Clinical trials: Genetic Conditions
Atopic Dermatitis
Acupressure in Pruritus for Atopic Dermatitis
The purpose of this study is to see if acupressure will be effective at reducing itch in people with atopic dermatitis (AD).
- Trial name
- NCT01074164
- Testing
- Standard-of-care treatment for AD, Accu-patch pellet
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Northwestern University
Hypertension, Angina Pectoris
Amlodipine/Atorvastatin (Caduet®) Drug Use Investigation (Regulatory Post Marketing Commitment Plan)
In this survey, to collect the safety and efficacy information of Amlodipine /Atorvastatin (Caduet® Combination Tablets) in daily medical practice will be examined.
- Conditions
- Hypertension, Angina Pectoris, Hypercholesterolemia, Familial Hypercholesterolemia
- Trial name
- NCT01107743
- Testing
- Amlodipine/Atorvastatin
- Volunteers
- 1,291 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Stage I Chronic Lymphocytic Leukemia, Stage II Chronic Lymphocytic Leukemia
Fludarabine and Rituximab With or Without Lenalidomide or Cyclophosphamide in Treating Patients With Symptomatic Chronic Lymphocytic Leukemia
This randomized phase II trial studies how well fludarabine (fludarabine phosphate) and rituximab with or without lenalidomide or cyclophosphamide work in treating patients with symptomatic chronic lymphocytic leukemia.
- Conditions
- Stage I Chronic Lymphocytic Leukemia, Stage II Chronic Lymphocytic Leukemia, Stage III Chronic Lymphocytic Leukemia, Stage IV Chronic Lymphocytic Leukemia
- Trial name
- NCT00602459
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- 364 sites in United States, Canada
- Volunteers
- 418 people
- Sponsor
- National Cancer Institute (NCI)
Prader-Willi Syndrome, Hyperphagia
Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome
This study is a phase 2 randomized double blind 8-week treatment trial of intranasal OXT vs. placebo in 50 subjects aged 5 to 17 years with PWS in order to assess IN-OXT's affect on measurements of (1) eating behaviors (2)…
- Trial name
- NCT03197662
- Testing
- Intranasal Oxytocin (IN-OXT)
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Eric Hollander
Anti-Psychotic, Management of Manifestations of Psychotic Disorders
Assessment of Intra-subject Variability in the Bioavailability of Chlorpromazine Hydrochloride
Cycle Pharmaceuticals Ltd. (Cycle) is developing an oral tablet formulation of Chlorpromazine Hydrochloride and intends to conduct bioequivalence trials to demonstrate its similarity to the RLD.
- Conditions
- Anti-Psychotic, Management of Manifestations of Psychotic Disorders, Treatment of Schizophrenia, Control Nausea and Vomiting
- Trial name
- NCT02943213
- Testing
- Chlorpromazine Hydrochloride
- Where
- South Africa
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Cycle Pharmaceuticals Ltd.
Cystic Fibrosis, Dysbiosis
Vitamin D and Prebiotics for Intestinal Health in Cystic Fibrosis
The study will assess if administration of high-dose vitamin D and a commonly used prebiotic (inulin) is effective to reduce gastrointestinal dysbiosis and to improve critical intestinal functions in Cystic Fibrosis with the…
- Trial name
- NCT04118010
- Testing
- Vitamin D3, Inulin
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Emory University
Tourette Syndrome
Repetitive Transcranial Magnetic Stimulation to Reduce Tics
Specific Aim1: Using a paradigm of Transcranial Magnetic Stimulation called Continuous Theta Burst Stimulation (cTBS) to reduce tics in Tourette Syndrome subjects Hypothesis1: cTBS, compared to sham stimulation, will reduce tic…
- Trial name
- NCT01258790
- Testing
- Active Repetitive Transcranial Magnetic Stimulation
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Fabry Disease
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
This was a randomized, double-blind, active control study of the enzyme replacement therapy (ERT) drug PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function.
- Trial name
- BALANCE (NCT02795676)
- Testing
- PRX-102 (pegunigalsidase alfa), agalsidase beta
- Where
- 29 sites in United States, Czechia and 10 more
- Volunteers
- 78 people
- Sponsor
- Protalix
Sickle Cell Disease, Chronic Kidney Diseases
Peer i-Coaching for Activated Self-Management Optimization in Adolescents and Young Adults With Chronic Conditions
The purpose of this study is to test the efficacy of a peer support coaching intervention to improve activated chronic illness self-management versus an attention control group in 225 adolescents and young adults with childhood…
- Conditions
- Sickle Cell Disease, Chronic Kidney Diseases, Systemic Lupus Erythematosus, Childhood Cancer
- Trial name
- PiCASO (NCT03938324)
- Testing
- PiCASO Intervention Group
- Where
- United States
- Volunteers
- 222 people
- Sponsor
- Duke University
Wolfram Syndrome, Diabetes Mellitus
A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
Wolfram syndrome is a rare genetic disorder characterized by juvenile-onset diabetes mellitus, diabetes insipidus, optic nerve atrophy, hearing loss, and neurodegeneration.
- Conditions
- Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Ataxia
- Trial name
- NCT02829268
- Testing
- dantrolene sodium
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Washington University School of Medicine
Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection
Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)
HLH, HLH-related disorders, Chronic Granulomatous (CGD), HIGM1, Immune dysregulation, polyendocrinopathy, enteropathy, and X-linked inheritance (IPEX) and severe LAD-I represent primary immune disorders that are typically fatal…
- Conditions
- Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection, Chronic Granulomatous Disease, HIGM-1
- Trial name
- RICHI (NCT01998633)
- Testing
- Hematopoietic Stem Cell Transplant
- Where
- 22 sites in United States, Canada
- Volunteers
- 47 people
- Sponsor
- Medical College of Wisconsin
Urea Cycle Disorders
Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders
Protocol HPN-100-005 was the first study of HPN-100 in pediatric subjects with urea cycle disorders (UCDs) and was a fixed-sequence, open-label, switch over study of HPN-100 with a long-term (12 month) safety extension designed…
- Trial name
- NCT00947544
- Testing
- HPN-100, NaPBA
- Where
- 8 sites in United States, Canada
- Volunteers
- 17 people
- Sponsor
- Amgen
Cystic Fibrosis
Supplementation of Oral Reduced Glutathione in Pediatric Cystic Fibrosis Patients
Many individuals with cystic fibrosis experience growth failure. The reasons are not clear, but inflammation of the gut in these patients seems to be one important reason.
- Trial name
- NCT02029521
- Testing
- Oral reduced l-glutathione
- Where
- Italy
- Volunteers
- 47 people
- Sponsor
- Clark Bishop
Sickle Cell Disease
Acupuncture for Adults With Sickle Cell Disease (SCD): A Feasibility Study
The investigators long-term goal is to demonstrate the effectiveness of acupuncture for the treatment of adults with chronic pain due to sickle cell disease (SCD), a debilitating pain syndrome characterized by acute and chronic…
- Trial name
- NCT04156399
- Testing
- Acupuncture
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Illinois Chicago
Constipation-predominant Irritable Bowel Syndrome
Cheno Effect on Transit in Health and IBS-C
The study hypothesis is that the naturally occurring bile acid, chenodeoxycholic acid, induces acceleration of colonic transit in health and in patients with constipation-predominant Irritable Bowel Syndrome (IBS-C).
- Trial name
- Chenotransit (NCT00912301)
- Testing
- Sodium chenodeoxycholate (NaCDC)
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Obesity Associated With Defects in Leptin-melanocortin Pathway
Long Term Extension Trial of Setmelanotide
This was a long-term extension trial to study the safety and tolerability of continued setmelanotide treatment in participants who had completed a previous clinical trial on treatment with setmelanotide for obesity associated…
- Trial name
- NCT03651765
- Testing
- Setmelanotide
- Where
- 27 sites in United States, Canada and 6 more
- Volunteers
- 205 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Skin Ulcers, Sickle Cell Anemia
Phase II Randomized Trial:Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers
OBJECTIVES: I. Compare the efficacy of local care alone vs local care plus arginine butyrate in terms of healing rate in patients with refractory sickle cell ulcers. II.
- Trial name
- NCT00004412
- Testing
- Arginine Butyrate, Standard local care dressing
- Where
- 4 sites in United States
- Volunteers
- 23 people
- Sponsor
- Susan P. Perrine
Alpha 1 Antitrypsin Deficiency
Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation.
The current treatment of individuals with alpha-1 antitrypsin deficiency (AATD) who develop lung disease (COPD) is the administration of intravenous purified alpha-1 antitrypsin (augmentation therapy) at a fixed dose of 60 mg/kg…
- Trial name
- NCT01669421
- Testing
- Alpha-1 Antitrypsin (human)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Michael Campos, MD
Angelman Syndrome, Nervous System Diseases
Dietary Supplements for the Treatment of Angelman Syndrome
Angelman syndrome (AS) is a complex genetic disorder that affects the nervous system. The purpose of this study is to determine the effectiveness of certain dietary supplements in treating the symptoms of AS.
- Trial name
- NCT00348933
- Testing
- Betaine, Creatine, Metafolin
- Where
- 4 sites in United States
- Volunteers
- 90 people
- Sponsor
- University of California, San Diego
Parkinson, Parkinson Disease
Parkinson Disease and DBS: Cognitive Effects in GBA Mutation Carriers
Every year, approximately 9,000 Parkinson disease (PD) patients undergo deep brain stimulator (DBS) placement into the subthalamic nucleus (STN-DBS).
- Conditions
- Parkinson, Parkinson Disease, Genetic Predisposition, GBA Gene Mutation
- Trial name
- NCT03234478
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Rutgers, The State University of New Jersey