Phase 2
Completed N=41
A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)
Muscular Dystrophy, Duchenne
Source: ClinicalTrials.gov NCT03769116 ↗
Enrolled (actual)
41
Serious AEs
8.1%
Results posted
Sep 2023
Primary outcomePrimary: Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content — 4.23; 1.91; 23.82; 0.14 percent normal — p=< 0.0001
Summary
The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD participants by measuring biological and clinical endpoints in three parts: two 48-week randomized, double-blinded, placebo-controlled periods (Part 1 and Part 2), and an open-label follow-up period (Part 3). Participants who are randomized to placebo in Part 1 will have the opportunity for treatment with delandistrogene moxeparvovec in Part 2.
In order to provide a uniform approach to monitoring long-term safety and efficacy in participants who received SRP-9001 in a clinical trial, the Sponsor has amended Study Completion for this study to occur at Week 130. Therefore, participants have transitioned and will complete the remainder of the Part 3 follow up visits in a long-term extension study, SRP-9001-305 (NCT05967351).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content |
4.23; 1.91; 23.82; 0.14 | < 0.0001 sig |
| PRIMARY Change From Baseline at Week 48 in NSAA Total Score |
1.7; 0.9 | = 0.3730 |
| SECONDARY Change From Baseline at Week 48 in Time to Rise From the Floor |
-0.15; 0.35 | — |
| SECONDARY Change From Baseline at Week 48 in Time to Ascend 4 Steps |
0.17; 0.03 | — |
| SECONDARY Change From Baseline at Week 48 in Time of 10-meter Timed Test |
0.59; 0.10 | — |
| SECONDARY Change From Baseline at Week 48 in Time of 100-meter Timed Test |
4.29; 6.28 | — |
| SECONDARY Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression Measured by Immunofluorescence (IF) Fiber Intensity |
0.06; 0.00 | — |
| SECONDARY Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression Measured by IF Percent Dystrophin Positive Fibers (PDPF) |
23.88; 5.09 | — |
| SECONDARY Participants Experiencing Adverse Events (AEs) Since Treatment With Delandistrogene Moxeparvovec |
41; 37; 33; 17; 3; 5 | — |
Eligibility Criteria
Inclusion Criteria
- Established clinical diagnosis of DMD and documented dystrophin gene mutation of DMD phenotype.
- Indication of symptomatic muscular dystrophy by protocol-specified criteria.
- Ability to cooperate with motor assessment testing.
- Stable dose equivalent of oral corticosteroids for at least 12 weeks.
- A frameshift mutation contained between exons 18 and 58 (inclusive).
Exclusion Criteria
- Impaired cardiovascular function on echocardiogram.
- Prior or ongoing medical condition on physical examination, electrocardiogram, or laboratory findings that could adversely affect participant safety, compromise completion of follow-up, or impair assessment of study results.
- Exposure to another investigational drug or exon skipping medication within 6 months of screening.
- Exposure to an investigational or commercial gene therapy product.
- Abnormal liver or renal function by protocol-specified criteria.
Other inclusion/exclusion criteria apply.
Data sourced from ClinicalTrials.gov (NCT03769116). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.