Trial Search Results
Study of SRP-4045 and SRP-4053 in DMD Patients
The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in Duchenne muscular dystrophy (DMD) patients 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
- Intact right and left biceps or 2 alternative upper muscle groups
- Mean 6MWT greater than or equal 300 meters and less than or equal to 450 meters
- Stable pulmonary function: forced vital capacity (FVC) equal to or greater than 50%
- Treatment with gene therapy at any time
- Previous treatment with SMT C1100, PRO045 (BMN 045), PRO053 (BMN 053) or PRO051 (BMN
051) within 24 weeks prior to Week 1
- Current or previous treatment with any other experimental treatment (other than
deflazacort) 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
7 Years - 13 Years
Genders Eligible for Study