cyrilzakka/clinical-trials huggingface dataset:
Lixivaptan in a Single Subject With Intractable Pain Due to Polycystic Kidney Disease

Abdominal pain, sometimes severe, is a known symptom of polycystic kidney disease (PKD) related to the cystic expansion. This study will investigate whether the vasopressin V2 receptor antagonist lixivaptan can alleviate intractable pain in a pediatric PKD patient who cannot tolerate the related vasopressin V2 receptor antagonist tolvaptan.


Eligibility Criteria:
Inclusion Criteria:

1. Capable of understanding the written informed consent/assent.
2. Provides signed and witnessed written informed consent/assent, and agrees to comply with protocol requirements and study-related procedures.
3. At least 15 years of age at the time of Screening.
4. Subject has been diagnosed with ADPKD by modified Ravine criteria:

   * If the subject has a family history of ADPKD, a minimum of 3 cysts per kidney by sonography or 5 cysts by computerized tomography or MRI; or
   * If the subject does not have a family history of ADPKD, a minimum of 10 cysts per kidney by any radiologic method and exclusion of other cystic kidney diseases.
5. Considered by the Investigator to be in good health relative to underlying CKD status and clinically stable with respect to underlying CKD, based on medical evaluation that includes medical and surgical history, as well as a complete physical examination including vital signs, ECG, and laboratory test results.

Exclusion Criteria:

1. Has a known sensitivity or idiosyncratic reaction to any compound present in lixivaptan or any compound listed as being present in the formulation.
2. Is pregnant or breast feeding.
3. Has taken any investigational drug or used an investigational device within 30 days or 5 half-lives, whichever is longer, prior to Screening.
4. Has taken tolvaptan, conivaptan, somatostatin analogs (e.g. lanreotide, pasireotide, octreotide, etc.), mTOR kinase inhibitors (e.g. everolimus, sirolimus, etc.), or oral or intravenous antibiotics within 30 days or 5 half-lives, whichever is longer, prior to Day 1.
5. Has a history of testing positive for hepatitis B surface antigen, hepatitis C, or human immunodeficiency virus (HIV).
6. Consumes grapefruit or Seville oranges (or their juices, or foods containing their extract) from 7 days prior to the first dose of study medication and until after the final dose.
7. Has clinically significant liver disease, or clinically significant liver function abnormalities or serology other than that expected for ADPKD with cystic liver disease at baseline.
8. Has any clinically significant laboratory abnormalities or concomitant conditions other than ADPKD (including treatment for such conditions) that, in the opinion of the Investigator, could either interfere with the study drug or pose an unacceptable risk to the subject.
Secondary Findings From High-throughput Sequencing: How to Announce Them With Respect to the Patient's Needs

High-throughput whole-genome sequencing (WGS) is bringing new opportunities in the diagnosis of rare diseases. It will more frequently lead to a primary diagnosis (aim of the genetic consultation), but it may also lead to the discovery of mutations not related to the patient's disease. These findings are called "incidental findings" (IF) and may give rise to preventive or curative interventions in a personalised medicine approach.

The question of proposing to patients access to all or part of these findings is a matter of debate in France and elsewhere. This question has given rise to new challenges and new needs that professionals must respond to by implementing appropriate management and new skills. It raises specific ethical issues, which require precise understanding of the expectations and experiences of patients. Patients' diagnostic trajectories must also meet criteria for efficacy and financial and organisational sustainability for the healthcare establishments and, for the healthcare system. Our project aims to assess the expectations of patients/parents with regard to this opportunity, and to determine how information should be provided to patients and how they should be accompanied to ensure efficient and appropriate management.


Eligibility Criteria:
Inclusion Criteria:

* Adults or parents of of deceased foetuses/children (alive or deceased) or adults living under guardianship or deceased, with Development Disorders(DD) who will undergo WES for the first time for diagnostic purposes
* Consent to take part in the study
* Desire to screen for at least one group of IF
* Able to speak fluent French.

Exclusion Criteria:

* Patients withough national health insurance cover
* Absence of consent from the patient or his/her legal representative
Cerebral Embolic Load in Patients Undergoing Surgical Aortic Valve Replacement: A Comparison of the Conventional With the Minimized Extracorporeal Circulation Technique Using Transcranial Doppler Ultrasound

In this study, the investigators aim to compare cerebral embolic load in patients undergoing surgical aortic valve replacement using either the minimized extracorporeal circulation or the conventional extracorporeal circulation technique. The detection of cerebral emboli is performed not-invasively by transcranial Doppler detection of high-intensity transient signals representing solid or gaseous microembolism in the middle cerebral arteries. The investigators hope to get more insight in the mechanism (incl. quantity) of cerebral embolism during aortic valve surgery using extracorporeal circulation.

Background

The gold standard to treat severe aortic valve stenosis is currently the surgical aortic valve replacement (SAVR) using conventional extracorporeal circulation (CECC). SAVR, however, can be performed also on minimized extracorporeal circulation (MECC), which is characterized by reduced priming volume and interfaces between blood and artificial surfaces and blood-air interface, respectively. Further technical developments of the MECC system together with reports on less induction of the coagulation cascade and activation of inflammatory systemic response may account for a reduced incidence of microbubble generation with MECC system.

Objective

The aim of the is to investigate the procedural-related incidence of high-intensity transient signals (HITS) representing solid or gaseous microembolism reaching the cerebral vessels.

Methods

Patients undergoing SAVR are included in the study and randomised to either MECC or CECC technique. HITS are continuously bilaterally detected during the entire intraoperative period by transcranial Doppler ultrasound.

Eligibility Criteria:
Inclusion Criteria:

* Isolated Severe Aortic Valve Stenosis
* No other cardiac disease
* No other coronary heart disease
* Written informed consent

Exclusion Criteria

* Double valve surgery
* Concomitant coronary artery bypass surgery
* Vascular surgery
* Age \< 18 yrs.
* Age \> 80 yrs.
Micronutrients in Management of Symptomatic Oral Lichen Planus

Treatment of oral lichen planus is challenging. Diverse therapeutic modalities have been suggested, but a permanent cure is not yet available. In some OLP patients, topical corticosteroid alone is not sufficiently enough, thus it may require a supplementation to augment its effect. Micronutrients are gaining more attention as therapeutic modalities in immunologic disorders. Researchers are recommended to conduct further clinical studies are to assess the role of these elements in management of OLP (Gholizadeh \& Sheykhbahaei, 2020). Among the less visited micronutrients are zinc and vitamin D. This trial will assess their role in management of OLP.

The enrolled patients will be divided randomly into three groups. One group will receive topical Corticosteroid (Kenacort A Orabase: triamcinolone acetonide 0.1% adhesive paste - Dermapharm) four times daily, the second group will receive topical Corticosteroid (Kenacort A Orabase: triamcinolone acetonide 0.1% adhesive paste - Dermapharm) four times daily with systemic zinc (Octozinc: Zinc sulphate heptahydrate 25 mg tablets - October Pharma) twice daily and the third group will receive topical Corticosteroid (Kenacort A Orabase: triamcinolone acetonide 0.1% adhesive paste - Dermapharm) four times daily with systemic vitamin D (Cholecalciferol: vitamin D3 15 ml oral solution - Medical Union Pharmaceuticals) once daily. The patients will be recalled weekly for eight weeks. For every four weeks of topical corticosteroid application, topical oral antifungal (Daktarin 2% oral gel - miconazole) will be prescribed, four times daily for one week, to all the patients in the trial, to avoid secondary infection with oral candidiasis. The patients will be asked to prohibit the use of any topical or systemic medication during the trial.

Eligibility Criteria:
Inclusion Criteria:

* Patients suffering from erosive or atrophic OLP
* Patients free from any visible oral lesions other than OLP.
* Patients who agreed to take the supplied interventions.
* Patient who will agree to participate in the study.
* Patients who will accept to sign the informed consent.

Exclusion Criteria:

* Patients suffering from any systemic disease.
* Treatment with any systemic treatment such as systemic steroids, other immunosuppressive drugs or non-steroidal anti-inflammatory drugs at least eight weeks.
* Treatment with any oral topical medications for at least four weeks prior to the study.
* Pregnant and lactating mothers.
Developing a Lifestyle Intervention for South Asians

This study aims to test the hypothesis that a culturally tailored lifestyle intervention can improve health behaviors among South Asians with prediabetes and diabetes

This pre-post pilot study is a 16 week lifestyle program for South Asians with prediabetes or diabetes in Atlanta. Patients will participate in physician-led group visits every other week focused on improving dietary and exercise practices to reduce weight.

Eligibility Criteria:
Inclusion Criteria:

1. age \> 18 years;
2. confirmed diagnosis of prediabetes or T2D (documented A1c of ≥ 5.7%, 2-hour post-load glucose of \>140, or FBG of \>100);
3. a family member or peer willing to participate and attend all sessions as a social influencer;
4. proficiency in English of at least one member of each dyad,
5. willingness to provide written consent.

Exclusion Criteria:

1. type 1 diabetes or diabetes secondary to other conditions (e.g. steroid-induced, pancreatic insufficiency, or chemotherapy-induced);
2. malignancy or life-threatening illness with life expectancy of \<5 years;
3. end-stage disease or serious illness that prohibits participation (e.g. end-stage renal disease or class IV congestive heart failure);
4. inability to perform unsupervised physical activity;
5. pregnancy;
6. diagnosed cognitive deficits or limited decision-making capacity;
7. alcohol or substance abuse;
8. homelessness or no fixed address.
Comparison of Diagnostic Accuracy Before or After Stricture Dilation in Biliary Stricture

Biliary strictures present a diagnostic and therapeutic challenge to clinicians due to unsatisfied accuracy of sampling modality. The major problem is very difficult to discern malignant from non-malignant strictures, such as patients with primary sclerosing cholangitis (PSC). With the poor prognosis and high mortality rate of advanced stage of hepatopancreaticobiliary malignancies, early and accurate diagnosis impacts patients' outcome and possible surgical candidacy. Therefore, a pre-operative determination of malignancy to help plan appropriate treatment is highly desirable.

Before 2000s, several diagnostic modalities, including laboratory tests, ultrasonography (US), computed tomography (CT) scan, cholangiography by percutaneous transhepatic cholangiography endoscopic (PTC) and endoscopic retrograde cholangiopancreatography (ERCP), and brushing cytology disclosed 13% to 24% false positive rate for suspicious malignant hilar strictures. Compared to recent studies, ERCP brushings still suffer from low sensitivity (41.6% ± 3.2% (99% CI)) and negative predictive value (58.0% ± 3.2% (99% CI)). In order to increase diagnostic accuracy, at least two sampling methods, including brushing cytology, biopsy, and fine-needle aspiration is therefore recommended. One article showed multimodal tissue-sampling (Brushing + Biopsy + Fine-needle aspiration) increased the sensitivity for diagnosis of malignant biliary stricture to 62%. However, no any literature demonstrate the best sequence of combined sampling modalities to yield the highest diagnostic accuracy. Besides, the role of stricture dilation before or after different tissue sampling modality is still uncertain.

In this study, the investigators want to compare stricture dilation before or after multimodal tissue-sampling, including brush cytology, intraductal suction and forceps biopsy for the diagnosis of malignant biliary stricture and also assess which kind of the sequence of combined tissue-sampling modalities could offer the highest diagnostic accuracy.


Eligibility Criteria:
Inclusion Criteria:

* Clinically suspicious biliary stricture that required tissue sampling as medically indicated were considered for the study

Exclusion Criteria:

* Biliary stricture caused by extra-luminal compression, such as pancreatic cancer and lymphadenopathy
* Contraindication for ERCP study
* Age younger than 20 years
* Prior tissue sampling had yielded a diagnosis of malignancy
* A guidewire could not be passed through the stricture
* Less than 6-month follow-up was available for patients with negative tissue sampling
A Study to Evaluate Caregiver Connections Via Technology for Patients With Alzheimer's and Other Types of Dementia

This research is being done to develop a unique matching process for caregivers of persons living with dementia, such as Alzheimer's disease, Lewy body dementia, frontotemporal degeneration, or other dementia syndromes. Dementia caregivers often assume greater caregiving burden than do non-dementia caregivers, and the caregiving duration tends to be longer. Many caregivers do not have the adequate support they need. Peer-to-peer support has been shown to improve quality of life, increase use of services, improve caregiver health, and reduce hospitalizations in the person they are caring for. This study will assess a technology platform and matching process for the purpose of peer-to-peer emotional support aimed at improving overall wellbeing in dementia care partners/caregivers.


Eligibility Criteria:
Individuals in a current or former caregiving role for a family member living with dementia will be recruited for this study. There will be no sex or gender restrictions, or exclusion based on race or ethnicity. Participants must be at least 18 years of age.

Inclusion Criteria:

* The participant must have a family member that has been diagnosed with dementia.
* The participant must identify as a Care Partner or Caregiver who has contact with their loved one with dementia, in person or by phone, and who provides social/emotional support and full or partial assistance with daily activities at least 3 days per week.
* The participant must have been caring for their loved one with dementia for at least 3 months.
* All participants must have access to a computer and be able to use the internet.
* All participants must be English speaking.
* All participants must agree to participate in the 15-month study, which includes completing questionnaires, brief "virtual" interactions with the study coordinator quarterly, and actively interacting with the website and potential matches.
* All participants must agree to follow-up contact throughout the duration of the study, which is anticipated to last 3 years.

Exclusion Criteria:

* Care Partner's loved one does not have a confirmed diagnosis of dementia.
* Care Partner is unable to provide consent.
* Care Partner was or has been in that role for less than 3 months.
A Study of LY3484356 in Chinese Participants With Advanced Breast Cancer

This is a study of LY3484356 in Chinese participants with advanced breast cancer. Participants must have breast cancer that is estrogen receptor positive (ER+), HER2 negative (HER2-). The purpose of this study is to measure how much LY3484356 gets into the bloodstream and how long it takes the body to remove it. The safety and effectiveness of LY3484356 will also be studied. Participation could last up to 28 months.


Eligibility Criteria:
Inclusion Criteria:

* Native Chinese participants must be of an acceptable age to provide informed consent
* Have locally advanced (not amenable to curative treatment by surgery) or metastatic disease and be an appropriate candidate for experimental therapy in the judgment of the investigator, after available standard therapies have ceased to provide clinical benefit
* Have a diagnosis of ER+, HER2- breast cancer
* Female participants have postmenopausal status due either surgical/natural menopause or ovarian suppression
* If postmenopausal status is due to ovarian suppression, participants must have a negative serum pregnancy test and agree to use highly effective, medically approved precautions to prevent pregnancy
* Have a performance status less than or equal to (≤)1 on the Eastern Cooperative Oncology Group (ECOG) scale
* Have adequate organ function
* Must be able to swallow capsules/tablets

Exclusion Criteria:

* Have symptomatic central nervous system (CNS) metastasis and/or carcinomatous meningitis
* Have a serious concomitant systemic disorder

  * Human immunodeficiency virus (HIV) positive patients are excluded unless they are well controlled on highly active antiretroviral therapy with no evidence of autoimmune deficiency syndrome-defining opportunistic infections within the last 2 years, and cluster of differentiation 4 (CD4) count greater than (\>)350 cells/microliter (μL)
  * Active hepatitis B or C virus infection
  * Severe renal impairment, interstitial lung disease, severe dyspnea at rest or requiring oxygen therapy, history of major surgical resection involving the stomach or small bowel, preexisting Crohn's disease or ulcerative colitis or a preexisting chronic condition resulting in clinically significant diarrhea
* Have visceral crisis
* Have a serious cardiac condition
* Have an acute leukemia or other relevant cancers
* Females who are pregnant or lactating
* Known allergic reaction against any of the components of the study drug
A Study of the Safety, Blood Levels and Biological Effects of GBT440 in Healthy Subjects and Subjects With Sickle Cell Disease

The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of GBT440 compared with placebo in healthy subjects and subjects with sickle cell disease (SCD).


Eligibility Criteria:
Inclusion Criteria:

* Healthy male or female of non-child bearing potential; 18 to 55 years old; are non-smokers and have not used nicotine products within 3 months prior to screening.
* Male or female, 18 to 60 years old, with sickle cell disease (hemoglobin SS, HbS/β0thalassemia, HbS/β+thalassemia, or HbSC) not requiring chronic blood transfusion therapy; without hospitalization in 30 days before screening or receiving blood transfusion within 30 days before screening; subjects are allowed concomitant use of hydroxyurea if the dose has been stable for the 3 months prior to screening.

Exclusion Criteria:

* Subjects who have a clinically relevant history or presence of respiratory, gastrointestinal, renal, hepatic, haematological, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological, dermatological, connective tissue diseases or disorders.
* Subjects who consume more than 14 (female subjects) or 21 (male subjects) units of alcohol a week.
* Subjects who have used any investigational product in any clinical trial within 30 days of screening
* Subjects with sickle cell disease who smoke \>10 cigarettes per day; have hemoglobin level \<6 g/dL or \>10.4 g/dL (\> ULN (appropriately corrected for gender) for Cohort 15) at screening; have aspartate aminotransferase (AST) \>4x upper limit of normal or alanine aminotransferase (ALT), or alkaline phosphatase (ALK) \>3x upper limit of normal reference range (ULN) at screening; have moderate or severe renal dysfunction
Attentional Focus Instructions and Conscious Movement Processing in Older Adults

This study aims to provide a novel scientific contribution through addressing critical knowledge gaps, examining the effects of attentional focus instructions on real-time (state) conscious movement processing propensity, gait parameters, and muscle efficiency in older adults in Hong Kong at risk of falling while walking in a challenging environment. The study results could update our scientific understanding of the mechanisms of conscious movement processing and the interventional effects of attentional focus instructions in older adults. It could ultimately enhance the methodology used for developing the most appropriate psychomotor gait re-education intervention in rehabilitation and provide clear guidelines on the exact attentional focus training that older adults require. Further, it could mitigate the effect of conscious movement processing and risk of falling in older adults.


Eligibility Criteria:
Inclusion Criteria:

* age 65 or above;
* able to walk independently indoor for at least 20 metres without a walking aid.

Exclusion Criteria:

* a total score of less than 24 on the MMSE-C (Chiu et al., 1994; Folstein et al., 1975);
* the presence of any untreated cerebral vascular disease, Parkinson's disease, or any other major neurological deficit;
* the presence of an unstable medical condition affecting safety while walking ;
* a history of a major fall incident within the last year;
* a static visual acuity poorer than 20/40 vision (assessed using the Tumbling E eye chart).
Volume 3D_US Kidney

The purpose of this study is to measure the volume of the kidney and tumors using 3D-US acquisition and to correlate these measurements to contrast-enhanced CT or MRI.

Additional objectives are comparison between true volume measurements and volume estimation using measurements of the three perpendicular diameters, comparison of inter and intra variability of 3D US measurements with inter and intra variability of 3D CT or MRI measurements, calculation of 3D and 2D volume measurements during contrast-enhanced US examination when the injection is performed for clinical routine purposes such as vascular and extension staging, during interventional procedures such as US guided biopsy targeting the non-necrotic tumour parenchyma. Concordance between 2D and 3D US acquisitions will be compared to CT and MRI measurements. Comparison of volume measurements between non-contrast and contrast-enhanced methods (US, CT and MRI) will be performed. The results will be compared to renal function before and after tumour treatment.

Eligibility Criteria:
Inclusion Criteria:

* Adult patient (age 18)
* Absence of participant's opposition
* No contra-indication to contrast-enhanced CT AND MRI (a single contrast-enhanced imaging modality is sufficient)
* Affiliated to Social Security

Exclusion Criteria:

- Contra-indication to both contrast-enhanced CT AND MRI