Clinical trials: Dystrophinopathies
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy
Registry for Duchenne and Becker Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is an X-linked, recessive, progressive, and degenerative neuromuscular disorder that affects approximately one in 5,000 newborn boys.
- Conditions
- Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy Symptomatic Female Carrier
- Trial name
- FAIR-DMD (NCT07402122)
- Where
- 30 sites in Austria, Germany
- Volunteers
- 1,500 people
- Sponsor
- Dr. Andreas Ziegler
Dystrophinopathies
Decoding Central Defects in Dystrophinopathies From Diagnostic to Remediation
The study aims to identify a genotype/phenotype correlation by analyzing more finely the neurodevelopmental disorders in DMD patients.
- Trial name
- DECODYS (NCT07125898)
- Where
- France
- Volunteers
- 110 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Dystrophinopathy, Muscular Dystrophies
VirtualPark_Pediatric
The goal of this clinical trial is to assess the effects of a dual-task, multimodal training program on slowing functional motor decline in children and adolescents with neuromuscular disorders.
- Conditions
- Dystrophinopathy, Muscular Dystrophies, Congenital Myopathies
- Trial name
- NCT07580365
- Testing
- Dual-task cycling, Standard therapy
- Where
- 4 sites in Italy
- Volunteers
- 22 people
- Sponsor
- Istituto di Sistemi e Tecnologie Industriali Intelligenti per il Manifatturiero Avanzato
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy
Duchenne Electronic Health Record Study
This study aims to collect retrospective and prospective, long-term data of patients with dystrophinopathy (including Duchenne, Becker, and female carriers) through electronic transfer.
- Conditions
- Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy, Dystrophinopathy Symptomatic Female Carrier
- Trial name
- NCT07609394
- Testing
- Observational study with patients who may be treated with various disease-modifying therapies
- Where
- 10 sites in United States
- Volunteers
- 2,500 people
- Sponsor
- The Duchenne Registry
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
The Duchenne Registry
The Duchenne Registry is an online, patient-report registry for individuals with Duchenne and Becker muscular dystrophy and carrier females.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy, Dystrophinopathy Symptomatic Female Carrier
- Trial name
- NCT02069756
- Where
- United States
- Volunteers
- 10,000 people
- Sponsor
- The Duchenne Registry
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
Duchenne/Becker muscular dystrophy (DBMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy
- Trial name
- NCT01557400
- Testing
- Ataluren
- Where
- 21 sites in Australia, Belgium and 8 more
- Volunteers
- 94 people
- Sponsor
- PTC Therapeutics
Dystrophinopathies
Clinical Evaluator Outcomes Reliability Study
This study outlines structured Clinical Evaluator (CE) testing techniques that are proposed to be implemented across all sites participating in the Cooperative International Neuromuscular Research Group (CINRG) research studies.
- Trial name
- NCT02146586
- Volunteers
- 55 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Dystrophinopathy, Duchenne Muscular Dystrophy
Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids
The purpose of the study is to assess the safety, tolerability, and effects of L-Arginine on muscles in boys with dystrophinopathy on corticosteroids.
- Conditions
- Dystrophinopathy, Duchenne Muscular Dystrophy, Becker's Muscular Dystrophy
- Trial name
- NCT01388764
- Testing
- L-arginine
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Massachusetts General Hospital