Status:
COMPLETED
A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Lead Sponsor:
Sarepta Therapeutics, Inc.
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
3-7 years
Phase:
PHASE1
PHASE2
Brief Summary
This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study was originally d...
Eligibility Criteria
Inclusion
- Cohort A participants: 3 months to 3 years of age, inclusive
- Cohort B participants: 4 to 7 years of age, inclusive
- Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Ability to cooperate with motor assessment testing.
- Cohort A participants: No previous treatment with corticosteroids.
- Cohort B participants: Stable dose equivalent of oral corticosteroids for at least 12 weeks prior to screening and the dose is expected to remain constant (except for potential modifications to accommodate changes in weight) throughout the first year of the study.
- Cohorts A \& B: A frameshift mutation contained between exons 18 and 58 (inclusive).
Exclusion
- 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.
Key Trial Info
Start Date :
January 4 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 25 2023
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT03375164
Start Date
January 4 2018
End Date
April 25 2023
Last Update
November 14 2024
Active Locations (1)
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1
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205