A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT ID: NCT04626674
Last Updated: 2025-12-29
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1
83 participants
INTERVENTIONAL
2020-11-23
2028-02-29
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Delandistrogene Moxeparvovec
Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.
delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec
Interventions
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delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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.
* Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥3 at the Screening visit and has a total PUL score of ≥20 and ≤40 at the time of Screening.
* Ability to cooperate with motor assessment testing.
* 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.
* 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.
* rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
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.
* Cohort 8: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients.
2 Years
MALE
No
Sponsors
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Hoffmann-La Roche
INDUSTRY
Sarepta Therapeutics, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Sarepta Therapeutics, Inc.
Locations
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Stanford University
Palo Alto, California, United States
University of California, Davis
Sacramento, California, United States
Washington University in St. Louis
St Louis, Missouri, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States
Countries
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Central Contacts
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Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
Role: CONTACT
Phone: 1-888-SAREPTA (1-888-727-3782)
Email: [email protected]
Facility Contacts
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Role: primary
Role: primary
Role: primary
References
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Potter RA, Moeller IH, Khan S, Haegel H, Hollenstein A, Steiner G, Wandel C, Murphy AP, Asher DR, Palatinsky E, Griffin DA, Mason S, Iannaccone ST, Zaidman CM, Rodino-Klapac LR. Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy. Sci Rep. 2025 Jan 2;15(1):4. doi: 10.1038/s41598-024-84077-w.
Zaidman CM, Proud CM, McDonald CM, Lehman KJ, Goedeker NL, Mason S, Murphy AP, Guridi M, Wang S, Reid C, Darton E, Wandel C, Lewis S, Malhotra J, Griffin DA, Potter RA, Rodino-Klapac LR, Mendell JR. Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged >/=4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR). Ann Neurol. 2023 Nov;94(5):955-968. doi: 10.1002/ana.26755. Epub 2023 Sep 7.
Other Identifiers
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SRP-9001-103
Identifier Type: -
Identifier Source: org_study_id