A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD)
- Duchenne Muscular Dystrophy (DMD)
Trial Status:
Active, not recruiting
This trial runs in
Countries
- United States
Trial Identifier:
NCT03375164 IRB17-00512 SRP-9001-101
Trial Summary
This is an open-label single-dose gene transfer therapy study evaluating the safety of SRP-9001 (delandistrogene moxeparvovec) intravenous (IV) administration in boys with DMD. This study will consist of 2 Cohorts. Cohort A will include participants ages 3 months to 3 years, and Cohort B will include participants ages 4 to 7 years old. All participants in the study will receive IV SRP-9001.
Sarepta Therapeutics, Inc.
Sponsor
Phase 1/Phase 2
Phase
NCT03375164 , SRP-9001-101 , IRB17-00512
Trial Identifier
SRP-9001
Treatments
Duchenne Muscular Dystrophy
Condition
Official Title
Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin (microDys-IV-001)
Eligibility Criteria
Male
Gender
≥3 Months & ≤ 7 Years
Age
No
Healthy Volunteers
Inclusion Criteria
- 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 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.
For the latest version of this information please go to www.forpatients.roche.com
