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

Plain-language summary

This study evaluates the safety and efficacy of the gene transfer therapy delandistrogene moxeparvovec (SRP-9001) in participants with Duchenne muscular dystrophy (DMD). It includes both non-ambulatory and ambulatory participants with a genetically confirmed diagnosis involving the DMD gene, with the ambulatory group aged 8 to 18. It is in Phase 3 and is active but not currently recruiting.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 3

Status

Active, not recruiting

Sponsor

Sarepta Therapeutics, Inc.

Intervention

delandistrogene moxeparvovec, placebo

Start date

5/31/2023

Estimated completion

6/30/2028

Countries

United States, Australia, Belgium, Canada, Germany, Hong Kong, Israel, Italy, Japan, South Korea, Spain, Sweden, Taiwan, United Kingdom

Related genes

Locations

• Little Rock, United States
• Palo Alto, United States
• Sacramento, United States
• San Diego, United States
• Gainesville, United States
• Chicago, United States
• Baltimore, United States
• Boston, United States
• St Louis, United States
• Rochester, United States
• Durham, United States
• Columbus, United States
• Philadelphia, United States
• Norfolk, United States
• Westmead, Australia
• Parkville, Australia
• Ghent, Belgium
• Ottawa, Canada
• Montreal, Canada
• Québec, Canada
• München, Germany
• Essen, Germany
• Hamburg, Germany
• Hong Kong, Hong Kong
• Petah Tikva, Israel
• Tel Aviv, Israel
• Genova, Italy
• Milan, Italy
• Milan, Italy
• Roma, Italy
• Toyonaka-shi, Japan
• Kodaira, Japan
• Shinjuku-ku, Japan
• Yangsan, South Korea
• Seoul, South Korea
• Daegu, South Korea
• Seoul, South Korea
• Esplugues de Llobregat, Spain
• Valencia, Spain
• Gothenburg, Sweden

+6

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
* Cohort 1 only: Non-ambulatory per protocol-specified criteria.
* Cohort 2 only: Ambulatory per protocol-specified criteria and ≥8 to \<18 years of age at the time of Screening.
* 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).
* Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are not elevated as per protocol-specified requirements.
* A pathogenic frameshift mutation or premature stop codon in the DMD gene, except for any deletion mutations in exon 8 and/or 9.

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.