Trial Search Results
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD)
This is an open-label gene transfer therapy study evaluating the safety of and expression from SRP-9001 (delandistrogene moxeparvovec) in participants with DMD over 260 weeks.
Stanford is currently accepting patients for this trial.
Sarepta Therapeutics, Inc.
Collaborator: Hoffmann-La Roche
- Genetic: SRP-9001
- Cohort 1 only: Is ambulatory, and ≥4 to <8 years of age at the time of Screening.
- Cohort 2 only: Is ambulatory, and ≥8 to <18 years of age at the time of Screening.
- Cohort 3 and Cohort 5b: Non-ambulatory per protocol specified criteria at the time of
- Cohort 4 only: Is ambulatory and ≥3 to <4 years of age at the time of Screening.
- Cohort 5a only: Is ambulatory and ≥4 to <9 years of age at the time of Screening
- For Cohort 1-5: Has a definitive diagnosis of DMD based on documented clinical
findings and prior genetic testing.
- Ability to cooperate with motor assessment testing.
- Cohorts 1, 2, 3, and 5 only: Stable dose equivalent of oral corticosteroids for at
least 12 weeks before screening and the dose is expected to remain constant (except
for modifications to accommodate changes in weight) throughout the first year of the
- Cohort 4: Do not yet require use of chronic steroids for treatment of their DMD, in
the opinion of the Investigator, and are not receiving steroids at the time of
- rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
- Genetic mutation inclusion criteria vary by cohort.
- Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or
cognitive delay/impairment that in the opinion of the Investigator creates unnecessary
risks for gene transfer.
- 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.
Other inclusion/exclusion criteria apply.
Ages Eligible for Study
3 Years - N/A
Genders Eligible for Study