Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping.
Conditions
Brief summary
Double-blind period: Change from Baseline at Week 96 in 4-step ascend velocity (step/second)
Detailed description
• Change from Baseline at Week 96 in 6MWT • Change from Baseline at Week 96 in rise from floor velocity (rise/second) • Change from Baseline at Week 144 in 4-step ascend velocity (step/second), • Change from Baseline at Week 96 in 10MWR velocity (meter/second) • Change from Baseline at Weeks 48 or 96 in the quantity of dystrophin protein expression as measured by Western blot of biopsied muscle tissue., • Change from Baseline at Weeks 48 or 96 in the intensity of dystrophin expression in biopsied muscle tissue, as measured by IHC. •Change from Baseline at Week 96 in: NSAA total score
Interventions
Sponsors
Sarepta Therapeutics Inc.
Eligibility
Sex/Gender
Male
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Double-blind period: Change from Baseline at Week 96 in 4-step ascend velocity (step/second) | — |
Secondary
| Measure | Time frame |
|---|---|
| • Change from Baseline at Week 96 in 6MWT • Change from Baseline at Week 96 in rise from floor velocity (rise/second) • Change from Baseline at Week 144 in 4-step ascend velocity (step/second), • Change from Baseline at Week 96 in 10MWR velocity (meter/second) • Change from Baseline at Weeks 48 or 96 in the quantity of dystrophin protein expression as measured by Western blot of biopsied muscle tissue., • Change from Baseline at Weeks 48 or 96 in the intensity of dystrophin expression in biopsied muscle tissue, as measured by IHC. •Change from Baseline at Week 96 in: NSAA total score | — |
Countries
Belgium, Bulgaria, Czechia, Denmark, Hungary, Ireland, Italy, Poland, Spain
Outcome results
None listed