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

NCT04626674 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 83

Last updated 2026-06-24

No results posted yet for this study

Summary

Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.

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

Conditions

Interventions

GENETIC

delandistrogene moxeparvovec

Single IV infusion of delandistrogene moxeparvovec

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Sarepta Therapeutics, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-11-23
Primary Completion
2027-12-31
Completion
2028-02-29
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04626674 on ClinicalTrials.gov