Tests a gene therapy in non-walking people with Duchenne muscular dystrophy
Tests delandistrogene moxeparvovec in non-ambulatory people with Duchenne muscular dystrophy aged 2 and older.
Plain-language title generated from the registry record
Registry title: A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort
Key facts
- Where
- Chicago, Columbus, Durham, Flower Mound, Gainesville, La Jolla, Little Rock, Los Angeles, Norfolk, Palo Alto, Sacramento, and St Louis, United StatesSee the 12 sites
- Phase
- Phase 1
Where in United States
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, United StatesTaking people
- Nationwide Children's HospitalColumbus, United StatesNo longer taking part
- Duke University Medical CenterDurham, United StatesTaking people
- Neurology Rare Disease CenterFlower Mound, United StatesTaking people
- University of FloridaGainesville, United StatesTaking people
- University of California San DiegoLa Jolla, United StatesTaking people
- Arkansas Children's HospitalLittle Rock, United StatesTaking people
- University of California, Los AngelesLos Angeles, United StatesTaking people
- Children's Hospital of The King's DaughtersNorfolk, United StatesTaking people
- Stanford UniversityPalo Alto, United StatesTaking people
- University of California - DavisSacramento, United StatesTaking people
- Washington University in St. LouisSt Louis, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Sarepta Therapeutics, Inc.
- Trial code
- NCT04626674
- Record updated
- 11 September 2026
- Original description
Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.Read full original descriptionShow less
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)
Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.
This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.
Inclusion: For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing; Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥2 at the Screening visit and has a total PUL score of ≥12 and ≤40 at the time of Screening; Cohorts 1, 2, 3, 5, 7 and 8 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 study; 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 with time to rise from the floor ≤7 seconds at the screening visit; 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; 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 Screening; Genetic mutation inclusion criteria vary by cohort; All Cohorts:; Ability to cooperate with motor assessment testing; rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
Exclusion: 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; Cohort 8: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients; Abnormality in protocol-specified diagnostic evaluations or laboratory tests; Note: Other inclusion/exclusion criteria apply.