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)
A Two-Part, Open-Label Systemic Gene Delivery Study to Evaluate the Safety and Expression of RO7494222 (SRP-9001) in Subjects Under the Age of Four With Duchenne Muscular Dystrophy
Sponsor: Hoffmann-La Roche + Sarepta Therapeutics, Inc.
No open prediction endpoints
Endpoints are classified and published by ProgramSignal analysts.
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Primary Endpoints (CT.gov)
Percentage of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
Time frame: Baseline up to Week 260
Secondary Endpoints
Change in Quantity of Delandistrogene Moxeparvovec Dystrophin as Measured by Western Blot
Eligibility Criteria
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 condi…
Read full criteria on CT.gov →✦ Analyst Commentary
Expert commentary on why this trial matters and what to watch for.
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