Clinical trials: Muscular Dystrophy, Duchenne and Becker Types
Duchenne Muscular Dystrophy
A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)
This Phase II study is an open-label, multiple dose study to evaluate the safety, tolerability, PK, PD, clinical efficacy, behavior and neuropsychology, and physical functioning vamorolone over a treatment period of 12 weeks in…
- Trial name
- NCT05185622
- Testing
- Vamorolone
- Where
- 5 sites in Canada
- Volunteers
- 54 people
- Sponsor
- Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory and non-ambulatory subjects with…
- Trial name
- NCT03362502
- Testing
- PF-06939926
- Where
- 22 sites in United States
- Volunteers
- 23 people
- Sponsor
- Pfizer
Duchenne Muscular Dystrophy
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
This study is designed to evaluate the ability of ataluren to increase dystrophin protein levels in muscle cells of participants with nmDMD.
- Trial name
- NCT03648827
- Testing
- Ataluren
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- PTC Therapeutics