Clinical trials: Inherited Genetic Conditions
Genetic Disorder, Prader-Willi Syndrome
Bioequivalence of Two Somatropin Products (Norditropin® Versus Genotropin®) in Healthy Adult Volunteers
This trial is conducted in United States of America (USA). The aim of this trial is to examine the bioequivalence of Norditropin® versus Genotropin® in healthy adult volunteers.
- Conditions
- Genetic Disorder, Prader-Willi Syndrome, Growth Disorder, Idiopathic Short Stature
- Trial name
- NCT01401244
- Testing
- somatropin
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Novo Nordisk A/S
Overweight and Obesity, Men
Evaluation of a Supplement for Weight Management in Obese and Overweight Individuals
The purpose of this trial is to examine the effects of a novel, orally consumed dietary supplement (composed of naturally occurring components) on weight management and metabolic rate in individuals who are overweight or obese…
- Conditions
- Overweight and Obesity, Men, Women
- Trial name
- NCT03812211
- Testing
- Active Supplement
- Where
- Canada
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- McMaster University
Cardiomyopathy, Hypertrophic, Familial
Signs and Symptoms of Genetic Abnormalities Linked to Inherited Heart Disease
Genetically inherited heart diseases (familial cardiomyopathies) are conditions affecting the heart passed on to family members through abnormalities in genetic information.
- Trial name
- NCT00001881
- Where
- United States
- Volunteers
- 9,999 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Kidney Failure, Chronic
Kidney Function in Sickle Cell Anemia
This is a prospective clinical cohort study that involves a baseline study visit followed by up to 3 annual follow-up study visits for a total follow-up of 36-48 months to evaluate the age- and sex-adjusted rate of change in…
- Trial name
- NCT03277547
- Where
- 3 sites in United States
- Volunteers
- 300 people
- Sponsor
- Kenneth Ataga MD
Down Syndrome, Trisomy 21
Down Syndrome Metabolic Health Study
The purpose of this research study is to determine which measures best capture cardiovascular disease (CVD) risk and type 2 diabetes (T2DM) risk in children and adolescents with Down syndrome (DS).
- Trial name
- NCT01821300
- Where
- 2 sites in United States
- Volunteers
- 257 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Cystinuria
TCUPS- Tolvaptan Use in Cystinuria and Urolithiasis: A Pilot Study
The goal of this research project is to establish that in short-term use, tolvaptan is a safe and potentially effective new therapy for cystinuria, by conducting a short-term pilot study of the safety and tolerability of this…
- Trial name
- TCUPS (NCT02538016)
- Testing
- Tolvaptan
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Caleb Nelson
Inherited Genetic Conditions
Preconception Carrier Screening at the Erasmus MC
Rationale A recent advisory report by the 'Health Council' has recommended that all prospective parents should have the opportunity to undergo preconception carrier screening.
- Trial name
- NCT06543784
- Where
- Netherlands
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Erasmus Medical Center
Pain
Evaluation of the Tolerability of Switching Subjects on Chronic ATC Opioid Therapy to Buprenorphine HCl Buccal Film
The primary aim of this study is to determine if chronic pain subjects on around-the-clock opioids who are receiving 100 to 220 mg oral morphine sulfate equivalent (MSE) can be safely transitioned on to buprenorphine…
- Trial name
- NCT01871285
- Testing
- Buprenorphine, Oxycodone, Morphine sulfate
- Where
- 3 sites in United States
- Volunteers
- 39 people
- Sponsor
- BioDelivery Sciences International
Atopic Dermatitis
A Study to Evaluate the Safety and Efficacy of Ruxolitinib Phosphate Cream Applied Topically to Adults With Atopic Dermatitis
The purpose of this study is to establish the efficacy of each strength of ruxolitinib cream once daily (QD) or twice daily (BID) in participants with atopic dermatitis as compared with vehicle cream BID.
- Trial name
- NCT03011892
- Testing
- Ruxolitinib 0.15% Cream QD, Ruxolitinib 0.5% Cream QD, Ruxolitinib 1.5% Cream QD
- Where
- 52 sites in United States, Canada
- Volunteers
- 307 people
- Sponsor
- Incyte Corporation
Cystic Fibrosis, Constipation
Effect of Lubiprostone on Nutritional Status and Pulmonary Function in Adults With Cystic Fibrosis
Cystic fibrosis (CF) results in thickened secretions in multiple organ systems including the lungs and gastrointestinal (GI) tract.
- Conditions
- Cystic Fibrosis, Constipation, Nutrition
- Trial name
- NCT00577499
- Volunteers
- 7 people
- Sponsor
- University of Arkansas
Autosomal Dominant Polycystic Kidney, Overweight and Obesity
Daily Caloric Restriction in ADPKD
This clinical trial will determine whether a daily-caloric restriction-based weight loss intervention can slow kidney growth in adults with autosomal dominant polycystic kidney disease who are overweight or obese.
- Trial name
- NCT04907799
- Testing
- Daily caloric restriction, Standard advice control
- Where
- United States
- Volunteers
- 125 people
- Sponsor
- University of Colorado, Denver
Autosomal Dominant Polycystic Kidney Disease
Repository Study of Autosomal Dominant Polycystic Kidney Disease
The design and establishment of the Polycystic Kidney Disease (PKD) Data Repository does not require, and may be constrained by, a narrowly conceived hypothesis.
- Trial name
- Repository (NCT01988038)
- Where
- United States
- Sponsor
- The Rogosin Institute
Unresectable or Metastatic Microsatellite Stable (MSS) Solid Tumor, Microsatellite Stable (MSS) Colon Cancer
Study of PI3Kinase Inhibition (Copanlisib) and Anti-PD-1 Antibody Nivolumab in Relapsed/Refractory Solid Tumors With Expansions in Mismatch-repair Proficient (MSS) Colorectal Cancer
A phase I/II study of PI3Kinase inhibition (copanlisib) and anti-PD-1 antibody nivolumab in relapsed/refractory solid tumors with expansions in mismatch-repair proficient (MSS) colorectal cancer.
- Trial name
- NCT03711058
- Testing
- Copanlisib, Nivolumab
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Analgesia
A Study Investigating the Pharmacokinetic Profiles of Four Extended Release Paracetamol Formulations
A study investigating the pharmacokinetic profiles of four extended release paracetamol formulations
- Trial name
- NCT01568749
- Testing
- Paracetamol, Paracetamol formulation 1, Paracetamol Formulation 2
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Pregnancy Related, Atopic Dermatitis
An Observational Retrospective Cohort Study Being Conducted in Women With Atopic Dermatitis Exposed to Ruxolitinib Cream Versus Topical Corticosteroids During Pregnancy: A US Claims Database Analysis
This study is to assess infant outcomes of women with AD who were exposed to ruxolitinib cream during pregnancy compared with a control cohort of women with AD who were exposed to a topical corticosteroid (TCS) during pregnancy.
- Trial name
- NCT06627335
- Testing
- Ruxolitinib Cream, TCS
- Where
- United States
- Volunteers
- 5,621 people
- Sponsor
- Incyte Corporation
Gout
Efficacy and Safety of Oral Febuxostat in Participants With Gout
The purpose of this study is to compare the efficacy and safety of febuxostat, once Daily (QD), to allopurinol in subjects with hyperuricemia and gout.
- Trial name
- CONFIRMS (NCT00430248)
- Testing
- Febuxostat, Allopurinol
- Where
- 258 sites in United States
- Volunteers
- 2,269 people
- Sponsor
- Takeda
Mucopolysaccharidosis II
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme…
- Trial name
- NCT04251026
- Testing
- tividenofusp alfa
- Where
- 7 sites in United States, Canada and 2 more
- Volunteers
- 47 people
- Sponsor
- Denali Therapeutics Inc.
Prader Willi Syndrome, Hyperphagia
Functional Connectome in Prader-Willi Syndrome: Neuroimaging and AI to Assess Therapeutic Impact
The goal of this observational study is to explore brain network changes and identify patterns related to hyperphagia, hormonal treatment effects, and cognitive deficits in adults with Prader-Willi Syndrome (PWS).
- Conditions
- Prader Willi Syndrome, Hyperphagia, Behavior Disorders
- Trial name
- NCT06900335
- Testing
- GH treatment (Somatropin), Eating
- Where
- Spain
- Volunteers
- 101 people, healthy volunteers welcome
- Sponsor
- Corporacion Parc Tauli
Diabetic Retinopathy, Sickle Cell Trait
Diabetic Retinopathy and Sickle Trait
To more clearly ascertain the relationship between ocular manifestations of sickle cell disease and diabetes, specifically; whether the presence of sickle cell trait exacerbates the disease progression of diabetic retinopathy.
- Trial name
- NCT00720317
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Medical University of South Carolina
Cystic Fibrosis (CF), Non Cystic Fibrosis Bronchiectasis
Feasibility and Acceptance of SonoHeal for Airway Clearance and Preliminary Comparison With the Standard of Care
The proposed study is to gauge the early feasibility and acceptance of the SonoHeal device. The study will evaluate the usability of the SonoHeal prototype using well-established Human Factors Engineering methods recommended by…
- Conditions
- Cystic Fibrosis (CF), Non Cystic Fibrosis Bronchiectasis, Chronic Obstructive Pulmonary Disease (COPD)
- Trial name
- NCT07568119
- Testing
- Airway clearance therapy through acoustic oscillations
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Cognita Labs LLC
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