Clinical trials: Genetic Conditions
Prader-Willi Syndrome, Hyperphagia
Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
The investigators propose a randomized double-blind 8 week treatment trial of intranasal oxytocin (IN-OXT) vs. placebo in 24 subjects aged 5 to 18 years with PWS in order to assess IN-OXT's affect on (1) Eating behaviors (2)…
- Trial name
- OXT-PWS (NCT02629991)
- Testing
- Intranasal Oxytocin (IN-OXT)
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Montefiore Medical Center
Abnormalities, Blood Coagulation Disorder
Study of Abnormal Blood Clotting in Children With Stroke
Effective treatment and prevention strategies for childhood stroke and porencephaly can only be developed once the causes are understood.
- Conditions
- Abnormalities, Blood Coagulation Disorder, Brain Disease, Cerebrovascular Accident
- Trial name
- NCT00001927
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Pulmonary Fibrosis
Genetic Polymorphisms in Idiopathic Pulmonary Fibrosis (IPF)
The purposes of this study are: to determine if there are specific genetic traits that might explain why patients have developed pulmonary fibrosis; to determine if specific genetic traits account for differing patterns of…
- Trial name
- GP (NCT00258570)
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- University of Pittsburgh
Colorectal Cancer, Precancerous Condition
Celecoxib in Preventing Colorectal Cancer in Young Patients With a Genetic Predisposition for Familial Adenomatous Polyposis
RATIONALE: Chemoprevention is the use of certain drugs to keep cancer from forming. The use of celecoxib may keep polyps and colorectal cancer from forming in patients with familial adenomatous polyposis.
- Trial name
- NCT00685568
- Testing
- celecoxib
- Where
- 4 sites in United States
- Volunteers
- 22 people
- Sponsor
- M.D. Anderson Cancer Center
Unverricht-Lundborg Disease
Brivaracetam as add-on Treatment of Unverricht-Lundborg Disease in Adolescents and Adults
The study will compare the efficacy and safety of brivaracetam with placebo in patients with Unverricht-Lundborg disease.
- Trial name
- NCT00357669
- Testing
- Brivaracetam 25 mg, Brivaracetam 50 mg
- Where
- 13 sites in Finland, France and 5 more
- Volunteers
- 50 people
- Sponsor
- UCB Pharma SA
Arrhythmia, Sudden Cardiac Death
Electrophysiological Phenotyping Of Patients at Risk of Ventricular Arrhythmia and Sudden Cardiac Death
Obesity, rheumatoid arthritis (RA) and gene-specific dilated cardiomyopathy (DCM) are common medical conditions. Small-scale studies have shown that these are associated with proarrhythmic changes on 12-lead electrocardiogram…
- Conditions
- Arrhythmia, Sudden Cardiac Death, Cardiomyopathy, Dilated, Obesity
- Trial name
- EPORVA (NCT03910725)
- Testing
- Electrocardiographic imaging
- Where
- 2 sites in United Kingdom
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Imperial College London
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
- Trial name
- NCT00837824
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Polycystic Liver Disease, Hepatomegaly
Open-Label Extension of LOCKCYST Trial
Positive data originating from two polycystic liver patients treated with somatostatin analogues, showed a volume reduction of 38.3% and 14.9%.
- Conditions
- Polycystic Liver Disease, Hepatomegaly, Liver Diseases, Polycystic Kidney
- Trial name
- LOCKCYST-ext (NCT00771888)
- Testing
- lanreotide
- Where
- 2 sites in Belgium, Netherlands
- Volunteers
- 54 people
- Sponsor
- Radboud University Medical Center
Prader-Willi Syndrome
Study of Tesomet With Open-label Extension in Subjects With Prader-Willi Syndrome
This study will evaluate the safety and efficacy of Tesomet (tesofensine + metoprolol) in subjects with PWS.
- Trial name
- PWS (NCT05198362)
- Testing
- Tesomet
- Where
- United States
- Sponsor
- Saniona
Bardet-Biedl Syndrome
The Effect of GLP1 Agonists on Weight Loss in BBS Cohort in the UK
The goal of this observational study is to learn about the long-term effects of GLP1 agonists (weight loss jab, including Mounjaro) in patients with Bardet-Biedl Syndrome (BBS) who take GLP1 agonists to treat high body weight.
- Trial name
- NCT07602803
- Where
- United Kingdom
- Volunteers
- 300 people
- Sponsor
- The Queen Elizabeth Hospital
Pancreas Cancer, Pancreatitis
Pancreas Registry and High Risk Registry
The purpose of this study is to establish a registry of patients with pancreatic diseases. Patients included in the registry may include those with: pancreatic cancer, precancerous lesions of the pancreas, inflammatory lesions of…
- Conditions
- Pancreas Cancer, Pancreatitis, Chronic Pancreatitis, Pancreatic Cyst
- Trial name
- NCT02775461
- Where
- 2 sites in United States
- Volunteers
- 1,368 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Duchenne Muscular Dystrophy
Tele-assessment of Functional Performance and Quality of Life in Patients With Duchenne Muscular Dystrophy: Validity and Reliability Study
Thanks to tele-assessment methods, it may be possible to evaluate DMD patients without traveling to clinical centers. In recent years, the applicability of remote assessment methods in DMD patients, as in many populations, is…
- Trial name
- NCT06295718
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people
- Sponsor
- Sahra Şirvan
Multiple Osteochondroma, Osteogenesis Imperfecta
Resilience and Coping in a Rare Skeletal Disease Population to Face Coronavirus (COVID-19) Outbreak Distress: a Longitudinal Study
In the COVID-19 outbreak context, people living with rare diseases have been highly troubled with anxiety, loneliness, and depression.
- Conditions
- Multiple Osteochondroma, Osteogenesis Imperfecta, Ollier Disease, Maffucci Syndrome
- Trial name
- RELOAD (NCT04844697)
- Where
- Italy
- Volunteers
- 15 people
- Sponsor
- Istituto Ortopedico Rizzoli
Chronic Kidney Diseases
Chronic Kidney Disease : Role of Biological Factors and Apolipoprotein L1 Encoding Gene (APOL1)
Chronic kidney disease (CKD) is a major global public health issue. The present project focuses on the role of apolipoprotein L1 (APOL1) in patients with stage 4 CKD (glomerular filtration rate between 15 and 29 mL/min/1.73 m²).
- Trial name
- APOL1 (NCT07288723)
- Where
- Guadeloupe
- Volunteers
- 88 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de la Guadeloupe
Gout, Coronary Artery Disease
Uric Acid Study in Healthy Male Volunteers
This study is being conducted to examine the effect of AZD6140 on the levels of certain chemicals in subjects' blood and urine.
- Trial name
- NCT00738842
- Testing
- AZD6140
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Smith-Magenis Syndrome (SMS)
Natural History Study of Smith-Magenis Syndrome
This study will examine how a rare disease called Smith-Magenis syndrome (SMS) affects people and how they change over time. SMS is caused by a small chromosome 17p11.2 deletion (missing piece).
- Trial name
- NCT00013559
- Where
- United States
- Volunteers
- 593 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Seemingly Healthy, Genetic Predisposition to Disease
Return of Genomic Results and Aggregate Penetrance in Population-Based Cohorts
The PopSeq Project is a prospective cohort study that will develop and implement a genomic return of results (gRoR) process in the Framingham Heart Study (FHS) and Jackson Heart Study (JHS) cohorts and explore associated medical…
- Trial name
- PopSeq (NCT04196374)
- Testing
- Genomic Sequencing
- Where
- 2 sites in United States
- Volunteers
- 7,603 people
- Sponsor
- Brigham and Women's Hospital
Obesity
Weight Loss Study: Genetics and Response to Naltrexone/Bupropion
The goal of this clinical trial is to understand if genetic variations are associated with the amount of weight loss with diet and while taking an FDA-approved medication for weight loss.
- Trial name
- NCT05919797
- Testing
- Naltrexone-Bupropion Combination
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- Columbia University
Human Immunodeficiency Virus Infection, Sexually Transmitted Diseases
Men Together Making a Difference: Reducing HIV/STD Risk Behavior Among South African Men
Sub-Saharan Africa has about 10% of the world's population, but was home to more than 60% of all people living with HIV in 2003.
- Trial name
- NCT01490359
- Testing
- Men Making a Difference HIV/STD Risk Reduction Intervention, Health Promotion Control
- Volunteers
- 1,181 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
Healthy Participants
Bioequivalence Study of Rivaroxaban 2.5 mg Tablets Under Fasting and Fed Conditions
This completed study compared how the body absorbed a single 2.5 mg dose of a test rivaroxaban tablet and the reference product (Xarelto) in healthy adults.
- Trial name
- NCT07807488
- Testing
- Rivaroxaban 2.5 mg Test Tablet, Rivaroxaban 2.5 mg Reference Tablet
- Where
- China
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- The Affiliated Hospital of Qingdao University
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