cyrilzakka/clinical-trials huggingface dataset:
Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease

The main purpose of this study was to determine the safety and tolerability of 3 different doses of duvoglustat (AT2220) in participants affected by Pompe disease. The study also evaluated the effects of duvoglustat on functional parameters in Pompe disease.

This was a Phase 2, open-label study in participants with Pompe disease, a lysosomal storage disorder. Duvoglustat is designed to act as a pharmacological chaperone of alpha-glucosidase, in order to restore enzyme activity. This study consisted of a 28-day screening period, an 11-week treatment period, and a 1-week follow-up period. Three dosing regimens of oral duvoglustat were to be evaluated (Cohort 1: 2.5 g daily for 3 days, followed by no study drug for 4 days; Cohort 2: 5 g daily for 3 days, followed by no study drug for 4 days; Cohort 3: 5 g daily for 7 days, followed by no study drug for 7 days).

Participants meeting all eligibility criteria underwent physical examination, electrocardiogram, spirometry, muscular strength test, functional muscle test, 6-minute walk test (when appropriate), laboratory tests, magnetic resonance imaging, and muscle (needle) biopsy. Quality of life was assessed via the 36-Item Short Form Health Survey questionnaire. Functional ability and level of handicap was assessed by Rotterdam handicap scale.

Eligibility Criteria:
Inclusion Criteria:

* Male or female, 18 to 74 years of age inclusive
* Diagnosis of Pompe disease based on clinical assessment, enzyme assay, and/or genotyping. Confirmatory genotyping will be performed on all participants who are screened for the study
* Naïve to enzyme replacement therapy (ERT) or has not received ERT in the 3 months prior to screening
* Willing not to initiate ERT or other prohibited treatment during study participation
* Functional grade for arms and/or legs ≥2 OR sitting forced vital capacity ≥30% and \<80% of predicted value, reproducible between screening and baseline (±15%)
* Participants of reproductive potential agree to use reliable methods of contraception during the study
* Participant or legal representative is willing and able to provide written informed consent

Exclusion Criteria:

* Any intercurrent condition that may preclude accurate interpretation of study data
* Obstructive pulmonary disease
* Invasive ventilatory support
* Use of noninvasive ventilatory support \>8 hours/day while awake
* History of QTc prolongation \>450 milliseconds (msec) for males and \>470 msec for females
* History of allergy or sensitivity to the study drug, including any prior serious adverse reaction to iminosugars (such as miglustat or miglitol)
* Pregnancy or breast-feeding
* Current or recent drug or alcohol abuse
* Treatment with another investigational drug within 30 days of study start
* Use of prohibited medications ≤3 months prior to screening
* Otherwise unsuitable for the study in the opinion of the Investigator
Workers' Acceptability of a Prototype Integrated and Interactive Pedal Desk

The purpose of this study is to assess full-time workers'impressions and acceptability of a prototype pedal desk. The pedal-desk is a semi-recumbent (upright) portable pedal mechanism that allows workers to complete tasks in a traditional seated position.

Researchers in recent years have begun to consider how modern occupational practices and environments could be enriched to facilitate increased energy expenditure by replacing sedentary behaviors with opportunities for non-exercise physical activity without competing with time committed to work tasks. The investigators have developed a pedal desk: a semi-recumbent (upright) portable pedal mechanism with a maneuverable and adaptable desktop. While utilizing the pedal desk, workers are able to complete their tasks in a more traditional seated position. Determining workers' acceptability is a key initial step in translating this "sedentary behavior countermeasure" to real workplace settings.

In addition to having their height, weight, body fat percentage, and bioelectrical impedance measured, eligible participants will be asked to complete three tasks while simultaneously pedaling at a sustainable and self-selected pace. The three tasks are: 1) compose and send an email; 2) search a topic on the internet, and 3) complete an on-line questionnaire. The questionnaire that participants will complete was developed specifically for this study, utilizes Likert scales, and is based on the Theory of Planned Behavior. Constructs tested focus on perceptions of using the pedal desk at work and include: behavioral beliefs, normative beliefs, control beliefs, intention, self-efficacy, and an open-ended question soliciting general impressions concerning using the pedal desk at work.

Eligibility Criteria:
Inclusion Criteria:

* full-time employee at Pennington Biomedical Research Center
* between ages 21 and 65
* self-report primarily sitting while at work
* comfortable sending emails and searching topics on the internet

Exclusion Criteria:

* weigh more than 250 pounds
* have physical limitations that prevent performance of pedaling motions
* pregnancy
* pacemaker or metal joint replacement
Validation of a New Prognostic Score for Adult Patients With RAS Wild-type mCRC Treated With Vectibix® and FOLFIRI or FOLFOX in First Line (VALIDATE)

A safety and efficacy study of first line therapy with Vectibix® in combination with FOLFIRI or FOLFOX to validate a prognostic score in adult patients with RAS wild-type metastatic colorectal cancer in a real world setting (VALIDATE)

This is a non-interventional, prospective, open-label, single-arm, 3-cohort, multicenter study in Germany and Austria. In total, 606 patients will be enrolled in approx. 120 oncological sites (office-based medical oncologists, oncology outpatient-centers, and university hospitals) in a time period of 48 months. Of these, 202 patients with high risk, 202 patients with intermediate risk, and 202 patients with low risk, as a priori assessed by the metastatic colorectal cancer prognostic score (mCCS), will be included. Overall survival of the pre-defined prognostic groups will be analyzed as primary endpoint to validate the mCCS.

Patients have been scheduled to receive first line combination therapy with panitumumab and FOLFIRI or FOLFOX according to the current SmPC valid for Germany and Austria, respectively. Data on efficacy in terms of tumor response evaluation / survival and safety (ADRs) will be collected during first line therapy. A subset of patients participating in the 'VALIDATE-PRO' project (n=303) will be assessed for general and health-related quality of life with patient questionnaires. Biomarker status beyond RAS will be collected at baseline. The documentation of defined patient data including survival will continue until the end of the individual study participation which is latest 36 months after last patient in. The end of study will be at latest at 36 months after last patient in (LPI).

Eligibility Criteria:
Inclusion Criteria:

* Eligibility according to current SmPC
* Signed written informed consent
* Diagnosis of RAS-WT mCRC
* No prior systemic therapy in the palliative setting
* Aged 18 years or older

Exclusion Criteria:

* Any contraindication according to current SmPC
* Participation in other clinical trials
A Study of Epacadostat in Combination With Pembrolizumab and Chemotherapy in Participants With Advanced or Metastatic Solid Tumors (ECHO-207/KEYNOTE-723)

This was an open-label, nonrandomized, Phase 1/2 study designed to determine the safety, tolerability, and efficacy of epacadostat when given in combination with pembrolizumab and 7 different chemotherapy regimens described as Treatment Groups A through G below (see Study Drug and Background Therapies, Dose, and Mode of Administration). Phase 1 consisted of a 3 + 3 + 3 design intended to determine the MTD or PAD of epacadostat when given in combination with pembrolizumab and chemotherapy; efficacy was also explored.

Phase 2 was designed to enroll efficacy expansion cohorts to further evaluate the safety, tolerability, and efficacy of epacadostat at the MTD or PAD (as selected in Phase 1) when given in combination with pembrolizumab and chemotherapy. Each efficacy expansion cohort was to enroll participants with 1 specific type of advanced or metastatic solid tumor. Additional cohorts (ie, the mandatory biopsy cohorts) were designed to evaluate changes in the tumor microenvironment in participants with any advanced or metastatic solid tumor who had progressed on previous therapy with a PD-1 or a PD-L1 inhibitor.

No participants were enrolled in any Phase 2 efficacy expansion cohort, or in any Phase 2 mandatory biopsy cohort receiving Treatment A, B, F, or G. Phase 2 mandatory biopsy cohort participants received Treatments C, D, or E (ie, were included in Treatment Groups C, D, or E). Participants were assigned to a treatment group based on the chemotherapy regimen most appropriate for their tumor type.


Eligibility Criteria:
Inclusion Criteria:

* Histologically or cytologically confirmed diagnosis of selected advanced or metastatic solid tumors.
* Presence of measurable disease per RECIST v1.1.
* Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.

Exclusion Criteria:

* Laboratory and medical history parameters not within the Protocol-defined range.
* Receipt of anticancer medications or investigational drugs within the Protocol-defined intervals before the first administration of study drug.
* Previous radiotherapy within 2 weeks of starting study therapy.
* Known active central nervous system (CNS) metastases and/or carcinomatous meningitis.
* Has not recovered to ≤ Grade 1 from toxic effects of previous therapy and/or complications from previous surgical intervention before starting study therapy.
* Receipt of a live vaccine within 30 days of planned start of study therapy.
* Active infection requiring systemic therapy.
* Subjects who have any active or inactive autoimmune disease or syndrome.
* Women who are pregnant or breastfeeding.
Banding Without Resection in Small Subepithelial Tumours

To analyse the effectiveness and safety of endoscopic band ligation without resection in small gastrointestinal subepithelial tumours.

Multicenter prospective study analysing the effectiveness and safety of endoscopic band ligation without resection in small (≤15-mm) gastrointestinal subepithelial tumours.

This endoscopic technique will be complemented with a SINK (single-incision needle-knife) biopsy for ensuring the pathological diagnosis of the treated subepithelial tumour.

Eligibility Criteria:
INCLUSION CRITERIA:

* Age between 18 and 80 years old.
* Endoscopic ultrasound (EUS) image of a gastrointestinal subepithelial tumour (SET) ≤15-mm.
* Patient capable of understanding and signing informed consent form.
* Patient understanding the type of study and complying with the follow-up of complementary tests during the study duration.

EXCLUSION CRITERIA:

* SETs \>15-mm.
* SETs with clear diagnosis of EUS benign semiology: ie., lipoma, ectopic pancreas.
* Patients with multiple SETs (e.g., carcinoids).
* Vascular SETs (Doppler effect by EUS).
* Severe coagulation disorder: INR \>1.5 not correctible with administration of plasma and/or platelets \<50,000/mm3.
* Conditions that preclude upper digestive endoscopy, such as stenosis.
* Pregnancy or breast-feeding.
* Failure to sign informed consent form.
* Patients with functional diversity who are unable to understand the nature and possible consequences of the study, unless there is a competent legal representative.
* Patients unable to adhere to subsequent follow-up requirements.
* Patients with a life expectancy of less than 12 months.
The Role of Maintenance Oral Clofarabine in Older Adults With Acute Myeloid Leukemia

The purpose of this study is to determine the side effects of the study drug, clofarabine, when given by mouth to patients with acute myeloid leukemia (AML), in remission.

Standard induction chemotherapy (chemotherapy given with the intent of inducing a remission/disappearance of the cancer) can lead to a complete remission (CR) in a large percentage (60% to 80%) of younger patients with newly diagnosed AML. However, the majority of patients relapse (their disease returns) despite intensive consolidation chemotherapy. In most chemotherapy studies, only a small percentage (30% to 40%) of patients who achieve a CR still are disease-free 5 years later. The outcome for older adults is even less favorable. Approximately 75% of elderly patients who achieve CR will relapse after 2 years or less and the toxicities seen with treatment are significant. A number of studies have shown that chemotherapy given to older adults after a remission has not improved this outcome; therefore new treatments need to be investigated. Clofarabine is a drug that has been studied in older adults who have achieved a CR. The treatment was found to have fewer side effects then other consolidation therapies. This study will give participants the drug for a longer period of time to see if doing this will extend the cancer remission. Clofarabine has been approved by the FDA for the treatment of acute lymphoblastic leukemia (ALL), another form of acute leukemia. The study drug is considered experimental for AML because it has not been approved by the Food and Drug Administration (FDA) for the treatment of AML.

Eligibility Criteria:
Inclusion Criteria:

* Diagnosis of acute myeloid leukemia, in first or second remission.
* Age 55 or older
* Patients must have given signed, informed consent prior to registration on study.
* Participants must have attained a complete remission (CR) to induction chemotherapy and are not good candidates for further intensive chemotherapy or a transplant.
* Female patients of childbearing potential must have a negative serum pregnancy test within 2 weeks prior to enrollment.
* Male and female patients must use an effective contraceptive method during the study and for a minimum of 6 months after study treatment.

Exclusion Criteria:

* Patients with a diagnosis of acute promyelocytic leukemia are not eligible.
* Participants cannot be currently receiving chemotherapy, radiation therapy, or immunotherapy.
* Patients must not be receiving any other investigational agents.
* Participants cannot have Hepatitis B or C or HIV.
* Patients must not have an uncontrolled infection
Venous Thromboembolism (VTE) Knowledge

Venous thromboembolism (VTE) is common especially among post-operative patients, and its occurrence is associated with significant morbidity and mortality. VTE is highly preventable with prophylactic modalities ranging from conservative measures such as early mobilization, to mechanical prophylaxis such as intermittent pneumatic compression (ICP), to pharmacological prophylaxis such as peri-operative anticoagulation. Despite established guidelines from various professional societies, the practice of VTE prophylaxis varied greatly from institute to institute. Evidence suggests that there is a significant gap between the optimal thromboprophylaxis as suggested by experts and real-work practice. Many healthcare-providers (HCP) and patients at risk of VTE are either not aware of the risk of VTE or not confident to apply the necessary thromboprophylaxis. The knowledge level of VTE appears to play a key role in affecting the level of compliance to the optimal thromboprophylaxis. Considerable improvement in VTE prophylaxis utilization may result from the implementation of a multifaceted educational program. By improving patient's awareness on VTE prophylaxis, the incidence of DVT might be reduced. The knowledge level of patients can be improved by various means including educational video, administrative measures, or continue medical education program. However, no prospective study has been performed to assess the impact of an educational program on the VTE knowledge level among post-operative patients and their HCP, and if any improvement of their knowledge level can be translated to a better adherence to VTE prophylaxis guidelines. We therefore propose to conduct a prospective non-randomized controlled study to evaluate the effect of an education materials for the patients as well as their HCP on their knowledge level as well as the utilization and adherence of VTE prophylaxis in these patients by comparing two groups of patients and HCP.


Eligibility Criteria:
Inclusion Criteria:

* Capable of providing written informed consent and willing and able to adhere to all protocol requirements
* Male or female at least 18 years of age at the time of providing written informed consent
* Adult patients age \>18 scheduled for surgical operations
* Healthcare Providers directedly involve in patients care of these surgical patients.

Exclusion criteria

* Unable to make their own informed consent
* Unable to understand the study protocol
* Visually or auditorily impaired.
Estimate The Effect Of Lersivirine On The Pharmacokinetics Of Abacavir/Lamivudine In Healthy Subjects

This will be an open-label, fixed-sequence, multiple dose crossover study in 14 healthy male and/or female subjects, to estimate the effect of steady state lersivirine on the steady state pharmacokinetics of abacavir/lamivudine.


Eligibility Criteria:
Inclusion Criteria:

* Healthy male and/or female subjects.
* Body Mass Index (BMI) of 17.5 to 30.5 kg/m2.
* Total body weight \>50 kg (110 lbs).

Exclusion Criteria:

* History of regular alcohol consumption exceeding 7 drinks/week for females or 14 drinks/week for males (1 drink = 5 ounces (150 mL) of wine or 12 ounces (360 mL) of beer or 1.5 ounces (45 mL) of hard liquor) within 6 months of screening.
* Use of tobacco- or nicotine-containing products in excess of the equivalent of 5 cigarettes per day.
* Treatment with an investigational drug within 30 days (or as determined by the local requirement, whichever is longer) or 5 half-lives preceding the first dose of study medication.
* History of hypersensitivity to abacavir, lamivudine and/or lersivirine.
A Whole-food, Plant-Based Nutrition Intervention in Women With Metastatic Breast Cancer

This research will examine the feasibility of conducting a strict whole-food, plant-based dietary intervention in women with stable metastatic breast cancer currently undergoing conventional treatments. In addition, this research will provide preliminary data on dietary intakes and the effect of plant-based nutrition on numerous outcomes reflecting cancer prognosis and overall health using advanced imaging, various blood biomarkers linked to cancer progression, and numerous symptom questionnaires.


Eligibility Criteria:
Inclusion Criteria:

* Women with a confirmed diagnosis of metastatic breast cancer with a stable treatment regimen, demonstrated by no changes in primary cancer therapy 6 weeks prior to consent and no anticipated changes in primary cancer therapy in the 4 weeks following consent.
* Systemic therapy may consist of any conventional treatment including anti-hormonal, cytotoxic, targeted monoclonal antibody or small molecule kinase inhibitors or any combination of the above. Women who have previously declined conventional cancer therapy are also eligible provided they meet all other eligibility criteria.
* Expected to survive for at least 6 months.
* Eligibility classification for enrollment into the study - T: any; N: any; M:1.
* Any ER/PR/HER2 status is eligible.
* Age \> 18 years.
* Must be willing to adopt a strict, whole-foods, plant-based diet.
* Participant must be willing and able to comply with the protocol for the duration of the study including scheduled testing and weekly office visits.
* Able to speak and read English fluently.

Exclusion Criteria:

* Inability to tolerate a normal diet.
* Current use of insulin or sulfonylureas.
* Active malabsorption syndrome at time of consent (ie. Crohn's disease, major bowel resection leading to permanent malabsorption).
* Current eating disorder.
* Uncontrolled diarrhea.
* Plant-based food allergies or intolerances.
* Recent consumption (in the past 6 months) of a vegan diet.
* GFR \< 30 on 2 or more lab tests in the past 90 days.
* Serum potassium \> 5.3 on 2 or more lab tests in the past 90 days.
* Major surgery within 2 months of starting study program.
* Psychiatric disorder that prohibits giving informed consent.
* Current smoking.
* Current high risk alcohol use (\> 7 drinks per week).
* Current illicit substance use.
* Current warfarin use.
Prospective Study of Particle Radiotherapy for Breast Cancer With Different Arm Position

The optical body surface image (C-RAD) was used to guide and track the change of positions, and multimodal images (including 2D X-ray images, CT images and PET-CT images) were used to verify the treatment positions. In order to explore the accuracy of breast cancer patients' treatment positions and its impact on dose of the new fixation device in two different fixed positions of arm up and arm down, optimize the position fixed equipment and methods of particle radiotherapy for breast cancer, and then establish the precision process of particle radiotherapy for breast cancer under the guidance of multimodal images. In a word, this is a dosimetry and prospective Phase II randomized clinical study of the accuracy of multi-modal guided particle radiotherapy for postoperative breast cancer with different arm position.

PET-CT: Positron Emission Tomography - Computed Tomography CT: Computed Tomography

Eligibility Criteria:
Inclusion Criteria:

1. Patients with pathologically (cytological or histological) confirmed breast cancer with positive lymph nodes and postoperative adjuvant radiotherapy
2. Exclude distant metastasis
3. No ipsilateral breast and breast radiation therapy
4. Exclude patients requiring axillary irradiation
5. Age 18-80 years old
6. General good health (Eastern Cooperative Oncology Group, ECOG 0-2)
7. No complications such as severe pulmonary hypertension, cardiovascular disease, peripheral vascular disease, or severe chronic heart disease that may affect radiotherapy.
8. Non-pregnant (confirmed by serum or urine β-HCG test) or lactating women
9. Patients must sign informed consent to receive radiotherapy.

Exclusion Criteria:

1. Pathology unapproved breast cancer
2. With distant metastasis
3. The ipsilateral breast or breast has received radiation therapy
4. Patients requiring axillary irradiation
5. The dose limit of organs at risk cannot reach the preset safe dose limit
6. Pregnancy (confirmed by serum or urine β-HCG test) or lactation
7. Poor general health, Karnofsky Performance Status (KPS)\<70，or ECOG\>2
8. Severe complications that may affect radiotherapy, including: a) Unstable angina pectoris, congestive heart failure, myocardial infarction requiring hospitalization in the past 6 months, b) acute bacterial or systemic fungal infection, c) hospitalization for exacerbations of chronic obstructive pulmonary disease (COPD) or other respiratory diseases
9. Unable to understand the purpose of treatment or unwilling to sign the treatment consent