Phase 3
N=125
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT05096221 ↗Enrolled (actual)
125
Serious AEs
12.9%
Results posted
Dec 2024
Primary outcome: Primary: Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 — 2.57; 1.92 scores on a scale — p=0.2441
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- delandistrogene moxeparvovec (Genetic); placebo (Genetic)
- Age
- Pediatric · 4+ yrs
- Sex
- Male
- Sponsor
- Sarepta Therapeutics, Inc.
- Primary completion
- Oct 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 |
2.57; 1.92 | 0.2441 |
| SECONDARY Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by Western Blot Adjusted by Muscle Content |
34.29; 0.00 | < 0.0001 sig |
| SECONDARY Part 1: Change From Baseline in Time to Rise From the Floor at Week 52 |
-0.27; 0.37 | 0.0025 sig |
| SECONDARY Part 1: Change From Baseline in Time to Complete 10 Meter Walk/Run (10MWR) at Week 52 |
-0.34; 0.08 | 0.0048 sig |
| SECONDARY Part 1: Change From Baseline in Time to Complete 100 Meter Walk/Run (100MWR) at Week 52 |
-6.57; -3.28 | 0.1942 |
| SECONDARY Part 1: Change From Baseline in the Timed Stair Ascend 4 Steps Test at Week 52 |
-0.44; -0.08 | 0.0412 sig |
| SECONDARY Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) at Week 52 |
0.06; -0.03 | 0.0402 sig |
| SECONDARY Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Score in Mobility to Week 52 |
0.05; -0.01 | 0.4272 |
| SECONDARY Part 1: Change From Baseline in PROMIS Score in Upper Extremity Function to Week 52 |
0.19; 0.23 | 0.7324 |
| SECONDARY Part 1: Number of Skills Gained or Improved at Week 52 as Measured by the NSAA |
4.18; 3.99 | 0.6554 |
| SECONDARY Parts 1 and 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
62; 57; 53; 56; 14; 5 | — |
| SECONDARY Parts 1 and 2: Number of Participants With Adverse Events of Special Interest (AESI) |
12; 0; 0; 14; 0; 0 | — |
Summary
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.
Eligibility Criteria
Inclusion Criteria
- Is ambulatory and from 4 to under 8 years of age at time of randomization.
- Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Ability to cooperate with motor assessment testing.
- Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
- rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
- A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive), with the exception of mutation fully contained within exon 45.
Exclusion Criteria
- Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
- Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
- Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.
Other inclusion or exclusion criteria could apply.
Data sourced from ClinicalTrials.gov (NCT05096221). 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.