A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT ID: NCT05096221
Last Updated: 2025-07-08
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
126 participants
INTERVENTIONAL
2021-10-27
2024-10-25
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Delandistrogene Moxeparvovec followed by Placebo
Participants will receive single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at Year 2.
delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec.
placebo
Single IV infusion of matching placebo.
Placebo followed by Delandistrogene Moxeparvovec
Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of delandistrogene moxeparvovec at Year 2.
delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec.
placebo
Single IV infusion of matching placebo.
Interventions
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delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec.
placebo
Single IV infusion of matching placebo.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
* Ability to cooperate with motor assessment testing.
* Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
* rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
* A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive), with the exception of mutation fully contained within exon 45.
Exclusion Criteria
* 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.
4 Years
7 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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Arkansas Children's
Little Rock, Arkansas, United States
UC San Diego Altman Clinical and Translational Research Institute
La Jolla, California, United States
UCLA Medical Center
Los Angeles, California, United States
Lucile Packard Children's Hospital at Stanford
Palo Alto, California, United States
University of California, Davis
Sacramento, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
University of Florida
Gainesville, Florida, United States
Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, United States
The Johns Hopkins Hospital
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Washington University of St. Louis
St Louis, Missouri, United States
Columbia University/NYPH
New York, New York, United States
University of Rochester
Rochester, New York, United States
Duke University Medical Center, Lenox Baker Children's Hospital
Durham, North Carolina, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
University of Texas Southwestern
Dallas, Texas, United States
University of Utah Hospital
Salt Lake City, Utah, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States
Children's Wisconsin
Milwaukee, Wisconsin, United States
University Hospital Ghent
Ghent, , Belgium
LMU - Klinikum der Universitaet Muenchen - Kinderklinik und
Bayern, , Germany
Universitätsklinikum Essen - Klinik für Kinderheilkunde I
Essen, , Germany
University Hospital Hamburg- Eppendorf
Hamburg, , Germany
Hong Kong Children's Hospital
Kowloon, , Hong Kong
IRCCS Istituto G.Gaslini, U.O.
Genoa, , Italy
UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, , Italy
UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gamelli IRCCS
Roma, , Italy
Kobe University Hospital
Kobe, , Japan
National Center for Child Health and Development
Tokyo, , Japan
Tokyo Women's Medical University Hospital - Pediatrics
Tokyo, , Japan
National Center of Neurology and Psychiatry
Tokyo, , Japan
Hospital Universitari i Politécnico La Fe
Valencia, Comunidad Valencia, Spain
Hospital Sant Joan de Déu
Barcelona, , Spain
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, , Taiwan
National Taiwan University Hospital
Taipei, , Taiwan
Oxford University Hospitals NHS Foundation Trust
Headington, Oxford, United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust
London, , United Kingdom
The Newcastle Upon Tyne NHS Hospital NHS Foundation Trust, Royal Victoria Infirmary
Newcastle upon Tyne, , United Kingdom
Countries
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References
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Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Guridi M, Murphy AP, Reid C, Wandel C, Asher DR, Darton E, Mason S, Potter RA, Singh T, Zhang W, Fontoura P, Elkins JS, Rodino-Klapac LR. AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial. Nat Med. 2025 Jan;31(1):332-341. doi: 10.1038/s41591-024-03304-z. Epub 2024 Oct 9.
Vandenborne K, Walter GA, Straub V, Willcocks RJ, Forbes SC, Mercuri EM, Muntoni F, Ding K, Ennamuri S, Reid C, Murphy AP, Manfrini M, Mendell JR, Elkins JS, Rodino-Klapac LR. Quantitative Muscle Magnetic Resonance Outcomes in Patients With Duchenne Muscular Dystrophy: An Exploratory Analysis From the EMBARK Randomized Clinical Trial. JAMA Neurol. 2025 Jul 1;82(7):734-744. doi: 10.1001/jamaneurol.2025.0992.
McDonald CM, Elkins JS, Dharmarajan S, Gooch K, Ciobanu T, Lansdall CJ, Murphy AP, McDougall F, Mercuri EM, Audhya I; EMBARK Study Group. Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy. Neurol Ther. 2025 Feb;14(1):211-225. doi: 10.1007/s40120-024-00685-8. Epub 2024 Nov 26.
Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Related Links
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SRP-9001-301 Plain Language Study Summary
Other Identifiers
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2019-003374-91
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
SRP-9001-301
Identifier Type: -
Identifier Source: org_study_id
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