A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) (EMBARK)
Duchenne Muscular Dystrophy
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Muscular Dystrophies, Duchenne Muscular Dystrophy, DMD, North Star Ambulatory Assessment (NSAA), Ambulatory, Pediatric, Gene-Delivery
Eligibility Criteria
Inclusion Criteria:
- Is ambulatory and from 4 to under 8 years of age at time of randomization.
- 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:
- 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.
- 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.
Other inclusion or exclusion criteria could apply.
Sites / Locations
- Arkansas Children's
- UC San Diego Altman Clinical and Translational Research Institute
- UCLA Medical Center
- Lucile Packard Children's Hospital at Stanford
- University of California, Davis
- Children's Hospital Colorado
- University of Florida
- Lurie Children's Hospital of Chicago
- University of Iowa Stead Family Children's Hospital
- The Johns Hopkins Hospital
- Boston Children's Hospital
- Washington University of St. Louis
- Columbia University/NYPH
- University of Rochester
- Duke University Medical Center, Lenox Baker Children's Hospital
- Nationwide Children's Hospital
- Oregon Health & Science University
- The Children's Hospital of Philadelphia
- Vanderbilt University Medical Center
- University of Texas Southwestern
- University of Utah Hospital
- Children's Hospital of the King's Daughters
- Children's Wisconsin
- University Hospital Ghent
- LMU - Klinikum der Universitaet Muenchen - Kinderklinik und
- Universitätsklinikum Essen - Klinik für Kinderheilkunde I
- University Hospital Hamburg- Eppendorf
- Hong Kong Children's Hospital
- IRCCS Istituto G.Gaslini, U.O.
- UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
- UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gamelli IRCCS
- Kobe University Hospital
- National Center for Child Health and Development
- Tokyo Women's Medical University Hospital - Pediatrics
- National Center of Neurology and Psychiatry
- Hospital Universitari i Politécnico La Fe
- Hospital Sant Joan de Déu
- Kaohsiung Medical University Chung-Ho Memorial Hospital
- National Taiwan University Hospital
- Oxford University Hospitals NHS Foundation Trust
- Great Ormond Street Hospital for Children NHS Foundation Trust
- The Newcastle Upon Tyne NHS Hospital NHS Foundation Trust, Royal Victoria Infirmary
Arms of the Study
Arm 1
Arm 2
Experimental
Placebo Comparator
Delandistrogene Moxeparvovec followed by Placebo
Placebo followed by Delandistrogene Moxeparvovec
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.
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.