A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)

• delandistrogene moxeparvovec (Genetic), also known as SRP-9001, ELEVIDYS, RO7494222, delandistrogene moxeparvovec-rokl: Single IV infusion of delandistrogene moxeparvovec

Primary outcomes

• Percentage of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) [Baseline up to Week 260]

Secondary outcomes

• Change in Quantity of Delandistrogene Moxeparvovec Dystrophin as Measured by Western Blot [Baseline, Week 12]

Sex: Male, ages: 2 Years to 3 Years

Inclusion Criteria:

* Cohort A: >=3 years of age to <4 years of age
* Cohort B: >=2 years of age to <3 years of age
* Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test
* Able to cooperate with age-appropriate motor assessment testing
* A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive)

Exclusion Criteria:

* Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression, within protocol-specified time limits
* Recombinant Adeno-Associated Virus Serotype rh74 (rAArh74) antibody titers are elevated, as per protocol-specified criteria
* Receiving regular oral corticosteroids as a treatment for DMD or planning to receive oral corticosteroids as a treatment for DMD within 1 year of baseline
* 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
* Medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the participant's ability to comply with the protocol required testing or procedures, or compromise the participant's well-being or safety, or clinical interpretability

Other inclusion or exclusion criteria could apply

• Clinical Trials, Study director, Hoffmann-La Roche

NIH grants: 2023