Clinical trials: Genetic Diseases
Metachromatic Leukodystrophy (MLD)
An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy
The purpose of this study is to collect long-term safety data in participants with metachromatic leukodystrophy (MLD) who are receiving HGT-1110 and have participated in Study HGT-MLD-070 (NCT01510028) through Week 40.
- Trial name
- NCT01887938
- Testing
- HGT-1110
- Where
- 15 sites in Australia, Brazil and 5 more
- Volunteers
- 24 people
- Sponsor
- Shire
Duchenne Muscular Dystrophy
A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy
This study aims to assess safety and effectivness of long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy (DMD) who have completed prior studies with vamorolone.
- Trial name
- GUARDIAN (NCT06713135)
- Testing
- vamorolone 40 mg/mL oral suspension
- Where
- 18 sites in Belgium, Czechia and 7 more
- Volunteers
- 80 people
- Sponsor
- Santhera Pharmaceuticals
Congenital Adrenal Hyperplasia
A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH
An investigation of the ability of Tildacerfont to reduce supraphysiologic glucocorticoid dosing in classic Congenital adrenal hyperplasia (CAH) subjects up to 76 weeks of treatment. Optional open label extension up to 240 weeks.
- Trial name
- NCT04544410
- Where
- 43 sites in United States, Australia and 14 more
- Volunteers
- 100 people
- Sponsor
- Spruce Biosciences
Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia
Pharmacokinetics of Oral Hydroxyurea Solution
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
- Trial name
- HUPK (NCT03763656)
- Testing
- Oral Hydroxyurea (100 mg/mL) Solution
- Where
- 6 sites in Jamaica, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Nova Laboratories Limited
Atopic Dermatitis
Long-term Trial of OPA-15406 Ointment in Adult and Pediatric Patients With Atopic Dermatitis
To demonstrate the safety of 1% OPA-15406 ointment in adult patients with AD and of 0.3% and 1% OPA-15406 ointments in pediatric patients with AD administered twice daily for 52 weeks.
- Trial name
- NCT03961529
- Testing
- 0.3% OPA-15406 ointment, 1% OPA-15406 ointment
- Where
- Japan
- Volunteers
- 366 people
- Sponsor
- Otsuka Pharmaceutical Co., Ltd.
Dermatitis, Atopic
Investigation of Topical SB705498 on Healthy Volunteers
A two part, randomized, double-blind, placebo controlled study to investigate the effects of topical doses of SB705498 in healthy volunteers.
- Trial name
- NCT01673529
- Testing
- SB705498
- Where
- United Kingdom
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Wilson's Disease
A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
The objectives of this clinical trial are to assess, for up to 5 years, the safety, tolerability and pharmacological activity of a single ascending doses of VTX-801, a gene therapy, administered intravenously (IV) to adult…
- Trial name
- GATEWAY (NCT04537377)
- Testing
- VTX-801
- Where
- 10 sites in United States, Denmark and 2 more
- Volunteers
- 4 people
- Sponsor
- Vivet Therapeutics SAS
Cystic Fibrosis Lung
4D-710 in Adult Patients With Cystic Fibrosis
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
- Trial name
- CF (NCT05248230)
- Testing
- 4D-710
- Where
- 20 sites in United States
- Volunteers
- 30 people
- Sponsor
- 4D Molecular Therapeutics
Atopic Dermatitis
A Study to Determine the Safety and Tolerability of Dupilumab (REGN668/SAR231893) in Patients Aged ≥6 to <18 Years With Atopic Dermatitis (Eczema)
The primary objective of the study is to characterize the safety and pharmacokinetics (PK) of dupilumab in pediatric patients with moderate-to-severe atopic dermatitis (AD) (for adolescents ≥12 to <18 years of age) or severe AD…
- Trial name
- NCT02407756
- Testing
- Dupilumab
- Where
- 26 sites in Canada, Czechia and 4 more
- Volunteers
- 78 people
- Sponsor
- Regeneron Pharmaceuticals
Atopic Dermatitis
Study of RVT-501 in Adult and Adolescent Subjects With Atopic Dermatitis
This is a multi-center, randomized, vehicle-controlled, double-blind Phase 2 study in adults and adolescent subjects with mild to moderate atopic dermatitis.
- Trial name
- NCT02950922
- Testing
- RVT-501 0.2% ointment, RVT-501 0.5% ointment, Vehicle ointment
- Where
- 20 sites in United States, Canada
- Volunteers
- 157 people
- Sponsor
- Dermavant Sciences GmbH
Usage of Prescription Opioids
Determining Genetic Biomarkers Predictive of the Development of Prescription Opioid Addiction
This is a clinical study on patients who have been prescribed opioids (narcotic pain relievers) for the treatment of pain, to determine what genetic factors influence outcome of treatment.
- Trial name
- NCT01562353
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Analgesic Solutions
Congenital Adrenal Hyperplasia
A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])
This pre-screening study is designed to determine potential eligibility of adults with classic CAH due to 21-hydroxylase deficiency (21-OHD) for participation in the CAH-301 [NCT04783181] gene therapy trial with BBP-631.
- Trial name
- NCT05101902
- Testing
- Pre-Screening
- Where
- United States
- Volunteers
- 65 people
- Sponsor
- Adrenas Therapeutics Inc
Atopic Dermatitis
A Study of How Safe Abrocitinib (CIBINQO) is in Pregnant Women and Their Offspring
The purpose of this observational study is to learn about the safety of abrocitinib (CIBINQO) in pregnant women and the offspring.
- Trial name
- NCT05721937
- Testing
- Abrocitinib
- Where
- United States
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- Pfizer
X-linked Hypophosphatemia
Study of the Anti-FGF23 Antibody, Burosumab, in Adults With XLH
This is phase 3b open-label, international, multicenter study to continue to monitor the long-term safety and efficacy of burosumab in adult patients with XLH that participated in previous clinical trials with burosumab…
- Trial name
- NCT03920072
- Testing
- Burosumab
- Where
- 10 sites in France, Ireland and 2 more
- Volunteers
- 35 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Osteoporosis
GRACE II (General Retrospective Analysis of Commercial Experience With AGN1 LOEP)
The study is designed as a retrospective, multicenter study for subjects previously treated with the AGN1 LOEP Kit in the proximal femur as part of standard hospital practice. This will be a non-randomized and non-blinded study.
- Trial name
- NCT07594639
- Testing
- AGN1 LOEP
- Where
- Belgium
- Volunteers
- 305 people
- Sponsor
- AgNovos Healthcare, LLC
Hereditary Angioedema
A Phase III, Crossover Trial Evaluating the Efficacy and Safety of KVD900 (Sebetralstat) for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)
This study is a randomized, double-blind, placebo-controlled, phase III, three-way crossover clinical trial evaluating the efficacy and safety of KVD900, in the treatment of hereditary angioedema attacks in adolescent and adult…
- Trial name
- NCT05259917
- Testing
- KVD900 600 mg, KVD900 300 mg
- Where
- 66 sites in United States, Australia and 19 more
- Volunteers
- 136 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Atopic Dermatitis
Study to Assess the Safety of Dupilumab (REGN668/SAR231893) Administered Concomitantly With Topical Corticosteroids (TCS) in Patients With Moderate-to-severe Atopic Dermatitis (AD)
The purpose of this study was to assess the safety of Dupilumab administered concomitantly with topical corticosteroids (TCS) in patients with moderate-to-severe atopic dermatitis (AD).
- Trial name
- NCT01639040
- Testing
- Dupilumab, Topical Corticosteroid (TCS)
- Where
- 13 sites in Germany, Hungary and 1 more
- Volunteers
- 31 people
- Sponsor
- Regeneron Pharmaceuticals
Cystic Fibrosis, Bronchiectasis
Safety, Tolerability and Efficacy of S-1226 in Cystic Fibrosis and Non CF Bronchiectasis
This is a single center, open label, Phase IIa, multiple-ascending dose study in which subjects with mild to moderate Cystic Fibrosis and non CF bronchiectasis (n≤12) will be enrolled.
- Trial name
- NCT03903913
- Testing
- S-1226
- Where
- 3 sites in Canada
- Volunteers
- 12 people
- Sponsor
- SolAeroMed Inc.
Skin Laxity, Wrinkle
Evaluation of Efficacy and Safety of the triLift™ System
Single center, single-arm, prospective, open Label with Before \& After Study Design.
- Trial name
- LUM-triLift (NCT05773924)
- Testing
- triLift system
- Where
- United States
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Lumenis Be Ltd.
Gout, Hyperuricemia
The Effect of Naproxen on the Pharmacokinetic Profile of LC350189 in Healthy Adults
This is a Phase 1, open-label, fixed-sequence, 3-period, 2-way drug interaction study designed to assess the pharmacokinetics, pharmacodynamics, safety, and tolerability of LC350189 and Naproxen when administered alone and in…
- Trial name
- NCT04139824
- Testing
- Xanthine Oxidase Inhibitor, nonsteroidal anti-inflammatory drug
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- LG Chem
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