Clinical trials: Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy
Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to <5 years old with Duchenne muscular dystrophy (DMD) caused by a…
- Trial name
- NCT02819557
- Testing
- Ataluren
- Where
- 6 sites in United States
- Volunteers
- 14 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain.
- Trial name
- NCT05693142
- Testing
- RGX-202
- Where
- 23 sites in United States, Canada
- Volunteers
- 65 people
- Sponsor
- REGENXBIO Inc.
Duchenne Muscular Dystrophy
Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose [MAD]) will be conducted to evaluate the safety and tolerability of vesleteplirsen at MAD levels to determine the maximum tolerated dose (MTD), and 2)…
- Trial name
- MOMENTUM (NCT04004065)
- Testing
- Vesleteplirsen
- Where
- 25 sites in United States, Belgium and 6 more
- Volunteers
- 62 people
- Sponsor
- Sarepta Therapeutics, Inc.