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A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Trial ID: NCT04626674
This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD. The maximum participant duration for this study is 156 weeks.
An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)
- For Cohorts 1-7: Has a definitive diagnosis of DMD based on documented clinical
findings and prior genetic testing.
- Cohort 1: Is ambulatory, and ≥4 to <8 years of age at the time of Screening.
- Cohort 2: Is ambulatory, and ≥8 to <18 years of age at the time of Screening.
- Cohort 3: Non-ambulatory per protocol specified criteria at the time of Screening.
- Cohort 4: Is ambulatory and ≥3 to <4 years of age at the time of Screening.
- Cohort 5a: Is ambulatory and ≥4 to <9 years of age.
- Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening.
- Cohort 6: Is ambulatory, and ≥2 to <3 years of age at the time of Screening.
- Cohort 7: Non-ambulatory per protocol-specified criteria at the time of Screening.
- Ability to cooperate with motor assessment testing.
- Cohorts 1, 2, 3, 5, and 7 only: Stable dose equivalent of oral glucocorticoids 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
- Cohorts 4 and 6: 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.
genetic: delandistrogene moxeparvovec