Trial Search Results
Study of SRP-4045 and SRP-4053 in Participants With Duchenne Muscular Dystrophy (DMD)
The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.
Stanford is currently accepting patients for this trial.
Sarepta Therapeutics, Inc.
- Drug: SRP-4045
- Drug: SRP-4053
- Drug: Placebo
- Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53
- Stable dose of oral corticosteroids for at least 24 weeks prior to Week 1, and the
dose is expected to remain constant throughout the study (except for modifications to
accommodate changes in weight).
- Intact right and left biceps or 2 alternative upper muscle groups
- Mean 6MWT ≥300 meters and ≤450 meters
- Stable pulmonary function: forced vital capacity (FVC) ≥50% predicted
- Treatment with gene therapy at any time
- Previous treatment with SMT C1100 within 1 week prior to Week 1 and previous treatment
with PRO045 (BMN 045), PRO053 (BMN 053), or PRO051 (BMN 051) within 24 weeks prior to
- Current or previous treatment with any other experimental treatment within 12 weeks
prior to Week 1
- Major surgery within 3 months prior to Week 1
- Presence of other clinically significant illness
Other inclusion/exclusion criteria may apply.
Ages Eligible for Study
6 Years - 13 Years
Genders Eligible for Study