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NCT04626674Phase 1RECRUITING

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

Study Summary

This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD. The maximum participant duration for this study is 156 weeks.

Conditions
Muscular Dystrophy, Duchenne
Interventions / Drugs
delandistrogene moxeparvovec
View on ClinicalTrials.gov ↗
Sponsor
SRPT
Sarepta Therapeutics
Gene Therapy
C54/100
Study Details
Enrollment83 participants
Primary Completion—
Lead SponsorSarepta Therapeutics, Inc.
Data Retrieved2026-04-11 02:55:56.327586+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 1 trials primarily test safety and dosing in a small number of participants (typically 20–100). Results do not yet speak to efficacy. FDA approval is typically many years away from this stage.

Status & Expected Data

Currently recruiting: the trial is enrolling patients. A data readout is not expected until after enrollment closes and the follow-up period is complete.

Enrolled: 83 participants
Sponsor Catalyst Profile

Sarepta Therapeutics (SRPT) is the sponsoring company for this trial. BiotechSign currently grades this company C (54/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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