Clinical trials: Genetic Disease
Dermatitis, Atopic, Pruritus
A Study to Evaluate the Anti-pruritic Effectiveness of ASN008 in Adults With Mild to Moderate Atopic Dermatitis
The goal of this clinical trial is to evaluate ASN008 in people with itch caused by eczema. The main questions it aims to answer are: What is the efficacy and safety of ASN008? What is the impact of ASN008 on itch in patients…
- Trial name
- NCT05870865
- Testing
- ASN008, ASN008 Matching Vehicle
- Where
- 27 sites in United States
- Volunteers
- 144 people
- Sponsor
- Formation Bio, Inc.
Muscular Dystrophy, Duchenne
Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients
The purpose of this study is to evaluate the effect of docosahexaenoic fatty acid and eicosapentaenoic fatty acid supplementation for six months on the inflammation state as well as the process of muscular regeneration and the…
- Trial name
- NCT01826422
- Testing
- EPA and DHA
- Where
- Mexico
- Volunteers
- 40 people
- Sponsor
- Coordinación de Investigación en Salud, Mexico
Vulvar Lichen Sclerosus
The CO2RE® System for Vulvar Lichen Sclerosus
Subjects with vulvar lichen sclerosus (VLS) will undergo fractional carbon dioxide (CO2) laser treatment to the vulvar area. Subjects will be evaluated for changes in clinical signs and architectural changes associated with VLS…
- Trial name
- NCT04148651
- Testing
- The CO2RE® System
- Where
- 4 sites in United States
- Volunteers
- 14 people
- Sponsor
- Candela Corporation
Fragile X Syndrome, Genetic Diseases
Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome
The purpose of the study is to test the efficacy of a 20 week multi-modal treatment comprised of lovastatin or placebo, and the Parent-implemented Language Intervention (PILI) in children with fragile X syndrome (FXS).
- Trial name
- NCT02642653
- Testing
- Lovastatin
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of California, Davis
Pompe Disease (Late-onset)
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)
This is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.
- Trial name
- NCT04138277
- Testing
- AT2221, ATB200
- Where
- 60 sites in United States, Argentina and 21 more
- Volunteers
- 119 people
- Sponsor
- Amicus Therapeutics
Renal Transplant
Evaluation of Calcineurin-inhibitor Reduction With Conversion at 2 Months to Everolimus/Reduced Tacrolimus in Renal Transplant Recipients Following Campath® Induction
The purpose of this study is to evaluate whether conversion to everolimus (Zortress®), allowing the elimination or reduction of calcineurin inhibitors, will reduce nephrotoxicity (measured by increased creatinine clearance) and…
- Trial name
- NCT01935128
- Testing
- Arm 1 Everolimus/Reduced dose tacrolimus
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- University of Toledo Health Science Campus
Hemophilia A
Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A
The primary purpose of this randomized, two-arm parallel clinical study in 66 previously treated patients with severe or moderately severe hemophilia A is to compare the rate of bleeding episodes for standard prophylaxis (20-40…
- Trial name
- NCT00243386
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 29 sites in United States, Austria and 8 more
- Volunteers
- 82 people
- Sponsor
- Baxalta now part of Shire
Sickle Cell Disease
Safety and Efficacy of Sodium Nitrite in Sickle Cell Disease
This study will determine if administration of sodium nitrite is safe and can improve small vessel blood flow and tissue oxygenation when given as an additional treatment in patients with acute vaso-occlusive crisis (pain crisis)…
- Trial name
- NCT01033227
- Testing
- sodium nitrite injection, usp
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's Hospital Los Angeles
Cystic Fibrosis
Human Factors Study to Validate the User Interface of TOBI Podhaler Using Placebo Capsules
The purpose of this study is to validate the user interface of TOBI Podhaler by establishing that the product can support safe and effective use for the intended users
- Trial name
- NCT03502070
- Testing
- Tobi Podhaler
- Where
- 9 sites in United States
- Volunteers
- 47 people
- Sponsor
- Mylan Inc.
Age-Related Macular Degeneration, Macular Degeneration
Repeated Eye Injections of Aflibercept for Treatment of Wet Age Related Macular Degeneration
To evaluate the visual outcome and number of injections required during an optical coherence tomography (OCT)-guided treat and extend regimen with intravitreal aflibercept for treatment of subfoveal neovascular age-related…
- Trial name
- ATLAS (NCT01773954)
- Testing
- Intravitreal Aflibercept Injection
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- MidAtlantic Retina
Atopic Dermatitis
A Study to Assess the Safety and Efficacy of Etrasimod in Subjects With Moderate-to-Severe Atopic Dermatitis
The purpose of this study is to determine whether etrasimod is a safe and effective treatment for moderate-to-severe atopic dermatitis (AD).
- Trial name
- ADVISE (NCT04162769)
- Testing
- Etrasimod 1 mg, Etrasimod 2 mg
- Where
- 38 sites in United States, Australia and 1 more
- Volunteers
- 140 people
- Sponsor
- Arena Pharmaceuticals
Cystic Fibrosis
Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor
Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109…
- Trial name
- NCT02544451
- Testing
- LUM/IVA
- Where
- 60 sites in United States, Australia and 7 more
- Volunteers
- 246 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Pruritus, Atopic Dermatitis
Blinded, Randomized Study Evaluating Aurstat Versus Control in the Treatment of Atopic Dermatitis Associated Pruritus
To demonstrate the ability of Aurstat to reduce pruritus in subjects with mild to moderate atopic dermatitis. Efficacy results will be based on subject assessment, IGA, and photographic evidence based on ordinal scales for…
- Trial name
- AUR0613 (NCT01905631)
- Testing
- Aurstat Anti-Itch Hydrogel (Aurstat)
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- The Center for Clinical and Cosmetic Research
Hemophilia A
Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies
The primary purpose of this study is to describe the outcome of Immune Tolerance Induction (ITI) treatment performed with rFVIIIFc within a timeframe of 60 weeks in patients with haemophilia A who have failed previous attempts at…
- Trial name
- ReITIrate (NCT03103542)
- Testing
- Recombinant coagulation factor (rFVIIIFc)
- Where
- 12 sites in United States, Canada and 5 more
- Volunteers
- 16 people
- Sponsor
- Swedish Orphan Biovitrum
Solid Tumor, Hematologic Malignancies
BGJ398 for Patients With Tumors With FGFR Genetic Alterations
The purpose of this signal seeking study was to determine whether treatment with BGJ398 demonstrates sufficient efficacy in select FGFR pathway-regulated solid tumors and/or hematologic malignancies to warrant further study.
- Trial name
- CBGJ398XUS04 (NCT02160041)
- Testing
- BGJ398
- Where
- 56 sites in United States
- Volunteers
- 84 people
- Sponsor
- Novartis Pharmaceuticals
Alcohol Dependence
Genetic and Brain Mechanisms of Naltrexone's Treatment Efficacy for Alcoholism
The overarching aim of this trial is to evaluate naltrexone's efficacy in light of genetic variation and brain response to alcohol cues utilizing a neuroimaging paradigm. This trial has four specific aims.
- Trial name
- NCT00920829
- Testing
- Naltrexone 50 Mg
- Where
- 2 sites in United States
- Volunteers
- 358 people
- Sponsor
- Medical University of South Carolina
Atopic Dermatitis
Study of the Safety, Tolerability and Efficacy of BTX 1204 in Patients With Moderate Atopic Dermatitis
This is a randomized, double-blind, vehicle-controlled, Phase 2 study in subjects with moderate AD.
- Trial name
- NCT03824405
- Testing
- BTX 1204, Vehicle
- Where
- 31 sites in United States, Australia and 1 more
- Volunteers
- 200 people
- Sponsor
- Botanix Pharmaceuticals
Hypothyroidism, Trisomy 21
Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients
Children with levothyroxine (T21) have developmental delay and other functional gastrointestinal (GI) issues that may negatively affect L-T4 tolerability and absorption.
- Trial name
- NCT04747275
- Testing
- Liquid stable levothyroxine (L-T4) Tirosint-SOL, Oral tablet levothyroxine (L-T4)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Children's Mercy Hospital Kansas City
Hemophilia A
A Study of BAX 888 in Male Adults With Severe Hemophilia A
The main aim of this study is to check if there are side effects from BAX 888 and to determine the dose of BAX 888 for treating severe hemophilia A in male adults.
- Trial name
- NCT03370172
- Testing
- BAX 888
- Where
- 26 sites in United States, Austria and 4 more
- Volunteers
- 4 people
- Sponsor
- Baxalta now part of Shire
Sickle Cell Disease
GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)
An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease
- Trial name
- NCT05632354
- Testing
- Osivelotor
- Where
- 9 sites in United States, Nigeria
- Volunteers
- 47 people
- Sponsor
- Pfizer
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