Clinical trials: Becker Muscular Dystrophy
Muscular Dystrophies
Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to determine whether GSK2402968 given as a continuous dose and as an intermittent dose is effective and safe in the treatment of Duchenne muscular dystrophy.
- Trial name
- NCT01153932
- Testing
- GSK2402968
- Where
- 13 sites in Australia, Belgium and 7 more
- Volunteers
- 53 people
- Sponsor
- GlaxoSmithKline
Duchenne Muscular Dystrophy
Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
This is a multicenter, open-label, long-term, extension, phase 2 study to evaluate the safety and efficacy of long-term treatment with DS-5141b in patients with DMD who have completed DS5141-A-J101.
- Trial name
- NCT04433234
- Testing
- DS-5141b
- Where
- 2 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Daiichi Sankyo Co., Ltd.
Duchenne Muscular Dystrophy
Treatment of a Single Patient With CRD-TMH-001
The study is a single patient study intended to understand the effects of a gene-editing therapeutic to treat a rare mutation of Duchenne muscular dystrophy.
- Trial name
- NCT05514249
- Testing
- CRD-TMH-001
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Cure Rare Disease, Inc
Duchenne Muscular Dystrophy
A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the…
- Trial name
- NCT01753804
- Testing
- Observational study
- Where
- 16 sites in United States, Argentina and 8 more
- Volunteers
- 269 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy (DMD)
A Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants
The purpose of this trial is to assess the safety, tolerability, and PK of 3 doses of GRT6019 in healthy male participants. This Phase I trial will be a multiple dose trial in healthy male participants with administration of…
- Trial name
- NCT07347548
- Testing
- GRT6019
- Where
- France
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Grünenthal GmbH
Duchenne Muscular Dystrophy, Amyotrophic Lateral Sclerosis
Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders
The hypothesis is that a mechanical insufflation-exsufflation (MI-E) is associated with a decrease in the number of intubations and more rapid clinical improvement in children and adults with neuromuscular disease who are…
- Conditions
- Duchenne Muscular Dystrophy, Amyotrophic Lateral Sclerosis, Neuromuscular Diseases
- Trial name
- Nemucough (NCT00839033)
- Testing
- mechanical insufflation - exsufflation, Standard respiratory physiotherapy
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris