fiducials, sepsis). ### Conditions Module **Conditions:** - Pancreatic Cancer ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 45 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants received 4 months of FOLFIRINOX or Gemcitabine-Abraxane before SBRT was administered. A 3-weeks break from chemotherapy and restaging with thorax-abdominal CT scan to confirm the absence of distant metastases was required before SBRT delivery. Before SBRT simulation, patients may will have implanted fiducial into the pancreatic tumor. The SBRT schedule will be \[6 x 9 Gy = 54 Gy\] delivered in consecutive days. **Intervention Names:** - Combination Product: SBRT and chemotherapy **Label:** SBRT and chemotherapy **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - SBRT and chemotherapy **Description:** Patients affected by locally unresectable pancreatic cancer receive integrated treatment of FOLFIRINOX or Gemcitabine-Abraxane and a Stereotactic Body Radiation Therapy with a total dose of 54 Gy in 6 fractions of 9 Gy /fractions. **Name:** SBRT and chemotherapy **Type:** COMBINATION_PRODUCT ### Outcomes Module #### Primary Outcomes **Description:** Evaluation of SBRT after induction CT with FOLFIRINOX or Gemcitabine-Abraxane in terms of overall survival time that will be calculated from the start of chemotherapy to death. **Measure:** Overall survival **Time Frame:** 2 years #### Secondary Outcomes **Description:** Evaluation of early post treatment complications according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) v3.0. **Measure:** Incidence of acute toxicities **Time Frame:** 2 years **Description:** Evaluation of late post treatment complications according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) v3.0. **Measure:** Incidence of late toxicities **Time Frame:** 4 years **Description:** Evaluation of proportion of patients free from local progression according to RECIST criteria **Measure:** Freedom from local progression in treated patients **Time Frame:** 2 years **Description:** Evaluation of proportions of patients alive and free form progression according to RECIST criteria **Measure:** Progression free-survival of treated patients **Time Frame:** 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Histologically and/or radiologically (CT scan and/or MRI scan and/or FDG-PET) proven unresectable locally advanced pancreatic cancer, discussed multidisciplinary by tumor board. * Eighteen years of age or older, * Minimum Karnofsky Performance Status of 70. * Lesions cannot exceed 5 cm in maximum diameter. * Absence of lymph-nodal metastases * Patients received 4-6 months of FOLFIRINOX or Gemcitabine-Abraxane before SBRT was administered. * Baseline total body CT scan performed no more than 2 months before treatment. * Placing fiducial markers through endoscopes is permitted (EUS-guided fiducials placement before the treatment). * Acceptable organ and bone marrow function. * Ability to maintain the set-up position during RT. * All patients give informed consent and sign a study-specific informed consent form. Exclusion Criteria: * Metastatic disease * Prior abdominal radiotherapy * Other malignancies diagnosed within 5 years * Gastric or duodenal obstruction. * Concurrent chemotherapy. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Rozzano **Country:** Italy **Facility:** Humanitas Research Hospital **State:** Milan **Zip:** 20089 #### Overall Officials **Official 1:** **Affiliation:** Istituto Clinico Humanitas **Name:** Marta Scorsetti, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004067 - Term: Digestive System Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000004701 - Term: Endocrine Gland Neoplasms - ID: D000004066 - Term: Digestive System Diseases - ID: D000010182 - Term: Pancreatic Diseases - ID: D000004700 - Term: Endocrine System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M13110 - Name: Pancreatic Neoplasms - Relevance: HIGH - As Found: Pancreatic Cancer - ID: M8886 - Name: Gastrointestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7256 - Name: Digestive System Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7863 - Name: Endocrine Gland Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M13102 - Name: Pancreatic Diseases - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown - ID: T4387 - Name: Pancreatic Cancer - Relevance: HIGH - As Found: Pancreatic Cancer ### Condition Browse Module - Meshes - ID: D000010190 - Term: Pancreatic Neoplasms ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M19537 - Name: Paclitaxel - Relevance: LOW - As Found: Unknown - ID: M231 - Name: Albumin-Bound Paclitaxel - Relevance: LOW - As Found: Unknown - ID: M2985 - Name: Gemcitabine - Relevance: LOW - As Found: Unknown - ID: M271136 - Name: Folfirinox - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03572179 **Brief Title:** Evaluation of Fixed Mandibular Retainer Using 3D Printed Positioning Tray Versus Direct Bonding Technique **Official Title:** Evaluation of Fixed Mandibular Retainer Using 3D Printed Positioning Tray Versus Direct Bonding Technique A Randomized Clinical Trial #### Organization Study ID Info **ID:** CEBD-CU-2018-04-28 #### Organization **Class:** OTHER **Full Name:** Cairo University ### Status Module #### Completion Date **Date:** 2020-03-28 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-09-05 **Type:** ACTUAL **Last Update Submit Date:** 2021-08-31 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-03-01 **Type:** ACTUAL #### Start Date **Date:** 2019-03-01 **Type:** ACTUAL **Status Verified Date:** 2021-08 #### Study First Post Date **Date:** 2018-06-28 **Type:** ACTUAL **Study First Submit Date:** 2018-06-09 **Study First Submit QC Date:** 2018-06-26 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Raghda Alaa #### Responsible Party **Investigator Affiliation:** Cairo University **Investigator Full Name:** Raghda Alaa **Investigator Title:** principal investigator **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Although there are many studies in the literature concerning methods of bonding of mandibular retainers whether directly or indirectly, there is a very limited evidence regarding their chair side time and bond failure. Also there are scarce data on fixed mandibular retainers bonded using 3D printed positioning tray.Thus, conducting a well-designed randomized clinical trial evaluating the chair side time and bond failure of 3d printed positioning tray and direct bonding method of fixed mandibular retainer.Null hypothesis is thatThe time needed for direct indirect bonding using 3D digital positioning tray of a mandibular fixed retainer is shorter than the time needed for direct bonding, and there is no difference in failures between the 2 bonding methods **Detailed Description:** Statement of the problem: - Although there are many studies in the literature concerning methods of bonding of mandibular retainers whether directly or indirectly, there is a very limited evidence regarding their chair side time and bond failure. Also there are scarce data on fixed mandibular retainers bonded using 3D printed positioning tray. Choice of the comparator: In the ongoing study, direct bonding of mandibular fixed retainer will be the control group. Thus, conducting a well-designed randomized clinical trial evaluating the chair side time and bond failure of 3d printed positioning tray and direct bonding method of fixed mandibular retainer. Study rationale: To evaluate and compare bond failure and chair side time by 3D printed positioning tray and direct bonding method of mandibular fixed retainer. Null hypothesis: 1. The time needed for direct indirect bonding using 3D digital positioning tray of a mandibular fixed retainer is shorter than the time needed for direct bonding, and (2) there is no difference in failures between the 2 bonding methods. Research question: Will the 3D digital positioning tray reduce chair side time, bond failure during retainer placement in patient with need of fixed bonded mandibular retainer, when compared to direct bonding method? Aim of the study: To evaluate chair side time and bond failure of 3D digital positioning tray during mandibular retainer placement and direct bonding of mandibular retainer. 2. Objectives Primary objective To evaluate effect of 3D Digital positioning Tray on chair side time Secondary outcome Immediate bond failure and bond failure during first 6 month of placement of mandibular fixed retainer. PICOTs format: P: patient with the need of fixed bonded mandibular retainer after orthodontic treatment I: 3D printed Digital positioning Tray for bonding mandibular retainer. C: conventional direct bonding of mandibular retainer O: chair side time, immediate bond failure and bond failure during first 6 month. Outcome Name,Measuring Tool and Measuring unit Primary is the Chair side Time measured by Digital stopwatch in Seconds Immediate Bond Failure is the Number of Debonded mandibular fixed retainer measured in Numerical value Bond failure during first 6 month follow up by the Number of Debonded mandibular fixed retainer measured in Numerical value T: Immediate and T2: 6 month follow up S: Randomized controlled trial. 3. Trial Design This trial is designed as a parallel, randomized, controlled trial with allocation ratio 1:1.It will be performed at the educational hospital of the Faculty of Oral \& Dental Medicine, Cairo University. Materials \& Methods: I. Participants, Intervention and Outcomes A) Study setting: * Source of patients: patients who finished their orthodontic treatment at the outpatient clinic in Orthodontic department, Faculty of Oral and Dental Medicine, Cairo University, Cairo government, Egypt. * Time: 2017-2018 The study will last for 6 months. C) Intervention Treatment Group After finishing orthodontic treatment, all patient will receive a modified indirect technique for bonding fixed mandibular retainer and the key of modification is the fabrication of 3D printed digital positioning tray for placement of retainer with holes for composite pads for its direct application using 3 shape Ortho analyser Software ® instead of conventional indirect retainer fabrication method The sample selection and screening for inclusion of patients in clinical trial will be performed at all patients at clinic of Orthodontic department, Faculty of Oral and Dental Medicine, Cairo University. Scanning of impression and designing the digital tray Lower impression is taken for each patient and the cast will be carefully scanned by desktop laser scanner for production of digital cast, using 3 shape ortho analyser Software®, the tray will be designed Fabrication and printing of the digital tray Using Dent1 3D printer® (Mogassam®, Greek campus, Cairo, Egypt), the tray will be printed Clinical application of the digital tray * Teeth to be bonded are polished and etched. * Teeth isolation \& moisture control are achieved. * Dead Soft Stainless Steel wire is placed and adapted in its place in the digital tray. * Placement of the tray in the patient mouth on the prepared teeth * Adhesive bond is applied to teeth and then curing of the adhesive bonding. * 3M ® composite (3M Unitek company , Monrovia, California, USA) is placed in the printed tray holes and any excess composite will be removed and then light curing is done. * Time will be recorded during this step Removal of digital tray After insuring complete curing of the composite the digital tray is then removed from patient mouth, Bond failure of composite pads will be checked in this step and recorded. Control Group All patients of this group will follow conventional steps of direct bonding procedure of fixed mandibular retainer * Pumice and polish the lingual surface of teeth on which retainer is to be bonded * The retainer wire is prefabricated by incorporating perpendicular pieces of 1.5"-2" ligature wire at interdental region of respective teeth * The retainer wire is then adapted on the tooth surface. The short ligatures are passed interdentally one occlusal to contact point and other gingival to contact point and tied over. * Bonding procedure after etching and application of sealant is carried out. * Enough adhesive should be used. Light curing is done. * 3M ® composite (3M Unitek company, Monrovia, California, USA) is placed then light curing is done. * Cut the ligature wires and pull them laically to disengage. * Time will be recorded during this step F) Sample Size calculation: the sample is increased to a total size of 20 participants; 10 in each arm. G) Recruitment strategy: * Patients will be selected from the outpatient clinic of the orthodontic Department - Cairo University * Consecutive sampling is done through screening of patients. This will continue until the target population is achieved. * Identifying and recruiting potential subjects is achieved through patient database. II. Assignment of Interventions A) Sequence generation: It will be performed as 1:1 allocation. The sequence of the two groups will be done by computer generated random numbers. This will be done by using Microsoft Office Excel 2013 sheet B) Allocation Concealment: Random numbers obtained by random sequence generation will be written on white papers, each paper will be kept in sealed envelope. Sealed envelopes will be kept in box at the secretary of orthodontic department office. C) Implementation: Implementation will be carried out in the secretary of the orthodontic department at the Faculty of Oral and Dental Medicine, Cairo University. D) Blinding: Participants Patients can detect the transfer tray so blinding is not possible Operator The main operator is responsible for the bonding so the blinding is not possible Assessor The assessor (different from main operator will carry out the measurement blindly ### Conditions Module **Conditions:** - Orthodontic Appliance Complication **Keywords:** - 3d printed positioning tray ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** all patient will receive a modified indirect technique for bonding fixed mandibular retainer and the key of modification is the fabrication of 3D printed digital positioning tray for placement of retainer with holes for composite pads for its direct application using 3 shape Ortho analyser Software ® instead of conventional indirect retainer fabrication method ##### Masking Info **Masking:** SINGLE **Masking Description:** Participants Patients can detect the transfer tray so blinding is not possible Operator The main operator is responsible for the bonding so the blinding is not possible Assessor The assessor (different from main operator will carry out the measurement blindly **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** OTHER #### Enrollment Info **Count:** 20 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** After finishing orthodontic treatment, all patient will receive a modified indirect technique for bonding fixed mandibular retainer and the key of modification is the fabrication of 3D printed digital positioning tray for placement of retainer with holes for composite pads for its direct application using 3 shape Ortho analyser Software ® instead of conventional indirect retainer fabrication method **Intervention Names:** - Device: 3d printed positioning tray **Label:** Treatment Group **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** All patients of this group will follow conventional steps of direct bonding procedure of fixed mandibular retainer **Label:** Control Group **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Treatment Group **Description:** 3d printed positioning tray for lingual fixed retainer placement in treatment group **Name:** 3d printed positioning tray **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** chairside time for bonding fixed lingual mandibular retainer using stopwatch in measured seconds **Measure:** chairside time **Time Frame:** follow up up to 6 month #### Secondary Outcomes **Description:** bond failure of composite per tooth measuered in numerical value **Measure:** bond failure **Time Frame:** 6 month follow up ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * • Patients with properly finished orthodontic treatment according to ABO standards. * the presence of the 4 mandibular incisors and the 2 mandibular canines, * No active caries, restorations, fractures, or periodontal disease of these teeth * Patient with Good oral hygiene Exclusion Criteria: * • Patients with no need of fixed bonded mandibular retainer * No or poor patient's co operation * Abnormal morphology of anterior teeth * Bad oral hygiene **Healthy Volunteers:** True **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Cairo **Country:** Egypt **Facility:** Cairo University Faculty of Oral and Dental Medicine **Zip:** 0202 ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04808479 **Acronym:** UCimFREE **Brief Title:** Effectiveness of a CBT-based mHealth Intervention Targeting MOUD Retention, Adherence, and Opioid Use **Official Title:** FOA Title: HEAL Initiative: Behavioral Research to Improve MAT: Behavioral and Social Interventions to Improve Adherence to Medication-Assisted Treatment for Opioid Use Disorders (R61/R33 Clinical Trial Optional #### Organization Study ID Info **ID:** UC imFREE phase1&RCT #### Organization **Class:** OTHER **Full Name:** University of California, Los Angeles ### Status Module #### Completion Date **Date:** 2026-08-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-05-14 **Type:** ACTUAL **Last Update Submit Date:** 2024-05-13 **Overall Status:** ENROLLING_BY_INVITATION #### Primary Completion Date **Date:** 2025-08-01 **Type:** ESTIMATED #### Start Date **Date:** 2019-12-10 **Type:** ACTUAL **Status Verified Date:** 2024-05 #### Study First Post Date **Date:** 2021-03-22 **Type:** ACTUAL **Study First Submit Date:** 2021-02-08 **Study First Submit QC Date:** 2021-03-17 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** University of California, San Diego #### Lead Sponsor **Class:** OTHER **Name:** University of California, Los Angeles #### Responsible Party **Investigator Affiliation:** University of California, Los Angeles **Investigator Full Name:** Suzette Glasner-Edwards **Investigator Title:** Principal Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** The focus of this study is to examine the effectiveness of imFREE relative to mHealth ED in facilitating treatment retention and adherence and reducing opioid use among adults with OUD initiating BUP treatment. There are two specific aims: (1) to test the effectiveness of imFREE, delivered in conjunction with medical management with buprenorphine (imFREE + MM), relative to mHealth ED + MM, in facilitating buprenorphine treatment retention and adherence in a population of individuals with OUD initiating MM (N=200). (2) To evaluate the cost-effectiveness of imFREE. ### Conditions Module **Conditions:** - Opioid-use Disorder ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 200 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The imFREE condition is a 32-week tailored, interactive text messaging intervention targeting buprenorphine treatment retention and adherence as well as opioid use and associated health consequences. Following a face-to-face CBT session with a clinician, participants receive CBT skills training via daily text messaging, with content themes around relapse prevention, adherence behaviors, and personalized plans to overcome risk factors for treatment discontinuation. **Intervention Names:** - Behavioral: imFREE mCBT **Label:** UC imFREE Smartphone application intervention **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** The mHealth condition will provide participants with a manualized health psychoeducation session of equivalent duration to the face-to-face CBT session delivered to imFREE participants. Content focuses on various dimensions of health and well-being. Subsequent to this session participants will receive an informational pamphlet regarding BUP and the importance of adherence. the mHealth component of the intervention comprises text reminders for scheduled MM appointments. **Intervention Names:** - Behavioral: mHealth ED **Label:** Health Education and pamphlet **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - UC imFREE Smartphone application intervention **Description:** The imFREE condition is a 32-week tailored, interactive text messaging intervention targeting buprenorphine treatment retention and adherence as well as opioid use and associated health consequences. Following a face-to-face CBT session with a clinician, participants receive CBT skills training via daily text messaging, with content themes around relapse prevention, adherence behaviors, and personalized plans to overcome risk factors for treatment discontinuation. **Name:** imFREE mCBT **Other Names:** - imFREE **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - Health Education and pamphlet **Description:** The mHealth condition will provide participants with a manualized health psychoeducation session of equivalent duration to the face-to-face CBT session delivered to imFREE participants. Content focuses on various dimensions of health and well-being. Subsequent to this session participants will receive an informational pamphlet regarding BUP and the importance of adherence. the mHealth component of the intervention comprises text reminders for scheduled MM appointments. **Name:** mHealth ED **Other Names:** - Manualized Health Psychoeducation **Type:** BEHAVIORAL ### Outcomes Module #### Other Outcomes **Description:** The Addiction Severity Index is a relatively brief, semi-structured interview designed to provide important information about aspects of a client's life that may contribute to his/her substance abuse syndrome.Assessment of addiction severity and frequency of alcohol/drug use. **Measure:** European Addiction Severity Index **Time Frame:** Baseline, month 3, month 6 **Description:** Confirming absence/presence of opiates and buprenorphine **Measure:** Urine drug screen **Time Frame:** Throughout six months, monthly **Description:** Retention in buprenorphine treatment my reviewing number of MM sessions attended **Measure:** Medical Management (MM) attendance **Time Frame:** Throughout six months, monthly #### Primary Outcomes **Description:** Self-report of Treatment adherence to buprenorphine collected in self-report forms. Unannounced medication count by phone. Research staff will call participants and ask them to count out the remaining buprenorphine films left in their prescription. **Measure:** Medication Adherence self-report form. Unannounced medication count by phone **Time Frame:** Monthly from date of randomization, for up to 6 months, or until study completion, whichever comes first. #### Secondary Outcomes **Description:** Assessing the presence of opiate use disorder **Measure:** DSM-V Checklist **Time Frame:** Baseline ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age \> 18 * DSM-5 diagnosis of OUD * no more than 2 weeks from the date of BUP induction * able to read and comprehend English at the 6th grade level (determined by REALM) * able to provide informed consent * presently owns a mobile phone that can send and receive text messages EXCLUSION CRITERIA Participants must not have: 1. Life threatening or unstable medical illness requiring treatment or making participation difficult 2. Dependence on alcohol or other illicit substances for which medical detoxification is imminently needed 3. Presence of acute psychiatric symptoms warranting intensive treatment or hospitalization (e.g., acute suicidality or mania). **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Tarzana **Country:** United States **Facility:** Tarzana Treatment Center **State:** California **Zip:** 91356 #### Overall Officials **Official 1:** **Affiliation:** University of California, Los Angeles **Name:** Suzette Glasner, PhD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000079524 - Term: Narcotic-Related Disorders - ID: D000019966 - Term: Substance-Related Disorders - ID: D000064419 - Term: Chemically-Induced Disorders - ID: D000001523 - Term: Mental Disorders ### Condition Browse Module - Browse Branches - Abbrev: BC25 - Name: Substance Related Disorders - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M12244 - Name: Opioid-Related Disorders - Relevance: HIGH - As Found: Opioid Use Disorder - ID: M2057 - Name: Narcotic-Related Disorders - Relevance: LOW - As Found: Unknown - ID: M21837 - Name: Substance-Related Disorders - Relevance: LOW - As Found: Unknown - ID: M30302 - Name: Chemically-Induced Disorders - Relevance: LOW - As Found: Unknown - ID: M4815 - Name: Mental Disorders - Relevance: LOW - As Found: Unknown - ID: M14473 - Name: Psychotic Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009293 - Term: Opioid-Related Disorders ### Intervention Browse Module - Browse Branches - Abbrev: Analg - Name: Analgesics - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: NarcAntag - Name: Narcotic Antagonists - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M5317 - Name: Buprenorphine - Relevance: LOW - As Found: Unknown - ID: M4033 - Name: Analgesics, Opioid - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00781079 **Acronym:** PEER **Brief Title:** Do Consumer Providers Enhance Recovery? **Official Title:** Promoting Recovery Using Mental Health Consumer Providers #### Organization Study ID Info **ID:** IIR 06-227 #### Organization **Class:** FED **Full Name:** VA Office of Research and Development ### Status Module #### Completion Date **Date:** 2012-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-10-11 **Type:** ACTUAL **Last Update Submit Date:** 2018-09-12 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2011-04 **Type:** ACTUAL #### Results First Post Date **Date:** 2014-10-06 **Type:** ESTIMATED **Results First Submit Date:** 2014-09-18 **Results First Submit QC Date:** 2014-10-03 #### Start Date **Date:** 2008-04 **Status Verified Date:** 2018-09 #### Study First Post Date **Date:** 2008-10-28 **Type:** ESTIMATED **Study First Submit Date:** 2008-10-27 **Study First Submit QC Date:** 2008-10-27 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** FED **Name:** VA Office of Research and Development #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Serious mental illness (SMI) is the second most costly disorder treated in the VHA, yet clinical outcomes for these patients in public sector settings are often poor due to a combination of low quality care and severe cognitive and functional impairments evidenced by this group. While these problems are multifaceted, studies outside the VHA have shown that using "consumer providers" (CPs) can improve and augment public care. Similar to recovering addiction counselors, CPs are individuals with SMI who use their lived experiences to provide services to others with SMI. CPs can reach out to patients that are difficult to engage, assist patients with tasks of daily living, offer a variety of rehabilitation (vocational, social, residential) services, be role models and offer hope for recovery, and facilitate support groups. Randomized controlled and quasi-experimental trials, all done outside the VHA, have shown that CPs can provide services that yield at least equivalent patient outcomes with particular benefits noted on intensive case management teams. Based on these successes both the President's New Freedom Commission and the Veteran Administration's Mental Health Strategic Plan call for broader dissemination of CPs as way to make mental health services more recovery-oriented, a recent national priority. Because of these recent calls, employing mentally ill veterans has just begun, although no effort has been made to evaluate their impact inside the VA mental health system. Yet its success outside the VHA and the recent emphasis on recovery-oriented care suggests the need to test this model in the VHA. **Detailed Description:** Background/Rationale Serious mental illness (SMI) is the second most costly disorder treated in the VHA, yet clinical outcomes for these patients are often poor due to a combination of low quality care and severe cognitive and functional impairments. While these problems are multifaceted, studies outside the VHA have shown that using "consumer providers" (CPs) can improve and augment care. Similar to recovering addiction counselors, CPs are individuals with SMI who use their lived experiences to provide services to others with SMI. CPs can reach out to patients that are difficult to engage, assist patients with tasks of daily living, offer a variety of rehabilitation (vocational, social, residential) services, be role models and offer hope for recovery, and facilitate support groups. Randomized controlled and quasi-experimental trials outside the VHA have shown that CPs can provide services that yield at least equivalent patient outcomes with particular benefits noted on intensive case management teams. VHA has hired about 250 CPs to date, although their impact has not been documented. Yet its success outside the VHA and the recent emphasis on recovery-oriented care suggests the need to test this model in the VHA. Objectives To conduct a randomized controlled trial testing the impact on patient level and team level outcomes of the implementation of CP services on six mental health intensive case management (MHICM) teams in VISN-22. The specific aims were to: 1) Evaluate the acceptability, facilitators of and barriers to the inclusion of two CPs to each intervention MHICM team. 2) Evaluate the effect of including CPs on the degree to which MHICM teams services are recovery-oriented. 3) Evaluate the effect of including CPs on veterans' clinical and recovery-focused outcomes. Our hypotheses were that CPs would be feasible and acceptable, that teams would become more recovery-oriented, and that the involvement of CPs would lead to greater gains in recovery, quality of life, empowerment with regard to illness, and to a lesser extent, symptoms compared to patients on teams without CPs. Methods This project was a "cluster randomized controlled trial" comparing 3 CP-MHICM teams (with CPs) to 3 control teams (i.e., without a CP), at MHICM sites within VISN-22. All patients on the 6 MHICM teams' caseload during each site's recruitment period were eligible. 282 MHICM patients were enrolled, 149 at the intervention sites and 133 at the control sites. Each intervention MHICM team used a strategic planning process to tailor the CP intervention to local priorities and structures, involving multiple meetings to discuss the CP's role, hiring, and incorporation of CPs into the team. The project hired the CPs, provided training, assisted in their implementation on the teams and provided ongoing supervision. The CPs worked for about 12 months. Using a patient survey, the study assessed impacts of CPs on patient level outcomes including recovery-orientation of their team (Recovery Self-assessment scale), individual recovery (Mental Health Recovery Measure, Illness Self-Management Scale), quality of life (Quality of Life Interview), symptoms (BASIS-24), and patient activation (Patient Activation Measure). The patient level outcome assessments were conducted pre and post intervention. Final follow-up assessment rates ranged from 71- 95% at intervention sites and 80 - 88% at control sites. We conducted site visits with all six MHICM teams using a validated, standardized protocol, rating each team's level of recovery orientation before and after the deployment of the CPs on the Recovery-Oriented Practices Index (ROPI). We conducted 23 focus groups and interviews with patients, providers and CPs at all intervention sites (about 8 per site) at the post time point to assess barriers and facilitators to CP implementation. All focus groups and interviews were recorded, transcribed, and coded using Atlas.ti. The analyses of the patient survey data were comparisons of changes between baseline and followup scores with regression analyses of the change scores for all the outcomes mentioned above. The first analyses was whether the intervention group was significantly related to change considering only treatment group and statistically controlling for baseline score. The second was the same as the first, adding demographics (age, gender, race, living situation and education level) and site as fixed covariates. The ROPI ratings were made on a very small number of units (6 teams), therefore the results were descriptive and involved a percent change from pre to post. The focus groups and interviews were each summarized and then summarized by site. The study team discussed the results. Using the constant comparison method, salient topics were compared within and across roles and sites. Status Completed. Major activities and accomplishments included hiring 6 CPs who together logged over 2000 clinical encounters over the funding period of the project. ### Conditions Module **Conditions:** - Mental Disorder **Keywords:** - Organization Innovation - Opinion Leaders - Schizophrenia ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** HEALTH_SERVICES_RESEARCH #### Enrollment Info **Count:** 285 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Adding a Consumer Provider to Intensive Case Management Teams (called MHICM in the VA) **Intervention Names:** - Behavioral: Consumer Provider **Label:** Consumer Provider **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Care as usual **Label:** Care as Usual **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Consumer Provider **Description:** Adding a Consumer Provider to Intensive Case Management Teams (called MHICM in the VA) **Name:** Consumer Provider **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** The BASIS-R is a 24 item, comprehensive instrument assessing a range of psychiatric symptoms and problems. It is valid and reliable in both inpatient and outpatient settings in populations with SMI. All items have five response options ranging from 0 to 4, with higher scores indicating more problems (range in possible scores is 0 to 96). **Measure:** BASIS-R **Time Frame:** immediately before the intervention (BL), and 12 months post intervention (Post). #### Secondary Outcomes **Description:** The Mental Health Recovery Measure (MHRM) is a 30-item, 5-point behaviorally-anchored self-report measure based upon recovery experiences of persons with psychiatric disabilities. The MHRM total score has good validity, correlating strongly with the Empowerment Scale and Community Living Skills Scales, yet assessing unique aspects of recovery. Range of total score is 0 to 144, with higher scores meaning better recovery. **Measure:** Mental Health Recovery Measure (MHRM) **Time Frame:** immediately before the intervention (BL), and 12 months post intervention (Post). **Description:** The mental health version of the Patient Activation Measure (PAM) is a single 13-item scale designed to assess patient's knowledge, skill, and confidence in health self-management. Respondents endorse items (e.g., "I know what each of my prescribed medications do") on a scale from 1 ("disagree strongly") to 4 ("agree strongly"). Raw scores are converted using the established methodology for the PAM to an activation score from 0 (lowest)-100 (highest). Identifying levels of activation is based on whether an activation score falls within a previously determined range of scores. Level 1, the lowest level of activation, includes activation scores of 47 or lower; Level 2 includes scores of 47.1 to 55.1; Level 3 includes scores of 55.2 to 67.0; and Level 4 (the highest activation level) includes scores of 67.1 or above. This version has similar psychometric properties as the original 13-item PAM and correlates with related constructs in other samples of people with SMI. **Measure:** Patient Activation Measure **Time Frame:** immediately before the intervention (BL), and 12 months post intervention (Post). **Description:** Perceptions of the recovery orientation of the program were assessed with the Recovery Self-Assessment (RSA), a 36 item survey that assesses domains of recovery-orientated practice (e.g., focus on life goals, involvement of patients in their own care). The RSA has high internal consistency and is thought to represent a more recovery-oriented or recovery-supportive environment. Each item ranges from 1 to 5. The total score (all 36 items averaged together) also is reported on that scale, with higher meaning more recovery. **Measure:** Recovery Self-Assessment: Person in Recovery Version **Time Frame:** immediately before the intervention (BL), and 12 months post intervention (Post) **Description:** The Illness Management and Recovery Scale (IMR) has 15 items (rated on 5-point behaviorally anchored scales) that assess progress toward goals, knowledge about mental illness, involvement with significant others and self-help, time in structured roles, impairment in functioning, symptom distress and coping, relapse prevention and hospitalizations, use of medications, and alcohol and drug use. A total IMR score is made of the mean of the items and has demonstrated good internal consistency, stability (test-retest after two weeks), and convergent validity, correlating with the Recovery Assessment Scale and the Colorado Symptom Index. The total score is reported on the scale of 1 to 5 with higher scores mean better recovery. **Measure:** Illness Management and Recovery Scale: Client Self-Rating **Time Frame:** 12 months prior to the intervention (BL1), immediately before the intervention (BL2), and 12 months post intervention (Post). **Description:** Subjective ratings of overall quality of life and the quality of social relationships, daily life, and family interactions was assessed using a combination of selected scales from the Quality of Life Instrument-Brief Version (QOLI), which been used extensively with a wide range of populations including those who are homeless, have a dual diagnosis, and are ethnic minorities. Because of low internal consistencies of subscales in our sample, a factor analysis was conducted which indicated that a larger scale that included the items from the overall quality of life, social relationships, daily life, and family interactions scales would be more reliable (all items averaged together). The score ranges from 1 to 5, with 1 meaning more quality of life. **Measure:** Quality of Life Interview, Brief Version **Time Frame:** immediately before the intervention (BL), and 12 months post intervention (Post). ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patient must have a Serious Mental Illness; * Patient must be working with a VA Intensive Case Management team Exclusion Criteria: * Prior exposure to intervention; Reduced capacity; * Patient is no longer working with a VA Intensive Case Management **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Loma Linda **Country:** United States **Facility:** VA Loma Linda Healthcare System, Loma Linda, CA **State:** California **Zip:** 92357 **Location 2:** **City:** Long Beach **Country:** United States **Facility:** VA Long Beach Healthcare System, Long Beach, CA **State:** California **Zip:** 90822 **Location 3:** **City:** San Diego **Country:** United States **Facility:** VA San Diego Healthcare System, San Diego, CA **State:** California **Zip:** 92161 **Location 4:** **City:** West Los Angeles **Country:** United States **Facility:** VA Greater Los Angeles Healthcare System, West Los Angeles, CA **State:** California **Zip:** 90073 **Location 5:** **City:** Indianapolis **Country:** United States **Facility:** Richard L. Roudebush VA Medical Center, Indianapolis, IN **State:** Indiana **Zip:** 46202-2884 **Location 6:** **City:** Las Vegas **Country:** United States **Facility:** VA Southern Nevada Healthcare System, North Las Vegas, NV **State:** Nevada **Zip:** 89106 **Location 7:** **City:** Pittsburgh **Country:** United States **Facility:** VA Pittsburgh Healthcare System University Drive Division, Pittsburgh, PA **State:** Pennsylvania **Zip:** 15240 #### Overall Officials **Official 1:** **Affiliation:** VA Pittsburgh Healthcare System University Drive Division, Pittsburgh, PA **Name:** Matthew J. Chinman, PhD **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** VA Greater Los Angeles Healthcare System, West Los Angeles, CA **Name:** Amy N. Cohen, PhD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Chinman M, Shoai R, Cohen A. Using organizational change strategies to guide peer support technician implementation in the Veterans Administration. Psychiatr Rehabil J. 2010 Spring;33(4):269-77. doi: 10.2975/33.4.2010.269.277. **PMID:** 20374985 **Citation:** Chinman M, Salzer M, O'Brien-Mazza D. National survey on implementation of peer specialists in the VA: implications for training and facilitation. Psychiatr Rehabil J. 2012 Dec;35(6):470-3. doi: 10.1037/h0094582. **PMID:** 23276242 **Citation:** Chinman M, Oberman RS, Hanusa BH, Cohen AN, Salyers MP, Twamley EW, Young AS. A cluster randomized trial of adding peer specialists to intensive case management teams in the Veterans Health Administration. J Behav Health Serv Res. 2015 Jan;42(1):109-21. doi: 10.1007/s11414-013-9343-1. Erratum In: J Behav Health Serv Res. 2015 Jan;42(1):122. **PMID:** 23657754 **Citation:** Hamilton AB, Chinman M, Cohen AN, Oberman RS, Young AS. Implementation of consumer providers into mental health intensive case management teams. J Behav Health Serv Res. 2015 Jan;42(1):100-8. doi: 10.1007/s11414-013-9365-8. **PMID:** 24091610 **Citation:** Chinman M, George P, Dougherty RH, Daniels AS, Ghose SS, Swift A, Delphin-Rittmon ME. Peer support services for individuals with serious mental illnesses: assessing the evidence. Psychiatr Serv. 2014 Apr 1;65(4):429-41. doi: 10.1176/appi.ps.201300244. **PMID:** 24549400 **Citation:** Chinman M, Oberman RS, Hanusa BH, Cohen AN, Salyers MP, Twamley EW, Young AS. Erratum to: A Cluster Randomized Trial of Adding Peer Specialists to Intensive Case Management Teams in the Veterans Health Administration. J Behav Health Serv Res. 2015 Jan;42(1):122. doi: 10.1007/s11414-013-9377-4. No abstract available. **PMID:** 24217992 #### See Also Links **Label:** Paper linked to the study **URL:** http://www.ncbi.nlm.nih.gov/pubmed/16645906 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M15376 - Name: Schizophrenia - Relevance: LOW - As Found: Unknown - ID: M4815 - Name: Mental Disorders - Relevance: HIGH - As Found: Mental Disorders - ID: M14473 - Name: Psychotic Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001523 - Term: Mental Disorders ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Consumer Provider **Description:** Adding a Consumer Provider to Intensive Case Management Teams (called MHICM in the VA) **ID:** EG000 **Other Num at Risk:** 149 **Serious Number Affected:** 85 **Serious Number At Risk:** 149 **Title:** Consumer Provider **Group ID:** EG001 **Title:** Case as Usual **Description:** Care as usual on case management teams **ID:** EG001 **Other Num at Risk:** 133 **Serious Number Affected:** 55 **Serious Number At Risk:** 133 **Title:** Case as Usual **Frequency Threshold:** 0 #### Serious Events **Term:** Death **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders ##### Stats **Group ID:** EG000 **Num Affected:** 10 **Num At Risk:** 149 **Num Events:** 10 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 133 **Num Events:** 3 **Term:** Psychiatric **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders ##### Stats **Group ID:** EG000 **Num Affected:** 41 **Num At Risk:** 149 **Num Events:** 105 **Group ID:** EG001 **Num Affected:** 23 **Num At Risk:** 133 **Num Events:** 74 **Term:** Cardiac **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders ##### Stats **Group ID:** EG000 **Num Affected:** 24 **Num At Risk:** 149 **Num Events:** 45 **Group ID:** EG001 **Num Affected:** 10 **Num At Risk:** 133 **Num Events:** 12 **Term:** other hospitalizations **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num Affected:** 10 **Num At Risk:** 149 **Num Events:** 40 **Group ID:** EG001 **Num Affected:** 19 **Num At Risk:** 133 **Num Events:** 19 **Time Frame:** 1 year ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 149 **Group ID:** BG001 **Value:** 133 **Group ID:** BG002 **Value:** 282 **Units:** Participants ### Group **ID:** BG000 **Title:** Consumer Provider **Description:** Adding a Consumer Provider to Intensive Case Management Teams (called MHICM in the VA) ### Group **ID:** BG001 **Title:** Care as Usual **Description:** Care as usual ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 9.19 **Value:** 54.59 #### Measurement **Group ID:** BG001 **Spread:** 11.13 **Value:** 51.89 #### Measurement **Group ID:** BG002 **Spread:** 10.15 **Value:** 52.95 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 15 #### Measurement **Group ID:** BG001 **Value:** 17 #### Measurement **Group ID:** BG002 **Value:** 32 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 134 #### Measurement **Group ID:** BG001 **Value:** 116 #### Measurement **Group ID:** BG002 **Value:** 250 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 39 #### Measurement **Group ID:** BG001 **Value:** 13 #### Measurement **Group ID:** BG002 **Value:** 52 **Class Title:** African American non-Hispanic #### Measurement **Group ID:** BG000 **Value:** 74 #### Measurement **Group ID:** BG001 **Value:** 78 #### Measurement **Group ID:** BG002 **Value:** 152 **Class Title:** White non hispanic #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 12 #### Measurement **Group ID:** BG002 **Value:** 22 **Class Title:** Bi racial non hispanic #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 2 **Class Title:** African American Hispanic #### Measurement **Group ID:** BG000 **Value:** 18 #### Measurement **Group ID:** BG001 **Value:** 21 #### Measurement **Group ID:** BG002 **Value:** 39 **Class Title:** White Hispanic #### Measurement **Group ID:** BG000 **Value:** 7 #### Measurement **Group ID:** BG001 **Value:** 8 #### Measurement **Group ID:** BG002 **Value:** 15 **Class Title:** Unknown ### Measure #### Measurement **Group ID:** BG000 **Value:** 149 #### Measurement **Group ID:** BG001 **Value:** 133 #### Measurement **Group ID:** BG002 **Value:** 282 **Class Title:** United States **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Description:** Age at time of enrollment **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** NUMBER **Title:** Race/Ethnicity, Customized **Unit of Measure:** participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Region of Enrollment **Unit of Measure:** Participants **Population Description:** Veteran patients of MHICM teams ## Results Section - More Information Module ### Certain Agreement **PI Sponsor Employee:** True ### Limitations and Caveats **Description:** 1. cluster randomized trial - teams may have exerted influence on the findings. 2. analyses were intent-to-treat, but about half the veterans in the PS group did not receive any PS services. 3. the study involved a small number of peers (n=6) ### Point of Contact **Email:** matthew.chinman@va.gov **Organization:** Pittsburgh VA **Phone:** 412 360-2438 **Title:** Matthew Chinman ## Results Section - Outcome Measures Module ### Outcome Measure 1 #### Analysis **CI Lower Limit:** **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** comparisons between the PS and Usual Care groups were completed with a regression testing the interaction of group (PS vs Usual Care) and time (baseline vs follow-up) for the outcome measure. The measure was analyzed with mixed effect hierarchical regressions which accounted for the nesting of site under treatment, subjects within sites, and subjects over time. **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** .5 **P-Value Comment:** Models included site, age, race, ethnicity. **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** Regression, Linear **Tested Non-Inferiority:** ### Outcome Measure 2 #### Analysis **CI Lower Limit:** **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY **Other Analysis Description:** **P-Value:** .56 **P-Value Comment:** **Parameter Type:** Z tests if Reg coeff are diff from 0 **Parameter Value:** -.58 **Statistical Comment:** **Statistical Method:** Regression, Linear **Tested Non-Inferiority:** ### Outcome Measure 3 #### Analysis **CI Lower Limit:** **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** comparisons between the PS and Usual Care groups were completed with a regression testing the interaction of group (PS vs Usual Care) and time (baseline vs follow-up) for the outcome measure. The measure was analyzed with mixed effect hierarchical regressions which accounted for the nesting of site under treatment, subjects within sites, and subjects over time. **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** .05 **P-Value Comment:** Models included site, age, race, ethnicity. This is the calculated p value. **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** Regression, Linear **Tested Non-Inferiority:** ### Outcome Measure 4 #### Analysis **CI Lower Limit:** **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY **Other Analysis Description:** **P-Value:** .58 **P-Value Comment:** **Parameter Type:** Z test if reg coeff is diff from 0 **Parameter Value:** .56 **Statistical Comment:** **Statistical Method:** Regression, Linear **Tested Non-Inferiority:** ### Outcome Measure 5 #### Analysis **CI Lower Limit:** **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY **Other Analysis Description:** **P-Value:** .87 **P-Value Comment:** **Parameter Type:** Z test if reg coeff is diff than 0 **Parameter Value:** -.16 **Statistical Comment:** **Statistical Method:** Regression, Linear **Tested Non-Inferiority:** ### Outcome Measure 6 #### Analysis **CI Lower Limit:** **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY **Other Analysis Description:** **P-Value:** .98 **P-Value Comment:** **Parameter Type:** Z test if ref coeff is diff than 0 **Parameter Value:** .03 **Statistical Comment:** **Statistical Method:** Regression, Linear **Tested Non-Inferiority:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 14.28 - **Upper Limit:** - **Value:** 26.15 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 14.24 - **Upper Limit:** - **Value:** 29.34 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 14.05 - **Upper Limit:** - **Value:** 24.57 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 15.11 - **Upper Limit:** - **Value:** 26.76 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 17.88 - **Upper Limit:** - **Value:** 85.00 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 18.79 - **Upper Limit:** - **Value:** 78.3 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 15.9 - **Upper Limit:** - **Value:** 85.66 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 16.54 - **Upper Limit:** - **Value:** 80.52 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 5.41 - **Upper Limit:** - **Value:** 39.31 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 5.37 - **Upper Limit:** - **Value:** 39.09 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 5.5 - **Upper Limit:** - **Value:** 40.34 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 5.01 - **Upper Limit:** - **Value:** 38.76 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** .53 - **Upper Limit:** - **Value:** 3.86 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** .61 - **Upper Limit:** - **Value:** 3.81 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** .61 - **Upper Limit:** - **Value:** 3.93 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** .52 - **Upper Limit:** - **Value:** 3.83 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** .58 - **Upper Limit:** - **Value:** 3.51 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** .56 - **Upper Limit:** - **Value:** 3.39 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** .56 - **Upper Limit:** - **Value:** 3.56 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** .54 - **Upper Limit:** - **Value:** 3.48 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.04 - **Upper Limit:** - **Value:** 4.64 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.03 - **Upper Limit:** - **Value:** 4.54 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.04 - **Upper Limit:** - **Value:** 4.72 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** .9 - **Upper Limit:** - **Value:** 4.71 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** The BASIS-R is a 24 item, comprehensive instrument assessing a range of psychiatric symptoms and problems. It is valid and reliable in both inpatient and outpatient settings in populations with SMI. All items have five response options ranging from 0 to 4, with higher scores indicating more problems (range in possible scores is 0 to 96). **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** immediately before the intervention (BL), and 12 months post intervention (Post). **Title:** BASIS-R **Type:** PRIMARY **Unit of Measure:** units on a scale ##### Group **Description:** Adding a Consumer Provider to Intensive Case Management Teams (called MHICM in the VA) **ID:** OG000 **Title:** Consumer Provider ##### Group **Description:** Care as usual on the case management teams **ID:** OG001 **Title:** Care as Usual #### Outcome Measure 2 **Description:** The Mental Health Recovery Measure (MHRM) is a 30-item, 5-point behaviorally-anchored self-report measure based upon recovery experiences of persons with psychiatric disabilities. The MHRM total score has good validity, correlating strongly with the Empowerment Scale and Community Living Skills Scales, yet assessing unique aspects of recovery. Range of total score is 0 to 144, with higher scores meaning better recovery. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** comparisons between the PS and Usual Care groups used a regression testing the interaction of group (PS vs Usual Care) and time (baseline vs follow-up) for the outcome measure. The measure was analyzed with mixed effect hierarchical regressions which accounted for the nesting of site under treatment, subjects within sites, and subjects over time. **Reporting Status:** POSTED **Time Frame:** immediately before the intervention (BL), and 12 months post intervention (Post). **Title:** Mental Health Recovery Measure (MHRM) **Type:** SECONDARY **Unit of Measure:** units on a scale ##### Group **Description:** Adding a Consumer Provider to Intensive Case Management Teams (called MHICM in the VA) **ID:** OG000 **Title:** Consumer Provider ##### Group **Description:** Care as usual on the case management teams **ID:** OG001 **Title:** Care as Usual #### Outcome Measure 3 **Description:** The mental health version of the Patient Activation Measure (PAM) is a single 13-item scale designed to assess patient's knowledge, skill, and confidence in health self-management. Respondents endorse items (e.g., "I know what each of my prescribed medications do") on a scale from 1 ("disagree strongly") to 4 ("agree strongly"). Raw scores are converted using the established methodology for the PAM to an activation score from 0 (lowest)-100 (highest). Identifying levels of activation is based on whether an activation score falls within a previously determined range of scores. Level 1, the lowest level of activation, includes activation scores of 47 or lower; Level 2 includes scores of 47.1 to 55.1; Level 3 includes scores of 55.2 to 67.0; and Level 4 (the highest activation level) includes scores of 67.1 or above. This version has similar psychometric properties as the original 13-item PAM and correlates with related constructs in other samples of people with SMI. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** comparisons between the PS and Usual Care groups used a regression testing the interaction of group (PS vs Usual Care) and time (baseline vs follow-up) for the outcome measure. The measure was analyzed with mixed effect hierarchical regressions which accounted for the nesting of site under treatment, subjects within sites, and subjects over time. **Reporting Status:** POSTED **Time Frame:** immediately before the intervention (BL), and 12 months post intervention (Post). **Title:** Patient Activation Measure **Type:** SECONDARY **Unit of Measure:** units on a scale ##### Group **Description:** Adding a Consumer Provider to Intensive Case Management Teams (called MHICM in the VA) **ID:** OG000 **Title:** Consumer Provider ##### Group **Description:** Care as usual on the case management teams **ID:** OG001 **Title:** Care as Usual #### Outcome Measure 4 **Description:** Perceptions of the recovery orientation of the program were assessed with the Recovery Self-Assessment (RSA), a 36 item survey that assesses domains of recovery-orientated practice (e.g., focus on life goals, involvement of patients in their own care). The RSA has high internal consistency and is thought to represent a more recovery-oriented or recovery-supportive environment. Each item ranges from 1 to 5. The total score (all 36 items averaged together) also is reported on that scale, with higher meaning more recovery. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** comparisons between the PS and Usual Care groups used a regression testing the interaction of group (PS vs Usual Care) and time (baseline vs follow-up) for the outcome measure. The measure was analyzed with mixed effect hierarchical regressions which accounted for the nesting of site under treatment, subjects within sites, and subjects over time. **Reporting Status:** POSTED **Time Frame:** immediately before the intervention (BL), and 12 months post intervention (Post) **Title:** Recovery Self-Assessment: Person in Recovery Version **Type:** SECONDARY **Unit of Measure:** units on a scale ##### Group **Description:** Adding a Consumer Provider to Intensive Case Management Teams (called MHICM in the VA) **ID:** OG000 **Title:** Consumer Provider ##### Group **Description:** Care as usual on the case management teams **ID:** OG001 **Title:** Care as Usual #### Outcome Measure 5 **Description:** The Illness Management and Recovery Scale (IMR) has 15 items (rated on 5-point behaviorally anchored scales) that assess progress toward goals, knowledge about mental illness, involvement with significant others and self-help, time in structured roles, impairment in functioning, symptom distress and coping, relapse prevention and hospitalizations, use of medications, and alcohol and drug use. A total IMR score is made of the mean of the items and has demonstrated good internal consistency, stability (test-retest after two weeks), and convergent validity, correlating with the Recovery Assessment Scale and the Colorado Symptom Index. The total score is reported on the scale of 1 to 5 with higher scores mean better recovery. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** comparisons between the PS and Usual Care groups used a regression testing the interaction of group (PS vs Usual Care) and time (baseline vs follow-up) for the outcome measure. The measure was analyzed with mixed effect hierarchical regressions which accounted for the nesting of site under treatment, subjects within sites, and subjects over time. **Reporting Status:** POSTED **Time Frame:** 12 months prior to the intervention (BL1), immediately before the intervention (BL2), and 12 months post intervention (Post). **Title:** Illness Management and Recovery Scale: Client Self-Rating **Type:** SECONDARY **Unit of Measure:** units on a scale ##### Group **Description:** Adding a Consumer Provider to Intensive Case Management Teams (called MHICM in the VA) **ID:** OG000 **Title:** Consumer Provider ##### Group **Description:** Care as usual on the case management teams **ID:** OG001 **Title:** Care as Usual #### Outcome Measure 6 **Description:** Subjective ratings of overall quality of life and the quality of social relationships, daily life, and family interactions was assessed using a combination of selected scales from the Quality of Life Instrument-Brief Version (QOLI), which been used extensively with a wide range of populations including those who are homeless, have a dual diagnosis, and are ethnic minorities. Because of low internal consistencies of subscales in our sample, a factor analysis was conducted which indicated that a larger scale that included the items from the overall quality of life, social relationships, daily life, and family interactions scales would be more reliable (all items averaged together). The score ranges from 1 to 5, with 1 meaning more quality of life. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** comparisons between the PS and Usual Care groups used a regression testing the interaction of group (PS vs Usual Care) and time (baseline vs follow-up) for the outcome measure. The measure was analyzed with mixed effect hierarchical regressions which accounted for the nesting of site under treatment, subjects within sites, and subjects over time. **Reporting Status:** POSTED **Time Frame:** immediately before the intervention (BL), and 12 months post intervention (Post). **Title:** Quality of Life Interview, Brief Version **Type:** SECONDARY **Unit of Measure:** units on a scale ##### Group **Description:** Adding a Consumer Provider to Intensive Case Management Teams (called MHICM in the VA) **ID:** OG000 **Title:** Consumer Provider ##### Group **Description:** Care as usual on the case management teams **ID:** OG001 **Title:** Care as Usual ### Participant Flow Module #### Group **Description:** Adding a Consumer Provider to Intensive Case Management Teams (called MHICM in the VA) **ID:** FG000 **Title:** Consumer Provider #### Group **Description:** Care as usual **ID:** FG001 **Title:** Care as Usual #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 149 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 133 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 122 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 116 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 27 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 17 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT00550979 **Brief Title:** Gestational Diabetes Mellitus and Implications for Cardiovascular Disease Risk **Official Title:** Gestational Diabetes Mellitus and Implications for Cardiovascular Disease Risk #### Organization Study ID Info **ID:** 2007P001406 #### Organization **Class:** OTHER **Full Name:** Brigham and Women's Hospital ### Status Module #### Completion Date **Date:** 2014-01 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2013-08-29 **Type:** ESTIMATED **Last Update Submit Date:** 2013-08-28 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2013-10 **Type:** ESTIMATED #### Start Date **Date:** 2007-09 **Status Verified Date:** 2013-08 #### Study First Post Date **Date:** 2007-10-30 **Type:** ESTIMATED **Study First Submit Date:** 2007-10-26 **Study First Submit QC Date:** 2007-10-26 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Massachusetts General Hospital **Class:** OTHER **Name:** Joslin Diabetes Center #### Lead Sponsor **Class:** OTHER **Name:** Brigham and Women's Hospital #### Responsible Party **Investigator Affiliation:** Brigham and Women's Hospital **Investigator Full Name:** Rhonda Bentley-Lewis, MD **Investigator Title:** Rhonda Bentley-Lewis, MD, MBA, MMSc **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this research study is to look at whether there are differences in blood vessel function, risk for developing diabetes (high blood sugar), lipid (blood fat) levels, and levels of other blood markers in women who have had a pregnancy complicated by diabetes as compared with women who have had a normal pregnancy. ### Conditions Module **Conditions:** - Gestational Diabetes Mellitus **Keywords:** - gestational diabetes mellitus - cardiovascular disease risk - heart disease risk ### Design Module #### Bio Spec **Description:** blood and urine **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** CASE_CONTROL **Time Perspective:** CROSS_SECTIONAL #### Enrollment Info **Count:** 96 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Black women with history of pregnancy/ies complicated by gestational diabetes mellitus **Label:** 1 #### Arm Group 2 **Description:** Black women with history of normal, uncomplicated pregnancy/ies **Label:** 2 #### Arm Group 3 **Description:** White women with a history of pregnancy/ies complicated by gestational diabetes. **Label:** 3 #### Arm Group 4 **Description:** White women with a history of normal, uncomplicated pregnancy/ies. **Label:** 4 ### Outcomes Module #### Primary Outcomes **Measure:** endothelial function **Time Frame:** same day ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Healthy women of self-identified African descent (black) and European (white) ancestry * 18-40 years of age * Regular menstrual cycles * 5 or less years postpartum * Have either a normal or a GDM index pregnancy history * Are in good health free of thyroid, cardiac, or renal disease Exclusion Criteria: * If you are pregnant * If you are lactating **Healthy Volunteers:** True **Maximum Age:** 40 Years **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** FEMALE **Standard Ages:** - ADULT **Study Population:** Black and white women who have had uncomplicated pregnancies or pregnancies complicated by gestational diabetes in the past 5 years ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** RBLResearchGroup@partners.org **Name:** Research Assistant **Phone:** 617-724-3504 **Role:** CONTACT **Contact 2:** **Email:** rbentleylewis@partners.org **Name:** Rhonda Bentley-Lewis, MD, MBA **Phone:** 617-726-2874 **Role:** CONTACT #### Locations **Location 1:** **City:** Boston **Contacts:** ***Contact 1:*** - **Name:** Rhonda Bentley-Lewis, MD - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 2:*** - **Name:** Florence Brown, MD - **Role:** SUB_INVESTIGATOR ***Contact 3:*** - **Name:** David Nathan, MD - **Role:** SUB_INVESTIGATOR ***Contact 4:*** - **Name:** Mark Creager, MD - **Role:** SUB_INVESTIGATOR **Country:** United States **Facility:** Brigham and Women's Hospital, Massachusetts General Hospital **State:** Massachusetts **Status:** RECRUITING **Zip:** 02115 **Location 2:** **City:** Boston **Country:** United States **Facility:** Brigham and Women's Hospital **State:** Massachusetts **Status:** RECRUITING **Zip:** 02115 #### Overall Officials **Official 1:** **Affiliation:** Brigham and Women's Hospital **Name:** Rhonda Bentley-Lewis, MD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000044882 - Term: Glucose Metabolism Disorders - ID: D000008659 - Term: Metabolic Diseases - ID: D000004700 - Term: Endocrine System Diseases - ID: D000011248 - Term: Pregnancy Complications - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions ### Condition Browse Module - Browse Leaves - ID: M7115 - Name: Diabetes Mellitus - Relevance: HIGH - As Found: Diabetes Mellitus - ID: M9419 - Name: Heart Diseases - Relevance: LOW - As Found: Unknown - ID: M19012 - Name: Diabetes, Gestational - Relevance: HIGH - As Found: Gestational Diabetes Mellitus - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: M25403 - Name: Glucose Metabolism Disorders - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000016640 - Term: Diabetes, Gestational - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000003920 - Term: Diabetes Mellitus ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02001779 **Acronym:** IRCSMBO **Brief Title:** IRS(Irradiation Stent) vs. CS(Conventional Stent) Insertion in Inoperable Malignant Biliary Obstruction **Official Title:** Study of Stent Insertion Combined With or Without Brachytherapy in Patients With Inoperable Malignant Biliary Obstruction #### Organization Study ID Info **ID:** IR-C-STENT-MBO-83272121 #### Organization **Class:** OTHER **Full Name:** Zhongda Hospital ### Status Module #### Completion Date **Date:** 2016-09 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-02-14 **Type:** ACTUAL **Last Update Submit Date:** 2017-02-12 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2016-09 **Type:** ACTUAL #### Start Date **Date:** 2013-10 **Type:** ACTUAL **Status Verified Date:** 2017-02 #### Study First Post Date **Date:** 2013-12-05 **Type:** ESTIMATED **Study First Submit Date:** 2013-11-22 **Study First Submit QC Date:** 2013-12-04 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Zhongda Hospital #### Responsible Party **Investigator Affiliation:** Zhongda Hospital **Investigator Full Name:** Gao-jun Teng **Investigator Title:** Professor of Radiology & Chair,Department of Radiology **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Stenting the malignant biliary obstruction is considered to be the preferred palliation modality to relieve pruritus,cholangitis,pain and jaundice in patients without surgical indications of malignant biliary obstruction.An unicentral clinical trial has demonstrated the safety and technical feasibility of an irradiation biliary stent insertion in patients with biliary obstruction caused by adenocarcinomas, such a treatment seems have benefits in relieving jaundice and extending survival when compared to a conventional biliary stent.However,the small sample size,the population distribution of two groups will influence the final results in obtaining a powerful statistical conclusion.Therefore,a multicentric study was designed to prospectively compared the responses to the treatment with this irradiation biliary stent versus the conventional biliary self-expandable stent in patients with inoperable malignant biliary obstruction. **Detailed Description:** Malignant biliary obstruction is a common condition caused by various carcinomas.The clinical process is usually silent and insidious,only 10-20% patients can be surgically removed once obstructive jaundice occurred.The long-term survival rate after resection remain dismal.Relief of obstructive jaundice plays major role on prognosis in patients without surgical indications.For unresectable patients,stenting the biliary obstruction is considered to be the preferred palliation modality to relieve pruritus,cholangitis,pain and jaundice.Although a number of new techniques for treatment of malignant biliary obstruction have been developed,there have been no significant differences in survival or quality of life comparing surgery bypass versus plastic stent insertion,metal stent insertion versus plastic stents insertion,or covered stent versus uncovered stents insertion in such patients.Our previous unicentric clinical trial has demonstrated the safety and technical feasibility of a biliary intraluminal irradiation stent insertion in patients with biliary obstruction caused by adenocarcinomas, such a treatment seems have benefits in relieving jaundice and extending survival when compared to a conventional biliary stent.However,the small sample size,the population distribution of two groups will influence the final results in obtaining a powerful statistical conclusion in the overall survival and stent patency between two groups.Therefore,a multicentric study was designed to prospectively compared the responses to the treatment with this irradiation biliary stent versus the conventional biliary self-expandable stent in patients with inoperable malignant biliary obstruction. ### Conditions Module **Conditions:** - Malignant Biliary Obstruction **Keywords:** - Malignant biliary obstruction - Obstruction/stricture - Stent - Brachytherapy - Bile duct ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 328 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** A self-expandable metallic biliary stent loaded with 125 iodine seeds is inserted in patients with inoperable malignant biliary obstruction. **Intervention Names:** - Device: Biliary SEMS loaded with 125I seeds **Label:** Biliary SEMS loaded with 125I seeds **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** A self-expandable metallic biliary stent is inserted in patients with inoperable malignant biliary obstruction. **Intervention Names:** - Device: Conventional biliary SEMS **Label:** Conventional biliary SEMS **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Biliary SEMS loaded with 125I seeds **Description:** A self-expandable metallic biliary stent loaded with 125 iodine seeds is inserted in patients with inoperable malignant biliary obstruction. **Name:** Biliary SEMS loaded with 125I seeds **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - Conventional biliary SEMS **Description:** A self-expandable metallic stent is inserted in patients with inoperable malignant biliary obstruction. **Name:** Conventional biliary SEMS **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Time from stenting to the day when stricture/obstruction of the stent occured or restricture/reobstruction after recanalization the obstructed stent. **Measure:** Patency **Time Frame:** Participants will be followed till die or lost to follow-up,an expected average of a year. #### Secondary Outcomes **Description:** Time from stenting to the day when the patients died or lost to the follow-up. **Measure:** Over survival **Time Frame:** Participants will be followed till die or lost to follow-up,an expected average of a year. **Description:** The rate of relief of the symptoms and signs of the patients. **Measure:** Clinical success **Time Frame:** Participants will be followed till die or lost to follow-up,an expected average of a year. **Description:** Including technique success rate,side effect/complication,radioactive safety. **Measure:** Safety **Time Frame:** Participants will be followed till die or lost to follow-up,an expected average of a year. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Clinical,histological or pathological diagnosis of malignant biliary obstruction * Unresectability or refusal to be surgically treated * The Eastern Cooperative Oncology Group(ECOG) scores from 0 to 3 * With symptoms such as jaundice related to biliary obstruction * Willing and able to comply with the study procedures and provide written informed consent to participate in the study * Biliary obstruction of Bismuthe-Corlette,type I and II Exclusion Criteria: * Benign biliary obstruction * The Eastern Cooperative Oncology Group(ECOG) score of 4 * Biliary tract stricture that could not be dilated enough to pass the delivery system * Presence of metallic biliary stent or bile duct surgery * Percutaneous transhepatic cholangiography(PTC) procedure was contraindicated * Active hepatitis * Biliary obstruction of Bismuthe-Corlette,type Ⅲ and Ⅳ * Uncooperative or could not provide authorization and signature **Maximum Age:** 90 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Nanjing **Country:** China **Facility:** Zhongda Hospital,Southeast University **State:** Jiangsu **Zip:** 210009 #### Overall Officials **Official 1:** **Affiliation:** Zhongda Hospital **Name:** Gao-jun Teng, Ph.D,MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Zhu HD, Guo JH, Huang M, Ji JS, Xu H, Lu J, Li HL, Wang WH, Li YL, Ni CF, Shi HB, Xiao EH, Lv WF, Sun JH, Xu K, Han GH, Du LA, Ren WX, Li MQ, Mao AW, Xiang H, Zhang KX, Min J, Zhu GY, Su C, Chen L, Teng GJ. Irradiation stents vs. conventional metal stents for unresectable malignant biliary obstruction: A multicenter trial. J Hepatol. 2018 May;68(5):970-977. doi: 10.1016/j.jhep.2017.12.028. Epub 2018 Jan 10. **PMID:** 29331343 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M6475 - Name: Constriction, Pathologic - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Browse Branches - Abbrev: Micro - Name: Micronutrients - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M10488 - Name: Iodine - Relevance: LOW - As Found: Unknown - ID: M229695 - Name: Cadexomer iodine - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05665179 **Acronym:** RB **Brief Title:** Removing Barriers: Community Partnering for Innovative Solutions to the Opioid Crisis **Official Title:** Removing Barriers: Community Partnering for Innovative Solutions to the Opioid Crisis #### Organization Study ID Info **ID:** HUM00152102 #### Organization **Class:** OTHER **Full Name:** University of Michigan ### Status Module #### Completion Date **Date:** 2024-07-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-02-12 **Type:** ACTUAL **Last Update Submit Date:** 2024-02-08 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-07-31 **Type:** ESTIMATED #### Start Date **Date:** 2021-12-23 **Type:** ACTUAL **Status Verified Date:** 2024-02 #### Study First Post Date **Date:** 2022-12-27 **Type:** ACTUAL **Study First Submit Date:** 2022-12-07 **Study First Submit QC Date:** 2022-12-16 ### Sponsor Collaborators Module #### Collaborators **Class:** FED **Name:** U.S. National Science Foundation #### Lead Sponsor **Class:** OTHER **Name:** University of Michigan #### Responsible Party **Investigator Affiliation:** University of Michigan **Investigator Full Name:** David Cordova **Investigator Title:** Associate Professor of Social Work, School of Social Work **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The opioid epidemic has become one of America's deadliest crises, surpassing car crashes, firearms, and HIV/AIDS as a leading cause of death for Americans under fifty years of age. People trying to recover from opioid-use disorder face many obstacles. Obstacles such as minor legal problems (e.g., arrest warrants for failure to pay a fine, failure to appear in court, or late child support payments) can undermine the stability needed to overcome opioid dependence. Outstanding legal obligations make it difficult to find jobs and to secure housing. They can result in removal from treatment programs as well as incarceration. Resolving these legal problems requires coordination, organization, preparation, travel, and time-expectations that may be problematic for many people in the early stages of recovery. Technology has the potential to make resolving these legal problems much easier. Online platform technology is now available that can guide people in recovery through the resolution of many legal problems at no cost and without an attorney, potentially doing so quickly, remotely, and at any time of day. This study of individuals in treatment in Michigan tests whether resolving outstanding legal issues improves drug treatment outcomes. The research also examines whether and to what extent resolving legal issues supports family reunification, reduces future criminal behavior, and improves access to jobs and housing for clients in treatment for opioid use disorder. A randomized controlled trial (RCT) is used to determine the effects of resolving legal issues on these outcomes. For identification, the investigators leverage the random assignment of legal services to treatment center clients, along with the random assignment of clients to treatment centers by birth month. We assemble a novel longitudinal dataset of hundreds of clients in treatment for substance use disorder and link these clients to several administrative datasets and qualitative data, which allows for measurement of: (1) substance use behaviors and (2) justice-system involvement, including civil and criminal legal system encounters. This study also uses linked client and administrative data to research the population in opioid treatment centers, follow-up behaviors, and whether the consequences of providing no-cost legal services differ by client background. Findings from this research will improve America's understanding of the acute socio-legal needs faced by those experiencing opioid use disorder and provide recommendations to help target resources toward the areas that best support long-term abstinence from opioids and other drugs. ### Conditions Module **Conditions:** - Opioid Use Disorder - Alcohol Use Disorder - Health Risk Behaviors - Criminal Behavior - Housing Problems - Overdose of Opiate ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 800 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Receives tutorial for free online legal resolution at treatment center **Intervention Names:** - Behavioral: Removing Barriers to Recovery **Label:** Treatment **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** The control group is not provided the experimental intervention. **Label:** Control **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Treatment **Description:** Experimental patients are provided no-cost legal services in-person at addiction treatment centers using an online platform **Name:** Removing Barriers to Recovery **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** Consulting electronic medical records, we measure how frequently the participant report using substance(s). Daily rates will be recorded or calculated if frequency of use is recorded in weekly, monthly, or annual terms. **Measure:** Frequency of substance misuse **Time Frame:** 1 year **Description:** Consulting electronic medical records, what is the mean quantity/measurement of substance(s) reported by participant per use. For alcohol, this is mean number of drinks per day(indicate beer, wine, liquor). For drugs this should be recorded in the standard measurement commonplace to that specific drug as recorded in the medical record, such as marijuana use in mean grams per day. **Measure:** Quantity of substance(s) **Time Frame:** 1 year #### Secondary Outcomes **Description:** Consulting electronic medical records, we measure how frequently the participant report using substance(s). Daily rates will be recorded or calculated if frequency of use is recorded in weekly, monthly, or annual terms. **Measure:** Frequency of substance misuse **Time Frame:** 2 years **Description:** Consulting electronic medical records, what is the mean quantity/measurement of substance(s) reported by participant per use. For alcohol, this is mean number of drinks per day(indicate beer, wine, liquor). For drugs this should be recorded in the standard measurement commonplace to that specific drug as recorded in the medical record, such as marijuana use in mean grams per day. **Measure:** Quantity of substance(s) **Time Frame:** 2 years **Description:** Utilizing State Court Administrative Office data, indicate count of all misdemeanor charges and count of felony charges. Using public records search on participant last known address, indicate count of civil case(s) including divorce, custody, eviction, bankruptcy, debt collection. **Measure:** Count of justice system involvement **Time Frame:** 1 year **Description:** Utilizing State Court Administrative Office data, indicate case type of misdemeanor charges and count of felony charges. Using public records search on participant last known address, indicate case type of civil case(s) including divorce, custody, eviction, bankruptcy, debt collection. **Measure:** Justice system involvement case type **Time Frame:** 1 year **Description:** Utilizing State Court Administrative Office data, indicate count of misdemeanor charges and count of felony charges. Using public records search on participant last known address, indicate count of civil case(s). **Measure:** Count of justice system involvement **Time Frame:** 2 years **Description:** Utilizing State Court Administrative Office data, indicate case type of all misdemeanor charges and count of felony charges. Using public records search on participant last known address, indicate case type of civil case(s) including divorce, custody, eviction, bankruptcy, debt collection. **Measure:** Case type of justice system involvement **Time Frame:** 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: \*Receiving substance use disorder treatment services Exclusion Criteria: \*Not receiving substance use disorder treatment services **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** cordovad@umich.edu **Name:** David Cordova, PhD **Phone:** 734-763-6201 **Role:** CONTACT #### Locations **Location 1:** **City:** Ann Arbor **Contacts:** ***Contact 1:*** - **Email:** ganderson@homeofnewvision.org - **Name:** Glynis Anderson, LMSW, ACSW - **Phone:** 734-975-1602 - **Role:** CONTACT ***Contact 2:*** - **Name:** David Cordova, PhD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Home of New Vision **State:** Michigan **Status:** RECRUITING **Zip:** 48104 **Location 2:** **City:** Ypsilanti **Contacts:** ***Contact 1:*** - **Email:** rdalton@dawnfarm.org - **Name:** Raymond Dalton - **Phone:** 734-485-8725 - **Role:** CONTACT ***Contact 2:*** - **Email:** SHAYES@dawnfarm.org - **Name:** Stacey Hayes - **Phone:** (734) 485-8725 - **Role:** CONTACT ***Contact 3:*** - **Name:** David Cordova, PhD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Dawn Farm **State:** Michigan **Status:** RECRUITING **Zip:** 48197 #### Overall Officials **Official 1:** **Affiliation:** Associate Professor **Name:** David Cordva, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Access Criteria:** public **Description:** deidentified data only **Info Types:** - STUDY_PROTOCOL - ICF **IPD Sharing:** YES **Time Frame:** within 1 year of study completion and follow-up administrative data linkages complete ## Derived Section ### Condition Browse Module - Ancestors - ID: D000019973 - Term: Alcohol-Related Disorders - ID: D000019966 - Term: Substance-Related Disorders - ID: D000064419 - Term: Chemically-Induced Disorders - ID: D000001523 - Term: Mental Disorders - ID: D000079524 - Term: Narcotic-Related Disorders - ID: D000062787 - Term: Drug Overdose - ID: D000063487 - Term: Prescription Drug Misuse - ID: D000076064 - Term: Drug Misuse ### Condition Browse Module - Browse Branches - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions - Abbrev: BC25 - Name: Substance Related Disorders ### Condition Browse Module - Browse Leaves - ID: M3774 - Name: Alcohol Drinking - Relevance: LOW - As Found: Unknown - ID: M30011 - Name: Drug Overdose - Relevance: LOW - As Found: Unknown - ID: M2423 - Name: Opiate Overdose - Relevance: HIGH - As Found: Overdose of Opiate - ID: M3783 - Name: Alcoholism - Relevance: HIGH - As Found: Alcohol Use Disorder - ID: M12244 - Name: Opioid-Related Disorders - Relevance: HIGH - As Found: Opioid Use Disorder - ID: M21842 - Name: Alcohol-Related Disorders - Relevance: LOW - As Found: Unknown - ID: M21837 - Name: Substance-Related Disorders - Relevance: LOW - As Found: Unknown - ID: M30302 - Name: Chemically-Induced Disorders - Relevance: LOW - As Found: Unknown - ID: M4815 - Name: Mental Disorders - Relevance: LOW - As Found: Unknown - ID: M14473 - Name: Psychotic Disorders - Relevance: LOW - As Found: Unknown - ID: M2057 - Name: Narcotic-Related Disorders - Relevance: LOW - As Found: Unknown - ID: M1557 - Name: Drug Misuse - Relevance: LOW - As Found: Unknown - ID: M30133 - Name: Prescription Drug Misuse - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000000437 - Term: Alcoholism - ID: D000009293 - Term: Opioid-Related Disorders - ID: D000083682 - Term: Opiate Overdose ### Intervention Browse Module - Browse Branches - Abbrev: PhSol - Name: Pharmaceutical Solutions - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Analg - Name: Analgesics - Abbrev: CNSDep - Name: Central Nervous System Depressants ### Intervention Browse Module - Browse Leaves - ID: M21860 - Name: Pharmaceutical Solutions - Relevance: LOW - As Found: Unknown - ID: M4033 - Name: Analgesics, Opioid - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02545179 **Brief Title:** Web-based Daily Care Training of Children With Cerebral Palsy **Official Title:** Ethics Committee of the University of Social Welfare and Rehabilitation Science #### Organization Study ID Info **ID:** IR.USWR.REC.1394.111 #### Organization **Class:** OTHER **Full Name:** University of Social Welfare and Rehabilitation Science ### Status Module #### Completion Date **Date:** 2016-12 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** NOT_YET_RECRUITING #### Last Update Post Date **Date:** 2015-09-10 **Type:** ESTIMATED **Last Update Submit Date:** 2015-09-09 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2016-01 **Type:** ESTIMATED #### Start Date **Date:** 2015-09 **Status Verified Date:** 2015-09 #### Study First Post Date **Date:** 2015-09-09 **Type:** ESTIMATED **Study First Submit Date:** 2015-09-07 **Study First Submit QC Date:** 2015-09-07 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Social Welfare and Rehabilitation Science #### Responsible Party **Investigator Affiliation:** University of Social Welfare and Rehabilitation Science **Investigator Full Name:** Zahra Nobakht **Investigator Title:** PhD student, occupational therapy department,University of Social Welfare and Rehabilitation Science **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Purpose:Effect of web-based daily care training on the quality of life of caregivers of children with cerebral palsy. study design: Single-blind clinical trial. study population: Caregivers of children with cerebral palsy. Inclusion criteria: caregiver -Mother of a child with cerebral palsy who spent most time on child care- Children with cerebral palsy aged 4 to 12 years old living in Alborz province that clinical diagnosis of cerebral palsy registered in their clinical records. Moderate and severe cerebral palsy (gross motor function levels 3 to 5). caregiver have facilities and basic knowledge of using Internet (Declared access to the Internet at least one hour a week). exclusion criteria: taking care of two or more children with disability. participated in previous face to face training programs. Sample size: 100. Intervention: Web-based daily care training. intervention time: 12-week. Outcome of the study: the quality of life of caregivers **Detailed Description:** step1: web development and design 1. development of educational content (4 weeks) 2. Create a paper version of the site (2 weeks) 3. Create a web-based version of the site (4 weeks) 4. pilot study (6 weeks) step 2: RCT (12 weeks) ### Conditions Module **Conditions:** - Cerebral Palsy ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 100 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** web-based daily care training 12 week interactive web based caring training education **Intervention Names:** - Other: web-based daily care training **Label:** intervention **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Previous therapy **Label:** control **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - intervention **Description:** 12 week interactive web based caring training education **Name:** web-based daily care training **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** quality of life of caregivers **Time Frame:** 3 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * caregiver_ Mother of a child with cerebral palsy who spent most time on child care Children with cerebral palsy aged 4 to 12 years old living in Alborz province that clinical diagnosis of cerebral palsy registered in their clinical records * Moderate and severe cerebral palsy (gross motor function levels 3 to 5) * caregiver have facilities and basic knowledge of using Internet (Declared access to the Internet at least one hour a week) Exclusion Criteria: * taking care of two or more children with disability * participated in previous training programs **Sex:** FEMALE **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** nobakht.zahra@gmail.com **Name:** Zahra Nobakht, PhD student **Phone:** 00982122180037 **Role:** CONTACT #### Overall Officials **Official 1:** **Affiliation:** Occupational therapy department, University of Social Welfare and Rehabilitation Sciences **Name:** Zahra Nobakht, PhD student **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001925 - Term: Brain Damage, Chronic - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M5796 - Name: Cerebral Palsy - Relevance: HIGH - As Found: Cerebral Palsy - ID: M13157 - Name: Paralysis - Relevance: LOW - As Found: Unknown - ID: M5207 - Name: Brain Injuries - Relevance: LOW - As Found: Unknown - ID: M5202 - Name: Brain Damage, Chronic - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002547 - Term: Cerebral Palsy ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06268379 **Brief Title:** BMA and Dynamic Nomogram for Survival Prediction in Patients With CRC **Official Title:** Developing a Clinician-friendly Online Tool for Survival Prediction in Colon Cancer Patients: A Bayesian Model Averaging for Risk Factor Selection and Dynamic Nomogram #### Organization Study ID Info **ID:** #08-01-03-23 #### Organization **Class:** OTHER **Full Name:** Cabrini Health ### Status Module #### Completion Date **Date:** 2021-12-15 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-02-20 **Type:** ACTUAL **Last Update Submit Date:** 2024-02-13 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2021-12-15 **Type:** ACTUAL #### Start Date **Date:** 2010-02-15 **Type:** ACTUAL **Status Verified Date:** 2024-02 #### Study First Post Date **Date:** 2024-02-20 **Type:** ACTUAL **Study First Submit Date:** 2024-02-13 **Study First Submit QC Date:** 2024-02-13 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Cabrini Health #### Responsible Party **Investigator Affiliation:** Cabrini Health **Investigator Full Name:** Mohammad Asghari Jafarabadi **Investigator Title:** A/Prof Mohamad Asghari Jafarabadi **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** This project will examine the outstanding statistical techniques for predicting the survival of patients with colorectal cancer (CRC) (colorectal neoplasia database). The motivating clinical question that led to proposing this project is based on the general assumption that: "Right-sided colorectal cancer (CRC) has worse survival than left-sided CRC." The question is, which aspects of the patient's characteristics are responsible for this difference? This led us to BMA model selection and provide a clinician-friendly online nomogram. **Detailed Description:** Translational statistics merges biostatistics and clinical research to communicate research findings effectively. Nomograms, graphical representations integrating independent prognostic factors, are valuable tools in colorectal cancer (CRC) research. Bayesian models for variable selection in survival outcome prediction offer advantages through Bayesian model averaging (BMA). This study aimed to utilise BMA for variable selection and develop a clinician-friendly online dynamic nomogram for survival prediction. A retrospective study utilised the Cabrini Monash colorectal neoplasia database, including colon cancer patients who underwent surgery. Data on demographics, perioperative risks, treatment details, mortality, morbidity, and survival were collected. BMA was employed for Bayesian variable selection to identify effective risk factors for survival prediction. Sensitivity analyses using Cox-LASSO and imputation of missing data were performed. Prognostic online dynamic nomograms were constructed using selected risk factors and the R-package DynNom. ### Conditions Module **Conditions:** - Colon Cancer - Model Disease **Keywords:** - Colorectal cancer; Translational statistics; Online dynamic nomogram; Bayesian variable selection; Survival prediction ### Design Module #### Design Info **Observational Model:** OTHER **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 2475 **Type:** ACTUAL **Patient Registry:** True **Study Type:** OBSERVATIONAL **Target Duration:** 11 Years ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** Not an interventional study, it is an observational, longitudinal study. **Name:** Surgery **Other Names:** - Observational study **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** Overall Survival, time from sugary to death or last follow up **Measure:** OS **Time Frame:** 2011-2021 **Description:** Relapse-free Survival, time from sugary to death or last follow up for those without relapse. **Measure:** RFS **Time Frame:** 2011-2021 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: In this study, patients were included based on specific selection criteria: being 18 years old or older, having a diagnosis of colon adenocarcinoma (or post polypectomy of the same condition), and having undergone surgery for colon cancer. Exclusion Criteria: Patients with rectal cancer, neuroendocrine tumours, lymphomas and those who underwent trans-anal surgery were not included in the study. **Maximum Age:** 101 Years **Minimum Age:** 22 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** A retrospective study was conducted with the Cabrini Monash Colorectal Neoplasia Database 15. This prospectively maintained database includes data from both private (Cabrini) and public (The Alfred) hospitals in Melbourne, Australia. The study focused on patients who underwent surgery for colon cancer from January 2010 to December 2021. ### Contacts Locations Module #### Locations **Location 1:** **City:** Melbourne **Country:** Australia **Facility:** Cabrini Health **State:** Victoria **Zip:** 3144 ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Sung H, Ferlay J, Siegel RL, Laversanne M, Soerjomataram I, Jemal A, Bray F. Global Cancer Statistics 2020: GLOBOCAN Estimates of Incidence and Mortality Worldwide for 36 Cancers in 185 Countries. CA Cancer J Clin. 2021 May;71(3):209-249. doi: 10.3322/caac.21660. Epub 2021 Feb 4. **PMID:** 33538338 **Citation:** Siegel RL, Miller KD, Goding Sauer A, Fedewa SA, Butterly LF, Anderson JC, Cercek A, Smith RA, Jemal A. Colorectal cancer statistics, 2020. CA Cancer J Clin. 2020 May;70(3):145-164. doi: 10.3322/caac.21601. Epub 2020 Mar 5. **PMID:** 32133645 **Citation:** Jalali A, Alvarez-Iglesias A, Roshan D, Newell J. Visualising statistical models using dynamic nomograms. PLoS One. 2019 Nov 15;14(11):e0225253. doi: 10.1371/journal.pone.0225253. eCollection 2019. **PMID:** 31730633 **Citation:** Borumandnia N, Doosti H, Jalali A, Khodakarim S, Charati JY, Pourhoseingholi MA, Talebi A, Agah S. Nomogram to Predict the Overall Survival of Colorectal Cancer Patients: A Multicenter National Study. Int J Environ Res Public Health. 2021 Jul 21;18(15):7734. doi: 10.3390/ijerph18157734. **PMID:** 34360026 **Citation:** Maity AK, Basu S, Ghosh S. Bayesian criterion-based variable selection. Journal of the Royal Statistical Society Series C: Applied Statistics. 2021;70(4):835-857. ## Derived Section ### Condition Browse Module - Ancestors - ID: D000015179 - Term: Colorectal Neoplasms - ID: D000007414 - Term: Intestinal Neoplasms - ID: D000005770 - Term: Gastrointestinal Neoplasms - ID: D000004067 - Term: Digestive System Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000004066 - Term: Digestive System Diseases - ID: D000005767 - Term: Gastrointestinal Diseases - ID: D000003108 - Term: Colonic Diseases - ID: D000007410 - Term: Intestinal Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M6338 - Name: Colonic Neoplasms - Relevance: HIGH - As Found: Colon Cancer - ID: M17890 - Name: Colorectal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M10448 - Name: Intestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M8886 - Name: Gastrointestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7256 - Name: Digestive System Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M6336 - Name: Colonic Diseases - Relevance: LOW - As Found: Unknown - ID: M10444 - Name: Intestinal Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000003110 - Term: Colonic Neoplasms ### Intervention Browse Module - Browse Branches - Abbrev: HB - Name: Herbal and Botanical - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: T120 - Name: Cola - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00864279 **Brief Title:** A Relative Bioavailability Study of Cetirizine HCl 10 mg Tablets Under Fasting Conditions **Official Title:** Single Dose Two-Way Crossover Fasted Bioequivalence Study of Cetirizine Hydrochloride 10 mg Tablets in Healthy Volunteers #### Organization Study ID Info **ID:** AAI-US-432 #### Organization **Class:** INDUSTRY **Full Name:** Actavis Inc. ### Status Module #### Completion Date **Date:** 2006-09 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2010-08-16 **Type:** ESTIMATED **Last Update Submit Date:** 2010-08-13 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2006-09 **Type:** ACTUAL #### Start Date **Date:** 2006-08 **Status Verified Date:** 2010-08 #### Study First Post Date **Date:** 2009-03-18 **Type:** ESTIMATED **Study First Submit Date:** 2009-03-17 **Study First Submit QC Date:** 2009-03-17 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Actavis Inc. #### Responsible Party **Old Name Title:** Meena Venugopal, Director, Clinical R&D **Old Organization:** Actavis Inc ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** To determine the pharmacokinetics and bioequivalence of cetirizine hydrochloride formulations after administration of single doses to normal healthy subjects under fasted conditions. These data were to be evaluated statistically to determine if the products meet bioequivalence criteria. **Detailed Description:** Study Type: Interventional Study Design: Single-dose two-way, crossover bioequivalence study with an adequate washout period (7 days) between the two periods of the study and with an equal number of subjects randomly assigned to receive the study test (Treatment A) and study reference (Treatment B). Official Title: Single Dose Two-Way Crossover Fasted Bioequivalence Study of Cetirizine Hydrochloride 10 mg Tablets in Healthy Volunteers Further study details as provided by Actavis Elizabeth LLC: Primary Outcome Measures: Rate and Extend of Absorption ### Conditions Module **Conditions:** - Healthy **Keywords:** - Bioequivalence - Cetirizine - Healthy subjects ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER ##### Masking Info **Masking:** NONE #### Enrollment Info **Count:** 26 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Cetirizine Hydrochloride 10 mg tablets, single dose **Intervention Names:** - Drug: Cetirizine Hydrochloride 10 mg tablets, single dose **Label:** A **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Zyrtec® 10 mg tablets, single dose **Intervention Names:** - Drug: Zyrtec® 10 mg tablets, single dose **Label:** B **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - A **Description:** A: Experimental Subjects received Shasun Chemicals and Drugs Ltd. formulated products under fast conditions **Name:** Cetirizine Hydrochloride 10 mg tablets, single dose **Other Names:** - Cetirizine **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - B **Description:** B: Active comparator Subjects received Pfizer Inc. formulated products under fast conditions **Name:** Zyrtec® 10 mg tablets, single dose **Other Names:** - Cetirizine **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Rate and Extend of Absorption **Time Frame:** 48 hours ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Healthy subjects at least 18 years of age. 2. Informed of the nature of the study and provide their written informed consent. 3. Have a body mass index2 between 18 and 30 and weighing at least 110 pound. 4. In good health as determined by lack of clinically significant abnormalities in health assessments performed at screening as judged by the physician. Exclusion Criteria: 1. Hypersensitivity to cetirizine hydrochloride (Zyrtec®) or related compounds. 2. Conditions that affect the absorption, metabolism or passage of drugs out of the body, e.g., sprue, celiac disease, Crohn's disease, colitis, liver, kidney or thyroid conditions. 3. Recent history (within 1 year) of mental illness, drug addiction, drug abuse or alcoholism. 4. A hematocrit value of ≤ 33.0 % for females and ≤ 37.0 % for males. 5. Donation of greater than 500 ml of blood in the past 4 weeks prior to study dosing or difficulty in donating blood. 6. Received an investigational drug within the 4 weeks prior to study dosing. 7. Currently taking any systemic prescription medications, except for oral/cutaneous/vaginal hormone contraceptives, within the 7 days prior to study dosing or over-the-counter medication within 3 days of study dosing. This prohibition does not include vitamins or herbal preparations taken as nutritional supplements for non-therapeutic indications as judged by the attending physician. Any nonprescription medication consumption reported will be reviewed by the investigator prior to dosing. At the discretion of the investigator these volunteers may be enrolled if the medication is not anticipated to alter study integrity. 8. Regular smoking of more than 5 cigarettes weekly or the regular daily use of nicotine-containing products beginning 3 months before study medication administration through the final evaluation. 9. If female, the subject is lactating or has a positive pregnancy test at screening and prior to each of the treatment periods. Females must use a medically acceptable method of contraception throughout the entire study period and for one week after the study is completed. Medically acceptable methods of contraception that may be used by the subject and/or her partner are: oral contraceptives/patches, progestin injection or implants, condom with spermicide, diaphragm with spermicide, IUD, vaginal spermicidal or hormonal suppository, surgical sterilization of themselves or their partner(s) or abstinence. Females taking oral contraceptives/patches must have taken them consistently for at least three months prior to receiving study medication. 10. Grapefruit beverages or foods beginning 7 days before each study medication administration and alcohol, caffeine or xanthine beverages or foods beginning 24 hours before each study medication administration through the last PK sample of each period. Such restricted items include coffee, tea, iced tea, Coke®, Pepsi®, Mountain DeW®, chocolate, brownies, etc. 11. Regular use of any drugs known to induce or inhibit hepatic drug metabolism (examples include barbiturates, carbamazepine, rifampin, phenylhydantoins, phenothiazines, cimetidine,omeprazole, macrolides, imidazoles, fluoroquinolones) within 30 days prior to study administration. 12. Positive test results for: HIV, Hepatitis B surface antigen, Hepatitis C antibody at screening. 13. Positive test results for: drugs of abuse or pregnancy at screening and prior to each dosing period. **Healthy Volunteers:** True **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Morrisville **Country:** United States **Facility:** AAIPharma Inc.- AAI Clinic **State:** North Carolina **Zip:** 27560 #### Overall Officials **Official 1:** **Affiliation:** AAI Clinic (AAIPharma Inc.) **Name:** Evin H. Sides III,, MD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Intervention Browse Module - Ancestors - ID: D000018926 - Term: Anti-Allergic Agents - ID: D000039563 - Term: Histamine H1 Antagonists, Non-Sedating - ID: D000006634 - Term: Histamine H1 Antagonists - ID: D000006633 - Term: Histamine Antagonists - ID: D000018494 - Term: Histamine Agents - ID: D000018377 - Term: Neurotransmitter Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: AAll - Name: Anti-Allergic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M19620 - Name: Cetirizine - Relevance: HIGH - As Found: Hearing loss - ID: M20962 - Name: Anti-Allergic Agents - Relevance: LOW - As Found: Unknown - ID: M9710 - Name: Histamine H1 Antagonists - Relevance: LOW - As Found: Unknown - ID: M9708 - Name: Histamine - Relevance: LOW - As Found: Unknown - ID: M212144 - Name: Histamine phosphate - Relevance: LOW - As Found: Unknown - ID: M24826 - Name: Histamine H1 Antagonists, Non-Sedating - Relevance: LOW - As Found: Unknown - ID: M9709 - Name: Histamine Antagonists - Relevance: LOW - As Found: Unknown - ID: M20504 - Name: Neurotransmitter Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000017332 - Term: Cetirizine ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04237779 **Brief Title:** The Effects of Intratesticular PRP Injection in Men With Azoospermia or Cryptozoospermia **Official Title:** The Effects of Intratesticular PRP Injection #### Organization Study ID Info **ID:** ATADEK 2019-4/23 #### Organization **Class:** OTHER **Full Name:** Acibadem University ### Status Module #### Completion Date **Date:** 2020-11-21 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-11-24 **Type:** ACTUAL **Last Update Submit Date:** 2020-11-21 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-10-31 **Type:** ACTUAL #### Start Date **Date:** 2020-01-27 **Type:** ACTUAL **Status Verified Date:** 2020-11 #### Study First Post Date **Date:** 2020-01-23 **Type:** ACTUAL **Study First Submit Date:** 2020-01-12 **Study First Submit QC Date:** 2020-01-19 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Acibadem University #### Responsible Party **Investigator Affiliation:** Acibadem University **Investigator Full Name:** Bulent Tiras **Investigator Title:** Prof. **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The study will be conducted in men with a diagnosis of non-obstructive azoospermia or cryptozoospermia after obtaining written informed consent. The diagnosis of azoospermia and cryptozoospermia will be based on two semen analyses performed at least 15 days apart, followed by a documented micro testicular sperm extraction (TESE). A detailed history will be obtained, physical examination and laboratory evaluation will be performed prior to treatment. In physical examination, testicular volumes will be evaluated using an orchiometer. Serum FSH and testosterone values will be determined. PRP will be prepared by centrifugation of approximately 20 ml autologous blood obtained by phlebotomy. PRP (3 ml) will be administered into the seminiferous tubule or interstitial space of each testis. Sperm analysis, testicular volume (using orchiometer), serum FSH and testosterone levels will be reevaluated at 8 weeks post-procedure. Micro TESE will performed on the third month after PRP procedure. **Detailed Description:** Platelet-rich plasma (PRP) is a unique autologous agent derived from blood that is rich in growth factors, cytokines, and hormones; it has been reported to reduce oxidative stress and reactive oxygen species generation and upregulate the expression of various antioxidant enzymes. PRP is used in a variety of clinical applications in cell therapy. Based on World Health Organization guidelines, azoospermia is diagnosed when the absence of sperm is observed in two semen samples. Based on World Health Organization (WHO), cryptozoospermia is diagnosed when spermatozoa cannot be observed in a fresh semen sample; however, it is found after an extended centrifugation and microscopic search. All patients require a clinical work-up with physical examination, endocrine evaluation (follicle stimulating hormone (FSH), luteinizing hormone (LH) and testosterone) and genetic analysis. Scrotal and transrectal ultrasounds will be performed as indicated. Platelet-rich plasma (PRP), with its rich growth factor composition, has proven beneficial in regenerative therapy. The potential therapeutic role of PRP has not been studied in testis for non-obstructive azoospermia and cryptozoospermia. The investigators will investigate sperm parameters in non-obstructive azoospermic and crypotozoospermic patients treated with PRP. ### Conditions Module **Conditions:** - Azoospermia, Nonobstructive ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 200 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Autologous blood obtained from peripheral vein will be used to prepare PRP following standard protocols. PRP injection will be performed under local anesthesia. Sperm analysis, testicular volumes (using orchiometer), serum FSH and testosterone measurements will be re-evaluated at 8 weeks post-procedure. On the third month after the procedure, presence of spermatozoa will be reassessed in microTESE material. **Intervention Names:** - Biological: PRP injection into at least one testis **Label:** PRP injection into at least one testis **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - PRP injection into at least one testis **Description:** Autologous blood obtained from peripheral vein will be used to prepare PRP following standard protocols. PRP injection will be performed under local anesthesia. Sperm analysis, testicular volumes (using orchiometer), serum FSH and testosterone measurements will be re-evaluated at 8 weeks post-procedure. On the third month after the procedure, presence of spermatozoa will be reassessed in micro testicular sperm extraction (TESE) material. **Name:** PRP injection into at least one testis **Type:** BIOLOGICAL ### Outcomes Module #### Primary Outcomes **Description:** Number of participants with testicular PRP treatment as assessed by testicular sperm extraction, change from azoospermia or cryptozoospermia to presence of sperm during TESE procedure **Measure:** sperm formation **Time Frame:** 6 months #### Secondary Outcomes **Description:** Number of participants with testicular PRP after TESE as assessed by number of clinical pregnancies, change in the clinical pregnancy rates with IVF treatment. **Measure:** IVF outcomes **Time Frame:** 12 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Azoospermia in at least 2 prior semen analysis. * Diagnosis of Non-Obstructive Azoospermia or Diagnosis of Cryptozoospermia Exclusion Criteria: * Obstructive Azospermia * Anatomical abnormalities in the genital tract, * Cancer, * Hepatitis * Patients with systemic medical problems * Patients with chromosomal disorders **Gender Based:** True **Gender Description:** Azoospermia in at least 2 prior semen analysis. Diagnosis of Non-Obstructive Azoospermia or Diagnosis of Cryptozoospermia **Maximum Age:** 65 Years **Minimum Age:** 25 Years **Sex:** MALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Istanbul **Country:** Turkey **Facility:** Acıbadem Maslak Hospital IVF Unit #### Overall Officials **Official 1:** **Affiliation:** Acibadem University **Name:** Yigit Cakiroglu, Assoc.Prof. **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Dhurat R, Sukesh M. Principles and Methods of Preparation of Platelet-Rich Plasma: A Review and Author's Perspective. J Cutan Aesthet Surg. 2014 Oct-Dec;7(4):189-97. doi: 10.4103/0974-2077.150734. **PMID:** 25722595 **Citation:** Dehghani F, Sotoude N, Bordbar H, Panjeshahin MR, Karbalay-Doust S. The use of platelet-rich plasma (PRP) to improve structural impairment of rat testis induced by busulfan. Platelets. 2019;30(4):513-520. doi: 10.1080/09537104.2018.1478400. Epub 2018 Jun 8. **PMID:** 29883240 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007248 - Term: Infertility, Male - ID: D000005832 - Term: Genital Diseases, Male - ID: D000091662 - Term: Genital Diseases - ID: D000091642 - Term: Urogenital Diseases - ID: D000007246 - Term: Infertility - ID: D000052801 - Term: Male Urogenital Diseases ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M27441 - Name: Azoospermia - Relevance: HIGH - As Found: Azoospermia - ID: M10290 - Name: Infertility - Relevance: LOW - As Found: Unknown - ID: M10292 - Name: Infertility, Male - Relevance: LOW - As Found: Unknown - ID: M2876 - Name: Genital Diseases - Relevance: LOW - As Found: Unknown - ID: M8944 - Name: Genital Diseases, Male - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000053713 - Term: Azoospermia ### Intervention Browse Module - Browse Branches - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown - ID: M16509 - Name: Testosterone - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06001879 **Acronym:** BLS-NEN-HCP **Brief Title:** The Study Focuses on Training Newly Employed Nurses With Two Groups Interventional (Simulation Training) & Control (Brochure) Group Using BLS -AHA 2020 Using Simulation, the Test Includes Pre-test & 2 Post-test Surveys to Assess Knowledge, Practice & Confidence Level. **Official Title:** Protocol Title : Development, Validation, and Evaluation of the Effectiveness of Simulation in Basic Life Support Training (SBLST) on Newly Employed Nurses in Governmental Jordanian Hospitals. #### Organization Study ID Info **ID:** JEPeM Code :USM/JEPeM/22110681 #### Organization **Class:** OTHER **Full Name:** Universiti Sains Malaysia ### Status Module #### Completion Date **Date:** 2024-06-01 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-08-21 **Type:** ACTUAL **Last Update Submit Date:** 2023-08-16 **Overall Status:** ACTIVE_NOT_RECRUITING #### Primary Completion Date **Date:** 2023-06-08 **Type:** ACTUAL #### Start Date **Date:** 2022-11-22 **Type:** ACTUAL **Status Verified Date:** 2023-08 #### Study First Post Date **Date:** 2023-08-21 **Type:** ACTUAL **Study First Submit Date:** 2023-07-22 **Study First Submit QC Date:** 2023-08-16 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** yousef Shukry Mohammad Abu-Wardeh #### Responsible Party **Investigator Affiliation:** Universiti Sains Malaysia **Investigator Full Name:** yousef Shukry Mohammad Abu-Wardeh **Investigator Title:** PHD STUDENTS aCUTE AND CRITICAL CARE IN NURSING **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** The general objective of this study is to develop, validate and evaluate the effectiveness of simulation in basic life support training (SBLST) among newly employed nurses in Jordanian governmental hospitals; the study design is a basic experimental study design, randomized control trial (RCT) design, the dependant variables measure in this study; knowledge, practice and confidence by using a pre-test and two follow up tests, two groups are participating in this study; experimental and control group. The control group treatment is the standard intervention (brochure), and the experimental group intervention is a simulation in basic life support training (SBLST). The study process includes four steps * Perform the pre-test (assess knowledge, practice confidence surveys * Education intervention knowledge and practice * Perform the post-test 1 (assess knowledge, practice confidence surveys * Perform post-test 2 (assess knowledge, practice confidence surveys **Detailed Description:** The general objective of this study is to develop, validate and evaluate the effectiveness of simulation in basic life support training (SBLST) among newly employed nurses in Jordanian governmental hospitals; the study design is a basic experimental study design, randomized control trial (RCT) design, the dependant variables measure in this study; knowledge, practice and confidence by using a pre-test and two follow up tests, two groups are participating in this study; experimental and control group. The control group treatment is the standard intervention (brochure), and the experimental group intervention is a simulation in basic life support training (SBLST); researchers' hypothesized that there are no significant differences between the control and experimental group in the inclusion criteria and pre-test score; furthermore, the simulation in basic life support training intervention significantly improves knowledge, practice and confidence level among newly employed nurses (NEN) in Jordanian governmental hospitals; moreover, the is a significant difference between control and experimental group in the post-test scores in all dependant variables. The study process include four steps * Perform the pre-test (assess knowledge, practice confidence surveys * Education intervention knowledge and practice * Perform the post-test 1 (assess knowledge, practice confidence surveys * Perform post-test 2 (assess knowledge, practice confidence surveys Study title: DEVELOPMENT, VALIDATION, AND EVALUATION OF THE EFFECTIVENESS OF SIMULATION IN BASIC LIFE SUPPORT TRAINING (SBLST) ON NEWLY EMPLOYED NURSES IN GOVERNMENTAL JORDANIAN HOSPITALS Research Hypotheses i. The simulation in basic life support training intervention effectively improves knowledge among newly employed nurses (NEN) in Jordanian governmental hospitals. ii. The simulation in basic life support training intervention effectively improves practice among newly employed nurses (NEN) in Jordanian governmental hospitals. iii. The simulation in basic life support training intervention effectively improves the confidence level among newly employed nurses (NEN) in Jordanian governmental hospitals. iv. There is no significant difference in means between the pre-test among the newly employed nurses (NEN) among interventional and the control groups in all dependent variables. v. There is a significant mean difference between PRE-SBLST and all POST-SBLST results among NEN in all dependent variables. Study Design The study design was a prospective, longitudinal, single-blind basic experimental design, randomized control trial (RCT) design. Study Location The study was carried out in Jordan, Amman and Al-Zarqa City. The accessible population was NEN in five hospitals. The researchers selected three Jordanian hospitals. The selection was according to the capacity, capability of the institution and eligibility criteria. Computer-generated randomization (Random Allocation Software, version 1.0) was applied in blocks to allocate the hospitals by distributing them into two arms. The first arm was the control group; selected from three hospitals; Prince Hamza Hospital, AL-Basheer Hospital, and AL-Zarqa Governmental Hospital (n=51); the second arm was the interventional group from two hospitals; Prince Faisal Governmental Hospital and Dr.Jameel AL-Totanji Hospital. Randomization of the Hospitals To prevent data contamination between the control and interventional groups, computer-generated randomization (Random Allocation Software, version 1.0) was applied in blocks to allocate the hospitals by distributing them into two arms. The first arm was the control group; selected from three hospitals; Prince Hamza Hospital, AL-Basheer Hospital, and AL-Zarqa Governmental Hospital (n=51); the second arm was the interventional group from two hospitals; Prince Faisal governmental hospital and Dr.Jameel AL-Totanji hospital. The control and interventional groups were selected from different hospitals because the researchers are always worried about data contamination and try to minimize it between the control and intervention groups; if the sample was selected from the same area and both groups worked closely together, the data would be contaminated. The study consists of a pre-test, intervention, immediate post-test-1 after the intervention, and post-test-2 three months after the intervention. This study measured knowledge, practice, and confidence through pre- and post-tests in control and experimental groups. This study used two interventions: SBLST for the interventional group and AHA-BLS 2020 brochure as a standard treatment for the control group. Data collection proceeded using the Google Form Platform (A web-based instrument Copyrighted by Google application). The evaluation process includes a pre-test and two follow-up assessments to measure knowledge, practice and confidence. The researcher will analyze the means of each test; the total dependent variables were 30 questions. Research Instruments Languages of the Tools According to the HCP educational level, all four sections of the tools assessment are written in English; furthermore, the researchers' assistance clarified any misunderstanding points or need for translation to the participants. Tools Sections The tools consist of five sections; research information sheet and consent forms, demographical data, knowledge assessment tools, skills assessment tools, and confidence level assessment tools. Research information sheet and Consent Forms The research information sheet is a piece of brief researcher information and contact details; it also includes subject information and a consent form with an area for the participant's signature when they are approved to participate in this study. Furthermore, the research information sheets include a participant's publication consent form. Demographical data Demographic parts are attached; these data consist of dichotomous questions (Yes\\NO), nominal data, ordinal data, and interval measurement questions. Nominal measurements include sex and ordinal assessment as educational level and area of experience; interval assessment questions include age and work experience. Dichotomous assessments (Yes\\NO), asking if the participant previously participated in a CPR episode or observed any CPR before, if the participants had finished training the BLS using simulation previously, and if the participant gained the certification of BLS training from formal and accredited institutions. Knowledge and practice Nursing knowledge is the interaction of science and research to improve practice. Researchers use a 13-question MCQ tool to assess knowledge. Nursing practice is performing the knowledge learned before; the researchers assessed skills using a tool consisting of ten MCQ. The participants got zero for each wrong answer and one mark for each correct answer. Confidence Confidence is the building and accomplishment of the nursing profession through knowledge acquisition, skills, and critical thinking. Confidence is the positive feeling of faith and dependability to perform BLS without fear and free from risk to the patient. The researchers assessed confidence level using seven statements rated as a dropdown in percent value from the lowest value of 5% to the highest value of 100%. Study process steps 1. Pre-test After NEN enrolled according to the inclusion criteria for both the control group and experimental group (n= 102), the participants started by signing the informed consent, filled out the demographical data and pre-test as proactive steps before the intervention; many sessions of pre-test data collection and intervention were performed according to the availability of NEN, the arrangement of the hospital's nursing director. The estimated time to fill in the demographical data and pre-test is around 30 minutes. Oermann et al. (2020) addressed that the trainer performs a pre-test assessment to assess the effectiveness of SBLST. The investigators discussed the purpose of the study with the control group and explained to them the procedure to prevent any data contamination and minimize the interpretation of the survey content by the other research assistants. 2. Intervention The control group read the brochure containing a brief guideline about basic life support for 30-60 minutes before taking the post-test. Standard treatment ran from Dec 2022 to Jan 2023. One full day for SBLST intervention, from 5 to 7 hours for SBLST intervention, is required based on the recent research and uses AHA-2020 guidelines. The number of participants was ten NEN in each session. The interventional treatment consists of theoretical and practical parts. The SBLST intervention was conducted in an educational lab according to the hospital's capability; SBLST ran for seven days, from 22 Nov 2022 to 20-Dec, 2022. Researchers combined two frameworks in the SBLST. Miller's Pyramid and Kolb's Cycle; Miller's Pyramid focuses on teaching theoretical and practical aspects of the task through simulation and allowing trainees to perform the procedure independently with guidance; Kolb's Cycle suggests giving learners a scenario to practice until they eliminate errors before moving to another scenario. The SBLST intervention was reviewed by three experts in BLS and approved by the Jordanian Ministry of Health-Life Support Center. It was found to be compliant, safe and covered all aspects of AHA-2020. English experts provided feedback to improve clarity. A pilot study was conducted on 20 nurses to calculate SBLST time, identify errors, monitor the progress and calculate Cronbach's Alpha.A pilot study was performed to check the stability of the tools. Cronbach's alpha of knowledge and skills assessment tools was 0.748, reflecting the suitability and strength of using these tools. Moreover, Cronbach's alpha of confidence tools was 0.731, reflecting the suitability and stability of the confidence tools. Adult half-body manikin and a pediatric full-body manikin are necessary; with lung bag inflation and deflation characteristics, palpable carotid pulse, the chest includes spring inside to facilitate chest recoil, and chest compression board, bag-valve-mask-ventilation, chest compression performed on the Charlie simulator to relieve choking. The principles investigator (PI) had a master's degree in critical care nursing, eighteen years of experience between ICUs, lecturers and clinical instructors, valid BLS and ACLS, training of trainers in nursing education, and randomized control trial training. The facilitator must have CPR certifications in BLS and ACLS, enough expertise in education and training, and good communication skills. The investigator had a master's degree in critical care. Two research assistants supported the PI in collecting data; one research assistant for the control group and the other for the interventional group. 3. post-test All participants completed the immediate post-test-1 after the interventions. The post-tets-2 was conducted by providing the participants with a Google Form Platform link through their phone and email addresses. The post-test after three months evaluates whether the participants' level stayed at the same level or minimized by comparing the mean value between all post-tests. Forty-eight participants in the experimental group completed post-test-2 with a response rate of 94%, and 45 participants in the control group with a response rate of 88%. Sample Size Estimation Sample size calculation is performed by using G\*POWER software (version 3.1); sample size calculation is summarized and consists of the following steps (1) Select the test family (F-test family), (2) Select the statistical test (Select ANOVA-repeated test, between factors), the preliminary test analysis the researchers used in this study, the rationale for using the Select ANOVA-repeated test between factors due to measuring many dependent variables means at different time points. (3) The parameters selected to calculate sample size include one side tail, alpha (α) and equals (0.05)- type one error, Power (P) and equal (0.8), effect size, the number of groups is two (each group = n\\2) n= the estimated sample size, number of measurements were four measurements (Pre-test, Post-test immediately, Post-test after two months, and Post-test after three months). Effect Size (ES) = 0.26, automatically calculated by G\*Power software, depending on the sample size of the previous study, the effect size is calculated, the Control group's post-test result M±SD was equal (25.03±3.04) with a sample size of 28 participants, and the interventional group (26.64±2.64) with sample size 29 participants. The sample size is 72 participants; because the research study continued for three months, the invistigators added a 10% drop rate for each month, so the drop rate is 30%. Final sample size estimation including drop rate (n\\1-dropout rate) = 72/(1-0.30) = 102 participants, the intervention group arm is 51 participants, and The largest sample size was obtained from the interventional group, more than the Control Group and the Control group arm 51 participants. Simple Random Sampling of the Participants After obtaining ethical approval and permission to conduct the study from the Ministry of Health in Jordan, approval letter (Education/Info\\ 15177) and ethical approval letter (MOH/REC/2022/340), The researchers started the formal face-to-face visits to arrange with the nursing director and a continuous education office in the selected hospitals about the study objective and how the study would proceed; The researchers received the activation letter to initiate the study and prepared the list of the available participants who met the study's eligibility criteria. The number of participants depends on the availability of newly employed nurses in the institution who meet the inclusion criteria. The researchers randomly selected the participants from the participants' list by randomly sampling from newly employed nurses in each hospital for interventional and control groups. . The response of the control group hospitals was as the following; the nursing director and continuous education in AL-Zarqa Governmental Hospital responded by providing all the information about participants and selecting a time and day to perform the Pre-test, standard treatment and the immediate post-test. Prince Hamzah Hospital and AL-Basheer Hospital gave the researcher the names of departments in the hospital, including those with NEN, and the invistigators visited these departments to select the available NEN randomly and performed Pre-test, standard treatment, and immediate post-test in their departments. The response of the interventional group hospitals was as the following; Prince Faisal Governmental Hospital and Dr.Jameel AL-Totanji Hospital scheduled many days and times to perform the intervention, and the hospitals arranged with the available participants who met the eligibility criteria; then, the researcher selected the participants randomly. Ethical Considerations 1. Permission to Conduct the Study Regarding ethical considerations, approval for the study was obtained from the Human Research Ethics Committee (HREC), Universiti Sains Malaysia (USM). Moreover, ethical approvals to initiate SBLST intervention in Jordanian Hospitals in Amman and AL-Zarqa city were obtained from the Ministry of Health in Jordan; the study protocol was revised and approved for implementation by Jawatankuasa Etika Penyilidikan Manusia Universiti Sains Malaysia (JEPeM-USM) with study protocol code USM/JEPeM/22110681, which compliance with the Declaration of Helsinki, International Conference on Harmonization (ICH) Guidelines, Good Clinical Practice (GCP) Standards, Council for International Organizations of Medical Sciences (CIOMS) Guidelines, World Health Organization (WHO) Standards and Operational Guidance for Ethics Review of Health-Related Research and Surveying and Evaluating Ethical Review Practices, EC/IRB Standard Operating Procedures (SOPs), and Local Regulations and Standards in Ethical Review. 2. Subject Vulnerability There is no vulnerability group in this study. All the participants were nurses over 20 years old and working in hospitals; no participants had handicaps or physical problems. The participants made the voluntary choice to take part in the trial. The researchers discussed the study's objectives, methods, benefits and risks. The study was free from any potential hazards or adverse effects on participants, no intervention was applied in emergencies, and no medicine product will be used in this study. The procedure was simple, the devices used in the intervention were safe, and the investigator was instructed to frequently ask the participant about health and physical condition, especially during chest compression. The participants who signed a hard copy of the consent are part of this trial study. During all study phases, the participants will be asked using the electronic method; Google Form Platform, if they would like to participate in this study, and they will answer (YES\\NO) before completing the questionnaires. Furthermore, the intervention will be applied in simulation; the participant voluntarily withdraws from the study without any influence or effect on annual evaluation or salary. The institutions took full responsibility for the trial and protecting the participants and treated the participants if there were unexpected adverse effects or physical harm. The research includes minimal risk toward participants, observable by the time length and components of the intervention; participants may be feeling discomfort. This risk is equal to the nurses' daily life activity. They can be adapted with this minimal risk; only four components require a long time to cover the knowledge and practice in the intervention, but the other four components are easy and need a short time. The researchers planned to minimize the participant's discomfort due to the time and length of the intervention by giving the participant "break times" between the components of the intervention. 3. Declaration of Absence of Conflict of Interest The researchers had declared that there was no conflict of interest, and all co-authors had no financial interest to report. The invistigators certify that the first author is purely a Ph.D. student's original work and has not been given to submit to other universities or journals. 4. Privacy and Confidentiality When the study was completed, the trial master files and documents were protected and kept safe, and all records were kept confidential. Furthermore, the data collected is not associated with individuals by name but only by phone number and email to contact them later to complete the post-test; data is kept properly and handled only in the research. In addition, the researchers also emphasized that only research groups can access the relevant data in this research and not use it in the future without consent from the participants; moreover, after finishing the study, the data will archive properly according to the policy. 5. Community Sensitivities and Benefits The intervention benefits of NEN as a part of a contentious education, and the knowledge and practice shared through the natural learning process in the classroom, face-to-face learning, and the questionnaires filled using an electronic method, Google Forms Platform; there is no sensitive information about the participants. The primary benefits of this study's findings toward participants are to increase newly employed nurses' knowledge, practice skills, and confidence level when facing critical situations inside or outside the hospitals, increases professionalism in all healthcare delivery systems, self-esteem, improve newly employed nurses' performance competency, decision-making, satisfaction, to decrease turnovers and dissatisfaction. Furthermore, the benefits of this study are to develop patient care processes and enhance the victims' survival and patient satisfaction, maintain patient safety, enhance the welfare and hospital discharge outcomes, become cost-effective for patients and their families, and keep patients away from hazards. 6. Incentive and Reimbursement The researcher verbally thanked all participants for their sincere cooperation following the completion. Furthermore, the researcher planned to give the control group an SBLST on a selected day; finally, the participant received feedback about the pre and post-result and a certification attendance of 4 hours from the researcher, adding to the required job development hours in the yearly self-evaluation form. 7. Collaborative Study Terms of Reference: None ### Conditions Module **Conditions:** - Healthy Nurses - Educational Activities **Keywords:** - pretest-post test and intervention ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** The study design is a basic experimental study design, randomized control trial (RCT) design, SBLST for the intervention group and standard intervention to the control group by using a brochure; the SBLST intervention among the control group was delayed until the end of data collection as an appreciation for their participation in the study, control group participants are wait-listed and exposed to the experimental treatment at a later point. Pre-prepared Basic Life Support Guidelines 2020 Treatment (A): Standard Treatment The investigator will give the participants in the control group; the standard treatment by using an AHA-BLS 2020 brochure. The participants will read and understand this brochure over 30 minutes Treatment (B): Simulation in basic life support training The interventional tools, simulation in basic life support training (SBLST), were prepared in English. The researcher will use an already established BLS training from the American Guidelines 2020 (AHA, 2020c) ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** HEALTH_SERVICES_RESEARCH #### Enrollment Info **Count:** 102 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Treatment (B): Simulation in basic life support training The interventional tools, simulation in basic life support training (SBLST), were prepared in English. The researcher will use an already established BLS training from the American Guidelines 2020, a simplified two-part; PowerPoint presentation and clinical simulation training. **Intervention Names:** - Other: Simulation in basic life support training (SBLST) **Label:** SBLST Group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** The investigator will give the participants in the control group; the standard treatment by using an AHA-BLS 2020 brochure. The participants will read and understand this brochure over 30 minutes, including a brief guideline about basic life support guidelines, and after these 30 minutes, the participants will move to the post-test. **Intervention Names:** - Other: standard treatment by using an AHA-BLS 2020 brochure **Label:** Standard Group **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - SBLST Group **Description:** The interventional tools, simulation in basic life support training (SBLST), were prepared in English. The researcher will use an already established BLS training from the American Guidelines 2020, a simplified two-part; PowerPoint presentation, and clinical simulation training. **Name:** Simulation in basic life support training (SBLST) **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Standard Group **Description:** The participants will read and understand this brochure over 30 minutes, including a brief guideline about basic life support guidelines, and after these 30 minutes, the participants will move to the post-test **Name:** standard treatment by using an AHA-BLS 2020 brochure **Type:** OTHER ### Outcomes Module #### Other Outcomes **Description:** The investigators selected inclusion criteria for both the control and experimental group, and both groups should be Homogenous in the demographical data by using Mann-Witney U-test and checking the median and IQ range. The intervention is educational, and there is no adverse effect or risk to the participant **Measure:** There are no differences between the control and experimental groups in the inclusion criteria **Time Frame:** 2 month after finsh the pre-test of both group #### Primary Outcomes **Description:** To evaluate the effectiveness of SMBST on knowledge scores, investigators will conduct three measurements: a pretest, an immediate post-test after the intervention, and a post-test 2 after three months for both experimental and control groups. Participants will be sent a questionnaire consisting of 13 MCQ three times via Google Form Platforms. These questions will be sent to their cell phone numbers. Repeated measure ANOVA will be conducted for both groups and the mean knowledge scores between the pretest and post-test will be compared. The level of significance will be determined by the p-value to accept or reject the alternative hypothesis (The simulation in basic life support training intervention improves knowledge among newly employed nurses (NEN) in a Jordanian governmental hospital). The intervention is educational and poses no risk or adverse effects to the participants. **Measure:** The Effectiveness of Simulation in basic life support training (SBLST) on the Knowledge Score **Time Frame:** 6 Months after finish all data collection **Description:** The investigators performed three measurements to assess the effectiveness of SMBST on practice score; Pretest, immediate-posttest after the intervention, and post-test-2 after three months of the intervention for both experimental and control groups. The practice questionnaires consist of ten MCQs, and the participants will answer this survey three times; these questions will send to the participant's cell phone numbers as a link from Google Form Platforms at all times off-test family, ANOVA for experimental and control groups, then comparing the mean between practice score in both groups and between pretest and post-test and finally identify the level of significant by p-value to reject or accept the alternative hypothesis" The simulation in basic life support training intervention effectively improves practice among newly employed nurses (NEN) in Jordanian governmental hospital." The intervention is educational, and there is no adverse effect or risk to the participant. **Measure:** The Effectiveness of Simulation in basic life support training (SBLST) on the Practice Score **Time Frame:** 6 moths after finish all data collection **Description:** The effectiveness of SMBST on confidence levels will be assessed by measuring pretest, immediate posttest, and post-test-2 results for both experimental and control groups. Participants will complete the survey three times using a Google Forms link sent to their cell phones. Repeated measure ANOVA will be used to analyze the data and compare mean confidence levels between the groups and pretest/post-test scores. The intervention has proven effective in improving confidence levels with no adverse effects or risks. **Measure:** The Effectiveness of Simulation in basic life support training (SBLST) on the Confidence Level **Time Frame:** 6 moths after finish all data collection #### Secondary Outcomes **Description:** Before implementing any interventions for the experimental and control groups, the investigator ensures that the pretest levels of both groups are significantly similar to ensure accurate measurement. After conducting an ANOVA analysis and pairwise comparison with means, standard deviation (SD), and P value results, the investigator will decide on this hypothesis. The intervention is educational and poses no adverse effects or risks to the participants. **Measure:** There are no significant differences between the control and experimental groups in the knowledge score pretest **Time Frame:** 2 month after finsh the pre-test of both group **Description:** Before implementing any interventions for the experimental or control groups, the investigator takes measures to ensure that both groups possess comparable pretest scores, thus guaranteeing precise measurements. The validity of this hypothesis will be substantiated through ANOVA analysis, whereby means, standard deviation (SD), and P-values will be compared using pairwise comparisons. It is important to note that the educational intervention provided does not carry any risk or adverse effects for the participants. **Measure:** There are no significant differences between the control and experimental groups in the practice score pretest **Time Frame:** 2 month after finsh the pre-test of both group **Description:** Before performing any intervention for the intervention; and experimental groups, the investigator makes sure that the pretest of both groups is significantly the same level to ensure the accuracy of the measurement; the investigator will decide on this hypothesis after ANOVA analysis and use pairwise comparison with means, standard deviation (SD), and P value results. The intervention is educational, and there is no adverse effect or risk to the participant. **Measure:** There are no significant differences between the control and experimental groups in the pretest regarding confidence level. **Time Frame:** 2 month after finsh the pre-test of both group **Description:** The investigator will assess the effectiveness of SBLST on the experimental group by comparing post-test mean and SD and assess the level of significance (p-value) for the control and experimental group to decide that the SBLST was effective on knowledge score using ANOVA repeated measure analysis. The intervention is educational, and there is no adverse effect or risk to the participant. **Measure:** Significant differences exist between the control and experimental groups in the post-tests on knowledge scores. **Time Frame:** 6 moths after finish all data collection **Description:** The investigator will assess the effectiveness of SBLST on the experimental group by comparing post-test mean and SD and assess the level of significance (p-value) for the control and experimental group to decide that the SBLST was effective on practice score using ANOVA repeated measure analysis. The intervention is educational, and there is no adverse effect or risk to the participant. **Measure:** Significant differences exist between the control and experimental groups in the post-test after the interventions on practice score **Time Frame:** 6 moths after finish all data collection **Description:** The investigator will assess the effectiveness of SBLST on the experimental group by comparing post-test mean and SD and assess the significance level (p-value) for the control and experimental group to decide that the SBLST was effective on confidence level using ANOVA repeated measure analysis. The intervention is educational, and there is no adverse effect or risk to the participant. **Measure:** Significant differences exist between the control and experimental groups in the post-test after the interventions on the confidence level **Time Frame:** 6 moths after finish all data collection ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria The researchers tried to maintain homogeneity and minimize any variations among the participants by 1. selecting both interventional and control groups from newly employed nurses 2. Male and female participants 3. Participants only who understand the questionnaires and interventions in English 4. Participants who were previously on-seat undergraduate nursing students last two years, 2020 (4th year nursing bachelor level) and 2021 (3rd year nursing bachelor level) in the period of the COVID-19 pandemic and received basic and clinical learning by online method, and now became a newly employed nurse (NEN) in hospitals and delivered care to the patient. 5. Participants can attend five to seven hours of SBLST sessions. 6. For more control, we selected nurses who rarely face CPR and do not attend BLS raining the last two years. Exclusion Criteria 1. The researchers excluded the NEN participants working in intensive care units because ICU nurses face CPR and perform BLS daily. 2. Excluded participants with medical or physical health problems (e.g., pregnant women and participants complaining of lower back pain). 3. Participants who attended CPR training less than two years previously were also excluded. Finally, 4. NEN who upgraded their educational degree from a diploma to a bachelor's degree were excluded. **Healthy Volunteers:** True **Minimum Age:** 17 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Amman **Country:** Jordan **Facility:** Ministry of Health **Zip:** 11118 ### IPD Sharing Statement Module **Access Criteria:** Data is kept properly and handled only in the research. In addition, the researchers also emphasized that only research groups can access the relevant data in this research and not use it in the future without consent from the participants; moreover, after finishing the study, the data will archive properly according to the policy. **Description:** Only the participant data related to the study after participate and publication consent without mentioned name, ID, email...and providing the privacy **Info Types:** - STUDY_PROTOCOL - SAP - ICF - CSR - ANALYTIC_CODE **IPD Sharing:** YES **Time Frame:** after finish and publish the study and will not use in any other study ### References Module #### References **Citation:** Pallas JD, Smiles JP, Zhang M. Cardiac Arrest Nurse Leadership (CANLEAD) trial: a simulation-based randomised controlled trial implementation of a new cardiac arrest role to facilitate cognitive offload for medical team leaders. Emerg Med J. 2021 Aug;38(8):572-578. doi: 10.1136/emermed-2019-209298. Epub 2021 Jan 26. **PMID:** 33500268 **Citation:** Nolan JP, Ornato JP, Parr MJA, Perkins GD, Soar J. Resuscitation highlights in 2019. Resuscitation. 2020 Mar 1;148:234-241. doi: 10.1016/j.resuscitation.2020.02.001. Epub 2020 Feb 7. No abstract available. **PMID:** 32044335 **Citation:** Zhou XL, Wang J, Jin XQ, Zhao Y, Liu RL, Jiang C. Quality retention of chest compression after repetitive practices with or without feedback devices: A randomized manikin study. Am J Emerg Med. 2020 Jan;38(1):73-78. doi: 10.1016/j.ajem.2019.04.025. Epub 2019 Apr 12. **PMID:** 31005392 **Citation:** Al Gharibi Msn KA, Arulappan MSc N PhD DNSc J. Repeated Simulation Experience on Self-Confidence, Critical Thinking, and Competence of Nurses and Nursing Students-An Integrative Review. SAGE Open Nurs. 2020 Jun 1;6:2377960820927377. doi: 10.1177/2377960820927377. eCollection 2020 Jan-Dec. **PMID:** 33415282 **Citation:** Abelsson A, Odestrand P, Nygardh A. To strengthen self-confidence as a step in improving prehospital youth laymen basic life support. BMC Emerg Med. 2020 Jan 30;20(1):8. doi: 10.1186/s12873-020-0304-8. **PMID:** 32000691 **Citation:** Aksoy ME. Comparing Basic Life Support Serious Gaming Scores With Hands-on Training Platform Performance Scores: Pilot Simulation Study for Basic Life Support Training. JMIR Serious Games. 2020 Nov 25;8(4):e24166. doi: 10.2196/24166. **PMID:** 33237035 **Citation:** Lott C, Truhlar A, Alfonzo A, Barelli A, Gonzalez-Salvado V, Hinkelbein J, Nolan JP, Paal P, Perkins GD, Thies KC, Yeung J, Zideman DA, Soar J; ERC Special Circumstances Writing Group Collaborators. European Resuscitation Council Guidelines 2021: Cardiac arrest in special circumstances. Resuscitation. 2021 Apr;161:152-219. doi: 10.1016/j.resuscitation.2021.02.011. Epub 2021 Mar 24. Erratum In: Resuscitation. 2021 Oct;167:91-92. **PMID:** 33773826 **Citation:** Aliyari S, Pishgooie AH, Abdi A, Mazhari MS, Nazari MR. Comparing two teaching methods based on concept map and lecture on the level of learning in basic life support. Nurse Educ Pract. 2019 Jul;38:40-44. doi: 10.1016/j.nepr.2019.05.008. Epub 2019 May 31. **PMID:** 31176242 **Citation:** Armstrong P, Peckler B, Pilkinton-Ching J, McQuade D, Rogan A. Effect of simulation training on nurse leadership in a shared leadership model for cardiopulmonary resuscitation in the emergency department. Emerg Med Australas. 2021 Apr;33(2):255-261. doi: 10.1111/1742-6723.13605. Epub 2020 Aug 27. **PMID:** 32856402 **Citation:** Banfai B, Musch J, Betlehem J, Santa E, Horvath B, Nemeth D, Banfai-Csonka H. How effective are chest compressions when wearing mask? A randomised simulation study among first-year health care students during the COVID-19 pandemic. BMC Emerg Med. 2022 May 8;22(1):82. doi: 10.1186/s12873-022-00636-2. **PMID:** 35527256 **Citation:** Barakat M, Farha RA, Muflih S, Al-Tammemi AB, Othman B, Allozi Y, Fino L. The era of E-learning from the perspectives of Jordanian medical students: A cross-sectional study. Heliyon. 2022 Jul 11;8(7):e09928. doi: 10.1016/j.heliyon.2022.e09928. eCollection 2022 Jul. **PMID:** 35874065 **Citation:** Borggreve AS, Meijer JMR, Schreuder HWR, Ten Cate O. Simulation-based trauma education for medical students: A review of literature. Med Teach. 2017 Jun;39(6):631-638. doi: 10.1080/0142159X.2017.1303135. Epub 2017 Mar 30. **PMID:** 28355934 **Citation:** Binkhorst M, van der Aar IM, Linders M, van Heijst AFJ, de Boode WP, Draaisma JMT, Hogeveen M. Training practices in neonatal and paediatric life support: A survey among healthcare professionals working in paediatrics. Resusc Plus. 2021 Jan 6;5:100063. doi: 10.1016/j.resplu.2020.100063. eCollection 2021 Mar. **PMID:** 34223335 **Citation:** Bottiger BW, Lockey A, Aickin R, Bertaut T, Castren M, de Caen A, Censullo E, Escalante R, Gent L, Georgiou M, Kern KB, Khan AMS, Lim SH, Nadkarni V, Nation K, Neumar RW, Nolan JP, Rao SSCC, Stanton D, Toporas C, Wang TL, Wong G, Perkins GD. Over 675,000 lay people trained in cardiopulmonary resuscitation worldwide - The "World Restart a Heart (WRAH)" initiative 2018. Resuscitation. 2019 May;138:15-17. doi: 10.1016/j.resuscitation.2019.02.033. Epub 2019 Mar 2. No abstract available. **PMID:** 30836172 **Citation:** Bukiran A, Erdur B, Ozen M, Bozkurt AI. Retention of nurses' knowledge after basic life support and advanced cardiac life support training at immediate, 6-month, and 12-month post-training intervals: a longitudinal study of nurses in Turkey. J Emerg Nurs. 2014 Mar;40(2):146-52. doi: 10.1016/j.jen.2012.08.011. Epub 2013 Jan 7. No abstract available. **PMID:** 23305947 **Citation:** Semeraro F, Greif R, Bottiger BW, Burkart R, Cimpoesu D, Georgiou M, Yeung J, Lippert F, S Lockey A, Olasveengen TM, Ristagno G, Schlieber J, Schnaubelt S, Scapigliati A, G Monsieurs K. European Resuscitation Council Guidelines 2021: Systems saving lives. Resuscitation. 2021 Apr;161:80-97. doi: 10.1016/j.resuscitation.2021.02.008. Epub 2021 Mar 24. **PMID:** 33773834 **Citation:** Soar J, Bottiger BW, Carli P, Couper K, Deakin CD, Djarv T, Lott C, Olasveengen T, Paal P, Pellis T, Perkins GD, Sandroni C, Nolan JP. European Resuscitation Council Guidelines 2021: Adult advanced life support. Resuscitation. 2021 Apr;161:115-151. doi: 10.1016/j.resuscitation.2021.02.010. Epub 2021 Mar 24. Erratum In: Resuscitation. 2021 Oct;167:105-106. **PMID:** 33773825 **Citation:** Charlier N, Van Der Stock L, Iserbyt P. Comparing student nurse knowledge and performance of basic life support algorithm actions: An observational post-retention test design study. Nurse Educ Pract. 2020 Feb 19;43:102714. doi: 10.1016/j.nepr.2020.102714. Online ahead of print. **PMID:** 32109754 **Citation:** Christmals CD, Gross J, Aziato L, Armstrong SJ. The State of Nursing Research in Ghana: An Integrative Literature Review. SAGE Open Nurs. 2018 Nov 18;4:2377960818783820. doi: 10.1177/2377960818783820. eCollection 2018 Jan-Dec. **PMID:** 33415196 **Citation:** Costa IKF, Tiburcio MP, Costa IKF, Dantas RAN, Galvao RN, Torres GV. Development of a virtual simulation game on basic life support. Rev Esc Enferm USP. 2018 Nov 1;52:e03382. doi: 10.1590/S1980-220X2017047903382. English, Portuguese. **PMID:** 30403269 **Citation:** Couloures KG, Allen C. Use of Simulation to Improve Cardiopulmonary Resuscitation Performance and Code Team Communication for Pediatric Residents. MedEdPORTAL. 2017 Mar 16;13:10555. doi: 10.15766/mep_2374-8265.10555. **PMID:** 30800757 **Citation:** Curl ED, Smith S, Ann Chisholm L, McGee LA, Das K. Effectiveness of Integrated Simulation and Clinical Experiences Compared to Traditional Clinical Experiences for Nursing Students. Nurs Educ Perspect. 2016 Mar-Apr;37(2):72-7. **PMID:** 27209864 **Citation:** Requena-Mullor MDM, Alarcon-Rodriguez R, Ventura-Miranda MI, Garcia-Gonzalez J. Effects of a Clinical Simulation Course about Basic Life Support on Undergraduate Nursing Students' Learning. Int J Environ Res Public Health. 2021 Feb 3;18(4):1409. doi: 10.3390/ijerph18041409. **PMID:** 33546328 **Citation:** Dick-Smith F, Power T, Martinez-Maldonado R, Elliott D. Basic Life Support Training for undergraduate nursing students: An integrative review. Nurse Educ Pract. 2021 Jan;50:102957. doi: 10.1016/j.nepr.2020.102957. Epub 2020 Dec 22. **PMID:** 33421680 **Citation:** Ekert JO, Luchesa Smith A, Ramsey CL, Robinson N, Love J, Gothard P, Armitage AJ. Medical student-led simulation in COVID-19 crisis. Clin Teach. 2021 Jun;18(3):252-257. doi: 10.1111/tct.13308. Epub 2020 Dec 6. **PMID:** 33280253 **Citation:** Elgohary M, Palazzo FS, Breckwoldt J, Cheng A, Pellegrino J, Schnaubelt S, Greif R, Lockey A. Blended learning for accredited life support courses - A systematic review. Resusc Plus. 2022 May 10;10:100240. doi: 10.1016/j.resplu.2022.100240. eCollection 2022 Jun. **PMID:** 35592876 **Citation:** Garcia-Suarez M, Mendez-Martinez C, Martinez-Isasi S, Gomez-Salgado J, Fernandez-Garcia D. Basic Life Support Training Methods for Health Science Students: A Systematic Review. Int J Environ Res Public Health. 2019 Mar 3;16(5):768. doi: 10.3390/ijerph16050768. **PMID:** 30832440 **Citation:** Gonzalez-Salvado V, Rodriguez-Ruiz E, Abelairas-Gomez C, Ruano-Ravina A, Pena-Gil C, Gonzalez-Juanatey JR, Rodriguez-Nunez A. Training adult laypeople in basic life support. A systematic review. Rev Esp Cardiol (Engl Ed). 2020 Jan;73(1):53-68. doi: 10.1016/j.rec.2018.11.013. Epub 2019 Feb 23. English, Spanish. **PMID:** 30808611 **Citation:** Goodloe JM, Topjian A, Hsu A, Dunne R, Panchal AR, Levy M, McEvoy M, Vaillancourt C, Cabanas JG, Eisenberg MS, Rea TD, Kudenchuk PJ, Gienapp A, Flores GE, Fuchs S, Adelgais KM, Owusu-Ansah S, Terry M, Sawyer KN, Fromm P, Panczyk M, Kurz M, Lindbeck G, Tan DK, Edelson DP, Sayre MR. Interim Guidance for Emergency Medical Services Management of Out-of-Hospital Cardiac Arrest During the COVID-19 Pandemic. Circ Cardiovasc Qual Outcomes. 2021 Jul;14(7):e007666. doi: 10.1161/CIRCOUTCOMES.120.007666. Epub 2021 Jun 23. No abstract available. **PMID:** 34157848 **Citation:** Grasner JT, Herlitz J, Tjelmeland IBM, Wnent J, Masterson S, Lilja G, Bein B, Bottiger BW, Rosell-Ortiz F, Nolan JP, Bossaert L, Perkins GD. European Resuscitation Council Guidelines 2021: Epidemiology of cardiac arrest in Europe. Resuscitation. 2021 Apr;161:61-79. doi: 10.1016/j.resuscitation.2021.02.007. Epub 2021 Mar 24. **PMID:** 33773833 **Citation:** Greif R, Lockey A, Breckwoldt J, Carmona F, Conaghan P, Kuzovlev A, Pflanzl-Knizacek L, Sari F, Shammet S, Scapigliati A, Turner N, Yeung J, Monsieurs KG. European Resuscitation Council Guidelines 2021: Education for resuscitation. Resuscitation. 2021 Apr;161:388-407. doi: 10.1016/j.resuscitation.2021.02.016. Epub 2021 Mar 24. **PMID:** 33773831 **Citation:** Gutierrez-Puertas L, Garcia-Viola A, Marquez-Hernandez VV, Garrido-Molina JM, Granados-Gamez G, Aguilera-Manrique G. Guess it (SVUAL): An app designed to help nursing students acquire and retain knowledge about basic and advanced life support techniques. Nurse Educ Pract. 2021 Jan;50:102961. doi: 10.1016/j.nepr.2020.102961. Epub 2020 Dec 29. **PMID:** 33421681 **Citation:** Gutierrez-Puertas L, Marquez-Hernandez VV, Gutierrez-Puertas V, Rodriguez-Garcia MC, Garcia-Viola A, Aguilera-Manrique G. Are You Prepared to Save a Life? Nursing Students' Experience in Advanced Life Support Practice. Int J Environ Res Public Health. 2021 Jan 31;18(3):1273. doi: 10.3390/ijerph18031273. **PMID:** 33572616 **Citation:** Haider AS, Al-Salman S. Dataset of Jordanian university students' psychological health impacted by using e-learning tools during COVID-19. Data Brief. 2020 Jul 31;32:106104. doi: 10.1016/j.dib.2020.106104. eCollection 2020 Oct. **PMID:** 32789158 **Citation:** Handeland JA, Prinz A, Ekra EMR, Fossum M. The role of manikins in nursing students' learning: A systematic review and thematic metasynthesis. Nurse Educ Today. 2021 Mar;98:104661. doi: 10.1016/j.nedt.2020.104661. Epub 2020 Nov 12. **PMID:** 33298327 **Citation:** Higgins BV, Medvedev MM, Spindler H, Ghosh R, Longkumer O, Cohen SR, Das A, Gore A, Mahapatra T, Walker DM. Cohort study of neonatal resuscitation skill retention in frontline healthcare facilities in Bihar, India, after PRONTO simulation training. BMJ Paediatr Open. 2020 Apr 20;4(1):e000628. doi: 10.1136/bmjpo-2019-000628. eCollection 2020. **PMID:** 32399505 **Citation:** Holm A, Jerkeman M, Sultanian P, Lundgren P, Ravn-Fischer A, Israelsson J, Giesecke J, Herlitz J, Rawshani A. Cohort study of the characteristics and outcomes in patients with COVID-19 and in-hospital cardiac arrest. BMJ Open. 2021 Nov 30;11(11):e054943. doi: 10.1136/bmjopen-2021-054943. **PMID:** 34848525 **Citation:** Irfan B, Zahid I, Khan MS, Khan OAA, Zaidi S, Awan S, Bilal S, Irfan O. Current state of knowledge of basic life support in health professionals of the largest city in Pakistan: a cross-sectional study. BMC Health Serv Res. 2019 Nov 21;19(1):865. doi: 10.1186/s12913-019-4676-y. **PMID:** 31752855 **Citation:** Iserbyt P, Theys L, Ward P, Charlier N. The effect of a specialized content knowledge workshop on teaching and learning Basic Life Support in elementary school: A cluster randomized controlled trial. Resuscitation. 2017 Mar;112:17-21. doi: 10.1016/j.resuscitation.2016.11.023. Epub 2016 Dec 20. **PMID:** 28011290 **Citation:** Jang K, Kim SH, Oh JY, Mun JY. Effectiveness of self-re-learning using video recordings of advanced life support on nursing students' knowledge, self-efficacy, and skills performance. BMC Nurs. 2021 Mar 31;20(1):52. doi: 10.1186/s12912-021-00573-8. **PMID:** 33789625 **Citation:** Jarrah S, Judeh M, AbuRuz ME. Evaluation of public awareness, knowledge and attitudes towards basic life support: a cross-sectional study. BMC Emerg Med. 2018 Oct 29;18(1):37. doi: 10.1186/s12873-018-0190-5. **PMID:** 30373529 **Citation:** Jung D, Lee SH, Kang SJ, Kim JH. Development and evaluation of a clinical simulation for new graduate nurses: A multi-site pilot study. Nurse Educ Today. 2017 Feb;49:84-89. doi: 10.1016/j.nedt.2016.11.010. Epub 2016 Nov 18. Erratum In: Nurse Educ Today. 2017 May;52:124. **PMID:** 27889583 **Citation:** Kei J, Mebust DP. Effects of cardiopulmonary resuscitation on direct versus video laryngoscopy using a mannequin model. Am J Emerg Med. 2021 Dec;50:587-591. doi: 10.1016/j.ajem.2021.09.031. Epub 2021 Sep 20. **PMID:** 34563941 **Citation:** Kim CH, Kim TH, Shin SD, Song KJ, Ro YS, Ahn KO, Hong KJ, Lee YJ, Lee EJ, Ha SY. The effect of automatic external defibrillator with a real-time feedback on quality of bystander cardiopulmonary resuscitation: A before-and-after simulation study. Health Soc Care Community. 2019 Sep;27(5):e744-e751. doi: 10.1111/hsc.12800. Epub 2019 Jun 21. **PMID:** 31225932 **Citation:** Kim YH, Jang KS. [Effect of a simulation-based education on cardio-pulmonary emergency care knowledge, clinical performance ability and problem solving process in new nurses]. J Korean Acad Nurs. 2011 Apr;41(2):245-55. doi: 10.4040/jkan.2011.41.2.245. Korean. **PMID:** 21551996 **Citation:** Kose S, Akin S, Mendi O, Goktas S. The effectiveness of basic life support training on nursing students' knowledge and basic life support practices: a non-randomized quasi-experimental study. Afr Health Sci. 2019 Jun;19(2):2252-2262. doi: 10.4314/ahs.v19i2.51. **PMID:** 31656511 **Citation:** La Cerra C, Dante A, Caponnetto V, Franconi I, Gaxhja E, Petrucci C, Alfes CM, Lancia L. Effects of high-fidelity simulation based on life-threatening clinical condition scenarios on learning outcomes of undergraduate and postgraduate nursing students: a systematic review and meta-analysis. BMJ Open. 2019 Feb 22;9(2):e025306. doi: 10.1136/bmjopen-2018-025306. **PMID:** 30798316 **Citation:** Laco RB, Stuart WP. Simulation-Based Training Program to Improve Cardiopulmonary Resuscitation and Teamwork Skills for the Urgent Care Clinic Staff. Mil Med. 2022 May 3;187(5-6):e764-e769. doi: 10.1093/milmed/usab198. **PMID:** 34050365 **Citation:** Lauridsen KG, Morgan RW, Dewan M, Gawronski O, Sen AI; PediRES-Q Investigators. In-hospital cardiac arrest characteristics, CPR quality, and outcomes in children with COVID-19. Resuscitation. 2021 Dec;169:39-40. doi: 10.1016/j.resuscitation.2021.10.013. Epub 2021 Oct 18. No abstract available. **PMID:** 34673154 **Citation:** Lee DK. Alternatives to P value: confidence interval and effect size. Korean J Anesthesiol. 2016 Dec;69(6):555-562. doi: 10.4097/kjae.2016.69.6.555. Epub 2016 Oct 25. **PMID:** 27924194 **Citation:** Liaw SY, Wong LF, Chan SW, Ho JT, Mordiffi SZ, Ang SB, Goh PS, Ang EN. Designing and evaluating an interactive multimedia Web-based simulation for developing nurses' competencies in acute nursing care: randomized controlled trial. J Med Internet Res. 2015 Jan 12;17(1):e5. doi: 10.2196/jmir.3853. Erratum In: J Med Internet Res. 2015 Mar 23;17(3):e75. **PMID:** 25583029 **Citation:** Magill N, Knight R, McCrone P, Ismail K, Landau S. A scoping review of the problems and solutions associated with contamination in trials of complex interventions in mental health. BMC Med Res Methodol. 2019 Jan 7;19(1):4. doi: 10.1186/s12874-018-0646-z. **PMID:** 30616508 **Citation:** Manolis AS, Manolis AA, Manolis TA, Apostolopoulos EJ, Papatheou D, Melita H. COVID-19 infection and cardiac arrhythmias. Trends Cardiovasc Med. 2020 Nov;30(8):451-460. doi: 10.1016/j.tcm.2020.08.002. Epub 2020 Aug 16. **PMID:** 32814095 **Citation:** Marijon E, Karam N, Jost D, Perrot D, Frattini B, Derkenne C, Sharifzadehgan A, Waldmann V, Beganton F, Narayanan K, Lafont A, Bougouin W, Jouven X. Out-of-hospital cardiac arrest during the COVID-19 pandemic in Paris, France: a population-based, observational study. Lancet Public Health. 2020 Aug;5(8):e437-e443. doi: 10.1016/S2468-2667(20)30117-1. Epub 2020 May 27. **PMID:** 32473113 **Citation:** Maxwell WD, Mohorn PL, Haney JS, Phillips CM, Lu ZK, Clark K, Corboy A, Ragucci KR. Impact of an Advanced Cardiac Life Support Simulation Laboratory Experience on Pharmacy Student Confidence and Knowledge. Am J Pharm Educ. 2016 Oct 25;80(8):140. doi: 10.5688/ajpe808140. **PMID:** 27899836 **Citation:** McKelvin R, McKelvin G. Immersive simulation training: Comparing the impact on midwifery and paramedic students' confidence to perform basic life support skills. Midwifery. 2020 Aug;87:102717. doi: 10.1016/j.midw.2020.102717. Epub 2020 Apr 12. **PMID:** 32353663 **Citation:** Mendez-Martinez C, Martinez-Isasi S, Garcia-Suarez M, Pena-Rodriguez MA, Gomez-Salgado J, Fernandez-Garcia D. Acquisition of Knowledge and Practical Skills after a Brief Course of BLS-AED in First-Year Students in Nursing and Physiotherapy at a Spanish University. Int J Environ Res Public Health. 2019 Mar 3;16(5):766. doi: 10.3390/ijerph16050766. **PMID:** 30832439 **Citation:** Mentzelopoulos SD, Couper K, Voorde PV, Druwe P, Blom M, Perkins GD, Lulic I, Djakow J, Raffay V, Lilja G, Bossaert L. European Resuscitation Council Guidelines 2021: Ethics of resuscitation and end of life decisions. Resuscitation. 2021 Apr;161:408-432. doi: 10.1016/j.resuscitation.2021.02.017. Epub 2021 Mar 24. **PMID:** 33773832 **Citation:** Muflih S, Abuhammad S, Al-Azzam S, Alzoubi KH, Muflih M, Karasneh R. Online learning for undergraduate health professional education during COVID-19: Jordanian medical students' attitudes and perceptions. Heliyon. 2021 Sep;7(9):e08031. doi: 10.1016/j.heliyon.2021.e08031. Epub 2021 Sep 20. **PMID:** 34568607 **Citation:** Nabolsi M, Abu-Moghli F, Khalaf I, Zumot A, Suliman W. Nursing Faculty Experience With Online Distance Education During COVID-19 Crisis: A Qualitative Study. J Prof Nurs. 2021 Sep-Oct;37(5):828-835. doi: 10.1016/j.profnurs.2021.06.002. Epub 2021 Jun 9. **PMID:** 34742511 **Citation:** Nassar AK, Al-Manaseer F, Knowlton LM, Tuma F. Virtual reality (VR) as a simulation modality for technical skills acquisition. Ann Med Surg (Lond). 2021 Oct 27;71:102945. doi: 10.1016/j.amsu.2021.102945. eCollection 2021 Nov. **PMID:** 34840738 **Citation:** Nishiyama C, Iwami T, Murakami Y, Kitamura T, Okamoto Y, Marukawa S, Sakamoto T, Kawamura T. Effectiveness of simplified 15-min refresher BLS training program: a randomized controlled trial. Resuscitation. 2015 May;90:56-60. doi: 10.1016/j.resuscitation.2015.02.015. Epub 2015 Feb 24. **PMID:** 25724354 **Citation:** Oermann MH, Krusmark MA, Kardong-Edgren S, Jastrzembski TS, Gluck KA. Training interval in cardiopulmonary resuscitation. PLoS One. 2020 Jan 16;15(1):e0226786. doi: 10.1371/journal.pone.0226786. eCollection 2020. **PMID:** 31945074 **Citation:** Oteir AO, Almhdawi KA, Kanaan SF, Alwidyan MT, Williams B. Cardiopulmonary resuscitation level of knowledge among allied health university students in Jordan: a cross-sectional study. BMJ Open. 2019 Nov 19;9(11):e031725. doi: 10.1136/bmjopen-2019-031725. **PMID:** 31748305 **Citation:** Paddock A. Using Simulation to Improve Adherence to Get With the Guidelines Time to First Shock. Crit Care Nurse. 2021 Dec 1;41(6):62-68. doi: 10.4037/ccn2021596. **PMID:** 34851386 **Citation:** Paliatsiou S, Xanthos T, Wyllie J, Volaki P, Sokou R, Bikouli D, Iliodromiti Z, Boutsikou T, Iacovidou N. Theoretical knowledge and skill retention 3 and 6 months after a European Newborn Life Support provider course. Am J Emerg Med. 2021 Nov;49:83-88. doi: 10.1016/j.ajem.2021.05.048. Epub 2021 May 20. **PMID:** 34089968 **Citation:** Panchal AR, Bartos JA, Cabanas JG, Donnino MW, Drennan IR, Hirsch KG, Kudenchuk PJ, Kurz MC, Lavonas EJ, Morley PT, O'Neil BJ, Peberdy MA, Rittenberger JC, Rodriguez AJ, Sawyer KN, Berg KM; Adult Basic and Advanced Life Support Writing Group. Part 3: Adult Basic and Advanced Life Support: 2020 American Heart Association Guidelines for Cardiopulmonary Resuscitation and Emergency Cardiovascular Care. Circulation. 2020 Oct 20;142(16_suppl_2):S366-S468. doi: 10.1161/CIR.0000000000000916. Epub 2020 Oct 21. No abstract available. **PMID:** 33081529 **Citation:** Parikh P, Samraj R, Ogbeifun H, Sumbel L, Brimager K, Alhendy M, McElroy J, Whitt D, Henderson C, Bhalala U. Simulation-Based Training in High-Quality Cardiopulmonary Resuscitation Among Neonatal Intensive Care Unit Providers. Front Pediatr. 2022 Mar 9;10:808992. doi: 10.3389/fped.2022.808992. eCollection 2022. **PMID:** 35356440 **Citation:** Parsons JR, Crichlow A, Ponnuru S, Shewokis PA, Goswami V, Griswold S. Filling the Gap: Simulation-based Crisis Resource Management Training for Emergency Medicine Residents. West J Emerg Med. 2018 Jan;19(1):205-210. doi: 10.5811/westjem.2017.10.35284. Epub 2017 Dec 14. **PMID:** 29383082 **Citation:** Partiprajak S, Thongpo P. Retention of basic life support knowledge, self-efficacy and chest compression performance in Thai undergraduate nursing students. Nurse Educ Pract. 2016 Jan;16(1):235-41. doi: 10.1016/j.nepr.2015.08.012. Epub 2015 Sep 3. **PMID:** 26553101 **Citation:** Pehlivan M, Mercan NC, Cinar I, Elmali F, Soyoz M. The evaluation of laypersons awareness of basic life support at the university in Izmir. Turk J Emerg Med. 2019 Jan 18;19(1):26-29. doi: 10.1016/j.tjem.2018.11.002. eCollection 2019 Jan. **PMID:** 30793062 **Citation:** Perkins GD, Couper K. COVID-19: long-term effects on the community response to cardiac arrest? Lancet Public Health. 2020 Aug;5(8):e415-e416. doi: 10.1016/S2468-2667(20)30134-1. Epub 2020 May 27. No abstract available. **PMID:** 32473112 **Citation:** Piryani RM, Piryani S, Shrestha U, Acharya A, Kanskar S, Shahi M, Kayastha J, Chaulagain A, Agarwal JP, Bajracharya SR. Simulation-based education workshop: perceptions of participants. Adv Med Educ Pract. 2019 Jul 23;10:547-554. doi: 10.2147/AMEP.S204816. eCollection 2019. **PMID:** 31413653 **Citation:** Rajaguru V, Park J. Contemporary Integrative Review in Simulation-Based Learning in Nursing. Int J Environ Res Public Health. 2021 Jan 15;18(2):726. doi: 10.3390/ijerph18020726. **PMID:** 33467730 **Citation:** Roedl K, Soffker G, Fischer D, Muller J, Westermann D, Issleib M, Kluge S, Jarczak D. Effects of COVID-19 on in-hospital cardiac arrest: incidence, causes, and outcome - a retrospective cohort study. Scand J Trauma Resusc Emerg Med. 2021 Feb 8;29(1):30. doi: 10.1186/s13049-021-00846-w. **PMID:** 33557923 **Citation:** Rushton MA, Drumm IA, Campion SP, O'Hare JJ. The Use of Immersive and Virtual Reality Technologies to Enable Nursing Students to Experience Scenario-Based, Basic Life Support Training-Exploring the Impact on Confidence and Skills. Comput Inform Nurs. 2020 Jun;38(6):281-293. doi: 10.1097/CIN.0000000000000608. **PMID:** 32149741 **Citation:** Ryan A, Rizwan R, Williams B, Benscoter A, Cooper DS, Iliopoulos I. Simulation Training Improves Resuscitation Team Leadership Skills of Nurse Practitioners. J Pediatr Health Care. 2019 May-Jun;33(3):280-287. doi: 10.1016/j.pedhc.2018.09.006. Epub 2018 Nov 27. **PMID:** 30497891 **Citation:** Ryzner DM, Kujath AS. Low-Fidelity Code Blue Simulation on the Orthopaedic Unit. Orthop Nurs. 2018 Jul/Aug;37(4):230-234. doi: 10.1097/NOR.0000000000000475. **PMID:** 30028424 **Citation:** Saramma PP, Raj LS, Dash PK, Sarma PS. Assessment of long-term impact of formal certified cardiopulmonary resuscitation training program among nurses. Indian J Crit Care Med. 2016 Apr;20(4):226-32. doi: 10.4103/0972-5229.180043. **PMID:** 27303137 **Citation:** Sarvan S, Efe E. The effect of neonatal resuscitation training based on a serious game simulation method on nursing students' knowledge, skills, satisfaction and self-confidence levels: A randomized controlled trial. Nurse Educ Today. 2022 Apr;111:105298. doi: 10.1016/j.nedt.2022.105298. Epub 2022 Feb 8. **PMID:** 35158135 **Citation:** Sawyer T, Eppich W, Brett-Fleegler M, Grant V, Cheng A. More Than One Way to Debrief: A Critical Review of Healthcare Simulation Debriefing Methods. Simul Healthc. 2016 Jun;11(3):209-17. doi: 10.1097/SIH.0000000000000148. **PMID:** 27254527 **Citation:** Schulz KF, Altman DG, Moher D; CONSORT Group. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. Int J Surg. 2011;9(8):672-7. doi: 10.1016/j.ijsu.2011.09.004. Epub 2011 Oct 13. No abstract available. **PMID:** 22019563 **Citation:** Perkins GD, Graesner JT, Semeraro F, Olasveengen T, Soar J, Lott C, Van de Voorde P, Madar J, Zideman D, Mentzelopoulos S, Bossaert L, Greif R, Monsieurs K, Svavarsdottir H, Nolan JP; European Resuscitation Council Guideline Collaborators. European Resuscitation Council Guidelines 2021: Executive summary. Resuscitation. 2021 Apr;161:1-60. doi: 10.1016/j.resuscitation.2021.02.003. Epub 2021 Mar 24. Erratum In: Resuscitation. 2021 May 4;163:97-98. **PMID:** 33773824 **Citation:** Sherwood RJ, Francis G. The effect of mannequin fidelity on the achievement of learning outcomes for nursing, midwifery and allied healthcare practitioners: Systematic review and meta-analysis. Nurse Educ Today. 2018 Oct;69:81-94. doi: 10.1016/j.nedt.2018.06.025. Epub 2018 Jun 30. **PMID:** 30015220 **Citation:** Shorey S, Pereira TL, Teo WZ, Ang E, Lau TC, Samarasekera DD. Navigating nursing curriculum change during COVID-19 pandemic: A systematic review and meta-synthesis. Nurse Educ Pract. 2022 Nov;65:103483. doi: 10.1016/j.nepr.2022.103483. Epub 2022 Oct 27. **PMID:** 36327596 **Citation:** Shrestha R, Badyal D, Shrestha AP, Shrestha A. In-situ Simulation-Based Module to Train Interns in Resuscitation Skills During Cardiac Arrest. Adv Med Educ Pract. 2020 Apr 8;11:271-285. doi: 10.2147/AMEP.S246920. eCollection 2020. **PMID:** 32308520 **Citation:** Sindiani AM, Obeidat N, Alshdaifat E, Elsalem L, Alwani MM, Rawashdeh H, Fares AS, Alalawne T, Tawalbeh LI. Distance education during the COVID-19 outbreak: A cross-sectional study among medical students in North of Jordan. Ann Med Surg (Lond). 2020 Nov;59:186-194. doi: 10.1016/j.amsu.2020.09.036. Epub 2020 Oct 2. **PMID:** 33042535 **Citation:** Sorensen JL, Ostergaard D, LeBlanc V, Ottesen B, Konge L, Dieckmann P, Van der Vleuten C. Design of simulation-based medical education and advantages and disadvantages of in situ simulation versus off-site simulation. BMC Med Educ. 2017 Jan 21;17(1):20. doi: 10.1186/s12909-016-0838-3. **PMID:** 28109296 **Citation:** Staerk M, Vammen L, Andersen CF, Krogh K, Lofgren B. Basic life support skills can be improved among certified basic life support instructors. Resusc Plus. 2021 Apr 14;6:100120. doi: 10.1016/j.resplu.2021.100120. eCollection 2021 Jun. **PMID:** 34223379 **Citation:** Suliman WA, Abu-Moghli FA, Khalaf I, Zumot AF, Nabolsi M. Experiences of nursing students under the unprecedented abrupt online learning format forced by the national curfew due to COVID-19: A qualitative research study. Nurse Educ Today. 2021 May;100:104829. doi: 10.1016/j.nedt.2021.104829. Epub 2021 Feb 27. **PMID:** 33740705 **Citation:** Tawalbeh LI, Tubaishat A. Effect of simulation on knowledge of advanced cardiac life support, knowledge retention, and confidence of nursing students in Jordan. J Nurs Educ. 2014 Jan 1;53(1):38-44. doi: 10.3928/01484834-20131218-01. Epub 2013 Dec 18. **PMID:** 24328248 **Citation:** Teoh SE, Masuda Y, Tan DJH, Liu N, Morrison LJ, Ong MEH, Blewer AL, Ho AFW. Impact of the COVID-19 pandemic on the epidemiology of out-of-hospital cardiac arrest: a systematic review and meta-analysis. Ann Intensive Care. 2021 Dec 7;11(1):169. doi: 10.1186/s13613-021-00957-8. **PMID:** 34874498 **Citation:** Thompson Bastin ML, Cook AM, Flannery AH. Use of simulation training to prepare pharmacy residents for medical emergencies. Am J Health Syst Pharm. 2017 Mar 15;74(6):424-429. doi: 10.2146/ajhp160129. **PMID:** 28274986 **Citation:** Torsy T, Deswarte W, Karlberg Traav M, Beeckman D. Effect of a dynamic mattress on chest compression quality during cardiopulmonary resuscitation. Nurs Crit Care. 2022 Mar;27(2):275-281. doi: 10.1111/nicc.12631. Epub 2021 Apr 21. **PMID:** 33884701 **Citation:** Toubasi S, Alosta MR, Darawad MW, Demeh W. Impact of simulation training on Jordanian nurses' performance of basic life support skills: A pilot study. Nurse Educ Today. 2015 Sep;35(9):999-1003. doi: 10.1016/j.nedt.2015.03.017. Epub 2015 Apr 16. **PMID:** 25935665 **Citation:** Umuhoza C, Chen L, Unyuzumutima J, McCall N. Impact of structured basic life-support course on nurses' cardiopulmonary resuscitation knowledge and skills: Experience of a paediatric department in low-resource country. Afr J Emerg Med. 2021 Sep;11(3):366-371. doi: 10.1016/j.afjem.2021.03.014. Epub 2021 Jul 22. **PMID:** 34367898 **Citation:** Uzen Cura S, Kocatepe V, Yildirim D, Kucukakgun H, Atay S, Unver V. Examining Knowledge, Skill, Stress, Satisfaction, and Self-Confidence Levels of Nursing Students in Three Different Simulation Modalities. Asian Nurs Res (Korean Soc Nurs Sci). 2020 Aug;14(3):158-164. doi: 10.1016/j.anr.2020.07.001. Epub 2020 Jul 9. **PMID:** 32653666 **Citation:** Van de Voorde P, Turner NM, Djakow J, de Lucas N, Martinez-Mejias A, Biarent D, Bingham R, Brissaud O, Hoffmann F, Johannesdottir GB, Lauritsen T, Maconochie I. European Resuscitation Council Guidelines 2021: Paediatric Life Support. Resuscitation. 2021 Apr;161:327-387. doi: 10.1016/j.resuscitation.2021.02.015. Epub 2021 Mar 24. **PMID:** 33773830 **Citation:** Waldner MH, Olson JK. Taking the patient to the classroom: applying theoretical frameworks to simulation in nursing education. Int J Nurs Educ Scholarsh. 2007;4:Article18. doi: 10.2202/1548-923X.1317. Epub 2007 Sep 17. **PMID:** 17910532 **Citation:** Williams KL, Rideout J, Pritchett-Kelly S, McDonald M, Mullins-Richards P, Dubrowski A. Mock Code: A Code Blue Scenario Requested by and Developed for Registered Nurses. Cureus. 2016 Dec 23;8(12):e938. doi: 10.7759/cureus.938. **PMID:** 28123919 **Citation:** Wilson C, Furness E, Proctor L, Sweetman G, Hird K. A randomised trial of the effectiveness of instructor versus automated manikin feedback for training junior doctors in life support skills. Perspect Med Educ. 2021 Mar;10(2):95-100. doi: 10.1007/s40037-020-00631-y. Epub 2020 Nov 26. **PMID:** 33242153 **Citation:** Wyckoff MH, Singletary EM, Soar J, Olasveengen TM, Greif R, Liley HG, Zideman D, Bhanji F, Andersen LW, Avis SR, Aziz K, Bendall JC, Berry DC, Borra V, Bottiger BW, Bradley R, Bray JE, Breckwoldt J, Carlson JN, Cassan P, Castren M, Chang WT, Charlton NP, Cheng A, Chung SP, Considine J, Costa-Nobre DT, Couper K, Dainty KN, Davis PG, de Almeida MF, de Caen AR, de Paiva EF, Deakin CD, Djarv T, Douma MJ, Drennan IR, Duff JP, Eastwood KJ, El-Naggar W, Epstein JL, Escalante R, Fabres JG, Fawke J, Finn JC, Foglia EE, Folke F, Freeman K, Gilfoyle E, Goolsby CA, Grove A, Guinsburg R, Hatanaka T, Hazinski MF, Heriot GS, Hirsch KG, Holmberg MJ, Hosono S, Hsieh MJ, Hung KKC, Hsu CH, Ikeyama T, Isayama T, Kapadia VS, Kawakami MD, Kim HS, Kloeck DA, Kudenchuk PJ, Lagina AT, Lauridsen KG, Lavonas EJ, Lockey AS, Malta Hansen C, Markenson D, Matsuyama T, McKinlay CJD, Mehrabian A, Merchant RM, Meyran D, Morley PT, Morrison LJ, Nation KJ, Nemeth M, Neumar RW, Nicholson T, Niermeyer S, Nikolaou N, Nishiyama C, O'Neil BJ, Orkin AM, Osemeke O, Parr MJ, Patocka C, Pellegrino JL, Perkins GD, Perlman JM, Rabi Y, Reynolds JC, Ristagno G, Roehr CC, Sakamoto T, Sandroni C, Sawyer T, Schmolzer GM, Schnaubelt S, Semeraro F, Skrifvars MB, Smith CM, Smyth MA, Soll RF, Sugiura T, Taylor-Phillips S, Trevisanuto D, Vaillancourt C, Wang TL, Weiner GM, Welsford M, Wigginton J, Wyllie JP, Yeung J, Nolan JP, Berg KM; COVID-19 Working Group. 2021 International Consensus on Cardiopulmonary Resuscitation and Emergency Cardiovascular Care Science With Treatment Recommendations: Summary From the Basic Life Support; Advanced Life Support; Neonatal Life Support; Education, Implementation, and Teams; First Aid Task Forces; and the COVID-19 Working Group. Resuscitation. 2021 Dec;169:229-311. doi: 10.1016/j.resuscitation.2021.10.040. Epub 2021 Nov 11. **PMID:** 34933747 **Citation:** Zideman DA, Singletary EM, Borra V, Cassan P, Cimpoesu CD, De Buck E, Djarv T, Handley AJ, Klaassen B, Meyran D, Oliver E, Poole K. European Resuscitation Council Guidelines 2021: First aid. Resuscitation. 2021 Apr;161:270-290. doi: 10.1016/j.resuscitation.2021.02.013. Epub 2021 Mar 24. **PMID:** 33773828 #### See Also Links **Label:** The Effect of Medium Fidelity Simulation on Perceived Competence Level of Novice Nursing Students **URL:** http://search.ebscohost.com/login.aspx?direct=true&db=jlh&AN=109860356&lang=es&site=ehost-live **Label:** Effects of in-situ simulation training intervention on Intensive Care Unit and Emergency Department nurses ' competency and confidence to ability to perform cardio- pulmonary resuscitation **URL:** https://www.theseus.fi/handle/10024/227686 **Label:** Descriptive comparative study of nursing faculty self-efficacy in the simulation setting. Descriptive Comparative Study of Nursing Faculty Self-Efficacy in the Simulation Setting **URL:** http://lib.tcu.edu/PURL/EZproxy_link.asp?url=https://search.ebscohost.com/login.aspx?direct=true&AuthType=cookie,ip,uid&db=ccm&AN=123295984&site=ehost-live **Label:** Brown, D. K., \& Benson, S. N. K. (2020). Does Time in Team Training Matter ? Evaluation of Team-Level Attitudes With Interprofessional Education. Clinical Simulation in Nursing, 48, 38-45 **URL:** https://doi.org/10.1016/j.ecns.2020.08.002 **Label:** To assess the effectiveness of simulation teaching of defibrillation on the knowledge and skill of nurses working in the critical care unit of a Tertiary Care Teaching Institute: Prospective, **URL:** https://doi.org/10.4103/ijcn.ijcn_31_20 **Label:** Simulation and audio-visual learning method for knowledge of cardiac pulmonary resuscitation skills in nursing students. Bali Medical Journal, 10(3Special issue), 1023-1028. https://doi.org/10.15562/bmj.v10i3.2816 **URL:** https://doi.org/10.15562/bmj.v10i3.2816 **Label:** The Effects of Repeated Basic Life Support Training on Teachers' Knowledge and Skill Levels: A Quasi-experimental Study. Eurasian Journal of Emergency Medicine, 19(1), 46-51 **URL:** https://doi.org/10.4274/eajem.galenos.2019.47450 **Label:** A Study on How Beneficial Simulation Experience Is For Nursing Staff's Advanced Cardiac Life Support Knowledge at the New Najran General Hospita. Annals of Case Reports, 6(02) **URL:** https://doi.org/10.29011/2574-7754.100596 **Label:** Purposeful Simulation Role Assignment. Clinical Simulation in Nursing **URL:** https://doi.org/10.1016/j.ecns.2020.07.008 **Label:** Exploring nurses' knowledge of basic life support guideline of american heart association: A local study. Journal of Emergency Practice and Trauma **URL:** https://doi.org/10.34172/jept.2021.02 **Label:** Knowledge and Practice of Basic Life Support ( BLS ) Among Registered Nurse at a Private Hospital in Seremban. 13(January), 58-64 **URL:** https://doi.org/10.31674/mjn.2022.v13i03.009 **Label:** Jawabreh, N., Ayed, A., \& Batran, A. (2019). Effect of High Fidelity Simulation on Nursing Students' Knowledge in Palestine. Open Journal of Nursing, 09(04), 364-369 **URL:** https://doi.org/10.4236/ojn.2019.94033 **Label:** Deliberate Practice in Simulation : Evaluation of Repetitive Code Training on Nursing Students ' BLS Team Skills. Clinical Simulation in Nursing, 48, 8-14 **URL:** https://doi.org/10.1016/j.ecns.2020.08.001 **Label:** High Fidelity Simulation Experience and Development of Clinical Judgment: Recent Graduate Nurses' Reflections. ProQuest Dissertations and Theses, 127. **URL:** https://manchester.idm.oclc.org/login?url=https://search.proquest.com/docview/2070939340?accountid=12253%0Ahttp://man-fe.hosted.exlibrisgroup.com/openurl/44MAN/44MAN_services_page?genre=dissertations+%26+theses&atitle=&author=Lawrence%2C+Martha+Kay&volume **Label:** Quality improvement for self-confidence, critical-thinking, and psychomotor skills in basic life support of nursing health professionals through case-scenario simulation training. Journal of Nursing Education **URL:** https://doi.org/10.5430/jnep.v11n8p23 **Label:** Guidance for Comprehensive Health Care Simulation Program Evaluation. Clinical Simulation in Nursing, 48, 20-28 **URL:** https://doi.org/10.1016/j.ecns.2020.08.003 **Label:** Mcdermott, D. S., \& Ludlow, J. (2022). A Prebriefing Guide for Online , Virtual , or Distant Simulation Experiences. Clinical Simulation in Nursing, 67, 1-5. **URL:** https://doi.org/10.1016/j.ecns.2022.03.004 **Label:** Assessing ventilation skills by nursing students in paediatric and adult basic life support: A crossover randomized simulation study using Bag-Val **URL:** https://doi.org/10.22514/sv.2020.16.0072 **Label:** In Situ Simulation : Challenges and Results (Vol.3: Performance and Tools). Approaches in Patient Safety: New Directions and Alternative Approaches, 1-18 **URL:** https://www.ncbi.nlm.nih.gov/books/NBK43682/pdf/Bookshelf_NBK43682.pdf **Label:** A study to assess knowledge andpractice of basic life support among nurses working in tertiary care hospital, New Delhi, India. Nursing \& Care Open Access Journal, 7(2), 48-52. **URL:** https://doi.org/10.15406/ncoaj.2020.07.00217 **Label:** Assessment of the knowledge level and its relevance in terms of CPR in medical personnel of the hospital emergency medical system of the Autonomous Community of the Region of Murcia **URL:** https://doi.org/10.6018/eglobal.14.3.197791 **Label:** The additional ımpact of simulation based medical training to traditional medical training alone in advanced cardiac life support: A scenario based evaluation **URL:** https://doi.org/10.22514/SV142.102018.10 **Label:** Atualização de trabalhadores de enfermagem em suporte básico de vida. Revista de Enfermagem UFPE on Line, 13 **URL:** https://doi.org/10.5205/1981-8963.2019.241981 **Label:** Suporte básico de vida: Avaliação da aprendizagem com uso de simulação e dispositivos de feedback imediato. Revista Latino-Americana de Enfermagem **URL:** https://doi.org/10.1590/1518-8345.1957.2942 **Label:** Knowledge Level of Healthcare Professionals on Basic and Advanced Life Support in Children. Eurasian Journal of Emergency Medicine **URL:** https://doi.org/10.4274/eajem.galenos.2020.05579 **Label:** Resuscitation and Emergency Cardiovascular Care Aid Task Forces ; and the COVID-19 Working Group International Liaison Committee on Resuscitation. 9. https://doi.org/10.1016/j.resuscitation.2021.10.040 **URL:** https://doi.org/10.1016/j.resuscitation.2021.10.040 **Label:** Knowledge, attitude and practice of basic life support among junior doctors and students in a tertiary care medical institute. International Journal of Research in Medical Sciences **URL:** https://doi.org/10.18203/2320-6012.ijrms20151416 **Label:** High Fidelity Simulation Improves Provider Confidence During ACLS Training Even Among Experienced Staff: Are We Missing an Opportunity? Emergency Medicine **URL:** https://doi.org/10.17140/emoj-2-120 ## Document Section ### Large Document Module #### Large Docs - Date: 2023-04-20 - Filename: Prot_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: False - Label: Study Protocol - Size: 629932 - Type Abbrev: Prot - Upload Date: 2023-08-16T00:40 - Date: 2023-04-20 - Filename: SAP_001.pdf - Has ICF: False - Has Protocol: False - Has SAP: True - Label: Statistical Analysis Plan - Size: 546211 - Type Abbrev: SAP - Upload Date: 2023-08-16T00:42 - Date: 2023-04-20 - Filename: ICF_002.pdf - Has ICF: True - Has Protocol: False - Has SAP: False - Label: Informed Consent Form - Size: 488560 - Type Abbrev: ICF - Upload Date: 2023-08-12T02:29 ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05705479 **Acronym:** STABLE-2 **Brief Title:** Shoulder Instability Trial Comparing Arthroscopic Stabilization Benefits Compared With Latarjet Procedure Evaluation - STABLE Definitive Trial **Official Title:** Shoulder Instability Trial Comparing Arthroscopic Stabilization Benefits Compared With Latarjet Procedure Evaluation #### Organization Study ID Info **ID:** STABLE-2 #### Organization **Class:** OTHER **Full Name:** McMaster University ### Status Module #### Completion Date **Date:** 2027-05-01 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-10-19 **Type:** ACTUAL **Last Update Submit Date:** 2023-10-18 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2025-05-01 **Type:** ESTIMATED #### Start Date **Date:** 2023-10-17 **Type:** ACTUAL **Status Verified Date:** 2023-10 #### Study First Post Date **Date:** 2023-01-30 **Type:** ACTUAL **Study First Submit Date:** 2023-01-17 **Study First Submit QC Date:** 2023-01-27 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** McMaster University #### Responsible Party **Investigator Affiliation:** McMaster University **Investigator Full Name:** Moin Khan **Investigator Title:** Associate Professor and Orthopaedic Surgeon **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The primary objective of the study is to evaluate the functional shoulder recovery of patients with recurrent shoulder dislocations at 24 months when treated with either arthroscopic capsuloligamentous repair (Bankart Procedure +/- Remplissage) or coracoid transfer (Latarjet procedure). **Detailed Description:** Background: Shoulder is a highly mobile joint with the most directional range of movement compared to other joints in the body. Thus, the surrounding supporting structures of the shoulder joint compromise on the stability of the joint, in order to accomplish this wide range of motion. Anterior dislocations, the most common type of shoulder dislocation, are often complicated by instability, and repeated dislocations. Shoulder instability results in pain and negatively impacts quality of life. Several long-term studies have demonstrated a relationship between the repeated dislocations and the risk of arthritis. Surgical stabilization of the shoulder improves function and may reduce the risk of developing degenerative arthritis. Two procedures are commonly performed in patients with repeated dislocations: a bony transfer procedure (Latarjet) or a soft tissue procedure (Bankart + Remplissage). The Latarjet procedure involves transferring bone to the front of the shoulder. The Bankart + Remplissage procedure involves tightening the soft tissues at the front of the shoulder joint. Although retrospective clinical studies have suggested a reduced recurrence rate with the Latarjet procedure, there is a higher reported complication rate and potential morbidity associated with the open procedure. Several case series from high-volume surgeons in Europe have suggested the Latarjet repair to be an acceptable and potentially favorable surgical approach for all cases of recurrent anterior shoulder dislocation, even in the primary setting and in the absence of significant glenoid cup bone loss. Retrospective analysis of soft tissue repair in comparison to open coracoid (Latarjet) procedure found at 10-year follow up, redislocation rates were 13% (36) of 271 shoulders with a Bankart repair and 1% (1) of the 93 shoulders with a Latarjet repair. Need for a Pilot Study Prior to a Large Trial: No comparative randomized control trial has been completed evaluating Bankart repair in comparison to Latarjet procedure in the setting of mild to moderate bone loss. Thus, surgeons face uncertainty regarding which procedure to perform. The Latarjet is more invasive (larger incision) and some research suggests it may be more effective at treating instability. The Bankart procedure, while minimally invasive (smaller incision), may result in higher rates of instability after surgery. Study Aims and Objectives: The primary objective of the study is to evaluate the functional shoulder recovery of patients with recurrent shoulder dislocations at 24 months when treated with either arthroscopic capsuloligamentous repair (Bankart Procedure +/- Remplissage) or coracoid transfer (Latarjet procedure). Our trial will compare arthroscopic capsuloligamentous repair vs. coracoid transfer (Latarjet procedure) on: 1. Rates of recurrent shoulder dislocations and symptoms of instability up to 24 months' post- surgery; 2. Clinical outcomes measured by American Shoulder and Elbow Society (ASES) score, Shoulder Activity Scale and EQ-5D and Patient Satisfaction Scale; 3. Physical examination: range of motion, strength, stability; 4. Return to previous level of activity; 5. Rate of shoulder-related complications and serious adverse events. Study Design: The investigators propose a multi-center pilot Randomized Clinical Trial of 126 patients across Canada, United States, Chile and Europe to compare the effect of capsuloligamentous repair (Bankart + Remplissage procedure) and coracoid transfer (Latarjet procedure) in patients with post-traumatic recurrent anterior dislocation. Eligible and consenting participants will be followed-up by the site for 24 months. Outcomes will be assessed at 2 weeks, 3 months, 6 months, 12 months, and 24 months post-surgery. Eligible participants will be randomized to one of two treatment groups: Arthroscopic capsuloligamentous repair (Bankart + Remplissage Procedure) Open or Arthroscopic coracoid transfer (Latarjet Procedure) Once participants have provided informed consent, baseline demographics, relevant medical history, and details regarding their diagnosis will be collected from the participant, the attending surgeon, their medical record and through physical examination. Participants will also complete The Western Ontario Shoulder Instability Index (WOSI) and he American Shoulder and Elbow Surgeons questionnaire (ASES) at the time of enrolment. After surgery, surgical and peri-operative details will be collected from the attending surgeon and the participant's medical records. Adverse events occurring during the surgical procedure or perioperative period will also be documented. ### Conditions Module **Conditions:** - Shoulder Dislocation - Sport Injuries - Anterior Dislocation ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 126 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Bankart Procedure: the participant will be placed in the lateral decubitus or beach chair position. Standard diagnostic arthroscopy will be performed. The anterior capsulolabral complex will be freed from the anterior aspect of the scapular neck. The anterior aspect of the scapular neck will be decorticated using a motorized burr. A capsuloligamentous repair will be performed with the capsule shifted from inferior to superior and repaired on the glenoid face. The number of anchors used for the repair will be left to the discretion of the surgeon. Patients will be given a sling for 4 weeks, and participation in sports will not be allowed for 6 months. **Intervention Names:** - Procedure: Bankart + Remplissage Procedure **Label:** Bankart + Remplissage Procedure **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Open or Arthroscopic coracoid transfer (Latarjet Procedure): This procedure may be performed through small incisions (minimally invasive) but may require a larger incision in some cases. It involves the transfer of a nearby bony structure (the coracoid process) to the front of the shoulder joint (glenoid). This bone will then provide support to prevent the shoulder joint from dislocating. **Intervention Names:** - Procedure: Latarjet Procedure **Label:** Latarjet Procedure **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Bankart + Remplissage Procedure **Description:** Participants will undergo arthroscopic stabilization. **Name:** Bankart + Remplissage Procedure **Type:** PROCEDURE #### Intervention 2 **Arm Group Labels:** - Latarjet Procedure **Description:** Participants will undergo open or arthroscopic Latarjet procedure. **Name:** Latarjet Procedure **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** The change from baseline to 24 months post-intervention difference in Western Ontario Shoulder Instability Index (WOSI) scores between patients with recurrent dislocations treated with Bankart Procedure + Remplissage versus Latarjet procedure. **Measure:** Clinical Outcome - Shoulder function **Time Frame:** 2 years #### Secondary Outcomes **Description:** Rate of recurrent dislocation and symptomatic instability between patients randomized to (capsuloligamentous repair + remplissage) and those receiving open Latarjet procedure. **Measure:** Rate of Recurrence **Time Frame:** 2 years **Description:** Measured by ASES questionnaire (American Shoulder and Elbow Surgeons) **Measure:** Clinical Outcome **Time Frame:** 2 years **Description:** Measured by EQ-5D **Measure:** Clinical Outcome - Quality of Life **Time Frame:** 2 years **Description:** Measured by Shoulder Activity Scale **Measure:** Clinical Outcome - Shoulder activity **Time Frame:** 2 years **Description:** Measured by apprehension-relocation physical examination maneuver **Measure:** Rate of participants with anterior shoulder instability **Time Frame:** 2 years **Description:** Assessed in forward flexion, abduction, external rotation and internal rotation. **Measure:** Rate of participants with full range of motion **Time Frame:** 2 years **Description:** Return to previous level of activity and sport (self reported) **Measure:** Rate of participants that return to previous level of activity **Time Frame:** 2 years **Description:** Adverse events **Measure:** Rate of major and minor shoulder-related complications and serious adverse events **Time Frame:** 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Men and women ages 18-50 years; 2. Diagnosis of post-traumatic recurrent anterior dislocation. This will require a minimum of 2 episodes of documented dislocations either by radiographic evidence or documented reduction of anterior shoulder dislocation as well as physical examination eliciting unwanted glenohumeral translation with reproduction of symptoms; 3. Mild glenoid bone loss as defined on CT by standardized and reproducible best-fit circle technique (\>10% but \<20%); 4. Provision of informed consent. Exclusion Criteria: 1. Patients with concomitant injuries (cuff tear); 2. Previous shoulder surgery; 3. Patients that will likely have problems, in the judgment of the investigators, with maintaining follow-up; 4. Epilepsy/seizure disorder; 5. Patients who are or at risk of being incarcerated; 6. Diagnosis of multidirectional instability; 7. Cases involving litigation or workplace insurance claims (e.g. WSIB); 8. Confirmed connective tissue disorder (Ehlers-Danlos, Marfans) or Beighton hypermobility score \> 6. 9. Pregnancy. **Maximum Age:** 50 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** mgarrido@stjoes.ca **Name:** Miriam Garrido Clua, MSc **Phone:** khanmm2@mcmaster.ca **Phone Ext:** 32953 **Role:** CONTACT **Contact 2:** **Email:** khanmm2@mcmaster.ca **Name:** Moin Khan, MD **Phone:** 905-522-1155 **Phone Ext:** 32953 **Role:** CONTACT #### Locations **Location 1:** **City:** Hamilton **Contacts:** ***Contact 1:*** - **Email:** mgarrido@stjoes.ca - **Name:** Miriam Garrido Clua, MSc - **Role:** CONTACT ***Contact 2:*** - **Name:** Moin Khan, MD, MSc,FRCSC - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Canada **Facility:** St. Joseph's Healthcare Hamilton **State:** Ontario **Status:** RECRUITING **Zip:** L8N 4A6 #### Overall Officials **Official 1:** **Affiliation:** McMaster University **Name:** Moin Khan, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007592 - Term: Joint Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000014947 - Term: Wounds and Injuries - ID: D000070599 - Term: Shoulder Injuries ### Condition Browse Module - Browse Branches - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: All - Name: All Conditions - Abbrev: BC05 - Name: Musculoskeletal Diseases ### Condition Browse Module - Browse Leaves - ID: M4570 - Name: Athletic Injuries - Relevance: HIGH - As Found: Sport Injury - ID: M15591 - Name: Shoulder Dislocation - Relevance: HIGH - As Found: Shoulder Dislocation - ID: M7385 - Name: Joint Dislocations - Relevance: HIGH - As Found: Dislocation - ID: M10621 - Name: Joint Diseases - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown - ID: M602 - Name: Shoulder Injuries - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000004204 - Term: Joint Dislocations - ID: D000012783 - Term: Shoulder Dislocation - ID: D000001265 - Term: Athletic Injuries ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01683279 **Brief Title:** A Pediatric Trial of Genetically Modified Autologous T Cells Directed Against CD19 for Relapsed CD19+ Acute Lymphoblastic Leukemia **Official Title:** Pediatric Leukemia Adoptive Therapy (PLAT)-01: A Phase 1 Feasibility and Safety Study of Cellular Immunotherapy for Relapsed Pediatric CD19+ Acute Lymphoblastic Leukemia Using Autologous T-cells Lentivirally Transduced To Express a CD19-Specific Chimeric Antigen Receptor #### Organization Study ID Info **ID:** PLAT-01 #### Organization **Class:** OTHER **Full Name:** Seattle Children's Hospital ### Status Module #### Completion Date **Date:** 2030-01-07 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-06-29 **Type:** ACTUAL **Last Update Submit Date:** 2023-06-27 **Overall Status:** ACTIVE_NOT_RECRUITING #### Primary Completion Date **Date:** 2015-01-07 **Type:** ACTUAL #### Start Date **Date:** 2012-03-25 **Type:** ACTUAL **Status Verified Date:** 2023-06 #### Study First Post Date **Date:** 2012-09-11 **Type:** ESTIMATED **Study First Submit Date:** 2012-09-07 **Study First Submit QC Date:** 2012-09-07 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Seattle Children's Hospital #### Responsible Party **Investigator Affiliation:** Seattle Children's Hospital **Investigator Full Name:** Colleen Annesley **Investigator Title:** Medical Director, Seattle Children's Therapeutics **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** True ### Description Module **Brief Summary:** Patients with relapsed leukemia often develop resistance to chemotherapy. For this reason, we are attempting to use a patient's own T cells, which can be genetically modified to expresses a chimeric antigen receptor(CAR). The CAR enables the T cell to recognize and kill the leukemic cells though the recognition of CD19, a protein expressed on the surface of the majority of pediatric ALL. This is a phase I study designed to determine the maximum tolerated dose of the CAR+ T cells and define the toxicity of the treatment. As a secondary aim, we will be looking at the efficacy of the T cells on eradicating the patient's leukemic cells. **Detailed Description:** Upon meeting the eligibility requirements and enrolling on study, subjects will undergo a blood draw to obtain the T cells for the generation of the CD19 CAR+ T cells. The T cells are isolated from the blood, transduced with a lentivirus to express the CD19 CAR, and expanded in culture over a three week period. During the process of cell generation, subjects will continue to be cared for by their primary oncologist and may undergo additional treatment directed at the leukemia during this time. After the CAR+ T cells have been generated, the subject undergoes a disease assessment and will be admitted to the hospital to receive 2 days of cyclophosphamide for lymphodepletion and reduction of disease burden. Several days later, the subject will receive an infusion of the CAR+ T cells. Following treatment with the CAR+ T cells, subjects will be intensely followed for 6 weeks with serial blood testing and re-evaluation of disease status with bone marrow aspirates. After 6 weeks, the subjects clinical care will be resumed by their primary oncologist, and it is possible that they would receive additionally chemotherapy or a stem cell transplant. Upon completion of the study, subjects will be followed at least annually with a either a medical history, physical exam and blood tests or a phone call/questionnaire for 15 years. This follow up will help to determine if the subject develops any long-term health problems related to the CAR+ T cells including a new cancer. ### Conditions Module **Conditions:** - B Cell Leukemia **Keywords:** - pediatric - acute lymphoblastic leukemia - CD19 - Chimeric Antigen Receptor ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 6 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Subjects will receive two days of cyclophosphamide for a total of 3g/m\^2 followed several days later by a single dose of Autologous CD19 CAR+ EGFTt + T cells **Intervention Names:** - Biological: Autologous CD19 CAR+ EGFTt + T cells **Label:** CAR+ T cells **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - CAR+ T cells **Description:** Autologous T cell modified to express a CD19 specific CAR and a truncated EGFRt tag **Name:** Autologous CD19 CAR+ EGFTt + T cells **Type:** BIOLOGICAL ### Outcomes Module #### Primary Outcomes **Description:** The safety of the T cell infusion will be described and the maximum tolerated dose determined. **Measure:** Number of Participant with Adverse Events **Time Frame:** 42 days #### Secondary Outcomes **Description:** Patients will be followed for 42 days to determine if the transferred T cells remain detectable in the blood and bone marrow **Measure:** Persistence of the CD19 CAR+ T cells **Time Frame:** 42 days **Description:** Patients will have their bone marrow assessed following the T cell infusion to determine if their disease responded to the treatment **Measure:** Determine if there is anti-leukemic activity of the CD19 CAR+ T cells **Time Frame:** 42 days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * CD19+ Leukemia in 1st marrow relapse with MRD at the end of 1st month of re-induction * CD19+ Leukemia in 2nd or greater relapse * CD19+ Leukemia with indication for HCT, but has contraindication * Age between 1 and 26 years of age * Karnofsky of \>50 or Lansky \>50 * Life Expectancy \>12 weeks * Able to tolerate a blood draw of 4-6mL/kg * Recovered from acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy * absolute lymphocyte count of \>/=750 cell/mm3 or \>/=500 is \>20kg * creatinine clearance or radioisotope GFR \>/= 70mL/min/1.73m2 OR normal serum creatinine based on age/gender * total bilirubin \</= 1.5x upper limit normal OR direct bilirubin \</= 1.5mg/dl * ALT \</= 3x upper limit normal * corrected QTc \<450msec of ECG * Shortening Fraction \>28% by ECHO or Ejection Fraction \>50% by MUGA * Documented negative HIV, Hep B and Hep C * Agree to long-term follow up for up to 15 years if they receive T cell infusion Exclusion Criteria: * Philadelphia Positive Leukemia * Prior Allogeneic Stem Cell Transplant * CNS 2 or 3 * prior cellular immunotherapy with chimeric antigen receptor modified T cells * fully humanized antibodies within three half lives * systemic corticosteroids within 7 days of enrollment * requires supplemental oxygen or has a chest X-ray with an infectious process * CNS pathology (seizure disorder, paresis, aphasia, cerebrovascular ischemia/hemorrhage, severe brain injuries, dementia, cerebellar disease, organic brain syndrome, psychosis, coordination or movement disorder) * Pregnant or breastfeeding women. Female participant of reproductive age must have a negative pregnancy test and agree to contraception for 1 year after T cell infusion. * Active Malignancy other than CD19+ Leukemia * Active severe infection defined as a positive blood culture within 48 hours of study enrollment or a fever \>38.2C AND clinical signs of infection within 48 hours of study enrollment * Patient has a concurrent medical condition, that in the opinion of the protocol PI or designee, would prevent the patient from undergoing protocol-based therapy. * Trisomy 21 * Primary immunodeficiency/bone marrow failure syndrome **Maximum Age:** 26 Years **Minimum Age:** 1 Year **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Seattle **Country:** United States **Facility:** Seattle Children's Hospital **State:** Washington **Zip:** 98105 #### Overall Officials **Official 1:** **Affiliation:** Seattle Children's Hospital **Name:** Rebecca Gardner, MD **Role:** STUDY_CHAIR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000006402 - Term: Hematologic Diseases - ID: D000008232 - Term: Lymphoproliferative Disorders - ID: D000008206 - Term: Lymphatic Diseases - ID: D000007160 - Term: Immunoproliferative Disorders - ID: D000007154 - Term: Immune System Diseases - ID: D000002908 - Term: Chronic Disease - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC15 - Name: Blood and Lymph Conditions - Abbrev: All - Name: All Conditions - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10945 - Name: Leukemia - Relevance: HIGH - As Found: Leukemia - ID: M10951 - Name: Leukemia, Lymphoid - Relevance: HIGH - As Found: Lymphoblastic Leukemia - ID: M18116 - Name: Leukemia, Lymphocytic, Chronic, B-Cell - Relevance: HIGH - As Found: B Cell Leukemia - ID: M27585 - Name: Precursor Cell Lymphoblastic Leukemia-Lymphoma - Relevance: HIGH - As Found: Acute Lymphoblastic Leukemia - ID: M18115 - Name: Leukemia, B-Cell - Relevance: HIGH - As Found: B Cell Leukemia - ID: M11220 - Name: Lymphoma - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M9490 - Name: Hematologic Diseases - Relevance: LOW - As Found: Unknown - ID: M11225 - Name: Lymphoproliferative Disorders - Relevance: LOW - As Found: Unknown - ID: M11203 - Name: Lymphatic Diseases - Relevance: LOW - As Found: Unknown - ID: M10206 - Name: Immunoproliferative Disorders - Relevance: LOW - As Found: Unknown - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown - ID: M6147 - Name: Chronic Disease - Relevance: LOW - As Found: Unknown - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown - ID: T1308 - Name: Chronic Lymphocytic Leukemia - Relevance: HIGH - As Found: B Cell Leukemia - ID: T175 - Name: Acute Lymphoblastic Leukemia - Relevance: HIGH - As Found: Acute Lymphoblastic Leukemia - ID: T3533 - Name: Lymphoblastic Lymphoma - Relevance: HIGH - As Found: Acute Lymphoblastic Leukemia - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown - ID: T3543 - Name: Lymphosarcoma - Relevance: LOW - As Found: Unknown - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007938 - Term: Leukemia - ID: D000054198 - Term: Precursor Cell Lymphoblastic Leukemia-Lymphoma - ID: D000007945 - Term: Leukemia, Lymphoid - ID: D000015448 - Term: Leukemia, B-Cell - ID: D000015451 - Term: Leukemia, Lymphocytic, Chronic, B-Cell ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: ARhu - Name: Antirheumatic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M6727 - Name: Cyclophosphamide - Relevance: LOW - As Found: Unknown - ID: M2853 - Name: Immunomodulating Agents - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00454779 **Brief Title:** PARTNER: Panitumumab Added to Regimen for Treatment of Head aNd Neck Cancer Evaluation of Response **Official Title:** A Randomized, Open-Label, Controlled, Phase II Trial of Combination Chemotherapy With or Without Panitumumab as First-line Treatment of Subjects With Metastatic or Recurrent Head and Neck Cancer, and Cross-over Second-line Panitumumab Monotherapy of Subjects Who Fail the Combination Chemotherapy… #### Organization Study ID Info **ID:** 20050236 #### Organization **Class:** INDUSTRY **Full Name:** Amgen ### Status Module #### Completion Date **Date:** 2014-01-01 **Type:** ACTUAL #### Disp First Post Date **Date:** 2011-08-23 **Type:** ESTIMATED **Disp First Submit Date:** 2011-08-16 **Disp First Submit QC Date:** 2011-08-16 #### Expanded Access Info #### Last Update Post Date **Date:** 2018-10-17 **Type:** ACTUAL **Last Update Submit Date:** 2018-09-20 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2012-06-11 **Type:** ACTUAL #### Results First Post Date **Date:** 2014-02-07 **Type:** ESTIMATED **Results First Submit Date:** 2013-10-11 **Results First Submit QC Date:** 2014-01-08 #### Start Date **Date:** 2007-01-01 **Type:** ACTUAL **Status Verified Date:** 2018-09 #### Study First Post Date **Date:** 2007-04-02 **Type:** ESTIMATED **Study First Submit Date:** 2007-03-29 **Study First Submit QC Date:** 2007-03-29 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Amgen #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** This is a randomized, open-label, 2-arm, controlled, phase 2, multi-center, estimation clinical trial of docetaxel and cisplatin combination chemotherapy with and without panitumumab in the first-line treatment of subjects with metastatic or recurrent head and neck cancer, as well as a cross-over second-line panitumumab monotherapy of subjects who fail the chemotherapy only arm. This study will be conducted in the United States. Approximately 150 subjects with histologically or cytologically confirmed metastatic and/or recurrent SCCHN. ### Conditions Module **Conditions:** - Metastatic or Recurrent Squamous Cell Carcinoma of Head and Neck **Keywords:** - SCCHNC - Metastatic - Recurrent ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 113 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Panitumumab + Docetaxel + Cisplatin **Intervention Names:** - Drug: Panitumumab - Drug: Cisplatin - Drug: Docetaxel **Label:** Arm 1 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** control **Intervention Names:** - Drug: Cisplatin - Drug: Docetaxel **Label:** Arm 2 **Type:** OTHER ### Interventions #### Intervention 1 **Arm Group Labels:** - Arm 2 **Description:** chemotherapy arm **Name:** Cisplatin **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Arm 1 **Description:** experimental arm **Name:** Panitumumab **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - Arm 2 **Description:** chemotherapy arm **Name:** Docetaxel **Type:** DRUG #### Intervention 4 **Arm Group Labels:** - Arm 1 **Description:** experimental arm **Name:** Cisplatin **Type:** DRUG #### Intervention 5 **Arm Group Labels:** - Arm 1 **Description:** experimental arm **Name:** Docetaxel **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** The time from the date of randomization to the date of first disease progression determined by the investigators per modified RECIST v1.0, or death within 60 days after the last evaluable tumor assessment or randomization date (whichever is later) during the first-line treatment phase. **Measure:** Progression Free Survival (PFS) During the First-line Treatment Phase **Time Frame:** Every 6 weeks until disease progression or death, up to 67 months #### Secondary Outcomes **Description:** Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter (SLD) of target lesions from baseline; Overall Response (OR) = CR + PR. An overall response of CR or PR must be confirmed at least 4 weeks after the criteria for response are first met. ORR is the percentage of subjects with an overall response among the analysis population. **Measure:** Overall Response Rate (ORR) During the First-line Treatment Phase **Time Frame:** Every 6 weeks until disease progression or death, up to 67 months **Description:** Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter (SLD) of target lesions from baseline; Disease Progression (PD), \>=20% increase in the SLD of target lesions from nadir; Stable Disease (SD), Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. An overall response of CR or PR must be confirmed at least 4 weeks after the criteria for response are first met. A best overall response of SD requires a visit response of SD or better no earlier than 35 days after randomization. RCD is the percentage of subjects with a best overall response of CR, PR or SD among the analysis population. **Measure:** Rate of Disease Control (RDC) During the First-line Treatment Phase **Time Frame:** Every 6 weeks until disease progression or death, up to 67 months **Description:** Calculated only for the subset of subjects who have an overall response of CR or PR while on first-line treatment phase (subsequently confirmed at least 4 weeks thereafter), and is defined as time from the first CR or PR to the first observed disease progression by a modified RECIST v1.0. Subjects not meeting the criteria for progression by the analysis data cutoff date will be censored at their last evaluable disease assessment date. **Measure:** Duration of Response (DOR) During the First-line Treatment Phase **Time Frame:** Every 6 weeks until disease progression or death, up to 67 months **Description:** Time from the date of randomization to the first CR or PR during first line treatment phase (subsequently confirmed at least 4 weeks thereafter) **Measure:** Time to Response (TTR) During the First-line Treatment Phase **Time Frame:** Every 6 weeks until disease progression or death, up to 67 months **Description:** Time from the date of randomization to the date of death during the entire study **Measure:** Overall Survival (OS) for the First-line Treatment **Time Frame:** Until death, up to 67 months **Description:** The time from the first dose of panitumumab monotherapy to the date of first disease progression determined by the investigators per modified RECIST v1.0, or death within 60 days after the last evaluable tumor assessment or the second-line first dose date (whichever is later) during the second-line treatment phase. **Measure:** Progression Free Survival (PFS) During the Second-line Treatment Phase **Time Frame:** Every 6 weeks until disease progression or death, up to 57 months **Description:** Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter (SLD) of target lesions from baseline; Overall Response (OR) = CR + PR. An overall response of CR or PR must be confirmed at least 4 weeks after the criteria for response are first met. ORR is the percentage of subjects with an overall response among the analysis population. **Measure:** Overall Response Rate (ORR) During the Second-line Treatment Phase **Time Frame:** Every 6 weeks until disease progression or death, up to 57 months **Description:** Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter (SLD) of target lesions from baseline; Disease Progression (PD), \>=20% increase in the SLD of target lesions from nadir; Stable Disease (SD), Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. An overall response of CR or PR must be confirmed at least 4 weeks after the criteria for response are first met. A best overall response of SD requires a visit response of SD or better no earlier than 35 days after the first dose date in second-line treatment. RCD is the percentage of subjects with a best overall response of CR, PR or SD among the analysis population. **Measure:** Rate of Disease Control (RDC) During the Second-line Treatment Phase **Time Frame:** Every 6 weeks until disease progression or death, up to 57 months **Description:** Time from the first CR or PR to the first observed disease progression by a modified RECIST v1.0. Subjects not meeting the criteria for progression by the analysis data cutoff date will be censored at their last evaluable disease assessment date. **Measure:** Duration of Response (DOR) During the Second-line Treatment Phase **Time Frame:** Every 6 weeks until disease progression or death, up to 57 months **Description:** Time from the first dose of panitumumab monotherapy to the first CR or PR during second-line treatment phase (subsequently confirmed at least 4 weeks thereafter) **Measure:** Time to Response (TTR) During the Second-line Treatment Phase **Time Frame:** Every 6 weeks until disease progression or death, up to 57 months **Description:** Time from the first dose of panitumumab monotherapy to the date of death during the entire study **Measure:** Overall Survival (OS) for the Second-line Treatment **Time Frame:** Until death, up to 57 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Histologically or cytologically confirmed metastatic and/or recurrent Squamous Cell Carcinoma of Head and Neck (SCCHN) determined to be incurable by surgery and/or radiation therapy. * Measurable disease by CT scan * Eastern Cooperative Oncology Group (ECOG) performance score of 0 or 1 * Age: 18 years or older * Adequate hematologic, renal, metabolic, hepatic \& thyroid function Exclusion Criteria: * Prior systemic treatment for metastatic and/or recurrent SCCHN * CNS metastases, or nasopharyngeal carcinoma * History of interstitial lung disease * History of another primary cancer * Any co-morbid disease that would increase risk of toxicity * Active infection requiring systemic treatment * Prior anti-Epidermal Growth Factor receptor (anti-EGFr) antibody therapy **Maximum Age:** 70 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Tucson **Country:** United States **Facility:** Research Site **State:** Arizona **Zip:** 85715 **Location 2:** **City:** Jonesboro **Country:** United States **Facility:** Research Site **State:** Arkansas **Zip:** 72401 **Location 3:** **City:** Duarte **Country:** United States **Facility:** Research Site **State:** California **Zip:** 91010 **Location 4:** **City:** La Jolla **Country:** United States **Facility:** Research Site **State:** California **Zip:** 92037 **Location 5:** **City:** La Verne **Country:** United States **Facility:** Research Site **State:** California **Zip:** 91750 **Location 6:** **City:** Los Angeles **Country:** United States **Facility:** Research Site **State:** California **Zip:** 90095 **Location 7:** **City:** Santa Cruz **Country:** United States **Facility:** Research Site **State:** California **Zip:** 95065 **Location 8:** **City:** Aurora **Country:** United States **Facility:** Research Site **State:** Colorado **Zip:** 80045 **Location 9:** **City:** Denver **Country:** United States **Facility:** Research Site **State:** Colorado **Zip:** 80210 **Location 10:** **City:** Norwich **Country:** United States **Facility:** Research Site **State:** Connecticut **Zip:** 06360 **Location 11:** **City:** Newark **Country:** United States **Facility:** Research Site **State:** Delaware **Zip:** 19713 **Location 12:** **City:** Washington **Country:** United States **Facility:** Research Site **State:** District of Columbia **Zip:** 20007 **Location 13:** **City:** Washington **Country:** United States **Facility:** Research Site **State:** District of Columbia **Zip:** 20010 **Location 14:** **City:** Washington **Country:** United States **Facility:** Research Site **State:** District of Columbia **Zip:** 20037 **Location 15:** **City:** Washington **Country:** United States **Facility:** Research Site **State:** District of Columbia **Zip:** 20422 **Location 16:** **City:** Lakeland **Country:** United States **Facility:** Research Site **State:** Florida **Zip:** 33805 **Location 17:** **City:** Miami **Country:** United States **Facility:** Research Site **State:** Florida **Zip:** 33176 **Location 18:** **City:** New Port Richey **Country:** United States **Facility:** Research Site **State:** Florida **Zip:** 34655 **Location 19:** **City:** Orlando **Country:** United States **Facility:** Research Site **State:** Florida **Zip:** 32806 **Location 20:** **City:** Athens **Country:** United States **Facility:** Research Site **State:** Georgia **Zip:** 30607 **Location 21:** **City:** Griffin **Country:** United States **Facility:** Research Site **State:** Georgia **Zip:** 30224 **Location 22:** **City:** Marietta **Country:** United States **Facility:** Research Site **State:** Georgia **Zip:** 30060 **Location 23:** **City:** Centralia **Country:** United States **Facility:** Research Site **State:** Illinois **Zip:** 62801 **Location 24:** **City:** Elk Grove Village **Country:** United States **Facility:** Research Site **State:** Illinois **Zip:** 60007 **Location 25:** **City:** Evanston **Country:** United States **Facility:** Research Site **State:** Illinois **Zip:** 60201 **Location 26:** **City:** Park Ridge **Country:** United States **Facility:** Research Site **State:** Illinois **Zip:** 60068 **Location 27:** **City:** Zion **Country:** United States **Facility:** Research Site **State:** Illinois **Zip:** 60099 **Location 28:** **City:** Wichita **Country:** United States **Facility:** Research Site **State:** Kansas **Zip:** 67214 **Location 29:** **City:** Ashland **Country:** United States **Facility:** Research Site **State:** Kentucky **Zip:** 41101 **Location 30:** **City:** Lexington **Country:** United States **Facility:** Research Site **State:** Kentucky **Zip:** 40536 **Location 31:** **City:** Louisville **Country:** United States **Facility:** Research Site **State:** Kentucky **Zip:** 40202-1703 **Location 32:** **City:** Louisville **Country:** United States **Facility:** Research Site **State:** Kentucky **Zip:** 40202 **Location 33:** **City:** Paducah **Country:** United States **Facility:** Research Site **State:** Kentucky **Zip:** 42003 **Location 34:** **City:** Baltimore **Country:** United States **Facility:** Research Site **State:** Maryland **Zip:** 21204 **Location 35:** **City:** Baltimore **Country:** United States **Facility:** Research Site **State:** Maryland **Zip:** 21237 **Location 36:** **City:** Frederick **Country:** United States **Facility:** Research Site **State:** Maryland **Zip:** 21701 **Location 37:** **City:** Boston **Country:** United States **Facility:** Research Site **State:** Massachusetts **Zip:** 02114 **Location 38:** **City:** Ann Arbor **Country:** United States **Facility:** Research Site **State:** Michigan **Zip:** 48106-0995 **Location 39:** **City:** Lansing **Country:** United States **Facility:** Research Site **State:** Michigan **Zip:** 48910 **Location 40:** **City:** Pascagoula **Country:** United States **Facility:** Research Site **State:** Mississippi **Zip:** 39581 **Location 41:** **City:** Columbia **Country:** United States **Facility:** Research Site **State:** Missouri **Zip:** 65201 **Location 42:** **City:** Jefferson City **Country:** United States **Facility:** Research Site **State:** Missouri **Zip:** 65109 **Location 43:** **City:** Saint Louis **Country:** United States **Facility:** Research Site **State:** Missouri **Zip:** 63110-1093 **Location 44:** **City:** Saint Louis **Country:** United States **Facility:** Research Site **State:** Missouri **Zip:** 63110 **Location 45:** **City:** Billings **Country:** United States **Facility:** Research Site **State:** Montana **Zip:** 59102 **Location 46:** **City:** Omaha **Country:** United States **Facility:** Research Site **State:** Nebraska **Zip:** 68114 **Location 47:** **City:** Henderson **Country:** United States **Facility:** Research Site **State:** Nevada **Zip:** 59074 **Location 48:** **City:** Hackensack **Country:** United States **Facility:** Research Site **State:** New Jersey **Zip:** 07601 **Location 49:** **City:** Binghamton **Country:** United States **Facility:** Research Site **State:** New York **Zip:** 13905 **Location 50:** **City:** Bronx **Country:** United States **Facility:** Research Site **State:** New York **Zip:** 10457 **Location 51:** **City:** Bronx **Country:** United States **Facility:** Research Site **State:** New York **Zip:** 10467 **Location 52:** **City:** Mineola **Country:** United States **Facility:** Research Site **State:** New York **Zip:** 11501 **Location 53:** **City:** New York **Country:** United States **Facility:** Research Site **State:** New York **Zip:** 10003 **Location 54:** **City:** New York **Country:** United States **Facility:** Research Site **State:** New York **Zip:** 10032 **Location 55:** **City:** Nyack **Country:** United States **Facility:** Research Site **State:** New York **Zip:** 10960 **Location 56:** **City:** Rochester **Country:** United States **Facility:** Research Site **State:** New York **Zip:** 14623 **Location 57:** **City:** Syracuse **Country:** United States **Facility:** Research Site **State:** New York **Zip:** 13210 **Location 58:** **City:** Canton **Country:** United States **Facility:** Research Site **State:** Ohio **Zip:** 44718 **Location 59:** **City:** Cincinnati **Country:** United States **Facility:** Research Site **State:** Ohio **Zip:** 45236 **Location 60:** **City:** Dayton **Country:** United States **Facility:** Research Site **State:** Ohio **Zip:** 45420 **Location 61:** **City:** Toledo **Country:** United States **Facility:** Research Site **State:** Ohio **Zip:** 43614 **Location 62:** **City:** Oklahoma City **Country:** United States **Facility:** Research Site **State:** Oklahoma **Zip:** 73104 **Location 63:** **City:** Langhorne **Country:** United States **Facility:** Research Site **State:** Pennsylvania **Zip:** 19047 **Location 64:** **City:** Philadelphia **Country:** United States **Facility:** Research Site **State:** Pennsylvania **Zip:** 19107 **Location 65:** **City:** Scranton **Country:** United States **Facility:** Research Site **State:** Pennsylvania **Zip:** 18510 **Location 66:** **City:** Charleston **Country:** United States **Facility:** Research Site **State:** South Carolina **Zip:** 29406 **Location 67:** **City:** Charleston **Country:** United States **Facility:** Research Site **State:** South Carolina **Zip:** 29425 **Location 68:** **City:** Greenville **Country:** United States **Facility:** Research Site **State:** South Carolina **Zip:** 29615 **Location 69:** **City:** Memphis **Country:** United States **Facility:** Research Site **State:** Tennessee **Zip:** 38120 **Location 70:** **City:** Arlington **Country:** United States **Facility:** Research Site **State:** Texas **Zip:** 76012 **Location 71:** **City:** Corpus Christi **Country:** United States **Facility:** Research Site **State:** Texas **Zip:** 78405 **Location 72:** **City:** Corpus Christi **Country:** United States **Facility:** Research Site **State:** Texas **Zip:** 78412 **Location 73:** **City:** Dallas **Country:** United States **Facility:** Research Site **State:** Texas **Zip:** 75201 **Location 74:** **City:** Dallas **Country:** United States **Facility:** Research Site **State:** Texas **Zip:** 75230 **Location 75:** **City:** Dallas **Country:** United States **Facility:** Research Site **State:** Texas **Zip:** 75234 **Location 76:** **City:** Galveston **Country:** United States **Facility:** Research Site **State:** Texas **Zip:** 77555-0158 **Location 77:** **City:** San Antonio **Country:** United States **Facility:** Research Site **State:** Texas **Zip:** 78229 **Location 78:** **City:** Abingdon **Country:** United States **Facility:** Research Site **State:** Virginia **Zip:** 24211 **Location 79:** **City:** Chesapeake **Country:** United States **Facility:** Research Site **State:** Virginia **Zip:** 23320 **Location 80:** **City:** Morgantown **Country:** United States **Facility:** Research Site **State:** West Virginia **Zip:** 26506 **Location 81:** **City:** Madison **Country:** United States **Facility:** Research Site **State:** Wisconsin **Zip:** 53792 **Location 82:** **City:** Marshfield **Country:** United States **Facility:** Research Site **State:** Wisconsin **Zip:** 54449 **Location 83:** **City:** Graz **Country:** Austria **Facility:** Research Site **Zip:** 8036 **Location 84:** **City:** Leoben **Country:** Austria **Facility:** Research Site **Zip:** 8700 **Location 85:** **City:** Linz **Country:** Austria **Facility:** Research Site **Zip:** 4010 **Location 86:** **City:** Wien **Country:** Austria **Facility:** Research Site **Zip:** 1090 **Location 87:** **City:** Brasschaat **Country:** Belgium **Facility:** Research Site **Zip:** 2930 **Location 88:** **City:** Brugge **Country:** Belgium **Facility:** Research Site **Zip:** 8000 **Location 89:** **City:** Kortrijk **Country:** Belgium **Facility:** Research Site **Zip:** 8500 **Location 90:** **City:** Libramont **Country:** Belgium **Facility:** Research Site **Zip:** 6800 **Location 91:** **City:** Liege **Country:** Belgium **Facility:** Research Site **Zip:** 4000 **Location 92:** **City:** Ottignies **Country:** Belgium **Facility:** Research Site **Zip:** 1340 **Location 93:** **City:** Wilrijk **Country:** Belgium **Facility:** Research Site **Zip:** 2610 **Location 94:** **City:** Brno **Country:** Czechia **Facility:** Research Site **Zip:** 656 53 **Location 95:** **City:** Praha 5 **Country:** Czechia **Facility:** Research Site **Zip:** 150 06 **Location 96:** **City:** Znojmo **Country:** Czechia **Facility:** Research Site **Zip:** 669 02 **Location 97:** **City:** Angers **Country:** France **Facility:** Research Site **Zip:** 49933 **Location 98:** **City:** Lens cedex **Country:** France **Facility:** Research Site **Zip:** 62307 **Location 99:** **City:** Perpignan **Country:** France **Facility:** Research Site **Zip:** 66000 **Location 100:** **City:** Vandoeuvre les Nancy **Country:** France **Facility:** Research Site **Zip:** 54511 **Location 101:** **City:** Kaunas **Country:** Lithuania **Facility:** Research Site **Zip:** 45434 **Location 102:** **City:** Vilnius **Country:** Lithuania **Facility:** Research Site **Zip:** 08660 **Location 103:** **City:** San Juan **Country:** Puerto Rico **Facility:** Research Site **Zip:** 00935 **Location 104:** **City:** Banska Bystrica **Country:** Slovakia **Facility:** Research Site **Zip:** 974 01 **Location 105:** **City:** Bratislava **Country:** Slovakia **Facility:** Research Site **Zip:** 812 50 **Location 106:** **City:** Nove Zamky **Country:** Slovakia **Facility:** Research Site **Zip:** 940 34 **Location 107:** **City:** Presov **Country:** Slovakia **Facility:** Research Site **Zip:** 080 01 **Location 108:** **City:** Spisska Nova Ves **Country:** Slovakia **Facility:** Research Site **Zip:** 052 01 **Location 109:** **City:** Santander **Country:** Spain **Facility:** Research Site **State:** Cantabria **Zip:** 39008 **Location 110:** **City:** Barcelona **Country:** Spain **Facility:** Research Site **State:** Cataluña **Zip:** 08025 **Location 111:** **City:** Girona **Country:** Spain **Facility:** Research Site **State:** Cataluña **Zip:** 17007 **Location 112:** **City:** L Hospitalet De Llobregat **Country:** Spain **Facility:** Research Site **State:** Cataluña **Zip:** 08907 **Location 113:** **City:** Valencia **Country:** Spain **Facility:** Research Site **State:** Comunidad Valenciana **Zip:** 46009 **Location 114:** **City:** Madrid **Country:** Spain **Facility:** Research Site **Zip:** 28040 #### Overall Officials **Official 1:** **Affiliation:** Amgen **Name:** MD **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** Wirth LJ. PARTNER: a study of panitumumab plus chemotherapy for first-line treatment of advanced head and neck cancer. Commun Oncol. 2008;5(Supp 14):1-4. #### See Also Links **Label:** AmgenTrials clinical trials website **URL:** http://www.amgentrials.com ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes - ID: D000002294 - Term: Carcinoma, Squamous Cell - ID: D000002277 - Term: Carcinoma - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000009371 - Term: Neoplasms by Site ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M9348 - Name: Head and Neck Neoplasms - Relevance: HIGH - As Found: Head and Neck Cancer - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - ID: M5550 - Name: Carcinoma, Squamous Cell - Relevance: LOW - As Found: Unknown - ID: M1689 - Name: Squamous Cell Carcinoma of Head and Neck - Relevance: HIGH - As Found: Squamous Cell Carcinoma of Head and Neck - ID: M14850 - Name: Recurrence - Relevance: HIGH - As Found: Recurrent - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: T4202 - Name: Oculocerebral Syndrome With Hypopigmentation - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006258 - Term: Head and Neck Neoplasms - ID: D000077195 - Term: Squamous Cell Carcinoma of Head and Neck - ID: D000012008 - Term: Recurrence ### Intervention Browse Module - Ancestors - ID: D000000970 - Term: Antineoplastic Agents - ID: D000050257 - Term: Tubulin Modulators - ID: D000050256 - Term: Antimitotic Agents - ID: D000050258 - Term: Mitosis Modulators - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000074322 - Term: Antineoplastic Agents, Immunological ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M6182 - Name: Cisplatin - Relevance: HIGH - As Found: High - ID: M1668 - Name: Docetaxel - Relevance: HIGH - As Found: Physical - ID: M1822 - Name: Panitumumab - Relevance: HIGH - As Found: 2/day - ID: M26197 - Name: Tubulin Modulators - Relevance: LOW - As Found: Unknown - ID: M26196 - Name: Antimitotic Agents - Relevance: LOW - As Found: Unknown - ID: M1346 - Name: Antineoplastic Agents, Immunological - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000002945 - Term: Cisplatin - ID: D000077143 - Term: Docetaxel - ID: D000077544 - Term: Panitumumab ### Misc Info Module #### Removed Countries - Country: Czech Republic - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events. #### Event Groups **Group ID:** EG000 **Title:** Panitumumab Plus Chemotherapy **Description:** Panitumumab + Docetaxel + Cisplatin, experiment **ID:** EG000 **Other Num Affected:** 54 **Other Num at Risk:** 56 **Serious Number Affected:** 34 **Serious Number At Risk:** 56 **Title:** Panitumumab Plus Chemotherapy **Group ID:** EG001 **Title:** Chemotherapy Alone **Description:** Docetaxel + Cisplatin, control **ID:** EG001 **Other Num Affected:** 53 **Other Num at Risk:** 55 **Serious Number Affected:** 27 **Serious Number At Risk:** 55 **Title:** Chemotherapy Alone **Frequency Threshold:** 5 #### Other Events **Term:** Anaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Febrile neutropenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Leukopenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Neutropenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Thrombocytopenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Tinnitus **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Ear and labyrinth disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Vertigo **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Ear and labyrinth disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Conjunctivitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Lacrimation increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Abdominal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Abdominal pain upper **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Constipation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Diarrhoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Dry mouth **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Dyspepsia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Dysphagia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Mouth ulceration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Rectal haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Stomatitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Vomiting **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Asthenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Chills **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Face oedema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Fatigue **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Mucosal inflammation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Oedema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Oedema peripheral **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Pyrexia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Bronchitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 **Term:** Candidiasis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 **Term:** Pneumonia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 **Term:** Urinary tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 **Term:** Laceration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 15.0 **Term:** Haemoglobin decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 15.0 **Term:** Weight decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 15.0 **Term:** Cachexia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Decreased appetite **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Dehydration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Hypoalbuminaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Hypocalcaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Hypokalaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Hypomagnesaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Hyponatraemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Hypophosphataemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Arthralgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Back pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Bone pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Muscular weakness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Neck pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Pain in extremity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Pain in jaw **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Dysgeusia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Neuropathy peripheral **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Paraesthesia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Peripheral sensory neuropathy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Somnolence **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Syncope **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Tremor **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Anxiety **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Depression **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Insomnia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Nocturia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Cough **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Dyspnoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Epistaxis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Haemoptysis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Hiccups **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Oropharyngeal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Pleural effusion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Rhinorrhoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Acne **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Alopecia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Decubitus ulcer **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Dermatitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Dermatitis acneiform **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Dry skin **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Erythema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Nail disorder **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Pruritus **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Rash **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Skin exfoliation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Hypertension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 15.0 **Term:** Hypotension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 15.0 #### Serious Events **Term:** Anaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 55 **Term:** Febrile neutropenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 4 **Num At Risk:** 55 **Term:** Neutropenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 55 **Term:** Pancytopenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Thrombocytopenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Acute myocardial infarction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Arteriosclerosis coronary artery **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Atrial fibrillation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Cardiac failure congestive **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Abdominal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Aphagia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Aphthous stomatitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Constipation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Diarrhoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 6 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 55 **Term:** Duodenal ulcer perforation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Dysphagia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 55 **Term:** Gastrointestinal haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Haematemesis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Ileus **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Intestinal obstruction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 4 **Num At Risk:** 55 **Term:** Oral cavity fistula **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Vomiting **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 4 **Num At Risk:** 55 **Term:** Asthenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Chills **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Device dislocation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** General physical health deterioration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Localised oedema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Malaise **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Mucosal inflammation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Oedema peripheral **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Pyrexia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 4 **Num At Risk:** 55 **Term:** Cholecystitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Hepatic failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Abdominal abscess **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Beta haemolytic streptococcal infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Cellulitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Clostridium difficile colitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Fungaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Neutropenic sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Oral infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Osteomyelitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Pneumonia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 4 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 6 **Num At Risk:** 55 **Term:** Pneumonia staphylococcal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Pseudomonal sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Pseudomonas infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Septic shock **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Staphylococcal infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 55 **Term:** Staphylococcal sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Stenotrophomonas infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Urinary tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Urosepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Wound infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Arterial injury **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Feeding tube complication **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Humerus fracture **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Post procedural discharge **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Post procedural haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Sternal fracture **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Blood creatine increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Hepatic enzyme increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** International normalised ratio increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Transaminases increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Decreased appetite **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Dehydration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 8 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 55 **Term:** Electrolyte imbalance **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Hypernatraemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Hypocalcaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Hypokalaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Hypomagnesaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 55 **Term:** Hyponatraemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Musculoskeletal chest pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Rhabdomyolysis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Head and neck cancer **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Malignant ascites **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Neoplasm malignant **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Neoplasm progression **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Tumour haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Tumour ulceration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Presyncope **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Syncope **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Mental status changes **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Acute prerenal failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Renal failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 55 **Term:** Renal failure acute **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Aspiration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Dyspnoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Haemoptysis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Hypoxia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Interstitial lung disease **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Pneumonia aspiration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Pulmonary embolism **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Respiratory failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 55 **Term:** Acne **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Arterial haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Deep vein thrombosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Term:** Hypotension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 15.0 ##### Stats **Group ID:** EG000 **Num Affected:** 5 **Num At Risk:** 56 **Group ID:** EG001 **Num At Risk:** 55 **Time Frame:** The time frame for adverse event reporting is from the first dose date to 30 days since the last dose date of the first-line treatment. The median time frame is 4.5 months for Panitumumab Plus Chemotherapy arm and 4.4 months for Chemotherapy Alone arm. ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 56 **Group ID:** BG001 **Value:** 57 **Group ID:** BG002 **Value:** 113 **Units:** Participants ### Group **ID:** BG000 **Title:** Panitumumab Plus Chemotherapy **Description:** Panitumumab + Docetaxel + Cisplatin, experiment ### Group **ID:** BG001 **Title:** Chemotherapy Alone **Description:** Docetaxel + Cisplatin, control ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 8.6 **Value:** 58.2 #### Measurement **Group ID:** BG001 **Spread:** 7.4 **Value:** 58.9 #### Measurement **Group ID:** BG002 **Spread:** 8.0 **Value:** 58.6 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 9 #### Measurement **Group ID:** BG001 **Value:** 5 #### Measurement **Group ID:** BG002 **Value:** 14 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 47 #### Measurement **Group ID:** BG001 **Value:** 52 #### Measurement **Group ID:** BG002 **Value:** 99 **Category Title:** Male **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** Years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement **Other Details:** The Clinical Trial Agreement generally does not restrict an investigator's discussion of trial results after completion. The Agreement permits Amgen a limited period of time to review material discussing trial results (typically up to 45 days and possible extension). Amgen may remove confidential information, but authors have final control and approval of publication content. For multicenter studies, the investigator agrees not to publish any results before the first multi-center publication. **Restriction Type:** OTHER **Restrictive Agreement:** True ### Point of Contact **Organization:** Amgen Inc. **Phone:** 866-572-6436 **Title:** Study Director ## Results Section - Outcome Measures Module ### Outcome Measure 1 #### Analysis **CI Lower Limit:** 0.395 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 1.002 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** Hazard ratio is presented as panitumumab plus chemotherapy:chemotherapy alone. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.051 **P-Value Comment:** **Parameter Type:** Hazard Ratio (HR) **Parameter Value:** 0.629 **Statistical Comment:** Stratified by IVRS randomization factors **Statistical Method:** Regression, Cox **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.048 **P-Value Comment:** **Parameter Type:** **Parameter Value:** **Statistical Comment:** Stratified by IVRS randomization factors **Statistical Method:** Log Rank **Tested Non-Inferiority:** ### Outcome Measure 2 #### Analysis **CI Lower Limit:** -11.67 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 24.12 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** With Mantel-Haenszel weights within strata defined by randomization factors recorded in the IVRS **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** **P-Value Comment:** **Parameter Type:** Mean Difference (Final Values) **Parameter Value:** 6.23 **Statistical Comment:** **Statistical Method:** **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** 0.57 **CI Number of Sides:** **CI Percentage Value:** 95 **CI Upper Limit:** 3.33 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** Calculated from a logistic regression model with treatment indicator and randomization factors (recorded on the CRF) as covariates **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** **P-Value Comment:** **Parameter Type:** Odds Ratio (OR) **Parameter Value:** 1.37 **Statistical Comment:** **Statistical Method:** **Tested Non-Inferiority:** ### Outcome Measure 3 #### Analysis **CI Lower Limit:** -8.29 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 23.85 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** With Mantel-Haenszel weights within strata defined by randomization factors recorded in the IVRS **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** **P-Value Comment:** **Parameter Type:** Mean Difference (Final Values) **Parameter Value:** 7.78 **Statistical Comment:** **Statistical Method:** **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** 0.62 **CI Number of Sides:** **CI Percentage Value:** 95 **CI Upper Limit:** 5.26 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** Calculated from a logistic regression model with treatment indicator and randomization factors (recorded on the CRF) as covariates **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** **P-Value Comment:** **Parameter Type:** Odds Ratio (OR) **Parameter Value:** 1.76 **Statistical Comment:** **Statistical Method:** **Tested Non-Inferiority:** ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 #### Analysis **CI Lower Limit:** 0.709 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 1.717 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** Hazard ratio is presented as panitumumab plus chemotherapy:chemotherapy alone **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.663 **P-Value Comment:** **Parameter Type:** Hazard Ratio (HR) **Parameter Value:** 1.103 **Statistical Comment:** Stratified by IVRS randomization factors **Statistical Method:** Regression, Cox **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.666 **P-Value Comment:** **Parameter Type:** **Parameter Value:** **Statistical Comment:** Stratified by IVRS randomization factors **Statistical Method:** Log Rank **Tested Non-Inferiority:** ### Outcome Measure 7 ### Outcome Measure 8 ### Outcome Measure 9 ### Outcome Measure 10 ### Outcome Measure 11 ### Outcome Measure 12 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 4.7 - **Spread:** - **Upper Limit:** 8.3 - **Value:** 6.9 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 4.1 - **Spread:** - **Upper Limit:** 6.8 - **Value:** 5.5 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 30.73 - **Spread:** - **Upper Limit:** 57.73 - **Value:** 44.23 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 23.99 - **Spread:** - **Upper Limit:** 50.52 - **Value:** 37.25 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 70.06 - **Spread:** - **Upper Limit:** 91.48 - **Value:** 80.77 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 60.30 - **Spread:** - **Upper Limit:** 84.80 - **Value:** 72.55 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 5.7 - **Spread:** - **Upper Limit:** 11.1 - **Value:** 8.0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 4.4 - **Spread:** - **Upper Limit:** 7.2 - **Value:** 5.1 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.9 - **Upper Limit:** - **Value:** 8.8 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 6.1 - **Upper Limit:** - **Value:** 10.6 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 9.4 - **Spread:** - **Upper Limit:** 18.5 - **Value:** 12.9 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 11.8 - **Spread:** - **Upper Limit:** 22.9 - **Value:** 13.8 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 1.5 - **Spread:** - **Upper Limit:** 7.6 - **Value:** 4.2 **Title:** #### Outcome Measure 8 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 1.17 - **Spread:** - **Upper Limit:** 25.50 - **Value:** 13.33 **Title:** #### Outcome Measure 9 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 35.48 - **Spread:** - **Upper Limit:** 71.19 - **Value:** 53.33 **Title:** #### Outcome Measure 10 **Class:** ##### Category **Measurements:** - **Comment:** Median is not reached due to censoring. - **Group ID:** OG001 - **Lower Limit:** 5.1 - **Spread:** - **Upper Limit:** NA - **Value:** NA **Title:** #### Outcome Measure 11 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 5.6 - **Upper Limit:** - **Value:** 10.6 **Title:** #### Outcome Measure 12 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 6.5 - **Spread:** - **Upper Limit:** 13.2 - **Value:** 8.5 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** The time from the date of randomization to the date of first disease progression determined by the investigators per modified RECIST v1.0, or death within 60 days after the last evaluable tumor assessment or randomization date (whichever is later) during the first-line treatment phase. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEDIAN **Population Description:** All randomized participants \< 70 years of age who provide informed consent and receive at least one dose of first-line treatment (chemotherapy and/or panitumumab) **Reporting Status:** POSTED **Time Frame:** Every 6 weeks until disease progression or death, up to 67 months **Title:** Progression Free Survival (PFS) During the First-line Treatment Phase **Type:** PRIMARY **Unit of Measure:** Months ##### Group **Description:** Panitumumab + Docetaxel + Cisplatin, experiment **ID:** OG000 **Title:** Panitumumab Plus Chemotherapy ##### Group **Description:** Docetaxel + Cisplatin, control **ID:** OG001 **Title:** Chemotherapy Alone #### Outcome Measure 2 **Description:** Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter (SLD) of target lesions from baseline; Overall Response (OR) = CR + PR. An overall response of CR or PR must be confirmed at least 4 weeks after the criteria for response are first met. ORR is the percentage of subjects with an overall response among the analysis population. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** all randomized subjects \< 70 years of age who provide informed consent and receive at least one dose of first-line treatment (chemotherapy and/or panitumumab), with at least one uni-dimensionally measurable lesion at baseline using a modified RECIST v1.0 per investigators' review **Reporting Status:** POSTED **Time Frame:** Every 6 weeks until disease progression or death, up to 67 months **Title:** Overall Response Rate (ORR) During the First-line Treatment Phase **Type:** SECONDARY **Unit of Measure:** Percentage of Participants ##### Group **Description:** Panitumumab + Docetaxel + Cisplatin, experiment **ID:** OG000 **Title:** Panitumumab Plus Chemotherapy ##### Group **Description:** Docetaxel + Cisplatin, control **ID:** OG001 **Title:** Chemotherapy Alone #### Outcome Measure 3 **Description:** Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter (SLD) of target lesions from baseline; Disease Progression (PD), \>=20% increase in the SLD of target lesions from nadir; Stable Disease (SD), Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. An overall response of CR or PR must be confirmed at least 4 weeks after the criteria for response are first met. A best overall response of SD requires a visit response of SD or better no earlier than 35 days after randomization. RCD is the percentage of subjects with a best overall response of CR, PR or SD among the analysis population. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** all randomized subjects \< 70 years of age who provide informed consent and receive at least one dose of first-line treatment (chemotherapy and/or panitumumab), with at least one uni-dimensionally measurable lesion at baseline using a modified RECIST v1.0 per investigators' review **Reporting Status:** POSTED **Time Frame:** Every 6 weeks until disease progression or death, up to 67 months **Title:** Rate of Disease Control (RDC) During the First-line Treatment Phase **Type:** SECONDARY **Unit of Measure:** Percentage of Participants ##### Group **Description:** Panitumumab + Docetaxel + Cisplatin, experiment **ID:** OG000 **Title:** Panitumumab Plus Chemotherapy ##### Group **Description:** Docetaxel + Cisplatin, control **ID:** OG001 **Title:** Chemotherapy Alone #### Outcome Measure 4 **Description:** Calculated only for the subset of subjects who have an overall response of CR or PR while on first-line treatment phase (subsequently confirmed at least 4 weeks thereafter), and is defined as time from the first CR or PR to the first observed disease progression by a modified RECIST v1.0. Subjects not meeting the criteria for progression by the analysis data cutoff date will be censored at their last evaluable disease assessment date. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEDIAN **Population Description:** All randomized participants \< 70 years of age who provide informed consent and receive at least one dose of first-line treatment (chemotherapy and/or panitumumab), with at least one uni-dimensionally measurable lesion at baseline using a modified RECIST v1.0 per investigators' review and objective response **Reporting Status:** POSTED **Time Frame:** Every 6 weeks until disease progression or death, up to 67 months **Title:** Duration of Response (DOR) During the First-line Treatment Phase **Type:** SECONDARY **Unit of Measure:** Months ##### Group **Description:** Panitumumab + Docetaxel + Cisplatin, experiment **ID:** OG000 **Title:** Panitumumab Plus Chemotherapy ##### Group **Description:** Docetaxel + Cisplatin, control **ID:** OG001 **Title:** Chemotherapy Alone #### Outcome Measure 5 **Description:** Time from the date of randomization to the first CR or PR during first line treatment phase (subsequently confirmed at least 4 weeks thereafter) **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** All randomized participants \< 70 years of age who provide informed consent and receive at least one dose of first-line treatment (chemotherapy and/or panitumumab), with at least one uni-dimensionally measurable lesion at baseline using a modified RECIST v1.0 per investigators' review and objective response **Reporting Status:** POSTED **Time Frame:** Every 6 weeks until disease progression or death, up to 67 months **Title:** Time to Response (TTR) During the First-line Treatment Phase **Type:** SECONDARY **Unit of Measure:** Weeks ##### Group **Description:** Panitumumab + Docetaxel + Cisplatin, experiment **ID:** OG000 **Title:** Panitumumab Plus Chemotherapy ##### Group **Description:** Docetaxel + Cisplatin, control **ID:** OG001 **Title:** Chemotherapy Alone #### Outcome Measure 6 **Description:** Time from the date of randomization to the date of death during the entire study **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEDIAN **Population Description:** All randomized participants \< 70 years of age who provide informed consent and receive at least one dose of first-line treatment (chemotherapy and/or panitumumab) **Reporting Status:** POSTED **Time Frame:** Until death, up to 67 months **Title:** Overall Survival (OS) for the First-line Treatment **Type:** SECONDARY **Unit of Measure:** Months ##### Group **Description:** Panitumumab + Docetaxel + Cisplatin, experiment **ID:** OG000 **Title:** Panitumumab Plus Chemotherapy ##### Group **Description:** Docetaxel + Cisplatin, control **ID:** OG001 **Title:** Chemotherapy Alone #### Outcome Measure 7 **Description:** The time from the first dose of panitumumab monotherapy to the date of first disease progression determined by the investigators per modified RECIST v1.0, or death within 60 days after the last evaluable tumor assessment or the second-line first dose date (whichever is later) during the second-line treatment phase. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEDIAN **Population Description:** Subjects who are randomized to docetaxel and cisplatin chemotherapy alone treatment for their first-line treatment and treated subsequently with at least 1 dose of second-line panitumumab monotherapy **Reporting Status:** POSTED **Time Frame:** Every 6 weeks until disease progression or death, up to 57 months **Title:** Progression Free Survival (PFS) During the Second-line Treatment Phase **Type:** SECONDARY **Unit of Measure:** Months ##### Group **Description:** Panitumumab + Docetaxel + Cisplatin, experiment **ID:** OG000 **Title:** Panitumumab Plus Chemotherapy ##### Group **Description:** Docetaxel + Cisplatin, control **ID:** OG001 **Title:** Chemotherapy Alone #### Outcome Measure 8 **Description:** Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter (SLD) of target lesions from baseline; Overall Response (OR) = CR + PR. An overall response of CR or PR must be confirmed at least 4 weeks after the criteria for response are first met. ORR is the percentage of subjects with an overall response among the analysis population. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** Subjects who are randomized to docetaxel and cisplatin chemotherapy alone treatment for their first-line treatment and treated subsequently with at least 1 dose of second-line panitumumab monotherapy **Reporting Status:** POSTED **Time Frame:** Every 6 weeks until disease progression or death, up to 57 months **Title:** Overall Response Rate (ORR) During the Second-line Treatment Phase **Type:** SECONDARY **Unit of Measure:** Percentage of Participants ##### Group **Description:** Panitumumab + Docetaxel + Cisplatin, experiment **ID:** OG000 **Title:** Panitumumab Plus Chemotherapy ##### Group **Description:** Docetaxel + Cisplatin, control **ID:** OG001 **Title:** Chemotherapy Alone #### Outcome Measure 9 **Description:** Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter (SLD) of target lesions from baseline; Disease Progression (PD), \>=20% increase in the SLD of target lesions from nadir; Stable Disease (SD), Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. An overall response of CR or PR must be confirmed at least 4 weeks after the criteria for response are first met. A best overall response of SD requires a visit response of SD or better no earlier than 35 days after the first dose date in second-line treatment. RCD is the percentage of subjects with a best overall response of CR, PR or SD among the analysis population. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** Subjects who are randomized to docetaxel and cisplatin chemotherapy alone treatment for their first-line treatment and treated subsequently with at least 1 dose of second-line panitumumab monotherapy **Reporting Status:** POSTED **Time Frame:** Every 6 weeks until disease progression or death, up to 57 months **Title:** Rate of Disease Control (RDC) During the Second-line Treatment Phase **Type:** SECONDARY **Unit of Measure:** Percentage of Participants ##### Group **Description:** Panitumumab + Docetaxel + Cisplatin, experiment **ID:** OG000 **Title:** Panitumumab Plus Chemotherapy ##### Group **Description:** Docetaxel + Cisplatin, control **ID:** OG001 **Title:** Chemotherapy Alone #### Outcome Measure 10 **Description:** Time from the first CR or PR to the first observed disease progression by a modified RECIST v1.0. Subjects not meeting the criteria for progression by the analysis data cutoff date will be censored at their last evaluable disease assessment date. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEDIAN **Population Description:** Subjects who are randomized to docetaxel and cisplatin chemotherapy alone treatment for their first-line treatment and treated subsequently with at least 1 dose of second-line panitumumab monotherapy, with at least one uni-dimensionally measurable lesion at baseline using a modified RECIST v1.0 per investigators' review and objective response **Reporting Status:** POSTED **Time Frame:** Every 6 weeks until disease progression or death, up to 57 months **Title:** Duration of Response (DOR) During the Second-line Treatment Phase **Type:** SECONDARY **Unit of Measure:** Months ##### Group **Description:** Panitumumab + Docetaxel + Cisplatin, experiment **ID:** OG000 **Title:** Panitumumab Plus Chemotherapy ##### Group **Description:** Docetaxel + Cisplatin, control **ID:** OG001 **Title:** Chemotherapy Alone #### Outcome Measure 11 **Description:** Time from the first dose of panitumumab monotherapy to the first CR or PR during second-line treatment phase (subsequently confirmed at least 4 weeks thereafter) **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Subjects who are randomized to docetaxel and cisplatin chemotherapy alone treatment for their first-line treatment and treated subsequently with at least 1 dose of second-line panitumumab monotherapy, with at least one uni-dimensionally measurable lesion at baseline using a modified RECIST v1.0 per investigators' review and objective response **Reporting Status:** POSTED **Time Frame:** Every 6 weeks until disease progression or death, up to 57 months **Title:** Time to Response (TTR) During the Second-line Treatment Phase **Type:** SECONDARY **Unit of Measure:** Weeks ##### Group **Description:** Panitumumab + Docetaxel + Cisplatin, experiment **ID:** OG000 **Title:** Panitumumab Plus Chemotherapy ##### Group **Description:** Docetaxel + Cisplatin, control **ID:** OG001 **Title:** Chemotherapy Alone #### Outcome Measure 12 **Description:** Time from the first dose of panitumumab monotherapy to the date of death during the entire study **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEDIAN **Population Description:** Subjects who are randomized to docetaxel and cisplatin chemotherapy alone treatment for their first-line treatment and treated subsequently with at least 1 dose of second-line panitumumab monotherapy **Reporting Status:** POSTED **Time Frame:** Until death, up to 57 months **Title:** Overall Survival (OS) for the Second-line Treatment **Type:** SECONDARY **Unit of Measure:** Months ##### Group **Description:** Panitumumab + Docetaxel + Cisplatin, experiment **ID:** OG000 **Title:** Panitumumab Plus Chemotherapy ##### Group **Description:** Docetaxel + Cisplatin, control **ID:** OG001 **Title:** Chemotherapy Alone ### Participant Flow Module #### Group **Description:** Panitumumab + Docetaxel + Cisplatin, experiment **ID:** FG000 **Title:** Panitumumab Plus Chemotherapy #### Group **Description:** Docetaxel + Cisplatin, control **ID:** FG001 **Title:** Chemotherapy Alone #### Period **Title:** First-line Treatment Phase ##### Withdraw **Type:** Lack of Efficacy ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 2 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 4 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ##### Withdraw **Type:** Protocol Violation ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ##### Withdraw **Type:** Ended follow-up prior to analysis ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ##### Withdraw **Type:** Ongoing ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 56 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 57 ##### Milestone **Type:** Received Study Medication ###### Achievement **Group ID:** FG000 **Number of Subjects:** 56 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 55 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 51 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 49 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 5 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 8 #### Period **Title:** Second-line Treatment Phase ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ##### Withdraw **Type:** Ended follow-up prior to analysis ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ##### Withdraw **Type:** Ongoing ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 30 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 27 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 3 **Pre-Assignment Details:** The planned number of subjects to enroll was 110. The actual number of subjects enrolled was 113. **Recruitment Details:** Subjects were enrolled from 29 January 2007 to 1 September 2010. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT03943979 **Brief Title:** Pilot Study of an Intervention Involving Cognitive Training and tDCS in Morbidly Obese Subjects **Official Title:** Pilot Study of an Intervention Involving Cognitive Training and Transcranial Direct-current Stimulation (tDCS) in Morbidly Obese Subjects #### Organization Study ID Info **ID:** IMIMFTLC/EC-TDCS OM #### Organization **Class:** OTHER **Full Name:** Parc de Salut Mar ### Status Module #### Completion Date **Date:** 2017-06-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2019-05-09 **Type:** ACTUAL **Last Update Submit Date:** 2019-05-08 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2017-05-26 **Type:** ACTUAL #### Start Date **Date:** 2016-02-25 **Type:** ACTUAL **Status Verified Date:** 2019-05 #### Study First Post Date **Date:** 2019-05-09 **Type:** ACTUAL **Study First Submit Date:** 2018-06-14 **Study First Submit QC Date:** 2019-05-08 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Neuroelectrics Corporation #### Lead Sponsor **Class:** OTHER **Name:** Parc de Salut Mar #### Responsible Party **Investigator Affiliation:** Parc de Salut Mar **Investigator Full Name:** Albert Goday Arno **Investigator Title:** Medical doctor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The study aims to: 1) investigate the effects of cognitive training (CT) and combined CT and transcranial direct current stimulation (tDCS) on food intake and 2) to further understand its neuropsychological and neurophysiological basis (i.e. EEG) as well as its impact in endocannabinoids (EC) in a sample of morbidly obese patients seeking for a gastric bypass surgery **Detailed Description:** The study will explore the impact of a 4-day intervention with either CT (Active control condition) or CT+tDCS (Active condition) stimulation on food intake in a sample of morbidly obese patients, as measured by dietary assessments the week before, during and the week after the intervention. Additionally, to further understand the neuropsychological and neurophysiological basis of its impact, measures of executive function and attention performance and EEG recordings, respectively, will be collected. Furthermore, we will explore the effect of the intervention on endocannabinoids previously related to eating behaviour. The Active Control condition will receive sham stimulation together with CT, through a computerized cognitive training platform (Guttmann NeuropersonalTrainer), including different tasks with designed to train executive functions and attention. Each session will last approximately 30-40 min. The Active condition will receive tDCS stimulation (20 min, multichannel with an excitatory target over the r-dlPFC) together with CT (same as for the Active Control condition). Participants will undergo a basal (the week before intervention) and a post treatment assessments (the day after finishing the intervention) that will include medical history, blood testing, anthropometric measures, a cognitive assessment battery and a 4-day dietary assessment. ### Conditions Module **Conditions:** - Obesity, Morbid - Obesity (BMI > 35) and Diabetes Mellitus - Obesity (BMI > 35) and High Blood Pressure - Obesity (BMI > 35) and Dyslipemia ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Active Control group: Subjects will receive CT and sham tDCS during four consecutive days. Active group: Subjects will receive CT and active tDCS during four consecutive days. ##### Masking Info **Masking:** QUADRUPLE **Masking Description:** The difference between the two groups is the tDCS therapy. To mask this difference, the duration time of the session will be the same under both conditions but a standard Sham tDCS protocol will be used for the Active Control group. That is, a 3-sec ramp-up and ramp-down current stimulation, so the subject perceives the current just like the real tDCS, but it is not strong nor durable enough to have any effect on the neuronal activity. **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 18 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** This group will receive CT and sham tDCS each day, for four days. **Intervention Names:** - Other: Cognitive Training **Label:** Active Control group **Type:** SHAM_COMPARATOR #### Arm Group 2 **Description:** This group will receive both CT and tDCS, each day, for four days. **Intervention Names:** - Other: Cognitive Training - Device: transcranial Direct-Current Stimulation (tDCS) **Label:** Active group **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Active Control group - Active group **Description:** Cognitive Training (CT): 4 consecutive days, 30-45 min session involving 5 different tasks designed to train executive functions and attention, available at the Guttmann Neuropersonaltrainer platform (computerized cognitive training tool certified by the Spanish Agency for Medicines and Health Products as a Class I Health Product). The difficulty level of all the tasks was automatically adjusted on a trial-by-trial basis for both Active and Active Control conditions. **Name:** Cognitive Training **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Active group **Description:** tDCS: 4 consecutive days, 20 min session, delivered through multichannel tDCS (Starstim, Neuroelectrics), with an excitatory target over the r-dlPFC, and an inhibitory target on the contralateral lobe (l-dlPFC). The positioning of the multichannel tCS (electrode location and currents) was solved using the Stimweaver (Ruffini 2013). The resulting tCS montage employed 8 gelled Ag/AgCl electrodes of π cm2 size (Pistim, Neuroelectrics) placed at AF3(-1093uA), AF4 (1178uA), F3 (-1161uA), F4 (1104uA), F7 (-414uA), F8 (530uA), FC5 (1189uA), FC6 (-1332uA). **Name:** transcranial Direct-Current Stimulation (tDCS) **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Changes in food consumption (medium kcal intake ) from baseline to the end of the treatment measured by 4-day food registers during the week before starting the treatment and during the 4 days of treatment. The dietary records were checked by a nutritionist and analyzed through the software PCN Pro 1.0. **Measure:** Change in food intake **Time Frame:** 4 consecutive days during the week before starting the treatment; 4 consecutive days during the week of treatment; **Description:** Changes in food consumption (medium kcal intake ) from baseline to the follow-up assessment measured by 4-day food registers during the week before starting the treatment and the week after finishing the treatment. The dietary records were checked by a nutritionist and analyzed through the software PCN Pro 1.0. **Measure:** Stability of changes in food intake **Time Frame:** 4 consecutive days during the week before starting the treatment and 4 consecutive days during the week after finishing the treatment #### Secondary Outcomes **Description:** EEG was recorded before and after tCS/sham using a Starstim device (Neuroelectrics), same electrode positions as for the stimulation with a sampling frequency of 500 S/s . Electrode impedance: below 10kΩ; electrical reference placed at the right earlobe. EEG data was analyzed offline by means of customized Matlab code (MathWorks Inc. Natick, MA, USA). Data was split into 1s non-overlapping epochs (epochs with amplitudes \>50 μV were rejected). EEG-metrics extracted: EEG-power and coherence. To compute EEG power, the power spectral density (PSD) was estimated for each epoch. Band Power was computed for the bands θ=\[4,8 Hz\], α=\[8,13 Hz\] β=\[13,25 Hz\], γ=\[30, 45 Hz\] and broadband=\[4-45Hz\] by integrating the PSD within the band frequency limits. Functional connectivity was estimated by means of coherence by the Welch method averaged over all electrodes. Frontal asymmetry (FA) computed as follows: log(avg(AF4 F4 F8 FC6))-log(avg(AF3 F3 F7 FC5); FA\<0 reflects dominance of left-hemisphere **Measure:** Change in electroencephalogram (EEG) power and coherence **Time Frame:** Every training session (4 consecutive days), 3 minutes before starting the training session and 3 minutes after finishing the training. **Description:** Change in BMI. BMI calculation: body weight divided by the square of the body height (expressed in units of kg/m2). Weight and height measures were taken by a nurse. **Measure:** Change in Body Mass Index (BMI) **Time Frame:** One day during the week before starting the training (pre-treatment) and the 1 day after finishing the treatment (post-treatment) **Description:** Change in endocannabinoids and related compounds (acylglycerols and fatty acid N-acylethanolamides) as measured in plasmatic concentrations and quantified by LC/MS-MS by a previously validated method (Pastor et al. 2014). **Measure:** Change in endocannabinoids (and related compounds) plasmatic concentrations. **Time Frame:** One day during the week before starting the training (pre-treatment) and the 1 day after finishing the treatment (post-treatment) **Description:** Measurement of change in hormones regulating the appetite and energic homeostasis of secretion in fatty tissues. **Measure:** Change in hormonal measurements : Leptin and adiponectin plasmatic/serum concentrations **Time Frame:** One day during the week before starting the training (pre-treatment) and the 1 day after finishing the treatment (post-treatment) **Description:** Executive functions: flexibility **Measure:** Change in neuropsychological test: Intra-extra dimension (IED-CANTAB Cambridge Cognition) **Time Frame:** One day during the week before starting the training (pre-treatment) and the 1 day after finishing the treatment (post-treatment) **Description:** Executive functions: inhibition **Measure:** Change in neuropsychological test: Stroop Colours and Words test (SCWT, Golden C.J., 1978; Stroop, 1935) **Time Frame:** One day during the week before starting the training (pre-treatment) and the 1 day after finishing the treatment (post-treatment) **Description:** Executive functions: decision-making, risky behaviour. **Measure:** Change in neuropsychological test: Iowa Gambling Task (IGT, Bechara et al., 1994, 2002) **Time Frame:** One day during the week before starting the training (pre-treatment) and the 1 day after finishing the treatment (post-treatment) **Description:** Executive functions: working memory **Measure:** Change in neuropsychological test: Spatial Span (SSP; CANTAB Cambridge Cognition) **Time Frame:** One day during the week before starting the training (pre-treatment) and the 1 day after finishing the treatment (post-treatment) **Description:** Executive functions: planning **Measure:** Change in neuropsychological test: Stockings of Cambridge (SOC; CANTAB Cambridge Cognition) **Time Frame:** One day during the week before starting the training (pre-treatment) and the 1 day after finishing the treatment (post-treatment) **Description:** Executive functions: Attention **Measure:** Change in neuropsychological test: Conners Continuous Performance Test (CPT, Conners & MHS Staff, 2000) **Time Frame:** One day during the week before starting the training (pre-treatment) and the 1 day after finishing the treatment (post-treatment) **Description:** Executive functions: Attention **Measure:** Change in neuropsychological test: Simple Reaction Time (SRT; CANTAB Cambridge Cognition) **Time Frame:** One day during the week before starting the training (pre-treatment) and the 1 day after finishing the treatment (post-treatment) **Description:** Executive functions: Attention and Processing speed **Measure:** Change in neuropsychological test:Symbol digit modalities test (SDMT; Smith A. 1982). **Time Frame:** One day during the week before starting the training (pre-treatment) and the 1 day after finishing the treatment (post-treatment) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Subjects of both gender, aged between 18 and 60 years-old * Having a BMI \> 40 kg/m2 or having a BMI\>35 and suffering from diabetes mellitus, HBP or LDP. * Obesity conventional treatment failure * Wish of bariatric surgery * Accepting the study and signing the Informed Consent Exclusion Criteria: * Do not meet inclusion criteria * Being left-handed * Using a pacemaker or deep cerebral stimulation device * Having a psychiatric disease or serious disease * Neurologic condition or learning issue or mental backwardness that could affect cognitive function * Use of psycho-stimulating medicines and/or drugs, abuse or dependance to a psychoactive substance (or during the last 6 months) * Dependance to alcohol or/and drugs (excepted from nicotina) * In treatment with benzodiazepines, antipsychotics, tricyclic antidepressants or topiramate, started in the last month * History of psychiatric disorders treated with lithio carbonate. * Cutaneous lesion on the area of using of electrodes * Contact allergy to material used in the used devices. **Healthy Volunteers:** True **Maximum Age:** 60 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ### References Module #### References **Citation:** Alonso-Alonso M. Translating tDCS into the field of obesity: mechanism-driven approaches. Front Hum Neurosci. 2013 Aug 27;7:512. doi: 10.3389/fnhum.2013.00512. eCollection 2013. **PMID:** 23986687 **Citation:** Barr MS, Fitzgerald PB, Farzan F, George TP, Daskalakis ZJ. Transcranial magnetic stimulation to understand the pathophysiology and treatment of substance use disorders. Curr Drug Abuse Rev. 2008 Nov;1(3):328-39. doi: 10.2174/1874473710801030328. **PMID:** 19630729 **Citation:** Boivin JR, Piscopo DM, Wilbrecht L. Brief cognitive training interventions in young adulthood promote long-term resilience to drug-seeking behavior. Neuropharmacology. 2015 Oct;97:404-13. doi: 10.1016/j.neuropharm.2015.05.036. Epub 2015 Jun 9. **PMID:** 26066577 **Citation:** Conti CL, Moscon JA, Fregni F, Nitsche MA, Nakamura-Palacios EM. Cognitive related electrophysiological changes induced by non-invasive cortical electrical stimulation in crack-cocaine addiction. Int J Neuropsychopharmacol. 2014 Sep;17(9):1465-75. doi: 10.1017/S1461145714000522. Epub 2014 Apr 28. **PMID:** 24776374 **Citation:** Corbett A, Owen A, Hampshire A, Grahn J, Stenton R, Dajani S, Burns A, Howard R, Williams N, Williams G, Ballard C. The Effect of an Online Cognitive Training Package in Healthy Older Adults: An Online Randomized Controlled Trial. J Am Med Dir Assoc. 2015 Nov 1;16(11):990-7. doi: 10.1016/j.jamda.2015.06.014. **PMID:** 26543007 **Citation:** Ditye T, Jacobson L, Walsh V, Lavidor M. Modulating behavioral inhibition by tDCS combined with cognitive training. Exp Brain Res. 2012 Jun;219(3):363-8. doi: 10.1007/s00221-012-3098-4. Epub 2012 Apr 25. **PMID:** 22532165 **Citation:** Gluck ME, Alonso-Alonso M, Piaggi P, Weise CM, Jumpertz-von Schwartzenberg R, Reinhardt M, Wassermann EM, Venti CA, Votruba SB, Krakoff J. Neuromodulation targeted to the prefrontal cortex induces changes in energy intake and weight loss in obesity. Obesity (Silver Spring). 2015 Nov;23(11):2149-56. doi: 10.1002/oby.21313. **PMID:** 26530931 **Citation:** Goldman RL, Borckardt JJ, Frohman HA, O'Neil PM, Madan A, Campbell LK, Budak A, George MS. Prefrontal cortex transcranial direct current stimulation (tDCS) temporarily reduces food cravings and increases the self-reported ability to resist food in adults with frequent food craving. Appetite. 2011 Jun;56(3):741-6. doi: 10.1016/j.appet.2011.02.013. Epub 2011 Feb 23. **PMID:** 21352881 **Citation:** Grall-Bronnec M, Sauvaget A. The use of repetitive transcranial magnetic stimulation for modulating craving and addictive behaviours: a critical literature review of efficacy, technical and methodological considerations. Neurosci Biobehav Rev. 2014 Nov;47:592-613. doi: 10.1016/j.neubiorev.2014.10.013. **PMID:** 25454360 **Citation:** Jauch-Chara K, Kistenmacher A, Herzog N, Schwarz M, Schweiger U, Oltmanns KM. Repetitive electric brain stimulation reduces food intake in humans. Am J Clin Nutr. 2014 Oct;100(4):1003-9. doi: 10.3945/ajcn.113.075481. Epub 2014 Aug 6. **PMID:** 25099550 **Citation:** Juarascio AS, Manasse SM, Espel HM, Kerrigan SG, Forman EM. Could training executive function improve treatment outcomes for eating disorders? Appetite. 2015 Jul;90:187-93. doi: 10.1016/j.appet.2015.03.013. Epub 2015 Mar 14. **PMID:** 25777264 **Citation:** Sauvaget A, Trojak B, Bulteau S, Jimenez-Murcia S, Fernandez-Aranda F, Wolz I, Menchon JM, Achab S, Vanelle JM, Grall-Bronnec M. Transcranial direct current stimulation (tDCS) in behavioral and food addiction: a systematic review of efficacy, technical, and methodological issues. Front Neurosci. 2015 Oct 9;9:349. doi: 10.3389/fnins.2015.00349. eCollection 2015. **PMID:** 26500478 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000050177 - Term: Overweight - ID: D000044343 - Term: Overnutrition - ID: D000009748 - Term: Nutrition Disorders - ID: D000001835 - Term: Body Weight - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M7115 - Name: Diabetes Mellitus - Relevance: LOW - As Found: Unknown - ID: M10024 - Name: Hypertension - Relevance: HIGH - As Found: High Blood Pressure - ID: M12701 - Name: Obesity - Relevance: HIGH - As Found: Obesity - ID: M12702 - Name: Obesity, Morbid - Relevance: HIGH - As Found: Obesity, Morbid - ID: M26186 - Name: Overweight - Relevance: LOW - As Found: Unknown - ID: M25307 - Name: Overnutrition - Relevance: LOW - As Found: Unknown - ID: M12684 - Name: Nutrition Disorders - Relevance: LOW - As Found: Unknown - ID: M5114 - Name: Body Weight - Relevance: LOW - As Found: Unknown - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006973 - Term: Hypertension - ID: D000009765 - Term: Obesity - ID: D000009767 - Term: Obesity, Morbid ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01252979 **Brief Title:** Ketones & Mitochondrial Heteroplasmy **Official Title:** Ketones & Mitochondrial Heteroplasmy #### Organization Study ID Info **ID:** HSC-MS-10-0091 #### Organization **Class:** OTHER **Full Name:** The University of Texas Health Science Center, Houston ### Status Module #### Completion Date **Date:** 2011-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2012-02-06 **Type:** ESTIMATED **Last Update Submit Date:** 2012-02-03 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2011-12 **Type:** ACTUAL #### Start Date **Date:** 2010-12 **Status Verified Date:** 2012-02 #### Study First Post Date **Date:** 2010-12-03 **Type:** ESTIMATED **Study First Submit Date:** 2010-12-01 **Study First Submit QC Date:** 2010-12-01 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** The University of Texas Health Science Center, Houston #### Responsible Party **Investigator Affiliation:** The University of Texas Health Science Center, Houston **Investigator Full Name:** Mary Kay Koenig **Investigator Title:** Principle Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The current study is a prospective evaluation of the ability of ketosis to shift mitochondrial DNA (mtDNA) heteroplasmy in subjects harboring a known mutation in their mtDNA at position 3243 (A\>G). Subjects will be given supplemental medium chain triglycerides (MCTs) for a period of 6 months. mtDNA heteroplasmy will be measured 3 months prior to treatment, at treatment initiation, and 6 months after initiation. The primary objective of the current study is to determine if there is a shift in heteroplasmy in patients harboring the 3243 A\>G mtDNA mutation to a more favorable (higher wild-type) profile while in a state of ketosis. **Detailed Description:** The current study is a prospective evaluation of the ability of ketosis to shift mitochondrial DNA (mtDNA) heteroplasmy in subjects harboring a known pathogenic mutation in their mtDNA at position 3243 (A\>G). Subjects will be induced in to ketosis by administration of supplemental medium chain triglycerides (MCTs) for a period of 6 months. mtDNA heteroplasmy will be assessed 3 months prior to treatment, at treatment initiation, and 6 months after initiation. The primary objective of the current study is to determine if there is a shift in heteroplasmy in patients harboring the 3243 A\>G mtDNA mutation to a more favorable (higher wild-type) genotypic profile while in a state of ketosis. ### Conditions Module **Conditions:** - MELAS Syndrome - Mitochondrial Diseases **Keywords:** - MELAS - Ketones - Medium Chain Triglycerides - MCT oil - Mitochondrial Disorder - Heteroplasmy - 3243AG - Subject is informed and given ample opportunity to consider his/her participation and has given his/her written consent. - Subject is willing and able to comply with all trial requirements. - Subject harbors the 3243 A>G mtDNA mutation at a level detectable in blood. - Female subjects of child-bearing potential must not be pregnant. - Subjects must not have Diabetes Mellitus. ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 13 **Type:** ACTUAL **Phases:** - EARLY_PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Dietary Supplement: Medium-Chain Triglycerides **Label:** Medium Chain Triglyceride **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Medium Chain Triglyceride **Description:** Subjects will take supplemental MCT oil 3 times a day for 6 months **Name:** Medium-Chain Triglycerides **Type:** DIETARY_SUPPLEMENT ### Outcomes Module #### Primary Outcomes **Description:** Blood samples will be used to measure the degree of mitochondrial DNA heteroplasmy **Measure:** Heteroplasmy **Time Frame:** 9 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Subject is informed and given ample opportunity to consider his/her participation and has given his/her written consent. 2. Subject is willing and able to comply with all trial requirements. 3. Subject harbors the 3243 A\>G mtDNA mutation at a level detectable in blood. 4. Female subjects of child-bearing potential must not be pregnant. Female subjects of child-bearing potential (not surgically sterile or 2 years post-menopausal) must also agree to use appropriate contraceptive methods (abstinence, oral, injectable, implantable, or barrier) for the duration of the trial. 5. Subject must not have diabetes mellitus. Exclusion Criteria: 1. Subject is currently participating or has participated within the last 2 months in any clinical trial involving treatment of mitochondrial disorders with MCT supplementation or induction of ketosis. 2. Subject has a medical condition that could reasonably be exacerbated by ketone supplementation (including diabetes mellitus). 3. Subject is unable to give reasonable informed consent/assent. 4. Subject is a pregnant or nursing female. **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Houston **Country:** United States **Facility:** The University of Texas Health Science Center at Houston **State:** Texas **Zip:** 77030 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000008659 - Term: Metabolic Diseases - ID: D000017237 - Term: Mitochondrial Encephalomyopathies - ID: D000017240 - Term: Mitochondrial Myopathies - ID: D000009135 - Term: Muscular Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000020739 - Term: Brain Diseases, Metabolic, Inborn - ID: D000001928 - Term: Brain Diseases, Metabolic - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000059345 - Term: Cerebral Small Vessel Diseases - ID: D000002561 - Term: Cerebrovascular Disorders - ID: D000009468 - Term: Neuromuscular Diseases - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000008661 - Term: Metabolism, Inborn Errors - ID: D000030342 - Term: Genetic Diseases, Inborn ### Condition Browse Module - Browse Branches - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M7115 - Name: Diabetes Mellitus - Relevance: LOW - As Found: Unknown - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M19539 - Name: MELAS Syndrome - Relevance: HIGH - As Found: MELAS Syndrome - ID: M23341 - Name: Mitochondrial Diseases - Relevance: HIGH - As Found: Mitochondrial Disease - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: M19535 - Name: Mitochondrial Encephalomyopathies - Relevance: LOW - As Found: Unknown - ID: M12092 - Name: Muscular Diseases - Relevance: LOW - As Found: Unknown - ID: M19538 - Name: Mitochondrial Myopathies - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5205 - Name: Brain Diseases, Metabolic - Relevance: LOW - As Found: Unknown - ID: M22498 - Name: Brain Diseases, Metabolic, Inborn - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M29437 - Name: Cerebral Small Vessel Diseases - Relevance: LOW - As Found: Unknown - ID: M5810 - Name: Cerebrovascular Disorders - Relevance: LOW - As Found: Unknown - ID: M12411 - Name: Neuromuscular Diseases - Relevance: LOW - As Found: Unknown - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown - ID: M11641 - Name: Metabolism, Inborn Errors - Relevance: LOW - As Found: Unknown - ID: M23686 - Name: Genetic Diseases, Inborn - Relevance: LOW - As Found: Unknown - ID: T3840 - Name: Mitochondrial Encephalomyopathy Lactic Acidosis and Stroke-like Episodes - Relevance: HIGH - As Found: MELAS Syndrome ### Condition Browse Module - Meshes - ID: D000017241 - Term: MELAS Syndrome - ID: D000028361 - Term: Mitochondrial Diseases ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04397679 **Brief Title:** Partial Brain RT, Temozolomide, Chloroquine, and TTF Therapy for the Treatment of Newly Diagnosed Glioblastoma **Official Title:** Treatment of Adults With Newly Diagnosed Glioblastoma With Partial Brain Radiation Therapy Plus Temozolomide and Chloroquine Followed by Tumor Treating Fields Plus Temozolomide and Chloroquine -- A Pilot Study #### Organization Study ID Info **ID:** 2018-148 #### Organization **Class:** OTHER **Full Name:** Barbara Ann Karmanos Cancer Institute #### Secondary ID Infos **ID:** P30CA022453 **Link:** https://reporter.nih.gov/quickSearch/P30CA022453 **Type:** NIH ### Status Module #### Completion Date **Date:** 2026-04-27 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-03-26 **Type:** ACTUAL **Last Update Submit Date:** 2024-03-25 **Overall Status:** ACTIVE_NOT_RECRUITING #### Primary Completion Date **Date:** 2025-04-27 **Type:** ESTIMATED #### Start Date **Date:** 2021-08-12 **Type:** ACTUAL **Status Verified Date:** 2024-03 #### Study First Post Date **Date:** 2020-05-21 **Type:** ACTUAL **Study First Submit Date:** 2020-05-18 **Study First Submit QC Date:** 2020-05-18 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Cancer Institute (NCI) #### Lead Sponsor **Class:** OTHER **Name:** Barbara Ann Karmanos Cancer Institute #### Responsible Party **Investigator Affiliation:** Barbara Ann Karmanos Cancer Institute **Investigator Full Name:** Michael Dominello **Investigator Title:** Principal Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** True **Is FDA Regulated Drug:** True **Is US Export:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** This trial studies the side effects of partial brain radiation therapy, temozolomide, chloroquine, and tumor treating fields therapy for the treatment of newly diagnosed glioblastoma. Radiation therapy uses high energy x-rays to kill tumor cells and shrink tumors. Chemotherapy drugs, such as temozolomide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Chloroquine is normally used to treat strains of malaria and prior preclinical and clinical data suggests that it may increase the efficacy of both radiation and tumor treating fields therapy. Tumor treating fields therapy uses electric fields tuned to specific frequencies to disrupt cell division, inhibiting tumor growth and potentially causing cancer cells to die. The purpose of this study is to determine the safety of partial brain radiation therapy, temozolomide, chloroquine, and tumor treating fields therapy in patients with gliobastoma **Detailed Description:** PRIMARY OBJECTIVE: I. To determine the safety of partial brain radiation therapy plus temozolomide and chloroquine followed by tumor treating fields (TTFs) plus temozolomide and chloroquine, specifically grade 3 or higher dermatitis within the first 3 months of the adjuvant therapy phase. SECONDARY OBJECTIVE: I. To measure treatment effect via advanced magnetic resonance imaging (MRI) and alpha-\[11C\]methyl-L-tryptophan (AMT)-positron emission tomography (PET). OUTLINE: Patients undergo 30 fractions of 3-dimensional conformal radiation therapy (3D CRT) or intensity-modulated radiation therapy (IMRT) and receive temozolomide orally (PO) and chloroquine PO daily from day 1 for the duration of radiation therapy up to day 49. Treatment continues in the absence of disease progression or unacceptable toxicity. ADJUVANT TREATMENT: Beginning 4 weeks after the last day of radiation therapy, patients receive temozolomide PO once daily (QD) on days 1-5 and chloroquine PO daily on days 1-28. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Patients demonstrating continued benefit may continue to receive temozolomide and chloroquine for up to 12 cycles. Patients also undergo TTF therapy over 18 hours or longer per day. After completion of study treatment, patients are followed up monthly for 3 months. ### Conditions Module **Conditions:** - Glioblastoma - Gliosarcoma ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 2 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients undergo 30 fractions of 3D CRT or Intensity-modulated radiation therapy (IMRT) and receive temozolomide by mouth (PO) and chloroquine PO daily from day 1 for the duration of radiation therapy up to day 49. Treatment continues in the absence of disease progression or unacceptable toxicity. ADJUVANT TREATMENT: Beginning 4 weeks after the last day of radiation therapy, patients receive temozolomide PO QD on days 1-5 and chloroquine PO daily on days 1-28. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Patients demonstrating continued benefit may continue to receive temozolomide and chloroquine for up to 12 cycles. Patients also undergo TTF therapy over 18 hours or longer per day. **Intervention Names:** - Radiation: 3-Dimensional Conformal Radiation Therapy - Radiation: Intensity-Modulated Radiation Therapy (IMRT) - Drug: Temozolomide - Drug: Chloroquine - Procedure: Tumor Treating Fields Therapy (TTF) **Label:** Treatment (radiation therapy, temozolomide, chloroquine, TTF) **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Treatment (radiation therapy, temozolomide, chloroquine, TTF) **Description:** Undergo 3D CRT **Name:** 3-Dimensional Conformal Radiation Therapy **Other Names:** - 3-dimensional radiation therapy - 3D CONFORMAL RADIATION THERAPY - 3D CRT - 3D-CRT - Conformal Therapy - Radiation Conformal Therapy - Radiation - 3D Conformal **Type:** RADIATION #### Intervention 2 **Arm Group Labels:** - Treatment (radiation therapy, temozolomide, chloroquine, TTF) **Description:** Undergo IMRT **Name:** Intensity-Modulated Radiation Therapy (IMRT) **Other Names:** - IMRT - Intensity Modulated RT - INTENSITY-MODULATED RADIATION THERAPY - Intensity-Modulated Radiotherapy - Radiation **Type:** RADIATION #### Intervention 3 **Arm Group Labels:** - Treatment (radiation therapy, temozolomide, chloroquine, TTF) **Description:** Given PO **Name:** Temozolomide **Other Names:** - 3,4-dihydro-3-methyl-4-oxoimidazo[5,1-d]-1,2,3,5-tetrazine-8-carboxamide, 362856 - 8-carbamoyl-3-methylimidazo[5,1-d]-1,2,3,5-tetrazin-4(3H)-one, 85622-93-1, CCRG-81045 - imidazo[5,1-d]-1,2,3,5-tetrazine-8-carboxamide, 3 - 4-dihydro-3-methyl-4-oxo-, Imidazo[5,1-d] - 1,2,3,5-tetrazine-8-carboxamide - 3, 4-dihydro-3-methyl-4-oxo-, M & B 39831, M and B 39831 - Methazolastone - RP-46161 - SCH 52365 - Temcad - Temodal - Temomedac - TMZ **Type:** DRUG #### Intervention 4 **Arm Group Labels:** - Treatment (radiation therapy, temozolomide, chloroquine, TTF) **Description:** Given PO **Name:** Chloroquine **Other Names:** - 54-05-7 **Type:** DRUG #### Intervention 5 **Arm Group Labels:** - Treatment (radiation therapy, temozolomide, chloroquine, TTF) **Description:** Undergo TTF **Name:** Tumor Treating Fields Therapy (TTF) **Other Names:** - Alternating Electric Field Therapy - TTF - TTFields **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** Will summarize dermatitis status **Measure:** Proportion of patients who develop a specific acute toxicity (dermatitis) **Time Frame:** First 3 months of adjuvant therapy phase #### Secondary Outcomes **Description:** Will detail the safety profile and measure (via magnetic resonance imaging and positron emission tomography scans) the uptake and retention of alpha-\[11C\]methyl-L-tryptophan. **Measure:** Incidence of adverse events **Time Frame:** First 3 months of adjuvant therapy phase ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Histologically confirmed newly diagnosed grade IV glioma (gliosarcoma allowed) * The subject must have recovered from the effects of surgery, postoperative infection, and other complications before enrollment. Post-operative unenhanced and contrast-enhanced MRI scan should be done within 72 hours after surgery. If it is not obtained within 72 hours post-resection, then an MRI obtained at least 2 weeks (or longer) after surgery is required * Karnofsky performance status \>= 70% * Absolute neutrophil count \>= 1,500/mm\^3 (=\< 21 days prior to registration) * Platelets \>= 100,000/mm\^3 (=\< 21 days prior to registration) * Hemoglobin (Hgb) \>= 9.0 g/dL (Note: The use of transfusion or other intervention to achieve Hgb \>= 9.0 g/dL is acceptable.) (=\< 21 days prior to registration) * Calculated creatinine clearance \>= 30 mL/min by Cockcroft-Gault formula (=\< 21 days prior to registration) * Total bilirubin =\< 1.5 times upper limit of normal (ULN) (=\< 21 days prior to registration) * Aspartate aminotransferase (AST)(serum glutamic-oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT)(serum glutamate pyruvate transaminase \[SGPT\]) =\< 1.5 times upper limit of normal (ULN) (=\< 21 days prior to registration) * Female subjects of childbearing potential (i.e., those who are not postmenopausal for at least 1 year or surgically sterile by bilateral tubal ligation, bilateral oophorectomy or hysterectomy) and their male partners should practice at least one of the methods of birth control listed below during study entry, for the entire duration of the study and for at least 6 months after treatment with temozolomide and chloroquine: \* A vasectomized male subject or a vasectomized partner of a female subject; hormonal contraceptives (oral, parenteral, or transdermal) for at least 3 months prior to study drug administration; intrauterine device (females); double-barrier method (condoms, contraceptive sponge, diaphragm or vaginal ring with spermicidal jellies or cream) * Female subjects of child-bearing potential must have a negative pregnancy test (urine or serum) within 3 days of registration * Must voluntarily sign and date informed consent form for study participation, approved by an Independent Ethics Committee (IEC)/Institutional Review Board (IRB), prior to the initiation of any screening or study-specific procedures Exclusion Criteria: * Gliomatosis cerebri (a diffuse glioma \[usually astrocytic\] growth pattern consisting of exceptionally extensive infiltration of a large region of the central nervous system, with involvement of at least 3 cerebral lobes, usually with bilateral involvement of the cerebral hemispheres and/or deep grey matter, and frequent extension to the brain stem, cerebellum, and even the spinal cord.) * Recurrent glioblastoma (GBM) * Metastatic GBM * Infratentorial tumor * Prior chemotherapy or radiosensitizers for cancers of the head and neck region; note that prior chemotherapy for a different cancer is allowable, except prior temozolomide. Implanted carmustine (BCNU) wafer is allowed * Prior radiotherapy to the head or neck (except for T1 glottic cancer or nonmelanomatous skin cancer), resulting in overlap of radiation fields * Any prior therapy for glioblastoma besides surgery (intra-operative techniques to guide resection and experimental imaging techniques are allowed). BCNU wafer is allowed * Prior invasive malignancy (except for non-melanomatous skin cancer; carcinoma in situ (CIS) of the breast, CIS oral cavity, or CIS cervix, T1 glottic cancer) unless disease free for \>= 5 years * Prior, concomitant, or planned concomitant treatment with bevacizumab, carmustine implant (Gliadel) wafers or other intratumoral or intracavitary anti-neoplastic therapy, or other experimental therapeutics intended to treat the tumor; the exceptions are diagnostic and operative guides to improve extent of resection or imaging studies, quality of life, biomarker, or epidemiological studies * History of hypersensitivity to temozolomide or excipients * Known glucose-6-phosphate dehydrogenase (G6PD) deficiency * Lactating or pregnant female * Severe, active, co-morbidity defined as follows: \* Moderate or severe hepatic impairment (Child-Pugh category B or higher \[score of 7 or higher \]); unstable angina and/or congestive heart failure within the last 6 months; transmural myocardial infarction within the last 6 months; evidence of recent myocardial infarction or ischemia by the findings of S-T elevations of \>= 2 mm using the analysis of an electrocardiogram (EKG) performed within 14 days prior to enrollment; New York Heart Association grade II or greater congestive heart failure requiring hospitalization within 12 months prior to enrollment * History of stroke, cerebral vascular accident (CVA), or transient ischemic attack within 6 months (except if intra- or post-operative); serious and inadequately controlled cardiac arrhythmia; acute bacterial or fungal infection requiring intravenous antibiotics at the time of enrollment; chronic obstructive pulmonary disease exacerbation or other respiratory illness requiring hospitalization or precluding study therapy at the time of enrollment; uncontrolled human immunodeficiency virus (HIV) with CD4 count \< 200; note, however, that HIV testing is not required for entry into this protocol * Any other major medical illnesses or psychiatric impairments that in the investigator's opinion will prevent administration or completion of protocol therapy * Subjects treated on any other therapeutic clinical protocols within 30 days prior to study entry or during participation in the study, except intra-operative therapy to guide resection or experimental imaging without therapeutic intent * Inability to undergo contrast-enhanced MRI scans * Presence of implanted pacemaker, programmable shunts, defibrillator, deep brain stimulator, or other implanted electronic devices in the brain * Documented clinically significant arrhythmia or severe ischemic heart disease * Patients with underlying ocular disorders, including but not limited to: maculopathy, macular degeneration, and retinopathy **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Detroit **Country:** United States **Facility:** Barbara Ann Karmanos Cancer Institute **State:** Michigan **Zip:** 48201 #### Overall Officials **Official 1:** **Affiliation:** Barbara Ann Karmanos Institute **Name:** Michael Dominello, M.D. **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001254 - Term: Astrocytoma - ID: D000005910 - Term: Glioma - ID: D000018302 - Term: Neoplasms, Neuroepithelial - ID: D000017599 - Term: Neuroectodermal Tumors - ID: D000009373 - Term: Neoplasms, Germ Cell and Embryonal - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009380 - Term: Neoplasms, Nerve Tissue ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M9019 - Name: Glioblastoma - Relevance: HIGH - As Found: Glioblastoma - ID: M20460 - Name: Gliosarcoma - Relevance: HIGH - As Found: Gliosarcoma - ID: M4561 - Name: Astrocytoma - Relevance: LOW - As Found: Unknown - ID: M9020 - Name: Glioma - Relevance: LOW - As Found: Unknown - ID: M20446 - Name: Neoplasms, Neuroepithelial - Relevance: LOW - As Found: Unknown - ID: M19845 - Name: Neuroectodermal Tumors - Relevance: LOW - As Found: Unknown - ID: M20388 - Name: Neuroectodermal Tumors, Primitive - Relevance: LOW - As Found: Unknown - ID: M12318 - Name: Neoplasms, Germ Cell and Embryonal - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12325 - Name: Neoplasms, Nerve Tissue - Relevance: LOW - As Found: Unknown - ID: T2518 - Name: Glioblastoma - Relevance: HIGH - As Found: Glioblastoma - ID: T2522 - Name: Gliosarcoma - Relevance: HIGH - As Found: Gliosarcoma - ID: T2519 - Name: Glioma - Relevance: LOW - As Found: Unknown - ID: T4092 - Name: Neuroepithelioma - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000005909 - Term: Glioblastoma - ID: D000018316 - Term: Gliosarcoma ### Intervention Browse Module - Ancestors - ID: D000018906 - Term: Antineoplastic Agents, Alkylating - ID: D000000477 - Term: Alkylating Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000000970 - Term: Antineoplastic Agents - ID: D000000563 - Term: Amebicides - ID: D000000981 - Term: Antiprotozoal Agents - ID: D000000977 - Term: Antiparasitic Agents - ID: D000000890 - Term: Anti-Infective Agents - ID: D000000962 - Term: Antimalarials - ID: D000018501 - Term: Antirheumatic Agents ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: ARhu - Name: Antirheumatic Agents - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: Analg - Name: Analgesics ### Intervention Browse Module - Browse Leaves - ID: M1692 - Name: Temozolomide - Relevance: HIGH - As Found: Skills - ID: M5979 - Name: Chloroquine - Relevance: HIGH - As Found: Metabolic Syndrome - ID: M244350 - Name: Chloroquine diphosphate - Relevance: LOW - As Found: Unknown - ID: M20942 - Name: Antineoplastic Agents, Alkylating - Relevance: LOW - As Found: Unknown - ID: M3820 - Name: Alkylating Agents - Relevance: LOW - As Found: Unknown - ID: M3904 - Name: Amebicides - Relevance: LOW - As Found: Unknown - ID: M4298 - Name: Antiprotozoal Agents - Relevance: LOW - As Found: Unknown - ID: M4294 - Name: Antiparasitic Agents - Relevance: LOW - As Found: Unknown - ID: M4214 - Name: Anti-Infective Agents - Relevance: LOW - As Found: Unknown - ID: M4280 - Name: Antimalarials - Relevance: LOW - As Found: Unknown - ID: M20604 - Name: Antirheumatic Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000002738 - Term: Chloroquine - ID: D000077204 - Term: Temozolomide ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02467179 **Acronym:** Amigo-AFL **Brief Title:** Evaluation of the Amigo Robotic System for Ablation of the Cavo-Tricuspid Isthmus **Official Title:** Evaluation of the Amigo Robotic System for Ablation of the Cavo-Tricuspid Isthmus #### Organization Study ID Info **ID:** 150318 #### Organization **Class:** OTHER **Full Name:** University of California, San Diego ### Status Module #### Completion Date **Date:** 2018-03-30 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2019-06-27 **Type:** ACTUAL **Last Update Submit Date:** 2019-06-07 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2017-10-30 **Type:** ACTUAL #### Results First Post Date **Date:** 2019-06-27 **Type:** ACTUAL **Results First Submit Date:** 2019-05-13 **Results First Submit QC Date:** 2019-06-07 #### Start Date **Date:** 2015-03 **Type:** ACTUAL **Status Verified Date:** 2019-06 #### Study First Post Date **Date:** 2015-06-09 **Type:** ESTIMATED **Study First Submit Date:** 2015-06-03 **Study First Submit QC Date:** 2015-06-05 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Catheter Robotics, Inc. #### Lead Sponsor **Class:** OTHER **Name:** University of California, San Diego #### Responsible Party **Investigator Affiliation:** University of California, San Diego **Investigator Full Name:** Gregory Kent Feld **Investigator Title:** Professor of Medicine **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to compare standard manual and robotically controlled catheter ablation of the cavo-tricuspid isthmus (CTI) as a treatment for atrial flutter. Ablation of the CTI is standard treatment for patients with a history of atrial flutter, and those undergoing ablation for atrial fibrillation. Both manual and robotic catheter manipulation are used in standard clinical practice at The University of California, San Diego (UCSD) for ablation. **Detailed Description:** Dr. Gregory Feld, M.D. is conducting a research study to find out more about the effects of using the Amigo™ Robotic System for ablation of the cavo-tricuspid isthmus (flutter isthmus) on catheter stability (stabilization of the catheter during the procedure in order to prevent movement and dislodgement) using a combination of contact force (a measurement of the force applied by the catheter tip against the tissue during the ablation procedure), location, and duration measurements during ablation of the atrial flutter circuit. Published studies have indicated that robotic catheter manipulation is safe, may reduce patient and physician x-ray exposure, and ablation procedure time. Measuring contact force has also been shown to improve both short-term and long-term procedure success. However, research on ablation of the CTI specifically, is limited. This is a randomized study comparing robotic catheter manipulation using the Amigo Robotic System to manual catheter manipulation for ablation of the CTI on measures including catheter stability, contact force, procedure time, and fluoroscopy (X-ray) time. The ablation procedure is standard of care, however, if patients chose to enroll in the study they will be randomized to one of two groups: either manual or robotic catheter manipulation. Throughout the procedure, research personnel will collect information from the procedure including measurements of contact force, catheter stability, procedure duration, and fluoroscopy (X-ray) time. If, in addition to ablation of the cavo-tricuspid isthmus for atrial flutter, a patient is scheduled to undergo ablation of the left atrium for atrial fibrillation or left atrial flutter, this will be performed subsequently in a standard manner, and will not be considered part of the research. Ablation of the CTI generally takes 45-60 minutes regardless of the technique used for manipulation of the catheters. The patient's participation in the study will only last one day, the length of study procedure. There is no follow up associated with this study. All subjects will received standard of care treatment following their ablation. ### Conditions Module **Conditions:** - Atrial Fibrillation - Atrial Flutter **Keywords:** - Arrhythmia - cavo-tricuspid isthmus - atrial fibrillation - atrial fibrillation ablation ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 50 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 25 subjects will be randomized to this arm. Manual catheter ablation of the cavo-tricuspid isthmus will be performed. **Intervention Names:** - Device: Manual Catheter Manipulation **Label:** Manual Catheter Manipulation **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** 25 subjects will be randomized to this arm. Robotic catheter ablation of the cavo-tricuspid isthmus with the Amigo Catheter System will be performed. **Intervention Names:** - Device: Amigo™ Robotic Catheter Manipulation **Label:** Amigo™ Robotic Catheter Manipulation **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Amigo™ Robotic Catheter Manipulation **Description:** Ablation is a standard procedure that patients may undergo for the atrial flutter. In this study, this group will be assigned to robotic catheter manipulation. **Name:** Amigo™ Robotic Catheter Manipulation **Other Names:** - cavo-tricuspid isthmus ablation - atrial flutter ablation - arrhythmia - radiofrequency catheter ablation **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - Manual Catheter Manipulation **Description:** Ablation is a standard procedure that patients may undergo for the atrial flutter. In this study, this group will be assigned to manual catheter manipulation. **Name:** Manual Catheter Manipulation **Other Names:** - cavo-tricuspid isthmus ablation - atrial flutter ablation - arrhythmia - radiofrequency catheter ablation **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Determination of the average total contact force (measured in gs) achieved during each ablation lesion using the Carto Mapping System **Measure:** Contact Force Through Measurement of Force-time Integral (FTI) **Time Frame:** At time of the ablation procedure, which typically lasts 30-60 minutes #### Secondary Outcomes **Description:** Determine fluoroscopy time to reach CTI block. **Measure:** Fluoroscopy Time Measurements **Time Frame:** at time of ablation procedure ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Must be scheduled to undergo radiofrequency catheter ablation of the cavo-tricuspid isthmus for atrial fibrillation (AF) or atrial flutter (AFL) according to appropriate clinical indications. * Must be able and willing to provide written informed consent * Must be at least 18 years old. Exclusion Criteria: * Patient's refusal to participate in the study * Lack of indication for CTI ablation (eg: prior CTI ablation with persistent bidirectional isthmus block) * Pregnancy **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** La Jolla **Country:** United States **Facility:** Sulpizio Cardiovascular Center **State:** California **Zip:** 92037 #### Overall Officials **Official 1:** **Affiliation:** UC San Diego **Name:** Gregory Feld, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Wood MA, Orlov M, Ramaswamy K, Haffajee C, Ellenbogen K; Stereotaxis Heart Study Investigators. Remote magnetic versus manual catheter navigation for ablation of supraventricular tachycardias: a randomized, multicenter trial. Pacing Clin Electrophysiol. 2008 Oct;31(10):1313-21. doi: 10.1111/j.1540-8159.2008.01183.x. **PMID:** 18811813 **Citation:** Proietti R, Pecoraro V, Di Biase L, Natale A, Santangeli P, Viecca M, Sagone A, Galli A, Moja L, Tagliabue L. Remote magnetic with open-irrigated catheter vs. manual navigation for ablation of atrial fibrillation: a systematic review and meta-analysis. Europace. 2013 Sep;15(9):1241-8. doi: 10.1093/europace/eut058. Epub 2013 Apr 12. **PMID:** 23585253 **Citation:** Hlivak P, Mlcochova H, Peichl P, Cihak R, Wichterle D, Kautzner J. Robotic navigation in catheter ablation for paroxysmal atrial fibrillation: midterm efficacy and predictors of postablation arrhythmia recurrences. J Cardiovasc Electrophysiol. 2011 May;22(5):534-40. doi: 10.1111/j.1540-8167.2010.01942.x. Epub 2010 Nov 23. **PMID:** 21091964 **Citation:** Feld G, Wharton M, Plumb V, Daoud E, Friehling T, Epstein L; EPT-1000 XP Cardiac Ablation System Investigators. Radiofrequency catheter ablation of type 1 atrial flutter using large-tip 8- or 10-mm electrode catheters and a high-output radiofrequency energy generator: results of a multicenter safety and efficacy study. J Am Coll Cardiol. 2004 Apr 21;43(8):1466-72. doi: 10.1016/j.jacc.2003.11.036. **PMID:** 15093885 **Citation:** Sawhney N, Anousheh R, Chen WC, Narayan S, Feld GK. Five-year outcomes after segmental pulmonary vein isolation for paroxysmal atrial fibrillation. Am J Cardiol. 2009 Aug 1;104(3):366-72. doi: 10.1016/j.amjcard.2009.03.044. Epub 2009 Jun 6. **PMID:** 19616669 **Citation:** Steinberg JS, Palekar R, Sichrovsky T, Arshad A, Preminger M, Musat D, Shaw RE, Mittal S. Very long-term outcome after initially successful catheter ablation of atrial fibrillation. Heart Rhythm. 2014 May;11(5):771-6. doi: 10.1016/j.hrthm.2014.02.003. Epub 2014 Feb 4. **PMID:** 24508206 **Citation:** Akca F, Janse P, Theuns DA, Szili-Torok T. A prospective study on safety of catheter ablation procedures: contact force guided ablation could reduce the risk of cardiac perforation. Int J Cardiol. 2015 Jan 20;179:441-8. doi: 10.1016/j.ijcard.2014.11.105. Epub 2014 Nov 13. **PMID:** 25465303 **Citation:** Sigmund E, Puererfellner H, Derndorfer M, Kollias G, Winter S, Aichinger J, Nesser HJ, Martinek M. Optimizing radiofrequency ablation of paroxysmal and persistent atrial fibrillation by direct catheter force measurement-a case-matched comparison in 198 patients. Pacing Clin Electrophysiol. 2015 Feb;38(2):201-8. doi: 10.1111/pace.12549. Epub 2014 Dec 2. **PMID:** 25469738 **Citation:** Jarman JWE, Panikker S, DAS M, Wynn GJ, Ullah W, Kontogeorgis A, Haldar SK, Patel PJ, Hussain W, Markides V, Gupta D, Schilling RJ, Wong T. Relationship between contact force sensing technology and medium-term outcome of atrial fibrillation ablation: a multicenter study of 600 patients. J Cardiovasc Electrophysiol. 2015 Apr;26(4):378-384. doi: 10.1111/jce.12606. Epub 2015 Feb 11. **PMID:** 25546580 **Citation:** le Polain de Waroux JB, Weerasooriya R, Anvardeen K, Barbraud C, Marchandise S, De Meester C, Goesaert C, Reis I, Scavee C. Low contact force and force-time integral predict early recovery and dormant conduction revealed by adenosine after pulmonary vein isolation. Europace. 2015 Jun;17(6):877-83. doi: 10.1093/europace/euu329. Epub 2015 Jan 24. **PMID:** 25618742 **Citation:** Arujuna A, Karim R, Zarinabad N, Gill J, Rhode K, Schaeffter T, Wright M, Rinaldi CA, Cooklin M, Razavi R, O'Neill MD, Gill JS. A randomized prospective mechanistic cardiac magnetic resonance study correlating catheter stability, late gadolinium enhancement and 3 year clinical outcomes in robotically assisted vs. standard catheter ablation. Europace. 2015 Aug;17(8):1241-50. doi: 10.1093/europace/euu364. Epub 2015 Feb 16. **PMID:** 25687748 **Citation:** Shurrab M, Danon A, Lashevsky I, Kiss A, Newman D, Szili-Torok T, Crystal E. Robotically assisted ablation of atrial fibrillation: a systematic review and meta-analysis. Int J Cardiol. 2013 Nov 5;169(3):157-65. doi: 10.1016/j.ijcard.2013.08.086. Epub 2013 Sep 10. **PMID:** 24063921 **Citation:** Lopez-Gil M, Salgado R, Merino JL, Datino T, Figueroa J, Arenal A, Mejia E, Salguero R, Fontenla A, Arribas F. Cavo-tricuspid isthmus radiofrequency ablation using a novel remote navigation catheter system in patients with typical atrial flutter. Europace. 2014 Apr;16(4):558-62. doi: 10.1093/europace/eut285. Epub 2013 Sep 20. **PMID:** 24058180 **Citation:** Datino T, Arenal A, Pelliza M, Hernandez-Hernandez J, Atienza F, Gonzalez-Torrecilla E, Avila P, Bravo L, Fernandez-Aviles F. Comparison of the safety and feasibility of arrhythmia ablation using the Amigo Robotic Remote Catheter System versus manual ablation. Am J Cardiol. 2014 Mar 1;113(5):827-31. doi: 10.1016/j.amjcard.2013.11.030. Epub 2013 Dec 12. **PMID:** 24440330 **Citation:** Zhang W, Jia N, Su J, Lin J, Peng F, Niu W. The comparison between robotic and manual ablations in the treatment of atrial fibrillation: a systematic review and meta-analysis. PLoS One. 2014 May 6;9(5):e96331. doi: 10.1371/journal.pone.0096331. eCollection 2014. **PMID:** 24800808 **Citation:** Wutzler A, Wolber T, Parwani AS, Huemer M, Attanasio P, Blaschke F, Haegeli L, Haverkamp W, Duru F, Boldt LH. Robotic ablation of atrial fibrillation with a new remote catheter system. J Interv Card Electrophysiol. 2014 Sep;40(3):215-9. doi: 10.1007/s10840-014-9895-x. Epub 2014 Apr 29. **PMID:** 24771262 **Citation:** Thomas D, Scholz EP, Schweizer PA, Katus HA, Becker R. Initial experience with robotic navigation for catheter ablation of paroxysmal and persistent atrial fibrillation. J Electrocardiol. 2012 Mar;45(2):95-101. doi: 10.1016/j.jelectrocard.2011.05.005. Epub 2011 Jun 28. **PMID:** 21714971 **Citation:** Hoffmayer KS, Krainski F, Shah S, Hunter J, Alegre M, Hsu JC, Feld GK. Randomized controlled trial of Amigo(R) robotically controlled versus manually controlled ablation of the cavo-tricuspid isthmus using a contact force ablation catheter. J Interv Card Electrophysiol. 2018 Mar;51(2):125-132. doi: 10.1007/s10840-018-0319-1. Epub 2018 Feb 12. **PMID:** 29435790 ## Document Section ### Large Document Module #### Large Docs - Date: 2015-03-19 - Filename: Prot_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: False - Label: Study Protocol - Size: 276389 - Type Abbrev: Prot - Upload Date: 2019-06-07T13:09 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001145 - Term: Arrhythmias, Cardiac - ID: D000006331 - Term: Heart Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M4453 - Name: Arrhythmias, Cardiac - Relevance: LOW - As Found: Unknown - ID: M4586 - Name: Atrial Fibrillation - Relevance: HIGH - As Found: Atrial Fibrillation - ID: M4587 - Name: Atrial Flutter - Relevance: HIGH - As Found: Atrial Flutter - ID: M9419 - Name: Heart Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001281 - Term: Atrial Fibrillation - ID: D000001282 - Term: Atrial Flutter ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Manual Catheter Manipulation **Deaths Num At Risk:** 25 **Description:** 25 subjects will be randomized to this arm. Manual catheter ablation of the cavo-tricuspid isthmus will be performed. Manual Catheter Manipulation: Ablation is a standard procedure that patients may undergo for the atrial flutter. In this study, this group will be assigned to manual catheter manipulation. **ID:** EG000 **Other Num at Risk:** 25 **Serious Number At Risk:** 25 **Title:** Manual Catheter Manipulation **Group ID:** EG001 **Title:** Amigo™ Robotic Catheter Manipulation **Deaths Num At Risk:** 25 **Description:** 25 subjects will be randomized to this arm. Robotic catheter ablation of the cavo-tricuspid isthmus with the Amigo Catheter System will be performed. Amigo™ Robotic Catheter Manipulation: Ablation is a standard procedure that patients may undergo for the atrial flutter. In this study, this group will be assigned to robotic catheter manipulation. **ID:** EG001 **Other Num at Risk:** 25 **Serious Number At Risk:** 25 **Title:** Amigo™ Robotic Catheter Manipulation **Frequency Threshold:** 0 **Time Frame:** From the time of enrollment to completion of the ablation procedure (which usually lasted 30-60 minutes). ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 25 **Group ID:** BG001 **Value:** 25 **Group ID:** BG002 **Value:** 50 **Units:** Participants ### Group **ID:** BG000 **Title:** Manual Catheter Manipulation **Description:** 25 subjects will be randomized to this arm. Manual catheter ablation of the cavo-tricuspid isthmus will be performed. Manual Catheter Manipulation: Ablation is a standard procedure that patients may undergo for the atrial flutter. In this study, this group will be assigned to manual catheter manipulation. ### Group **ID:** BG001 **Title:** Amigo™ Robotic Catheter Manipulation **Description:** 25 subjects will be randomized to this arm. Robotic catheter ablation of the cavo-tricuspid isthmus with the Amigo Catheter System will be performed. Amigo™ Robotic Catheter Manipulation: Ablation is a standard procedure that patients may undergo for the atrial flutter. In this study, this group will be assigned to robotic catheter manipulation. ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 25 #### Measurement **Group ID:** BG001 **Value:** 25 #### Measurement **Group ID:** BG002 **Value:** 50 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** >=65 years #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 25 **Group ID:** BG001 **Value:** 25 **Group ID:** BG002 **Value:** 50 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 12 **Value:** 63 #### Measurement **Group ID:** BG001 **Spread:** 12 **Value:** 63 #### Measurement **Group ID:** BG002 **Spread:** 12 **Value:** 63 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 25 **Group ID:** BG001 **Value:** 25 **Group ID:** BG002 **Value:** 50 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 8 #### Measurement **Group ID:** BG001 **Value:** 6 #### Measurement **Group ID:** BG002 **Value:** 14 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 17 #### Measurement **Group ID:** BG001 **Value:** 19 #### Measurement **Group ID:** BG002 **Value:** 36 **Category Title:** Male #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 25 **Group ID:** BG001 **Value:** 25 **Group ID:** BG002 **Value:** 50 **Class Title:** ### Measure #### Measurement **Group ID:** BG002 **Value:** 0 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 0 **Group ID:** BG001 **Value:** 0 **Group ID:** BG002 **Value:** 0 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 25 #### Measurement **Group ID:** BG001 **Value:** 25 #### Measurement **Group ID:** BG002 **Value:** 50 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 25 **Group ID:** BG001 **Value:** 25 **Group ID:** BG002 **Value:** 50 **Class Title:** United States **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Age, Categorical **Unit of Measure:** Participants ### Measure 2 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Race and Ethnicity were not collected from any participant. **Title:** Race and Ethnicity Not Collected **Unit of Measure:** Participants ### Measure 5 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants ## Results Section - More Information Module ### Certain Agreement ### Point of Contact **Email:** gfeld@ucsd.edu **Organization:** University of California San Diego **Phone:** (858) 246-2972 **Title:** Dr. Gregory Feld ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 179 - **Upper Limit:** - **Value:** 471 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 278 - **Upper Limit:** - **Value:** 571 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.3 - **Upper Limit:** - **Value:** 3.8 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 4.4 - **Upper Limit:** - **Value:** 6.8 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Determination of the average total contact force (measured in gs) achieved during each ablation lesion using the Carto Mapping System **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** At time of the ablation procedure, which typically lasts 30-60 minutes **Title:** Contact Force Through Measurement of Force-time Integral (FTI) **Type:** PRIMARY **Unit of Measure:** gm/s ##### Group **Description:** 25 subjects will be randomized to this arm. Manual catheter ablation of the cavo-tricuspid isthmus will be performed. Manual Catheter Manipulation: Ablation is a standard procedure that patients may undergo for the atrial flutter. In this study, this group will be assigned to manual catheter manipulation. **ID:** OG000 **Title:** Manual Catheter Manipulation ##### Group **Description:** 25 subjects will be randomized to this arm. Robotic catheter ablation of the cavo-tricuspid isthmus with the Amigo Catheter System will be performed. Amigo™ Robotic Catheter Manipulation: Ablation is a standard procedure that patients may undergo for the atrial flutter. In this study, this group will be assigned to robotic catheter manipulation. **ID:** OG001 **Title:** Amigo™ Robotic Catheter Manipulation #### Outcome Measure 2 **Description:** Determine fluoroscopy time to reach CTI block. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** at time of ablation procedure **Title:** Fluoroscopy Time Measurements **Type:** SECONDARY **Unit of Measure:** minutes ##### Group **Description:** 25 subjects will be randomized to this arm. Manual catheter ablation of the cavo-tricuspid isthmus will be performed. Manual Catheter Manipulation: Ablation is a standard procedure that patients may undergo for the atrial flutter. In this study, this group will be assigned to manual catheter manipulation. **ID:** OG000 **Title:** Manual Catheter Manipulation ##### Group **Description:** 25 subjects will be randomized to this arm. Robotic catheter ablation of the cavo-tricuspid isthmus with the Amigo Catheter System will be performed. Amigo™ Robotic Catheter Manipulation: Ablation is a standard procedure that patients may undergo for the atrial flutter. In this study, this group will be assigned to robotic catheter manipulation. **ID:** OG001 **Title:** Amigo™ Robotic Catheter Manipulation ### Participant Flow Module #### Group **Description:** 25 subjects will be randomized to this arm. Manual catheter ablation of the cavo-tricuspid isthmus will be performed. Manual Catheter Manipulation: Ablation is a standard procedure that patients may undergo for the atrial flutter. In this study, this group will be assigned to manual catheter manipulation. **ID:** FG000 **Title:** Manual Catheter Manipulation #### Group **Description:** 25 subjects will be randomized to this arm. Robotic catheter ablation of the cavo-tricuspid isthmus with the Amigo Catheter System will be performed. Amigo™ Robotic Catheter Manipulation: Ablation is a standard procedure that patients may undergo for the atrial flutter. In this study, this group will be assigned to robotic catheter manipulation. **ID:** FG001 **Title:** Amigo™ Robotic Catheter Manipulation #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 25 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 25 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 25 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 25 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT03872479 **Brief Title:** Single Ascending Dose Study in Participants With LCA10 **Official Title:** Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Efficacy of EDIT-101 in Adult and Pediatric Participants With Leber Congenital Amaurosis Type 10 (LCA10), With Centrosomal Protein 290 (CEP290)-Related Retinal Degeneration Caused by a Compound Heterozygous or Homozygous Mutation Involving c.2991+1655A>G in Intron 26 (IVS26) of the CEP290 Gene ("LCA10-IVS26") #### Organization Study ID Info **ID:** 1991-201-008 #### Organization **Class:** INDUSTRY **Full Name:** Editas Medicine, Inc. ### Status Module #### Completion Date **Date:** 2025-05-23 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2022-12-05 **Type:** ACTUAL **Last Update Submit Date:** 2022-12-02 **Overall Status:** ACTIVE_NOT_RECRUITING #### Primary Completion Date **Date:** 2025-05-23 **Type:** ESTIMATED #### Start Date **Date:** 2019-09-26 **Type:** ACTUAL **Status Verified Date:** 2022-12 #### Study First Post Date **Date:** 2019-03-13 **Type:** ACTUAL **Study First Submit Date:** 2018-10-12 **Study First Submit QC Date:** 2019-03-11 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Editas Medicine, Inc. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A\>G in intron 26 of the CEP290 gene ("LCA10-IVS26"). **Detailed Description:** This is an open-label, single ascending dose study of EDIT-101 in adult and pediatric (ie, ages 3 to 17) participants with LCA10-IVS26. Up to 34 participants will be enrolled in up to 5 cohorts to evaluate up to 3 dose levels of EDIT-101 in this study. EDIT-101 is a novel gene editing product designed to eliminate the mutation on the CEP290 gene that results in the retinal degeneration that defines LCA10-IVS26. ### Conditions Module **Conditions:** - Leber Congenital Amaurosis 10 - Inherited Retinal Dystrophies - Eye Diseases, Hereditary - Retinal Disease - Retinal Degeneration - Vision Disorders - Eye Disorders Congenital **Keywords:** - CEP290 - LCA10 - Retinal degenerative diseases (RDD) - Leber congenital amaurosis (LCA) - Congenital Retinal Blindness - p.Cys998X - c.2991+1655A>G - CRISPR Treatment - Cas9 Protein - Eye Diseases Signs and Symptoms - Genetic Diseases, Inborn - Congenital Abnormalities - Eye Abnormalities - CRISPR-Cas9 - CRISPR Associated Protein 9 - Cas9 Enzyme ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** SEQUENTIAL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 34 **Type:** ESTIMATED **Phases:** - PHASE1 - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Single dose of EDIT-101 administered by subretinal injection surgery **Intervention Names:** - Drug: EDIT-101 **Label:** Adults Low Dose **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Single dose of EDIT-101 administered by subretinal injection surgery **Intervention Names:** - Drug: EDIT-101 **Label:** Adults Middle Dose **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Single dose of EDIT-101 administered by subretinal injection surgery **Intervention Names:** - Drug: EDIT-101 **Label:** Adults High Dose **Type:** EXPERIMENTAL #### Arm Group 4 **Description:** Single dose of EDIT-101 administered by subretinal injection surgery **Intervention Names:** - Drug: EDIT-101 **Label:** Pediatric Middle Dose **Type:** EXPERIMENTAL #### Arm Group 5 **Description:** Single dose of EDIT-101 administered by subretinal injection surgery **Intervention Names:** - Drug: EDIT-101 **Label:** Pediatric High Dose **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Adults High Dose - Adults Low Dose - Adults Middle Dose - Pediatric High Dose - Pediatric Middle Dose **Description:** Participants will receive a single dose of EDIT-101 administered via subretinal injection in the study eye. Up to 5 cohorts across 3 doses will be enrolled in this study. **Name:** EDIT-101 **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Frequency of Adverse Events related to EDIT-101 **Time Frame:** 1 year **Measure:** Number of participants experiencing procedural related adverse events **Time Frame:** 1 year **Measure:** Incidence of dose limiting toxicities **Time Frame:** 1 year #### Secondary Outcomes **Measure:** Maximum tolerated dose as determined by occurrence of dose limiting toxicities **Time Frame:** 1 year **Description:** Testing the subjects visual function by having the subject walk through obstacle courses. Courses will have different levels of difficulty depending on the light levels of the room and the contrast of the objects in the room. **Measure:** Change from baseline in Mobility course score **Time Frame:** 1 year **Description:** The test will evaluate visual acuity in ranges from light perception to normal vision. **Measure:** Change from baseline in LogMAR measurement of BCVA **Time Frame:** 1 year **Description:** Measuring the change in pupil diameter in response to a light stimulus. **Measure:** Change from baseline in pupillary response **Time Frame:** 1 year **Description:** Flashes of light of varying luminance are presented to the eye and the subject reports is the flash was seen. **Measure:** Change from baseline in dark adapted visual sensitivity using Full field light sensitivity threshold (FST) **Time Frame:** 1 year **Measure:** Change from baseline in macula thickness **Time Frame:** 1 year **Description:** The Lea symbols chart will be used for subjects under age 6 and the Pelli-Robson chart for all other subjects. The images or letters on the charts are in decreasing contrast. **Measure:** Change from baseline in contrast sensitivity **Time Frame:** 1 year **Description:** Visual field test measuring the amount of light perceived in specific parts of the macula. **Measure:** Change from baseline in macular sensitivity as measured by microperimetry **Time Frame:** 1 year **Description:** The Farnsworth D15 tests for congenital and acquired color vision defects. Fifteen color discs will be arranged by the subject. Scoring is accomplished by recording the sequence selected by the patient on a copy of the score sheet. A patient with a color vision deficiency will arrange the color discs in a different order than a person with normal color vision. **Measure:** Change from baseline in color vision score using the Farnsworth 15 score **Time Frame:** 1 year **Measure:** Change from baseline in QOL score for Age <8 years using the Children's Visual Function Questionnaire **Time Frame:** 1 year **Measure:** Change from baseline in QOL score for Age 8 to <18 years using the Impact of Vision Impairment for Children **Time Frame:** 1 year **Measure:** Change from baseline in QOL score for Age >18 years if BCVA is worse than 1.0 logMAR in both eyes using the Impact of Vision Impairment for Very Low Vision **Time Frame:** 1 year **Measure:** Change from baseline in QOL score for Age >18 years if BCVA is 1.0 logMAR or better in both eyes using the Impact of Vision Impairment **Time Frame:** 1 year **Description:** Kinetic perimetry looks as the visual field to identify regions of normal and abnormal sensitivity to light **Measure:** Change from baseline in visual field using kinetic perimetry **Time Frame:** 1 year **Description:** This QOL has 5 non-numeric choices for the subject to select how they believe their condition has changed. **Measure:** Change from baseline in Patient Global Impressions of Change score **Time Frame:** 1 year **Description:** Video clips of the eyes are used to measure eye position and stability over time. **Measure:** Change from baseline in gaze tracking **Time Frame:** 1 year ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Male or female * At least 3 years of age at screening with CEP290-related retinal degeneration caused by a homozygous or compound heterozygous mutation involving c.2991+1655A\>G in IVS26 of the CEP290 gene. * Visual Acuity: * Sentinel participant will have severe vision loss with a logMAR BCVA of ≥1.6 to 3.9 (20/800 or worse to LP) in the study eye * Non-sentinel participants must have BCVA between 1.0 - 3.0 logMAR in the study eye Exclusion Criteria: * Other known disease-causing mutations * Achieves a passing score for the mobility course at the most difficult level * In either eye, active systemic or ocular/intraocular infection or inflammation * In either eye, history of steroid-responsive intraocular pressure with increases \> 25 mm Hg following corticosteroid exposure * Any vaccination/immunization in the last 28 days before screening * Inability or unwillingness to take oral prednisone * Prior gene therapy or oligonucleotide treatment **Minimum Age:** 3 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Miami **Country:** United States **Facility:** Bascom Palmer Eye Institute **State:** Florida **Zip:** 33136 **Location 2:** **City:** Boston **Country:** United States **Facility:** Massachusetts Eye and Ear Infirmary **State:** Massachusetts **Zip:** 02114 **Location 3:** **City:** Ann Arbor **Country:** United States **Facility:** W.K. Kellogg Eye Center - University of Michigan **State:** Michigan **Zip:** 48105 **Location 4:** **City:** Portland **Country:** United States **Facility:** Casey Eye Institute - OSHU **State:** Oregon **Zip:** 97239 **Location 5:** **City:** Philadelphia **Country:** United States **Facility:** University of Pennsylvania **State:** Pennsylvania **Zip:** 19104 ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Harvey JP, Sladen PE, Yu-Wai-Man P, Cheetham ME. Induced Pluripotent Stem Cells for Inherited Optic Neuropathies-Disease Modeling and Therapeutic Development. J Neuroophthalmol. 2022 Mar 1;42(1):35-44. doi: 10.1097/WNO.0000000000001375. Epub 2021 Sep 30. **PMID:** 34629400 **Citation:** Zhang X, Zhang D, Thompson JA, Chen SC, Huang Z, Jennings L, McLaren TL, Lamey TM, De Roach JN, Chen FK, McLenachan S. Gene correction of the CLN3 c.175G>A variant in patient-derived induced pluripotent stem cells prevents pathological changes in retinal organoids. Mol Genet Genomic Med. 2021 Mar;9(3):e1601. doi: 10.1002/mgg3.1601. Epub 2021 Jan 26. **PMID:** 33497524 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012678 - Term: Sensation Disorders - ID: D000009461 - Term: Neurologic Manifestations - ID: D000009422 - Term: Nervous System Diseases - ID: D000000013 - Term: Congenital Abnormalities ### Condition Browse Module - Browse Branches - Abbrev: BC11 - Name: Eye Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M8271 - Name: Eye Diseases - Relevance: HIGH - As Found: Eye Disease - ID: M12 - Name: Congenital Abnormalities - Relevance: LOW - As Found: Unknown - ID: M14999 - Name: Retinal Diseases - Relevance: HIGH - As Found: Retinal Disease - ID: M5047 - Name: Blindness - Relevance: HIGH - As Found: Amaurosis - ID: M8267 - Name: Eye Abnormalities - Relevance: HIGH - As Found: Eye Disorders Congenital - ID: M17530 - Name: Vision Disorders - Relevance: HIGH - As Found: Vision Disorders - ID: M23686 - Name: Genetic Diseases, Inborn - Relevance: HIGH - As Found: Diseases, Hereditary - ID: M14997 - Name: Retinal Degeneration - Relevance: HIGH - As Found: Retinal Degeneration - ID: M29107 - Name: Retinal Dystrophies - Relevance: HIGH - As Found: Retinal Dystrophy - ID: M18339 - Name: Eye Diseases, Hereditary - Relevance: HIGH - As Found: Eye Diseases, Hereditary - ID: M28730 - Name: Leber Congenital Amaurosis - Relevance: HIGH - As Found: Leber Congenital Amaurosis - ID: M15490 - Name: Sensation Disorders - Relevance: LOW - As Found: Unknown - ID: M12404 - Name: Neurologic Manifestations - Relevance: LOW - As Found: Unknown - ID: T3335 - Name: Leber Congenital Amaurosis - Relevance: HIGH - As Found: Leber Congenital Amaurosis - ID: T3337 - Name: Leber Congenital Amaurosis 10 - Relevance: HIGH - As Found: Leber Congenital Amaurosis 10 ### Condition Browse Module - Meshes - ID: D000001766 - Term: Blindness - ID: D000014786 - Term: Vision Disorders - ID: D000005128 - Term: Eye Diseases - ID: D000012164 - Term: Retinal Diseases - ID: D000012162 - Term: Retinal Degeneration - ID: D000058499 - Term: Retinal Dystrophies - ID: D000057130 - Term: Leber Congenital Amaurosis - ID: D000015785 - Term: Eye Diseases, Hereditary - ID: D000005124 - Term: Eye Abnormalities - ID: D000030342 - Term: Genetic Diseases, Inborn ### Intervention Browse Module - Browse Branches - Abbrev: AA - Name: Amino Acids - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: T4 - Name: Cysteine - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05168579 **Brief Title:** Developing a Deliberate Practice Intervention to Recalibrate Physician Heuristics in Trauma Triage **Official Title:** Developing a Deliberate Practice Intervention to Recalibrate Physician Heuristics in Trauma Triage #### Organization Study ID Info **ID:** STUDY20120026 #### Organization **Class:** OTHER **Full Name:** University of Pittsburgh #### Secondary ID Infos **ID:** R21AG072072 **Link:** https://reporter.nih.gov/quickSearch/R21AG072072 **Type:** NIH ### Status Module #### Completion Date **Date:** 2022-12-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-04-05 **Type:** ACTUAL **Last Update Submit Date:** 2023-03-13 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-03-11 **Type:** ACTUAL #### Results First Post Date **Date:** 2023-04-05 **Type:** ACTUAL **Results First Submit Date:** 2023-01-19 **Results First Submit QC Date:** 2023-03-13 #### Start Date **Date:** 2021-12-15 **Type:** ACTUAL **Status Verified Date:** 2023-03 #### Study First Post Date **Date:** 2021-12-23 **Type:** ACTUAL **Study First Submit Date:** 2021-12-08 **Study First Submit QC Date:** 2021-12-09 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Institute on Aging (NIA) #### Lead Sponsor **Class:** OTHER **Name:** University of Pittsburgh #### Responsible Party **Investigator Affiliation:** University of Pittsburgh **Investigator Full Name:** Deepika Mohan **Investigator Title:** Associate Professor of Surgery **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The objective of this study is to test the feasibility of using deliberate practice - goal-oriented training in the presence of a coach who can provide personalized, immediate feedback - to increase engagement. The research design involves recruitment of a national convenience sample of board-certified emergency physicians who will serve as trainees (n=30), pairing of the trainees with a coach, delivery of three 30-minute coaching sessions using the existing games as the training task, and assessment of the effect of the combined intervention on performance in the laboratory. The specific aims are: 1. To assess the fidelity of intervention delivery by measuring coaching skill acquisition, coaching skill drift and protocol adherence. 2. To assess the potential effect size of the intervention by comparing trainee performance on a validated virtual simulation with a control group of physicians (n=30). 3. To assess the acceptability of the intervention by using a mixture of validated instruments and semi-structured debriefing interviews with trainees to assess their engagement with the intervention. **Detailed Description:** Deliberate practice - goal-oriented training in the presence of a coach who can provide personalized, immediate feedback - has successfully improved performance across multiple domains, including sports, music, and combat. When used in conjunction with simulation to improve surgical skill, it has a large effect on educational outcomes. It has characteristics that make its application in this context potentially powerful (e.g. personalized feedback/relationship with coach increase engagement) but also potentially challenging (e.g. the diagnostic process does not lend itself easily to assessment). The objective of this study is to test the feasibility of using deliberate practice to amplify the effect of a video game intervention. The team will recruit a national sample of board-certified emergency physicians (n=30) to serve as trainees, with members of the team (n=3) serving as coaches. Trainee-coach dyads will meet for 30 minutes/week for 3 weeks, by video-conferencing, to play one of the existing video games and to use it to practice pattern recognition. Aims are: 1. To assess the fidelity of intervention delivery. Approach: the team will standardize coaching skill during an 'on-boarding session,' measure skill drift over the course of training sessions, and measure protocol adherence (primary outcome). Hypothesis: \>90% of dyads will complete three training sessions. 2. To assess the potential effect size of the intervention. Approach: the team will compare performance of trainees (n=30) with a control group of physicians (n=30) on a validated virtual simulation. Hypothesis: Trainees will make ≥25% fewer diagnostic errors than control physicians (large effect size). 3. To assess the acceptability of the intervention. Approach: the team will conduct semi-structured debriefing interviews with trainees, assessing elements of the intervention that promote engagement. This proposal will inform a future Stage III trial to compare the effect of different interventions on diagnostic error in trauma triage. If successful, this program of research will have an impact on patients by reducing the burden imposed by injury and by addressing the refractory problem of diagnostic error. It is novel conceptually in its effort to make heuristics a source of power, methodologically in its use of deliberate practice to improve diagnosis, and translationally in its use of video game technology. It is feasible because the investigative team has clinical and behavioral science expertise, experience developing deliberate practice interventions, and a track record of successfully building video games that can transform physician behavior. It responds to two national research priorities: 1) improving the diagnostic process; 2) maintaining health and independent living among the aging. ### Conditions Module **Conditions:** - Trauma Injury ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** two arm randomized trial ##### Masking Info **Masking:** SINGLE **Masking Description:** Allocation to treatment v. control group will be masked to the person doing the analysis until the data is locked. **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 72 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Trainees will be paired with a coach, and will meet, once per week for thirty minutes, over a three week period. During the training session, coach-trainee dyads will play a puzzle video game, and will discuss contextual cues that should inform triage decisions. At the completion of the three weeks, trainees will complete a semi-structured, debriefing interview and a virtual simulation to assess triage performance. **Intervention Names:** - Behavioral: Deliberate practice **Label:** Deliberate practice **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants in the control group will complete a virtual simulation. **Label:** Control **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Deliberate practice **Description:** As above **Name:** Deliberate practice **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** A summary of the number of trainees who signed up for coaching sessions and completed all three sessions as assigned. **Measure:** Percentage of Participants Who Signed up for the Intervention That Completed All 3 Coaching Sessions. **Time Frame:** 3 week intervention period #### Secondary Outcomes **Description:** Proportion of severely injured patients NOT transferred to trauma centers/total number of severely injured patients evaluated during a validated virtual simulation **Measure:** Under-triage **Time Frame:** 3 weeks post-intervention (deliberate practice) or 3 weeks post-enrollment (control) **Description:** Derived from semi-structured interviews conducted by members of the investigative team at the conclusion of the 3 week training period. These interviews will focus on participant perceptions of the acceptability of the intervention. **Measure:** Number of Participants Who Describe Coaching as Acceptable. **Time Frame:** 2- weeks post intervention ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Board certified physicians who work at non-trauma centers in the United States Exclusion Criteria: Physicians without board certification (i.e., residents). Physicians who work only at trauma centers in the US. **Healthy Volunteers:** True **Maximum Age:** 70 Years **Minimum Age:** 25 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Pittsburgh **Country:** United States **Facility:** University of Pittsburgh **State:** Pennsylvania **Zip:** 15213 #### Overall Officials **Official 1:** **Affiliation:** University of Pittsburgh **Name:** Deepika Mohan, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Access Criteria:** Access will be provided to researchers and interested parties. **Description:** De-identified participant data will be shared with researchers conditional on approval from the University of Pittsburgh Office of Research **Info Types:** - STUDY_PROTOCOL - SAP - ICF - CSR **IPD Sharing:** YES **Time Frame:** Data will be made available at the conclusion of the analytic phase of the trial, and for 3 years thereafter. ## Document Section ### Large Document Module #### Large Docs - Date: 2021-01-06 - Filename: Prot_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: False - Label: Study Protocol - Size: 224872 - Type Abbrev: Prot - Upload Date: 2023-01-12T17:19 - Date: 2022-11-25 - Filename: SAP_001.pdf - Has ICF: False - Has Protocol: False - Has SAP: True - Label: Statistical Analysis Plan - Size: 104759 - Type Abbrev: SAP - Upload Date: 2023-01-12T17:22 - Date: 2021-01-06 - Filename: ICF_002.pdf - Has ICF: True - Has Protocol: False - Has SAP: False - Label: Informed Consent Form - Size: 101037 - Type Abbrev: ICF - Upload Date: 2023-01-17T16:25 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M17685 - Name: Wounds and Injuries - Relevance: HIGH - As Found: Trauma ### Condition Browse Module - Meshes - ID: D000014947 - Term: Wounds and Injuries ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Deliberate Practice **Deaths Num At Risk:** 36 **Description:** Trainees will be paired with a coach, and will meet, once per week for thirty minutes, over a three week period. During the training session, coach-trainee dyads will play a puzzle video game, and will discuss contextual cues that should inform triage decisions. At the completion of the three weeks, trainees will complete a semi-structured, debriefing interview and a virtual simulation to assess triage performance. Deliberate practice: As above **ID:** EG000 **Other Num at Risk:** 36 **Serious Number At Risk:** 36 **Title:** Deliberate Practice **Group ID:** EG001 **Title:** Control **Deaths Num At Risk:** 36 **Description:** Participants in the control group will complete a virtual simulation. **ID:** EG001 **Other Num at Risk:** 36 **Serious Number At Risk:** 36 **Title:** Control **Frequency Threshold:** 0 **Time Frame:** 3 months ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 36 **Group ID:** BG001 **Value:** 36 **Group ID:** BG002 **Value:** 72 **Units:** Participants ### Group **ID:** BG000 **Title:** Deliberate Practice **Description:** Trainees will be paired with a coach, and will meet, once per week for thirty minutes, over a three week period. During the training session, coach-trainee dyads will play a puzzle video game, and will discuss contextual cues that should inform triage decisions. At the completion of the three weeks, trainees will complete a semi-structured, debriefing interview and a virtual simulation to assess triage performance. Deliberate practice: As above ### Group **ID:** BG001 **Title:** Control **Description:** Participants in the control group will complete a virtual simulation. ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 35 #### Measurement **Group ID:** BG001 **Value:** 35 #### Measurement **Group ID:** BG002 **Value:** 70 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 2 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 13 #### Measurement **Group ID:** BG001 **Value:** 15 #### Measurement **Group ID:** BG002 **Value:** 28 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 23 #### Measurement **Group ID:** BG001 **Value:** 21 #### Measurement **Group ID:** BG002 **Value:** 44 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 4 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 35 #### Measurement **Group ID:** BG001 **Value:** 33 #### Measurement **Group ID:** BG002 **Value:** 68 **Category Title:** Not Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 1 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 17 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 3 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 26 #### Measurement **Group ID:** BG001 **Value:** 25 #### Measurement **Group ID:** BG002 **Value:** 51 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 9 #### Measurement **Group ID:** BG002 **Value:** 19 **Category Title:** Northeast #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 9 #### Measurement **Group ID:** BG002 **Value:** 19 **Category Title:** Southeast #### Measurement **Group ID:** BG000 **Value:** 4 #### Measurement **Group ID:** BG001 **Value:** 5 #### Measurement **Group ID:** BG002 **Value:** 9 **Category Title:** Midwest #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 5 #### Measurement **Group ID:** BG002 **Value:** 8 **Category Title:** Southwest #### Measurement **Group ID:** BG000 **Value:** 9 #### Measurement **Group ID:** BG001 **Value:** 8 #### Measurement **Group ID:** BG002 **Value:** 17 **Category Title:** West **Class Title:** United States ### Measure #### Measurement **Group ID:** BG000 **Value:** 31 #### Measurement **Group ID:** BG001 **Value:** 33 #### Measurement **Group ID:** BG002 **Value:** 64 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 29 #### Measurement **Group ID:** BG001 **Value:** 30 #### Measurement **Group ID:** BG002 **Value:** 59 **Class Title:** Emergency Medicine #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 4 **Class Title:** Family Practice #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 2 #### Measurement **Group ID:** BG002 **Value:** 3 **Class Title:** Internal Medicine #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 6 **Class Title:** Other (e.g. general surgery) **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Age, Categorical **Unit of Measure:** Participants ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Ethnicity (NIH/OMB) **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race (NIH/OMB) **Unit of Measure:** Participants ### Measure 5 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Region of Enrollment **Unit of Measure:** Participants ### Measure 6 **Description:** Completion of Advanced Trauma Life Support (ATLS) course, which instructs learners in the American College of Surgeons guidelines for the triage of trauma patients. **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** ATLS Certification **Unit of Measure:** Participants ### Measure 7 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Specialty **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement **PI Sponsor Employee:** True ### Limitations and Caveats **Description:** First, we used a passive rather than an active control as the comparator, which may have magnified the effect of the intervention. Second, the differential rate of attrition for completing the virtual simulation in the control and intervention groups could introduce bias, if missingness was not random. Third, we used a simulation to assess behavior, albeit one with evidence of criterion validity, which limited inference about the efficacy of the intervention. ### Point of Contact **Email:** dem73@pitt.edu **Organization:** University of Pittsburgh **Phone:** 404-375-9325 **Title:** Dr. Deepika Mohan ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 #### Analysis **CI Lower Limit:** 2.76 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 69.6 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** Odds Ratio (OR) **Parameter Value:** 13.8 **Statistical Comment:** **Statistical Method:** Mixed Models Analysis **Tested Non-Inferiority:** ### Outcome Measure 3 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 28 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** Completed 3 coaching sessions ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** Completed 2 coaching sessions ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** Completed 1 coaching session ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 6 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 36 **Title:** Not assigned to coaching sessions #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.36 - **Upper Limit:** - **Value:** 0.85 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.50 - **Upper Limit:** - **Value:** 0.51 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 26 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** Participants received the intervention and who described it acceptable ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** Participants who received the intervention and did not describe it as acceptable. ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** Participants who received the intervention but did not complete an interview ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 6 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 36 **Title:** Participants who did not receive the intervention ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** A summary of the number of trainees who signed up for coaching sessions and completed all three sessions as assigned. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Board-certified emergency physicians who triage and treat adult patients in the emergency department of either a non-trauma center or a Level III/IV trauma center in the United States. **Reporting Status:** POSTED **Time Frame:** 3 week intervention period **Title:** Percentage of Participants Who Signed up for the Intervention That Completed All 3 Coaching Sessions. **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Trainees will be paired with a coach, and will meet, once per week for thirty minutes, over a three week period. During the training session, coach-trainee dyads will play a puzzle video game, and will discuss contextual cues that should inform triage decisions. At the completion of the three weeks, trainees will complete a semi-structured, debriefing interview and a virtual simulation to assess triage performance. Deliberate practice: As above **ID:** OG000 **Title:** Deliberate Practice ##### Group **Description:** Participants in the control group who enrolled in the trial. **ID:** OG001 **Title:** Control #### Outcome Measure 2 **Description:** Proportion of severely injured patients NOT transferred to trauma centers/total number of severely injured patients evaluated during a validated virtual simulation **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Physicians who used the virtual simulation. We excluded physicians enrolled in the trial but who did not provide outcome data for analysis. **Reporting Status:** POSTED **Time Frame:** 3 weeks post-intervention (deliberate practice) or 3 weeks post-enrollment (control) **Title:** Under-triage **Type:** SECONDARY **Unit of Measure:** proportion of patients under-triaged ##### Group **Description:** Participants in the control group who completed a virtual simulation. **ID:** OG000 **Title:** Control ##### Group **Description:** Participants in the intervention arm who completed a virtual simulation **ID:** OG001 **Title:** Deliberate Practice #### Outcome Measure 3 **Description:** Derived from semi-structured interviews conducted by members of the investigative team at the conclusion of the 3 week training period. These interviews will focus on participant perceptions of the acceptability of the intervention. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Participants assigned to receive the deliberate practice intervention who completed an interview after the training sessions. **Reporting Status:** POSTED **Time Frame:** 2- weeks post intervention **Title:** Number of Participants Who Describe Coaching as Acceptable. **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Trainees will be paired with a coach, and will meet, once per week for thirty minutes, over a three week period. During the training session, coach-trainee dyads will play a puzzle video game, and will discuss contextual cues that should inform triage decisions. At the completion of the three weeks, trainees will complete a semi-structured, debriefing interview and a virtual simulation to assess triage performance. Deliberate practice: As above **ID:** OG000 **Title:** Deliberate Practice ##### Group **Description:** Patients who enrolled in the trial and were randomized to not receive the intervention **ID:** OG001 **Title:** Control ### Participant Flow Module #### Group **Description:** Trainees will be paired with a coach, and will meet, once per week for thirty minutes, over a three week period. During the training session, coach-trainee dyads will play a puzzle video game, and will discuss contextual cues that should inform triage decisions. At the completion of the three weeks, trainees will complete a semi-structured, debriefing interview and a virtual simulation to assess triage performance. Deliberate practice: As above **ID:** FG000 **Title:** Deliberate Practice #### Group **Description:** Participants in the control group will complete a virtual simulation. **ID:** FG001 **Title:** Control #### Period **Title:** Overall Study ##### Withdraw **Type:** Lack of Consent ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ##### Withdraw **Type:** Conflicts with time ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ##### Withdraw **Type:** Time slots were full ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 15 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 36 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 36 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 30 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 21 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 6 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 15 **Recruitment Details:** A single email invitation was sent to 633 physicians who had previously participated in research (with valid contact information), asking them to forward the invitation to two colleagues. 78 responses were received. Five were excluded because they lived outside the contiguous 48 US States (e.g., in Canada or Puerto Rico) and one was excluded for refusing to consent to videotaping. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT03049579 **Brief Title:** Immune System Stimulation by Probiotic Food Supplementation **Official Title:** Immune System Stimulation by Probiotic Food Supplementation: a Double-blind, Randomized, Controlled, Parallel-designed, Prospective Trial #### Organization Study ID Info **ID:** 15-SC-09-WQ-002 #### Organization **Class:** INDUSTRY **Full Name:** Hangzhou Wei Chuan Foods Co., Ltd. ### Status Module #### Completion Date **Date:** 2016-05-02 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-02-10 **Type:** ACTUAL **Last Update Submit Date:** 2017-02-09 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2016-04-10 **Type:** ACTUAL #### Start Date **Date:** 2016-01-08 **Type:** ACTUAL **Status Verified Date:** 2017-02 #### Study First Post Date **Date:** 2017-02-10 **Type:** ACTUAL **Study First Submit Date:** 2017-02-08 **Study First Submit QC Date:** 2017-02-09 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Hangzhou Wei Chuan Foods Co., Ltd. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** The purpose of this study is to investigate the effects of a probiotic supplementation on adult volunteers with having caught the common cold more than 4 times in the past year. This study is a single center, double-blind, randomized, controlled, parallel-designed, prospective trial. Subjects received a probiotic drink containing probiotics of Lactobacillus paracasei (108 colony forming units (CFU)/ml), Lactobacilluscasei431® (108CFU/ml) and Lactobacillus fermentiumPCC® (106CFU/ml) or an identical placebo without probiotics for a 12-week study period. The incidence of flue and cold during the study period were compared between study groups. Blood and fecal samples were collected at baseline and at the end of the intervention. Fecal samples were collected for the secretory immunoglobulin A (sIgA) analysis. Blood sample was drawn for interferon γ (IFN-γ), interleukin 4 (IL-4), interleukin 10 (IL-10), immunoglobulin A (IgA), immunoglobulin G (IgG) and immunoglobulin M (IgM) analysis. ### Conditions Module **Conditions:** - Flu Symptom ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** TRIPLE **Who Masked:** - PARTICIPANT - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 136 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Weiquan Yogurt with probiotics contained Lactobacillus paracasei (108 colony forming units (CFU)/ml), Lactobacillus casei 431® (108 CFU/ml) and Lactobacillus fermentum PCC® (106 CFU/ml) **Intervention Names:** - Dietary Supplement: Weiquan Yogurt with probiotics **Label:** Weiquan Yogurt with probiotics **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Weiquan Yogurt devoid of probiotics, but otherwise similar to the experimental product. **Intervention Names:** - Dietary Supplement: Weiquan Yogurt without probiotics **Label:** Weiquan Yogurt without probiotics **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Weiquan Yogurt with probiotics **Description:** 150 ml daily consumption for a total of 12 weeks **Name:** Weiquan Yogurt with probiotics **Type:** DIETARY_SUPPLEMENT #### Intervention 2 **Arm Group Labels:** - Weiquan Yogurt without probiotics **Description:** 150 ml daily consumption for a total of 12 weeks **Name:** Weiquan Yogurt without probiotics **Type:** DIETARY_SUPPLEMENT ### Outcomes Module #### Primary Outcomes **Description:** Body temperature≥38.0℃, and have one or more symptoms of cough, runny nose, throat pain, headache, muscle pain, weak feeling, hard to breath, chest pain or loss of appetite. **Measure:** Incidence of flue symptoms during the study **Time Frame:** end of week 12 #### Secondary Outcomes **Description:** Body temperature\<38.0℃, and have one or more symptoms of cough, runny nose, throat pain, headache, muscle pain, weak feeling, hard to breath, chest pain or loss of appetite. **Measure:** Incidence of cold symptoms during the study **Time Frame:** end of week 12 **Description:** The total number of days of having one or more symptoms of cough, runny nose, throat pain, headache, muscle pain, weak feeling, hard to breath, chest pain or loss of appetite during the 12 week study period. **Measure:** Number of accumulated days of having cold symptoms during the study **Time Frame:** End of week 12 **Description:** Interferon gamma (unit: pg/ml) **Measure:** Serum IFN-γ concentration **Time Frame:** Baseline, end of week 12 **Description:** Interleukin 4 (unit: ng/ml) **Measure:** Serum IL-4 concentration **Time Frame:** Baseline, end of week 12 **Description:** Interleukin 10 (unit: pg/ml) **Measure:** Serum IL-10 concentration **Time Frame:** Baseline, end of week 12 **Description:** Immunoglobulin A (unit: g/L) **Measure:** Serum IgA concentration **Time Frame:** Baseline, end of week 12 **Description:** Immunoglobulin G (unit: g/L) **Measure:** Serum IgG concentration **Time Frame:** Baseline, end of week 12 **Description:** Immunoglobulin M (unit: g/L) **Measure:** Serum IgM concentration **Time Frame:** Baseline, end of week 12 **Description:** Secretory Immunoglobulin A (unit: ng/ml) **Measure:** Fecal sIgA concentration **Time Frame:** Baseline, end of week 12 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * male or female between 25 to 45 years old; * having caught the common cold or flu at least 4 to 6 times in the past calendar year; * signed the informed consent forms before entering the study; * fully understood the risks and potential benefits in participating this study. Exclusion Criteria: * were diagnosed with the decreased immunity caused by any diagnosed chronic illness; * having any gastrointestinal illness with medical treatment at the time of being enrolled; * having any diagnosed respiratory illness with similar symptoms as the common cold and flu; * currently taking any pain killer drug; * having received any vaccine for the upper respiratory infection within 6 months before enrollment; * having received any purgative drug or digestion related drug within 2 weeks before enrollment; * having taken any dairy product containing prebiotics and probiotics within 10 days before enrollment; * currently taking any preventive drug for upper respiratory infection; * having received any drug which has impact with the immune system such as antibiotics within 3 months before enrollment; * alcoholic or addicted to any drug; * pregnant or breastfeeding mothers; * having participated another clinical trial within 3 months before enrollment. **Healthy Volunteers:** True **Maximum Age:** 45 Years **Minimum Age:** 25 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Overall Officials **Official 1:** **Affiliation:** Sprim (Shanghai) Consulting Co., Ltd. **Name:** Li Zhang, MD **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M10295 - Name: Influenza, Human - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Browse Branches - Abbrev: Ot - Name: Other Dietary Supplements - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: T355 - Name: Acidophilus - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06121479 **Brief Title:** Oral Branched-chain Amino Acid Supplementation for Decompensated Cirrhotic Patients **Official Title:** Oral Branched-chain Amino Acid Supplementation for Decompensated Cirrhotic Patients: a Pilot Study #### Organization Study ID Info **ID:** SI 608/2023 #### Organization **Class:** OTHER **Full Name:** Mahidol University ### Status Module #### Completion Date **Date:** 2024-12-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-11-18 **Type:** ACTUAL **Last Update Submit Date:** 2023-11-14 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-06-30 **Type:** ESTIMATED #### Start Date **Date:** 2023-11-13 **Type:** ACTUAL **Status Verified Date:** 2023-11 #### Study First Post Date **Date:** 2023-11-08 **Type:** ACTUAL **Study First Submit Date:** 2023-11-01 **Study First Submit QC Date:** 2023-11-07 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Mahidol University #### Responsible Party **Investigator Affiliation:** Mahidol University **Investigator Full Name:** Phunchai Charatcharoenwitthaya **Investigator Title:** Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Is US Export:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The goal of this clinical trial is to compare the nutritional parameters after 12-week supplementation of branched-chain amino acids in cirrhotic patients with ascites and serum albumin less than 3 g/dL. The main questions it aims to answer are: 1. Would thigh muscle thickness change after 12-week supplementation of branched-chain amino acids in cirrhotic patients with ascites and serum albumin less than 3 g/dL? 2. Would triceps skin fold thickness, mid-arm circumferences, mid-arm muscle circumferences, skeletal muscle mass, appendicular skeletal muscle mass, skeletal muscle index and fat mass change after 12-week supplementation of branched-chain amino acids in cirrhotic patients with ascites and serum albumin less than 3 g/dL? 3. Would handgrip strength change after 12-week supplementation of branched-chain amino acids in cirrhotic patients with ascites and serum albumin less than 3 g/dL? 4. Would serum albumin change after 12-week supplementation of branched-chain amino acids in cirrhotic patients with ascites and serum albumin less than 3 g/dL? 5. Would score for cirrhotic severity such as Model for End-Stage Liver Disease-Sodium Score (MELD-Na score) and Child Turcotte Pugh Score change after 12-week supplementation of branched-chain amino acids in cirrhotic patients with ascites and serum albumin less than 3 g/dL? Participants will be asked to do following tasks: 1. Participants will be asked for basic information such as age, place of residence, and contact telephone number. 2. Participants will undergo measurements of weight, height, body mass index, skinfold thickness on the arms, circumference of the arms and legs, muscle mass, and body fat content using a body composition analyzer, both at the beginning and end of the research study. 3. Participants will perform grip strength measurements, at both the beginning and end of the research study. 4. Participants will undergo laboratory tests, including a complete blood count, liver and kidney function tests, blood clotting factors, and blood mineral levels, with a total blood volume of approximately 15 milliliters (1 tablespoon), collected twice during the study (at the beginning and end). 5. Participants will be administered supplements containing branched-chain amino acids (BCAA) twice a day for a total of 12 weeks. 6. Participants will be appointed for follow-up during the study, totaling 2 appointments at weeks 4 and 12. Side effects related to medication will be asked. 7. Participants will undergo ultrasound measurements of the right thigh to assess thigh muscle thickness, both at the beginning and end of the research study. 8. Participants will will complete questionnaires to assess your overall quality of life twice, both at the beginning and end of the research study. **Detailed Description:** The goal of this clinical trial is to compare the nutritional parameters after 12-week supplementation of branched-chain amino acids in cirrhotic patients with ascites and serum albumin less than 3 g/dL. The main questions it aims to answer are: 1. Would thigh muscle thickness change after 12-week supplementation of branched-chain amino acids in cirrhotic patients with ascites and serum albumin less than 3 g/dL? 2. Would triceps skin fold thickness, mid-arm circumferences, mid-arm muscle circumferences, skeletal muscle mass, appendicular skeletal muscle mass, skeletal muscle index and fat mass change after 12-week supplementation of branched-chain amino acids in cirrhotic patients with ascites and serum albumin less than 3 g/dL? 3. Would handgrip strength change after 12-week supplementation of branched-chain amino acids in cirrhotic patients with ascites and serum albumin less than 3 g/dL? 4. Would serum albumin change after 12-week supplementation of branched-chain amino acids in cirrhotic patients with ascites and serum albumin less than 3 g/dL? 5. Would score for cirrhotic severity such as Model for End-Stage Liver Disease-Sodium Score (MELD-Na score) and Child Turcotte Pugh Score change after 12-week supplementation of branched-chain amino acids in cirrhotic patients with ascites and serum albumin less than 3 g/dL? Participants will be asked to do following tasks: 1. Participants will be asked for basic information such as age, place of residence, and contact telephone number. 2. Participants will undergo measurements of weight, height, body mass index, skinfold thickness on the arms, circumference of the arms and legs, muscle mass, and body fat content using a body composition analyzer, both at the beginning and end of the research study. 3. Participants will perform grip strength measurements, at both the beginning and end of the research study. 4. Participants will undergo laboratory tests, including a complete blood count, liver and kidney function tests, blood clotting factors, and blood mineral levels, with a total blood volume of approximately 15 milliliters (1 tablespoon), collected twice during the study (at the beginning and end). 5. Participants will be administered supplements containing branched-chain amino acids (BCAA) twice a day for a total of 12 weeks. 6. Participants will be appointed for follow-up during the study, totaling 2 appointments at weeks 4 and 12. Side effects related to medication will be asked. 7. Participants will undergo ultrasound measurements of the right thigh to assess thigh muscle thickness, both at the beginning and end of the research study. 8. Participants will will complete questionnaires to assess your overall quality of life twice, both at the beginning and end of the research study. ### Conditions Module **Conditions:** - Decompensated Cirrhosis and Ascites **Keywords:** - Decompensated cirrhosis - Branched-chain amino acid - Nutrition - Ascites ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP **Intervention Model Description:** Oral branched-chain amino acid (BCAA) is provided to the patients with a recommended daily intake of approximately 13.68 grams per day. Each sachet contains 6.84 grams of BCAA (valine 1.82 grams, leucine 3.29 grams, isoleucine 1.72 grams), total protein 17.08 grams, carbohydrates 25.48 grams, fat 5.66 grams, providing 221.2 kcal of energy. Each sachet weighs 52 grams and should be mixed with 150 ml of water. The recommended daily intake is 2 sachets, to be consumed after breakfast and dinner. The BCAA provided to the patients comes in pre-packaged silver sachets, with the manufacturing date and expiration date indicated. ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 30 **Type:** ESTIMATED **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Oral branched-chain amino acid (BCAA) is provided to the patients with a recommended daily intake of approximately 13.68 grams per day. Each sachet contains 6.84 grams of BCAA (valine 1.82 grams, leucine 3.29 grams, isoleucine 1.72 grams), total protein 17.08 grams, carbohydrates 25.48 grams, fat 5.66 grams, providing 221.2 kcal of energy. Each sachet weighs 52 grams and should be mixed with 150 ml of water. The recommended daily intake is 2 sachets, to be consumed after breakfast and dinner. The BCAA provided to the patients comes in pre-packaged silver sachets, with the manufacturing date and expiration date indicated. **Intervention Names:** - Drug: Branched-chain amino acid **Label:** branched-chain amino acid **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - branched-chain amino acid **Description:** Oral branched-chain amino acid is provided to the patients with a recommended daily intake of approximately 13.68 grams per day. Each sachet contains 6.84 grams of BCAA (valine 1.82 grams, leucine 3.29 grams, isoleucine 1.72 grams), total protein 17.08 grams, carbohydrates 25.48 grams, fat 5.66 grams, providing 221.2 kcal of energy. Each sachet weighs 52 grams and should be mixed with 150 ml of water. The recommended daily intake is 2 sachets, to be consumed after breakfast and dinner. The BCAA provided to the patients comes in pre-packaged silver sachets, with the manufacturing date and expiration date indicated. **Name:** Branched-chain amino acid **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Thigh muscle thickness (centimeters) by ultrasound of right thigh will be performed according to these instructions 1. Measure the thickness of the right thigh muscles, which are the rectus femoris and vastus intermedius, at a position one-third between the upper edge of the patella bone to the iliac crest, with the patient lying flat. In that position, measure the thigh muscle thickness using two methods: a. Compression reading: Measure the muscle thickness by pressing the probe head until it cannot be compressed further. b. Feather weight reading: Measure the muscle thickness without applying pressure to the probe head. Take the muscle thickness measurements twice for each method, calculate their averages. Increase in thigh muscle thickness implies better nutritional status. **Measure:** thigh muscle thickness by ultrasound **Time Frame:** 12 weeks **Description:** The Average Feather Index (cm/m2) will be calculated by taking average of thigh muscle thickness (centimeter) measured by feather weight reading divided by height (m\^2). Increase in average feather index implies better nutritional status. **Measure:** Average Feather Index **Time Frame:** 12 weeks **Description:** The Average Compression Index (cm/m2) will be calculated by taking average of thigh muscle thickness (centimeter) measured by compression weight reading divided by height (m\^2). Increase in average compression index implies better nutritional status. **Measure:** Average Compression Index **Time Frame:** 12 weeks #### Secondary Outcomes **Description:** Triceps skin fold thickness (TSF, millimeter) will be measured using Lange skin fold caliper at midpoint of non dominant arm. **Measure:** triceps skin fold thickness **Time Frame:** 12 weeks **Description:** Mid-arm circumferences (MAC, centimeters) will be measured using standard measuring tape at midpoint of non dominant arm. Increase of mid-arm circumferences implies better nutritional status. **Measure:** mid-arm circumferences **Time Frame:** 12 weeks **Description:** Mid-arm muscle circumferences (MAMC, centimeters) will be obtained from calculating the following formula: MAMC (cm) = MAC - (0.314 × TSF \[mm\]). Increase of mid-arm muscle circumferences implies better nutritional status. **Measure:** mid-arm muscle circumferences **Time Frame:** 12 weeks **Description:** Skeletal muscle mass (kilograms) will be measured using bioelectrical impedance analysis. Increase of skeletal muscle mass implies better nutritional status. **Measure:** skeletal muscle mass **Time Frame:** 12 weeks **Description:** Appendicular skeletal muscle mass (kilograms) will be measured using bioelectrical impedance analysis. Increase of appendicular skeletal muscle mass implies better nutritional status. **Measure:** appendicular skeletal muscle mass **Time Frame:** 12 weeks **Description:** Skeletal muscle index (kilograms/m\^2) will be measured using bioelectrical impedance analysis. Increase of skeletal muscle index implies better nutritional status. **Measure:** skeletal muscle index **Time Frame:** 12 weeks **Description:** Fat mass (kilograms) will be measured using bioelectrical impedance analysis. **Measure:** Fat mass **Time Frame:** 12 weeks **Description:** Handgrip strength (kilograms) will be measured three times for each hand using digital handgrip strength dynamometer. Increase of handgrip strength implies better physical performance. **Measure:** Handgrip strength **Time Frame:** 12 weeks **Description:** MELD Na Score will be calculated at the start and end of study. Decrease of MELD Na Score implies improving of cirrhotic severity. **Measure:** MELD Na Score **Time Frame:** 12 weeks **Description:** Child Turcotte Pugh Score will be calculated at the start and end of study. Decrease of Child Turcotte Pugh Score implies improving of cirrhotic severity. **Measure:** Child Turcotte Pugh Score **Time Frame:** 12 weeks **Description:** Serum albumin (gram/deciliter) will be measured at the start and end of study. Increase of serum albumin implies better nutritional status. **Measure:** Serum albumin **Time Frame:** 12 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Cirrhotic patients of all etiology with aged of 18-85 years 2. Clinically detectable ascites 3. Serum albumin \< 3 g/dL Exclusion Criteria: 1. Patients with hepatocellular carcinoma outside the Milan criteria. 2. Patients with history of gastrointestinal bleeding and spontaneous bacterial peritonitis (SBP) within the last 3 months. 3. Patients with acute on chronic liver failure (ACLF). 4. Patients with uncontrollable chronic comorbidities, including chronic heart failure in NYHA stages 3-4, end stage kidney disease requiring dialysis, and chronic obstructive pulmonary disease Gold D. 5 .Patients who have undergone liver or kidney transplant. 6. Patients with Human Immunodeficiency Virus (HIV). 7. Pregnant or lactating patients. 8. Patients who have undergone large volume paracentesis (\>5 liters) with intravenous albumin administration on the day of study enrollment. 9. Patients with a history of above-knee amputation surgery. 10. Patients with a history of receiving intravenous albumin administration every 1-2 weeks within the last one month. **Maximum Age:** 85 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** WASIT.WONGTRAKUL@GMAIL.COM **Name:** WASIT WONGTRAKUL, M.D. **Phone:** +66822702898 **Role:** CONTACT #### Locations **Location 1:** **City:** Bangkok **Contacts:** ***Contact 1:*** - **Email:** wasit.wongtrakul@gmail.com - **Name:** WASIT WONGTRAKUL, M.D. - **Phone:** 66822702898 - **Role:** CONTACT ***Contact 2:*** - **Name:** Phunchai Charatcharoenwitthaya, M.D. - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Thailand **Facility:** Division of Gastroenterology, Department of Medicine, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand **Status:** RECRUITING **Zip:** 10700 #### Overall Officials **Official 1:** **Affiliation:** Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand **Name:** Phunchai Charatcharoenwitthaya, M.D. **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Description:** In order to protect patients' confidentiality, we will not share individual participant data. **IPD Sharing:** NO ### References Module #### References **Citation:** Liu YB, Chen MK. Epidemiology of liver cirrhosis and associated complications: Current knowledge and future directions. World J Gastroenterol. 2022 Nov 7;28(41):5910-5930. doi: 10.3748/wjg.v28.i41.5910. **PMID:** 36405106 **Citation:** D'Amico G, Garcia-Tsao G, Pagliaro L. Natural history and prognostic indicators of survival in cirrhosis: a systematic review of 118 studies. J Hepatol. 2006 Jan;44(1):217-31. doi: 10.1016/j.jhep.2005.10.013. Epub 2005 Nov 9. No abstract available. **PMID:** 16298014 **Citation:** European Association for the Study of the Liver. Electronic address: easloffice@easloffice.eu; European Association for the Study of the Liver. EASL Clinical Practice Guidelines on nutrition in chronic liver disease. J Hepatol. 2019 Jan;70(1):172-193. doi: 10.1016/j.jhep.2018.06.024. Epub 2018 Aug 23. **PMID:** 30144956 **Citation:** Maharshi S, Sharma BC, Srivastava S. Malnutrition in cirrhosis increases morbidity and mortality. J Gastroenterol Hepatol. 2015 Oct;30(10):1507-13. doi: 10.1111/jgh.12999. **PMID:** 25974421 **Citation:** Soeters PB, Fischer JE. Insulin, glucagon, aminoacid imbalance, and hepatic encephalopathy. Lancet. 1976 Oct 23;2(7991):880-2. doi: 10.1016/s0140-6736(76)90541-9. **PMID:** 62115 **Citation:** Tajiri K, Shimizu Y. Branched-chain amino acids in liver diseases. Transl Gastroenterol Hepatol. 2018 Jul 30;3:47. doi: 10.21037/tgh.2018.07.06. eCollection 2018. **PMID:** 30148232 **Citation:** Gluud LL, Dam G, Les I, Marchesini G, Borre M, Aagaard NK, Vilstrup H. Branched-chain amino acids for people with hepatic encephalopathy. Cochrane Database Syst Rev. 2017 May 18;5(5):CD001939. doi: 10.1002/14651858.CD001939.pub4. **PMID:** 28518283 **Citation:** Sirisunhirun P, Bandidniyamanon W, Jrerattakon Y, Muangsomboon K, Pramyothin P, Nimanong S, Tanwandee T, Charatcharoenwitthaya P, Chainuvati S, Chotiyaputta W. Effect of a 12-week home-based exercise training program on aerobic capacity, muscle mass, liver and spleen stiffness, and quality of life in cirrhotic patients: a randomized controlled clinical trial. BMC Gastroenterol. 2022 Feb 14;22(1):66. doi: 10.1186/s12876-022-02147-7. **PMID:** 35164698 **Citation:** Hernandez-Conde M, Llop E, Gomez-Pimpollo L, Fernandez Carrillo C, Rodriguez L, Van Den Brule E, Perello C, Lopez-Gomez M, Abad J, Martinez-Porras JL, Fernandez-Puga N, Ferre C, Trapero M, Fraga E, Calleja JL. Adding Branched-Chain Amino Acids to an Enhanced Standard-of-Care Treatment Improves Muscle Mass of Cirrhotic Patients With Sarcopenia: A Placebo-Controlled Trial. Am J Gastroenterol. 2021 Nov 1;116(11):2241-2249. doi: 10.14309/ajg.0000000000001301. **PMID:** 34074812 **Citation:** Siramolpiwat S, Limthanetkul N, Pornthisarn B, Vilaichone RK, Chonprasertsuk S, Bhanthumkomol P, Nunanan P, Issariyakulkarn N. Branched-chain amino acids supplementation improves liver frailty index in frail compensated cirrhotic patients: a randomized controlled trial. BMC Gastroenterol. 2023 May 15;23(1):154. doi: 10.1186/s12876-023-02789-1. **PMID:** 37189033 **Citation:** Haj Ali S, Abu Sneineh A, Hasweh R. Nutritional assessment in patients with liver cirrhosis. World J Hepatol. 2022 Sep 27;14(9):1694-1703. doi: 10.4254/wjh.v14.i9.1694. **PMID:** 36185724 **Citation:** Tandon P, Low G, Mourtzakis M, Zenith L, Myers RP, Abraldes JG, Shaheen AA, Qamar H, Mansoor N, Carbonneau M, Ismond K, Mann S, Alaboudy A, Ma M. A Model to Identify Sarcopenia in Patients With Cirrhosis. Clin Gastroenterol Hepatol. 2016 Oct;14(10):1473-1480.e3. doi: 10.1016/j.cgh.2016.04.040. Epub 2016 May 14. Erratum In: Clin Gastroenterol Hepatol. 2022 Jun;20(6):1423. **PMID:** 27189915 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M11103 - Name: Liver Cirrhosis - Relevance: LOW - As Found: Unknown - ID: M4509 - Name: Ascites - Relevance: HIGH - As Found: Ascites - ID: M8485 - Name: Fibrosis - Relevance: HIGH - As Found: Cirrhosis ### Condition Browse Module - Meshes - ID: D000005355 - Term: Fibrosis - ID: D000001201 - Term: Ascites ### Intervention Browse Module - Browse Branches - Abbrev: AA - Name: Amino Acids - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: T10 - Name: Leucine - Relevance: LOW - As Found: Unknown - ID: T23 - Name: Valine - Relevance: LOW - As Found: Unknown - ID: T9 - Name: Isoleucine - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04515979 **Brief Title:** Vactosertib in Combination With Pembrolizumab for PD-L1 Positive Non-small Cell Lung Cancer (NSCLC) Subjects **Official Title:** A Phase 2, Open-label, Multicenter Study to Assess the Efficacy and Safety of Vactosertib in Combination With Pembrolizumab as a First-line Treatment for Subjects With PD-L1 Positive Advanced Non-Small Cell Lung Cancer #### Organization Study ID Info **ID:** MP-VAC-205 #### Organization **Class:** INDUSTRY **Full Name:** MedPacto, Inc. #### Secondary ID Infos **Domain:** Merck **ID:** MK3475 B37 **Type:** OTHER ### Status Module #### Completion Date **Date:** 2025-12-30 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2021-09-24 **Type:** ACTUAL **Last Update Submit Date:** 2021-09-22 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2022-08-01 **Type:** ESTIMATED #### Start Date **Date:** 2020-12-17 **Type:** ACTUAL **Status Verified Date:** 2021-09 #### Study First Post Date **Date:** 2020-08-17 **Type:** ACTUAL **Study First Submit Date:** 2020-08-11 **Study First Submit QC Date:** 2020-08-13 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** MedPacto, Inc. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** This is Phase 2, open label, multi-center study to assess safety and efficacy of vactosertib in combination with pembrolizumab as 1st line treatment for subjects with advanced or metastatic, PD-L1 positive, non-small cell lung cancer (NSCLC) who have not previously received systemic therapy for advanced disease and in whom EGFR, ALK, BRAF, ROS1-directed therapy is not indicated. **Detailed Description:** Approximately 55 NSCLC subjects with PD-L1 tumor proportion score (TPS) ≥ 1% are expected to be enrolled in this study. Subjects' TPS will be determined by PD-L1 IHC 22C3 pharmDx assay performed according to local laboratory regulations prior to study enrollment. Subjects who received adjuvant or neoadjuvant therapy are permitted onto the study if the therapy was completed at least 12 months prior to the development of metastatic disease. Eligible subjects will receive: • Vactosertib 300 mg orally (PO) BID for 5 days with 2 days off period (5 days on/2days off) and pembrolizumab 200 mg IV on Day 1 of every 3-week cycle (Q3W). ### Conditions Module **Conditions:** - Carcinoma, Non-Small-Cell Lung ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 55 **Type:** ESTIMATED **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Vactosertib (5days on and 2days off) Pembrolizumab 200mg Q3Weeks **Intervention Names:** - Drug: Vactosertib 300 mg BID and pembrolizumab 200 mg IV **Label:** vactosertib+Pembrolizumab **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - vactosertib+Pembrolizumab **Description:** Vactosertib 300 mg orally (PO) BID(5 days on/2days off) and pembrolizumab 200 mg IV (Q3W). **Name:** Vactosertib 300 mg BID and pembrolizumab 200 mg IV **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** ORR in PD-L1≥1% and PD-L1≥50% population per RECIST 1.1 by investigators **Measure:** ORR per RECIST 1.1 **Time Frame:** From date of enrollment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 3 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Have a histologically- or cytologically-documented NSCLC advanced or stage IV lung cancer * Have confirmation that EGFR, ALK, BRAF, ROS1-directed therapy is not indicated * Have measurable disease based on RECIST 1.1 * PD-L1 expression is ≥1% as determined by the PD-L1 IHC 22C3 pharmDx assay * Have a life expectancy of at least 3 months. * ECOG 0 or 1 * Subjects must be able to swallow tablets and absorb vactosertib. * Have adequate organ function as indicated by the following laboratory values in Exclusion Criteria: * Is currently participating in a study of an investigational agent * Has received prior systemic cytotoxic chemotherapy for metastatic disease/ antineoplastic biological therapy /Had major surgery / radiation therapy to the lung * Has received a live vaccine within 30 days prior to the first dose of study drug. * Is taking prohibited medications * Has a diagnosis of immunodeficiency or is receiving chronic systemic steroid therapy * Has known active central nervous system (CNS) metastases and/or carcinomatous meningitis. * Had a severe hypersensitivity reaction to treatment with another mAb previously. * Has severe hypersensitivity to vactosertib and/or any of its excipients **Minimum Age:** 19 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** kyounghee.kim@medpacto.com **Name:** kyounghee kim **Phone:** 82-2-6938-0235 **Role:** CONTACT #### Locations **Location 1:** **City:** Seoul **Contacts:** ***Contact 1:*** - **Name:** Sㄷ-hoon Lee, Phd - **Role:** CONTACT **Country:** Korea, Republic of **Facility:** Samsung Medical Center **Status:** RECRUITING **Zip:** 06351 #### Overall Officials **Official 1:** **Affiliation:** MedPacto, Inc. **Name:** Bitna Oh, MD **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002283 - Term: Carcinoma, Bronchogenic - ID: D000001984 - Term: Bronchial Neoplasms - ID: D000008175 - Term: Lung Neoplasms - ID: D000012142 - Term: Respiratory Tract Neoplasms - ID: D000013899 - Term: Thoracic Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases ### Condition Browse Module - Browse Leaves - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - ID: M11172 - Name: Lung Neoplasms - Relevance: LOW - As Found: Unknown - ID: M5546 - Name: Carcinoma, Non-Small-Cell Lung - Relevance: HIGH - As Found: Carcinoma, Non-Small-Cell Lung - ID: M5540 - Name: Carcinoma, Bronchogenic - Relevance: LOW - As Found: Unknown - ID: M5260 - Name: Bronchial Neoplasms - Relevance: LOW - As Found: Unknown - ID: M14979 - Name: Respiratory Tract Neoplasms - Relevance: LOW - As Found: Unknown - ID: M16658 - Name: Thoracic Neoplasms - Relevance: LOW - As Found: Unknown - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002289 - Term: Carcinoma, Non-Small-Cell Lung ### Intervention Browse Module - Ancestors - ID: D000074322 - Term: Antineoplastic Agents, Immunological - ID: D000000970 - Term: Antineoplastic Agents - ID: D000082082 - Term: Immune Checkpoint Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000047428 - Term: Protein Kinase Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M349416 - Name: Pembrolizumab - Relevance: HIGH - As Found: Blind - ID: M96450 - Name: Vactosertib - Relevance: HIGH - As Found: Evidence for - ID: M1346 - Name: Antineoplastic Agents, Immunological - Relevance: LOW - As Found: Unknown - ID: M2342 - Name: Immune Checkpoint Inhibitors - Relevance: LOW - As Found: Unknown - ID: M25820 - Name: Protein Kinase Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000582435 - Term: Pembrolizumab - ID: C000590371 - Term: Vactosertib ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00885079 **Brief Title:** Confirmatory Study of OPC-12759 Ophthalmic Suspension **Official Title:** Confirmatory Study of OPC-12759 Ophthalmic Suspension in Dry Eye Patients #### Organization Study ID Info **ID:** 037E-08-001 #### Organization **Class:** INDUSTRY **Full Name:** Otsuka Pharmaceutical Co., Ltd. ### Status Module #### Completion Date **Date:** 2010-04 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2013-12-12 **Type:** ESTIMATED **Last Update Submit Date:** 2013-11-18 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2010-03 **Type:** ACTUAL #### Results First Post Date **Date:** 2013-12-12 **Type:** ESTIMATED **Results First Submit Date:** 2013-09-12 **Results First Submit QC Date:** 2013-11-18 #### Start Date **Date:** 2009-05 **Status Verified Date:** 2013-11 #### Study First Post Date **Date:** 2009-04-21 **Type:** ESTIMATED **Study First Submit Date:** 2009-04-19 **Study First Submit QC Date:** 2009-04-20 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Otsuka Pharmaceutical Co., Ltd. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to verify whether OPC-12759 ophthalmic suspension is effective compared with active control in dry eye patients. ### Conditions Module **Conditions:** - Dry Eye Syndromes ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 188 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Instillation,4 times/day for 4 weeks **Intervention Names:** - Drug: OPC-12759 Ophthalmic suspension **Label:** Rebamipide **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Instillation,6 times/day for 4 weeks **Intervention Names:** - Drug: Hyalein Mini Ophthalmic solution **Label:** Hyaluronate **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Rebamipide **Description:** OPC-12759 Ophthalmic suspension 2% **Name:** OPC-12759 Ophthalmic suspension **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Hyaluronate **Description:** Hyalein Mini Ophthalmic solution 0.1% **Name:** Hyalein Mini Ophthalmic solution **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** FCS indicates the damage to the corneal epithelium. Per the National Eye Institute/Industry Workshop report, the cornea was divided into 5 fractions, each of which was given a staining score from 0 to 3, and the total score was calculated (0-15). 0 is better. Noninferiority for change from baseline in the FCS score (LOCF) was determined by comparing the noninferiority margin (0.4) with the upper limit of the 95% confidence interval (CI) of the difference between the 2 treatment groups **Measure:** Change in Fluorescein Corneal Staining (FCS) Score From Baseline **Time Frame:** Baseline, Weeks4 **Description:** LGCS indicates the damage to the conjunctival epithelium. Per the National Eye Institute/Industry Workshop report, the conjunctiva was divided into 6 fractions, each of which was given a staining sdore from 0 to 3, and the total score was calculated (0-18). 0 is better. Superiority was verified by comparing t-test results for change from baseline in the LGCS score (LOCF) between 2 treatment groups. **Measure:** Change in Lissamine Green Conjunctival Staining (LGCS) Score From Baseline **Time Frame:** Baseline, Weeks4 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Out patient 2. Subjective complaint of dry eye that has been present for minimum 20 months 3. Ocular discomfort severity is moderate to severe 4. Corneal - conjunctival damage is moderate to severe 5. Unanesthetized Schirmer's test score of 5mm/5minutes or less 6. Best corrected visual acuity of 0.2 or better in both eyes Exclusion Criteria: 1. Presence of anterior segment disease or disorder other than that associated with keratoconjunctivitis sicca 2. Ocular hypertension patient or glaucoma patient with ophthalmic solution 3. Anticipated use of any topically-instilled ocular medications or patients who cannot discontinue the use during the study 4. Anticipated use of contact lens during the study 5. Patient with punctal plug 6. Any history of ocular surgery within 12 months 7. Female patients who are pregnant,possibly pregnant or breast feeding 8. Known hypersensitivity to any component of the study drug or procedural medications 9. Receipt of any investigational product within 4 months. **Minimum Age:** 20 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Kagoshima **Country:** Japan **Facility:** Kyushu region **Location 2:** **City:** Matsuyama **Country:** Japan **Facility:** Chushikoku region **Location 3:** **City:** Nagoya **Country:** Japan **Facility:** Tokai region **Location 4:** **City:** Osaka **Country:** Japan **Facility:** Kansai region **Location 5:** **City:** Tokyo **Country:** Japan **Facility:** Kanto region #### Overall Officials **Official 1:** **Affiliation:** OPCJ-DDO **Name:** Eiji Murakami **Role:** STUDY_CHAIR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007766 - Term: Lacrimal Apparatus Diseases - ID: D000005128 - Term: Eye Diseases - ID: D000007637 - Term: Keratoconjunctivitis - ID: D000003231 - Term: Conjunctivitis - ID: D000003229 - Term: Conjunctival Diseases - ID: D000007634 - Term: Keratitis - ID: D000003316 - Term: Corneal Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC11 - Name: Eye Diseases ### Condition Browse Module - Browse Leaves - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M18040 - Name: Dry Eye Syndromes - Relevance: HIGH - As Found: Dry Eye Syndrome - ID: M10664 - Name: Keratoconjunctivitis Sicca - Relevance: HIGH - As Found: Dry Eye - ID: M10663 - Name: Keratoconjunctivitis - Relevance: LOW - As Found: Unknown - ID: M10786 - Name: Lacrimal Apparatus Diseases - Relevance: LOW - As Found: Unknown - ID: M8271 - Name: Eye Diseases - Relevance: LOW - As Found: Unknown - ID: M6455 - Name: Conjunctivitis - Relevance: LOW - As Found: Unknown - ID: M6453 - Name: Conjunctival Diseases - Relevance: LOW - As Found: Unknown - ID: M10660 - Name: Keratitis - Relevance: LOW - As Found: Unknown - ID: M6539 - Name: Corneal Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000015352 - Term: Dry Eye Syndromes - ID: D000007638 - Term: Keratoconjunctivitis Sicca ### Intervention Browse Module - Ancestors - ID: D000019999 - Term: Pharmaceutical Solutions - ID: D000000897 - Term: Anti-Ulcer Agents - ID: D000005765 - Term: Gastrointestinal Agents - ID: D000000975 - Term: Antioxidants - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000020011 - Term: Protective Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000004791 - Term: Enzyme Inhibitors ### Intervention Browse Module - Browse Branches - Abbrev: PhSol - Name: Pharmaceutical Solutions - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Gast - Name: Gastrointestinal Agents ### Intervention Browse Module - Browse Leaves - ID: M21860 - Name: Pharmaceutical Solutions - Relevance: LOW - As Found: Unknown - ID: M12814 - Name: Ophthalmic Solutions - Relevance: HIGH - As Found: Start - ID: M246061 - Name: Rebamipide - Relevance: HIGH - As Found: Information and Communication - ID: M4188 - Name: Antacids - Relevance: LOW - As Found: Unknown - ID: M4219 - Name: Anti-Ulcer Agents - Relevance: LOW - As Found: Unknown - ID: M8881 - Name: Gastrointestinal Agents - Relevance: LOW - As Found: Unknown - ID: M4292 - Name: Antioxidants - Relevance: LOW - As Found: Unknown - ID: M21869 - Name: Protective Agents - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000052785 - Term: Rebamipide - ID: D000009883 - Term: Ophthalmic Solutions ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Rebamipide **Description:** Instillation,4 times/day for 4 weeks OPC-12759 Ophthalmic suspension : OPC-12759 Ophthalmic suspension 2% **ID:** EG000 **Other Num Affected:** 27 **Other Num at Risk:** 93 **Serious Number At Risk:** 93 **Title:** Rebamipide **Group ID:** EG001 **Title:** Hyaluronate **Description:** Instillation,6 times/day for 4 weeks Hyalein Mini Ophthalmic solution : Hyalein Mini Ophthalmic solution 0.1% **ID:** EG001 **Other Num Affected:** 19 **Other Num at Risk:** 95 **Serious Number At Risk:** 95 **Title:** Hyaluronate **Frequency Threshold:** 1.0 #### Other Events **Term:** Blepharitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Chalazion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Conjunctival haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Conjunctivitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Erythema of eyelid **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Eye discharge **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Eye irritation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Eye pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Eyelid oedema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Ocular hyperaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Vision blurred **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Visual impairment **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Conjunctival hyperaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Eye pruritus **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Ocular discomfort **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Abdominal discomfort **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Abdominal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Gastritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Chest discomfort **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Thirst **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Hepatic function abnormal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Hordeolum **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (12.1) **Term:** Nasopharyngitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (12.1) **Term:** Onychomycosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (12.1) **Term:** Sinusitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (12.1) **Term:** Foreign body in eye **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (12.1) **Term:** Aspartate aminotransferase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (12.1) **Term:** Blood bilirubin increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (12.1) **Term:** Blood potassium increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (12.1) **Term:** Blood urea increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (12.1) **Term:** Blood urine present **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (12.1) **Term:** White blood cell count decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (12.1) **Term:** Back pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Dysgeusia (bitter taste) **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Oropharyngeal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Rash **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Dry mouth **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (12.1) ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 93 **Group ID:** BG001 **Value:** 95 **Group ID:** BG002 **Value:** 188 **Units:** Participants ### Group **ID:** BG000 **Title:** Rebamipide **Description:** Instillation,4 times/day for 4 weeks OPC-12759 Ophthalmic suspension : OPC-12759 Ophthalmic suspension 2% ### Group **ID:** BG001 **Title:** Hyaluronate **Description:** Instillation,6 times/day for 4 weeks Hyalein Mini Ophthalmic solution : Hyalein Mini Ophthalmic solution 0.1% ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 55 #### Measurement **Group ID:** BG001 **Value:** 59 #### Measurement **Group ID:** BG002 **Value:** 114 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 38 #### Measurement **Group ID:** BG001 **Value:** 36 #### Measurement **Group ID:** BG002 **Value:** 74 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 17.7 **Value:** 58.0 #### Measurement **Group ID:** BG001 **Spread:** 16.9 **Value:** 55.2 #### Measurement **Group ID:** BG002 **Spread:** 17.4 **Value:** 56.6 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 83 #### Measurement **Group ID:** BG001 **Value:** 80 #### Measurement **Group ID:** BG002 **Value:** 163 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 15 #### Measurement **Group ID:** BG002 **Value:** 25 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 93 #### Measurement **Group ID:** BG001 **Value:** 95 #### Measurement **Group ID:** BG002 **Value:** 188 **Class Title:** Japan **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Age, Categorical **Unit of Measure:** Participants ### Measure 2 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age Continuous **Unit of Measure:** years ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants ## Results Section - More Information Module ### Certain Agreement ### Point of Contact **Organization:** Otsuka Pharmaceutical Co., Ltd. **Phone:** -81-6-6943-7722 **Title:** Mr. Kyoji Imaoka ## Results Section - Outcome Measures Module ### Outcome Measure 1 #### Analysis **CI Lower Limit:** -1.47 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** -0.24 **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_DEVIATION **Dispersion Value:** 2.1 **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** Non-inferiority for change from baseline in the FCS score was determined by comparing the non-inferiority margin (0.4) with the upper limit of the confidence interval of the difference between the 2 treatment groups. **Non-Inferiority Type:** NON_INFERIORITY_OR_EQUIVALENCE **Other Analysis Description:** **P-Value:** <0.05 **P-Value Comment:** An analysis of change from baseline of FCS was performed using t-test. The level of singnificanse was 5 % (2-sided). **Parameter Type:** Mean Difference (Final Values) **Parameter Value:** -0.9 **Statistical Comment:** **Statistical Method:** t-test, 2 sided **Tested Non-Inferiority:** True ### Outcome Measure 2 #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** <0.05 **P-Value Comment:** **Parameter Type:** Mean Difference (Final Values) **Parameter Value:** -2.1 **Statistical Comment:** **Statistical Method:** t-test, 2 sided **Tested Non-Inferiority:** False ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.3 - **Upper Limit:** - **Value:** -3.7 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.2 - **Upper Limit:** - **Value:** -2.9 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.2 - **Upper Limit:** - **Value:** -4.5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 2.5 - **Upper Limit:** - **Value:** -2.4 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** FCS indicates the damage to the corneal epithelium. Per the National Eye Institute/Industry Workshop report, the cornea was divided into 5 fractions, each of which was given a staining score from 0 to 3, and the total score was calculated (0-15). 0 is better. Noninferiority for change from baseline in the FCS score (LOCF) was determined by comparing the noninferiority margin (0.4) with the upper limit of the 95% confidence interval (CI) of the difference between the 2 treatment groups **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** Baseline, Weeks4 **Title:** Change in Fluorescein Corneal Staining (FCS) Score From Baseline **Type:** PRIMARY **Unit of Measure:** units on a scale ##### Group **Description:** Instillation,4 times/day for 4 weeks OPC-12759 Ophthalmic suspension : OPC-12759 Ophthalmic suspension 2% **ID:** OG000 **Title:** Rebamipide ##### Group **Description:** Instillation,6 times/day for 4 weeks Hyalein Mini Ophthalmic solution : Hyalein Mini Ophthalmic solution 0.1% **ID:** OG001 **Title:** Hyaluronate #### Outcome Measure 2 **Description:** LGCS indicates the damage to the conjunctival epithelium. Per the National Eye Institute/Industry Workshop report, the conjunctiva was divided into 6 fractions, each of which was given a staining sdore from 0 to 3, and the total score was calculated (0-18). 0 is better. Superiority was verified by comparing t-test results for change from baseline in the LGCS score (LOCF) between 2 treatment groups. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** Baseline, Weeks4 **Title:** Change in Lissamine Green Conjunctival Staining (LGCS) Score From Baseline **Type:** PRIMARY **Unit of Measure:** units on a scale ##### Group **Description:** Instillation,4 times/day for 4 weeks OPC-12759 Ophthalmic suspension : OPC-12759 Ophthalmic suspension 2% **ID:** OG000 **Title:** Rebamipide ##### Group **Description:** Instillation,6 times/day for 4 weeks Hyalein Mini Ophthalmic solution : Hyalein Mini Ophthalmic solution 0.1% **ID:** OG001 **Title:** Hyaluronate ### Participant Flow Module #### Group **Description:** Instillation,4 times/day for 4 weeks OPC-12759 Ophthalmic suspension : OPC-12759 Ophthalmic suspension 2% **ID:** FG000 **Title:** Rebamipide #### Group **Description:** Instillation,6 times/day for 4 weeks Hyalein Mini Ophthalmic solution : Hyalein Mini Ophthalmic solution 0.1% **ID:** FG001 **Title:** Hyaluronate #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 93 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 95 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 91 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 91 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 2 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 4 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT00857779 **Brief Title:** Study on Pharmacodynamic Parameter and Tolerability With Subcutaneous Immunotherapy in Grass Pollen Allergic Patients **Official Title:** A Multicentre Randomised Phase II Clinical Trial to Demonstrate Equivalent Pharmacodynamic Efficacy and Tolerability of Two Updosing Schedules for ALK-Flex SQ #### Organization Study ID Info **ID:** AF-H-01 #### Organization **Class:** INDUSTRY **Full Name:** ALK-Abelló A/S ### Status Module #### Completion Date **Date:** 2009-10 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2013-02-08 **Type:** ESTIMATED **Last Update Submit Date:** 2013-02-07 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2009-08 **Type:** ACTUAL #### Start Date **Date:** 2009-02 **Status Verified Date:** 2013-02 #### Study First Post Date **Date:** 2009-03-09 **Type:** ESTIMATED **Study First Submit Date:** 2009-03-05 **Study First Submit QC Date:** 2009-03-05 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** ALK-Abelló A/S #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The aim of this study is to test the pharmacodynamic equivalence with respect to IgE-blocking factor and to compare the tolerability of two different updosing schedules with ALK-Flex SQ ### Conditions Module **Conditions:** - Rhinitis - Conjunctivitis - Asthma **Keywords:** - allergen immunotherapy - allergy to grass pollen ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 473 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** subcutaneous immunotherapy using a slow updosing schedule **Intervention Names:** - Biological: subcutaneous immunotherapy **Label:** subcutaneous immunotherapy **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** subcutaneous immunotherapy using a fast updosing schedule **Intervention Names:** - Biological: subcutaneous immunotherapy **Label:** subcutaneous injections **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - subcutaneous immunotherapy - subcutaneous injections **Description:** 7 injections **Name:** subcutaneous immunotherapy **Other Names:** - AVANZ **Type:** BIOLOGICAL ### Outcomes Module #### Primary Outcomes **Measure:** IgE-blocking factor **Time Frame:** start of treatment and 1 week after end of treatment #### Secondary Outcomes **Measure:** Tolerability of two different updosing schedules **Time Frame:** throughout treatment ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * A clinical history of grass pollen induced allergic rhinoconjunctivitis of two years or more requiring treatment during the grass pollen season * Lack of adequate relief with symptomatic medication during the previous grass pollen season * Positive Skin Prick Test (SPT) response to Phleum pratense (wheal diameter \>= 3mm) currently performed or not older than 60 days before screening Exclusion Criteria: * FEV1 \< 70% of predicted value at screening * Bronchial asthma corresponding to GINA step 3 or more, even if controlled * History of asthma exacerbation or emergency visit or admission for asthma in the previous 12 months * Previous treatment by immunotherapy with grass pollen allergen or any other allergen within the previous 5 years * Exclusion criteria in accordance with contraindications in the SPC of ALK-Flex SQ **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Erfurt **Country:** Germany **Facility:** Allergists' practice Dr. Kirsten Jung **State:** Thueringen **Zip:** D-99084 #### Overall Officials **Official 1:** **Affiliation:** Private practice, Erfurt, Germany **Name:** Kirsten Jung, MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Pfaar O, Jung K, Wolf H, Decot E, Kleine-Tebbe J, Klimek L, Wustenberg E. Immunological effects and tolerability of a new fast updosed immunologically enhanced subcutaneous immunotherapy formulation with optimized allergen/adjuvant ratio. Allergy. 2012 May;67(5):630-7. doi: 10.1111/j.1398-9995.2012.02801.x. Epub 2012 Mar 3. **PMID:** 22385366 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012141 - Term: Respiratory Tract Infections - ID: D000007239 - Term: Infections - ID: D000009668 - Term: Nose Diseases - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000010038 - Term: Otorhinolaryngologic Diseases - ID: D000003229 - Term: Conjunctival Diseases - ID: D000005128 - Term: Eye Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC01 - Name: Infections - Abbrev: BC09 - Name: Ear, Nose, and Throat Diseases - Abbrev: BC11 - Name: Eye Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M10018 - Name: Hypersensitivity - Relevance: LOW - As Found: Unknown - ID: M4556 - Name: Asthma - Relevance: LOW - As Found: Unknown - ID: M15049 - Name: Rhinitis - Relevance: HIGH - As Found: Rhinitis - ID: M6455 - Name: Conjunctivitis - Relevance: HIGH - As Found: Conjunctivitis - ID: M10283 - Name: Infections - Relevance: LOW - As Found: Unknown - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: M14978 - Name: Respiratory Tract Infections - Relevance: LOW - As Found: Unknown - ID: M12604 - Name: Nose Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M12961 - Name: Otorhinolaryngologic Diseases - Relevance: LOW - As Found: Unknown - ID: M6453 - Name: Conjunctival Diseases - Relevance: LOW - As Found: Unknown - ID: M8271 - Name: Eye Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000012220 - Term: Rhinitis - ID: D000003231 - Term: Conjunctivitis ### Intervention Browse Module - Ancestors - ID: D000007155 - Term: Immunologic Factors - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M2853 - Name: Immunomodulating Agents - Relevance: HIGH - As Found: Computer - ID: M10201 - Name: Immunologic Factors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000091369 - Term: Immunomodulating Agents ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01522079 **Brief Title:** Spinal Muscular Atrophy and Cardiac Autonomic Function **Official Title:** Spinal Muscular Atrophy and Cardiac Autonomic Function #### Organization Study ID Info **ID:** Dias #### Organization **Class:** OTHER **Full Name:** Centro Universitário Augusto Motta ### Status Module #### Completion Date **Date:** 2011-10 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2012-01-31 **Type:** ESTIMATED **Last Update Submit Date:** 2012-01-26 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2011-08 **Type:** ACTUAL #### Start Date **Date:** 2011-01 **Status Verified Date:** 2012-01 #### Study First Post Date **Date:** 2012-01-31 **Type:** ESTIMATED **Study First Submit Date:** 2012-01-24 **Study First Submit QC Date:** 2012-01-26 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Centro Universitário Augusto Motta #### Responsible Party **Investigator Affiliation:** Centro Universitário Augusto Motta **Investigator Full Name:** Cristina Marcia Dias **Investigator Title:** Professor DSc **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Respiratory dysfunction is the major cause of morbidity and mortality in patients with spinal muscular atrophy (SMA). Air stacking is a clearance airway technique frequently used but its effects on cardiac autonomic function in patients with spinal muscle atrophy is not clear. Objective: To evaluate the acute effect of air stacking and posture on cardiac autonomic function in patients with spinal muscular atrophy types II and III. Methods: Patients with spinal muscle atrophy type II and III will be recruited. Electrocardiogram signals will be recorded for analyses of heart rate variability during air stacking in supine and sitting position. **Detailed Description:** Background: Respiratory dysfunction is the major cause of morbidity and mortality in patients with spinal muscular atrophy. Air stacking is a clearance airway technique frequently used but its effects on cardiac autonomic function in patients with spinal muscle atrophy is not clear. Objective: To evaluate the acute effect of air stacking and posture on cardiac autonomic function in patients with spinal muscular atrophy types II and III. Methods: Patients with spinal muscle atrophy type II and III will be recruited. Electrocardiogram signals will be recorded for analyses of heart rate variability during air stacking in supine and sitting position. Data will collected before, during and after air stacking and will be compared using Anova Repeated Measures or Kruskal-Wallis Anova on Ranks, followed by Tukey test. The relationship between heart rate variability indexes and age will be evaluated by Pearson correlation. Significant level will be set at 5%. ### Conditions Module **Conditions:** - Spinal Muscular Atrophy **Keywords:** - spinal muscular atrophy - heart rate variability - posture - air stacking ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 9 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Electrocardiogram signals were recorded for analyses of heart rate variability during air stacking in supine and sitting position. **Intervention Names:** - Procedure: Air stacking manuever **Label:** Air stacking **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Air stacking **Description:** Electrocardiogram signals were recorded for analyses of heart rate variability during air stacking in supine and sitting position **Name:** Air stacking manuever **Other Names:** - HRV during air stacking **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** Evaluation of heart rate variability in patients with spinal muscular atrophy **Measure:** Cardiac autonomic function **Time Frame:** Thirty minutes during air stacking manuever ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * diagnosis of SMA types II and III, * absence of acute respiratory disease in the last two weeks, * agreement to participate in the study, * according written informed consent and * at least 5 years old. Exclusion Criteria: * respiratory disease in the last two weeks **Healthy Volunteers:** True **Maximum Age:** 25 Years **Minimum Age:** 5 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### Contacts Locations Module #### Overall Officials **Official 1:** **Affiliation:** Centro Universitário Augusto Motta **Name:** Cristina M Dias, PT - PhD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020763 - Term: Pathological Conditions, Anatomical - ID: D000020879 - Term: Neuromuscular Manifestations - ID: D000009461 - Term: Neurologic Manifestations - ID: D000009422 - Term: Nervous System Diseases - ID: D000013118 - Term: Spinal Cord Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000016472 - Term: Motor Neuron Disease - ID: D000019636 - Term: Neurodegenerative Diseases - ID: D000009468 - Term: Neuromuscular Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M12090 - Name: Muscular Atrophy - Relevance: HIGH - As Found: Muscular Atrophy - ID: M12091 - Name: Muscular Atrophy, Spinal - Relevance: HIGH - As Found: Spinal Muscular Atrophy - ID: M4589 - Name: Atrophy - Relevance: HIGH - As Found: Atrophy - ID: M22519 - Name: Pathological Conditions, Anatomical - Relevance: LOW - As Found: Unknown - ID: M22619 - Name: Neuromuscular Manifestations - Relevance: LOW - As Found: Unknown - ID: M12404 - Name: Neurologic Manifestations - Relevance: LOW - As Found: Unknown - ID: M15915 - Name: Spinal Cord Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M18879 - Name: Motor Neuron Disease - Relevance: LOW - As Found: Unknown - ID: M4024 - Name: Amyotrophic Lateral Sclerosis - Relevance: LOW - As Found: Unknown - ID: M21558 - Name: Neurodegenerative Diseases - Relevance: LOW - As Found: Unknown - ID: M12411 - Name: Neuromuscular Diseases - Relevance: LOW - As Found: Unknown - ID: T5342 - Name: Spinal Muscular Atrophy - Relevance: HIGH - As Found: Spinal Muscular Atrophy - ID: T349 - Name: Amyotrophic Lateral Sclerosis - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009133 - Term: Muscular Atrophy - ID: D000009134 - Term: Muscular Atrophy, Spinal - ID: D000001284 - Term: Atrophy ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00428779 **Brief Title:** Neuromuscular and Cognitive Fatigue During a 24 Hour Treadmill Running Exercise **Official Title:** Neuromuscular and Cognitive Fatigue During a 24h Treadmill Running Exercise. #### Organization Study ID Info **ID:** 0601095 #### Organization **Class:** OTHER **Full Name:** Centre Hospitalier Universitaire de Saint Etienne #### Secondary ID Infos **ID:** DGS 2006-0111 **ID:** 2006-A00312-49 ### Status Module #### Completion Date **Date:** 2007-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2009-06-11 **Type:** ESTIMATED **Last Update Submit Date:** 2009-06-10 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2007-12 **Type:** ACTUAL #### Start Date **Date:** 2007-09 **Status Verified Date:** 2009-06 #### Study First Post Date **Date:** 2007-01-30 **Type:** ESTIMATED **Study First Submit Date:** 2007-01-29 **Study First Submit QC Date:** 2007-01-29 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Centre Hospitalier Universitaire de Saint Etienne #### Responsible Party **Old Name Title:** Clément CAILLAUX **Old Organization:** CHU de Saint-Etienne ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The main purpose of the present study is to determine the relative contributions of central and peripheral fatigue during an ultra-endurance exercise. **Detailed Description:** For that purpose, 14 subjects running 24 hours on a treadmill will be compared with a control group being awake during the same period. Subjects who running will have electrically evoked forces on the muscles associated EMG activity, blood samples and muscle biopsy. ### Conditions Module **Conditions:** - Healthy **Keywords:** - Running during 24 hours - Voluntary evoked force on the muscles - Electrically evoked force on the muscles - EMG activity - Muscle biopsy - subjects will be recruited among experienced ultra-runners ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** HEALTH_SERVICES_RESEARCH #### Enrollment Info **Count:** 30 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** patients running during 24 hours without sleep **Intervention Names:** - Procedure: running during 24 hours without sleep **Label:** 1 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** patients without sleep during 24 hours **Intervention Names:** - Other: no sleeping **Label:** 2 **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - 1 **Description:** patients running during 24 hours without sleep **Name:** running during 24 hours without sleep **Type:** PROCEDURE #### Intervention 2 **Arm Group Labels:** - 2 **Description:** patients without sleep during 24 hours **Name:** no sleeping **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** -Voluntary and electrically evoked forces on the contracted muscles **Time Frame:** 24 hours #### Secondary Outcomes **Measure:** Electrically evoked forces on the relaxed muscles and associated EMG activity **Time Frame:** 24 hours **Measure:** Blood samples before the exercise **Time Frame:** every 4 hours **Measure:** Muscle biopsy (vastus lateralis) **Time Frame:** before and after the exercise ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * The subjects of the experimental group will be recruited among experienced ultra-runners . They all have run at least a 12 hour race or longer. * The control group will be recruited among experienced ultra-runners . Age of control group will be age experimental group more or less 2 years. Exclusion Criteria: * The subjects will not be selected if: * they were injured in the 3 months before the experiment * they had a tendon or joint pathology that could impair strength measurements. * they participate to an other experiment at the same time **Healthy Volunteers:** True **Maximum Age:** 60 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Saint-etienne **Country:** France **Facility:** Pr André GEYSSANT **Zip:** 42055 #### Overall Officials **Official 1:** **Affiliation:** Service de médecine du sport, CHU SAINT-ETIENNE **Name:** DENIS Christian, Professor **Role:** STUDY_CHAIR ### References Module #### References **Citation:** Morin JB, Samozino P, Feasson L, Geyssant A, Millet G. Effects of muscular biopsy on the mechanics of running. Eur J Appl Physiol. 2009 Jan;105(2):185-90. doi: 10.1007/s00421-008-0888-2. Epub 2008 Oct 8. **PMID:** 18841378 **Citation:** Jamart C, Francaux M, Millet GY, Deldicque L, Frere D, Feasson L. Modulation of autophagy and ubiquitin-proteasome pathways during ultra-endurance running. J Appl Physiol (1985). 2012 May;112(9):1529-37. doi: 10.1152/japplphysiol.00952.2011. Epub 2012 Feb 16. **PMID:** 22345427 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M8364 - Name: Fatigue - Relevance: HIGH - As Found: Fatigue ### Condition Browse Module - Meshes - ID: D000005221 - Term: Fatigue ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01914679 **Acronym:** PERRFECT-UM **Brief Title:** A Prospective Evaluation of RINCE to Reduce Fibromyalgia Effects - University of Michigan **Official Title:** A Phase 2 Clinical Trial Evaluating Use of the NeuroPoint Medical Device as a Treatment for Fibromyalgia. #### Organization Study ID Info **ID:** NPT-201-UM #### Organization **Class:** OTHER **Full Name:** University of Michigan ### Status Module #### Completion Date **Date:** 2015-02 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-04-19 **Type:** ACTUAL **Last Update Submit Date:** 2017-03-07 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2015-02 **Type:** ACTUAL #### Results First Post Date **Date:** 2017-04-19 **Type:** ACTUAL **Results First Submit Date:** 2017-01-04 **Results First Submit QC Date:** 2017-03-07 #### Start Date **Date:** 2013-07 **Status Verified Date:** 2017-03 #### Study First Post Date **Date:** 2013-08-02 **Type:** ESTIMATED **Study First Submit Date:** 2013-07-23 **Study First Submit QC Date:** 2013-07-30 **Why Stopped:** Sponsor terminated the study ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Cerephex Corporation #### Lead Sponsor **Class:** OTHER **Name:** University of Michigan #### Responsible Party **Investigator Affiliation:** University of Michigan **Investigator Full Name:** Daniel Clauw, MD **Investigator Title:** Professor of Anesthesiology **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to evaluate the mechanisms of noninvasive cortical electrostimulation therapy known as "Reduced Impedance Noninvasive Cortical Electrostimulation" RINCE)in the management of fibromyalgia. Patients who meet the 1990 American College of Rheumatology criteria for fibromyalgia will receive up to 24 RINCE treatments delivered by a medical device called "NeuroPoint". Approximately 20 patients will receive a combination of active and inactive (sham) therapy treatments over a 16-week period followed by a 4 week post-treatment evaluation. Patients will also undergo three (3) functional brain imaging scans while participating in the study: the first prior to the commencement of treatment, another mid-treatment; and the third at the completion of the treatment period. The study's primary outcome measure will be the change from baseline in self-reported 24-hour average pain intensity. The study's hypothesis is that there will be a change in pain intensity as well brain functioning. We do not expect there to be a statistically significant improvement in pain intensity due to the small sample but do expect to see statistically significant changes in cortical function as measured by EEG and fMRI ### Conditions Module **Conditions:** - Fibromyalgia **Keywords:** - Fibromyalgia - Pain - Brain - Stimulation - Device - Treatment ### Design Module #### Design Info **Intervention Model:** SEQUENTIAL **Intervention Model Description:** All participants went through a 4 week sham period, followed by a 12 week intervention with the device. ##### Masking Info **Masking:** SINGLE **Masking Description:** Participants did not know when the stimulation was occurring or not. **Who Masked:** - PARTICIPANT **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 17 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 4 weeks of inactive (sham) RINCE therapy involving no RINCE therapy followed by 12 weeks of RINCE therapy involving 24 total treatments **Intervention Names:** - Device: RINCE **Label:** Sham followed by device **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Sham followed by device **Description:** The intervention is repeat applications of RINCE therapy. The sham is created by not delivering the therapy stimulation signal. **Name:** RINCE **Other Names:** - RINCE therapy delivered by NeuroPoint Device **Type:** DEVICE ### Outcomes Module #### Other Outcomes **Description:** EEGs will be measured at the baseline, week 4, week 18 and week 21 visits. **Measure:** Change in Network Connectivity as Measured by EEG **Time Frame:** Baseline (week 1), week 6, week 18 and week 21 **Description:** The MASQ and MCS questionnaires will be administered at Baseline (week 1), week 6, week 10, week 14, week 18 and week 21. **Measure:** Investigate Changes in Neurocognitive Functioning Using the MASQ and MCS Assessments. **Time Frame:** Baseline and up to 21 weeks **Description:** Subjects will undergo a neuroimaging scan at Baseline (week 1), week 6, and week 18. The scan will measure network connectivity during stimuli. **Measure:** fMRI Measures of Network Connectivity **Time Frame:** Baseline (week 1), week 6, and week 18 #### Primary Outcomes **Description:** The units of measure represent self-reported average pain over the last 24 hours on a 0-100 pain rating scale where 0 is no pain and 100 is the worst pain imaginable. . **Measure:** Change in Patient 24-hour Recall Average Pain Intensity **Time Frame:** Assessed at Baseline (Week 1), Post-Sham (Week 5), Mid-Treatment (Week 10), Mid-Treatment (Week 14), Post-Treatment (Week 18) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patient must provide written informed consent and privacy authorization prior to participation in the study. Patient must have the ability to read and/or follow written and oral instructions, abide by the study restrictions, and agree to return for the required assessments. * Patient is female, 18-65 years of age (inclusive) at the time of consent. * Patient must have a confirmed diagnosis of fibromyalgia meeting the ACR 1990 diagnostic criteria for fibromyalgia. * Patients must have a 24-hour recall pain intensity score at both the screening and baseline visits between 40 and 90 inclusive on a 100 mm VAS scale. * Female patient of childbearing potential must be willing to use an acceptable method of birth control for the duration of their study participation. * Patients must be willing to refrain from all excluded therapies for the duration of the study. * In the opinion of the Investigator, the patient is willing and able to comply with all protocol-specified requirements. * Participants undergoing fMRI and 1H-MRS must be predominantly right handed (i.e. the subject writes with their right hand). Exclusion Criteria: The patient will not be eligible for enrollment if there is any history of, or in the opinion of the investigator, any of the following criteria are met: * Patient has a current significant psychological or psychiatric disorder (e.g., severe, unstable or poorly controlled depression, severe anxiety or obsessive-compulsive disorder; history of suicide attempt within preceding 5 years or suicidal ideation within preceding 6 months; or any history of bipolar disorder, schizophrenia, schizoaffective or other psychotic disorder). * Patient has a total Hospital and Anxiety Depression score of 11-21 for either anxiety or depression, or, based on the investigator's judgment, the patient is at risk of suicidal ideation or behavior. * Patient is currently using prohibited medications or treatments (see Prohibited Concomitant Therapy section of protocol) including stimulants, anesthetic patches, CPAP and/or TENS therapy. * Patient has an active diagnosis and is being treated for chronic infection or chronic condition such as lupus, rheumatoid arthritis, Parkinson's disease, multiple sclerosis, hepatitis, polio, seizures, or cancer (other than basal or squamous cell skin cancer). * Patient has any other chronic pain condition other than fibromyalgia that, in the Investigator's opinion, would interfere with the assessment of fibromyalgia (e.g., rheumatoid arthritis, post herpetic neuralgia, pain associated with diabetic neuropathy, severe pain due to degenerative joint disease, etc.) * Patient has history of seizure disorder, dementia or epilepsy anytime during his or her life except pediatric febrile seizures. * Female patient who is pregnant, planning a pregnancy, or breastfeeding. * Patient has any other disease or medical condition that, in the opinion of the investigator, would interfere with the evaluation of study device efficacy or safety, or would compromise the patient's ability to participate in or complete the study. * Patient has a history of other cranial electrical stimulation device use, or electroconvulsive therapy. * Patient has any metal implant, such as stents, aneurysm clips, shunts, pacemakers, defibrillators, neurostimulators or other contraindications with fMRI and 1H-MRS. Long-bone implants are not excluded. * Any anticipated need for surgery that might confound results or interfere with patient's ability to comply with the protocol. * Myocardial infarction during preceding 12 months, uncontrolled hypertension, active cardiac disease (American Heart Association Functional Class 2, 3 or 4 or Objective Class C or D), clinically significant cardiac rhythm or conduction abnormality, or anticipation of bypass or other cardiac surgery within the next 12 months. * Current systemic infection (e.g., HIV, hepatitis). * Patients receiving systemic corticosteroids (\> 5 mg prednisone or equivalent per day). * Pending or current litigation or disability claim (including Workman's Compensation). Patients currently receiving disability benefits will require medical monitor approval on a case-by-case basis. * Patient has history of alcohol and/or drug abuse. * Patient has participated in any investigational study within 30 days prior to Screening visit or is currently participating in another clinical trial. * Patient has received any prior experimental treatment or therapy that, in the opinion of the medical monitor, would compromise the patient's ability to participate in the study. * Patient is a staff member or relative of a staff member at either the investigative site or the Cerephex Corporation. * Body Mass Index (BMI) of greater than approximately 40 kg/m2. * Claustrophobia or any other factor sufficiently significant that it is likely to prevent successful completion of fMRI and 1H-MRS procedures. **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Ann Arbor **Country:** United States **Facility:** University of Michigan **State:** Michigan **Zip:** 48106 #### Overall Officials **Official 1:** **Affiliation:** University of Michigan **Name:** Daniel J Clauw, MD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009135 - Term: Muscular Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000012216 - Term: Rheumatic Diseases - ID: D000009468 - Term: Neuromuscular Diseases - ID: D000009422 - Term: Nervous System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases ### Condition Browse Module - Browse Leaves - ID: M8486 - Name: Fibromyalgia - Relevance: HIGH - As Found: Fibromyalgia - ID: M12161 - Name: Myofascial Pain Syndromes - Relevance: HIGH - As Found: Fibromyalgia - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M12092 - Name: Muscular Diseases - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: M15045 - Name: Rheumatic Diseases - Relevance: LOW - As Found: Unknown - ID: M6323 - Name: Collagen Diseases - Relevance: LOW - As Found: Unknown - ID: M12411 - Name: Neuromuscular Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000005356 - Term: Fibromyalgia - ID: D000009209 - Term: Myofascial Pain Syndromes ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Sham and RINCE Treatment **Description:** 4 weeks of inactive (sham) RINCE therapy involving no RINCE therapy (8 treatments) and 12 weeks of RINCE therapy (24 treatments). RINCE: The intervention is repeat applications of RINCE therapy. The sham is created by not delivering the therapy stimulation signal. **ID:** EG000 **Other Num Affected:** 14 **Other Num at Risk:** 17 **Serious Number At Risk:** 17 **Title:** Sham and RINCE Treatment **Frequency Threshold:** 5 #### Other Events **Term:** Loss of Balance Resulting in Fall **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** **Term:** Leg cramp **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** **Term:** Cough/Congestion **Organ System:** Infections and infestations **Source Vocabulary:** **Term:** Night Sweats **Organ System:** General disorders **Source Vocabulary:** **Term:** Chest Pain **Organ System:** Cardiac disorders **Source Vocabulary:** **Term:** Stomach Flu (achiness, diarrhea, nausea, vomiting) **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Rash **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** **Term:** Tinnitis **Organ System:** Ear and labyrinth disorders **Source Vocabulary:** **Term:** Skin Tenderness **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** **Term:** Yeast Infection **Organ System:** Infections and infestations **Source Vocabulary:** **Term:** Mental Fogginess **Organ System:** Psychiatric disorders **Source Vocabulary:** **Term:** Food Poisoning **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Sinus Infection **Organ System:** Infections and infestations **Source Vocabulary:** **Term:** Migraine **Organ System:** General disorders **Source Vocabulary:** **Term:** Vomiting **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Itchy Ears/Throat **Organ System:** Infections and infestations **Source Vocabulary:** **Term:** Sore Throat **Organ System:** Infections and infestations **Source Vocabulary:** **Term:** Bad Dreams **Organ System:** Psychiatric disorders **Source Vocabulary:** **Term:** Stomach Pain/Constipation **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Ankle Pain **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** **Term:** Stomach Cramps **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Vertigo **Organ System:** Ear and labyrinth disorders **Source Vocabulary:** **Term:** Myalgia **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** **Term:** Difficulty Focusing Vision **Organ System:** Eye disorders **Source Vocabulary:** **Term:** Shaking/Dizziness **Organ System:** General disorders **Source Vocabulary:** ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 17 **Units:** Participants ### Group **ID:** BG000 **Title:** Sham and RINCE Treatment **Description:** 4 weeks of inactive (sham) RINCE therapy involving no RINCE therapy (8 treatments) and 12 weeks of RINCE therapy (24 treatments). RINCE: The intervention is repeat applications of RINCE therapy. The sham is created by not delivering the therapy stimulation signal. ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 17 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 17 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Male **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Age, Categorical **Unit of Measure:** Participants ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement ### Limitations and Caveats **Description:** This mechanistic study wasn't powered to show statistically significant clinical improvement, even as originally planned. Sponsor terminated study early when they realized the devices were not delivering enough current to be of therapeutic benefit. ### Point of Contact **Email:** dclauw@med.umich.edu **Organization:** University of Michigan **Phone:** 734-998-6901 **Title:** Daniel Clauw, MD ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 15.68 - **Upper Limit:** - **Value:** 66.42 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 12 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 29.46 - **Upper Limit:** - **Value:** 49.83 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 12 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 24.34 - **Upper Limit:** - **Value:** 37.50 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 12 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 23.17 - **Upper Limit:** - **Value:** 32.18 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 11 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 24.52 - **Upper Limit:** - **Value:** 32.58 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 11 **Units:** Participants #### Outcome Measure 2 #### Outcome Measure 3 #### Outcome Measure 4 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** The units of measure represent self-reported average pain over the last 24 hours on a 0-100 pain rating scale where 0 is no pain and 100 is the worst pain imaginable. . **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** One participant missing from analysis at Week 14/Mid-Treatment/Visit 27 due to missed visit. **Reporting Status:** POSTED **Time Frame:** Assessed at Baseline (Week 1), Post-Sham (Week 5), Mid-Treatment (Week 10), Mid-Treatment (Week 14), Post-Treatment (Week 18) **Title:** Change in Patient 24-hour Recall Average Pain Intensity **Type:** PRIMARY **Unit of Measure:** units on a scale ##### Group **Description:** 4 weeks of inactive (sham) RINCE therapy involving no RINCE therapy (8 treatments) and 12 weeks of RINCE therapy (24 treatments). RINCE: The intervention is repeat applications of RINCE therapy. The sham is created by not delivering the therapy stimulation signal. **ID:** OG000 **Title:** Sham and RINCE Treatment #### Outcome Measure 2 **Description:** EEGs will be measured at the baseline, week 4, week 18 and week 21 visits. **Reporting Status:** NOT_POSTED **Time Frame:** Baseline (week 1), week 6, week 18 and week 21 **Title:** Change in Network Connectivity as Measured by EEG **Type:** OTHER_PRE_SPECIFIED #### Outcome Measure 3 **Description:** The MASQ and MCS questionnaires will be administered at Baseline (week 1), week 6, week 10, week 14, week 18 and week 21. **Reporting Status:** NOT_POSTED **Time Frame:** Baseline and up to 21 weeks **Title:** Investigate Changes in Neurocognitive Functioning Using the MASQ and MCS Assessments. **Type:** OTHER_PRE_SPECIFIED #### Outcome Measure 4 **Description:** Subjects will undergo a neuroimaging scan at Baseline (week 1), week 6, and week 18. The scan will measure network connectivity during stimuli. **Reporting Status:** NOT_POSTED **Time Frame:** Baseline (week 1), week 6, and week 18 **Title:** fMRI Measures of Network Connectivity **Type:** OTHER_PRE_SPECIFIED ### Participant Flow Module #### Group **Description:** 4 weeks of inactive (sham) RINCE therapy involving no RINCE therapy (8 treatments), and 12 weeks of RINCE therapy (24 treatments). RINCE: The intervention is repeat applications of RINCE therapy. The sham is created by not delivering the therapy stimulation signal. **ID:** FG000 **Title:** Sham and RINCE Treatment #### Period **Title:** Overall Study ##### Withdraw **Type:** Lack of Efficacy ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ##### Withdraw **Type:** Sponsor terminated study ###### Reason **Group ID:** FG000 **Number of Subjects:** 4 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 17 ##### Milestone **Type:** Began 12 Week Actual RINCE Treatment ###### Achievement **Group ID:** FG000 **Number of Subjects:** 17 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 12 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 5 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT05773079 **Brief Title:** Toripalimab Maintenance for Locally Advanced Head and Neck Squamous Cell Carcinoma **Official Title:** Toripalimab Maintenance After First-line Comprehensive Therapy for Locally Advanced Head and Neck Squamous Cell Carcinoma: a Single-arm, Phase II Study #### Organization Study ID Info **ID:** NFEC-2022-371 #### Organization **Class:** OTHER **Full Name:** Nanfang Hospital, Southern Medical University ### Status Module #### Completion Date **Date:** 2028-06 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-04-01 **Type:** ACTUAL **Last Update Submit Date:** 2024-03-28 **Overall Status:** NOT_YET_RECRUITING #### Primary Completion Date **Date:** 2026-02 **Type:** ESTIMATED #### Start Date **Date:** 2024-05 **Type:** ESTIMATED **Status Verified Date:** 2024-03 #### Study First Post Date **Date:** 2023-03-17 **Type:** ACTUAL **Study First Submit Date:** 2023-03-06 **Study First Submit QC Date:** 2023-03-06 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Nanfang Hospital, Southern Medical University #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** The guidelines for locally advanced head and neck squamous cell carcinoma currently recommend surgery / radiotherapy / chemotherapy / targeted therapy. However, the median PFS of patients with high risk factors after comprehensive treatment was about 17 months, and the 2-year PFS rate was about 40 %. The KEYNOTE-048 study showed that PD-1 monoclonal antibody alone or in combination with chemotherapy significantly improved survival and was safe for recurrent / metastatic head and neck squamous cell carcinoma. Therefore, PD-1 monoclonal antibody has become the first-line treatment of metastatic head and neck squamous cell carcinoma. For locally advanced head and neck squamous cell carcinoma, the existing studies on immunotherapy for neoadjuvant or concurrent chemoradiotherapy have not been clearly concluded. We previously used PD-1 monoclonal antibody for the maintenance treatment of patients after the first-line treatment of locally advanced head and neck squamous cell carcinoma, without residual tumor, which showed a trend of prolonged survival. Therefore, this study intends to explore whether the maintenance treatment of PD-1 monoclonal antibody terripril can further improve the survival of patients with locally advanced head and neck squamous cell carcinoma with high risk factors and no residual tumor after first-line comprehensive treatment, and the safety is good. ### Conditions Module **Conditions:** - Locally Advanced Head and Neck Squamous Cell Carcinoma **Keywords:** - Squamous Cell Carcinoma of Head and Neck - Toripalimab - PFS - Immunotherapy ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 28 **Type:** ESTIMATED **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Toripalimab 240mg, VD, Q3W (treatment time: maintain for 1 year or until disease progression or intolerance requires drug withdrawal) **Intervention Names:** - Drug: Toripalimab **Label:** Toripalimab **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Toripalimab **Description:** Within 4 to 12 weeks after the first line of treatment, the maintenance treatment of Toripalimab was started, and 240 mg of Toripalimab was injected intravenously every 3 weeks (the maintenance treatment time was not more than 1 year, until there was an intolerable toxic reaction, or disease progression, or withdrawal of consent, or the investigator judged that it was necessary to withdraw from the treatment, or the subject had received treprizumab treatment for a cumulative period of 1 year or other reasons specified in the protocol). During the treatment period, tumor imaging evaluation shall be conducted every 3 months to see the research process description for details, and the evaluation shall be conducted according to the evaluation criteria of recist. If the evaluation is progress, the treatment shall be stopped. **Name:** Toripalimab **Type:** DRUG ### Outcomes Module #### Other Outcomes **Description:** Any adverse signs ( including abnormal laboratory results ), symptoms or diseases that are time-related to the use of the study drug, regardless of whether there is a causal relationship with the study drug **Measure:** ≥Grade 3 Treatment-Related Adverse Events **Time Frame:** 2 years #### Primary Outcomes **Description:** The time from the day of first-line systemic treatment to disease progression or death. **Measure:** 2-year progression-free survival **Time Frame:** 2 years #### Secondary Outcomes **Description:** The time from the day of first-line treatment to the death of any cause. **Measure:** 2-year Overall Survival **Time Frame:** 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Locally advanced squamous cell carcinoma of the head and neck (AJCC8thIII-IV) confirmed by pathology in the initial treatment; * No residual lesions after first-line surgery/radiotherapy/chemotherapy/targeted comprehensive treatment; * Associated high risk factors: T3-4, regional lymph node positive, vascular invasion, neural invasion, lymph node capsular invasion, incisional margin positive; * The patients' age is between 18 and 70 years old; * The ECOG physical fitness status score is 0 or 1; * Estimated survival period ≥ 3 months; * The main organ functions meet the following standards: (1) Blood routine (without blood transfusion within 14 days): HGB ≥ 110g /L, WBC ≥ 3.0 × 10\^9\\/L， NEUT≥1.5 × 10\^9\\/L，PLT ≥75 × 10\^9\\/L； (2) Biochemical: BIL ≤ 1.5 times the upper limit of normal value (ULN), ALT and AST ≤ 2.0 × ULN, serum Cr ≤ 1.5 × ULN or endogenous creatinine clearance ≥ 50ml /min; (3) Occult blood in stool (-); (4) Normal urine routine, or urine protein\&lt;(++), or 24-hour urine protein\&lt;1.0g; (5) Left ventricular ejection fraction (LVEF) ≥ 50%. (6) The blood coagulation function is normal, and there is no active bleeding or thrombosis disease. A. International standardized ratio INR ≤ 1.5 × ULN； B. Partial thromboplastin time APTT ≤ 1.5 × ULN； C. Prothrombin time PT ≤ 1.5ULN. (7) Thyroid stimulating hormone (TSH) ≤ 1.5ULN; If the T3 and T4 levels are abnormal, they should be investigated. If the T3 and T4 levels are normal, they can be selected; * Women of childbearing age should agree to use contraceptives (such as intrauterine devices, birth control pills, or condoms) during and within 3 months after the end of medication; "Within 7 days prior to study enrollment, the serum or urine pregnancy test was negative and must be a non lactating patient. The male should agree to use contraception during the study period and within 3 months after the end of the study period; * Patients with hepatitis B virus (HBV) infection, inactive/asymptomatic HBV carriers, or patients with chronic or active HBV who received antiviral therapy for\&gt;1 week at the time of screening are allowed to be enrolled and continue treatment for more than 6 months after the study drug treatment. Patients with positive hepatitis C antibodies who have started anti hepatitis C virus treatment at the time of screening will be allowed to participate in the group; * Subjects voluntarily joined the study, signed an informed consent form, had good compliance, and cooperated with follow-up. Exclusion Criteria: * Distant metastasis of the tumor was detected upon enrollment; * Have a history of allergy to PD-1 monoclonal antibody or drug components; * There has been a history of other malignant tumors within the past 5 years or at the same time, except for cured skin basal cell carcinoma, cervical carcinoma in situ, and thyroid papillary carcinoma; * Uncontrolled clinical cardiac symptoms or diseases, such as: (1) heart failure above NYHA class II; (2) unstable angina; (3) myocardial infarction within 1 year; (4) patients with clinically significant supraventricular or ventricular arrhythmias requiring clinical intervention; * Have received any of the following treatments: a. Have previously received treatment with immunosuppressive drugs; B. Have received any investigational drug within 4 weeks before the first use of the investigational drug; C. Joining another clinical study at the same time, unless it is an observational (non intervention) clinical study or an intervention clinical study follow-up; D. Subjects who require systemic treatment with corticosteroids (greater than 10 mg prednisone equivalent dose per day) or other immunosuppressants within 2 weeks prior to the first use of the study drug, excluding the use of corticosteroids for local inflammation and the prevention of allergies, nausea, and vomiting. In the absence of active autoimmune diseases, it is allowed to inhale or locally use steroids and adrenal cortical hormone replacement with a dose greater than 10 mg/day of prednisone; E. Have received an anti-tumor vaccine or a live vaccine within 4 weeks before the first administration of the study drug; F. Major surgery or severe trauma requiring removal of the disease within 4 weeks before the first use of the study drug; * Serious infections (CTCAE greater than Level 2) occurred within 4 weeks before the first use of the study drug, such as severe pneumonia, intracranial infection, etc. that require hospitalization; * Have a history of active autoimmune diseases and autoimmune diseases, but does not include autoimmune mediated hypothyroidism treated with stable doses of thyroid replacement hormone; Type I diabetes with a stable dose of insulin; Patients with vitiligo or recovered childhood asthma/allergies who do not require any intervention in adulthood; * Have a history of immunodeficiency, including HIV testing positive, or have other acquired or congenital immunodeficiency diseases, or have a history of organ transplantation and bone marrow transplantation; * Have a history of interstitial lung disease and non infectious pneumonia; * Patients who have a history of active pulmonary tuberculosis infection through medical history or CT examination, or who have a history of active pulmonary tuberculosis infection within 1 year before enrollment, or who have a history of active pulmonary tuberculosis infection before 1 year but have not received formal treatment; * Subjects with active hepatitis (HBV DNA ≥ 2000 IU /ml or 10000 copies /ml) who have not been treated, and hepatitis C (hepatitis C antibody positive, and HCV-RNA above the detection limit of the analytical method) who have not been treated; * Known history of abuse, alcoholism, and drug abuse of psychotropic substances; * KPS score\<60, intolerance to anti-tumor therapy; * Pregnant or lactating women; * Engaging or expected to participate in other clinical studies; * Researchers believe that it is not suitable for inclusion. **Maximum Age:** 70 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000018307 - Term: Neoplasms, Squamous Cell - ID: D000006258 - Term: Head and Neck Neoplasms - ID: D000009371 - Term: Neoplasms by Site ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M5534 - Name: Carcinoma - Relevance: HIGH - As Found: Carcinoma - ID: M5550 - Name: Carcinoma, Squamous Cell - Relevance: HIGH - As Found: Squamous Cell Carcinoma - ID: M1689 - Name: Squamous Cell Carcinoma of Head and Neck - Relevance: HIGH - As Found: Head and Neck Squamous Cell Carcinoma - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M20451 - Name: Neoplasms, Squamous Cell - Relevance: LOW - As Found: Unknown - ID: M9348 - Name: Head and Neck Neoplasms - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002277 - Term: Carcinoma - ID: D000002294 - Term: Carcinoma, Squamous Cell - ID: D000077195 - Term: Squamous Cell Carcinoma of Head and Neck ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: Ot - Name: Other Dietary Supplements ### Intervention Browse Module - Browse Leaves - ID: M2853 - Name: Immunomodulating Agents - Relevance: LOW - As Found: Unknown - ID: M4854 - Name: Benzocaine - Relevance: LOW - As Found: Unknown - ID: T433 - Name: Tannic Acid - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03553979 **Acronym:** Grip&Health **Brief Title:** Grip&Health: Behavioural Intervention to Reduce Smoking, Stress and Improve Financial Health in Low-SES in Rotterdam **Official Title:** Project Grip&Health: A Behavioural Intervention Which Uses an Integral Approach to Reduce Stress, Smoking, Improve Financial Health and Self-perceived Health of Low SES-residents in Rotterdam #### Organization Study ID Info **ID:** FNO projectnr. 102291 #### Organization **Class:** OTHER **Full Name:** Erasmus Medical Center ### Status Module #### Completion Date **Date:** 2019-05-01 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2018-06-12 **Type:** ACTUAL **Last Update Submit Date:** 2018-05-30 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2018-10-01 **Type:** ESTIMATED #### Start Date **Date:** 2018-01-09 **Type:** ACTUAL **Status Verified Date:** 2018-05 #### Study First Post Date **Date:** 2018-06-12 **Type:** ACTUAL **Study First Submit Date:** 2018-01-08 **Study First Submit QC Date:** 2018-05-30 ### Sponsor Collaborators Module #### Collaborators **Class:** UNKNOWN **Name:** Indigo Rijnmond, the Netherlands **Class:** UNKNOWN **Name:** Avant Sanare, The Netherlands **Class:** OTHER **Name:** Erasmus University Rotterdam #### Lead Sponsor **Class:** OTHER **Name:** Erasmus Medical Center #### Responsible Party **Investigator Affiliation:** Erasmus Medical Center **Investigator Full Name:** Ingmar Franken **Investigator Title:** Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** Grip\&Health: randomised trial which will examine the effect of theory-based multicomponent behavioural intervention for reducing stress, smoking and improving financial health and perceived health of low-SES residents in Rotterdam. Between January 2018 and July 2018, a total of 300 participants will be recruited and randomised either to a stress management program (SM), stress management with a buddy program (SM-B) or a control condition. The investigators hypothesise that compared to participants in the control condition, participants in the intervention arms will demonstrate reduced stress, reduced smoking and improved financial health and perceived health. ### Conditions Module **Conditions:** - Smoking Cessation - Smoking, Cigarette - Smoking - Stress - Stress, Psychological - Health Behavior - Smoking, Tobacco **Keywords:** - stress management - stress intervention - smoking intervention - lower SES - lower income ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** SEQUENTIAL **Intervention Model Description:** Stress management program (SM) Stress management and Buddy program (SM-B) ##### Masking Info **Masking:** NONE **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 300 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The stress management program (SM) is a program which has been tailored to meet the specific needs of low-SES participants. The SM consists of 4-weekly sessions (1.5hours/session) and a follow-up session 8 weeks later. A core element of SM is its group-based format in which psycho-educative topics on stress responses and coping and motivation to stop smoking link up with cognitive and behavioural technique activities. **Intervention Names:** - Behavioral: Stress management program (SM) **Label:** Stress management program (SM) **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** The stress management + buddy program (SM-B) includes the same psycho-educative topics and exercises, cognitive and behavioural technique activities as the SM condition. The SM-B in addition to SM utilises one-to-one support through a buddy selected by a participant. A buddy, 18 year or older is a student or a volunteer who is recruited and trained by Indigo Rijnmond. The buddy pairs up with a participant and provides the following: supports participant in managing and filling in tax/welfare papers; 2) helps a participant to get a grip over his/her personal finances; and 3) helps a participant to overcome daily barriers (eg. arranging childcare). Over the duration of the course, the buddy meets up 6 times with a participant every second week in a public area. **Intervention Names:** - Behavioral: Stress management + Buddy program (SM-B) **Label:** Stress management + Buddy program (SM-B) **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Participants in the control condition are instructed to continue with their normal daily behaviour. They will be invited to complete the questionnaires and objective measurements at the equivalent times as the intervention groups, thus at baseline, 4 weeks after baseline and 12 weeks after baseline. After the control period, participants in the control condition will be offered the intervention. **Label:** Control **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Stress management program (SM) **Description:** Participants follow the SM program. **Name:** Stress management program (SM) **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - Stress management + Buddy program (SM-B) **Description:** In addition to receiving the SM, participants in the SM-B will also receive one-to-one support through a buddy. **Name:** Stress management + Buddy program (SM-B) **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** Change in self-reported Stress. The Depression, Anxiety and Stress (DASS) questionnaire will be used to measure change in self-reported stress from baseline (T0), at 4 weeks after baseline (T1) and at 12 weeks after baseline (T2). The DASS is a 21-item questionnaire with three self-report scales (Depression, Anxiety and Stress). Scores for depression, anxiety and stress are determined by summing the scores of the 21 items. Ranges for depression, anxiety and stress are 0-28, 0-20, and 0-33, respectively. Lower scores indicate less severity. In this study participants will be asked to complete the DASS-21 questionnaire at three different time points (T0, T1 and T2). This is done so the investigators can assess the change in scores of self-reported stress across time points (T0, T1 and T2). **Measure:** Self-reported Stress **Time Frame:** at baseline (T0), 4 weeks after baseline (T1) and 12 weeks after baseline (T2) **Description:** Change in objective evaluations of stress based on heart rate variability (HRV) from baseline (T0), at 4 weeks after baseline (T1) and at 12 weeks after baseline (T2). HRV indicates variability of time intervals between two consecutive heartbeats with each heartbeat having a R-wave which peaks in the R peak. The variations between two RR intervals are defined as HRV. A higher HRV value reflects a greater variation of the RR whereas a lower HRV indicates small variation. Participants beat-to-beat heart rate is measured using wrist wearables provided by Philips. During the measurements, the wearables are mounted on a participant's wrist and the participant is instructed to sit in a resting state for five minutes while the beat-to-beat heart rate is recorded. HRV analysis is derived from the recorded data using an appropriate software program. To assess change over time points (T0, T1 and T2), participants's beat-to-beat heart rate will be measured at three time points **Measure:** Objective Stress **Time Frame:** at baseline (T0), 4 weeks after baseline (T1) and 12 weeks after baseline (T2) **Description:** Change in self-reported Smoking. The Global Adult Tobacco Survey (GATS) questionnaire will be used to measure change in self-reported smoking from baseline (T0), at 4 weeks after baseline (T1) and at 12 weeks after baseline (T2). Participants will be asked to complete the questionnaire at three time points (T0, T1 and T2). This is done so the investigators can assess the change in self-reported smoking across time points. **Measure:** Self-reported Smoking **Time Frame:** at baseline (T0), 4 weeks after baseline (T1) and 12 weeks after baseline (T2) **Description:** Fagerstrom Test for Nicotine Dependence (FTND) questionnaire will be used to measure change in level of nicotine dependence from baseline (T0), at 4 weeks after baseline (T1) and at 12 weeks after baseline (T2). The FTND test is used to assess the intensity of physical addiction to nicotine. The test is designed to provide an ordinal measure of nicotine dependence related to cigarette smoking. It contains six items that evaluates the quantity of cigarette consumption, the compulsion to use and dependence. Yes/No items on FTND are scores from 0-1 and multiple choice items are scores from 0-3. The items are summed to yield a total score of 0-10. The higher the FTND, the more intense a participant's physical dependence on nicotine. In this study, participants will be asked to complete the FTND at three time points (T0, T1 and T2). This is done so the investigators can assess the change in the level of nicotine dependence across time points. **Measure:** Dependence of nicotine **Time Frame:** at baseline (T0), 4 weeks after baseline (T1) and 12 weeks after baseline (T2) **Description:** Change in exhaled Carbon Monoxide (CO) levels from baseline (T0), at 4 weeks after baseline (T1) and at 12 weeks after baseline (T2). The CO breath test is a biological method used to verify smoking or non-smoking status of participants. In this study, participants's exhaled CO levels will be measured (Micro SmokeLyzer; Bedfont, UK). A CO level ≥10 ppm reflects a heavy smoker. During the measurements, participants are instructed to hold their breath for 15 seconds and subsequently to slowly exhale into a mouthpiece connected to the SmokeLyzer device until their lungs are empty. Participants CO levels will be recorded at three time points (T0, T1 and T2). This is done so the investigators can assess the change in C0 levels across time points. **Measure:** Carbon Monoxide breath test **Time Frame:** at baseline (T0), 4 weeks after baseline (T1) and 12 weeks after baseline (T2) **Description:** The Short Form Health Survey version 2 (SF-12v2) questionnaire will be used to measure change in self-perceived health from baseline (T0), at 4 weeks after baseline (T1) and at 12 weeks after baseline (T2). The SF-12v2 is a 12-item with three dimensions both for functioning (physical, social and role) and for wellbeing (mental health, general health perceptions and pain). In this study, participants will be asked to rate their general health on a Likert-type scale (1=poor, 2=fair, 3= good, 4= very good, and 5=excellent). Low scores (poor) on the general health scale represent a person who believes his/her health to be poor and high score (excellent) represents someone who sees his/her health as excellent. Participants will be asked to complete the SF-12v2 questionnaire at three time points (T0, T1 and T2). This is done so the investigators can assess the change in health perceptions across time points. **Measure:** Self-perceived health **Time Frame:** at baseline (T0), 4 weeks after baseline (T1) and 12 weeks after baseline (T2) **Description:** Change in participant's's financial health will be measured from baseline (T0), at 4 weeks after baseline (T1) and at 12 weeks after baseline (T2). In this study, participants's financial health will be measured by asking them to report: 1) their total monthly income; 2) their spouse/partner's total monthly income; and 3) money shortages experienced in the past month. Participants will be asked to fill this questionnaire in at three time points (T0, T1 and T2). This is done to assess changes in financial health across time points. **Measure:** Financial health **Time Frame:** at baseline (T0), 4 weeks after baseline (T1) and 12 weeks after baseline (T2) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * perceive stress * smoke * poor financial health Exclusion Criteria: * follow other stress management course * follow smoking cessation course * receive help from debt services for their financial problems * have health problems which hamper their ability to take part in the study **Healthy Volunteers:** True **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Rotterdam **Contacts:** ***Contact 1:*** - **Email:** schop@euc.eur.nl - **Name:** Astrid Schop-Etman, Dr. - **Phone:** 0031657779496 - **Role:** CONTACT ***Contact 2:*** - **Email:** s.s.shagiwal@gmail.com - **Name:** Sara Shagiwal, MSc. - **Phone:** 0031650744268 - **Role:** CONTACT **Country:** Netherlands **Facility:** Erasmus University College **State:** Zuid-Holland **Status:** RECRUITING **Zip:** 3011 HP ### References Module #### References **Citation:** Shagiwal SS, Schop-Etman A, Bergwerff I, Vrencken W, Denktas S. The BeHealthyR Study: a randomized trial of a multicomponent intervention to reduce stress, smoking and improve financial health of low-income residents in Rotterdam. BMC Public Health. 2018 Jul 18;18(1):891. doi: 10.1186/s12889-018-5728-7. **PMID:** 30021551 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001526 - Term: Behavioral Symptoms ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: All - Name: All Conditions - Abbrev: BXM - Name: Behaviors and Mental Disorders ### Condition Browse Module - Browse Leaves - ID: M10295 - Name: Influenza, Human - Relevance: LOW - As Found: Unknown - ID: M16105 - Name: Stress, Psychological - Relevance: HIGH - As Found: Stress, Psychological - ID: M4818 - Name: Behavioral Symptoms - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000013315 - Term: Stress, Psychological ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04167579 **Acronym:** TVT **Brief Title:** STS & ACC Foundation's Transcutaneous Valve Therapy (TVT) Registry Foundation's Transcutaneous Valve Therapy (TVT) Registry **Official Title:** The Society of Thoracic Surgeons and American College of Cardiology Foundation's Transcutaneous Valve Therapy (TVT) Registry #### Organization Study ID Info **ID:** 172-19-CV #### Organization **Class:** OTHER **Full Name:** Hoag Memorial Hospital Presbyterian ### Status Module #### Completion Date **Date:** 2025-05-20 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-02-28 **Type:** ACTUAL **Last Update Submit Date:** 2023-02-27 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2025-05-20 **Type:** ESTIMATED #### Start Date **Date:** 2019-05-20 **Type:** ACTUAL **Status Verified Date:** 2023-02 #### Study First Post Date **Date:** 2019-11-19 **Type:** ACTUAL **Study First Submit Date:** 2019-08-08 **Study First Submit QC Date:** 2019-11-15 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Hoag Memorial Hospital Presbyterian #### Responsible Party **Investigator Affiliation:** Hoag Memorial Hospital Presbyterian **Investigator Full Name:** Colleen Balius **Investigator Title:** CLINICAL DATA COORDINATOR (RN) **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** True **Is FDA Regulated Drug:** False **Is US Export:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The TVT Registry was designed to support a national surveillance system to assess the characteristics, treatments, and outcomes of patients receiving transcutaneous valve therapies. **Detailed Description:** The TVT Registry was designed to support a national surveillance system to assess the characteristics, treatments, and outcomes of patients receiving transcutaneous valve therapies. Patient-level data are submitted by participating hospitals to The Society of Thoracic Surgeons (STS) and American College of Cardiology Foundation's (ACCF) joint TVT Registry. The purposes of the TVT Registry include: (i) collecting pertinent and standardized data elements from participating hospitals, health care providers and others that measure and assess the quality of care for patients receiving TVT; (ii) providing confidential periodic reports to participating hospitals, health care providers and others, to evaluate and improve the quality of care in these areas; and (iii) permitting and fostering appropriate research based upon the data collected by means of the TVT Registry. The secondary aim of the TVT Registry is to serve as a scalable data infrastructure forpost market studies. ### Conditions Module **Conditions:** - Transcutaneous Valve Therapies ### Design Module #### Design Info **Observational Model:** CASE_ONLY **Time Perspective:** OTHER #### Enrollment Info **Count:** 5000 **Type:** ESTIMATED **Patient Registry:** True **Study Type:** OBSERVATIONAL **Target Duration:** 1 Year ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** Any patient who undergoes an attempted transcutaneous aortic or mitral valve therapy procedure, with an FDA approved device. **Name:** Transcutaneous Valve Therapy **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** increase in the 30-day risk of adverse events among patients receiving TAVR **Measure:** Major adverse cardiac and cerebrovascular events **Time Frame:** 30 days from procedure #### Secondary Outcomes **Description:** Proportion of patients who are alive with at least moderate functional improvement (defined as at least 10 point improvement in Kansas City Cardiomyopathy Questionnaire (KCCQ) from baseline) at 1 year after receiving TAVR **Measure:** Mortality **Time Frame:** 1 year **Description:** increase in the 1 year risk of adverse events among patients receiving TAVR **Measure:** Major adverse cardiac and cerebrovascular events **Time Frame:** 1 year from procedure ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Adult patients (age 18 and older) who undergo an attempted transcutaneous aortic or mitral valve therapy procedure, with an FDA approved device. Exclusion Criteria: - **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Any patient who undergoes an attempted transcutaneous aortic or mitral valve therapy procedure, with an FDA approved device. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** colleen.balius@hoag.org **Name:** Colleen Balius **Phone:** 9497641497 **Role:** CONTACT #### Locations **Location 1:** **City:** Newport Beach **Contacts:** ***Contact 1:*** - **Email:** colleen.balius@hoag.org - **Name:** Colleen Balius, BSN, RN - **Phone:** 949-764-1497 - **Role:** CONTACT ***Contact 2:*** - **Name:** Colleen Balius, BSN, RN - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Hoah Memorial Hospital Presbyterian **State:** California **Status:** RECRUITING **Zip:** 92663 ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M18592 - Name: Ichthyosis, X-Linked - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05945979 **Brief Title:** Evaluation of the Efficacy of a New Supplement for Hair and Nails - in Vitro, Clinical, Subjective and Instrumental Study **Official Title:** Avaliação da eficácia de um Novo Suplemento Alimentar Para Cabelos e Unhas- Estudo in Vitro, clínico, Subjetivo e Instrumental #### Organization Study ID Info **ID:** FQM166-20 #### Organization **Class:** INDUSTRY **Full Name:** Farmoquimica S.A. ### Status Module #### Completion Date **Date:** 2022-06-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-07-14 **Type:** ACTUAL **Last Update Submit Date:** 2023-07-13 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-06-01 **Type:** ACTUAL #### Start Date **Date:** 2022-03-07 **Type:** ACTUAL **Status Verified Date:** 2023-06 #### Study First Post Date **Date:** 2023-07-14 **Type:** ACTUAL **Study First Submit Date:** 2023-05-26 **Study First Submit QC Date:** 2023-07-13 ### Sponsor Collaborators Module #### Collaborators **Class:** UNKNOWN **Name:** Kosmoscience Ciência e Tecnologia Cosmética Ltda #### Lead Sponsor **Class:** INDUSTRY **Name:** Farmoquimica S.A. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** A unicentric, exploratory, prospective clinical an in vitro study to evaluate efficacy and safety of a Supplement in improving the quality and general aspects of hair and nails in female participants through clinical,in vitro, subjective and instrumental evaluations **Detailed Description:** A unicentric, exploratory, prospective clinical an in vitro study to evaluate efficacy and safety of a Supplement in improving the quality and general aspects of hair and nails in female participants through clinical, in vitro, subjective and instrumental evaluations. It will be necessary 40 female participants aged between 25 and 55 years old with a clinical and in vitro diagnosis of telogen effluvium presenting at least one of the following characteristics: hair loss problems caused by stress, poor diet and excess of tinctures and chemicals procedures applied to hair, and menopausal women. The participants will remain in the study for 93 days using the product. Visits will be scheduled in D-3, D0, D45, D48, D90, D93. Instrumental evaluations: Evaluated on each visit: * To analyze the biological cycle of hair growth using Trichogram test - D-3, D45, D90; * Evaluation of hair growth kinetics using phototrichogram - D-3,D0, D45, D48, D90, D93; * Evaluation of the mechanical strength of the hair structure using EMIC DL 500- D-3, D90 * Evaluation of the dermal density of the scalp using the high-frequency ultrasound- D-3, D45, D90 * Participants will respond to a subjective assessment using a questionnaire to capture a subjective efficacy- D45, D90 ### Conditions Module **Conditions:** - Hair Loss - Nails, Ingrown **Keywords:** - capillary density - strengthening - hair loss ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 40 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 40 participants using the product for 90 days. Aims to evaluate the clinical, subjective and instrumental usage **Intervention Names:** - Dietary Supplement: A power supplement with biotin **Label:** A power supplement with biotin **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - A power supplement with biotin **Description:** A power dietary supplement containing aminoacids and vitamins **Name:** A power supplement with biotin **Type:** DIETARY_SUPPLEMENT ### Outcomes Module #### Primary Outcomes **Description:** Evaluate the effectiveness of the product investigational in providing reduction of hair loss through thrichogram analysis. If the ratio between anagen/telogen hairs strands is \> 4, it indicates improvement in reducing hair loss after daily and continuous use for a period of up to 90 days. **Measure:** Evaluate efficacy of a power supplement in reducing hair loss throgh thichogram analysis **Time Frame:** Day 93 #### Secondary Outcomes **Description:** Evaluate the phase of the biological cycle of hair growth (anagen, telogen or catagen) and determine the ratio between anagen/telogen hair strands. If the ratio between anagen/telogen hairs strands is ≤ 4, it indicates positive symptoms for androgenetic alopecia or telogen effluvium and the partipant will be included in the study.The trichogram was performed three days before the beginning of product intake, and after 45 days and 90 days of continuous use. **Measure:** Analysis of the biological cycle of hair growth- Trichogram **Time Frame:** Three days before the beginning of product intake, Day 45 and Day 90 **Description:** Evaluate the growth kinetics of hair strands after shaving a standardized area of 1 cm² of the scalp and taking images on the same day and three days after. In total, three scrapings were performed on three days before intervention, day 45 and day 90, totaling six image collections. A microscope (i-Scope USB, Moritex, JP) with a 30x lens and polarized light was used to obtain the images. In each collection, three microimages of the evaluated area on the head of each participant were recorded. **Measure:** Evaluation of hair growth kinetics- Phototrichogram **Time Frame:** Three days before the beginning of product intake, Day 0, Day 45, Day 48, Day 90 and Day 93 **Description:** Evaluation of the dermal thickness of hair strands through specific software. Patients will be submitted to the Ultrascan UC 22 MHz equipment (Courage + Khazaka electronic GmbH), on day 0 and after 45 days and 90 days of continuous use of the investigational product. **Measure:** Monitoring of hair growth by photographic documentation **Time Frame:** Day 0, Day 45 and Day 90 **Description:** Evaluation of the mechanical resistance of the hair. 50 hairs strands were collected from each participant and cut with the aid of scissors to avoid possible damage to the hair shaft resulting from traction for pulling out. The test was performed three days before the beginning of product intake and after 90 days of continuous use. **Measure:** Evaluation of the mechanical strength of the hair structure using EMIC DL 500 **Time Frame:** Three days before the beginning of product intake and Day 90 **Description:** Evaluate the perceived efficacy of the product from the participant's point of view through a subjective questionnaire after 45 days e 90 days of continuous use of the product. The questionnaire uses a 7-point scale, where 1 meaning poor and 7 means excellent. **Measure:** Subjective efficacy of the participants by questionnaire **Time Frame:** Day 45 and Day 90 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Phototype (Fitzpatrick): I to VI. * Have intact skin in the test region: scalp. * Present a clinical diagnosis of telogen effluvium. * Agree to adhere to the study procedures and requirements: study time, returns (D-3, D0, D45, D48,D90 and D93) to the laboratory to carry out the study procedures, administration of the product research, fill the use diary, fill the perceived efficacy questionnaire, carrying out the collection of hair samples (hair cut, close to the scalp - beginning of the study and after 90 days), not change hair habits during the study period. * Agree not to carry out chemical hair treatments (hair coloring, straightening, among others) and/or treatment to control/reduce hair loss and/or dandruff, among others, until the end of the study (90 days). * Agree with the procedure for collecting hair samples: trichogram and hair cut, close to the leather hairy. * Agree to administer 01 capsule/day of the investigational product. Present a minimum length of hair (3 cm) to carry out the procedure for collecting samples of hair * Present hair and brittleness for at least 3 months before the start of the study. * Signature of the Free and Informed Consent Term (TCLE). Exclusion Criteria: * Pregnancy * During the course of the study, the subject develops symptoms of COVID-19, such as: fever (temperature above 37.5°C), cough; dyspnoea (difficulty breathing characterized by rapid, short breaths, usually associated with heart or lung disease); myalgia (muscle pain); upper respiratory symptoms; fatigue and more rarely, gastrointestinal symptoms. * Appearance of disease that requires the use of medications prohibited in this protocol: use of corticoids and anti-inflammatories for a period longer than 15 days and/or in high doses (above 1mg/kg). * Use of immunosuppressants during the study period. * Use of Vitamin A or its derivatives and other vitamins and minerals. * Beginning or changing hormone treatments after signing the TCLE. * Swallowing difficulty: dysphagia. * Presence of dermatoses or active skin lesions (local and/or disseminated) in the study region. * Hypersensitivity reactions (allergic reactions, irritation or feelings of discomfort) caused by actives or excipients from the investigational product. * Vaccine use during the study period. * Diagnosis of diabetes or nephropathies during the study period. **Gender Based:** True **Healthy Volunteers:** True **Maximum Age:** 55 Years **Minimum Age:** 25 Years **Sex:** FEMALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Valinhos **Country:** Brazil **Facility:** Kosmoscience Ciência e Tecnologia Cosmética Ltda **State:** São Paulo **Zip:** 13270-180 ### IPD Sharing Statement Module **Info Types:** - STUDY_PROTOCOL - SAP - CSR **IPD Sharing:** YES **Time Frame:** D93 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007039 - Term: Hypotrichosis - ID: D000006201 - Term: Hair Diseases - ID: D000012871 - Term: Skin Diseases - ID: D000020763 - Term: Pathological Conditions, Anatomical - ID: D000009260 - Term: Nail Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M3846 - Name: Alopecia - Relevance: HIGH - As Found: Hair Loss - ID: M3847 - Name: Alopecia Areata - Relevance: HIGH - As Found: Hair Loss - ID: M12214 - Name: Nails, Ingrown - Relevance: HIGH - As Found: Nails, Ingrown - ID: M10089 - Name: Hypotrichosis - Relevance: LOW - As Found: Unknown - ID: M9293 - Name: Hair Diseases - Relevance: LOW - As Found: Unknown - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown - ID: M22519 - Name: Pathological Conditions, Anatomical - Relevance: LOW - As Found: Unknown - ID: M12211 - Name: Nail Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000000505 - Term: Alopecia - ID: D000000506 - Term: Alopecia Areata - ID: D000009263 - Term: Nails, Ingrown ### Intervention Browse Module - Ancestors - ID: D000014803 - Term: Vitamin B Complex - ID: D000014815 - Term: Vitamins - ID: D000018977 - Term: Micronutrients - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: Micro - Name: Micronutrients - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Hemat - Name: Hematinics - Abbrev: Vi - Name: Vitamins ### Intervention Browse Module - Browse Leaves - ID: M17558 - Name: Vitamins - Relevance: LOW - As Found: Unknown - ID: M4992 - Name: Biotin - Relevance: HIGH - As Found: Perianal - ID: M8618 - Name: Folic Acid - Relevance: LOW - As Found: Unknown - ID: M17546 - Name: Vitamin B Complex - Relevance: LOW - As Found: Unknown - ID: M21009 - Name: Micronutrients - Relevance: LOW - As Found: Unknown - ID: M16885 - Name: Trace Elements - Relevance: LOW - As Found: Unknown - ID: T439 - Name: Biotin - Relevance: HIGH - As Found: Perianal - ID: T473 - Name: Vitamin B7 - Relevance: LOW - As Found: Unknown - ID: T446 - Name: Folic Acid - Relevance: LOW - As Found: Unknown - ID: T448 - Name: Folate - Relevance: LOW - As Found: Unknown - ID: T475 - Name: Vitamin B9 - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000001710 - Term: Biotin ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03817879 **Brief Title:** Double-lumen Tubes (DLT) - Health Economic Study **Official Title:** A Randomized Controlled Study Comparing the VivaSight Double-lumen Tube With a Conventional Double-lumen Tube in Adult Patients Undergoing Thoracic Surgery #### Organization Study ID Info **ID:** CIS-009 #### Organization **Class:** INDUSTRY **Full Name:** Ambu A/S ### Status Module #### Completion Date **Date:** 2019-01-31 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-02-13 **Type:** ACTUAL **Last Update Submit Date:** 2020-02-03 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-01-31 **Type:** ACTUAL #### Results First Post Date **Date:** 2020-02-13 **Type:** ACTUAL **Results First Submit Date:** 2019-10-14 **Results First Submit QC Date:** 2020-02-03 #### Start Date **Date:** 2018-11-02 **Type:** ACTUAL **Status Verified Date:** 2019-10 #### Study First Post Date **Date:** 2019-01-28 **Type:** ACTUAL **Study First Submit Date:** 2019-01-09 **Study First Submit QC Date:** 2019-01-23 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Ambu A/S #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Is US Export:** False ### Description Module **Brief Summary:** The aim of this study is to make a health economic evaluation comparing novice physicians use of VivaSight double-lumen tube and a conventional double-lumen tube for single-lung ventilation during thoracic surgery at a teaching hospital. The hypothesis is, that both double-lumen tubes are equally cost-effective and the the incidence of fiberoptic bronchoscope use it the same for both tubes. **Detailed Description:** A randomized, controlled single-centre investigation comparing the VivaSight double-lumen tube and the conventional double-lumen tube at a teaching hospital. A pilot study including up to 10 subjects will be performed prior to the investigation is initiated. The investigation will include a total of 50 adult subjects (25 subjects in each group) admitted to the investigational site with established indication of single lung ventilation. The objective of the investigation is to compare the number of times the tube position needs to be verified with a scope and relevant costs between VivaSight double lumen tube and conventional double lument tube in a cost-effectiveness analysis. ### Conditions Module **Conditions:** - Single Lung Ventilation - Thoracic Surgery - Anesthesia - Cost-effectiveness ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** HEALTH_SERVICES_RESEARCH #### Enrollment Info **Count:** 70 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Procedure: VivaSight double-lumen tube for single-lung ventilation **Label:** VivaSight double-lumen tube **Type:** EXPERIMENTAL #### Arm Group 2 **Intervention Names:** - Procedure: Conventional double-lument tube for single-lung ventilation **Label:** Conventional double-lumen tube **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - VivaSight double-lumen tube **Description:** Procedure using a tube with a camera **Name:** VivaSight double-lumen tube for single-lung ventilation **Type:** PROCEDURE #### Intervention 2 **Arm Group Labels:** - Conventional double-lumen tube **Description:** Procedure using a tube without a camera **Name:** Conventional double-lument tube for single-lung ventilation **Type:** PROCEDURE ### Outcomes Module #### Other Outcomes **Description:** Qualitative assessment (face-validated and testet during pilot test) using a five-point scale (1: very easy/very good, 3: acceptable, 5: very difficult/very poor) **Measure:** Obtain User Perspective of the Device (VivaSight Double Lumen Tube or Conventional Double Lumen Tube) Used During Procedure. **Time Frame:** During procedure, up to 4 hours **Description:** Qualitative assessment (face-validated and testet during pilot test) using a questionnaire registrering postoperative outcomes (Yes/No) and degree of postoperative outcomes (mild/moderate/severe) **Measure:** Patient Reported Post Operative Outcomes **Time Frame:** Within 48 hours after the procedure #### Primary Outcomes **Measure:** Number of Times Bronchoscope is Used **Time Frame:** During procedure, up to 4 hours #### Secondary Outcomes **Measure:** Intubation Time **Time Frame:** During procedure, up to 4 hours **Measure:** Number of Intubation Attempts **Time Frame:** During procedure, up to 4 hours **Measure:** Number of Time the Tube Was Repositioned **Time Frame:** During procedure, up to 4 hours **Measure:** Number of Times Repositioning of the Tube Was Prevented **Time Frame:** During procedure, up to 4 hours **Measure:** Cost Per Procedure **Time Frame:** An average of 1 year ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Oral explanation of the investigation and Patient Information has been given to the subject or legal representative * The subject or legal representative has signed the Informed Consent * The subject is admitted at Odense University Hospital (OUH), department V * Subjects evaluated as eligible for single-lung ventilation with the use of a left sided DLT * Subjects \> 18 years of age Exclusion Criteria: * Subjects with known tracheobronchial anatomic anomalies * Subjects going for emergency procedures * Subjects with anticipated difficult airways * Subjects with known tracheal pathology * Subjects requiring rapid sequence induction * Surgeries in which other lung isolation devices or techniques may be warranted (e.g. tracheostomy, nasal intubation, bronchial blockers) * Subjects who cannot be intubated with a double-lumen tube (VivaSight-DL or conventional DLT) * Subjects requiring a right-sided DLT * Subjects who had participated in the study before **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Odense **Country:** Denmark **Facility:** Odense University Hospital **Zip:** 5000 ### IPD Sharing Statement Module **Description:** Data will described in groups **IPD Sharing:** NO ### References Module #### References **Citation:** Larsen S, Holm JH, Sauer TN, Andersen C. A Cost-Effectiveness Analysis Comparing the VivaSight Double-Lumen Tube and a Conventional Double-Lumen Tube in Adult Patients Undergoing Thoracic Surgery Involving One-Lung Ventilation. Pharmacoecon Open. 2020 Mar;4(1):159-169. doi: 10.1007/s41669-019-0163-y. **PMID:** 31297752 ## Document Section ### Large Document Module #### Large Docs - Date: 2018-06-26 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 1329803 - Type Abbrev: Prot_SAP - Upload Date: 2020-02-03T07:03 ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** VivaSight Double-lumen Tube **Deaths Num At Risk:** 30 **Description:** VivaSight double-lumen tube for single-lung ventilation: Procedure using a tube with a camera **ID:** EG000 **Other Num at Risk:** 30 **Serious Number At Risk:** 30 **Title:** VivaSight Double-lumen Tube **Group ID:** EG001 **Title:** Conventional Double-lumen Tube **Deaths Num At Risk:** 22 **Description:** Conventional double-lument tube for single-lung ventilation: Procedure using a tube without a camera **ID:** EG001 **Other Num at Risk:** 22 **Serious Number At Risk:** 22 **Title:** Conventional Double-lumen Tube **Frequency Threshold:** 0 **Time Frame:** Up to 48 hours post procedure. ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 30 **Group ID:** BG001 **Value:** 22 **Group ID:** BG002 **Value:** 52 **Units:** Participants ### Group **ID:** BG000 **Title:** VivaSight Double-lumen Tube **Description:** VivaSight double-lumen tube for single-lung ventilation: Procedure using a tube with a camera ### Group **ID:** BG001 **Title:** Conventional Double-lumen Tube **Description:** Conventional double-lument tube for single-lung ventilation: Procedure using a tube without a camera ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 10.02 **Value:** 65.80 #### Measurement **Group ID:** BG001 **Spread:** 7.16 **Value:** 68.50 #### Measurement **Group ID:** BG002 **Spread:** 8.94 **Value:** 66.94 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 30 **Group ID:** BG001 **Value:** 22 **Group ID:** BG002 **Value:** 52 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 13 #### Measurement **Group ID:** BG001 **Value:** 12 #### Measurement **Group ID:** BG002 **Value:** 25 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 17 #### Measurement **Group ID:** BG001 **Value:** 10 #### Measurement **Group ID:** BG002 **Value:** 27 **Category Title:** Male #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 30 **Group ID:** BG001 **Value:** 22 **Group ID:** BG002 **Value:** 52 **Class Title:** ### Measure #### Measurement **Group ID:** BG002 **Value:** 0 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 0 **Group ID:** BG001 **Value:** 0 **Group ID:** BG002 **Value:** 0 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 9.32 **Value:** 172.47 #### Measurement **Group ID:** BG001 **Spread:** 9.56 **Value:** 172.77 #### Measurement **Group ID:** BG002 **Spread:** 9.33 **Value:** 172.60 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 30 **Group ID:** BG001 **Value:** 22 **Group ID:** BG002 **Value:** 52 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 19.14 **Value:** 74.73 #### Measurement **Group ID:** BG001 **Spread:** 17.12 **Value:** 76.23 #### Measurement **Group ID:** BG002 **Spread:** 18.15 **Value:** 75.37 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 30 **Group ID:** BG001 **Value:** 22 **Group ID:** BG002 **Value:** 52 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 9 #### Measurement **Group ID:** BG001 **Value:** 4 #### Measurement **Group ID:** BG002 **Value:** 13 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 30 **Group ID:** BG001 **Value:** 22 **Group ID:** BG002 **Value:** 52 **Class Title:** Left #### Measurement **Group ID:** BG000 **Value:** 21 #### Measurement **Group ID:** BG001 **Value:** 18 #### Measurement **Group ID:** BG002 **Value:** 39 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 30 **Group ID:** BG001 **Value:** 22 **Group ID:** BG002 **Value:** 52 **Class Title:** Right **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Race and Ethnicity were not collected from any participant. **Title:** Race and Ethnicity Not Collected **Unit of Measure:** Participants ### Measure 4 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Height **Unit of Measure:** cm ### Measure 5 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Weight **Unit of Measure:** kg ### Measure 6 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Surgical lung **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement ### Point of Contact **Email:** aclu@ambu.com **Organization:** Ambu A/S **Phone:** +4529643748 **Title:** Anna Charlotte Lundgaard ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 ### Outcome Measure 7 ### Outcome Measure 8 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.25 - **Upper Limit:** - **Value:** 0.07 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.58 - **Upper Limit:** - **Value:** 3.14 **Title:** #### Outcome Measure 2 #### Outcome Measure 3 #### Outcome Measure 4 #### Outcome Measure 5 #### Outcome Measure 6 #### Outcome Measure 7 #### Outcome Measure 8 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** During procedure, up to 4 hours **Title:** Number of Times Bronchoscope is Used **Type:** PRIMARY **Unit of Measure:** Reported uses of bronchoscopes ##### Group **Description:** VivaSight double-lumen tube for single-lung ventilation: Procedure using a tube with a camera **ID:** OG000 **Title:** VivaSight Double-lumen Tube ##### Group **Description:** Conventional double-lument tube for single-lung ventilation: Procedure using a tube without a camera **ID:** OG001 **Title:** Conventional Double-lumen Tube #### Outcome Measure 2 **Reporting Status:** NOT_POSTED **Time Frame:** During procedure, up to 4 hours **Title:** Intubation Time **Type:** SECONDARY #### Outcome Measure 3 **Reporting Status:** NOT_POSTED **Time Frame:** During procedure, up to 4 hours **Title:** Number of Intubation Attempts **Type:** SECONDARY #### Outcome Measure 4 **Reporting Status:** NOT_POSTED **Time Frame:** During procedure, up to 4 hours **Title:** Number of Time the Tube Was Repositioned **Type:** SECONDARY #### Outcome Measure 5 **Reporting Status:** NOT_POSTED **Time Frame:** During procedure, up to 4 hours **Title:** Number of Times Repositioning of the Tube Was Prevented **Type:** SECONDARY #### Outcome Measure 6 **Reporting Status:** NOT_POSTED **Time Frame:** An average of 1 year **Title:** Cost Per Procedure **Type:** SECONDARY #### Outcome Measure 7 **Description:** Qualitative assessment (face-validated and testet during pilot test) using a five-point scale (1: very easy/very good, 3: acceptable, 5: very difficult/very poor) **Reporting Status:** NOT_POSTED **Time Frame:** During procedure, up to 4 hours **Title:** Obtain User Perspective of the Device (VivaSight Double Lumen Tube or Conventional Double Lumen Tube) Used During Procedure. **Type:** OTHER_PRE_SPECIFIED #### Outcome Measure 8 **Description:** Qualitative assessment (face-validated and testet during pilot test) using a questionnaire registrering postoperative outcomes (Yes/No) and degree of postoperative outcomes (mild/moderate/severe) **Reporting Status:** NOT_POSTED **Time Frame:** Within 48 hours after the procedure **Title:** Patient Reported Post Operative Outcomes **Type:** OTHER_PRE_SPECIFIED ### Participant Flow Module #### Group **Description:** VivaSight double-lumen tube for single-lung ventilation: Procedure using a tube with a camera **ID:** FG000 **Title:** VivaSight Double-lumen Tube #### Group **Description:** Conventional double-lument tube for single-lung ventilation: Procedure using a tube without a camera **ID:** FG001 **Title:** Conventional Double-lumen Tube #### Period **Title:** Overall Study ##### Withdraw **Type:** Drop out ###### Reason **Group ID:** FG000 **Number of Subjects:** 5 ###### Reason **Group ID:** FG001 **Number of Subjects:** 12 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 35 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 35 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 30 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 22 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 5 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 13 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT01155479 **Acronym:** PARADYSE **Brief Title:** A Placebo- and Active-Controlled Study of Preladenant in Early Parkinson's Disease (PD) (P05664) **Official Title:** A Phase 3, Double-Blind, Double-Dummy, Placebo- and Active-Controlled Dose-Range-Finding Efficacy and Safety Study of Preladenant in Subjects With Early Parkinson's Disease #### Organization Study ID Info **ID:** P05664 #### Organization **Class:** INDUSTRY **Full Name:** Merck Sharp & Dohme LLC #### Secondary ID Infos **ID:** 2009-013552-72 **Type:** EUDRACT_NUMBER **Domain:** Merck Study Number **ID:** MK-3814-024 **Type:** OTHER ### Status Module #### Completion Date **Date:** 2013-07-16 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-11-07 **Type:** ACTUAL **Last Update Submit Date:** 2018-10-09 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2013-07-16 **Type:** ACTUAL #### Results First Post Date **Date:** 2016-07-28 **Type:** ESTIMATED **Results First Submit Date:** 2016-06-15 **Results First Submit QC Date:** 2016-06-15 #### Start Date **Date:** 2010-07-06 **Type:** ACTUAL **Status Verified Date:** 2018-10 #### Study First Post Date **Date:** 2010-07-01 **Type:** ESTIMATED **Study First Submit Date:** 2010-06-30 **Study First Submit QC Date:** 2010-06-30 **Why Stopped:** Termininated for business reasons ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Merck Sharp & Dohme LLC #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** This is a one year, 2-part study to determine the efficacy and safety of preladenant, an adenosine type 2a (A2a) receptor antagonist. The purpose of Part 1 (first 26 weeks) is to determine if preladenant is effective in the treatment of early Parkinson's Disease. The purpose of Part 2 (second 26 weeks) is to determine if preladenant is safe and well tolerated. The primary efficacy hypothesis is that at least the 10 mg twice daily dose of preladenant is superior to placebo as measured by the change from Baseline to Week 26 in the sum of Unified Parkinson's Disease Rating Scale (UPDRS) Parts 2 and 3 scores (UPDRS2+3). ### Conditions Module **Conditions:** - Parkinson Disease ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 1022 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Preladenant 2 mg oral tablet and placebo for rasagiline taken in the morning (AM) followed by preladenant 2 mg oral tablet taken in the evening (PM) for 26 weeks (Part 1) and then for another 26 weeks (Part 2). **Intervention Names:** - Drug: Preladenant 2 mg tablet - Drug: Placebo for Rasagiline 1 mg capsule **Label:** Preladenant 2 mg **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Preladenant 5 mg oral tablet and placebo for rasagiline taken in the AM followed by preladenant 5 mg taken in the PM for 26 weeks (Part 1) and then for another 26 weeks (Part 2). **Intervention Names:** - Drug: Preladenant 5 mg tablet - Drug: Placebo for Rasagiline 1 mg capsule **Label:** Preladenant 5 mg **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Preladenant 10 mg oral tablet and placebo for rasagiline taken in the AM followed by preladenant 10 mg taken in the PM for 26 weeks (Part 1) and then for another 26 weeks (Part 2). **Intervention Names:** - Drug: Preladenant 10 mg tablet - Drug: Placebo for Rasagiline 1 mg capsule **Label:** Preladenant 10 mg **Type:** EXPERIMENTAL #### Arm Group 4 **Description:** Placebo for preladenant and placebo for rasagiline taken in the AM followed by placebo for preladenant taken in the PM for 26 weeks (Part 1); preladenant 5 mg was taken twice daily for 26 weeks (Part 2). **Intervention Names:** - Drug: Preladenant 5 mg tablet - Drug: Placebo for Rasagiline 1 mg capsule - Drug: Placebo for Preladenant **Label:** Placebo **Type:** PLACEBO_COMPARATOR #### Arm Group 5 **Description:** Rasagiline 1 mg oral capsule and placebo for preladenant taken in the AM followed by placebo for preladenant taken in the PM for 26 weeks (Part 1) and then for another 26 weeks (Part 2). **Intervention Names:** - Drug: Rasagiline 1 mg capsule - Drug: Placebo for Preladenant **Label:** Rasagiline **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Preladenant 2 mg **Description:** Preladenant 2 mg oral tablet taken twice daily **Name:** Preladenant 2 mg tablet **Other Names:** - SCH 420814 **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo - Preladenant 5 mg **Description:** Preladenant 5 mg oral tablet taken twice daily **Name:** Preladenant 5 mg tablet **Other Names:** - SCH 420814 **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - Preladenant 10 mg **Description:** Preladenant 10 mg oral tablet taken twice daily **Name:** Preladenant 10 mg tablet **Other Names:** - SCH 420814 **Type:** DRUG #### Intervention 4 **Arm Group Labels:** - Rasagiline **Description:** Rasagiline 1 mg oral capsule taken once daily **Name:** Rasagiline 1 mg capsule **Other Names:** - Azilect **Type:** DRUG #### Intervention 5 **Arm Group Labels:** - Placebo - Preladenant 10 mg - Preladenant 2 mg - Preladenant 5 mg **Description:** Placebo for rasagiline 1 mg oral capsule taken once daily **Name:** Placebo for Rasagiline 1 mg capsule **Type:** DRUG #### Intervention 6 **Arm Group Labels:** - Placebo - Rasagiline **Description:** Placebo for preladenant 2 mg, 5 mg, or 10 mg oral tablet taken twice daily **Name:** Placebo for Preladenant **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** The UPDRS is a clinician based rating scale used to measure motor impairments and disability. The UPDRS assesses six features of PD impairment. These are evaluated using a combination of data collected by interview and examination of the participant. The UPDRS Part 2 is the Activities of Daily Living (ADL) score and can range from 0-52 as determined by the physician. The UPDRS Part 3 is the Motor Examination (Total Motor Score \[TMS\]) and is defined as the total score, ranging from 0-108 as determined by the physician, of the tests given in the motor examination section. The combined scores of Parts 2 and 3 can range from 0-160 with the higher score indicating the worse condition. Change from baseline was analyzed using a constrained longitudinal analysis (cLDA) model with treatment, time, strata and treatment-by-time interaction as fixed effects and participant as a random effect. **Measure:** Change From Baseline in the Sum of Unified Parkinson's Disease Rating Scale Parts 2 and 3 Scores (UPDRS2+3) **Time Frame:** Baseline and Week 26 **Description:** An adverse event (AE) is defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to this medicinal product. **Measure:** Number of Participants With Adverse Events (AEs) in Part 1 **Time Frame:** Day 1 to Week 26 **Description:** An AE is defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to this medicinal product. **Measure:** Number of Participants Who Discontinued Study Due to an AE in Part 1 **Time Frame:** Day 1 to Week 26 **Description:** An adverse event (AE) is defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to this medicinal product. **Measure:** Number of Participants With Adverse Events (AEs) in Part 2 **Time Frame:** Week 27 to Week 52 **Description:** An AE is defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to this medicinal product. **Measure:** Number of Participants Who Discontinued Study Due to an AE in Part 2 **Time Frame:** Week 27 to Week 52 #### Secondary Outcomes **Description:** UPDRS is a clinician based rating scale used to measure motor impairments and disability; it assesses 6 features of PD impairment. These are evaluated using a combination of data collected by interview and examination of the participant. UPDRS Part 2 is Activities of Daily Living score and ranges from 0-52. UPDRS Part 3 is Motor Examination and ranges from 0-108. The combined scores of Parts 2 and 3 can range from 0-160 with the higher score indicating the worse condition. A Responder is defined as a participant with at least 20% improvement in UPDRS2+3 from Baseline to Week 26 (End of Part 1 Treatment); a participant with at least a 20% decrease from Baseline score in UPDRS2+3 is defined as a responder. The proportion of Responders was analyzed using a generalized linear mixed model with treatment effect, strata and Baseline UPDRS2+3 as a covariate, and treatment-by-time interaction as fixed effects and subject as random effect. **Measure:** Percentage of Responders (Participants With a ≥20% Improvement in UPDRS2+3) **Time Frame:** Baseline and Week 26 **Description:** The UPDRS is a clinician based rating scale used to measure motor impairments and disability. The UPDRS assesses six features of PD impairment. These are evaluated using a combination of data collected by interview and examination of the participant. The UPDRS Part 2 is the Activities of Daily Living (ADL) score and can range from 0-52 as determined by the physician with the higher score indicating the worse condition. Change from baseline was analyzed using a cLDA model with treatment, time, strata and treatment-by-time interaction as fixed effects and participant as a random effect. **Measure:** Change From Baseline in the UPDRS Part 2 Score (Activities of Daily Living [ADL]) **Time Frame:** Baseline and Week 26 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Has a diagnosis of idiopathic PD for \< 5 years. * If receiving amantadine and/or anticholinergics, must have been on a stable regimen of treatment for at least the 5 weeks immediately before Screening. (Note: Participants who are not taking any medications for PD are permitted to enroll in this trial.) * Must have a UPDRS Part 3 score of ≥10, a Hoehn and Yahr Stage ≤3, be ≥30 to ≤85 years of age, and have results of Screening clinical laboratory tests drawn within 5 weeks prior to randomization, clinically acceptable to the investigator, and not within the parameters specified for exclusion. * If sexually active or plan to be sexually active, must agree to use a highly effective method of birth control while the participant is in the study and for 2 weeks after the last dose of study drug. A male participant must also not donate sperm during the trial and within 2 weeks after the last dose of study drug. Exclusion Criteria: * Must not have a form of drug-induced or atypical Parkinsonism, cognitive impairment (ie, Montreal Cognitive Assessment \[MoCA\] score \<22), bipolar disorder, untreated major depressive disorder, schizophrenia, or other psychotic disorder; history of exposure to a known neurotoxin, or any neurological features not consistent with the diagnosis of PD as assessed by the investigator. * Must not have had surgery for PD. * Must not have a history of repeated strokes with stepwise progression of Parkinsonism or head injuries, or a stroke within 6 months of screening; poorly controlled diabetes; abnormal renal function; or a severe or ongoing unstable medical condition. * Must not have failed to show a therapeutic response if a diagnostic levodopa (L dopa) challenge had been done with a large test dose (\>500 mg) of L dopa (if malabsorption excluded), or failed to respond to an adequate previous treatment with dopaminergic therapy. * Must not have been treated with L dopa or dopamine agonists for 30 days or more. A participant who has been treated with L-dopa or dopamine agonists for \<30 days will be allowed to enter the study. These participants must stop taking dopaminergic medication 30 days prior to Randomization. * Must not be at imminent risk of self-harm or harm to others. * Must not have elevated blood pressure (BP) (systolic BP ≥150 mm Hg or diastolic BP ≥95 mm Hg) that cannot be adequately controlled with antihypertensive medication, as demonstrated by 2 BP measurements meeting acceptable BP criterion at consecutive scheduled or unscheduled visits between Screening and Randomization (a 5-6 week period), one of which must be the Randomization visit. * Must not have had any clinically significant cardiovascular event or procedure for 6 months prior to Randomization, including, but not limited to, myocardial infarction, angioplasty, unstable angina, or heart failure; and a participant must not have heart failure staged New York Heart Association Class III or IV. * Must not have an alanine aminotransferase (ALT) or aspartate amino transferase (AST) ≥ 3 x the upper limit of normal (ULN) or total bilirubin (T BIL) ≥ 1.5 x ULN. * Must not have active serologically-confirmed hepatic dysfunction (defined as viral infection \[Hepatitis B, C, or E; Epstein-Barr virus (EBV)\]; cytomegalovirus \[CMV\] or a history of diagnosis of drug- or alcohol-induced hepatic toxicity or frank hepatitis, or a history of diagnosis of drug- or alcohol-induced hepatic toxicity or frank hepatitis.) * Must not have a history within the past 5 years of a primary or recurrent malignant disease with the exception of adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, or in situ prostate cancer with a normal prostate-specific antigen (PSA) post resection. * Must not have received certain prespecified medications or ingested high tyramine-containing aged cheeses (eg, Stilton) for a prespecified time window before the trial, during the trial, and for 2 weeks after the trial. * Must not have an average daily consumption of more than three 4 ounce glasses (118 mL) of wine or the equivalent. * Must not have a severe or ongoing unstable medical condition (eg, any form of clinically significant cardiac disease, symptomatic orthostatic hypotension, seizures, or alcohol/drug dependence.) * Must not have allergy/sensitivity to investigational product(s) or its/their excipients. * Must not be breast-feeding, considering breast-feeding, pregnant or intending to become pregnant. * Must not have used preladenant ever, or any investigational drugs within 90 days immediately before screening. **Maximum Age:** 85 Years **Minimum Age:** 30 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Overall Officials **Official 1:** **Affiliation:** Merck Sharp & Dohme LLC **Name:** Medical Director **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **Description:** https://www.merck.com/clinical-trials/pdf/ProcedureAccessClinicalTrialData.pdf **IPD Sharing:** YES **URL:** http://engagezone.msd.com/ds_documentation.php ### References Module #### Available IPDs **Type:** CSR Synopsis **URL:** http://www.merck.com/clinical-trials/study.html?id=P05664&kw=P05664&tab=access #### References **Citation:** Stocchi F, Rascol O, Hauser RA, Huyck S, Tzontcheva A, Capece R, Ho TW, Sklar P, Lines C, Michelson D, Hewitt DJ; Preladenant Early Parkinson Disease Study Group. Randomized trial of preladenant, given as monotherapy, in patients with early Parkinson disease. Neurology. 2017 Jun 6;88(23):2198-2206. doi: 10.1212/WNL.0000000000004003. Epub 2017 May 10. **PMID:** 28490648 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020734 - Term: Parkinsonian Disorders - ID: D000001480 - Term: Basal Ganglia Diseases - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000009069 - Term: Movement Disorders - ID: D000080874 - Term: Synucleinopathies - ID: D000019636 - Term: Neurodegenerative Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases ### Condition Browse Module - Browse Leaves - ID: M13213 - Name: Parkinson Disease - Relevance: HIGH - As Found: Parkinson's Disease - ID: M22494 - Name: Parkinsonian Disorders - Relevance: LOW - As Found: Unknown - ID: M25603 - Name: Ganglion Cysts - Relevance: LOW - As Found: Unknown - ID: M16358 - Name: Synovial Cyst - Relevance: LOW - As Found: Unknown - ID: M4774 - Name: Basal Ganglia Diseases - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M12029 - Name: Movement Disorders - Relevance: LOW - As Found: Unknown - ID: M2217 - Name: Synucleinopathies - Relevance: LOW - As Found: Unknown - ID: M21558 - Name: Neurodegenerative Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010300 - Term: Parkinson Disease ### Intervention Browse Module - Ancestors - ID: D000008996 - Term: Monoamine Oxidase Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000018696 - Term: Neuroprotective Agents - ID: D000020011 - Term: Protective Agents - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: NeuroAg - Name: Neuroprotective Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M213846 - Name: Rasagiline - Relevance: HIGH - As Found: Sudden - ID: M11960 - Name: Monoamine Oxidase Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M20773 - Name: Neuroprotective Agents - Relevance: LOW - As Found: Unknown - ID: M21869 - Name: Protective Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000031967 - Term: Rasagiline ### Misc Info Module #### Removed Countries - Country: Argentina - Country: Bulgaria - Country: Canada - Country: Chile - Country: Colombia - Country: Czech Republic - Country: Finland - Country: France - Country: Germany - Country: Hungary - Country: India - Country: Israel - Country: Italy - Country: Mexico - Country: Peru - Country: Poland - Country: Russian Federation - Country: Spain - Country: Sweden - Country: Turkey - Country: United Kingdom - Country: United States - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** All Participants as Treated (APaT) - includes all participants who received at least one (1) dose of study drug. #### Event Groups **Group ID:** EG000 **Title:** Preladenant 2 mg - Part 1 **Description:** Participants received preladenant 2 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 2 mg oral tablet in PM for 26 weeks (Part 1). **ID:** EG000 **Other Num Affected:** 33 **Other Num at Risk:** 200 **Serious Number Affected:** 4 **Serious Number At Risk:** 200 **Title:** Preladenant 2 mg - Part 1 **Group ID:** EG001 **Title:** Preladenant 5 mg - Part 1 **Description:** Participants received preladenant 5 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 5 mg in the PM for 26 weeks (Part 1). **ID:** EG001 **Other Num Affected:** 39 **Other Num at Risk:** 202 **Serious Number Affected:** 5 **Serious Number At Risk:** 202 **Title:** Preladenant 5 mg - Part 1 **Group ID:** EG002 **Title:** Preladenant 10 mg - Part 1 **Description:** Participants received preladenant 10 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 10 mg in the PM for 26 weeks (Part 1). **ID:** EG002 **Other Num Affected:** 40 **Other Num at Risk:** 204 **Serious Number Affected:** 8 **Serious Number At Risk:** 204 **Title:** Preladenant 10 mg - Part 1 **Group ID:** EG003 **Title:** Placebo - Part 1 **Description:** Participants received placebo for preladenant and placebo for rasagiline in the AM followed by placebo for preladenant in the PM for 26 weeks (Part 1). **ID:** EG003 **Other Num Affected:** 36 **Other Num at Risk:** 198 **Serious Number Affected:** 3 **Serious Number At Risk:** 198 **Title:** Placebo - Part 1 **Group ID:** EG004 **Title:** Rasagiline - Part 1 **Description:** Participants received rasagiline 1 mg oral capsule and placebo for preladenant in the AM followed by placebo for preladenant in PM for 26 weeks (Part 1). **ID:** EG004 **Other Num Affected:** 34 **Other Num at Risk:** 203 **Serious Number Affected:** 9 **Serious Number At Risk:** 203 **Title:** Rasagiline - Part 1 **Group ID:** EG005 **Title:** Preladenant 2 mg - Part 2 **Description:** Participants received preladenant 2 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 2 mg oral tablet in PM for 26 weeks (Part 2). **ID:** EG005 **Other Num Affected:** 18 **Other Num at Risk:** 166 **Serious Number Affected:** 7 **Serious Number At Risk:** 166 **Title:** Preladenant 2 mg - Part 2 **Group ID:** EG006 **Title:** Preladenant 5 mg - Part 2 **Description:** Participants received preladenant 5 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 5 mg in the PM for 26 weeks (Part 2). **ID:** EG006 **Other Num Affected:** 22 **Other Num at Risk:** 177 **Serious Number Affected:** 1 **Serious Number At Risk:** 177 **Title:** Preladenant 5 mg - Part 2 **Group ID:** EG007 **Title:** Preladenant 10 mg - Part 2 **Description:** Participants received preladenant 10 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 10 mg in the PM for 26 weeks (Part 2). **ID:** EG007 **Other Num Affected:** 17 **Other Num at Risk:** 167 **Serious Number Affected:** 8 **Serious Number At Risk:** 167 **Title:** Preladenant 10 mg - Part 2 **Group ID:** EG008 **Title:** Placebo/Preladenant 5 Mg-Part 2 **Description:** Participants received preladenant 5 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 5 mg oral tablet in the PM for 26 weeks (Part 2). **ID:** EG008 **Other Num Affected:** 16 **Other Num at Risk:** 177 **Serious Number Affected:** 4 **Serious Number At Risk:** 177 **Title:** Placebo/Preladenant 5 Mg-Part 2 **Group ID:** EG009 **Title:** Rasagiline - Part 2 **Description:** Participants received rasagiline 1 mg oral capsule and placebo for preladenant in the AM followed by placebo for preladenant in PM for 26 weeks (Part 2). **ID:** EG009 **Other Num Affected:** 19 **Other Num at Risk:** 181 **Serious Number Affected:** 8 **Serious Number At Risk:** 181 **Title:** Rasagiline - Part 2 **Frequency Threshold:** 5 #### Other Events **Term:** Constipation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Back Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Tremor **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Hypertension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 17.0 #### Serious Events **Term:** Atrial Fibrillation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 204 **Num Events:** 1 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num Affected:** 1 **Num At Risk:** 166 **Num Events:** 1 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Acute Coronary Syndrome **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 202 **Num Events:** 1 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Acute Myocardial Infarction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num Affected:** 1 **Num At Risk:** 167 **Num Events:** 1 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Angina Pectoris **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num Affected:** 1 **Num At Risk:** 181 **Num Events:** 1 **Term:** Angina Unstable **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num Affected:** 1 **Num At Risk:** 203 **Num Events:** 1 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num Affected:** 1 **Num At Risk:** 181 **Num Events:** 1 **Term:** Cardiac Failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 204 **Num Events:** 1 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num Affected:** 1 **Num At Risk:** 177 **Num Events:** 1 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Coronary Artery Disease **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num Affected:** 1 **Num At Risk:** 203 **Num Events:** 1 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Left Ventricular Failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num Affected:** 1 **Num At Risk:** 177 **Num Events:** 1 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Myocardial Infarction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num Affected:** 1 **Num At Risk:** 203 **Num Events:** 1 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Constipation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num Affected:** 1 **Num At Risk:** 167 **Num Events:** 1 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Pancreatitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num Affected:** 1 **Num At Risk:** 198 **Num Events:** 1 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Pancreatitis Acute **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num Affected:** 1 **Num At Risk:** 203 **Num Events:** 1 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Chest Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num Affected:** 1 **Num At Risk:** 203 **Num Events:** 1 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Local Swelling **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num Affected:** 1 **Num At Risk:** 181 **Num Events:** 1 **Term:** Cholecystitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num Affected:** 1 **Num At Risk:** 198 **Num Events:** 1 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num Affected:** 1 **Num At Risk:** 177 **Num Events:** 1 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Cholecystitis Acute **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num Affected:** 1 **Num At Risk:** 198 **Num Events:** 1 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Jaundice Cholestatic **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num Affected:** 1 **Num At Risk:** 198 **Num Events:** 1 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Appendicitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 204 **Num Events:** 1 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num Affected:** 1 **Num At Risk:** 166 **Num Events:** 1 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Erysipelas **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num Affected:** 1 **Num At Risk:** 167 **Num Events:** 1 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Gastroenteritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 204 **Num Events:** 1 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Lower Respiratory Tract Infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num Affected:** 1 **Num At Risk:** 177 **Num Events:** 1 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Pneumonia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num Affected:** 1 **Num At Risk:** 166 **Num Events:** 1 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Pneumonia Bacterial **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num Affected:** 1 **Num At Risk:** 203 **Num Events:** 1 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num Affected:** 1 **Num At Risk:** 203 **Num Events:** 1 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num Affected:** 1 **Num At Risk:** 177 **Num Events:** 1 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Upper Respiratory Tract Infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 204 **Num Events:** 1 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Urinary Tract Infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 200 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Brain Contusion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 204 **Num Events:** 1 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Femur Fracture **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num Affected:** 1 **Num At Risk:** 166 **Num Events:** 1 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Fibula Fracture **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num Affected:** 1 **Num At Risk:** 177 **Num Events:** 1 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Hip Fracture **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num Affected:** 1 **Num At Risk:** 181 **Num Events:** 1 **Term:** Pelvic Fracture **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num Affected:** 1 **Num At Risk:** 181 **Num Events:** 1 **Term:** Tendon Rupture **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num Affected:** 1 **Num At Risk:** 203 **Num Events:** 1 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Tibia Fracture **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num Affected:** 1 **Num At Risk:** 177 **Num Events:** 1 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Alanine Aminotransferase Increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 202 **Num Events:** 2 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Antinuclear Antibody Increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 204 **Num Events:** 1 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Aspartate Aminotransferase Increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 202 **Num Events:** 1 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Hepatic Enzyme Increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 202 **Num Events:** 1 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Hyponatraemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 204 **Num Events:** 1 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Vitamin B12 Deficiency **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 204 **Num Events:** 1 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Back Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 202 **Num Events:** 1 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Colon Cancer **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 204 **Num Events:** 1 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Osteoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num Affected:** 1 **Num At Risk:** 167 **Num Events:** 1 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Sinonasal Papilloma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num Affected:** 1 **Num At Risk:** 181 **Num Events:** 1 **Term:** Uterine Leiomyoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num Affected:** 1 **Num At Risk:** 166 **Num Events:** 1 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Cerebrovascular Accident **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num Affected:** 1 **Num At Risk:** 167 **Num Events:** 1 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Haemorrhagic Stroke **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 204 **Num Events:** 1 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Parkinson's Disease **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num Affected:** 1 **Num At Risk:** 167 **Num Events:** 1 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num Affected:** 1 **Num At Risk:** 181 **Num Events:** 1 **Term:** Sciatica **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num Affected:** 1 **Num At Risk:** 198 **Num Events:** 1 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Vertebrobasilar Insufficiency **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num Affected:** 1 **Num At Risk:** 177 **Num Events:** 1 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Depression Suicidal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num Affected:** 1 **Num At Risk:** 203 **Num Events:** 1 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Suicidal Ideation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 200 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Suicide Attempt **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num Affected:** 1 **Num At Risk:** 166 **Num Events:** 1 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Cystitis Haemorrhagic **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num Affected:** 1 **Num At Risk:** 166 **Num Events:** 1 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Nephrolithiasis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 204 **Num Events:** 1 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Renal Failure Acute **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 200 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Renal Failure Chronic **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 202 **Num Events:** 1 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Benign Prostatic Hyperplasia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Reproductive system and breast disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 200 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num Affected:** 1 **Num At Risk:** 167 **Num Events:** 1 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Pulmonary Embolism **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num Affected:** 1 **Num At Risk:** 166 **Num Events:** 1 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num Affected:** 1 **Num At Risk:** 181 **Num Events:** 1 **Term:** Pulmonary Fibrosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num Affected:** 1 **Num At Risk:** 177 **Num Events:** 1 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Pregnancy of Partner **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Social circumstances **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num Affected:** 1 **Num At Risk:** 203 **Num Events:** 1 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Phlebitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num Affected:** 1 **Num At Risk:** 166 **Num Events:** 1 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num At Risk:** 167 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Term:** Sudden Cardiac Death **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num At Risk:** 200 **Group ID:** EG001 **Num At Risk:** 202 **Group ID:** EG002 **Num At Risk:** 204 **Group ID:** EG003 **Num At Risk:** 198 **Group ID:** EG004 **Num At Risk:** 203 **Group ID:** EG005 **Num At Risk:** 166 **Group ID:** EG006 **Num At Risk:** 177 **Group ID:** EG007 **Num Affected:** 1 **Num At Risk:** 167 **Num Events:** 1 **Group ID:** EG008 **Num At Risk:** 177 **Group ID:** EG009 **Num At Risk:** 181 **Time Frame:** Up to 54 weeks (including 2 weeks of follow-up) ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 204 **Group ID:** BG001 **Value:** 204 **Group ID:** BG002 **Value:** 206 **Group ID:** BG003 **Value:** 204 **Group ID:** BG004 **Value:** 204 **Group ID:** BG005 **Value:** 1022 **Units:** Participants ### Group **ID:** BG000 **Title:** Preladenant 2 mg **Description:** Participants received preladenant 2 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 2 mg oral tablet in PM for 26 weeks (Part 1) and then for another 26 weeks (Part 2). ### Group **ID:** BG001 **Title:** Preladenant 5 mg **Description:** Participants received preladenant 5 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 5 mg in the PM for 26 weeks (Part 1) and then for another 26 weeks (Part 2). ### Group **ID:** BG002 **Title:** Preladenant 10 mg **Description:** Participants received preladenant 10 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 10 mg in the PM for 26 weeks (Part 1) and then for another 26 weeks (Part 2). ### Group **ID:** BG003 **Title:** Placebo **Description:** Participants received placebo for preladenant and placebo for rasagiline in the AM followed by placebo for preladenant in the PM for 26 weeks (Part 1); preladenant 5 mg was taken twice daily for 26 weeks (Part 2). ### Group **ID:** BG004 **Title:** Rasagiline **Description:** Participants received rasagiline 1 mg oral capsule and placebo for preladenant in the AM followed by placebo for preladenant in PM for 26 weeks (Part 1) and then for another 26 weeks (Part 2). ### Group **ID:** BG005 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 10.5 **Value:** 63.0 #### Measurement **Group ID:** BG001 **Spread:** 10.2 **Value:** 62.3 #### Measurement **Group ID:** BG002 **Spread:** 11.1 **Value:** 63.8 #### Measurement **Group ID:** BG003 **Spread:** 10.0 **Value:** 63.3 #### Measurement **Group ID:** BG004 **Spread:** 10.2 **Value:** 62.9 #### Measurement **Group ID:** BG005 **Spread:** 10.4 **Value:** 63.1 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 78 #### Measurement **Group ID:** BG001 **Value:** 90 #### Measurement **Group ID:** BG002 **Value:** 90 #### Measurement **Group ID:** BG003 **Value:** 82 #### Measurement **Group ID:** BG004 **Value:** 85 #### Measurement **Group ID:** BG005 **Value:** 425 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 126 #### Measurement **Group ID:** BG001 **Value:** 114 #### Measurement **Group ID:** BG002 **Value:** 116 #### Measurement **Group ID:** BG003 **Value:** 122 #### Measurement **Group ID:** BG004 **Value:** 119 #### Measurement **Group ID:** BG005 **Value:** 597 **Category Title:** Male **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** Years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants **Population Description:** All Participants as Randomized ## Results Section - More Information Module ### Certain Agreement **Other Details:** The Investigator agrees not to publish or publicly present any interim results of the trial without the prior written consent of the sponsor. The Investigator further agrees to provide the sponsor 45 days prior to submission for publication or presentation, review copies of abstracts or manuscripts for publication that report any results of the trial. The sponsor shall have the right to review and comment. **Restriction Type:** OTHER **Restrictive Agreement:** True ### Point of Contact **Email:** ClinicalTrialsDisclosure@merck.com **Organization:** Merck Sharp & Dohme Corp. **Phone:** 1-800-672-6372 **Title:** Senior Vice President, Global Clinical Development ## Results Section - Outcome Measures Module ### Outcome Measure 1 #### Analysis **CI Lower Limit:** 0.86 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 4.30 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Preladenant 2 mg (Part 1) vs Placebo (Part 1) **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.0033 **P-Value Comment:** **Parameter Type:** Difference in Estimated Means **Parameter Value:** 2.60 **Statistical Comment:** **Statistical Method:** constrained longitudinal analysis **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** -0.41 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 2.94 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Preladenant 5 mg (Part 1) vs Placebo (Part 1) **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.1382 **P-Value Comment:** **Parameter Type:** Difference in Estimated Means **Parameter Value:** 1.30 **Statistical Comment:** **Statistical Method:** constrained longitudinal analysis **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** -1.29 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 2.11 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Preladenant 10 mg (Part 1) vs Placebo (Part 1) **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.6378 **P-Value Comment:** **Parameter Type:** Difference in Estimated Means **Parameter Value:** 0.40 **Statistical Comment:** **Statistical Method:** constrained longitudinal analysis **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** -1.35 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 2.03 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Rasagiline (Part 1) vs Placebo (Part 1) **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.6923 **P-Value Comment:** **Parameter Type:** Difference in Estimated Means **Parameter Value:** 0.30 **Statistical Comment:** **Statistical Method:** constrained longitudinal analysis **Tested Non-Inferiority:** ### Outcome Measure 2 #### Analysis **CI Lower Limit:** -21.0 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 1.82 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Preladenant 2 mg (Part 1) vs Placebo (Part 1) **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.0785 **P-Value Comment:** p-value is for the estimated Odds Ratio based on a generalized linear mixed model with baseline UPDRS2+3 as a covariate and treatment-by-time interaction as fixed effect and participant as random effect. **Parameter Type:** Difference vs Placebo (%) **Parameter Value:** -9.7 **Statistical Comment:** **Statistical Method:** generalized linear mixed model **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** -17.6 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 5.05 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Preladenant 5 mg (Part 1) vs Placebo (Part 1) **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.2735 **P-Value Comment:** p-value is for the estimated Odds Ratio based on a generalized linear mixed model with baseline UPDRS2+3 as a covariate and treatment-by-time interaction as fixed effect and participant as random effect. **Parameter Type:** Difference vs Placebo (%) **Parameter Value:** -6.3 **Statistical Comment:** **Statistical Method:** generalized linear mixed model **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** -15.2 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 7.99 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Preladenant 10 mg (Part 1) vs Placebo (Part 1) **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.4823 **P-Value Comment:** p-value is for the estimated Odds Ratio based on a generalized linear mixed model with baseline UPDRS2+3 as a covariate and treatment-by-time interaction as fixed effect and participant as random effect. **Parameter Type:** Difference vs Placebo (%) **Parameter Value:** -3.7 **Statistical Comment:** **Statistical Method:** generalized linear mixed model **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** -13.9 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 9.24 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Rasagiline (Part 1) vs Placebo (Part 1) **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.6827 **P-Value Comment:** p-value is for the estimated Odds Ratio based on a generalized linear mixed model with baseline UPDRS2+3 as a covariate and treatment-by-time interaction as fixed effect and participant as random effect. **Parameter Type:** Difference vs Placebo (%) **Parameter Value:** -2.3 **Statistical Comment:** **Statistical Method:** generalized linear mixed model **Tested Non-Inferiority:** ### Outcome Measure 3 #### Analysis **CI Lower Limit:** 0.09 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 1.27 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Preladenant 2 mg (Part 1) vs Placebo (Part 1) **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.0235 **P-Value Comment:** **Parameter Type:** Difference in Estimated Means **Parameter Value:** 0.70 **Statistical Comment:** **Statistical Method:** constrained longitudinal analysis **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** -0.11 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 1.04 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Preladenant 5 mg (Part 1) vs Placebo (Part 1) **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.1093 **P-Value Comment:** **Parameter Type:** Difference in Estimated Means **Parameter Value:** 0.50 **Statistical Comment:** **Statistical Method:** constrained longitudinal analysis **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** -0.42 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 0.75 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Preladenant 10 mg (Part 1) vs Placebo (Part 1) **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.5756 **P-Value Comment:** **Parameter Type:** Difference in Estimated Means **Parameter Value:** 0.20 **Statistical Comment:** **Statistical Method:** constrained longitudinal analysis **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** -0.45 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 0.70 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Rasagiline (Part 1) vs Placebo (Part 1) **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.6657 **P-Value Comment:** **Parameter Type:** Difference in Estimated Means **Parameter Value:** 0.10 **Statistical Comment:** **Statistical Method:** constrained longitudinal analysis **Tested Non-Inferiority:** ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 ### Outcome Measure 7 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.63 - **Upper Limit:** - **Value:** 0.3 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.60 - **Upper Limit:** - **Value:** -1.0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 0.61 - **Upper Limit:** - **Value:** -1.8 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** 0.61 - **Upper Limit:** - **Value:** -2.2 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** 0.61 - **Upper Limit:** - **Value:** -1.9 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 18.95 - **Spread:** - **Upper Limit:** 34.33 - **Value:** 25.90 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 22.50 - **Spread:** - **Upper Limit:** 37.72 - **Value:** 29.50 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 24.06 - **Spread:** - **Upper Limit:** 40.02 - **Value:** 31.50 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** 27.49 - **Spread:** - **Upper Limit:** 43.82 - **Value:** 35.20 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** 25.60 - **Spread:** - **Upper Limit:** 41.51 - **Value:** 33.10 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.22 - **Upper Limit:** - **Value:** 0.30 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.21 - **Upper Limit:** - **Value:** 0.10 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 0.21 - **Upper Limit:** - **Value:** -0.20 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** 0.21 - **Upper Limit:** - **Value:** -0.40 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** 0.21 - **Upper Limit:** - **Value:** -0.20 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 108 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 110 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 121 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 102 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 105 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 13 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 8 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 20 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 8 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 6 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 116 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 120 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 113 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 120 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 119 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 7 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 8 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** The UPDRS is a clinician based rating scale used to measure motor impairments and disability. The UPDRS assesses six features of PD impairment. These are evaluated using a combination of data collected by interview and examination of the participant. The UPDRS Part 2 is the Activities of Daily Living (ADL) score and can range from 0-52 as determined by the physician. The UPDRS Part 3 is the Motor Examination (Total Motor Score \[TMS\]) and is defined as the total score, ranging from 0-108 as determined by the physician, of the tests given in the motor examination section. The combined scores of Parts 2 and 3 can range from 0-160 with the higher score indicating the worse condition. Change from baseline was analyzed using a constrained longitudinal analysis (cLDA) model with treatment, time, strata and treatment-by-time interaction as fixed effects and participant as a random effect. **Dispersion Type:** Standard Error **Parameter Type:** MEAN **Population Description:** Full Analysis Set (FAS): All randomized and treated participants with at least one post-baseline value. **Reporting Status:** POSTED **Time Frame:** Baseline and Week 26 **Title:** Change From Baseline in the Sum of Unified Parkinson's Disease Rating Scale Parts 2 and 3 Scores (UPDRS2+3) **Type:** PRIMARY **Unit of Measure:** Score on a Scale ##### Group **Description:** Participants received preladenant 2 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 2 mg oral tablet in PM for 26 weeks. **ID:** OG000 **Title:** Preladenant 2 mg (Part 1) ##### Group **Description:** Participants received preladenant 5 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 5 mg in the PM for 26 weeks. **ID:** OG001 **Title:** Preladenant 5 mg (Part 1) ##### Group **Description:** Participants received preladenant 10 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 10 mg in the PM for 26 weeks. **ID:** OG002 **Title:** Preladenant 10 mg (Part 1) ##### Group **Description:** Participants received placebo for preladenant and placebo for rasagiline in the AM followed by placebo for preladenant in the PM for 26 weeks. **ID:** OG003 **Title:** Placebo (Part 1) ##### Group **Description:** Participants received rasagiline 1 mg oral capsule and placebo for preladenant in the AM followed by placebo for preladenant in PM for 26 weeks. **ID:** OG004 **Title:** Rasagiline (Part 1) #### Outcome Measure 2 **Description:** UPDRS is a clinician based rating scale used to measure motor impairments and disability; it assesses 6 features of PD impairment. These are evaluated using a combination of data collected by interview and examination of the participant. UPDRS Part 2 is Activities of Daily Living score and ranges from 0-52. UPDRS Part 3 is Motor Examination and ranges from 0-108. The combined scores of Parts 2 and 3 can range from 0-160 with the higher score indicating the worse condition. A Responder is defined as a participant with at least 20% improvement in UPDRS2+3 from Baseline to Week 26 (End of Part 1 Treatment); a participant with at least a 20% decrease from Baseline score in UPDRS2+3 is defined as a responder. The proportion of Responders was analyzed using a generalized linear mixed model with treatment effect, strata and Baseline UPDRS2+3 as a covariate, and treatment-by-time interaction as fixed effects and subject as random effect. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** Full Analysis Set (FAS): All randomized and treated participants with at least one post-baseline value. **Reporting Status:** POSTED **Time Frame:** Baseline and Week 26 **Title:** Percentage of Responders (Participants With a ≥20% Improvement in UPDRS2+3) **Type:** SECONDARY **Unit of Measure:** Percentage of Responders ##### Group **Description:** Participants received preladenant 2 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 2 mg oral tablet in PM for 26 weeks. The number of participants analyzed (195) represents the number of randomized and treated participants with at least one post-treatment value. **ID:** OG000 **Title:** Preladenant 2 mg (Part 1) ##### Group **Description:** Participants received preladenant 5 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 5 mg in the PM for 26 weeks. The number of participants analyzed (202) represents the number of randomized and treated participants with at least one post-treatment value. **ID:** OG001 **Title:** Preladenant 5 mg (Part 1) ##### Group **Description:** Participants received preladenant 10 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 10 mg in the PM for 26 weeks. The number of participants analyzed (200) represents the number of randomized and treated participants with at least one post-treatment value. **ID:** OG002 **Title:** Preladenant 10 mg (Part 1) ##### Group **Description:** Participants received placebo for preladenant and placebo for rasagiline in the AM followed by placebo for preladenant in the PM for 26 weeks. The number of participants analyzed (195) represents the number of randomized and treated participants with at least one post-treatment value. **ID:** OG003 **Title:** Placebo (Part 1) ##### Group **Description:** Participants received rasagiline 1 mg oral capsule and placebo for preladenant in the AM followed by placebo for preladenant in PM for 26 weeks. The number of participants analyzed (195) represents the number of randomized and treated participants with at least one post-treatment value. **ID:** OG004 **Title:** Rasagiline (Part 1) #### Outcome Measure 3 **Description:** The UPDRS is a clinician based rating scale used to measure motor impairments and disability. The UPDRS assesses six features of PD impairment. These are evaluated using a combination of data collected by interview and examination of the participant. The UPDRS Part 2 is the Activities of Daily Living (ADL) score and can range from 0-52 as determined by the physician with the higher score indicating the worse condition. Change from baseline was analyzed using a cLDA model with treatment, time, strata and treatment-by-time interaction as fixed effects and participant as a random effect. **Dispersion Type:** Standard Error **Parameter Type:** MEAN **Population Description:** Full Analysis Set (FAS): All randomized and treated participants with at least one post-baseline value. **Reporting Status:** POSTED **Time Frame:** Baseline and Week 26 **Title:** Change From Baseline in the UPDRS Part 2 Score (Activities of Daily Living [ADL]) **Type:** SECONDARY **Unit of Measure:** Score on a Scale ##### Group **Description:** Participants received preladenant 2 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 2 mg oral tablet in PM for 26 weeks. **ID:** OG000 **Title:** Preladenant 2 mg (Part 1) ##### Group **Description:** Participants received preladenant 5 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 5 mg in the PM for 26 weeks. **ID:** OG001 **Title:** Preladenant 5 mg (Part 1) ##### Group **Description:** Participants received preladenant 10 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 10 mg in the PM for 26 weeks. **ID:** OG002 **Title:** Preladenant 10 mg (Part 1) ##### Group **Description:** Participants received placebo for preladenant and placebo for rasagiline in the AM followed by placebo for preladenant in the PM for 26 weeks. **ID:** OG003 **Title:** Placebo (Part 1) ##### Group **Description:** Participants received rasagiline 1 mg oral capsule and placebo for preladenant in the AM followed by placebo for preladenant in PM for 26 weeks (Part 1) and then for another 26 weeks (Part 2). **ID:** OG004 **Title:** Rasagiline (Part 1) #### Outcome Measure 4 **Description:** An adverse event (AE) is defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to this medicinal product. **Parameter Type:** NUMBER **Population Description:** All Participants as Treated: All participants who received at least one dose of study treatment. **Reporting Status:** POSTED **Time Frame:** Day 1 to Week 26 **Title:** Number of Participants With Adverse Events (AEs) in Part 1 **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Participants received preladenant 2 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 2 mg oral tablet in PM for 26 weeks. **ID:** OG000 **Title:** Preladenant 2 mg (Part 1) ##### Group **Description:** Participants received preladenant 5 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 5 mg in the PM for 26 weeks. **ID:** OG001 **Title:** Preladenant 5 mg (Part 1) ##### Group **Description:** Participants received preladenant 10 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 10 mg in the PM for 26 weeks. **ID:** OG002 **Title:** Preladenant 10 mg (Part 1) ##### Group **Description:** Participants received placebo for preladenant and placebo for rasagiline in the AM followed by placebo for preladenant in the PM for 26 weeks. **ID:** OG003 **Title:** Placebo (Part 1) ##### Group **Description:** Participants received rasagiline 1 mg oral capsule and placebo for preladenant in the AM followed by placebo for preladenant in PM for 26 weeks. **ID:** OG004 **Title:** Rasagiline (Part 1) #### Outcome Measure 5 **Description:** An AE is defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to this medicinal product. **Parameter Type:** NUMBER **Population Description:** All Participants as Treated: All participants who received at least one dose of study treatment. **Reporting Status:** POSTED **Time Frame:** Day 1 to Week 26 **Title:** Number of Participants Who Discontinued Study Due to an AE in Part 1 **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Participants received preladenant 2 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 2 mg oral tablet in PM for 26 weeks. **ID:** OG000 **Title:** Preladenant 2 mg (Part 1) ##### Group **Description:** Participants received preladenant 5 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 5 mg in the PM for 26 weeks. **ID:** OG001 **Title:** Preladenant 5 mg (Part 1) ##### Group **Description:** Participants received preladenant 10 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 10 mg in the PM for 26 weeks. **ID:** OG002 **Title:** Preladenant 10 mg (Part 1) ##### Group **Description:** Participants received placebo for preladenant and placebo for rasagiline in the AM followed by placebo for preladenant in the PM for 26 weeks. **ID:** OG003 **Title:** Placebo (Part 1) ##### Group **Description:** Participants received rasagiline 1 mg oral capsule and placebo for preladenant in the AM followed by placebo for preladenant in PM for 26 weeks. **ID:** OG004 **Title:** Rasagiline (Part 1) #### Outcome Measure 6 **Description:** An adverse event (AE) is defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to this medicinal product. **Parameter Type:** NUMBER **Population Description:** All Participants as Treated: All participants who received at least one dose of study treatment. **Reporting Status:** POSTED **Time Frame:** Week 27 to Week 52 **Title:** Number of Participants With Adverse Events (AEs) in Part 2 **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Participants received preladenant 2 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 2 mg oral tablet in PM for 26 weeks. **ID:** OG000 **Title:** Preladenant 2 mg (Part 2) ##### Group **Description:** Participants received preladenant 5 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 5 mg in the PM for 26 weeks. **ID:** OG001 **Title:** Preladenant 5 mg (Part 2) ##### Group **Description:** Participants received preladenant 10 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 10 mg in the PM for 26 weeks. **ID:** OG002 **Title:** Preladenant 10 mg (Part 2) ##### Group **Description:** Participants who had received placebo to preladenant in Part 1 received preladenant 5 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 5 mg in the PM for 26 weeks. **ID:** OG003 **Title:** Placebo (Part 2) ##### Group **Description:** Participants received rasagiline 1 mg oral capsule and placebo for preladenant in the AM followed by placebo for preladenant in PM for 26 weeks. **ID:** OG004 **Title:** Rasagiline (Part 2) #### Outcome Measure 7 **Description:** An AE is defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to this medicinal product. **Parameter Type:** NUMBER **Population Description:** All Participants as Treated (APaT): All participants who received at least one dose of study treatment. **Reporting Status:** POSTED **Time Frame:** Week 27 to Week 52 **Title:** Number of Participants Who Discontinued Study Due to an AE in Part 2 **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Participants received preladenant 2 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 2 mg oral tablet in PM for 26 weeks. **ID:** OG000 **Title:** Preladenant 2 mg (Part 2) ##### Group **Description:** Participants received preladenant 5 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 5 mg in the PM for 26 weeks. **ID:** OG001 **Title:** Preladenant 5 mg (Part 2) ##### Group **Description:** Participants received preladenant 10 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 10 mg in the PM for 26 weeks. **ID:** OG002 **Title:** Preladenant 10 mg (Part 2) ##### Group **Description:** Participants who had received placebo to preladenant in Part 1 received preladenant 5 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 5 mg in the PM for 26 weeks. **ID:** OG003 **Title:** Placebo (Part 2) ##### Group **Description:** Participants received rasagiline 1 mg oral capsule and placebo for preladenant in the AM followed by placebo for preladenant in PM for 26 weeks. **ID:** OG004 **Title:** Rasagiline (Part 2) ### Participant Flow Module #### Group **Description:** Participants received preladenant 2 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 2 mg oral tablet in PM for 26 weeks (Part 1) and then for another 26 weeks (Part 2). **ID:** FG000 **Title:** Preladenant 2 mg #### Group **Description:** Participants received preladenant 5 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 5 mg in the PM for 26 weeks (Part 1) and then for another 26 weeks (Part 2). **ID:** FG001 **Title:** Preladenant 5 mg #### Group **Description:** Participants received preladenant 10 mg oral tablet and placebo for rasagiline in the AM followed by preladenant 10 mg in the PM for 26 weeks (Part 1) and then for another 26 weeks (Part 2). **ID:** FG002 **Title:** Preladenant 10 mg #### Group **Description:** Participants received placebo for preladenant and placebo for rasagiline in the AM followed by placebo for preladenant in the PM for 26 weeks (Part 1); preladenant 5 mg was taken twice daily for 26 weeks (Part 2). **ID:** FG003 **Title:** Placebo #### Group **Description:** Participants received rasagiline 1 mg oral capsule and placebo for preladenant in the AM followed by placebo for preladenant in PM for 26 weeks (Part 1) and then for another 26 weeks (Part 2). **ID:** FG004 **Title:** Rasagiline #### Period **Title:** Part I ##### Withdraw **Type:** Adverse Event ###### Reason **Group ID:** FG000 **Number of Subjects:** 13 ###### Reason **Group ID:** FG001 **Number of Subjects:** 8 ###### Reason **Group ID:** FG002 **Number of Subjects:** 18 ###### Reason **Group ID:** FG003 **Number of Subjects:** 7 ###### Reason **Group ID:** FG004 **Number of Subjects:** 6 ##### Withdraw **Type:** Administrative ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 2 ###### Reason **Group ID:** FG002 **Number of Subjects:** 2 ###### Reason **Group ID:** FG003 **Number of Subjects:** 0 ###### Reason **Group ID:** FG004 **Number of Subjects:** 0 ##### Withdraw **Type:** Did Not Meet Protocol Eligibility ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ###### Reason **Group ID:** FG002 **Number of Subjects:** 2 ###### Reason **Group ID:** FG003 **Number of Subjects:** 1 ###### Reason **Group ID:** FG004 **Number of Subjects:** 3 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 11 ###### Reason **Group ID:** FG001 **Number of Subjects:** 13 ###### Reason **Group ID:** FG002 **Number of Subjects:** 8 ###### Reason **Group ID:** FG003 **Number of Subjects:** 8 ###### Reason **Group ID:** FG004 **Number of Subjects:** 9 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ###### Reason **Group ID:** FG002 **Number of Subjects:** 1 ###### Reason **Group ID:** FG003 **Number of Subjects:** 1 ###### Reason **Group ID:** FG004 **Number of Subjects:** 0 ##### Withdraw **Type:** Treatment Failure ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ###### Reason **Group ID:** FG002 **Number of Subjects:** 3 ###### Reason **Group ID:** FG003 **Number of Subjects:** 1 ###### Reason **Group ID:** FG004 **Number of Subjects:** 2 ##### Withdraw **Type:** Did Not Receive Treatment ###### Reason **Group ID:** FG000 **Number of Subjects:** 4 ###### Reason **Group ID:** FG001 **Number of Subjects:** 2 ###### Reason **Group ID:** FG002 **Number of Subjects:** 2 ###### Reason **Group ID:** FG003 **Number of Subjects:** 6 ###### Reason **Group ID:** FG004 **Number of Subjects:** 1 ##### Withdraw **Type:** Non-Compliance with Protocol ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ###### Reason **Group ID:** FG002 **Number of Subjects:** 3 ###### Reason **Group ID:** FG003 **Number of Subjects:** 3 ###### Reason **Group ID:** FG004 **Number of Subjects:** 2 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 204 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 204 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 206 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 204 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 204 ##### Milestone **Type:** Treated ###### Achievement **Group ID:** FG000 **Number of Subjects:** 200 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 202 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 204 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 198 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 203 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 166 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 177 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 167 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 177 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 181 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 38 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 27 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 39 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 27 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 23 #### Period **Title:** Part II ##### Withdraw **Type:** Adverse Event ###### Reason **Group ID:** FG000 **Number of Subjects:** 7 ###### Reason **Group ID:** FG001 **Number of Subjects:** 6 ###### Reason **Group ID:** FG002 **Number of Subjects:** 8 ###### Reason **Group ID:** FG003 **Number of Subjects:** 3 ###### Reason **Group ID:** FG004 **Number of Subjects:** 4 ##### Withdraw **Type:** Administrative ###### Reason **Group ID:** FG000 **Number of Subjects:** 43 ###### Reason **Group ID:** FG001 **Number of Subjects:** 49 ###### Reason **Group ID:** FG002 **Number of Subjects:** 36 ###### Reason **Group ID:** FG003 **Number of Subjects:** 38 ###### Reason **Group ID:** FG004 **Number of Subjects:** 46 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 8 ###### Reason **Group ID:** FG001 **Number of Subjects:** 4 ###### Reason **Group ID:** FG002 **Number of Subjects:** 12 ###### Reason **Group ID:** FG003 **Number of Subjects:** 7 ###### Reason **Group ID:** FG004 **Number of Subjects:** 3 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ###### Reason **Group ID:** FG002 **Number of Subjects:** 0 ###### Reason **Group ID:** FG003 **Number of Subjects:** 0 ###### Reason **Group ID:** FG004 **Number of Subjects:** 1 ##### Withdraw **Type:** Treatment Failure ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ###### Reason **Group ID:** FG002 **Number of Subjects:** 2 ###### Reason **Group ID:** FG003 **Number of Subjects:** 0 ###### Reason **Group ID:** FG004 **Number of Subjects:** 1 ##### Withdraw **Type:** Non-Compliance with Protocol ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ###### Reason **Group ID:** FG002 **Number of Subjects:** 0 ###### Reason **Group ID:** FG003 **Number of Subjects:** 2 ###### Reason **Group ID:** FG004 **Number of Subjects:** 0 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 166 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 177 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 167 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 177 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 181 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 107 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 116 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 109 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 127 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 126 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 59 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 61 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 58 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 50 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 55 **Pre-Assignment Details:** In Part 1, participants were randomized to one of five treatment groups and treated for 26 weeks. In Part 2, which was conducted for an additional 26 weeks, participants continued taking the same study treatment from Part 1, except for placebo participants who were re-assigned to receive preladenant 5 mg twice daily. **Recruitment Details:** Participants with a diagnosis of idiopathic PD for less than 5 years were selected to participate in this study. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT05297279 **Brief Title:** OMEGA - Dietary Intervention - COPD Trial **Official Title:** OMEGA - Dietary Intervention - COPD Trial #### Organization Study ID Info **ID:** IRB00302442 #### Organization **Class:** OTHER **Full Name:** Johns Hopkins University ### Status Module #### Completion Date **Date:** 2026-09 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-03-21 **Type:** ACTUAL **Last Update Submit Date:** 2024-03-20 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2026-03 **Type:** ESTIMATED #### Start Date **Date:** 2022-03-01 **Type:** ACTUAL **Status Verified Date:** 2024-03 #### Study First Post Date **Date:** 2022-03-28 **Type:** ACTUAL **Study First Submit Date:** 2022-03-16 **Study First Submit QC Date:** 2022-03-25 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Heart, Lung, and Blood Institute (NHLBI) #### Lead Sponsor **Class:** OTHER **Name:** Johns Hopkins University #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** A randomized controlled trial of a food delivery dietary intervention targeting increased omega-3 intake to determine whether dietary modifications can improve Chronic Obstructive Pulmonary Disease (COPD) outcomes and attenuate the adverse effects of particulate matter on respiratory health. Investigators believe that study results will comprehensively address the impact of an evidence-based nutrition intervention on COPD health and provide a framework for dietary intervention within other chronic diseases disproportionately impacting susceptible, low-income populations. **Detailed Description:** COPD is a leading cause of death in the US with low-income individuals experiencing increased prevalence and morbidity. Poor dietary intake is also prevalent in low-income communities and has been associated with adverse outcomes in populations with respiratory disease. Data generated from the investigators Johns Hopkins NIH/EPA funded Environmental Health Disparities Center was instrumental in showing that poor dietary patterns, and in particular low omega-3 polyunsaturated fatty acid intake, are prevalent in a low income population with COPD. Low omega-3 intake was associated with poor respiratory outcomes and exacerbated the adverse effects of indoor air pollution exposure on respiratory symptoms. Accordingly, diet likely represents an important modifiable risk factor in low income individuals with COPD. Investigators proposed a 12-week (3 months) randomized controlled intervention trial of a home food delivery dietary intervention targeting increased omega-3 intake to determine whether dietary modifications can improve COPD outcomes and attenuate the adverse effects of particulate matter (PM) on respiratory health. Willing participants with low omega-3 intake assessed during the screening visit will have 1:1 randomization to one of the treatment arms. Outcomes will be assessed monthly and dietary intake will be assessed at 3 months. After the 12-week (3 months) intervention, participants will be followed for an additional 3 months to assess sustainability of the intervention. Participants will be part of this study for 6 months. The proposed research represents new lines of investigation to test a dietary intervention aimed at: (1) improving respiratory health, and (2) protecting against adverse effects of environmental exposures in low-income adults with COPD. Investigators will also explore barriers and facilitators of the intervention in order to optimize the sustainability of future implementation strategies. ### Conditions Module **Conditions:** - COPD, Chronic Obstructive Pulmonary Disease ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** OTHER #### Enrollment Info **Count:** 200 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants will receive weekly food voucher (with a specified amount) for home delivery of omega-3 rich food (with a minimum of 4 grams of eicosapentaenoic acid (EPA) + docosahexaenoic acid (DHA) in the weekly food order) and personalized dietary coaching. Participant will also receive a single 1-hour one-on-one session (dietary motivational coaching) by a dietary coach to guide participants to consume at least 500 mg of EPA+DHA daily at the beginning of the study, followed by weekly 30-minute calls during the 12-week intervention study period. **Intervention Names:** - Behavioral: Home delivery Omega-3 rich Food - Behavioral: Dietary Motivational Coaching **Label:** Active/Intervention Treatment Arm **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Participants will receive a voucher of weekly food voucher (with a specified amount) for home delivery. Participants in the control arm will also receive a single one-on-one session by a trained research staff member at the beginning of the study, which will be followed by weekly 30-minute calls with the participant during the 12-week intervention study period. The trained research staff member will assist with the online ordering of foods and will provide calls centered on general publicly available, guideline-based dietary recommendations without tailoring or personalization (no dietary coaching). This group will not receive guidance specifically about omega-3 fatty acids. **Intervention Names:** - Behavioral: Home delivery Food **Label:** Control Treatment Arm **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Active/Intervention Treatment Arm **Description:** The active treatment arm will receive voucher (with a specified amount) weekly home delivery omega-3 rich food for the 12-weeks (3 months) intervention. Participants will be limited to purchasing foods rich in omega-3 only. **Name:** Home delivery Omega-3 rich Food **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - Active/Intervention Treatment Arm **Description:** The active treatment arm will receive weekly personalize dietary couching by a dietary coach on omega-3 rich food for the 12-weeks intervention. Participant will also receive educational materials and guidance about what kind of food to order, how to cook, prepare (recipes) and store omega-3 rich foods. **Name:** Dietary Motivational Coaching **Type:** BEHAVIORAL #### Intervention 3 **Arm Group Labels:** - Control Treatment Arm **Description:** The control treatment arm will receive voucher (with a specified amount) weekly home delivery food (any kind of food) for the 12-weeks (3 months) intervention. Participant will not receive dietary coaching on food choices and will not be limited to purchasing foods rich in omega-3. **Name:** Home delivery Food **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** COPD health status will be assessed with the COPD assessment test (CAT). The total score is from 0 to 40. Higher scores indicate worse COPD control. **Measure:** Change in COPD health status as assessed by the COPD assessment test **Time Frame:** Baseline, months 1 and 2 follow up calls, 3 months and 6 months **Description:** The Clinical COPD Questionnaire (CCQ) health-related quality of life questionnaire (HRQoL) with good psychometric properties. The CCQ consists of 10 items with an overall score and 3 domains: Symptoms (4 items), Functional state (4 items) and Mental state (2 items). High scores indicate worse quality of life. **Measure:** Change in COPD health status/health-related quality of life as assessed by the Clinical COPD Questionnaire health-related quality of life questionnaire **Time Frame:** Baseline, months 1 and 2 follow up calls, 3 months and 6 months #### Secondary Outcomes **Description:** Investigators will collect participant's self-report number of exacerbation episodes due to moderate and/or severe COPD-related exacerbation including need for oral corticosteroids or antibiotics for worsening respiratory symptoms, emergency department (ED) visit or hospitalization. **Measure:** Change in the number of exacerbation episodes reported due to moderate and/or severe COPD-related exacerbations **Time Frame:** Baseline, months 1 and 2 follow up calls, 3 months and 6 months **Description:** Pulmonary function testing will be assessed as FEV1, that is FEV1 (adjusted for age, height, race and sex) according to the American Thoracic Society (ATS) guidelines. **Measure:** Change in Lung Function as assessed by Forced Expiratory Volume in the First Second (FEV1) **Time Frame:** Baseline, 3 months and 6 months **Description:** Pulmonary function testing will be assessed as FEV1% predicted. **Measure:** Change in Lung Function as assessed by Forced Expiratory Volume in the First Second (FEV1) percent predicted **Time Frame:** Baseline, 3 months and 6 months **Description:** The Six-Minute Walk Distance (6MWD) is a test used as a measure of exercise capacity and functional status of participants. **Measure:** Change in exercise capacity as assessed by the Six-Minute Walk Distance **Time Frame:** Baseline, 3 months and 6 months **Description:** St. George's Respiratory Questionnaire (SGRQ). The total score is from 0 to 100. Higher scores indicate more limitations. **Measure:** Change in Quality of Life as assessed by the St. George's Respiratory Questionnaire **Time Frame:** Baseline, 3 months and 6 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age ≥ 40 years, * Physician diagnosis of COPD, * Global Initiative for Obstructive Lung Disease (GOLD) Stage II-IV disease with Forced Expiratory Volume in the First Second (FEV1)/ Forced Vital Capacity (FVC) \<70% and FEV1 (% predicted) \<80%. IF FEV1/FVC \<70% and FEV1 (% predicted) ≥ 80%, additional requirement will be asked: CAT score ≥ 10. Also, IF available for screening purposes: participant can provide a previous pulmonary function testing (PFT) report within the last 6 months. * Tobacco exposure ≥ 10 pack-years, * Poverty criteria as determined by residing in a neighborhood with ≥10% of residents living in poverty, consistent with the definition of poverty area OR not access to private health insurance, OR only completed high school education or less. * Low omega-3 intake (reported daily intake of EPA+DHA intake \<500 mg via diet and/or supplement) at the screening visit, and * Willing to comply with dietary recommendations Exclusion Criteria: * Participant planning to change residence during study period, * Other chronic lung disease, except those with history of asthma if it felt by the investigator not to be a primary diagnosis. * Pregnancy or breastfeeding and * Reported unwillingness to eat seafood. **Minimum Age:** 40 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** wlorizi1@jhmi.edu **Name:** Wendy Lorizio, MD, MPH **Phone:** 410-550-2449 **Role:** CONTACT **Contact 2:** **Email:** mmaly1@jhmi.edu **Name:** Maggie Maly **Phone:** 410-550-9527 **Role:** CONTACT #### Locations **Location 1:** **City:** Baltimore **Contacts:** ***Contact 1:*** - **Email:** mmaly1@jhmi.edu - **Name:** Maggie Maly - **Phone:** 410-550-9527 - **Role:** CONTACT ***Contact 2:*** - **Name:** Nadia Hansel, MD, MPH - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Johns Hopkins Bayview Campus **State:** Maryland **Status:** RECRUITING **Zip:** 21224 #### Overall Officials **Official 1:** **Affiliation:** Johns Hopkins University **Name:** Nadia Hansel, MD, MPH **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000002908 - Term: Chronic Disease - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M11170 - Name: Lung Diseases, Obstructive - Relevance: HIGH - As Found: Obstructive Pulmonary Disease - ID: M23449 - Name: Pulmonary Disease, Chronic Obstructive - Relevance: HIGH - As Found: Chronic Obstructive Pulmonary Disease - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M6147 - Name: Chronic Disease - Relevance: LOW - As Found: Unknown - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000008173 - Term: Lung Diseases, Obstructive - ID: D000029424 - Term: Pulmonary Disease, Chronic Obstructive ### Intervention Browse Module - Browse Branches - Abbrev: Ot - Name: Other Dietary Supplements - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: T415 - Name: Omega 3 Fatty Acid - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04896879 **Brief Title:** Experiences and Needs of Patients, Their Informal Caregiver and Healthcare Professionals Regarding LARS **Official Title:** Rectal Cancer Survivorship, the Struggle of the Low Anterior Resection Syndrome (LARS). Experiences and Needs of Patients, Their Informal Caregiver and Healthcare Professionals #### Organization Study ID Info **ID:** EC2016/1016 #### Organization **Class:** OTHER **Full Name:** University Hospital, Ghent ### Status Module #### Completion Date **Date:** 2020-12-31 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-05-21 **Type:** ACTUAL **Last Update Submit Date:** 2021-05-20 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-12-31 **Type:** ACTUAL #### Start Date **Date:** 2016-11-22 **Type:** ACTUAL **Status Verified Date:** 2021-05 #### Study First Post Date **Date:** 2021-05-21 **Type:** ACTUAL **Study First Submit Date:** 2020-12-31 **Study First Submit QC Date:** 2021-05-20 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Kom Op Tegen Kanker **Class:** OTHER **Name:** AZ Sint-Jan AV **Class:** OTHER **Name:** AZ Delta **Class:** OTHER **Name:** AZ Sint-Lucas Gent #### Lead Sponsor **Class:** OTHER **Name:** University Hospital, Ghent #### Responsible Party **Investigator Affiliation:** University Hospital, Ghent **Investigator Full Name:** GIHeelkunde **Investigator Title:** Principal investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Exploration of the experiences and needs of patients with the low anterior resection syndrome, their informal caregiver and health care professionals. This is a qualitative study where semi-structured interviews will be conducted with patients and informal caregivers. next to that focus groups will be performed with healthcare professionals from different professions. ### Conditions Module **Conditions:** - Low Anterior Resection Syndrome - Rectal Cancer **Keywords:** - low anterior resection syndrome ### Design Module #### Design Info **Observational Model:** OTHER **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 69 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients with major LARS **Intervention Names:** - Behavioral: interviews **Label:** Patients #### Arm Group 2 **Description:** Informal caregivers of patients with major LARS **Intervention Names:** - Behavioral: interviews **Label:** Informal caregivers #### Arm Group 3 **Description:** Healthcare professionals taking care for patients with LARS **Intervention Names:** - Behavioral: interviews **Label:** Healthcare professionals ### Interventions #### Intervention 1 **Arm Group Labels:** - Healthcare professionals - Informal caregivers - Patients **Description:** semi-structured interviews will be conducted in patients with major LARS and their informal caregiver to explore their experiences and needs. Focus groups will be perfomed with healthcare professionals of several professions. **Name:** interviews **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** Exploring needs and experiences of patients with major lars and their informal caregiver through individual semistructured interviews **Measure:** needs and experiences of patients with major lars and their informal caregiver through individual semistructered interviews **Time Frame:** Interview will last about 60 minutes **Description:** Exploring needs and experiences of healthcare professionals caring for patients with LARS **Measure:** Needs and experiences of healthcare professionals caring for patients with LARS through focus groups **Time Frame:** Focus group will last about 60 minutes ### Eligibility Module **Eligibility Criteria:** PATIENTS Inclusion Criteria: * adult patients (18+) * confronted with major low anterior resection syndrome after rectal cancer surgery Exclusion Criteria: * stoma at time interview * abdominal perineal resection INFORMAL CAREGIVERS Inclusion criteria * Adults (18+) * Nominated by the participating patient as their principal caregiver Exclusion criteria None HEALTHCARE PROFESSIONALS inclusion criteria * Adults (18+) * Caring for patients with LARS Exclusion criteria None **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** The participants will be patients with major LARS, their informal caregivers and healthcare professionals ### References Module #### References **Citation:** Pape E, Decoene E, Debrauwere M, Van Nieuwenhove Y, Pattyn P, Feryn T, Pattyn PRL, Verhaeghe S, Van Hecke A; Belgian LARS collaborative group. Information and counselling needs of patients with major low anterior resection syndrome: A qualitative study. J Clin Nurs. 2023 Apr;32(7-8):1240-1250. doi: 10.1111/jocn.16277. Epub 2022 Mar 6. **PMID:** 35253296 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004194 - Term: Disease - ID: D000010335 - Term: Pathologic Processes - ID: D000015179 - Term: Colorectal Neoplasms - ID: D000007414 - Term: Intestinal Neoplasms - ID: D000005770 - Term: Gastrointestinal Neoplasms - ID: D000004067 - Term: Digestive System Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000004066 - Term: Digestive System Diseases - ID: D000005767 - Term: Gastrointestinal Diseases - ID: D000007410 - Term: Intestinal Diseases - ID: D000012002 - Term: Rectal Diseases - ID: D000003108 - Term: Colonic Diseases - ID: D000011183 - Term: Postoperative Complications ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: BC04 - Name: Neoplasms ### Condition Browse Module - Browse Leaves - ID: M16355 - Name: Syndrome - Relevance: HIGH - As Found: Syndrome - ID: M3018 - Name: Low Anterior Resection Syndrome - Relevance: HIGH - As Found: Low Anterior Resection Syndrome - ID: M14846 - Name: Rectal Neoplasms - Relevance: HIGH - As Found: Rectal Cancer - ID: M17890 - Name: Colorectal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M10448 - Name: Intestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M8886 - Name: Gastrointestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7256 - Name: Digestive System Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M10444 - Name: Intestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M14844 - Name: Rectal Diseases - Relevance: LOW - As Found: Unknown - ID: M6336 - Name: Colonic Diseases - Relevance: LOW - As Found: Unknown - ID: M14065 - Name: Postoperative Complications - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000012004 - Term: Rectal Neoplasms - ID: D000094123 - Term: Low Anterior Resection Syndrome - ID: D000013577 - Term: Syndrome ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02993679 **Brief Title:** Stability of the Knee Joint After Anterior Cruciate Ligament and Anterolateral Ligament Reconstruction **Official Title:** Biomechanical Assessment of the Reconstruction of the Anterolateral Ligament During ACL Surgery. The In-vivo Study #### Organization Study ID Info **ID:** Hospital Znojmo #### Organization **Class:** OTHER **Full Name:** Hospital Znojmo ### Status Module #### Completion Date **Date:** 2016-11 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2016-12-15 **Type:** ESTIMATED **Last Update Submit Date:** 2016-12-14 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2016-07 **Type:** ACTUAL #### Start Date **Date:** 2014-07 **Status Verified Date:** 2016-12 #### Study First Post Date **Date:** 2016-12-15 **Type:** ESTIMATED **Study First Submit Date:** 2016-12-04 **Study First Submit QC Date:** 2016-12-14 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Hospital Znojmo #### Responsible Party **Investigator Affiliation:** Hospital Znojmo **Investigator Full Name:** Komzak Martin, M.D. **Investigator Title:** General Hospital Znojmo **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The objective of this randomised cohort study was to evaluate the knee rotational stability after the single-bundle ACL reconstruction (SB) with addition of the ALL reconstruction and to compare it with the double-bundle ACL reconstruction technique (DB) and to analyse when the ALL is necessary to reconstruct. **Detailed Description:** Background Rotational instability after the anterior cruciate ligament (ACL) injury and subsequent reconstruction may be caused by the rupture of the anterolateral structures of the knee, specifically the anterolateral ligament (ALL). There are more techniques to improve the rotational stability of the knee. The objective of this randomised cohort study was: (1) To evaluate the knee rotational stability after the single-bundle ACL reconstruction (SB) with addition of the ALL reconstruction and to compare it with the double-bundle ACL reconstruction technique (DB). (2) To analyse when the ALL is necessary to reconstruct. Methods 60 patients underwent the ACL reconstruction with the average age of 29.5 years. In thirty patient's knees the ACL was replaced with quadriceps muscle graft using the SB technique in combination with the ALL reconstruction by the gracilis graft (ALL group). With another thirty patients the ACL was reconstructed performing DB technique with the use of hamstring tendons (DB group). The rotational stability was studied before and after the reconstruction of the ACL in time "zero" using the computer navigation system. In the ALL group, the rotational stability was also analysed after the ALL reconstruction. ### Conditions Module **Conditions:** - Cruciate Ligament Rupture ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 60 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** surgical reconstruction of the anterior cruciate ligament **Intervention Names:** - Procedure: reconstruction of the ACL and anterolateral ligament **Label:** double-bundle ACL reconstruction **Type:** OTHER #### Arm Group 2 **Description:** surgical reconstruction of the anterolateral ligament **Intervention Names:** - Procedure: reconstruction of the ACL and anterolateral ligament **Label:** anterolateral ligament reconstruction **Type:** OTHER ### Interventions #### Intervention 1 **Arm Group Labels:** - anterolateral ligament reconstruction - double-bundle ACL reconstruction **Description:** reconstruction of the ACL and anterolateral ligament **Name:** reconstruction of the ACL and anterolateral ligament **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** Analyse of the rotational stability of the knee joint 24 months after the operation by the computerised navigation system. **Measure:** Description of the change in the rotational stability of the knee joint after ACL and anterolateral ligament reconstruction. **Time Frame:** 24 months after the operation ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * The isolated ACL lesion * The absence of the previous surgery on the knee joint * The body mass index (BMI) less than 30. Exclusion Criteria: * Associated injuries of the tissue around the knee joint **Maximum Age:** 40 Years **Minimum Age:** 17 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014947 - Term: Wounds and Injuries ### Condition Browse Module - Browse Branches - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M15241 - Name: Rupture - Relevance: HIGH - As Found: Rupture - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000012421 - Term: Rupture ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01597479 **Brief Title:** Ultrasound Guided Distal Peripheral Nerve Blocks and Postoperative Pain. **Official Title:** Effectiveness of Distal Peripheral Nerve Blocks on Postoperative Pain Management After Ambulatory Thumb Resection Arthroplasty. #### Organization Study ID Info **ID:** IIBSP-LEV-2011-21 #### Organization **Class:** OTHER **Full Name:** Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau ### Status Module #### Completion Date **Date:** 2014-04 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2016-03-21 **Type:** ESTIMATED **Last Update Submit Date:** 2016-02-19 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2014-04 **Type:** ACTUAL #### Results First Post Date **Date:** 2016-03-21 **Type:** ESTIMATED **Results First Submit Date:** 2015-05-06 **Results First Submit QC Date:** 2016-02-19 #### Start Date **Date:** 2012-02 **Status Verified Date:** 2016-01 #### Study First Post Date **Date:** 2012-05-14 **Type:** ESTIMATED **Study First Submit Date:** 2012-05-07 **Study First Submit QC Date:** 2012-05-10 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The aim of this clinical trial is to determinate if distal ultrasound guided peripheral nerve blocks on target nerves (radial and median nerve blocks at the elbow), using low volume and low concentration of long acting local anesthetic provide better postoperative pain control compare with systemic analgesia alone after thumb resection arthroplasty (TRA) due to a prolonged selective sensitive block on the tissue trauma. **Detailed Description:** We designed a prospective randomized controlled trial, with nursing blinded evaluation. We enrolled 52 patients scheduled for elective ambulatory TRA. 2 patients were excluded after randomization. Patients were randomized into two groups: A. Group A= no distal peripheral nerve blocks (no dPNBs group; n = 24 ): We performed usual anesthetic technique for surgery: an AXILLARY BRAQUIAL PLEXUS BLOCK using SHORT ACTING local anesthetic (mepivacaine 1%). Patients allocated in this group didn't received any additional intervention in the postoperative period. B. Group B (dPNBs group; n = 26): We performed the same anesthetic technique for surgery (AXILLARY BLOCK with 1% of mepivacaine) with an additional intervention. Patients allocated in this group received postoperatively dPNBs on target nerves. Based on surgical approached and technique we evaluated that radial and median nerves were responsible for the innervation of the surgical area, and therefore responsible for the postoperative pain. We performed dPNBs ON RADIAL AND MEDIAN NERVES (TARGET NERVES) WITH LONG ACTING AND LOW CONCENTRATION LOCAL ANESTHETIC (0,125% levobupivacaine 5ml/nerve). All blocks were performed under ultrasound guidance. Analgesic regime prescribed at discharge was the same in both groups: dexketoprofen with tramadol for rescue analgesia. The primary outcome was to evaluate the proportion of patients experienced moderate to severe pain during first and second day postoperatively, mesured using a numerical visual scale (NVS) of 0 to 10 (0= no pain and 10= worst pain imaginable). We defined mild pain (NVS 0-3), moderate pain (NVS 7-10) and severe pain (NVS 7-10). We considered dPNBs effective when patients experienced mild pain (NVS 0-3) for at least 6 hours after dPNBs puncture. Secondary outcomes included: 1. Maximum pain intensity during first and second day postoperatively. 2. Duration of dPNBs, defined as the interval between dPNBs performance and the occurrence of first pain. 3. Time to discharge, defined as the interval since patient arrived at postoperative care unit (PACU) until discharge home. 4. Presence of distal hand motor block after dPNBs puncture. 5. Needed for rescue analgesia and total consumption of tramadol during first and second day postoperatively 6. Incidence of postoperative nausea and vomiting during 1st and 2nd day postoperatively 7. Needed for rescue antiemetic therapy, total consumption of ondansetron and effectiveness of treatment during 1st and 2nd day postoperatively. Patients were contacted by phone first and second day postoperatively from a blinded PACU nursing staff (all outcome data were collected by PACU nursing staff blinded to group allocation). ### Conditions Module **Conditions:** - Postoperative Pain **Keywords:** - Ultrasound guided distal peripheral nerve blocks ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 52 **Type:** ACTUAL **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients in this group didn't receive any intervention in the postoperative period. **Label:** No dPNBs group **Type:** NO_INTERVENTION #### Arm Group 2 **Description:** Patients in this group received ultrasound guided dPNBs on radial and median nerves (target nerves of TRA) in the postoperative period, before discharge. The procedural objective of dPNBs was to place local anesthetic around the target nerves to achieve a long lasting, sensitive and selective block in the surgical area. dPNBs were performed with 5 ml/nerve of levobupivacaine 0,125%. Ultrasound guidance allowed us to verify the correct distribution of LA around the target nerves target and optimize needle position if it was necessary, always avoiding the intraneural injection. **Intervention Names:** - Drug: Levobupivacaine **Label:** dPNBs group **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - dPNBs group **Description:** In dPNBs group, we performed ultrasound guided dPNBs on radial and median nerves using low concentration and low volume of long acting local anesthetic (0.125% levobupivacaine, 5 ml per nerve). Using low concentrations of levobupivacaine on target nerves could achieve a prolonged sensitive block in the surgical area without motor block of the hand. **Name:** Levobupivacaine **Other Names:** - long acting local anesthetics **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Pain scores assessed using pain numerical visual scale (NVS) of 0-10 (o= no pain and 10= worst pain imaginable). We defined mild pain (NVS 0-3); moderate pain (NVS 4-6) and severe pain (NVS 7-10).The analysis of this variable at the end of the study will confirm or not the effectiveness of dPNBs for management of postoperative pain after TRA. **Measure:** Proportion of Patients Who Experienced Moderate to Severe Pain During First and Second Postoperative Day **Time Frame:** Up to 48 hours #### Secondary Outcomes **Description:** Number of participants with Maximum pain intensity NVS \> 3; Rescue analgesia; Nausea and Vomiting incidence; use of ondansetron for NVPO; Ondansetron being effective (number of participants for whom ondansetron was effective to stop NVPO). **Measure:** Maximum Pain Intensity, Rescue Analgesia, Nausea and Vomiting Incidence, Use of Ondansetron for NVPO, Efectiveness of Ondansetron **Time Frame:** Up to 48 hours ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age more than 18 years. * Ambulatory TRA. * Free acceptance to participate in the study, with informed consent signed by patient, guardian or family member. Exclusion Criteria: * Age less than 18 years * Pregnancy. * Inability to provide informed consent. * Allergy to amide local anesthetics/NSAIDs * Preexisting chronic pain treated with opioids. * Neuropathy involving the extremity undergoing surgery or neurological-cognitive deficits that may interfere in the assessement. * Contraindications to dPNBs **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Barcelona **Country:** Spain **Facility:** Hospital de la Santa Creu i Sant Pau **Zip:** 08025 #### Overall Officials **Official 1:** **Affiliation:** Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau **Name:** Mireia Rodriguez, anesthesia **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ### References Module #### References **Citation:** Dufeu N, Marchand-Maillet F, Atchabahian A, Robert N, Ait Yahia Y, Milan D, Robert C, Coroir M, Beaussier M. Efficacy and safety of ultrasound-guided distal blocks for analgesia without motor blockade after ambulatory hand surgery. J Hand Surg Am. 2014 Apr;39(4):737-43. doi: 10.1016/j.jhsa.2014.01.011. Epub 2014 Feb 28. **PMID:** 24582844 **Citation:** Fredrickson MJ, Ting FS, Chinchanwala S, Boland MR. Concomitant infraclavicular plus distal median, radial, and ulnar nerve blockade accelerates upper extremity anaesthesia and improves block consistency compared with infraclavicular block alone. Br J Anaesth. 2011 Aug;107(2):236-42. doi: 10.1093/bja/aer101. Epub 2011 May 15. **PMID:** 21576095 **Citation:** Fredrickson MJ, Wolstencroft PJ, Chinchanwala S, Boland MR. Does motor block related to long-acting brachial plexus block cause patient dissatisfaction after minor wrist and hand surgery? A randomized observer-blinded trial. Br J Anaesth. 2012 Nov;109(5):809-15. doi: 10.1093/bja/aes266. Epub 2012 Aug 2. **PMID:** 22864520 **Citation:** Bouaziz H, Narchi P, Mercier FJ, Khoury A, Poirier T, Benhamou D. The use of a selective axillary nerve block for outpatient hand surgery. Anesth Analg. 1998 Apr;86(4):746-8. doi: 10.1097/00000539-199804000-00013. **PMID:** 9539595 **Citation:** Vial F, Bouaziz H, Mekler G, Cornet C, Merle M, Laxenaire MC. [Postoperative pain and surgical treatment of trapeziometacarpal osteoarthritis of the thumb after ambulatory surgery]. Ann Fr Anesth Reanim. 2000 Nov;19(9):643-8. doi: 10.1016/s0750-7658(00)00292-6. French. **PMID:** 11244701 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000011183 - Term: Postoperative Complications - ID: D000010335 - Term: Pathologic Processes - ID: D000010146 - Term: Pain - ID: D000009461 - Term: Neurologic Manifestations ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases ### Condition Browse Module - Browse Leaves - ID: M13069 - Name: Pain, Postoperative - Relevance: HIGH - As Found: Postoperative Pain - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown - ID: M14065 - Name: Postoperative Complications - Relevance: LOW - As Found: Unknown - ID: M12404 - Name: Neurologic Manifestations - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010149 - Term: Pain, Postoperative ### Intervention Browse Module - Ancestors - ID: D000002492 - Term: Central Nervous System Depressants - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018689 - Term: Sensory System Agents - ID: D000018373 - Term: Peripheral Nervous System Agents ### Intervention Browse Module - Browse Branches - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M4107 - Name: Anesthetics - Relevance: HIGH - As Found: Virus - ID: M4109 - Name: Anesthetics, Local - Relevance: HIGH - As Found: Until disease progression - ID: M1832 - Name: Levobupivacaine - Relevance: HIGH - As Found: Coronary Artery Disease ### Intervention Browse Module - Meshes - ID: D000000777 - Term: Anesthetics - ID: D000077554 - Term: Levobupivacaine - ID: D000000779 - Term: Anesthetics, Local ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Distal Peripheral Nerve Block Group **Description:** Any patient undergoing ultrasound guided distal peripheral nerve block reported any complication. **ID:** EG000 **Other Num at Risk:** 24 **Serious Number At Risk:** 24 **Title:** Distal Peripheral Nerve Block Group **Frequency Threshold:** 0 **Time Frame:** Up to 48 hours ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 24 **Group ID:** BG001 **Value:** 26 **Group ID:** BG002 **Value:** 50 **Units:** Participants ### Group **ID:** BG000 **Title:** dPNBs Group **Description:** dPNBs on radial and median nerves at the elbow in postoperative period, before discharge ### Group **ID:** BG001 **Title:** Non dPNBs Group **Description:** Non intervention in postoperative period ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 6.5 **Value:** 67.4 #### Measurement **Group ID:** BG001 **Spread:** 7.5 **Value:** 64.2 #### Measurement **Group ID:** BG002 **Spread:** 7.2 **Value:** 65.7 **Class Title:** years ### Measure #### Measurement **Group ID:** BG000 **Value:** 21 #### Measurement **Group ID:** BG001 **Value:** 21 #### Measurement **Group ID:** BG002 **Value:** 42 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 5 #### Measurement **Group ID:** BG002 **Value:** 8 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 24 #### Measurement **Group ID:** BG001 **Value:** 26 #### Measurement **Group ID:** BG002 **Value:** 50 **Class Title:** Spain **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants **Population Description:** Patients undergoing Ambulatory TRA ## Results Section - More Information Module ### Certain Agreement ### Limitations and Caveats **Description:** We could included another group: distal blocks with 0,125% levobupivacaine+ 4mg dexamethasone; register patient satisfaction with anesthetic technique or patient risks factors for nausea and vomiting. ### Point of Contact **Email:** mrodriguezpr@santpau.cat **Organization:** Hospital de la Santa Creu i Sant Pau **Phone:** 677896054 **Title:** Dra. Mireia Rodriguez Prieto ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 8.3 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 80.8 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4.2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 42.3 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 8 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 24 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 21 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 17 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 17 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 10 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Pain scores assessed using pain numerical visual scale (NVS) of 0-10 (o= no pain and 10= worst pain imaginable). We defined mild pain (NVS 0-3); moderate pain (NVS 4-6) and severe pain (NVS 7-10).The analysis of this variable at the end of the study will confirm or not the effectiveness of dPNBs for management of postoperative pain after TRA. **Parameter Type:** NUMBER **Population Description:** Patients undergoing ambulatory thumb resection arthroplasty (TRA) **Reporting Status:** POSTED **Time Frame:** Up to 48 hours **Title:** Proportion of Patients Who Experienced Moderate to Severe Pain During First and Second Postoperative Day **Type:** PRIMARY **Unit of Measure:** percentage of patients ##### Group **Description:** We practiced ultrasound guided dPNBs on radial and median nerves, after surgery, before discharge. * Distal median nerve block was performed at the elbow, in the internal bicipital channel. * Distal radial nerve block was performed at approximately the junction of the middle and distal thirds of the arm. We use 5ml per nerve of levobupivacaine 0,125% for target nerve blocks. **ID:** OG000 **Title:** Distal Peripheral Nerve Blocks Group (dPNBs Group) ##### Group **Description:** Patients in non dPNBs didn't received any intervention in the postoperatively period. **ID:** OG001 **Title:** Non Distal Peripheral Nerve Blocks (Non dPNBs Group) #### Outcome Measure 2 **Description:** Number of participants with Maximum pain intensity NVS \> 3; Rescue analgesia; Nausea and Vomiting incidence; use of ondansetron for NVPO; Ondansetron being effective (number of participants for whom ondansetron was effective to stop NVPO). **Parameter Type:** NUMBER **Population Description:** Patients undergoing ambulatory thumb resection arthroplasty **Reporting Status:** POSTED **Time Frame:** Up to 48 hours **Title:** Maximum Pain Intensity, Rescue Analgesia, Nausea and Vomiting Incidence, Use of Ondansetron for NVPO, Efectiveness of Ondansetron **Type:** SECONDARY **Unit of Measure:** participants ##### Group **Description:** we practiced ultrasound guided dPNBs on radial and median nerves using a long acting and low concentration local anesthetic (0.125% levobupivacaine, 5 ml per nerve). **ID:** OG000 **Title:** dPNBs Group ##### Group **Description:** In this group any intervention was done. **ID:** OG001 **Title:** Non dPNBs Group ### Participant Flow Module #### Group **Description:** In dPNBs group, we practice distal peripheral nerves blocks guided by ultrasound and neurostimulator. Levobupivacaine: In dPNBs group, we practice ultrasound guided distal peripheral nerve blocks on radial and median nerves using low volume and low concentration of long acting local anesthetic (0.125% levobupivacaine, 5 ml per nerve). We performed dPNBs in the postoperative period. Deffered dPNBs under the influence of axillary block didn't cause patient disconfort. We considered the technique safety due to ultrasound guidance. **ID:** FG000 **Title:** Distal Peripheral Nerve Blocks Group (dPNBs Group) #### Group **Description:** In patients of no dPNBs group didn't performed any intervention after surgery. **ID:** FG001 **Title:** No Intervention (no dPNBs Group) #### Period **Title:** Overall Study ##### Withdraw **Type:** Protocol Violation ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 26 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 26 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 24 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 26 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 2 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 **Recruitment Details:** Between february 2010 and april 2014, we enrolled 52 patients scheduled for elective ambulatory TRA under rutine axillary braquial plexus block. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT03006679 **Acronym:** TANGOIII **Brief Title:** A Study of Meropenem-Vaborbactam Versus Piperacillin/Tazobactam in Participants With Hospital-Acquired and Ventilator-Associated Bacterial Pneumonia **Official Title:** A Phase IIIb, Multicenter, Double-Blind, Randomized, Comparative Study to Evaluate the Efficacy, Safety, and Tolerability of Meropenem-Vaborbactam Versus Piperacillin/Tazobactam in the Treatment of Hospital-Acquired Bacterial Pneumonia and Ventilator-Associated Bacterial Pneumonia in Hospitalized Adults (TANGO III) #### Organization Study ID Info **ID:** Rempex 509 #### Organization **Class:** INDUSTRY **Full Name:** Melinta Therapeutics, Inc. ### Status Module #### Completion Date **Date:** 2020-12 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2019-01-11 **Type:** ACTUAL **Last Update Submit Date:** 2019-01-09 **Overall Status:** WITHDRAWN #### Primary Completion Date **Date:** 2020-06 **Type:** ESTIMATED #### Start Date **Date:** 2018-08 **Type:** ESTIMATED **Status Verified Date:** 2019-01 #### Study First Post Date **Date:** 2016-12-30 **Type:** ESTIMATED **Study First Submit Date:** 2016-12-15 **Study First Submit QC Date:** 2016-12-27 **Why Stopped:** Sponsor Decision ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Melinta Therapeutics, Inc. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to determine the efficacy, safety, tolerability, and pharmacokinetics (PK) of meropenem-vaborbactam compared to piperacillin/tazobactam for 7 to 14 days in the treatment of hospitalized adults who meet clinical, radiographic, and microbiological criteria for hospital-acquired bacterial pneumonia (HABP) or ventilator-associated bacterial pneumonia (VABP). ### Conditions Module **Conditions:** - Hospital-Acquired Bacterial Pneumonia - Ventilator-Associated Bacterial Pneumonia - Hospital-Acquired Pneumonia - Ventilator-Associated Pneumonia ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants with a clinical diagnosis of HABP will be treated with meropenem 2 grams (g) and vaborbactam 2 g in 250 milliliters (mL) infused intravenously for 3 hours every 8 hours for 7 days to a maximum of 14 days. **Intervention Names:** - Drug: Meropenem-Vaborbactam **Label:** Meropenem-Vaborbactam HABP **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants with a clinical diagnosis of HABP will be treated with piperacillin 4 g and tazobactam 0.5 g in 100 mL infused intravenously for 30 minutes every 6 hours for 7 days to a maximum of 14 days. **Intervention Names:** - Drug: Piperacillin/Tazobactam **Label:** Piperacillin/Tazobactam HABP **Type:** ACTIVE_COMPARATOR #### Arm Group 3 **Description:** Participants with a clinical diagnosis of VABP will be treated with meropenem 2 g and vaborbactam 2 g in 250 mL infused intravenously for 3 hours every 8 hours for 7 days to a maximum of 14 days. **Intervention Names:** - Drug: Meropenem-Vaborbactam **Label:** Meropenem-Vaborbactam VABP **Type:** EXPERIMENTAL #### Arm Group 4 **Description:** Participants with a clinical diagnosis of VABP will be treated with piperacillin 4 g and tazobactam 0.5 g in 100 mL infused intravenously for 30 minutes every 6 hours for 7 days to a maximum of 14 days. **Intervention Names:** - Drug: Piperacillin/Tazobactam **Label:** Piperacillin/Tazobactam VABP **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Meropenem-Vaborbactam HABP - Meropenem-Vaborbactam VABP **Description:** meropenem 2 g and vaborbactam 2 g **Name:** Meropenem-Vaborbactam **Other Names:** - Carbavance **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Piperacillin/Tazobactam HABP - Piperacillin/Tazobactam VABP **Description:** piperacillin 4 g and tazobactam 0.5 g **Name:** Piperacillin/Tazobactam **Other Names:** - Zosyn **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Rate of Participants with All-Cause Mortality at Day 28 in the Intent-to-Treat (ITT) Population **Time Frame:** Day 28 **Measure:** Percentage of Participants who Achieve a Clinical Outcome of Cure at the Test of Cure (TOC) Visit in the ITT and Clinically Evaluable (CE) Populations **Time Frame:** 12-23 days (TOC visit) after first dose of study drug #### Secondary Outcomes **Measure:** Rate of Participants with All-Cause Mortality at Day 14 in the ITT, microbiological Modified ITT (mMITT), CE, and Microbiologically Evaluable (ME) Populations **Time Frame:** Day 14 **Measure:** Rate of Participants with All-Cause Mortality at Day 28 in the mMITT, CE, and ME Populations **Time Frame:** Day 28 **Measure:** Percentage of Participants with Clinical Outcome of Cure at EOT, TOC, and Last Follow Up (LFU) Visits in the ITT, CE, and ME Populations **Time Frame:** 1 day (EOT visit), 12-23 days (TOC visit), and 19-30 days (LFU visit) after first dose of study drug **Measure:** Percentage of Participants with Clinical Outcome of Cure per Pathogen at EOT, TOC, and LFU Visits in the mMITT and ME Populations **Time Frame:** 1 day (EOT visit), 12-23 days (TOC visit), and 19-30 days (LFU visit) after first dose of study drug **Measure:** Percentage of Participants with Microbiological Eradication Per-Participant and Per-Pathogen Microbiological Response at EOT and TOC visits in the mMITT and ME Populations **Time Frame:** 1 day (EOT visit) and 12-23 days (TOC visit) after first dose of study drug **Measure:** Percentage of Participants who Survive and Did Not Have a "Major Nonfatal Event" (Treatment-Emergent Events of Acute Respiratory Distress Syndrome, Septic Shock, or Pleural Empyema) up to Day 28 in the ITT and CE Populations **Time Frame:** Up to Day 28 **Measure:** Pharmacokinetics: Area Under the Concentration-Time Curve (AUC) of Meropenem **Time Frame:** Days 1 and 3 **Measure:** Pharmacokinetics: Maximum Plasma Concentration (Cmax) of Meropenem **Time Frame:** Days 1 and 3 **Measure:** Pharmacokinetics: Time to Maximum Plasma Concentration (Tmax) of Meropenem **Time Frame:** Days 1 and 3 **Measure:** Pharmacokinetics: Area Under the Concentration-Time Curve (AUC) of Vaborbactam **Time Frame:** Days 1 and 3 **Measure:** Pharmacokinetics: Maximum Plasma Concentration (Cmax) of Vaborbactam **Time Frame:** Days 1 and 3 **Measure:** Pharmacokinetics: Time to Maximum Plasma Concentration (Tmax) of Vaborbactam **Time Frame:** Days 1 and 3 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Willingness to comply with all study procedures and provide a signed written informed consent prior to any study-specific procedures; however, if unable to do so, the participant's legally authorized representative may provide written consent as approved by institutional-specific guidelines. Participants who are unconscious or considered by the investigator to be clinically unable to consent at Screening and who are entered into the study by the consent of a legally authorized representative, should provide their own written informed consent for continuing to participate in the study as soon as possible on recovery, as applicable in accordance with local regulations. 2. Hospitalized male or female participants, ≥18 years of age. 3. Females must be surgically sterile or at least 2 years postmenopausal, or if of childbearing potential, have a negative screening urine pregnancy test and be willing to practice sexual abstinence or use an accepted form of contraception with her partner (for example, barrier or hormonal methods) during treatment and for at least 28 days after the last dose of study drug. 4. Expectation, in the opinion of the Investigator, that the participant's infection will require treatment with IV antibiotics for a minimum of 7 days. 5. Have a confirmed diagnosis of HABP or VABP requiring antibiotic therapy by meeting all clinical, microbiological, and radiographic criteria as defined in the following: For HABP participants: To meet the study definition of HABP, participants must meet all of the following clinical, microbiological, and radiographic criteria: 1. A chest radiograph (chest X-ray \[CXR\], magnetic resonance imaging \[MRI\] or computed tomography \[CT\]) reveals the presence of new or progressive pulmonary infiltrate(s) consistent with bacterial pneumonia within 48 hours prior to randomization. 2. Onset of symptoms at least 48 hours after hospitalization or within 7 days after discharge from an inpatient acute or chronic care facility (for example, long-term care, rehabilitation center, hospital, or skilled nursing home). 3. Have at least one of the following: 1. Temperature ≥38.0 degrees Celsius (100.4 degrees Fahrenheit) or ≤35 degrees Celsius (95.0 degrees Fahrenheit). 2. Peripheral white blood cell (WBC) count ≥10,000 cells/cubic millimeter (mm\^3) or ≤4,500 cells/mm\^3. 3. ≥15 percent immature neutrophils (band forms) regardless of total WBC count. 4. Have at least one of the following: 1. New onset of cough or expectorated sputum production (or worsening of baseline cough). 2. Auscultatory findings on pulmonary examination consistent with bacterial pneumonia or pulmonary consolidation (for example, rales, dullness on percussion, bronchial breath sounds, or ego phony). 3. Dyspnea or tachypnea (that is, respiratory rate greater than 25 breaths/minute). 4. Hypoxemia (O2 saturation ≤90 percent or pO2 ≤60 millimeters of mercury \[mmHg\] while breathing room air, or worsening of the O2 saturation/FiO2). 5. New onset need for mechanical ventilation. 6. A deep respiratory secretion specimen that was collected within 48 hours prior to randomization and after development of clinical signs and symptoms of HABP (ideally prior to administration of systemic antimicrobial therapy). This can be obtained via a sputum sample (expectorated sputum), bronchoalveolar lavage (BAL) (including protected BAL or mini-BAL), protected specimen brush (PSB), endotracheal tube aspirate (ETA), pleural fluid, or lung parenchyma (open-lung, transthoracic, or transbronchial biopsy). 7. This deep respiratory secretion sample must meet adequacy criteria for testing, and be sent to the local or regional laboratory for Gram stain and culture (results of Gram stain and culture do not have to be available for enrollment). For VABP participants: To meet the study definition of VABP, participants must meet all of the following clinical, microbiological, and radiographic criteria: 1. A chest radiograph (CXR, MRI or CT) revealing the presence of new or progressive pulmonary infiltrate(s) consistent with bacterial pneumonia within 48 hours prior to randomization. 2. Receiving mechanical ventilation via endotracheal intubation or tracheostomy for greater than or equal to 48 hours. 3. Have at least one of the following: 1. Temperature ≥38.0 degrees Celsius (100.4 degrees Fahrenheit) or ≤35 degrees Celsius (95.0 degrees Fahrenheit). 2. Peripheral white blood cell (WBC) count ≥10,000 cells/mm\^3 or ≤4,500 cells/mm\^3. 3. ≥15 percent immature neutrophils (band forms) regardless of total WBC count. 4. Have at least one of the following: 1. New onset of purulent respiratory secretions from the lungs or new onset of or increased need for respiratory secretion suctioning. 2. Auscultatory findings on pulmonary examination consistent with bacterial pneumonia or pulmonary consolidation (for example, rales, dullness on percussion, bronchial breath sounds, or egophony). 3. Worsening gas exchange (ratio of partial pressure of arterial oxygen to fraction of inspired oxygen \[PaO2/FiO2\] ≤240 or PaO2 ≤60 mmHg) leading to acute changes to the ventilator support system (minimum daily FiO2 values increased by at least 0.20 over the daily minimum FiO2 in the preceding 48 hours, or minimum daily positive end- expiratory pressure (PEEP) values increased by at least 3 centimeters (cm) H2O over the daily minimum PEEP in the preceding 48 hours). 5. A deep respiratory secretion specimen must be collected within 48 hours prior to randomization and after development of clinical signs and symptoms of VABP (ideally prior to administration of systemic antimicrobial therapy). This can be obtained via BAL (including protected BAL or mini-BAL), PSB, or ETA, pleural fluid, or lung parenchyma (open-lung, transthoracic, or transbronchial biopsy). 6. The above deep respiratory secretion sample must meet adequacy criteria for testing and be sent to the local or regional laboratory for Gram stain and culture (results of Gram stain and culture do not have to be available for enrollment). Exclusion Criteria: Participants who meet any of the following exclusion criteria will not be enrolled in the study: 1. History of any severe hypersensitivity to any beta-lactam antibiotic (for example, cephalosporins, penicillins, or carbapenems). 2. History of any severe allergic reaction that would preclude the use of either all aminoglycosides or adjunctive gram-positive antimicrobials (that is, allergy to both glycopeptides and oxazolidinones). 3. Requirement or anticipated need for additional systemic antibiotic (other than study drug) or antifungal, including prophylactic antimicrobials and antifungals. 4. Requirement or anticipated need for more than 14 days of systemic antimicrobial therapy to treat HABP or VABP. 5. Known deep-tissue infection (including undrained abscess, meningitis, endocarditis, or osteomyelitis) within 7 days prior to randomization. 6. Participant has received more than 24 hours of any potentially effective systemic antibacterial therapy for the current episode of HABP or VABP within 72 hours before randomization. Exceptions: 1. Evidence of clinical failure of the current episode of HABP or VABP (for example, worsening signs and symptoms) following at least 48 hours of prior systemic antimicrobial therapy (participants with evidence of clinical failure of piperacillin/tazobactam are not eligible for inclusion), or 2. The clinical symptoms and signs of the current episode of HABP or VABP started at least 48 hours after the prior antibacterial therapy was initiated. 7. Pulmonary disease that precludes evaluation of a therapeutic response (including, but not limited to, lung cancer, active tuberculosis, cystic fibrosis, granulomatous disease, fungal pulmonary infection, pulmonary embolism, lung abscess, pleural empyema, or post obstructive pneumonia). 8. Known human immunodeficiency virus (HIV) positivity and meets an acquired immune deficiency syndrome (AIDS)-defining illness or has a documented CD4 count \<200/ microliter (μL) within the past year. 9. Treatment within 30 days prior to enrollment with bone-marrow suppressive chemotherapy (non-bone marrow suppressive chemotherapy is permitted), high dose steroids, immunosuppressive medications for transplantation, or medications for rejection of transplantation. 10. Fulminant hepatitis; current cirrhosis or clinical manifestations of end-stage liver disease (for example, ascites or hepatic encephalopathy); acute hepatic failure or acute decompensation of chronic hepatic failure; or aspartate aminotransferase (AST) or alanine aminotransferase (ALT) level greater than 5-fold the upper limit of normal or total bilirubin greater than 3-fold the upper limit of normal using local or regional laboratory reference values. 11. Requirement for peritoneal dialysis or continuous renal replacement therapy (including continuous venovenous hemofiltration \[CVVH\], continuous venovenous hemodialysis \[CVVHD\], and continuous venovenous hemodiafiltration \[CVVHDF\]) (Note, standard intermittent hemodialysis is not exclusionary). 12. Females who are pregnant or breastfeeding. 13. Participation in any study involving administration of an investigational agent or device within 30 days prior to randomization into this study or previous participation in the current study or any study of vaborbactam or meropenem vaborbactam. 14. Any condition that would make the participant, in the opinion of the Investigator, unsuitable for the study (for example, would place a participant at risk or compromise the quality of the data), including participants with a high likelihood of death within 72 hours after randomization despite adequate antimicrobial therapy for HABP or VABP or participants with a "do not resuscitate" order. 15. An employee of the Investigator or study center with direct involvement in the proposed study or other studies under the direction of that Investigator or study center, or a family member of the employee or the Investigator. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012141 - Term: Respiratory Tract Infections - ID: D000007239 - Term: Infections - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000003428 - Term: Cross Infection - ID: D000007049 - Term: Iatrogenic Disease - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes - ID: D000001424 - Term: Bacterial Infections - ID: D000001423 - Term: Bacterial Infections and Mycoses ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M13904 - Name: Pneumonia - Relevance: HIGH - As Found: Pneumonia - ID: M27445 - Name: Pneumonia, Ventilator-Associated - Relevance: HIGH - As Found: Ventilator Associated Pneumonia - ID: M20529 - Name: Pneumonia, Bacterial - Relevance: HIGH - As Found: Bacterial Pneumonia - ID: M1752 - Name: Healthcare-Associated Pneumonia - Relevance: HIGH - As Found: Hospital-acquired Pneumonia - ID: M10283 - Name: Infections - Relevance: LOW - As Found: Unknown - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: M14978 - Name: Respiratory Tract Infections - Relevance: LOW - As Found: Unknown - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M6642 - Name: Cross Infection - Relevance: LOW - As Found: Unknown - ID: M10099 - Name: Iatrogenic Disease - Relevance: LOW - As Found: Unknown - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown - ID: M4722 - Name: Bacterial Infections - Relevance: LOW - As Found: Unknown - ID: M12136 - Name: Mycoses - Relevance: LOW - As Found: Unknown - ID: M4721 - Name: Bacterial Infections and Mycoses - Relevance: LOW - As Found: Unknown - ID: T4202 - Name: Oculocerebral Syndrome With Hypopigmentation - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000011014 - Term: Pneumonia - ID: D000053717 - Term: Pneumonia, Ventilator-Associated - ID: D000018410 - Term: Pneumonia, Bacterial - ID: D000077299 - Term: Healthcare-Associated Pneumonia ### Intervention Browse Module - Ancestors - ID: D000000900 - Term: Anti-Bacterial Agents - ID: D000000890 - Term: Anti-Infective Agents - ID: D000065093 - Term: beta-Lactamase Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M1889 - Name: Meropenem - Relevance: HIGH - As Found: Every 12 hours - ID: M1938 - Name: Tazobactam - Relevance: HIGH - As Found: Ancillary studies - ID: M13772 - Name: Piperacillin - Relevance: HIGH - As Found: Neoadjuvant therapy - ID: M1885 - Name: Piperacillin, Tazobactam Drug Combination - Relevance: HIGH - As Found: Xenograft - ID: M255430 - Name: Vaborbactam - Relevance: HIGH - As Found: Aboriginal - ID: M4222 - Name: Anti-Bacterial Agents - Relevance: LOW - As Found: Unknown - ID: M4214 - Name: Anti-Infective Agents - Relevance: LOW - As Found: Unknown - ID: M30450 - Name: beta-Lactamase Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000077731 - Term: Meropenem - ID: D000078142 - Term: Tazobactam - ID: D000010878 - Term: Piperacillin - ID: D000077725 - Term: Piperacillin, Tazobactam Drug Combination - ID: C000626994 - Term: Vaborbactam ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00216879 **Brief Title:** Efficacy and Safety of Calcipotriol Plus Betamethasone Dipropionate Gel for up to a Year in Scalp Psoriasis **Official Title:** Long-term Treatment of Scalp Psoriasis With Calcipotriol Plus Betamethasone Dipropionate Gel #### Organization Study ID Info **ID:** MBL 0407 INT #### Organization **Class:** INDUSTRY **Full Name:** LEO Pharma ### Status Module #### Completion Date **Date:** 2006-07 #### Last Update Post Date **Date:** 2015-03-26 **Type:** ESTIMATED **Last Update Submit Date:** 2015-03-25 **Overall Status:** COMPLETED #### Start Date **Date:** 2005-02 **Status Verified Date:** 2015-03 #### Study First Post Date **Date:** 2005-09-22 **Type:** ESTIMATED **Study First Submit Date:** 2005-09-15 **Study First Submit QC Date:** 2005-09-15 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** LEO Pharma ### Description Module **Brief Summary:** The purpose of the trial is to study the safety and efficacy of long term use of once daily applications, as needed, of calcipotriol plus betamethasone dipropionate gel, as compared to calcipotriol alone in the same gel. The primary response criteria will be the incidence of adverse drug reactions of any type, and the incidence of adverse events of concern associated with long-term corticosteroid use on the scalp. ### Conditions Module **Conditions:** - Psoriasis of Scalp ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 800 **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Name:** Calcipotriol plus betamethasone dipropionate gel (LEO 80185) **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** The proportions of patients who experience adverse drug reactions and the proportion of patients who experience adverse events of concern associated with long-term topical corticosteroid use on the scalp during the study #### Secondary Outcomes **Measure:** Percentage of post-baseline satisfactorily controlled assessments according to the Investigators' Global Assessment of disease severity during the study ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Scalp psoriasis amenable to topical treatment with a maximum of 100 g of study medication per week * Clinical signs of psoriasis vulgaris on trunk and/or limbs, or earlier diagnosed with psoriasis vulgaris on trunk and/or limbs * Extent of scalp psoriasis involving more than 10% of the total scalp area * Disease severity on the scalp graded as Moderate, Severe or Very Severe according to the Investigator's Global Assessment of disease severity Exclusion Criteria: * PUVA or Grenz ray therapy anywhere on the patient within 28 days prior to randomisation * UVB therapy anywhere on the patient within 14 days prior to randomisation * Systemic use of biological treatments, whether marketed or not, directed against or with a potential effect on, scalp psoriasis (e.g., alefacept, efalizumab, etanercept, infliximab) within 6 months prior to randomisaiton * Systemic treatments with a potential effect on scalp psoriasis vulgaris (e.g., corticosteroids, retinoids, immunosuppressants) within 28 days prior to randomisation * Any topical treatment for scalp psoriasis or any other skin disease on the scalp (excluding medicated shampoos, emollients and hair conditioners) within 14 days prior to randomisaiton * Topical treatment for other skin disorders with very potent WHO group IV corticosteroids within 14 days prior to randomisation * Planned initiation of, or changes in dose of concomitant medication that could affect scalp psoriasis (e.g., beta blockers, antimalarial drugs, lithium) during the study * Current diagnosis of guttate, pustular, exfoliative or erythrodermic psoriasis * Patients with any of the following conditions present on the scalp area: viral lesions, fungal and bacterial skin infections, parasitic infections and atrophic skin * Known or suspected severe renal insufficiency or severe hepatic disorders * Patiens with history/signs/symptoms suggestive of an abnormality of calcium homeostasis associated with clinically significant hypercalcaemia * Trial subjects should be using an adequate method of contraception **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Moncton **Country:** Canada **Facility:** Clinique de Dermatologie **Zip:** E1C 8X3 **Location 2:** **City:** Hørsholm **Country:** Denmark **Facility:** Hørsholm Hospital, Dermatological Department **Zip:** 2970 **Location 3:** **City:** Saint-Etienne **Country:** France **Facility:** Hôpital Nord, Service de Dermatologie **Zip:** 42055 **Location 4:** **City:** Münster **Country:** Germany **Facility:** Universitätsklinikum Münster, Klinik und Poliklinik für Hautkrankheiten **Zip:** 48179 **Location 5:** **City:** Airdrie **Country:** United Kingdom **Facility:** Monklands Hospital, Department of Dermatology **Zip:** ML6 6JS #### Overall Officials **Official 1:** **Affiliation:** Universitätsklinikum Münster, Klinik und Poliklinik für Hautkrankheiten **Name:** T A Luger, Dr. med. **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000017444 - Term: Skin Diseases, Papulosquamous - ID: D000012871 - Term: Skin Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M14422 - Name: Psoriasis - Relevance: HIGH - As Found: Psoriasis - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown - ID: M19713 - Name: Skin Diseases, Papulosquamous - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000011565 - Term: Psoriasis ### Intervention Browse Module - Ancestors - ID: D000000893 - Term: Anti-Inflammatory Agents - ID: D000005938 - Term: Glucocorticoids - ID: D000006728 - Term: Hormones - ID: D000006730 - Term: Hormones, Hormone Substitutes, and Hormone Antagonists - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018927 - Term: Anti-Asthmatic Agents - ID: D000019141 - Term: Respiratory System Agents - ID: D000003879 - Term: Dermatologic Agents ### Intervention Browse Module - Browse Branches - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: Resp - Name: Respiratory System Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Derm - Name: Dermatologic Agents ### Intervention Browse Module - Browse Leaves - ID: M4909 - Name: Betamethasone - Relevance: HIGH - As Found: 1/2 - ID: M350710 - Name: Calcipotriene - Relevance: HIGH - As Found: Grip - ID: M4910 - Name: Betamethasone Valerate - Relevance: HIGH - As Found: Brain Imaging - ID: M229285 - Name: Betamethasone-17,21-dipropionate - Relevance: HIGH - As Found: Brain Imaging - ID: M259190 - Name: Betamethasone benzoate - Relevance: HIGH - As Found: Brain Imaging - ID: M248787 - Name: Betamethasone sodium phosphate - Relevance: HIGH - As Found: Brain Imaging - ID: M4217 - Name: Anti-Inflammatory Agents - Relevance: LOW - As Found: Unknown - ID: M9047 - Name: Glucocorticoids - Relevance: LOW - As Found: Unknown - ID: M9789 - Name: Hormones - Relevance: LOW - As Found: Unknown - ID: M9788 - Name: Hormone Antagonists - Relevance: LOW - As Found: Unknown - ID: M20963 - Name: Anti-Asthmatic Agents - Relevance: LOW - As Found: Unknown - ID: M21137 - Name: Respiratory System Agents - Relevance: LOW - As Found: Unknown - ID: M7074 - Name: Dermatologic Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000001623 - Term: Betamethasone - ID: D000001624 - Term: Betamethasone Valerate - ID: C000011175 - Term: Betamethasone-17,21-dipropionate - ID: C000015706 - Term: Betamethasone benzoate - ID: C000055085 - Term: Calcipotriene - ID: C000028994 - Term: Betamethasone sodium phosphate ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06371079 **Brief Title:** Safety and Suitability of ICL for Correction of Refractive Errors Without the Use of Dispersive OVDs **Official Title:** Safety and Suitability of Implantable Collamer Lens (ICL) Implantation for Correction of Refractive Errors Without the Use of Dispersive Ophthalmic Viscosurgical Devices (OVDs) #### Organization Study ID Info **ID:** NO-OVD-ICL #### Organization **Class:** OTHER **Full Name:** University of Basrah ### Status Module #### Completion Date **Date:** 2024-06 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-04-17 **Type:** ACTUAL **Last Update Submit Date:** 2024-04-16 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-05-30 **Type:** ESTIMATED #### Start Date **Date:** 2024-03-01 **Type:** ACTUAL **Status Verified Date:** 2024-04 #### Study First Post Date **Date:** 2024-04-17 **Type:** ACTUAL **Study First Submit Date:** 2024-04-06 **Study First Submit QC Date:** 2024-04-16 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Loay Abdulmutalib Almusawi #### Responsible Party **Investigator Affiliation:** University of Basrah **Investigator Full Name:** Loay Abdulmutalib Almusawi **Investigator Title:** Ophthalmology Lecturer **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** True **Is FDA Regulated Drug:** False **Is US Export:** True ### Description Module **Brief Summary:** The goal of this observational study is to test whether surgeries for lenses designed to be implanted in the eye to correct refractive error can be done without the need for using viscoelastic substances that are used routinely nowadays to make it easier to introduce them inside the human eye and protect the inside of the eye during the operation. The main question it aims to answer is that is it safe to do the surgery without using them? to answer this question researchers will access recorded data of patients that underwent refractive surgeries in a private clinic since 2017 and compare them as two groups: those who underwent the traditional procedures and those who had it without the use of dispersive viscoelastics in regard to their vision before and after surgery, their ocular pressure and biomicroscopic analysis of the inside of their corneas before and after surgery. **Detailed Description:** Records of patients that had undergone ICL implantation surgery in Al-Ferdows private eye hospital in Baghdad between 2017 and 2023 were accessed. Two groups of patients were identified, for the first group the ICL was implanted with the use of both dispersive and cohesive OVDs (traditional OVD group) and for the second one a novel method of implantation was used without utilizing dispersive OVD (reduced OVD) group. Both types of surgery were done by the same surgeon and in the same settings. The operative notes of the OVD group were as follows: under topical anesthesia if the ICL is toric, manual corneal marking is done in the sitting position using pendular marker. After loading of the ICL, two-step clear corneal main incision 2.8 mm in width with a bit long track of 1.5-2 mm to enhance its valve action was fashioned. Intracameral injection of dispersive OVD. The ICL was implanted with mouth-to-mouth technique. Anterior chamber reformation with cohesive OVD, then haptics are gently pushed behind the iris using an olive-tipped manipulator, ensuring alignment to proper axis in case of toric ICL, otherwise spherical ICL is placed directly at 180° axis. After that, AC (anterior chamber) wash with irrigation/aspiration is performed then stromal hydration was done to seal the surgical wound. In the reduced OVD group, modification to the traditional method involved omitting the step of dispersive OVD injection and instead utilizing Intracameral injection of 1:1 mixture of 1:1000 adrenaline and 2% lidocaine in an overfilling manner. All other surgical steps are performed in an identical manner. The study was ethically approved by the institutional review committee at the respective hospital and a similar committee at the college of medicine of university of Basrah according to the local guidelines and protocols. Written informed consent was obtained from each patient before the surgery. The study followed tenets of declaration of Helsinki. For both groups, records involving preoperative and postoperative assessments such as uncorrected and best corrected visual acuity, refractive error quantification both objectively utilizing an autorefractometer autorefractor Nidek ARK 1 (Nidek Inc, Gamagori, Japan) and subjectively as manifest refraction, clinical slit-lamp examination notes, intraocular pressure with non-contact air puff tonometer Topcon CT-1P (Topcon Inc., Tokyo, Japan), AC depth assessment by Pentacam Scheimpflug (Oculus Optikgeräte GmbH Inc., Wetzlar, Germany), specular microscopy study of corneal endothelial cells (endothelial cell density (ECD), coefficient of variation (CV) and hexagonality) using Topcon SP-1p Specular Microscope (Topcon Corporation, Tokyo, Japan), were accessed and analyzed using the latest software in SPSS. Patients with missing or incomplete data were excluded from the study. Exclusion criteria also included patients with severe ocular surface disease, unstable refraction, glaucoma, cataract, retinal detachment and uveitis. Unpaired t test or Mann-Whitney test was used to compare the two groups according to fulfilled statistical assumptions. Significance was considered at P value less than 0.05. ### Conditions Module **Conditions:** - Refractive Errors - Myopia - Myopic Astigmatism - Hypermetropia **Keywords:** - refractive errors - myopia - hypermetropia - astigmatism - refractive surgery - ICL - ophthalmic viscosurgical device ### Design Module #### Design Info **Observational Model:** CASE_CONTROL **Time Perspective:** CROSS_SECTIONAL #### Enrollment Info **Count:** 400 **Type:** ESTIMATED **Patient Registry:** True **Study Type:** OBSERVATIONAL **Target Duration:** 1 Year ### Arms Interventions Module #### Arm Group 1 **Description:** In this group of patients, the ICL was implanted utilizing both cohesive and dispersive ophthalmic viscosurgical device. **Intervention Names:** - Device: phakic intraocular lens implantation traditional **Label:** Traditional OVD group #### Arm Group 2 **Description:** In this group of patients, the ICL was implanted with the use of cohesive OVD only. **Intervention Names:** - Device: phakic intraocular lens implantation reduced OVD **Label:** Reduced OVD group ### Interventions #### Intervention 1 **Arm Group Labels:** - Traditional OVD group **Description:** patients with refractive errors undergo surgical implantation of a lens inside the eye (implantable collamer lens or ICL) to correct these refractive errors. The procedure is done with help of use of both cohesive and dispersive OVD **Name:** phakic intraocular lens implantation traditional **Other Names:** - ICL implantation traditional - Intraocular refractive surgery **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - Reduced OVD group **Description:** patients with refractive errors undergo surgical implantation of a lens inside the eye (implantable collamer lens or ICL) to correct these refractive errors. The procedure is done with help of use of only cohesive OVD without using dispersive OVD. **Name:** phakic intraocular lens implantation reduced OVD **Other Names:** - ICL implantation reduced OVD - Intraocular refractive surgery **Type:** DEVICE ### Outcomes Module #### Other Outcomes **Description:** such as glaucoma, cataract, persistent uveitis, macular edema, endophthalmitis..etc. **Measure:** postoperative complications **Time Frame:** any time post op within the maximum 2 years follow up period #### Primary Outcomes **Description:** visual acuity (vision of the patient) without use of any glasses measured using LogMar charts. This measure has no specified units **Measure:** unaided visual acuity after the procedure **Time Frame:** 1-2 years after the surgery **Description:** visual acuity with use of full correction with glasses measured using LogMar charts. This measure has no specified units **Measure:** best corrected visual acuity after the procedure **Time Frame:** 1-2 years after the surgery #### Secondary Outcomes **Description:** intraocular pressure in mmHg as measured by non-contact airpuff tonometer Topcon CT-1P (Topcon Inc., Tokyo, Japan). intraocular pressure is measured in millimeters of mercury **Measure:** Intraocular pressure postoperatively **Time Frame:** first day, first week and first month after the procedure **Description:** defined as the number of corneal endothelial cells per square millimeter of area, measured using the automated machine from Topcon SP-1P (Topcon Inc, Tokyo, Japan) **Measure:** Specular microscopy parameter: endothelial cell density **Time Frame:** 1-2 years after the surgery **Description:** defined as the percentage of endothelial cells having 6 borders and shaped like a hexagon. this measure reflects the overall health of the endothelial cells and decreased hexagonality below 58-60% indicates corneal enodthelial pleomorphism, also measured using the automated machine from Topcon SP-1P (Topcon Inc, Tokyo, Japan) **Measure:** Specular microscopy parameter: endothelial cells hexagonality **Time Frame:** 1-2 years after the surgery **Description:** The standard deviation of the mean cell area divided by the mean cell area gives the coefficient of variation, a unitless number that is normally less than 0.30. it reflects the health of endothelial cells and measured using the automated machine from Topcon SP-1P (Topcon Inc, Tokyo, Japan). **Measure:** Specular microscopy parameter: coefficient of variation of corneal endothelial cells **Time Frame:** 1-2 years after the surgery ### Eligibility Module **Eligibility Criteria:** * Inclusion Criteria: * patients with refractive errors and stable refraction seeking refractive surgery who are fit for phakic IOL (intraocular lens) implantation and having reasonable improvement of visual acuity with refractive correction. Exclusion Criteria: * patients with severe ocular surface disease, unstable refraction, glaucoma, cataract, retinal detachment and uveitis. **Maximum Age:** 50 Years **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT **Study Population:** Records of patients that had undergone ICL implantation surgery in Al-Ferdows private eye hospital in Baghdad between 2017 and 2023 were accessed. Two groups of patients were identified, for the first group the ICL was implanted with the use of both dispersive and cohesive OVDs (traditional OVD group) and for the second one, a novel method of implantation was used without utilizing dispersive OVD (reduced OVD) group. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** loay.almusawi@uobasrah.edu.iq **Name:** Loay Almusawi, FIBMS **Phone:** 009647705559205 **Role:** CONTACT **Contact 2:** **Email:** sohaib.mahmood@meciq.edu.iq **Name:** Sohaib A Mahmood, CABMS **Phone:** 00971585358111 **Role:** CONTACT #### Locations **Location 1:** **City:** Baghdad **Contacts:** ***Contact 1:*** - **Email:** sohaib.mahmood@meciq.edu.iq - **Name:** Sohaib A Mahmood, CABMS - **Phone:** 00971585358111 - **Role:** CONTACT ***Contact 2:*** - **Email:** Ferdouseyeshospital@yahoo.com - **Name:** Sami A Hasoon - **Phone:** +9647901867279 - **Role:** CONTACT ***Contact 3:*** - **Name:** Sohaib A Mahmood, CABMS - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 4:*** - **Name:** Loay A Almusawi, FIBMS - **Role:** SUB_INVESTIGATOR ***Contact 5:*** - **Name:** Hamid M Altaha, CABMS - **Role:** SUB_INVESTIGATOR ***Contact 6:*** - **Name:** Bashar Alwash, CABMS - **Role:** SUB_INVESTIGATOR **Country:** Iraq **Facility:** Al-Ferdows private eye hospital **Status:** RECRUITING #### Overall Officials **Official 1:** **Affiliation:** Ibn Al-Haithem eye teaching hospital **Name:** Sohaib A Mahmood, FIBMS **Role:** STUDY_CHAIR **Official 2:** **Affiliation:** University of Basrah **Name:** Loay A Almusawi, FIBMS **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** Peng M, Tang Q, Zhao L, Khan MA, Lin D. Safety of implantable Collamer lens implantation without ophthalmic viscosurgical device: A retrospective cohort study. Medicine (Baltimore). 2020 Jun 12;99(24):e20691. doi: 10.1097/MD.0000000000020691. **PMID:** 32541520 **Citation:** Zhang Z, Niu L, Zhao J, Miao H, Chen Z, Shen Y, Chen X, Ye Y, Wang X, Zhou X. Safety of EVO ICL Implantation With an Ophthalmic Viscosurgical Device-Free Technique in the Early 24 h After Surgery. Front Med (Lausanne). 2021 Nov 17;8:764653. doi: 10.3389/fmed.2021.764653. eCollection 2021. **PMID:** 34869472 **Citation:** Qin Q, Bao L, He Z, Chen F, Zhu D, Zhang S, Zhang W, Liu Y, Gao R, Xie Z. Pure ICL Implantation: A Novel Ophthalmic Viscosurgical Device-Free Method. J Ophthalmol. 2021 Oct 6;2021:7363267. doi: 10.1155/2021/7363267. eCollection 2021. **PMID:** 34659826 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000005128 - Term: Eye Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC11 - Name: Eye Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M12168 - Name: Myopia - Relevance: HIGH - As Found: Myopia - ID: M4558 - Name: Astigmatism - Relevance: HIGH - As Found: Astigmatism - ID: M10007 - Name: Hyperopia - Relevance: HIGH - As Found: Hypermetropia - ID: M14872 - Name: Refractive Errors - Relevance: HIGH - As Found: Refractive Errors - ID: M8271 - Name: Eye Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009216 - Term: Myopia - ID: D000001251 - Term: Astigmatism - ID: D000012030 - Term: Refractive Errors - ID: D000006956 - Term: Hyperopia ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05732779 **Brief Title:** Mobile Video Directly Observed Therapy (DOT) for Immunosuppression Medication Adherence in Adolescent Heart Transplant Recipients **Official Title:** Mobile Video Directly Observed Therapy (DOT) for Immunosuppression Medication Adherence in Adolescent Heart Transplant Recipients: Enhancing Technological Innovation and Strengthening the Role of Small Businesses in Meeting Needs of Adolescent Organ Transplant #### Organization Study ID Info **ID:** IRB202201809 #### Organization **Class:** OTHER **Full Name:** University of Florida #### Secondary ID Infos **ID:** R44HL167591-01A1 **Link:** https://reporter.nih.gov/quickSearch/R44HL167591-01A1 **Type:** NIH ### Status Module #### Completion Date **Date:** 2026-12-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-03-29 **Type:** ACTUAL **Last Update Submit Date:** 2024-03-27 **Overall Status:** NOT_YET_RECRUITING #### Primary Completion Date **Date:** 2025-03-31 **Type:** ESTIMATED #### Start Date **Date:** 2024-03-31 **Type:** ESTIMATED **Status Verified Date:** 2024-03 #### Study First Post Date **Date:** 2023-02-17 **Type:** ACTUAL **Study First Submit Date:** 2023-02-06 **Study First Submit QC Date:** 2023-02-08 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** emocha Mobile Health, Inc. **Class:** NIH **Name:** National Heart, Lung, and Blood Institute (NHLBI) #### Lead Sponsor **Class:** OTHER **Name:** University of Florida #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** We will conduct a two-group randomized controlled trial to examine the eMocha DOT intervention with pediatric HT recipients.In this population, medication nonadherence remains a primary cause of late acute rejection (LAR) episodes, increased number of hospitalizations, graft failure, and patient mortality. Herein, we propose an innovative approach to promote medication adherence and improve patient and graft outcomes. **Detailed Description:** Few interventions have proven to be successful in promoting medication adherence and impacting short- and long-term post-transplant outcomes in adolescent heart transplant (HT) recipients. Improving adherence is a persistent challenge with youth experiencing chronic health conditions, especially among adolescent transplant recipients. Adolescent organ transplant recipients experience unique challenges remaining adherent to the complex post-transplant regimen, with rates of non-adherence as high as 40% to 60%. In this population, medication non-adherence remains a primary cause of late acute rejection (LAR) episodes, increased number of hospitalizations, graft failure, and patient mortality. Herein, we propose an innovative approach to promote medication adherence and improve patient and graft outcomes. ### Conditions Module **Conditions:** - Heart Transplant Rejection - Immune Suppression - Pediatric Heart Transplant - Medication Nonadherence - Health Behavior - Remote Monitoring - Patient Engagement ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 100 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Adolescent patients randomized to the use of asynchronous mobile video directly observed therapy (DOT) intervention (eMocha DOT app) **Intervention Names:** - Other: DOT intervention **Label:** eMocha intervention **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Adolescent patients who continue enhanced goal-setting standard of care **Label:** Standard of care **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - eMocha intervention **Description:** A mobile health application developed by eMocha Health Inc. facilitates asynchronous mobile video directly observed therapy (DOT) intervention, empowering users to track dose-by-dose medication adherence **Name:** DOT intervention **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** medication level variability index (MLVI). MLVI is calculated as the Standard Deviation of a set of at least 3 tacrolimus trough blood levels for each participant. **Measure:** Medication adherence based on medication level variability index **Time Frame:** 12 weeks **Description:** Number of doses observed and reviewed by nursing staff compare to number of prescribed doses per day **Measure:** Directly observed medication adherence **Time Frame:** 1 weeks **Description:** Number of biopsy-proven rejection, clinician-assigned rejection **Measure:** Late Acute Rejection **Time Frame:** 6 months post-intervention **Description:** Number of hospitalizations **Measure:** Hospitalization **Time Frame:** 6 months post-intervention **Description:** A 13-item scale designed to measure a patient's perception of their knowledge, skill and confidence in managing their chronic health condition **Measure:** Patient Activation Measure **Time Frame:** Pretest at enrollment and posttest at 12 weeks **Description:** A 20-item scale with five subscales (scale of 1 (none) to 5 (always), total score 20 to 200, higher score being better outcome) that assess patient activation, delivery system design, goal setting, problem-solving and contextual counseling, and follow-up and coordination. **Measure:** Patient Assessment of Chronic Illness Care (PACIC) **Time Frame:** Pretest at enrollment and posttest at 12 weeks **Description:** Collected using a modified version of the engagement index questionnaire used **Measure:** Self-reported user satisfaction with the DOT app **Time Frame:** Pretest at enrollment and posttest at 12 weeks **Description:** Standard engagement metrics or paradata (e.g., amount, frequency, duration, and depth of use) **Measure:** Engagement metrics **Time Frame:** Pretest at enrollment and posttest at 12 weeks **Description:** HRQOL **Measure:** PedsQL 3.0 Transplant Module (PedsQL-TM) **Time Frame:** Pretest at enrollment and posttest at 12 weeks **Description:** To assess adolescent perceived barriers to medication adherence **Measure:** Adolescent Medication Barriers Scale (AMBS) **Time Frame:** Pretest, posttest **Description:** To assess parental perceived barriers to their child taking their medication **Measure:** Parent Medication Barriers Scale (PMBS) **Time Frame:** Pretest at enrollment and posttest at 12 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Eligible participants are 10-21 years of age * Have received a heart transplant and are followed participating pediatric heart transplant centers * English-speaking or Spanish-speaking * Own a smart-phone or have access to the mobile app through other devices * Are willing to receive information through it * Have a MLVI score of greater than 2.0 over the last year Exclusion Criteria: • Those with cognitive impairments will not be eligible for enrollment due to inability to provide informed assent **Maximum Age:** 21 Years **Minimum Age:** 10 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** dgupta@ufl.edu **Name:** Dipankar Gupta, MD **Phone:** 352-273-5422 **Role:** CONTACT **Contact 2:** **Email:** dalylc@ufl.edu **Name:** Dalia Lopez-Colon, PhD **Phone:** 352-281-6723 **Role:** CONTACT #### Locations **Location 1:** **City:** Coral Gables **Contacts:** ***Contact 1:*** - **Email:** osa5040@med.miami.edu - **Name:** Olena Ashworth, MD - **Phone:** 801-310-9452 - **Role:** CONTACT ***Contact 2:*** - **Name:** Paolo Rosconi, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** University of Miami, Miller School of Medicine **State:** Florida **Zip:** 33146 **Location 2:** **City:** Gainesville **Country:** United States **Facility:** University of Florida **State:** Florida **Zip:** 32610 **Location 3:** **City:** Hollywood **Contacts:** ***Contact 1:*** - **Email:** sshugh@mhs.net - **Name:** Svetlana B Shugh, MD - **Phone:** 954-265-3437 - **Role:** CONTACT ***Contact 2:*** - **Name:** Svetlana B Shuh, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Joe DiMaggio Children's Hospital **State:** Florida **Zip:** 33021 **Location 4:** **City:** Tallahassee **Contacts:** ***Contact 1:*** - **Email:** mkillian@fsu.edu - **Name:** Mike Killian, PhD - **Phone:** 850-644-7094 - **Role:** CONTACT ***Contact 2:*** - **Email:** smayewski@fsu.edu - **Name:** Sonnie Mayewski - **Role:** CONTACT ***Contact 3:*** - **Name:** Mike Killian, PhD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** FSU College of Medicine **State:** Florida **Zip:** 32306 #### Overall Officials **Official 1:** **Affiliation:** University of Florida **Name:** Dipankar Gupta, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04894279 **Acronym:** SMİLE-C **Brief Title:** Reliability and Validity of the Turkish Version of SMİLE-C **Official Title:** A Study of the Reliability and Validity of the Turkish Version of Short Multidimensional Inventory Lifestyle Evaluation - Confinement #### Organization Study ID Info **ID:** SMİLE-C #### Organization **Class:** OTHER **Full Name:** Marmara University ### Status Module #### Completion Date **Date:** 2022-02-20 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-03-11 **Type:** ACTUAL **Last Update Submit Date:** 2022-03-10 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-02-01 **Type:** ACTUAL #### Start Date **Date:** 2021-04-30 **Type:** ACTUAL **Status Verified Date:** 2021-05 #### Study First Post Date **Date:** 2021-05-20 **Type:** ACTUAL **Study First Submit Date:** 2021-05-16 **Study First Submit QC Date:** 2021-05-16 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Marmara University #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** How the life style changed multidimensionally with home isolation in the period of COVID-19 is a new research question. Physical and social isolation, home isolation, and quarantine policies applied during epidemic periods such as SARS, MERS, Ebola have been associated with lifestyle changes (Lippi, 2019). Much of the research on the health impact of epidemics and isolation has focused on psychological issues such as stress-related symptoms and disorders and to a lesser extent social support and stress management. In contrast, the epidemic and its associated lifestyle issues during quarantine and isolation, including the impact of dietary changes, restricted physical activity, and increased indoor and screen time, are still not investigated. A limited number of studies have examined lifestyle changes during COVID-19 and previous epidemic periods. However, there is no Turkish survey that comprehensively deals with the lifestyle, the scope of which is expanding, today. The Brief Multidimensional Lifestyle Assessment Scale, which we planned to validate in our study, is a scale developed by Balanzá-Martínez et al to multi-dimensionally evaluate the lifestyle changes that occurred during the COVID-19 outbreak. It consists of 7 parts (Diet and Nutrition, Substance addiction, Physical activity, Stress management, Restorative sleep, Social support and Environmental exposure) and a total of 27 items. There are response options measured using a 4-point Likert scale. High scores indicate a healthy lifestyle (Balanzá-Martínez, 2021). In this context, we think that adapting the Short Multidimensional Lifestyle Assessment Scale / Short Multidimensional Inventory Lifestyle Evaluation - Confinement (SMILE-C) questionnaire into Turkish will make a significant contribution to the literature. ### Conditions Module **Conditions:** - Covid19 **Keywords:** - Covid-19, pandemic, lifestyle ### Design Module #### Design Info **Observational Model:** OTHER **Time Perspective:** CROSS_SECTIONAL #### Enrollment Info **Count:** 166 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Outcomes Module #### Primary Outcomes **Description:** Questionnaire **Measure:** Short Multidimensional Inventory Lifestyle Evaluation - Confinement **Time Frame:** 5 minutes **Description:** Questionnaire **Measure:** Health Promoting Life-Style Profile **Time Frame:** 10 minutes **Description:** Quesitonnaire **Measure:** International Physical Activity Questionnaire - Short Form **Time Frame:** 5 minutes ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Female and male participants aged 18-65, Volunteering to participate in research Exclusion Criteria: Inability to use a computer, tablet or mobile phone, Not answering all the questions in questionnaire **Healthy Volunteers:** True **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** healthy people between 18-65 years old. ### Contacts Locations Module #### Locations **Location 1:** **City:** İ̇stanbul **Country:** Turkey **Facility:** Berivan Beril KILIÇ ### IPD Sharing Statement Module **Info Types:** - STUDY_PROTOCOL **IPD Sharing:** YES ### References Module #### References **Citation:** Balanza-Martinez V, Kapczinski F, de Azevedo Cardoso T, Atienza-Carbonell B, Rosa AR, Mota JC, De Boni RB. The assessment of lifestyle changes during the COVID-19 pandemic using a multidimensional scale. Rev Psiquiatr Salud Ment (Engl Ed). 2021 Jan-Mar;14(1):16-26. doi: 10.1016/j.rpsm.2020.07.003. Epub 2020 Aug 29. **PMID:** 32962948 **Citation:** Firth J, Ward PB, Stubbs B. Editorial: Lifestyle Psychiatry. Front Psychiatry. 2019 Aug 26;10:597. doi: 10.3389/fpsyt.2019.00597. eCollection 2019. No abstract available. **PMID:** 31507466 **Citation:** He H, Harris L. The impact of Covid-19 pandemic on corporate social responsibility and marketing philosophy. J Bus Res. 2020 Aug;116:176-182. doi: 10.1016/j.jbusres.2020.05.030. Epub 2020 May 21. **PMID:** 32457556 **Citation:** Jiao WY, Wang LN, Liu J, Fang SF, Jiao FY, Pettoello-Mantovani M, Somekh E. Behavioral and Emotional Disorders in Children during the COVID-19 Epidemic. J Pediatr. 2020 Jun;221:264-266.e1. doi: 10.1016/j.jpeds.2020.03.013. Epub 2020 Apr 3. No abstract available. **PMID:** 32248989 **Citation:** Lippi G, Henry BM, Sanchis-Gomar F. Physical inactivity and cardiovascular disease at the time of coronavirus disease 2019 (COVID-19). Eur J Prev Cardiol. 2020 Jun;27(9):906-908. doi: 10.1177/2047487320916823. Epub 2020 Apr 9. No abstract available. **PMID:** 32270698 **Citation:** Pinar R, Celik R, Bahcecik N. Reliability and construct validity of the Health-Promoting Lifestyle Profile II in an adult Turkish population. Nurs Res. 2009 May-Jun;58(3):184-93. doi: 10.1097/NNR.0b013e31819a8248. **PMID:** 19448522 **Citation:** Orru G, Ciacchini R, Gemignani A, Conversano C. Psychological Intervention Measures During the Covid-19 Pandemic. Clin Neuropsychiatry. 2020 Apr;17(2):76-79. doi: 10.36131/CN20200208. **PMID:** 34908972 **Citation:** GBD 2017 Risk Factor Collaborators. Global, regional, and national comparative risk assessment of 84 behavioural, environmental and occupational, and metabolic risks or clusters of risks for 195 countries and territories, 1990-2017: a systematic analysis for the Global Burden of Disease Study 2017. Lancet. 2018 Nov 10;392(10159):1923-1994. doi: 10.1016/S0140-6736(18)32225-6. Epub 2018 Nov 8. Erratum In: Lancet. 2019 Jan 12;393(10167):132. Lancet. 2019 Jun 22;393(10190):e44. **PMID:** 30496105 **Citation:** Tabachnick, B. G., & Fidell, L. S. (2018). Using multivariate statistics (7th ed.). **Citation:** Walker, S. N., & Hill-Polerecky, D. M. (1997). Psychometric evaluation of Health Promoting Lifestyle Profile II. Unpublished manuscript, University of Nebraska Medical Center, College of Nursing, Lincoln. **Citation:** Yılmaz, Ö , Boz, H , Arslan, A . (2017). DEPRESYON ANKSİYETE STRES ÖLÇEĞİNİN(DASS 21) TÜRKÇE KISA FORMUNUN GEÇERLİLİK-GÜVENİLİRLİK ÇALIŞMASI . Finans Ekonomi ve Sosyal Araştırmalar Dergisi , 2 (2) , 78-91 . Retrieved from. https://dergipark.org.tr/tr/pub/fesa/issue/30912/323190 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000011024 - Term: Pneumonia, Viral - ID: D000011014 - Term: Pneumonia - ID: D000012141 - Term: Respiratory Tract Infections - ID: D000007239 - Term: Infections - ID: D000014777 - Term: Virus Diseases - ID: D000018352 - Term: Coronavirus Infections - ID: D000003333 - Term: Coronaviridae Infections - ID: D000030341 - Term: Nidovirales Infections - ID: D000012327 - Term: RNA Virus Infections - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M2561 - Name: COVID-19 - Relevance: HIGH - As Found: COVID-19 - ID: M13904 - Name: Pneumonia - Relevance: LOW - As Found: Unknown - ID: M13914 - Name: Pneumonia, Viral - Relevance: LOW - As Found: Unknown - ID: M10283 - Name: Infections - Relevance: LOW - As Found: Unknown - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: M14978 - Name: Respiratory Tract Infections - Relevance: LOW - As Found: Unknown - ID: M17522 - Name: Virus Diseases - Relevance: LOW - As Found: Unknown - ID: M20490 - Name: Coronavirus Infections - Relevance: LOW - As Found: Unknown - ID: M6555 - Name: Coronaviridae Infections - Relevance: LOW - As Found: Unknown - ID: M23685 - Name: Nidovirales Infections - Relevance: LOW - As Found: Unknown - ID: M15149 - Name: RNA Virus Infections - Relevance: LOW - As Found: Unknown - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000086382 - Term: COVID-19 ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00648479 **Brief Title:** New Method for Distal Interlocking of Cannulated Intramedullary Nails **Official Title:** New Method for Distal Interlocking of Cannulated Intramedullary Nails #### Organization Study ID Info **ID:** EKL-001-HMO-CTIL #### Organization **Class:** OTHER **Full Name:** Hadassah Medical Organization ### Status Module #### Expanded Access Info #### Last Update Post Date **Date:** 2011-12-28 **Type:** ESTIMATED **Last Update Submit Date:** 2011-12-25 **Overall Status:** TERMINATED **Status Verified Date:** 2011-12 #### Study First Post Date **Date:** 2008-04-01 **Type:** ESTIMATED **Study First Submit Date:** 2008-03-17 **Study First Submit QC Date:** 2008-03-31 **Why Stopped:** the company does not want to proceed yet with the study ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Hadassah Medical Organization #### Responsible Party **Old Name Title:** Hadassah Medical Organization ### Description Module **Brief Summary:** We intend to include10 patients in this study and trial. Prior to treatment they will sign an informed consent form, stating that they are acquainted with the surgical procedure and study, and that they consent to participate in the study. Five patients will undergo regular operation with Universal tibial reamed nail (AO-Synthes) using the fluoroscopy device: OEC 9800 (GENERAL ELECTRIC). five patients will undergo the same operation using the new navigation system:"The Guiding star" method. The allocation will be random. ### Conditions Module **Conditions:** - Fracture **Keywords:** - Fracture Fixation ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** DIAGNOSTIC #### Enrollment Info **Count:** 10 **Type:** ESTIMATED **Phases:** - PHASE1 - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Device: The Guiding Star **Label:** 1 **Type:** EXPERIMENTAL #### Arm Group 2 **Intervention Names:** - Device: fluoroscopy (OEC 9800 - General Electric) **Label:** 2 **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - 1 **Name:** The Guiding Star **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - 2 **Name:** fluoroscopy (OEC 9800 - General Electric) **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Measure:** the outcome will be asses using regular fluoroscopy in the operation room. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Signing Informed consent. 2. Conscious patients. Exclusion Criteria: 1. Hemodynamic Instability. 2. Multiple system injuries. 3. Pregnancy. 4. Soldiers. **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014947 - Term: Wounds and Injuries ### Condition Browse Module - Browse Branches - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M26370 - Name: Fractures, Bone - Relevance: HIGH - As Found: Fracture - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000050723 - Term: Fractures, Bone ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03205579 **Brief Title:** Pain Education to Improve Cancer Pain Management Patients **Official Title:** Effectiveness of Pain Education to Improve Cancer Pain Management in Hospitalized Patients #### Organization Study ID Info **ID:** Si231/2017 #### Organization **Class:** OTHER **Full Name:** Mahidol University ### Status Module #### Completion Date **Date:** 2022-07-18 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-08-05 **Type:** ACTUAL **Last Update Submit Date:** 2022-08-03 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-09-30 **Type:** ACTUAL #### Start Date **Date:** 2018-09-20 **Type:** ACTUAL **Status Verified Date:** 2022-08 #### Study First Post Date **Date:** 2017-07-02 **Type:** ACTUAL **Study First Submit Date:** 2017-06-29 **Study First Submit QC Date:** 2017-06-29 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Mahidol University #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Cancer pain is one of the problems of treating cancer pain. Although, there is a WHO analgesic ladder to improve this problem, it is still inadequate pain control. Pain does not affect only physical but also emotional and quality of life. From review literatures we found that patients' knowledge about cancer pain management is inaccurate; for example, fear to use opioid, try to patience of pain, concerning only cancer treatments, which can cause of unfavorable pain management outcome. Therefore, we will conduct the RCT of using pain education by video comparing to conventional face to face pain education by nurse in hospitalized cancer pain patients.We will use 25 MCQs examination for testing pre-post intervention to test level of understanding of patients. The measurements are NRS, ThaiHADs and FACT-G at the first and last day of study. We expect that NRS should improve more than 50% at the seven day of study. **Detailed Description:** protocol Randomized controlled trial study Sample size is 70 patients divided to 2 groups (35 for video group and 35 for conventional face to face group) Inclusion criteria 1. patients' age 18-70 years old 2. moderate to severe cancer pain 3. ECOG(Eastern cooperative Oncology Group) performance status \<= 3 4. Patients can write and read Thai language Exclusion criteria 1.Clinical unstability 2.Confusion and delirium 3.Bed ridden 4.Psychotic problem We will randomly allocate patients by using computer program nQuery advice 6.0. After patients sign inform consent we will record baseline characteristics, assess baseline Numerical rating scale (NRS), emotional status (ThaiHADs), Quality of life status (FACT-G). All patients will do the 25 MCQs test within 30 minutes before intervention. Video group will watch video 10 minutes and conventional group will receive pain education from trained nurse in the same period. The knowledge includes cancer pain definition, cancer pain management, pain assessment and role of patients in cancer pain management. After finishing intervention patients will the same 25 MCQs test (30 minutes) and patients can ask questions to the trained nurse. All patients will be educated to record pain diary everyday for seven days. All patients will receive standard pain management from the physicians. At the day seven we will assess NRS, ThaiHADs and FACT-G and finish the study. ### Conditions Module **Conditions:** - Pain - Inpatient - Cancer ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - CARE_PROVIDER **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 48 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Video cancer pain education (10 minutes ) the knowledge includes cancer pain definition, cancer pain treatment, pain assessment and role of patient in cancer pain management. **Intervention Names:** - Other: Video group **Label:** Video group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Face to face cancer pain education by trained nurse (10 minutes)the knowledge includes cancer pain definition, cancer pain treatment, pain assessment and role of patient in cancer pain management. **Intervention Names:** - Other: conventional group **Label:** Conventional group **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Video group **Description:** Using video for educate cancer pain patients **Name:** Video group **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Conventional group **Description:** Face to face cancer pain education by trained nurse **Name:** conventional group **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** The pain reduction will be assessed and compared using numerical rating (NRS) from 0-10; 0 designating "no pain" and 10 designating " worst possible pain" scale between the first day and the seventh day of study **Measure:** Pain intensity **Time Frame:** seven days #### Secondary Outcomes **Description:** The emotional status of participants will be assessed and compared using Thai-HADs (Hospital Anxiety and Depression scale) between the first day and the seventh day of study. The score will be analyzed into depression and anxiety. the score 0-7 for either sub scale being in normal range, 8-10; being just suggestive of the presence of the respective state, a score of 11 or higher indicating probable presence of the mood disorder(Depression and Anxiety). **Measure:** Emotional status **Time Frame:** seven days **Description:** Functional status will be assessed and compared using FACT-G between the first day and the seventh day of study. The subscale including physical well-being, social/family well-being, emotional well-being and functional well-being. Each items scored from 0-4; from 0 means "not at all" to 4 means "very much". The higher score would indicate better quality of life. **Measure:** Functional assessment **Time Frame:** seven days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * moderate to severe pain (NRS\>4) cancer pain * ECOG (Eastern cooperative Oncology group) performance status \<=3 * Can read, listen, write Thai language Exclusion Criteria: * Clinical instability * Confusion and delirium * Bed ridden * Psychotic problem **Maximum Age:** 70 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Bangkoknoi **Country:** Thailand **Facility:** Faculty of medicine Siriraj Hospital Mahidol University **State:** Bangkok **Zip:** 10700 **Location 2:** **City:** Bangkok **Country:** Thailand **Facility:** Siriraj Hospital Mahidol University **Zip:** 10700 #### Overall Officials **Official 1:** **Affiliation:** Department of Anesthesiology, Faculty of Medicine Siriraj Hospital, Mahidol University **Name:** Suratsawadee Wangnamthip, md **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ### References Module #### References **Citation:** Wangnamthip S, Euasobhon P, Siriussawakul A, Jirachaipitak S, Laurujisawat J, Vimolwattanasarn K. Effective Pain Management for Inpatients at Siriraj Hospital: A Retrospective Study. J Med Assoc Thai. 2016 May;99(5):565-71. **PMID:** 27501612 **Citation:** van den Beuken-van Everdingen MH, de Rijke JM, Kessels AG, Schouten HC, van Kleef M, Patijn J. Prevalence of pain in patients with cancer: a systematic review of the past 40 years. Ann Oncol. 2007 Sep;18(9):1437-49. doi: 10.1093/annonc/mdm056. Epub 2007 Mar 12. **PMID:** 17355955 **Citation:** Yates P, Dewar A, Edwards H, Fentiman B, Najman J, Nash R, Richardson V, Fraser J. The prevalence and perception of pain amongst hospital in-patients. J Clin Nurs. 1998 Nov;7(6):521-30. doi: 10.1046/j.1365-2702.1998.00192.x. **PMID:** 10222947 **Citation:** Gureje O, Von Korff M, Simon GE, Gater R. Persistent pain and well-being: a World Health Organization Study in Primary Care. JAMA. 1998 Jul 8;280(2):147-51. doi: 10.1001/jama.280.2.147. Erratum In: JAMA 1998 Oct 7;280(13):1142. **PMID:** 9669787 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010146 - Term: Pain - ID: D000009461 - Term: Neurologic Manifestations ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases ### Condition Browse Module - Browse Leaves - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown - ID: M1091 - Name: Cancer Pain - Relevance: HIGH - As Found: Cancer Pain - ID: M12404 - Name: Neurologic Manifestations - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000072716 - Term: Cancer Pain ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00953979 **Brief Title:** Efficacy and Safety of Kunxian Capsule in Treatment Patients With Early Ankylosing Spondylitis **Official Title:** A Clinical Trial About the Efficacy and Safety of Kunxian Capsule in Treatment Patients With Early Ankylosing Spondylitis: a 12-week, Multi-center, Randomized, Double-blind, Active Drug and Placebo Compared Clinical Trial #### Organization Study ID Info **ID:** 2008-2 #### Organization **Class:** OTHER **Full Name:** Sun Yat-sen University ### Status Module #### Completion Date **Date:** 2009-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2012-01-19 **Type:** ESTIMATED **Last Update Submit Date:** 2012-01-18 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2009-09 **Type:** ACTUAL #### Start Date **Date:** 2008-01 **Status Verified Date:** 2012-01 #### Study First Post Date **Date:** 2009-08-06 **Type:** ESTIMATED **Study First Submit Date:** 2009-08-05 **Study First Submit QC Date:** 2009-08-05 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** China-Japan Friendship Hospital **Class:** OTHER_GOV **Name:** Dongguan People's Hospital **Class:** OTHER **Name:** Zhujiang Hospital **Class:** OTHER **Name:** Huashan Hospital #### Lead Sponsor **Class:** OTHER **Name:** Gu Jieruo #### Responsible Party **Investigator Affiliation:** Sun Yat-sen University **Investigator Full Name:** Gu Jieruo **Investigator Title:** Department of Rheumatology, the Third Affiliated Hospital of Sun Yat-sen University **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** This is a prospective randomized controlled study to evaluated the efficacy and safety of kunxian capsule in treating of patients with early ankylosing spondylitis (AS). The major outcome index is proportion of patients achieving ASAS20 response at week12, and minor outcome indexes include ASAS50 and ASAS70, BASDAI20,BASDAI50 and BASDAI70. The adverse events at any time were recorded to evaluate the safety. ### Conditions Module **Conditions:** - Ankylosing Spondylitis - Treatment **Keywords:** - ankylosing spondylitis - treatment - Sulfasalazine - kunxian capsule ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 126 **Type:** ACTUAL **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Sulfasalazine is a drug in treatment RA, AS and ulcerative colonitis. In this study, Sulfasalazine is used to act as an active comparator to access the efficacy of kunxian capsule. **Intervention Names:** - Drug: kunxian capsule **Label:** Sulfasalazine **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** The placebo capsule was used to be a comparator of kunxian capsule **Intervention Names:** - Drug: kunxian capsule **Label:** placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Sulfasalazine - placebo **Description:** The main element of kunxian capsule is Tripterygium. Patients should take 2 tablets per time and 3 times per day for 12 weeks. **Name:** kunxian capsule **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** proportion of patients achieving ASAS20 response **Time Frame:** 12th week #### Secondary Outcomes **Measure:** proportion of patients achieving BASDAI20/50/70 response **Time Frame:** 12th week ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 16 to 65 years old, having signed the informed consent; * fulfill the ESSG criteria for diagnosis of SpA; not fulfill the 1984 modified NewYork criteria for AS; * BASDAI score more than 4; * stop taking DMARDs for at least 4 weeks; * NSAIDs dosage has been stable for at least 4 weeks; Exclusion Criteria: * Intra-articular injection of cortisone within 3 months. * History of heart failure, multiple sclerosis, COPD, lymphoma or other tumors; * Accompanied by fibromyalgia or other rheumatic diseases; * Female of pregnancy or breast feeding; * History of mental disease and poor compliance. * History of drug abuse or alcoholism. **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Guangzhou **Country:** China **Facility:** Rheumatology Department, the Third Affiliated Hospital of Sun Yat-sen University **State:** Guangdong **Zip:** 510630 #### Overall Officials **Official 1:** **Affiliation:** Rheumatology Department, Third Affiliated Hospital of Sun Yat-sen University **Name:** Jieruo Gu, M.D. **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Li Q, Li L, Bi L, Xiao C, Lin Z, Cao S, Liao Z, Gu J. Kunxian capsules in the treatment of patients with ankylosing spondylitis: a randomized placebo-controlled clinical trial. Trials. 2016 Jul 22;17(1):337. doi: 10.1186/s13063-016-1438-6. **PMID:** 27449221 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001850 - Term: Bone Diseases, Infectious - ID: D000007239 - Term: Infections - ID: D000001847 - Term: Bone Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000013122 - Term: Spinal Diseases - ID: D000001168 - Term: Arthritis - ID: D000007592 - Term: Joint Diseases - ID: D000089183 - Term: Axial Spondyloarthritis - ID: D000025242 - Term: Spondylarthropathies - ID: D000000844 - Term: Ankylosis ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M15961 - Name: Spondylitis - Relevance: HIGH - As Found: Spondylitis - ID: M23035 - Name: Spondylarthritis - Relevance: HIGH - As Found: Spondylitis - ID: M15962 - Name: Spondylitis, Ankylosing - Relevance: HIGH - As Found: Ankylosing Spondylitis - ID: M10283 - Name: Infections - Relevance: LOW - As Found: Unknown - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: M5126 - Name: Bone Diseases - Relevance: LOW - As Found: Unknown - ID: M5129 - Name: Bone Diseases, Infectious - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: M15919 - Name: Spinal Diseases - Relevance: LOW - As Found: Unknown - ID: M4476 - Name: Arthritis - Relevance: LOW - As Found: Unknown - ID: M10621 - Name: Joint Diseases - Relevance: LOW - As Found: Unknown - ID: M2697 - Name: Axial Spondyloarthritis - Relevance: LOW - As Found: Unknown - ID: M23036 - Name: Spondylarthropathies - Relevance: LOW - As Found: Unknown - ID: M4170 - Name: Ankylosis - Relevance: LOW - As Found: Unknown - ID: T5412 - Name: Spondylarthropathy - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000013166 - Term: Spondylitis - ID: D000025241 - Term: Spondylarthritis - ID: D000013167 - Term: Spondylitis, Ankylosing ### Intervention Browse Module - Browse Branches - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: ARhu - Name: Antirheumatic Agents - Abbrev: Analg - Name: Analgesics - Abbrev: Gast - Name: Gastrointestinal Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: HB - Name: Herbal and Botanical ### Intervention Browse Module - Browse Leaves - ID: M15280 - Name: Sulfasalazine - Relevance: LOW - As Found: Unknown - ID: T318 - Name: Tripterygium - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02793479 **Acronym:** RABE **Brief Title:** Radiofrequency Ablation in Patients With Barrett's Esophagus **Official Title:** National Registry for Radiofrequency Ablation in Patients With Barrett's Esophagus #### Organization Study ID Info **ID:** AUT-RFA-Registry-01 #### Organization **Class:** OTHER **Full Name:** Austrian Society Of Surgical Oncology ### Status Module #### Completion Date **Date:** 2021-12 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2019-07-05 **Type:** ACTUAL **Last Update Submit Date:** 2019-07-02 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2020-12 **Type:** ESTIMATED #### Start Date **Date:** 2017-03-01 **Type:** ACTUAL **Status Verified Date:** 2019-07 #### Study First Post Date **Date:** 2016-06-08 **Type:** ESTIMATED **Study First Submit Date:** 2016-06-01 **Study First Submit QC Date:** 2016-06-07 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Medtronic - MITG #### Lead Sponsor **Class:** OTHER **Name:** Matthias Paireder #### Responsible Party **Investigator Affiliation:** Austrian Society Of Surgical Oncology **Investigator Full Name:** Matthias Paireder **Investigator Title:** Medical Doctor **Type:** SPONSOR_INVESTIGATOR ### Description Module **Brief Summary:** The aim of this study is to establish a nationwide registry to collect data regarding the treatment of Barrett's Esophagus (BE) with radiofrequency ablation. The objective of this registry is to increase the number of data and therefore obtain a raise of quality assurance and improve outcome and patient security. Furthermore to provide participating physicians information and experience for treatment details in the therapy of BE. **Detailed Description:** For treatment of Barrett's Esophagus (BE), a condition considered as a complication of the gastroesophageal reflux disease (GERD), radiofrequency ablation (RFA) was introduced as a new method over 10 years ago. This method is already established in treating low- and high-grade dysplastic BE, but there is also a recommendation in therapy of non-dysplastic Barrett's esophagus (NDBE), if patients provide a increased cancer risk profile, such as long history of GERD (over 10 years), large hiatal hernia (over 3cm), esophagitis, history of BE with dysplasia or positive family history for gastrointestinal cancer. In Austria 1 - 3% of the population is estimated to develop BE. The treatment numbers in each specialized centers therefore is low. To guarantee a better and more potent data analysis, the establishment of a nationwide registry is crucial. Furthermore this is essential to raise quality assurance and improve outcome and patients will benefit eventually. The aim is the participation of about 10 departments, which are all qualified centers and experts, who offer RFA as a treatment for BE. Thus data can be collected and interpreted more exact due to the increased number of treated patients. For the purpose of data collection a structured electronic form will be used. This will further improve treatment documentation and quality. Documentation will be performed in participating local centers. High priority is patient safety and data de-personalization. Data administration and data input should be performed through qualified medical personal via an online documentation platform. Original patients charts are archived at each center and data de-personalization is guaranteed via consecutive numbering when submitted to the study center. Therefore protection of privacy of participating patients can be assured. The registry should contain basis data of the patient as well as specific treatment data. 1. Patient data Personal identification number (PIN) Sex Age Place of residence - county 2. GERD history Typical, atypical symptoms Leading symptom Since when symptomatically Since when proton-pump inhibitor (PPI) treatment, dose Fundoplication y/n, Type, Date 3. Endoscopy Date of diagnosis - Date of endoscopy Endoscopic Classification (Prague) Histological Classification 4. Treatment Data Catheter type: 360°, 90° Energy: 10/12 J/cm2 Ablation catheter 18, 22, 25, 28, 31mm Ablation from ... to ... ab ore, length Complications 5. Follow Up Date Endoscopic Classification (Prague) Histological Classification Complete remission of intestinal metaplasia (CR-IM) Proton-pump inhibitor (PPI), Dose Inclusion of 500 patients a study period of two years is planned. There will be a possible extension of the study period, if the registry is successful. ### Conditions Module **Conditions:** - Barrett's Esophagus - Radiofrequency Ablation - Gastroesophageal Reflux Disease ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 500 **Type:** ESTIMATED **Patient Registry:** True **Study Type:** OBSERVATIONAL **Target Duration:** 5 Years ### Arms Interventions Module #### Arm Group 1 **Description:** Patients presenting Barrett's Esophagus as a complication of a gastroesophageal reflux disease. **Label:** BE ### Outcomes Module #### Primary Outcomes **Measure:** Number of patients treated with Radiofrequency Ablation for Barrett's Esophagus in Austria **Time Frame:** 5 years **Measure:** Elimination Rate of Barrett's Esophagus in the study population **Time Frame:** 5 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients with a histological confirmed BE, who give their informed consent for participation in an anonymized registry. Exclusion Criteria: * Patients, who do not give informed consent. **Maximum Age:** 75 Years **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Each center should define a system for identification and eligibility screening of patients. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** matthias.paireder@meduniwien.ac.at **Name:** Matthias Paireder, MD **Phone:** +43 1 40400 **Phone Ext:** 56210 **Role:** CONTACT #### Locations **Location 1:** **City:** Vienna **Contacts:** ***Contact 1:*** - **Email:** matthias.paireder@meduniwien.ac.at - **Name:** Matthias Paireder, MD - **Phone:** 0043140400 - **Phone Ext:** 56210 - **Role:** CONTACT ***Contact 2:*** - **Email:** sebastian.schoppmann@meduniwien.ac.at - **Name:** Sebastian F Schoppmann, MD - **Phone:** 0043140400 - **Phone Ext:** 56210 - **Role:** CONTACT **Country:** Austria **Facility:** Medical University of Vienna **Status:** RECRUITING **Zip:** 1090 #### Overall Officials **Official 1:** **Affiliation:** Medical University of Vienna **Name:** Sebastian F Schoppmann, MD **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** Chandrasoma P, Makarewicz K, Wickramasinghe K, Ma Y, Demeester T. A proposal for a new validated histological definition of the gastroesophageal junction. Hum Pathol. 2006 Jan;37(1):40-7. doi: 10.1016/j.humpath.2005.09.019. Epub 2005 Nov 28. **PMID:** 16360414 **Citation:** Fleischer DE, Odze R, Overholt BF, Carroll J, Chang KJ, Das A, Goldblum J, Miller D, Lightdale CJ, Peters J, Rothstein R, Sharma VK, Smith D, Velanovich V, Wolfsen H, Triadafilopoulos G. The case for endoscopic treatment of non-dysplastic and low-grade dysplastic Barrett's esophagus. Dig Dis Sci. 2010 Jul;55(7):1918-31. doi: 10.1007/s10620-010-1218-1. Epub 2010 Apr 20. **PMID:** 20405211 **Citation:** Fleischer DE, Overholt BF, Sharma VK, Reymunde A, Kimmey MB, Chuttani R, Chang KJ, Muthasamy R, Lightdale CJ, Santiago N, Pleskow DK, Dean PJ, Wang KK. Endoscopic radiofrequency ablation for Barrett's esophagus: 5-year outcomes from a prospective multicenter trial. Endoscopy. 2010 Oct;42(10):781-9. doi: 10.1055/s-0030-1255779. Epub 2010 Sep 20. **PMID:** 20857372 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000015154 - Term: Esophageal Motility Disorders - ID: D000003680 - Term: Deglutition Disorders - ID: D000004935 - Term: Esophageal Diseases - ID: D000005767 - Term: Gastrointestinal Diseases - ID: D000004066 - Term: Digestive System Diseases - ID: D000011230 - Term: Precancerous Conditions - ID: D000009369 - Term: Neoplasms ### Condition Browse Module - Browse Branches - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC09 - Name: Ear, Nose, and Throat Diseases ### Condition Browse Module - Browse Leaves - ID: M8880 - Name: Gastroesophageal Reflux - Relevance: HIGH - As Found: Gastroesophageal Reflux - ID: M4765 - Name: Barrett Esophagus - Relevance: HIGH - As Found: Barrett's Esophagus - ID: M17874 - Name: Esophageal Motility Disorders - Relevance: LOW - As Found: Unknown - ID: M17875 - Name: Esophageal Spasm, Diffuse - Relevance: LOW - As Found: Unknown - ID: M6882 - Name: Deglutition Disorders - Relevance: LOW - As Found: Unknown - ID: M8085 - Name: Esophageal Diseases - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M14111 - Name: Precancerous Conditions - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001471 - Term: Barrett Esophagus - ID: D000005764 - Term: Gastroesophageal Reflux ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01516879 **Acronym:** DESCARTES **Brief Title:** Durable Effect of PCSK9 Antibody CompARed wiTh placEbo Study **Official Title:** A Double-Blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Long-Term Tolerability and Durable Efficacy of AMG 145 (Evolocumab) on LDL-C in Hyperlipidemic Subjects #### Organization Study ID Info **ID:** 20110109 #### Organization **Class:** INDUSTRY **Full Name:** Amgen ### Status Module #### Completion Date **Date:** 2013-10-14 **Type:** ACTUAL #### Disp First Post Date **Date:** 2014-05-28 **Type:** ESTIMATED **Disp First Submit Date:** 2014-05-15 **Disp First Submit QC Date:** 2014-05-15 #### Expanded Access Info #### Last Update Post Date **Date:** 2022-07-22 **Type:** ACTUAL **Last Update Submit Date:** 2022-07-15 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2013-10-14 **Type:** ACTUAL #### Results First Post Date **Date:** 2015-09-29 **Type:** ESTIMATED **Results First Submit Date:** 2015-08-28 **Results First Submit QC Date:** 2015-08-28 #### Start Date **Date:** 2012-01-05 **Type:** ACTUAL **Status Verified Date:** 2022-07 #### Study First Post Date **Date:** 2012-01-25 **Type:** ESTIMATED **Study First Submit Date:** 2012-01-18 **Study First Submit QC Date:** 2012-01-20 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Amgen #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** To evaluate the efficacy, safety, and tolerability of 52 weeks of subcutaneous (SC) evolocumab (AMG 145) compared with placebo when added to assigned background lipid-lowering therapy. **Detailed Description:** Eligible participants with screening central laboratory low-density lipoprotein cholesterol (LDL-C) values ≥ 75 mg/dL (1.9 mmol/L) were instructed to follow National Cholesterol Education Program (NCEP) Adult Treatment Panel III (ATP) Therapeutic Lifestyle Changes (TLC) diet and were assigned to 1 of the following 4 background lipid-lowering therapies for a 4-week stabilization period based upon their screening LDL-C and its distance from the individual's required goal as stipulated by their NCEP ATP III risk category: 1. no drug therapy required - diet alone 2. low dose drug therapy required - diet plus atorvastatin 10 mg orally (PO) once daily (QD) 3. high dose drug therapy required - diet plus atorvastatin 80 mg PO QD 4. maximal drug therapy required - diet plus atorvastatin 80 mg PO QD plus ezetimibe 10 mg PO QD. If the participant met entry criteria at the end of the lipid stabilization period they were randomized 2:1 to receive evolocumab 420 mg or placebo subcutaneously once a month for 52 weeks in addition to their background therapy. ### Conditions Module **Conditions:** - Hypercholesterolemia **Keywords:** - Cholesterol - High Cholesterol - Elevated Cholesterol - Raised Cholesterol ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 905 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **Intervention Names:** - Biological: Evolocumab - Drug: Atorvastatin - Drug: Ezetimibe - Other: Diet Only **Label:** Evolocumab **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **Intervention Names:** - Biological: Placebo - Drug: Atorvastatin - Drug: Ezetimibe - Other: Diet Only **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Evolocumab **Description:** Administered by subcutaneous injection once a month **Name:** Evolocumab **Other Names:** - AMG 145 - Repatha **Type:** BIOLOGICAL #### Intervention 2 **Arm Group Labels:** - Placebo **Description:** Administered by subcutaneous injection once a month **Name:** Placebo **Type:** BIOLOGICAL #### Intervention 3 **Arm Group Labels:** - Evolocumab - Placebo **Description:** Background lipid lowering therapy: 10 mg or 80 mg atorvastatin orally once daily. **Name:** Atorvastatin **Type:** DRUG #### Intervention 4 **Arm Group Labels:** - Evolocumab - Placebo **Description:** Background lipid lowering therapy: ezetimibe 10 mg orally once a day **Name:** Ezetimibe **Type:** DRUG #### Intervention 5 **Arm Group Labels:** - Evolocumab - Placebo **Description:** Diet only, no lipid lowering background drug given **Name:** Diet Only **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Cholesterol was measured by means of ultracentrifugation. **Measure:** Percent Change From Baseline in LDL-C at Week 52 **Time Frame:** Baseline and Week 52 #### Secondary Outcomes **Description:** Cholesterol was measured by means of ultracentrifugation. **Measure:** Change From Baseline in LDL-C at Week 52 **Time Frame:** Baseline and Week 52 **Description:** An LDL-C response is defined as LDL-C level \< 70 mg/dL (1.8 mmol/L) at Week 52. **Measure:** Percentage of Participants With an LDL-C Response at Week 52 **Time Frame:** Week 52 **Description:** Cholesterol was measured by means of ultracentrifugation. **Measure:** Percent Change From Baseline in LDL-C at Week 12 **Time Frame:** Baseline and Week 12 **Measure:** Percent Change From Baseline in Total Cholesterol at Week 12 **Time Frame:** Baseline and Week 12 **Measure:** Percent Change From Baseline in Total Cholesterol at Week 52 **Time Frame:** Baseline and Week 52 **Measure:** Percent Change From Baseline in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) at Week 52 **Time Frame:** Baseline and Week 52 **Measure:** Percent Change From Baseline in Apolipoprotein B at Week 52 **Time Frame:** Baseline and Week 52 **Measure:** Percent Change From Baseline in the Total Cholesterol/HDL-C Ratio at Week 52 **Time Frame:** Baseline and Week 52 **Measure:** Percent Change From Baseline in Apolipoprotein B/Apolipoprotein A1 Ratio at Week 52 **Time Frame:** Baseline and Week 52 **Measure:** Percent Change From Baseline in Lipoprotein(a) at Week 52 **Time Frame:** Baseline and Week 52 **Measure:** Percent Change From Baseline in Triglycerides at Week 52 **Time Frame:** Baseline and Week 52 **Measure:** Percent Change From Baseline in High-density Lipoprotein Cholesterol (HDL-C) at Week 52 **Time Frame:** Baseline and Week 52 **Description:** Cholesterol was measured by means of ultracentrifugation. **Measure:** Percent Change From Baseline in Very Low-density Lipoprotein Cholesterol (VLDL-C) at Week 52 **Time Frame:** Baseline and Week 52 **Description:** Cholesterol was measured by means of ultracentrifugation. **Measure:** Percent Change From Week 12 to Week 52 in LDL-C **Time Frame:** Week 12 and Week 52 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Subject has provided informed consent. * Fasting LDL-C ≥ 75 mg/dL and meeting the following LDL-C values on background lipid-lowering therapy: * \< 100 mg/dL for subjects with diagnosed coronary heart disease (CHD) or CHD risk equivalent * \< 130 mg/dL for subjects without diagnosed CHD or CHD risk equivalent * OR on maximal background lipid-lowering therapy defined as atorvastatin 80 mg PO QD and ezetimibe 10 mg PO QD * Fasting triglycerides ≤ 400 mg/dL Exclusion Criteria: * New York Heart Association (NYHA) II-IV heart failure, or last known left ventricular ejection fraction \< 30% * Uncontrolled cardiac arrhythmia * Myocardial infarction, unstable angina, percutaneous coronary intervention (PCI), coronary artery bypass graft (CABG) or stroke within 3 months prior to randomization, type 1 diabetes, newly diagnosed or poorly controlled type 2 diabetes * Uncontrolled hypertension **Maximum Age:** 80 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Birmingham **Country:** United States **Facility:** Research Site **State:** Alabama **Zip:** 35216 **Location 2:** **City:** Little Rock **Country:** United States **Facility:** Research Site **State:** Arkansas **Zip:** 72205 **Location 3:** **City:** Anaheim **Country:** United States **Facility:** Research Site **State:** California **Zip:** 92801 **Location 4:** **City:** Encinitas **Country:** United States **Facility:** Research Site **State:** California **Zip:** 92024 **Location 5:** **City:** Spring Valley **Country:** United States **Facility:** Research Site **State:** California **Zip:** 91978 **Location 6:** **City:** Westlake Village **Country:** United States **Facility:** Research Site **State:** California **Zip:** 91361 **Location 7:** **City:** DeLand **Country:** United States **Facility:** Research Site **State:** Florida **Zip:** 32720 **Location 8:** **City:** Jacksonville **Country:** United States **Facility:** Research Site **State:** Florida **Zip:** 32204 **Location 9:** **City:** Jacksonville **Country:** United States **Facility:** Research Site **State:** Florida **Zip:** 32216 **Location 10:** **City:** Ponte Vedra **Country:** United States **Facility:** Research Site **State:** Florida **Zip:** 32081 **Location 11:** **City:** Atlanta **Country:** United States **Facility:** Research Site **State:** Georgia **Zip:** 30338 **Location 12:** **City:** Atlanta **Country:** United States **Facility:** Research Site **State:** Georgia **Zip:** 30342 **Location 13:** **City:** Savannah **Country:** United States **Facility:** Research Site **State:** Georgia **Zip:** 31406 **Location 14:** **City:** Chicago **Country:** United States **Facility:** Research Site **State:** Illinois **Zip:** 60610 **Location 15:** **City:** Indianapolis **Country:** United States **Facility:** Research Site **State:** Indiana **Zip:** 46260 **Location 16:** **City:** Louisville **Country:** United States **Facility:** Research Site **State:** Kentucky **Zip:** 40213 **Location 17:** **City:** Auburn **Country:** United States **Facility:** Research Site **State:** Maine **Zip:** 04210 **Location 18:** **City:** Bethesda **Country:** United States **Facility:** Research Site **State:** Maryland **Zip:** 20817 **Location 19:** **City:** Chevy Chase **Country:** United States **Facility:** Research Site **State:** Maryland **Zip:** 20815 **Location 20:** **City:** Columbia **Country:** United States **Facility:** Research Site **State:** Maryland **Zip:** 21045 **Location 21:** **City:** Brockton **Country:** United States **Facility:** Research Site **State:** Massachusetts **Zip:** 02301 **Location 22:** **City:** Saint Paul **Country:** United States **Facility:** Research Site **State:** Minnesota **Zip:** 55114 **Location 23:** **City:** Olive Branch **Country:** United States **Facility:** Research Site **State:** Mississippi **Zip:** 38654 **Location 24:** **City:** Las Vegas **Country:** United States **Facility:** Research Site **State:** Nevada **Zip:** 89148 **Location 25:** **City:** Endwell **Country:** United States **Facility:** Research Site **State:** New York **Zip:** 13760 **Location 26:** **City:** New Windsor **Country:** United States **Facility:** Research Site **State:** New York **Zip:** 12553 **Location 27:** **City:** Raleigh **Country:** United States **Facility:** Research Site **State:** North Carolina **Zip:** 27609 **Location 28:** **City:** Raleigh **Country:** United States **Facility:** Research Site **State:** North Carolina **Zip:** 27612 **Location 29:** **City:** Fargo **Country:** United States **Facility:** Research Site **State:** North Dakota **Zip:** 58103 **Location 30:** **City:** Akron **Country:** United States **Facility:** Research Site **State:** Ohio **Zip:** 44311 **Location 31:** **City:** Cincinnati **Country:** United States **Facility:** Research Site **State:** Ohio **Zip:** 45219 **Location 32:** **City:** Cincinnati **Country:** United States **Facility:** Research Site **State:** Ohio **Zip:** 45227 **Location 33:** **City:** Cincinnati **Country:** United States **Facility:** Research Site **State:** Ohio **Zip:** 45246 **Location 34:** **City:** Norman **Country:** United States **Facility:** Research Site **State:** Oklahoma **Zip:** 73069 **Location 35:** **City:** Duncansville **Country:** United States **Facility:** Research Site **State:** Pennsylvania **Zip:** 16635 **Location 36:** **City:** Mount Pleasant **Country:** United States **Facility:** Research Site **State:** South Carolina **Zip:** 29464 **Location 37:** **City:** Rapid City **Country:** United States **Facility:** Research Site **State:** South Dakota **Zip:** 57702 **Location 38:** **City:** Houston **Country:** United States **Facility:** Research Site **State:** Texas **Zip:** 77030 **Location 39:** **City:** Richmond **Country:** United States **Facility:** Research Site **State:** Virginia **Zip:** 23294 **Location 40:** **City:** Renton **Country:** United States **Facility:** Research Site **State:** Washington **Zip:** 98057 **Location 41:** **City:** Seattle **Country:** United States **Facility:** Research Site **State:** Washington **Zip:** 98104 **Location 42:** **City:** Camperdown **Country:** Australia **Facility:** Research Site **State:** New South Wales **Zip:** 2015 **Location 43:** **City:** Maroubra **Country:** Australia **Facility:** Research Site **State:** New South Wales **Zip:** 2035 **Location 44:** **City:** Carina Heights **Country:** Australia **Facility:** Research Site **State:** Queensland **Zip:** 4152 **Location 45:** **City:** Milton **Country:** Australia **Facility:** Research Site **State:** Queensland **Zip:** 4064 **Location 46:** **City:** Fitzroy **Country:** Australia **Facility:** Research Site **State:** Victoria **Zip:** 3065 **Location 47:** **City:** Perth **Country:** Australia **Facility:** Research Site **State:** Western Australia **Zip:** 6000 **Location 48:** **City:** Feldkirch **Country:** Austria **Facility:** Research Site **Zip:** 6807 **Location 49:** **City:** Innsbruck **Country:** Austria **Facility:** Research Site **Zip:** 6020 **Location 50:** **City:** Salzburg **Country:** Austria **Facility:** Research Site **Zip:** 5020 **Location 51:** **City:** Wels **Country:** Austria **Facility:** Research Site **Zip:** 4600 **Location 52:** **City:** Anthée **Country:** Belgium **Facility:** Research Site **Zip:** 5520 **Location 53:** **City:** Bruxelles **Country:** Belgium **Facility:** Research Site **Zip:** 1200 **Location 54:** **City:** Gent **Country:** Belgium **Facility:** Research Site **Zip:** 9000 **Location 55:** **City:** Gozee **Country:** Belgium **Facility:** Research Site **Zip:** 6534 **Location 56:** **City:** Ham **Country:** Belgium **Facility:** Research Site **Zip:** 3945 **Location 57:** **City:** Oostende **Country:** Belgium **Facility:** Research Site **Zip:** 8400 **Location 58:** **City:** Victoria **Country:** Canada **Facility:** Research Site **State:** British Columbia **Zip:** V8T 5G4 **Location 59:** **City:** Bay Roberts **Country:** Canada **Facility:** Research Site **State:** Newfoundland and Labrador **Zip:** A0A 1G0 **Location 60:** **City:** Cambridge **Country:** Canada **Facility:** Research Site **State:** Ontario **Zip:** N1R 6V6 **Location 61:** **City:** London **Country:** Canada **Facility:** Research Site **State:** Ontario **Zip:** N5W 6A2 **Location 62:** **City:** Newmarket **Country:** Canada **Facility:** Research Site **State:** Ontario **Zip:** L3Y 5G8 **Location 63:** **City:** Sudbury **Country:** Canada **Facility:** Research Site **State:** Ontario **Zip:** P3C 5K7 **Location 64:** **City:** Toronto **Country:** Canada **Facility:** Research Site **State:** Ontario **Zip:** M9V 4B4 **Location 65:** **City:** Toronto **Country:** Canada **Facility:** Research Site **State:** Ontario **Zip:** M9W 4L6 **Location 66:** **City:** Pointe-Claire **Country:** Canada **Facility:** Research Site **State:** Quebec **Zip:** H9R 3J1 **Location 67:** **City:** Quebec **Country:** Canada **Facility:** Research Site **Zip:** G1V 4M6 **Location 68:** **City:** Brno **Country:** Czechia **Facility:** Research Site **Zip:** 602 00 **Location 69:** **City:** Brno **Country:** Czechia **Facility:** Research Site **Zip:** 625 00 **Location 70:** **City:** Chomutov **Country:** Czechia **Facility:** Research Site **Zip:** 430 02 **Location 71:** **City:** Hradec Kralove **Country:** Czechia **Facility:** Research Site **Zip:** 500 05 **Location 72:** **City:** Pardubice **Country:** Czechia **Facility:** Research Site **Zip:** 530 02 **Location 73:** **City:** Plzen **Country:** Czechia **Facility:** Research Site **Zip:** 305 99 **Location 74:** **City:** Praha 2 **Country:** Czechia **Facility:** Research Site **Zip:** 120 00 **Location 75:** **City:** Praha 4 **Country:** Czechia **Facility:** Research Site **Zip:** 140 21 **Location 76:** **City:** Praha 5 **Country:** Czechia **Facility:** Research Site **Zip:** 150 06 **Location 77:** **City:** Slany **Country:** Czechia **Facility:** Research Site **Zip:** 274 01 **Location 78:** **City:** Aalborg **Country:** Denmark **Facility:** Research Site **Zip:** 9000 **Location 79:** **City:** Ballerup **Country:** Denmark **Facility:** Research Site **Zip:** 2750 **Location 80:** **City:** Vejle **Country:** Denmark **Facility:** Research Site **Zip:** 7100 **Location 81:** **City:** Baja **Country:** Hungary **Facility:** Research Site **Zip:** 6500 **Location 82:** **City:** Budapest **Country:** Hungary **Facility:** Research Site **Zip:** 1085 **Location 83:** **City:** Budapest **Country:** Hungary **Facility:** Research Site **Zip:** 1115 **Location 84:** **City:** Budapest **Country:** Hungary **Facility:** Research Site **Zip:** 1125 **Location 85:** **City:** Komarom **Country:** Hungary **Facility:** Research Site **Zip:** 2991 **Location 86:** **City:** Pecs **Country:** Hungary **Facility:** Research Site **Zip:** 7624 **Location 87:** **City:** Szeged **Country:** Hungary **Facility:** Research Site **Zip:** 6720 **Location 88:** **City:** Zalaegerszeg **Country:** Hungary **Facility:** Research Site **Zip:** 8900 **Location 89:** **City:** Lyttelton **Country:** South Africa **Facility:** Research Site **State:** Gauteng **Zip:** 0140 **Location 90:** **City:** Amanzimtoti **Country:** South Africa **Facility:** Research Site **State:** KwaZulu-Natal **Zip:** 4126 **Location 91:** **City:** Chatsworth, Durban **Country:** South Africa **Facility:** Research Site **State:** KwaZulu-Natal **Zip:** 4092 **Location 92:** **City:** Observatory **Country:** South Africa **Facility:** Research Site **State:** Western Cape **Zip:** 7925 **Location 93:** **City:** Paarl **Country:** South Africa **Facility:** Research Site **State:** Western Cape **Zip:** 7646 **Location 94:** **City:** Parow **Country:** South Africa **Facility:** Research Site **State:** Western Cape **Zip:** 7505 **Location 95:** **City:** Somerset West **Country:** South Africa **Facility:** Research Site **State:** Western Cape **Zip:** 7130 **Location 96:** **City:** Bloemfontein **Country:** South Africa **Facility:** Research Site **Zip:** 9301 #### Overall Officials **Official 1:** **Affiliation:** Amgen **Name:** MD **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** Toth PP, Sattar N, Blom DJ, Martin SS, Jones SR, Monsalvo ML, Elliott M, Davis M, Somaratne R, Preiss D. Effect of Evolocumab on Lipoprotein Particles. Am J Cardiol. 2018 Feb 1;121(3):308-314. doi: 10.1016/j.amjcard.2017.10.028. Epub 2017 Nov 8. **PMID:** 29221604 **Citation:** Blom DJ, Koren MJ, Roth E, Monsalvo ML, Djedjos CS, Nelson P, Elliott M, Wasserman SM, Ballantyne CM, Holman RR. Evaluation of the efficacy, safety and glycaemic effects of evolocumab (AMG 145) in hypercholesterolaemic patients stratified by glycaemic status and metabolic syndrome. Diabetes Obes Metab. 2017 Jan;19(1):98-107. doi: 10.1111/dom.12788. Epub 2016 Oct 14. **PMID:** 27619750 **Citation:** Blom DJ, Djedjos CS, Monsalvo ML, Bridges I, Wasserman SM, Scott R, Roth E. Effects of Evolocumab on Vitamin E and Steroid Hormone Levels: Results From the 52-Week, Phase 3, Double-Blind, Randomized, Placebo-Controlled DESCARTES Study. Circ Res. 2015 Sep 25;117(8):731-41. doi: 10.1161/CIRCRESAHA.115.307071. Epub 2015 Jul 30. **PMID:** 26228031 **Citation:** Blom DJ, Hala T, Bolognese M, Lillestol MJ, Toth PD, Burgess L, Ceska R, Roth E, Koren MJ, Ballantyne CM, Monsalvo ML, Tsirtsonis K, Kim JB, Scott R, Wasserman SM, Stein EA; DESCARTES Investigators. A 52-week placebo-controlled trial of evolocumab in hyperlipidemia. N Engl J Med. 2014 May 8;370(19):1809-19. doi: 10.1056/NEJMoa1316222. Epub 2014 Mar 29. **PMID:** 24678979 **Citation:** Daviglus ML, Ferdinand KC, Lopez JAG, Wu Y, Monsalvo ML, Rodriguez CJ. Effects of Evolocumab on Low-Density Lipoprotein Cholesterol, Non-High Density Lipoprotein Cholesterol, Apolipoprotein B, and Lipoprotein(a) by Race and Ethnicity: A Meta-Analysis of Individual Participant Data From Double-Blind and Open-Label Extension Studies. J Am Heart Assoc. 2021 Jan 5;10(1):e016839. doi: 10.1161/JAHA.120.016839. Epub 2020 Dec 16. **PMID:** 33325247 **Citation:** Kuchimanchi M, Grover A, Emery MG, Somaratne R, Wasserman SM, Gibbs JP, Doshi S. Population pharmacokinetics and exposure-response modeling and simulation for evolocumab in healthy volunteers and patients with hypercholesterolemia. J Pharmacokinet Pharmacodyn. 2018 Jun;45(3):505-522. doi: 10.1007/s10928-018-9592-y. Epub 2018 May 7. **PMID:** 29736889 **Citation:** Kasichayanula S, Grover A, Emery MG, Gibbs MA, Somaratne R, Wasserman SM, Gibbs JP. Clinical Pharmacokinetics and Pharmacodynamics of Evolocumab, a PCSK9 Inhibitor. Clin Pharmacokinet. 2018 Jul;57(7):769-779. doi: 10.1007/s40262-017-0620-7. **PMID:** 29353350 **Citation:** Sattar N, Toth PP, Blom DJ, Koren MJ, Soran H, Uhart M, Elliott M, Cyrille M, Somaratne R, Preiss D. Effect of the Proprotein Convertase Subtilisin/Kexin Type 9 Inhibitor Evolocumab on Glycemia, Body Weight, and New-Onset Diabetes Mellitus. Am J Cardiol. 2017 Nov 1;120(9):1521-1527. doi: 10.1016/j.amjcard.2017.07.047. Epub 2017 Jul 31. **PMID:** 28844508 **Citation:** Toth PP, Descamps O, Genest J, Sattar N, Preiss D, Dent R, Djedjos C, Wu Y, Geller M, Uhart M, Somaratne R, Wasserman SM; PROFICIO Investigators. Pooled Safety Analysis of Evolocumab in Over 6000 Patients From Double-Blind and Open-Label Extension Studies. Circulation. 2017 May 9;135(19):1819-1831. doi: 10.1161/CIRCULATIONAHA.116.025233. Epub 2017 Mar 1. **PMID:** 28249876 **Citation:** Toth PP, Jones SR, Monsalvo ML, Elliott-Davey M, Lopez JAG, Banach M. Effect of Evolocumab on Non-High-Density Lipoprotein Cholesterol, Apolipoprotein B, and Lipoprotein(a): A Pooled Analysis of Phase 2 and Phase 3 Studies. J Am Heart Assoc. 2020 Mar 3;9(5):e014129. doi: 10.1161/JAHA.119.014129. Epub 2020 Mar 2. **PMID:** 32114889 **Citation:** Wasserman SM, Sabatine MS, Koren MJ, Giugliano RP, Legg JC, Emery MG, Doshi S, Liu T, Somaratne R, Gibbs JP. Comparison of LDL-C Reduction Using Different Evolocumab Doses and Intervals: Biological Insights and Treatment Implications. J Cardiovasc Pharmacol Ther. 2018 Sep;23(5):423-432. doi: 10.1177/1074248418774043. Epub 2018 May 16. **PMID:** 29768954 **Citation:** Schludi B, Giugliano RP, Sabatine MS, Raal FJ, Teramoto T, Koren MJ, Stein EA, Wang H, Monsalvo ML. Time-averaged low-density lipoprotein cholesterol lowering with evolocumab: Pooled analysis of phase 2 trials. J Clin Lipidol. 2022 Jul-Aug;16(4):538-543. doi: 10.1016/j.jacl.2022.05.069. Epub 2022 Jun 6. **PMID:** 35760720 #### See Also Links **Label:** AmgenTrials clinical trials website **URL:** http://www.amgentrials.com ## Derived Section ### Condition Browse Module - Ancestors - ID: D000006949 - Term: Hyperlipidemias - ID: D000050171 - Term: Dyslipidemias - ID: D000052439 - Term: Lipid Metabolism Disorders - ID: D000008659 - Term: Metabolic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M9988 - Name: Hypercholesterolemia - Relevance: HIGH - As Found: Hypercholesterolemia - ID: M10000 - Name: Hyperlipidemias - Relevance: LOW - As Found: Unknown - ID: M10002 - Name: Hyperlipoproteinemias - Relevance: LOW - As Found: Unknown - ID: M26181 - Name: Dyslipidemias - Relevance: LOW - As Found: Unknown - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: M27029 - Name: Lipid Metabolism Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006937 - Term: Hypercholesterolemia ### Intervention Browse Module - Ancestors - ID: D000000924 - Term: Anticholesteremic Agents - ID: D000000960 - Term: Hypolipidemic Agents - ID: D000000963 - Term: Antimetabolites - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000057847 - Term: Lipid Regulating Agents - ID: D000019161 - Term: Hydroxymethylglutaryl-CoA Reductase Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000091362 - Term: PCSK9 Inhibitors - ID: D000015842 - Term: Serine Proteinase Inhibitors - ID: D000011480 - Term: Protease Inhibitors ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Lipd - Name: Lipid Regulating Agents - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: AA - Name: Amino Acids ### Intervention Browse Module - Browse Leaves - ID: M4225 - Name: Antibodies - Relevance: LOW - As Found: Unknown - ID: M351 - Name: Atorvastatin - Relevance: HIGH - As Found: Of each - ID: M10184 - Name: Immunoglobulins - Relevance: LOW - As Found: Unknown - ID: M409 - Name: Ezetimibe - Relevance: HIGH - As Found: Amount - ID: M348166 - Name: Evolocumab - Relevance: HIGH - As Found: Pragmatic - ID: M4243 - Name: Anticholesteremic Agents - Relevance: LOW - As Found: Unknown - ID: M4278 - Name: Hypolipidemic Agents - Relevance: LOW - As Found: Unknown - ID: M4281 - Name: Antimetabolites - Relevance: LOW - As Found: Unknown - ID: M28883 - Name: Lipid Regulating Agents - Relevance: LOW - As Found: Unknown - ID: M21155 - Name: Hydroxymethylglutaryl-CoA Reductase Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M2849 - Name: PCSK9 Inhibitors - Relevance: LOW - As Found: Unknown - ID: M14343 - Name: Protease Inhibitors - Relevance: LOW - As Found: Unknown - ID: M18391 - Name: Serine Proteinase Inhibitors - Relevance: LOW - As Found: Unknown - ID: M19609 - Name: HIV Protease Inhibitors - Relevance: LOW - As Found: Unknown - ID: T18 - Name: Serine - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000069059 - Term: Atorvastatin - ID: D000069438 - Term: Ezetimibe - ID: C000577155 - Term: Evolocumab ### Misc Info Module #### Removed Countries - Country: Czech Republic - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold. #### Event Groups **Group ID:** EG000 **Title:** Placebo **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** EG000 **Other Num Affected:** 76 **Other Num at Risk:** 302 **Serious Number Affected:** 13 **Serious Number At Risk:** 302 **Title:** Placebo **Group ID:** EG001 **Title:** Evolocumab **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** EG001 **Other Num Affected:** 177 **Other Num at Risk:** 599 **Serious Number Affected:** 33 **Serious Number At Risk:** 599 **Title:** Evolocumab **Frequency Threshold:** 5 #### Other Events **Term:** Influenza **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 **Term:** Nasopharyngitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 **Term:** Upper respiratory tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 **Term:** Back pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 #### Serious Events **Term:** Angina pectoris **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 599 **Term:** Angina unstable **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Atrial fibrillation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 302 **Group ID:** EG001 **Num At Risk:** 599 **Term:** Cardiac failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Myocardial infarction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Palpitations **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 599 **Term:** Sinus bradycardia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 302 **Group ID:** EG001 **Num At Risk:** 599 **Term:** Ventricular extrasystoles **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 599 **Term:** Exostosis of external ear canal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Ear and labyrinth disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Vertigo positional **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Ear and labyrinth disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 599 **Term:** Gastritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Haemorrhoids **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Chest pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 302 **Group ID:** EG001 **Num At Risk:** 599 **Term:** Device breakage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Non-cardiac chest pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Biliary tract disorder **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Cholelithiasis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Appendicitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Pneumonia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Skin infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Joint injury **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 302 **Group ID:** EG001 **Num At Risk:** 599 **Term:** Multiple fractures **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 302 **Group ID:** EG001 **Num At Risk:** 599 **Term:** Overdose **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 302 **Group ID:** EG001 **Num At Risk:** 599 **Term:** Road traffic accident **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 302 **Group ID:** EG001 **Num At Risk:** 599 **Term:** Skull fracture **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 302 **Group ID:** EG001 **Num At Risk:** 599 **Term:** Alanine aminotransferase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Aspartate aminotransferase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Blood creatine phosphokinase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Hepatic enzyme increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 302 **Group ID:** EG001 **Num At Risk:** 599 **Term:** Hypomagnesaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Back pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 599 **Term:** Intervertebral disc protrusion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Osteoarthritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Spinal osteoarthritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 302 **Group ID:** EG001 **Num At Risk:** 599 **Term:** Breast cancer **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Ovarian cancer metastatic **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Renal neoplasm **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Uterine cancer **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Convulsion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 302 **Group ID:** EG001 **Num At Risk:** 599 **Term:** Migraine with aura **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Peripheral sensory neuropathy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 302 **Group ID:** EG001 **Num At Risk:** 599 **Term:** Syncope **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Ovarian cyst **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Reproductive system and breast disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Asthma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Pleurisy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Pulmonary embolism **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 599 **Term:** Breast prosthesis implantation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Term:** Hypotension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num At Risk:** 302 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 599 **Time Frame:** 52 weeks ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 303 **Group ID:** BG001 **Value:** 602 **Group ID:** BG002 **Value:** 905 **Units:** Participants ### Group **ID:** BG000 **Title:** Placebo **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. ### Group **ID:** BG001 **Title:** Evolocumab **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 10.3 **Value:** 56.6 #### Measurement **Group ID:** BG001 **Spread:** 10.9 **Value:** 55.9 #### Measurement **Group ID:** BG002 **Spread:** 10.7 **Value:** 56.1 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 162 #### Measurement **Group ID:** BG001 **Value:** 312 #### Measurement **Group ID:** BG002 **Value:** 474 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 141 #### Measurement **Group ID:** BG001 **Value:** 290 #### Measurement **Group ID:** BG002 **Value:** 431 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 3 **Class Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 16 #### Measurement **Group ID:** BG001 **Value:** 41 #### Measurement **Group ID:** BG002 **Value:** 57 **Class Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 23 #### Measurement **Group ID:** BG001 **Value:** 53 #### Measurement **Group ID:** BG002 **Value:** 76 **Class Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 1 **Class Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 249 #### Measurement **Group ID:** BG001 **Value:** 478 #### Measurement **Group ID:** BG002 **Value:** 727 **Class Title:** White #### Measurement **Group ID:** BG000 **Value:** 13 #### Measurement **Group ID:** BG001 **Value:** 26 #### Measurement **Group ID:** BG002 **Value:** 39 **Class Title:** Other #### Measurement **Group ID:** BG000 **Value:** 2 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 2 **Class Title:** Mixed Race ### Measure #### Measurement **Group ID:** BG000 **Value:** 17 #### Measurement **Group ID:** BG001 **Value:** 33 #### Measurement **Group ID:** BG002 **Value:** 50 **Class Title:** Hispanic/Latino #### Measurement **Group ID:** BG000 **Value:** 286 #### Measurement **Group ID:** BG001 **Value:** 569 #### Measurement **Group ID:** BG002 **Value:** 855 **Class Title:** Not Hispanic/Latino ### Measure #### Measurement **Group ID:** BG000 **Value:** 38 #### Measurement **Group ID:** BG001 **Value:** 74 #### Measurement **Group ID:** BG002 **Value:** 112 **Class Title:** Diet Only #### Measurement **Group ID:** BG000 **Value:** 129 #### Measurement **Group ID:** BG001 **Value:** 256 #### Measurement **Group ID:** BG002 **Value:** 385 **Class Title:** Diet + Atorvastatin 10 mg #### Measurement **Group ID:** BG000 **Value:** 73 #### Measurement **Group ID:** BG001 **Value:** 146 #### Measurement **Group ID:** BG002 **Value:** 219 **Class Title:** Diet + Atorvastatin 80 mg #### Measurement **Group ID:** BG000 **Value:** 63 #### Measurement **Group ID:** BG001 **Value:** 126 #### Measurement **Group ID:** BG002 **Value:** 189 **Class Title:** Diet + Atorvastatin 80 mg + Ezetimibe 10 mg ### Measure #### Measurement **Group ID:** BG000 **Spread:** 21.6 **Value:** 104.0 #### Measurement **Group ID:** BG001 **Spread:** 22.1 **Value:** 104.2 #### Measurement **Group ID:** BG002 **Spread:** 22.0 **Value:** 104.1 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 27.2 **Value:** 179.1 #### Measurement **Group ID:** BG001 **Spread:** 27.5 **Value:** 176.8 #### Measurement **Group ID:** BG002 **Spread:** 27.4 **Value:** 177.6 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 26.9 **Value:** 125.6 #### Measurement **Group ID:** BG001 **Spread:** 25.6 **Value:** 124.2 #### Measurement **Group ID:** BG002 **Spread:** 26.1 **Value:** 124.6 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 16.3 **Value:** 87.5 #### Measurement **Group ID:** BG001 **Spread:** 16.3 **Value:** 87.0 #### Measurement **Group ID:** BG002 **Spread:** 16.3 **Value:** 87.2 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 1.11 **Value:** 3.603 #### Measurement **Group ID:** BG001 **Spread:** 1.04 **Value:** 3.597 #### Measurement **Group ID:** BG002 **Spread:** 1.06 **Value:** 3.599 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 0.170 **Value:** 0.586 #### Measurement **Group ID:** BG001 **Spread:** 0.170 **Value:** 0.593 #### Measurement **Group ID:** BG002 **Spread:** 1.70 **Value:** 0.590 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 108.6 **Value:** 89.3 #### Measurement **Group ID:** BG001 **Spread:** 98.5 **Value:** 84.0 #### Measurement **Group ID:** BG002 **Spread:** 102.0 **Value:** 85.8 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 65.8 **Value:** 127.8 #### Measurement **Group ID:** BG001 **Spread:** 63.2 **Value:** 119.8 #### Measurement **Group ID:** BG002 **Spread:** 64.1 **Value:** 122.5 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 16.1 **Value:** 53.5 #### Measurement **Group ID:** BG001 **Spread:** 15.5 **Value:** 52.6 #### Measurement **Group ID:** BG002 **Spread:** 15.7 **Value:** 52.9 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 13.4 **Value:** 21.5 #### Measurement **Group ID:** BG001 **Spread:** 11.4 **Value:** 20.0 #### Measurement **Group ID:** BG002 **Spread:** 12.1 **Value:** 20.5 **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** NUMBER **Title:** Race/Ethnicity, Customized **Unit of Measure:** participants ### Measure 4 **Parameter Type:** NUMBER **Title:** Race/Ethnicity, Customized **Unit of Measure:** participants ### Measure 5 **Parameter Type:** NUMBER **Title:** Background Therapy **Unit of Measure:** participants ### Measure 6 **Description:** Cholesterol was measured by means of ultracentrifugation. Data are provided for the Full Analysis Set (all participants who were randomized and received at least 1 dose of study treatment). **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Low-density Lipoprotein Cholesterol (LDL-C) Concentration **Unit of Measure:** mg/dL ### Measure 7 **Description:** Data are provided for the Full Analysis Set **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Total Cholesterol **Unit of Measure:** mg/dL ### Measure 8 **Description:** Data are provided for the Full Analysis Set **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) Concentration **Unit of Measure:** mg/dL ### Measure 9 **Description:** Data are provided for the Full Analysis Set **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Apolipoprotein B Concentration **Unit of Measure:** mg/dL ### Measure 10 **Description:** Data are provided for the Full Analysis Set **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Total Cholesterol/High Density Lipoprotein-Cholesterol (HDL-C) Ratio **Unit of Measure:** ratio ### Measure 11 **Description:** Data are provided for the Full Analysis Set **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Apolipoprotein B/Apolipoprotein A-1 Ratio **Unit of Measure:** ratio ### Measure 12 **Description:** Data are provided for the Full Analysis Set **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Lipoprotein(a) Concentration **Unit of Measure:** nmol/L ### Measure 13 **Description:** Data are provided for the Full Analysis Set **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Triglycerides Concentration **Unit of Measure:** mg/dL ### Measure 14 **Description:** Data are providedfor the Full Analysis Set **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** High-density Lipoprotein Cholesterol (HDL-C) Concentration **Unit of Measure:** mg/dL ### Measure 15 **Description:** Data are provided for the Full Analysis Set **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Very Low-density Lipoprotein Cholesterol (VLDL-C) Concentration **Unit of Measure:** mg/dL ## Results Section - More Information Module ### Certain Agreement **Other Details:** The Clinical Trial Agreement generally does not restrict an investigator's discussion of trial results after completion. The Agreement permits Amgen a limited period of time to review material discussing trial results (typically up to 45 days and possible extension). Amgen may remove confidential information, but authors have final control and approval of publication content. For multicenter studies, the investigator agrees not to publish any results before the first multi-center publication. **Restriction Type:** OTHER **Restrictive Agreement:** True ### Point of Contact **Organization:** Amgen Inc. **Phone:** 866-572-6436 **Title:** Study Director ## Results Section - Outcome Measures Module ### Outcome Measure 1 #### Analysis **CI Lower Limit:** -61.08 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** -52.85 **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 2.10 **Estimate Comment:** Treatment difference using placebo as the reference. **Group Description:** The null hypothesis was that there was no mean difference in the percent change from Baseline at Week 52 in LDL-C between evolocumab 420 mg and placebo, and the alternative hypothesis was that a mean difference did exist. **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** LS Mean Treatment Difference **Parameter Value:** -56.97 **Statistical Comment:** The model included treatment group, stratification factor, scheduled visit and the interaction of treatment with scheduled visit as covariates. **Statistical Method:** Repeated measures linear effects model **Tested Non-Inferiority:** ### Outcome Measure 2 #### Analysis **CI Lower Limit:** -62.3 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** -53.3 **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 2.3 **Estimate Comment:** Treatment difference using placebo as the reference. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** LS Mean Treatment Difference **Parameter Value:** -57.8 **Statistical Comment:** The model includes treatment group, stratification factor, scheduled visit and the interaction of treatment with scheduled visit as covariates. **Statistical Method:** Repeated measures linear effects model **Tested Non-Inferiority:** ### Outcome Measure 3 #### Analysis **CI Lower Limit:** 70.8 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 79.7 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** Treatment difference using placebo as the reference. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** Treatment Difference **Parameter Value:** 75.8 **Statistical Comment:** CMH test stratified by the stratification factor. For testing, non-achievement was imputed for participants with a missing value at Week 52. **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** ### Outcome Measure 4 #### Analysis **CI Lower Limit:** -60.57 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** -54.45 **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 1.56 **Estimate Comment:** Treatment difference using placebo as the reference. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** LS Mean Treatment Difference **Parameter Value:** -57.51 **Statistical Comment:** The model includes treatment group, stratification factor, scheduled visit and the interaction of treatment with scheduled visit as covariates. **Statistical Method:** Repeated measures linear effects model **Tested Non-Inferiority:** ### Outcome Measure 5 #### Analysis **CI Lower Limit:** -37.19 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** -33.11 **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 1.04 **Estimate Comment:** Treatment difference using placebo as the reference. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** LS Mean Treatment Difference **Parameter Value:** -35.15 **Statistical Comment:** The model includes treatment group, stratification factor, scheduled visit and the interaction of treatment with scheduled visit as covariates. **Statistical Method:** Repeated measures linear effects model **Tested Non-Inferiority:** ### Outcome Measure 6 #### Analysis **CI Lower Limit:** -36.21 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** -30.68 **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 1.41 **Estimate Comment:** Treatment difference using placebo as the reference. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** LS Mean Treatment Difference **Parameter Value:** -33.45 **Statistical Comment:** The model includes treatment group, stratification factor, scheduled visit and the interaction of treatment with scheduled visit as covariates. **Statistical Method:** Repeated measures linear effects model **Tested Non-Inferiority:** ### Outcome Measure 7 #### Analysis **CI Lower Limit:** -54.25 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** -46.28 **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 2.03 **Estimate Comment:** Treatment difference using placebo as the reference. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** LS Mean Treatment Difference **Parameter Value:** -50.27 **Statistical Comment:** The model includes treatment group, stratification factor, scheduled visit and the interaction of treatment with scheduled visit as covariates. **Statistical Method:** Repeated measures linear effects model **Tested Non-Inferiority:** ### Outcome Measure 8 #### Analysis **CI Lower Limit:** -47.56 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** -40.85 **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 1.71 **Estimate Comment:** Treatment difference using placebo as the reference. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** LS Mean Treatment Difference **Parameter Value:** -44.21 **Statistical Comment:** The model includes treatment group, stratification factor, scheduled visit and the interaction of treatment with scheduled visit as covariates. **Statistical Method:** Repeated measures linear effects model **Tested Non-Inferiority:** ### Outcome Measure 9 #### Analysis **CI Lower Limit:** -40.41 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** -33.87 **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 1.67 **Estimate Comment:** Treatment difference using placebo as the reference. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** LS Mean Treatment Difference **Parameter Value:** -37.14 **Statistical Comment:** The model includes treatment group, stratification factor, scheduled visit and the interaction of treatment with scheduled visit as covariates. **Statistical Method:** Repeated measures linear effects model **Tested Non-Inferiority:** ### Outcome Measure 10 #### Analysis **CI Lower Limit:** -49.79 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** -42.63 **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 1.82 **Estimate Comment:** Treatment difference using placebo as the reference. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** LS Mean Treatment Difference **Parameter Value:** -46.21 **Statistical Comment:** The model includes treatment group, stratification factor, scheduled visit and the interaction of treatment with scheduled visit as covariates. **Statistical Method:** Repeated measures linear effects model **Tested Non-Inferiority:** ### Outcome Measure 11 #### Analysis **CI Lower Limit:** -26.15 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** -18.55 **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 1.94 **Estimate Comment:** Treatment difference using placebo as the reference. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** LS Mean Treatment Difference **Parameter Value:** -22.35 **Statistical Comment:** The model includes treatment group, stratification factor, scheduled visit and the interaction of treatment with scheduled visit as covariates. **Statistical Method:** Repeated measures linear effects model **Tested Non-Inferiority:** ### Outcome Measure 12 #### Analysis **CI Lower Limit:** -17.21 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** -5.86 **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 2.89 **Estimate Comment:** Treatment difference using placebo as the reference. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** LS Mean Treatment Difference **Parameter Value:** -11.54 **Statistical Comment:** The model includes treatment group, stratification factor, scheduled visit and the interaction of treatment with scheduled visit as covariates. **Statistical Method:** Repeated measures linear effects model **Tested Non-Inferiority:** ### Outcome Measure 13 #### Analysis **CI Lower Limit:** 3.28 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 7.56 **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 1.09 **Estimate Comment:** Treatment difference using placebo as the reference. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** LS Mean Treatment Difference **Parameter Value:** 5.42 **Statistical Comment:** The model includes treatment group, stratification factor, scheduled visit and the interaction of treatment with scheduled visit as covariates. **Statistical Method:** Repeated measures linear effects model **Tested Non-Inferiority:** ### Outcome Measure 14 #### Analysis **CI Lower Limit:** -40.23 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** -18.08 **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 5.64 **Estimate Comment:** Treatment difference using placebo as the reference. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** <0.001 **P-Value Comment:** **Parameter Type:** LS Mean Treatment Difference **Parameter Value:** -29.15 **Statistical Comment:** The model includes treatment group, stratification factor, scheduled visit and the interaction of treatment with scheduled visit as covariates. **Statistical Method:** Repeated measures linear effects model **Tested Non-Inferiority:** ### Outcome Measure 15 #### Analysis **CI Lower Limit:** -3.76 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 3.48 **CI Upper Limit Comment:** **Dispersion Type:** STANDARD_ERROR_OF_MEAN **Dispersion Value:** 1.84 **Estimate Comment:** Treatment difference using placebo as the reference. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER_LEGACY **Other Analysis Description:** **P-Value:** 0.94 **P-Value Comment:** **Parameter Type:** LS Mean Treatment Difference **Parameter Value:** -0.14 **Statistical Comment:** The ANCOVA model includes treatment group and stratification factor as covariates. **Statistical Method:** ANCOVA **Tested Non-Inferiority:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.75 - **Upper Limit:** - **Value:** 6.83 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.24 - **Upper Limit:** - **Value:** -50.14 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.9 - **Upper Limit:** - **Value:** 5.1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.4 - **Upper Limit:** - **Value:** -52.7 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 4.1 - **Spread:** - **Upper Limit:** 10.1 - **Value:** 6.4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 78.8 - **Spread:** - **Upper Limit:** 85.3 - **Value:** 82.3 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.31 - **Upper Limit:** - **Value:** 3.17 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.96 - **Upper Limit:** - **Value:** -54.35 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.87 - **Upper Limit:** - **Value:** 2.85 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.63 - **Upper Limit:** - **Value:** -32.30 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.16 - **Upper Limit:** - **Value:** 5.26 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.84 - **Upper Limit:** - **Value:** -28.18 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.68 - **Upper Limit:** - **Value:** 8.44 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.21 - **Upper Limit:** - **Value:** -41.82 **Title:** #### Outcome Measure 8 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.41 - **Upper Limit:** - **Value:** 2.94 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.02 - **Upper Limit:** - **Value:** -41.26 **Title:** #### Outcome Measure 9 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.37 - **Upper Limit:** - **Value:** 6.47 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.99 - **Upper Limit:** - **Value:** -30.67 **Title:** #### Outcome Measure 10 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.50 - **Upper Limit:** - **Value:** 4.46 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.09 - **Upper Limit:** - **Value:** -41.75 **Title:** #### Outcome Measure 11 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.62 - **Upper Limit:** - **Value:** -5.37 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.19 - **Upper Limit:** - **Value:** -27.72 **Title:** #### Outcome Measure 12 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.39 - **Upper Limit:** - **Value:** 8.99 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.72 - **Upper Limit:** - **Value:** -2.55 **Title:** #### Outcome Measure 13 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.90 - **Upper Limit:** - **Value:** 0.35 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.65 - **Upper Limit:** - **Value:** 5.77 **Title:** #### Outcome Measure 14 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 4.69 - **Upper Limit:** - **Value:** 31.89 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 3.36 - **Upper Limit:** - **Value:** 2.74 **Title:** #### Outcome Measure 15 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.56 - **Upper Limit:** - **Value:** 2.57 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.14 - **Upper Limit:** - **Value:** 2.44 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Cholesterol was measured by means of ultracentrifugation. **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Full Analysis Set (all randomized subjects who received at least 1 dose of study drug). **Reporting Status:** POSTED **Time Frame:** Baseline and Week 52 **Title:** Percent Change From Baseline in LDL-C at Week 52 **Type:** PRIMARY **Unit of Measure:** percent change ##### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG000 **Title:** Placebo ##### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG001 **Title:** Evolocumab #### Outcome Measure 2 **Description:** Cholesterol was measured by means of ultracentrifugation. **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Full Analysis Set **Reporting Status:** POSTED **Time Frame:** Baseline and Week 52 **Title:** Change From Baseline in LDL-C at Week 52 **Type:** SECONDARY **Unit of Measure:** mg/dL ##### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG000 **Title:** Placebo ##### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG001 **Title:** Evolocumab #### Outcome Measure 3 **Description:** An LDL-C response is defined as LDL-C level \< 70 mg/dL (1.8 mmol/L) at Week 52. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** Full Analysis Set **Reporting Status:** POSTED **Time Frame:** Week 52 **Title:** Percentage of Participants With an LDL-C Response at Week 52 **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG000 **Title:** Placebo ##### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG001 **Title:** Evolocumab #### Outcome Measure 4 **Description:** Cholesterol was measured by means of ultracentrifugation. **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Full Analysis set **Reporting Status:** POSTED **Time Frame:** Baseline and Week 12 **Title:** Percent Change From Baseline in LDL-C at Week 12 **Type:** SECONDARY **Unit of Measure:** percent change ##### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG000 **Title:** Placebo ##### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG001 **Title:** Evolocumab #### Outcome Measure 5 **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Full Analysis Set **Reporting Status:** POSTED **Time Frame:** Baseline and Week 12 **Title:** Percent Change From Baseline in Total Cholesterol at Week 12 **Type:** SECONDARY **Unit of Measure:** percent change ##### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG000 **Title:** Placebo ##### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG001 **Title:** Evolocumab #### Outcome Measure 6 **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Full Analysis Set **Reporting Status:** POSTED **Time Frame:** Baseline and Week 52 **Title:** Percent Change From Baseline in Total Cholesterol at Week 52 **Type:** SECONDARY **Unit of Measure:** percent change ##### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG000 **Title:** Placebo ##### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG001 **Title:** Evolocumab #### Outcome Measure 7 **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Full Analysis Set **Reporting Status:** POSTED **Time Frame:** Baseline and Week 52 **Title:** Percent Change From Baseline in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) at Week 52 **Type:** SECONDARY **Unit of Measure:** percent change ##### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG000 **Title:** Placebo ##### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG001 **Title:** Evolocumab #### Outcome Measure 8 **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Full analysis set **Reporting Status:** POSTED **Time Frame:** Baseline and Week 52 **Title:** Percent Change From Baseline in Apolipoprotein B at Week 52 **Type:** SECONDARY **Unit of Measure:** percent change ##### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG000 **Title:** Placebo ##### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG001 **Title:** Evolocumab #### Outcome Measure 9 **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Full Analysis Set **Reporting Status:** POSTED **Time Frame:** Baseline and Week 52 **Title:** Percent Change From Baseline in the Total Cholesterol/HDL-C Ratio at Week 52 **Type:** SECONDARY **Unit of Measure:** percent change ##### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG000 **Title:** Placebo ##### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG001 **Title:** Evolocumab #### Outcome Measure 10 **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Full Analysis Set **Reporting Status:** POSTED **Time Frame:** Baseline and Week 52 **Title:** Percent Change From Baseline in Apolipoprotein B/Apolipoprotein A1 Ratio at Week 52 **Type:** SECONDARY **Unit of Measure:** percent change ##### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG000 **Title:** Placebo ##### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG001 **Title:** Evolocumab #### Outcome Measure 11 **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Full Analysis Set **Reporting Status:** POSTED **Time Frame:** Baseline and Week 52 **Title:** Percent Change From Baseline in Lipoprotein(a) at Week 52 **Type:** SECONDARY **Unit of Measure:** percent change ##### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG000 **Title:** Placebo ##### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG001 **Title:** Evolocumab #### Outcome Measure 12 **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Full Analysis Set **Reporting Status:** POSTED **Time Frame:** Baseline and Week 52 **Title:** Percent Change From Baseline in Triglycerides at Week 52 **Type:** SECONDARY **Unit of Measure:** percent change ##### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG000 **Title:** Placebo ##### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG001 **Title:** Evolocumab #### Outcome Measure 13 **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Full Analysis Set **Reporting Status:** POSTED **Time Frame:** Baseline and Week 52 **Title:** Percent Change From Baseline in High-density Lipoprotein Cholesterol (HDL-C) at Week 52 **Type:** SECONDARY **Unit of Measure:** percent change ##### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG000 **Title:** Placebo ##### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG001 **Title:** Evolocumab #### Outcome Measure 14 **Description:** Cholesterol was measured by means of ultracentrifugation. **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** Full Analysis Set **Reporting Status:** POSTED **Time Frame:** Baseline and Week 52 **Title:** Percent Change From Baseline in Very Low-density Lipoprotein Cholesterol (VLDL-C) at Week 52 **Type:** SECONDARY **Unit of Measure:** percent change ##### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG000 **Title:** Placebo ##### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG001 **Title:** Evolocumab #### Outcome Measure 15 **Description:** Cholesterol was measured by means of ultracentrifugation. **Dispersion Type:** Standard Error **Parameter Type:** LEAST_SQUARES_MEAN **Population Description:** The Effect Durability Analysis Set included participants in the FAS who adhered to the scheduled study drug and had nonmissing LDL-C values at Baseline, Week 12 and Week 52. **Reporting Status:** POSTED **Time Frame:** Week 12 and Week 52 **Title:** Percent Change From Week 12 to Week 52 in LDL-C **Type:** SECONDARY **Unit of Measure:** percent change ##### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG000 **Title:** Placebo ##### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** OG001 **Title:** Evolocumab ### Participant Flow Module #### Group **Description:** Participants received placebo subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** FG000 **Title:** Placebo #### Group **Description:** Participants received evolocumab 420 mg subcutaneously once a month for 52 weeks in addition to background lipid-lowering therapy. **ID:** FG001 **Title:** Evolocumab #### Period **Title:** Overall Study ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 9 ###### Reason **Group ID:** FG001 **Number of Subjects:** 11 ##### Withdraw **Type:** Death ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 2 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 11 ##### Withdraw **Type:** Other ###### Reason **Group ID:** FG000 **Number of Subjects:** 5 ###### Reason **Group ID:** FG001 **Number of Subjects:** 10 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 303 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 602 ##### Milestone **Type:** Received Treatment ###### Achievement **Group ID:** FG000 **Number of Subjects:** 302 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 599 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 287 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 568 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 16 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 34 **Pre-Assignment Details:** Patients were assigned to 1 of 4 background lipid-lowering regimens for a 4-12 week stabilization period: diet alone, diet and 10 mg atorvastatin daily, diet and 80 mg atorvastatin daily, or diet, 80 mg atorvastatin and 10 mg ezetimibe daily. Patients meeting criteria were randomized 2:1 to evolocumab or placebo, stratified by background therapy. **Recruitment Details:** Adults with fasting low-density lipoprotein cholesterol (LDL-C) ≥ 75 mg/dL and triglycerides ≤ 400 mg/dL were eligible. The first patient enrolled on 5 January 2012 and the last patient enrolled on 12 October 2012. All patients were counseled on the National Cholesterol Education Program Adult Treatment Panel III Therapeutic Lifestyle Changes diet. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT04678479 **Acronym:** CDD **Brief Title:** Ganaxolone Expanded Access Program Compassionate Use **Official Title:** Expanded Access With Adjunctive Ganaxolone Treatment in Children and Young Adults With Cyclin Dependent Kinase-like 5 Deficiency Disorder #### Organization Study ID Info **ID:** 1042-CDD-EAP-3005 #### Organization **Class:** INDUSTRY **Full Name:** Marinus Pharmaceuticals ### Status Module #### Expanded Access Info #### Last Update Post Date **Date:** 2024-01-22 **Type:** ACTUAL **Last Update Submit Date:** 2024-01-18 **Overall Status:** NO_LONGER_AVAILABLE **Status Verified Date:** 2024-01 #### Study First Post Date **Date:** 2020-12-22 **Type:** ACTUAL **Study First Submit Date:** 2020-12-16 **Study First Submit QC Date:** 2020-12-16 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Marinus Pharmaceuticals #### Responsible Party **Type:** SPONSOR ### Description Module **Brief Summary:** The primary objective is to provide GNX to patients ≥ 2 years with CDD-related seizures who are refractory to, or intolerant of, standard therapy. **Detailed Description:** This is a multi-center, long-term, open-label, expanded access protocol of adjunctive GNX treatment in children, adolescents, and adults with CDD. Patients with inadequate seizure control on their current anti seizure medications at therapeutic doses will be eligible for enrollment. ### Conditions Module **Conditions:** - CDKL5 Disorder ### Design Module #### Expanded Access Types **Individual:** True **Treatment:** True **Study Type:** EXPANDED_ACCESS ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** ganaxolone **Name:** Ganaxolone **Type:** DRUG ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Molecular confirmation of a pathogenic CDKL5 variant, early onset, difficult to control seizures, and neurodevelopmental impairment are required. Male or female patients aged ≥ 2 years. In the opinion of the investigator, the patient has inadequate seizure control on current anti-seizure medications at therapeutic doses. Exclusion Criteria: - **Minimum Age:** 2 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Overall Officials **Official 1:** **Affiliation:** Marinus Pharmaceuticals, Inc. **Name:** Paula Bokesch, MD, FAAP **Role:** STUDY_DIRECTOR **Official 2:** **Affiliation:** Marinus Pharmaceuticals, Inc. **Name:** Igor Grachev, MD, PhD **Role:** STUDY_DIRECTOR ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: Rare - Name: Rare Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: T1938 - Name: Dravet Syndrome - Relevance: HIGH - As Found: CDKL5 Disorder - ID: T1024 - Name: CDKL5 Deficiency Disorder - Relevance: HIGH - As Found: CDKL5 Disorder ### Intervention Browse Module - Ancestors - ID: D000081227 - Term: Neurosteroids - ID: D000018377 - Term: Neurotransmitter Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018757 - Term: GABA Modulators - ID: D000018682 - Term: GABA Agents ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M347103 - Name: Ganaxolone - Relevance: HIGH - As Found: Meningococcal Vaccine - ID: M2291 - Name: Neurosteroids - Relevance: LOW - As Found: Unknown - ID: M20504 - Name: Neurotransmitter Agents - Relevance: LOW - As Found: Unknown - ID: M20827 - Name: GABA Modulators - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000105051 - Term: Ganaxolone ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06352879 **Brief Title:** Prevalence, Types and Risk Factors of Urinary Incontinence Among Women in Baghdad, Iraq. **Official Title:** Prevalence, Types and Risk Factors of Urinary Incontinence Among Women Attending Primary Healthcare Centers in Baghdad, Iraq. #### Organization Study ID Info **ID:** 18-Al-KindyCM #### Organization **Class:** OTHER **Full Name:** Al-Kindy College of Medicine ### Status Module #### Completion Date **Date:** 2024-06-15 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-04-08 **Type:** ACTUAL **Last Update Submit Date:** 2024-04-03 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-05-31 **Type:** ESTIMATED #### Start Date **Date:** 2024-03-01 **Type:** ACTUAL **Status Verified Date:** 2024-04 #### Study First Post Date **Date:** 2024-04-08 **Type:** ACTUAL **Study First Submit Date:** 2024-04-03 **Study First Submit QC Date:** 2024-04-03 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Al-Kindy College of Medicine #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The goal of this observational study is to observe and describe the prevalence, types, and risk factors of urinary incontinence (UI) in adult women attending primary healthcare centers in Baghdad, Iraq. The main questions it aims to answer are: * What is the prevalence of incontinence among the targeted group? * What are the types of incontinence and what is the frequency of each type among the targeted group? * What is the effect of several risk factors on the occurrence and severity of urinary incontinence (including age, job, sexual activity, chronic constipation, chronic cough, parity, largest birth weight, and body mass index)? Participants will be asked to fill out a questionnaire consisting of the Arabic International Consultation On Incontinence Questionnaire-Urinary Incontinence Short Form (Arabic ICIQ-UI SF) in addition to questions about the aforementioned risk factors. ### Conditions Module **Conditions:** - Urinary Incontinence - Urge Incontinence - Stress Incontinence **Keywords:** - Urinary Incontinence - Urge incontinence - Stress incontinence - Mixed incontinence ### Design Module #### Design Info **Observational Model:** CASE_CONTROL **Time Perspective:** CROSS_SECTIONAL #### Enrollment Info **Count:** 384 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Outcomes Module #### Primary Outcomes **Description:** Arabic International Consultation On Incontinence Questionnaire-Urinary Incontinence Short Form (ICIQ-UI SF) consists of three-scored items designed to evaluate the frequency of urine leaks (score 0-5), the amount of leaked urine (score 0-6), and the effect urine leakage on the quality of life of the participant (score 0-10). In addition, an unscored item to evaluate the sensed causes of leakage. The scores of the scored items are added together to provide a total score, which ranges from zero to 21. The higher the score, the more severe the urine leakage. **Measure:** Arabic International Consultation On Incontinence Questionnaire-Urinary Incontinence Short Form (Arabic ICIQ-UI SF) **Time Frame:** 3 months #### Secondary Outcomes **Description:** the study aim to study the effect of several risk factors on the occurrence and severity of urinary incontinence (including age, job, sexual activity, chronic constipation, chronic cough, parity, largest birth weight, and body mass index). **Measure:** Risk factors for urinary incontinence **Time Frame:** 3 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * women * Attending the primary healthcare centers in Baghdad, Iraq * older than 18 years Exclusion Criteria: * Patients who refuse to participate in the study. **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Adult women attending primary healthcare centers in Baghdad, Iraq ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** harthkamber@kmc.uobaghdad.edu.iq **Name:** Harth Mohamed Kamber, FICMS (Uro.) **Phone:** 07706029071 **Role:** CONTACT #### Locations **Location 1:** **City:** Baghdad **Contacts:** ***Contact 1:*** - **Email:** harthkamber@kmc.uobaghdad.edu.iq - **Name:** Harth Mohamed Kamber, FICMS (Uro.) - **Phone:** 009647706029071 - **Role:** CONTACT ***Contact 2:*** - **Name:** Shahad Hameed Kadhom, MBChB - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Iraq **Facility:** Baghdad Al- Karkh Health Directorate **Status:** RECRUITING **Location 2:** **City:** Baghdad **Contacts:** ***Contact 1:*** - **Email:** harthkamber@kmc.uobaghdad.edu.iq - **Name:** Harth Mohamed Kamber, FICMS (Uro.) - **Phone:** 009647706029071 - **Role:** CONTACT ***Contact 2:*** - **Name:** Shahad Hameed Kadhom, MBChB - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Iraq **Facility:** Baghdad Al-Russafa Health Directorate **Status:** RECRUITING #### Overall Officials **Official 1:** **Affiliation:** University of Baghdad / Alkindy collage of medicine **Name:** Harth Mohamed Kamber, FICMS (Uro.) **Role:** STUDY_CHAIR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014555 - Term: Urination Disorders - ID: D000014570 - Term: Urologic Diseases - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000052801 - Term: Male Urogenital Diseases - ID: D000059411 - Term: Lower Urinary Tract Symptoms - ID: D000020924 - Term: Urological Manifestations - ID: D000001526 - Term: Behavioral Symptoms - ID: D000019960 - Term: Elimination Disorders - ID: D000001523 - Term: Mental Disorders ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BXM - Name: Behaviors and Mental Disorders ### Condition Browse Module - Browse Leaves - ID: M17299 - Name: Urinary Incontinence - Relevance: HIGH - As Found: Urinary Incontinence - ID: M7936 - Name: Enuresis - Relevance: HIGH - As Found: Urinary Incontinence - ID: M17300 - Name: Urinary Incontinence, Stress - Relevance: HIGH - As Found: Stress Incontinence - ID: M27168 - Name: Urinary Incontinence, Urge - Relevance: HIGH - As Found: Urge Incontinence - ID: M27171 - Name: Nocturnal Enuresis - Relevance: LOW - As Found: Unknown - ID: M17305 - Name: Urination Disorders - Relevance: LOW - As Found: Unknown - ID: M17319 - Name: Urologic Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M29464 - Name: Lower Urinary Tract Symptoms - Relevance: LOW - As Found: Unknown - ID: M22659 - Name: Urological Manifestations - Relevance: LOW - As Found: Unknown - ID: M4818 - Name: Behavioral Symptoms - Relevance: LOW - As Found: Unknown - ID: M21832 - Name: Elimination Disorders - Relevance: LOW - As Found: Unknown - ID: M4815 - Name: Mental Disorders - Relevance: LOW - As Found: Unknown - ID: M14473 - Name: Psychotic Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000014549 - Term: Urinary Incontinence - ID: D000004775 - Term: Enuresis - ID: D000014550 - Term: Urinary Incontinence, Stress - ID: D000053202 - Term: Urinary Incontinence, Urge ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02120079 **Brief Title:** The Utility of IVCM to Assess Cellular Response and Efficacy of Long-term Topical Steroid Treatment in Patients With DED **Official Title:** The Utility of in Vivo Confocal Microscopy (IVCM) to Assess Cellular Response and Efficacy of Long-term Topical Steroid Treatment in Patients With Dry Eye Disease (DED) **NCT ID Aliases:** - NCT02106377 #### Organization Study ID Info **ID:** 13-150H #### Organization **Class:** OTHER **Full Name:** Tufts Medical Center ### Status Module #### Completion Date **Date:** 2018-01-28 **Type:** ACTUAL #### Disp First Post Date **Date:** 2018-05-08 **Type:** ACTUAL **Disp First Submit Date:** 2018-05-02 **Disp First Submit QC Date:** 2018-05-02 #### Expanded Access Info #### Last Update Post Date **Date:** 2022-03-23 **Type:** ACTUAL **Last Update Submit Date:** 2022-02-24 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2017-04 **Type:** ACTUAL #### Results First Post Date **Date:** 2022-03-23 **Type:** ACTUAL **Results First Submit Date:** 2022-02-24 **Results First Submit QC Date:** 2022-02-24 #### Start Date **Date:** 2014-02 **Status Verified Date:** 2022-02 #### Study First Post Date **Date:** 2014-04-22 **Type:** ESTIMATED **Study First Submit Date:** 2014-02-12 **Study First Submit QC Date:** 2014-04-18 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Tufts Medical Center #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** This research study is looking to see if in vivo confocal microscopy (IVCM) imaging can be used to confirm clinical findings (which are noted by the doctor during an eye exam) and measure the immune response to the inflammation in the subject's cornea (the front part of the eyeball). Additionally, this study is trying to determine the effectiveness of two eye-drops, Lotemax and artificial tears, in treating the inflammation associated with DED by measuring changes in immune cells with IVCM imaging. The subject will be treated with either Lotemax (loteprednol) or artificial tears (a lubricating eye drop with no medication). Lotemax is an FDA-approved steroid eye-drop that is often used to treat inflammation associated with DED. Artificial tears are approved by the FDA for treatment of dryness associated with DED. Thus, this study is designed to determine the effects of the administration of a topical steroid, Lotemax, over a treatment period of 6 weeks, using novel methods of detecting efficacy. In order to achieve the aforementioned goal, subjects will be prospectively randomized to one of two treatment arms - Lotemax or artificial tear. Both groups will follow the same study schedule. **Detailed Description:** IVCM is a non-invasive imaging technique that images the cornea at a cellular level with 800x magnification, using a scanning laser. The laser is used to map the cornea, and will not damage or harm the subject's eye. Studies have shown that IVCM can be used to study cells and nerves within the cornea, providing a better understanding of how the cornea reacts to irritants. IVCM has recently been used by the investigator to assess the extent of eye inflammation in cases of dry eye patients. Current steroid therapy in dry eye disease (DED) is comprised of a 2 week duration of pulse therapy, administered twice daily (to avoid adverse effects associated with long-term steroid use). This time frame is often too short to meaningfully resolve the inflammation associated with DED. DED often occurs when there is a decrease in the eye's tear production or if there is an increase in the evaporation of the tear film (a thin layer of tears that keep the eye moist). Eye irritation and inflammation (swelling) is often associated with DED because the surface of the eye is unable to maintain a normal level of moisture. ### Conditions Module **Conditions:** - Dry Eye Disease **Keywords:** - Dry Eye ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 38 **Type:** ACTUAL **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension supplied by Bausch \& Lomb, Inc. Lotemax (loteprednol etabonate) 0.5% has been approved by the FDA for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. The medication will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. **Intervention Names:** - Drug: Lotemax - Diagnostic Test: In Vivo Confocal Microscopy (IVCM) **Label:** Lotemax **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. **Intervention Names:** - Drug: Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) - Diagnostic Test: In Vivo Confocal Microscopy (IVCM) **Label:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Lotemax **Description:** Lotemax (loteprednol etabonate) 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. **Name:** Lotemax **Other Names:** - Lotemax (loteprednol etabonate) 0.5% **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) **Description:** Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. **Name:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) **Other Names:** - Soothe Tired Eyes Lubricant Eye Drop (Bausch & Lomb Inc.) **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - Lotemax - Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) **Description:** In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **Name:** In Vivo Confocal Microscopy (IVCM) **Type:** DIAGNOSTIC_TEST ### Outcomes Module #### Primary Outcomes **Description:** Density (in cells/mm2) of Corneal Immune Dendritiform Cells **Measure:** IVCM for Density of Corneal Immune Dendritiform Cells **Time Frame:** 2 Weeks **Description:** Density (in cells/mm2) of corneal immune dendritiform cells **Measure:** IVCM for Density of Corneal Immune Dendritiform Cells **Time Frame:** 6 Weeks **Description:** Morphology (in cells/mm2) of Corneal Immune Dendritiform Cells **Measure:** IVCM for Corneal Immune Dendritiform Cell (DC) Morphology **Time Frame:** 2 Weeks **Description:** Morphology (in cells.mm2) of Corneal Immune Dendritiform Cells **Measure:** IVCM for Corneal Immune Dendritiform Cell (DC) Morphology **Time Frame:** 6 Weeks #### Secondary Outcomes **Description:** Corneal Fluorescein Staining Using the National Eye Institute (NEI) Grading Scheme Minimum score-0 Maximum score-15 Higher score means worse outcome **Measure:** Ocular Signs: Corneal Epitheliopathy **Time Frame:** 2 Weeks **Description:** Corneal Fluorescein Staining Using the National Eye Institute (NEI) Grading Scheme Minimum score-0 Maximum score-15 Higher score means worse outcome **Measure:** Ocular Signs: Corneal Epitheliopathy **Time Frame:** 6 Weeks **Description:** Conjunctival Lissamine Green Staining Using National Eye Institute (NEI) Grading Scheme Minimum score-0 Maximum score-18 Higher score means worse outcome **Measure:** Ocular Signs: Conjunctival Epitheliopathy **Time Frame:** 2 Weeks **Description:** Conjunctival Lissamine Green Staining Using National Eye Institute (NEI) Grading Scheme Minimum score-0 Maximum score-18 Higher score means worse outcome **Measure:** Ocular Signs: Conjunctival Epitheliopathy **Time Frame:** 6 Weeks **Description:** Total Score of the Ocular Surface Disease Index (OSDI) Questionnaire Minimum score- 0 Maximum score-100 Higher score means worse outcome **Measure:** Ocular Symptoms: Ocular Surface Disease Index (OSDI) Questionnaire **Time Frame:** 2 Weeks **Description:** Total Score of the Ocular Surface Disease Index (OSDI) Questionnaire Minimum score- 0 Maximum score-100 Higher score means worse outcome **Measure:** Ocular Symptoms: Ocular Surface Disease Index (OSDI) Questionnaire **Time Frame:** 6 Weeks **Description:** Intraocular Pressure (IOP) will be measured via Applanation and the result will have these units: mmHg **Measure:** Ocular Signs: Intraocular Pressure (IOP) by Measure of Applanation Tonometry **Time Frame:** 2 Weeks **Description:** Intraocular Pressure (IOP) will be measured via Applanation and the result will have these units: mmHg **Measure:** Ocular Signs: Intraocular Pressure (IOP) by Measure of Applanation Tonometry **Time Frame:** 6 Weeks **Description:** Tear Break Up Time (TBUT) will be recorded in seconds **Measure:** Ocular Signs: Tear Break Up Time (TBUT) **Time Frame:** 2 Weeks **Description:** Tear Break Up Time (TBUT) will be recorded in seconds **Measure:** Ocular Signs: Tear Break Up Time (TBUT) **Time Frame:** 6 Weeks **Description:** The Schirmer's Test (performed using Anesthesia) will be measured in mm. **Measure:** Ocular Signs: Schirmer's Test With Anesthesia **Time Frame:** 2 Weeks **Description:** The Schirmer's Test (performed using Anesthesia) will be measured in mm. **Measure:** Ocular Signs: Schirmer's Test With Anesthesia **Time Frame:** 6 Weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age 18-89 years. * Willing and able to provide written informed consent. * Willing and able to comply with study assessments for the full duration of the study. * In good stable overall health. * Corneal dendritiform cell count by confocal microscopy of \>=75/mm2 (13 immune cells per image) * Diagnosis of dry eye disease based on the followings: * Symptoms of dry eye disease such as foreign body sensation, burning, stinging, light sensitivity for at least 6 months. * Two or more of the following objective signs: * Schirmer test with anesthesia \<10 mm at 5 minutes \[mean Schirmer between eyes. * Tear break-up time (TBUT) of \<10 seconds. * Corneal fluorescein staining of 4 (NEI grading scheme, 0-15) in at least one eye * Lissamine green staining of the nasal and temporal conjunctiva (NEI grading scheme, 0-18) in at least one eye Exclusion Criteria: * Central corneal subbasal dendritic cell count by in vivo confocal microscopy of \<75/mm2 in both eyes * Active ocular allergies * Active allergies to steroids, aminoglycosides, or benzalkonium chloride (BAK) * History of contact lens wear within 3 months before enrollment. * Intraocular surgery or ocular laser surgery within 3 months before enrollment. * History of ocular infection within 3 months before enrollment. * History of topical (for ophthalmic use) or systemic steroid treatment (Loteprednol (other than Lotemax suspension used in our study), Difluprednate, Fluorometholone, Prednisolone, Dexamethasone, Triamcinolone, Rimexolone, Medrysone) within 1 month before enrollment. In case of topical ophthalmic steroid use, a wash-out period of 1 month is required. * History of increased intraocular pressure after using topical steroids (steroid responsive) * Change in systemic immunosuppression medication in the past 3 months. * History of any change in the frequency of topical cyclosporine or oral tetracycline compounds (tetracycline, doxycycline, and minocycline) within 1 month before enrollment. * Any condition (including language barrier) that precludes subject's ability to comply with study requirements including completion of study. **Maximum Age:** 89 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Boston **Country:** United States **Facility:** Tufts Medical Center **State:** Massachusetts **Zip:** 02111 #### Overall Officials **Official 1:** **Affiliation:** Tufts Medical Center **Name:** Pedram Hamrah, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Document Section ### Large Document Module #### Large Docs - Date: 2017-01-13 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 1120235 - Type Abbrev: Prot_SAP - Upload Date: 2017-12-07T14:48 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007766 - Term: Lacrimal Apparatus Diseases - ID: D000007637 - Term: Keratoconjunctivitis - ID: D000003231 - Term: Conjunctivitis - ID: D000003229 - Term: Conjunctival Diseases - ID: D000007634 - Term: Keratitis - ID: D000003316 - Term: Corneal Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC11 - Name: Eye Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M18040 - Name: Dry Eye Syndromes - Relevance: HIGH - As Found: Dry Eye Disease - ID: M10664 - Name: Keratoconjunctivitis Sicca - Relevance: HIGH - As Found: Dry Eye Disease - ID: M8271 - Name: Eye Diseases - Relevance: HIGH - As Found: Eye Disease - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M10663 - Name: Keratoconjunctivitis - Relevance: LOW - As Found: Unknown - ID: M10786 - Name: Lacrimal Apparatus Diseases - Relevance: LOW - As Found: Unknown - ID: M6455 - Name: Conjunctivitis - Relevance: LOW - As Found: Unknown - ID: M6453 - Name: Conjunctival Diseases - Relevance: LOW - As Found: Unknown - ID: M10660 - Name: Keratitis - Relevance: LOW - As Found: Unknown - ID: M6539 - Name: Corneal Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000015352 - Term: Dry Eye Syndromes - ID: D000007638 - Term: Keratoconjunctivitis Sicca - ID: D000005128 - Term: Eye Diseases ### Intervention Browse Module - Ancestors - ID: D000019999 - Term: Pharmaceutical Solutions - ID: D000018926 - Term: Anti-Allergic Agents ### Intervention Browse Module - Browse Branches - Abbrev: PhSol - Name: Pharmaceutical Solutions - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: AAll - Name: Anti-Allergic Agents ### Intervention Browse Module - Browse Leaves - ID: M12814 - Name: Ophthalmic Solutions - Relevance: HIGH - As Found: Cerebral - ID: M4217 - Name: Anti-Inflammatory Agents - Relevance: LOW - As Found: Unknown - ID: M30506 - Name: Lubricant Eye Drops - Relevance: HIGH - As Found: Employment - ID: M468 - Name: Loteprednol Etabonate - Relevance: HIGH - As Found: Autologous Transplantation - ID: M21860 - Name: Pharmaceutical Solutions - Relevance: LOW - As Found: Unknown - ID: M20962 - Name: Anti-Allergic Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000069559 - Term: Loteprednol Etabonate - ID: D000009883 - Term: Ophthalmic Solutions - ID: D000065346 - Term: Lubricant Eye Drops ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Lotemax **Deaths Num At Risk:** 17 **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax (loteprednol etabonate) 0.5% has been approved by the FDA for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. The medication will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Lotemax: Lotemax (loteprednol etabonate) 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of **ID:** EG000 **Other Num at Risk:** 17 **Serious Number At Risk:** 17 **Title:** Lotemax **Group ID:** EG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) **Deaths Num At Risk:** 20 **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** EG001 **Other Num at Risk:** 20 **Serious Number At Risk:** 20 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) **Frequency Threshold:** 0 **Time Frame:** 6 Weeks ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 17 **Group ID:** BG001 **Value:** 20 **Group ID:** BG002 **Value:** 37 **Units:** Participants **Group ID:** BG000 **Value:** 28 **Group ID:** BG001 **Value:** 34 **Group ID:** BG002 **Value:** 62 **Units:** Eyes ### Group **ID:** BG000 **Title:** Lotemax **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. ### Group **ID:** BG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 12.62 **Value:** 57.29 #### Measurement **Group ID:** BG001 **Spread:** 15.78 **Value:** 55.35 #### Measurement **Group ID:** BG002 **Spread:** 14.25 **Value:** 56.24 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 12 #### Measurement **Group ID:** BG001 **Value:** 17 #### Measurement **Group ID:** BG002 **Value:** 29 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 5 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 8 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG002 **Value:** 0 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 14.98 **Value:** 26.83 #### Measurement **Group ID:** BG001 **Spread:** 29.15 **Value:** 34.59 #### Measurement **Group ID:** BG002 **Spread:** 23.76 **Value:** 30.97 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 16.67 **Value:** 90.79 #### Measurement **Group ID:** BG001 **Spread:** 23.82 **Value:** 92.29 #### Measurement **Group ID:** BG002 **Spread:** 20.64 **Value:** 91.59 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 2.50 **Value:** 3.39 #### Measurement **Group ID:** BG001 **Spread:** 3.27 **Value:** 4.06 #### Measurement **Group ID:** BG002 **Spread:** 2.94 **Value:** 3.76 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 29.36 **Value:** 56.93 #### Measurement **Group ID:** BG001 **Spread:** 20.34 **Value:** 38.46 #### Measurement **Group ID:** BG002 **Spread:** 26.35 **Value:** 47.18 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 2.71 **Value:** 3.57 #### Measurement **Group ID:** BG001 **Spread:** 4.26 **Value:** 5.74 #### Measurement **Group ID:** BG002 **Spread:** 3.78 **Value:** 4.76 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 3.18 **Value:** 14.50 #### Measurement **Group ID:** BG001 **Spread:** 2.46 **Value:** 14.97 #### Measurement **Group ID:** BG002 **Spread:** 2.79 **Value:** 14.76 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 2.18 **Value:** 3.99 #### Measurement **Group ID:** BG001 **Spread:** 3.30 **Value:** 4.17 #### Measurement **Group ID:** BG002 **Spread:** 2.82 **Value:** 4.09 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 8.94 **Value:** 10.44 #### Measurement **Group ID:** BG001 **Spread:** 9.35 **Value:** 10.57 #### Measurement **Group ID:** BG002 **Spread:** 9.09 **Value:** 10.51 **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years #### Denomination 1 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 17 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 20 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 37 **Units:** Participants #### Denomination 2 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 28 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 34 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 62 **Units:** Eyes ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants #### Denomination 1 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 17 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 20 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 37 **Units:** Participants #### Denomination 2 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 28 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 34 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 62 **Units:** Eyes ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Race and Ethnicity were not collected from any participant. **Title:** Race and Ethnicity Not Collected **Unit of Measure:** Participants #### Denomination 1 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 0 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 0 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 0 **Units:** Participants #### Denomination 2 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 28 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 34 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 62 **Units:** Eyes ### Measure 4 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Corneal immune dendritiform cell (DC) density at Baseline **Unit of Measure:** cells/mm2 #### Denomination 1 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 17 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 20 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 37 **Units:** Participants #### Denomination 2 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 28 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 34 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 62 **Units:** Eyes ### Measure 5 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Corneal immune dendritiform cell (DC) morphology at Baseline **Unit of Measure:** cells/mm2 #### Denomination 1 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 17 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 20 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 37 **Units:** Participants #### Denomination 2 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 28 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 34 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 62 **Units:** Eyes ### Measure 6 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Corneal fluorescein staining at Baseline **Unit of Measure:** Score #### Denomination 1 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 17 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 20 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 37 **Units:** Participants #### Denomination 2 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 28 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 34 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 62 **Units:** Eyes ### Measure 7 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** OSDI at Baseline **Unit of Measure:** Score #### Denomination 1 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 17 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 20 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 37 **Units:** Participants #### Denomination 2 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 28 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 34 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 62 **Units:** Eyes ### Measure 8 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Lissamine Green at Baseline **Unit of Measure:** Score #### Denomination 1 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 17 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 20 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 37 **Units:** Participants #### Denomination 2 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 28 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 34 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 62 **Units:** Eyes ### Measure 9 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** IOP at Baseline **Unit of Measure:** mmHG #### Denomination 1 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 17 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 20 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 37 **Units:** Participants #### Denomination 2 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 28 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 34 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 62 **Units:** Eyes ### Measure 10 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** TBUT at Baseline **Unit of Measure:** Seconds #### Denomination 1 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 17 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 20 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 37 **Units:** Participants #### Denomination 2 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 28 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 34 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 62 **Units:** Eyes ### Measure 11 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Schirmer's at Baseline **Unit of Measure:** mm in 10 min #### Denomination 1 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 17 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 20 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 37 **Units:** Participants #### Denomination 2 ##### Denomination Count 1 **Group ID:** BG000 **Value:** 28 ##### Denomination Count 2 **Group ID:** BG001 **Value:** 34 ##### Denomination Count 3 **Group ID:** BG002 **Value:** 62 **Units:** Eyes **Type Units Analyzed:** Eyes ## Results Section - More Information Module ### Certain Agreement ### Point of Contact **Email:** phamrah@tuftsmedicalcenter.org **Organization:** Tufts Medical Center **Phone:** 617-636-5321 **Title:** Dr. Pedram Hamrah ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 ### Outcome Measure 7 ### Outcome Measure 8 ### Outcome Measure 9 ### Outcome Measure 10 ### Outcome Measure 11 ### Outcome Measure 12 ### Outcome Measure 13 ### Outcome Measure 14 ### Outcome Measure 15 ### Outcome Measure 16 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 5.13 - **Upper Limit:** - **Value:** 9.47 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 26.09 - **Upper Limit:** - **Value:** 28.60 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 5.10 - **Upper Limit:** - **Value:** 9.80 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 31.92 - **Upper Limit:** - **Value:** 34.03 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 40.56 - **Upper Limit:** - **Value:** 109.50 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 30.92 - **Upper Limit:** - **Value:** 97.95 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 26.25 - **Upper Limit:** - **Value:** 102.31 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 24.73 - **Upper Limit:** - **Value:** 89.34 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.12 - **Upper Limit:** - **Value:** 1.86 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 2.55 - **Upper Limit:** - **Value:** 3.18 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.98 - **Upper Limit:** - **Value:** 2.00 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 2.75 - **Upper Limit:** - **Value:** 3.59 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.47 - **Upper Limit:** - **Value:** 3.79 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 4.13 - **Upper Limit:** - **Value:** 4.50 **Title:** #### Outcome Measure 8 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.86 - **Upper Limit:** - **Value:** 2.46 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 3.55 - **Upper Limit:** - **Value:** 3.44 **Title:** #### Outcome Measure 9 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 27.36 - **Upper Limit:** - **Value:** 47.25 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 23.55 - **Upper Limit:** - **Value:** 33.16 **Title:** #### Outcome Measure 10 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 29.15 - **Upper Limit:** - **Value:** 46.63 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 23.30 - **Upper Limit:** - **Value:** 36.04 **Title:** #### Outcome Measure 11 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.87 - **Upper Limit:** - **Value:** 15.52 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 3.32 - **Upper Limit:** - **Value:** 14.50 **Title:** #### Outcome Measure 12 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.30 - **Upper Limit:** - **Value:** 16.88 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 3.32 - **Upper Limit:** - **Value:** 13.76 **Title:** #### Outcome Measure 13 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.48 - **Upper Limit:** - **Value:** 5.20 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.91 - **Upper Limit:** - **Value:** 3.93 **Title:** #### Outcome Measure 14 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.68 - **Upper Limit:** - **Value:** 4.83 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 2.04 - **Upper Limit:** - **Value:** 3.44 **Title:** #### Outcome Measure 15 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 4.98 - **Upper Limit:** - **Value:** 7.98 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 5.92 - **Upper Limit:** - **Value:** 6.88 **Title:** #### Outcome Measure 16 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 4.78 - **Upper Limit:** - **Value:** 8.16 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 6.06 - **Upper Limit:** - **Value:** 8.18 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Density (in cells/mm2) of Corneal Immune Dendritiform Cells **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 2 Weeks **Title:** IVCM for Density of Corneal Immune Dendritiform Cells **Type:** PRIMARY **Type Units Analyzed:** eyes **Unit of Measure:** cells/mm2 ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Outcome Measure 2 **Description:** Density (in cells/mm2) of corneal immune dendritiform cells **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 6 Weeks **Title:** IVCM for Density of Corneal Immune Dendritiform Cells **Type:** PRIMARY **Type Units Analyzed:** eyes **Unit of Measure:** cells/mm2 ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Outcome Measure 3 **Description:** Morphology (in cells/mm2) of Corneal Immune Dendritiform Cells **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 2 Weeks **Title:** IVCM for Corneal Immune Dendritiform Cell (DC) Morphology **Type:** PRIMARY **Type Units Analyzed:** Eyes **Unit of Measure:** cells/mm2 ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Outcome Measure 4 **Description:** Morphology (in cells.mm2) of Corneal Immune Dendritiform Cells **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 6 Weeks **Title:** IVCM for Corneal Immune Dendritiform Cell (DC) Morphology **Type:** PRIMARY **Type Units Analyzed:** Eyes **Unit of Measure:** Cells/mm2 ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Outcome Measure 5 **Description:** Corneal Fluorescein Staining Using the National Eye Institute (NEI) Grading Scheme Minimum score-0 Maximum score-15 Higher score means worse outcome **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 2 Weeks **Title:** Ocular Signs: Corneal Epitheliopathy **Type:** SECONDARY **Type Units Analyzed:** Eyes **Unit of Measure:** Score ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Outcome Measure 6 **Description:** Corneal Fluorescein Staining Using the National Eye Institute (NEI) Grading Scheme Minimum score-0 Maximum score-15 Higher score means worse outcome **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 6 Weeks **Title:** Ocular Signs: Corneal Epitheliopathy **Type:** SECONDARY **Type Units Analyzed:** Eyes **Unit of Measure:** Score ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension supplied by Bausch \& Lomb, Inc. Lotemax (loteprednol etabonate) 0.5% has been approved by the FDA for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. The medication will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Lotemax: Lotemax (loteprednol etabonate) 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Outcome Measure 7 **Description:** Conjunctival Lissamine Green Staining Using National Eye Institute (NEI) Grading Scheme Minimum score-0 Maximum score-18 Higher score means worse outcome **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 2 Weeks **Title:** Ocular Signs: Conjunctival Epitheliopathy **Type:** SECONDARY **Type Units Analyzed:** Eyes **Unit of Measure:** Score ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Outcome Measure 8 **Description:** Conjunctival Lissamine Green Staining Using National Eye Institute (NEI) Grading Scheme Minimum score-0 Maximum score-18 Higher score means worse outcome **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 6 Weeks **Title:** Ocular Signs: Conjunctival Epitheliopathy **Type:** SECONDARY **Type Units Analyzed:** Eyes **Unit of Measure:** Score ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Outcome Measure 9 **Description:** Total Score of the Ocular Surface Disease Index (OSDI) Questionnaire Minimum score- 0 Maximum score-100 Higher score means worse outcome **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 2 Weeks **Title:** Ocular Symptoms: Ocular Surface Disease Index (OSDI) Questionnaire **Type:** SECONDARY **Unit of Measure:** Score ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Outcome Measure 10 **Description:** Total Score of the Ocular Surface Disease Index (OSDI) Questionnaire Minimum score- 0 Maximum score-100 Higher score means worse outcome **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 6 Weeks **Title:** Ocular Symptoms: Ocular Surface Disease Index (OSDI) Questionnaire **Type:** SECONDARY **Unit of Measure:** Score ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Outcome Measure 11 **Description:** Intraocular Pressure (IOP) will be measured via Applanation and the result will have these units: mmHg **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 2 Weeks **Title:** Ocular Signs: Intraocular Pressure (IOP) by Measure of Applanation Tonometry **Type:** SECONDARY **Type Units Analyzed:** Eyes **Unit of Measure:** mmHg ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Outcome Measure 12 **Description:** Intraocular Pressure (IOP) will be measured via Applanation and the result will have these units: mmHg **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 6 Weeks **Title:** Ocular Signs: Intraocular Pressure (IOP) by Measure of Applanation Tonometry **Type:** SECONDARY **Type Units Analyzed:** Eyes **Unit of Measure:** mmHg ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Outcome Measure 13 **Description:** Tear Break Up Time (TBUT) will be recorded in seconds **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 2 Weeks **Title:** Ocular Signs: Tear Break Up Time (TBUT) **Type:** SECONDARY **Type Units Analyzed:** Eyes **Unit of Measure:** Seconds ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Outcome Measure 14 **Description:** Tear Break Up Time (TBUT) will be recorded in seconds **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 6 Weeks **Title:** Ocular Signs: Tear Break Up Time (TBUT) **Type:** SECONDARY **Type Units Analyzed:** Eyes **Unit of Measure:** Seconds ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Outcome Measure 15 **Description:** The Schirmer's Test (performed using Anesthesia) will be measured in mm. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 2 Weeks **Title:** Ocular Signs: Schirmer's Test With Anesthesia **Type:** SECONDARY **Type Units Analyzed:** Eyes **Unit of Measure:** mm ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Outcome Measure 16 **Description:** The Schirmer's Test (performed using Anesthesia) will be measured in mm. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** 6 Weeks **Title:** Ocular Signs: Schirmer's Test With Anesthesia **Type:** SECONDARY **Type Units Analyzed:** Eyes **Unit of Measure:** mm ##### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG000 **Title:** Lotemax ##### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** OG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) ### Participant Flow Module #### Group **Description:** Lotemax (loteprednol etabonate) 0.5% is a prescription-only, preserved ophthalmic suspension. Lotemax 0.5% is FDA approved for treatment of ocular inflammation with a maximum dosing frequency of 24 drops per eye per day. It is a C-20 ester-based corticosteroid, with a potent anti-inflammatory efficacy, but decreased impact on intraocular pressure (IOP) compared to other corticosteroids, which may increase IOP. Lotemax 0.5% will be applied topically to both eyes for 6 weeks with the following regimen: four times daily for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): IVCM is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** FG000 **Title:** Lotemax #### Group **Description:** Soothe Tired Eyes Lubricant Eye Drop (Bausch \& Lomb Inc.) is a preserved artificial tear which is used to relieve the dryness of the eye and to prevent further irritation. Its active ingredient is glycerin 1%. The artificial tear will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears): Soothe Tired Eyes Lubricant Eye Drop will be applied topically to both eyes for 6 weeks with the following regimen: four times a day for 2 weeks, twice daily for 2 weeks, and once daily for 2 weeks. In Vivo Confocal Microscopy (IVCM): In vivo confocal microscopy (IVCM) is a new imaging method, which allows visualization of the corneal structures at the cellular level. With a magnification of 800 times, it makes it possible to detect and quantify changes in the epithelial layers and sub-basal nerve plexus. **ID:** FG001 **Title:** Soothe Tired Eyes Lubricant Eye Drop (Artificial Tears) #### Period **Title:** Baseline ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 17 **Number of Units:** 28 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 20 **Number of Units:** 34 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 17 **Number of Units:** 28 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 20 **Number of Units:** 34 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 **Number of Units:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 **Number of Units:** 0 #### Period **Title:** 2 Weeks ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 17 **Number of Units:** 28 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 20 **Number of Units:** 34 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 17 **Number of Units:** 28 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 20 **Number of Units:** 34 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 **Number of Units:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 **Number of Units:** 0 #### Period **Title:** 6 Weeks ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 17 **Number of Units:** 28 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 20 **Number of Units:** 34 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 17 **Number of Units:** 28 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 20 **Number of Units:** 34 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 **Number of Units:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 **Number of Units:** 0 **Type Units Analyzed:** eyes **Has Results:** True