Phase 2
N=4
A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT03375164 ↗Enrolled (actual)
4
Serious AEs
0.0%
Results posted
May 2024
Primary outcome: Primary: Number of Participants With Adverse Events (AEs) — 4 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- delandistrogene moxeparvovec (Genetic)
- Age
- Pediatric · 0+ yrs
- Sex
- Male
- Sponsor
- Sarepta Therapeutics, Inc.
- Primary completion
- Apr 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events (AEs) |
4 | — |
| SECONDARY Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Western Blot |
70.52 | — |
| SECONDARY Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Immunofluorescence (IF) Fiber Intensity |
93.59 | — |
| SECONDARY Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by IF Percent Dystrophin Positive Fibers (PDPF) |
81.18 | — |
| SECONDARY Change From Baseline at Year 5 in the 100 Meter Timed Test |
-4.02 | — |
Summary
This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study was originally designed to consist of 12 patients across 2 Cohorts. Cohort A would have included participants ages 3 months to 3 years, and Cohort B included participants ages 4 to 7 years old. No participants were enrolled in Cohort A.
Eligibility Criteria
Inclusion Criteria
- Cohort A participants: 3 months to 3 years of age, inclusive
- Cohort B participants: 4 to 7 years of age, inclusive
- Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Ability to cooperate with motor assessment testing.
- Cohort A participants: No previous treatment with corticosteroids.
- Cohort B participants: Stable dose equivalent of oral corticosteroids for at least 12 weeks prior to screening and the dose is expected to remain constant (except for potential modifications to accommodate changes in weight) throughout the first year of the study.
- Cohorts A & B: A frameshift mutation contained between exons 18 and 58 (inclusive).
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 (NCT03375164). 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.