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

Completed · Phase 3 · Has a placebo group

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID: NCT05096221
Run by: Sarepta Therapeutics, Inc.
People needed: 126
Starts: 2021-10-27
Expected to finish: 2024-10-25
Last updated by the study team: 2025-07-08

Who can join

Age: 4 and older, up to 7. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

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.

You may not qualify if…

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.

Where it is running

Arkansas Children's — Little Rock, Arkansas, United States
UC San Diego Altman Clinical and Translational Research Institute — La Jolla, California, United States
UCLA Medical Center — Los Angeles, California, United States
Lucile Packard Children's Hospital at Stanford — Palo Alto, California, United States
University of California, Davis — Sacramento, California, United States
Children's Hospital Colorado — Aurora, Colorado, United States
University of Florida — Gainesville, Florida, United States
Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
University of Iowa Stead Family Children's Hospital — Iowa City, Iowa, United States
The Johns Hopkins Hospital — Baltimore, Maryland, United States
Boston Children's Hospital — Boston, Massachusetts, United States
Washington University of St. Louis — St Louis, Missouri, United States
Columbia University/NYPH — New York, New York, United States
University of Rochester — Rochester, New York, United States
Duke University Medical Center, Lenox Baker Children's Hospital — Durham, North Carolina, United States
Nationwide Children's Hospital — Columbus, Ohio, United States
Oregon Health & Science University — Portland, Oregon, United States
The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
Vanderbilt University Medical Center — Nashville, Tennessee, United States
University of Texas Southwestern — Dallas, Texas, United States
University of Utah Hospital — Salt Lake City, Utah, United States
Children's Hospital of the King's Daughters — Norfolk, Virginia, United States
Children's Wisconsin — Milwaukee, Wisconsin, United States
University Hospital Ghent — Ghent, Belgium
LMU - Klinikum der Universitaet Muenchen - Kinderklinik und — Bayern, Germany

Full record on ClinicalTrials.gov

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