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Clinical Trials in the USA / NCT05881408
Active, not recruiting Phase 3

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

NCT05881408 · tracked via the Priya Life Science USA tracker
Sponsor
Sarepta Therapeutics, Inc.
Phase
Phase 3
Started
2023-05-31
Last updated
2026-05-22

Condition(s) studied

Duchenne Muscular Dystrophy

Investigational drug(s) / intervention(s)

delandistrogene moxeparvovecplacebo

delandistrogene moxeparvovec: Single IV infusion of delandistrogene moxeparvovec

placebo: Single IV infusion of matching placebo

Study summary

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.

Eligibility

Sex
MALE
Min age
Max age
Healthy volunteers
No
Inclusion Criteria: * Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing. * Cohort 1 only: Non-ambulatory per protocol-specified criteria. * Cohort 2 only: Ambulatory per protocol-specified criteria and ≥8 to \<18 years of age at the time of Screening. * 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). * Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are not elevated as per protocol-specified requirements. * A pathogenic frameshift mutation or premature stop codon in the DMD gene, except for any deletion mutations in exon 8 and/or 9. 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.

Primary outcome measure(s)

Trial sites (46)

FacilityCityRegionStatus
Arkansas Children's Hospital Little Rock Arkansas
Lucile Packard Children's Hospital Stanford Palo Alto California
University of California at Davis Medical Center Sacramento California
Rady Children's Hospital-San Diego San Diego California
University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases Gainesville Florida
Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois
The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit Baltimore Maryland
Boston Children's Hospital Boston Massachusetts
Washington University of St. Louis, St. Louis Children's Hospital St Louis Missouri
University of Rochester, Department of Neurology Rochester New York
Lenox Baker Children's Hospital (Duke University) Durham North Carolina
Nationwide Children's Hospital Columbus Ohio
Children's Hospital of Philadelphia Philadelphia Pennsylvania
Children's Hospital of the King's Daughters Norfolk Virginia
The Children's Hospital at Westmead Westmead New South Wales
The Royal Children's Hospital Parkville Victoria
Universitair Ziekenhuis Gent Ghent Oost-Vlaanderen
The Children's Hospital of Eastern Ontario Ottawa Ontario
Research Institute McGill University Health Centre Montreal Quebec
Centre Hospitalier Universitaire de Québec - Université Laval (pavillon Centre Hospitalier Universitaire Laval) Québec Canada
LMU- Klinikum der Universitat Munchen, Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital, Abeteilung Neuropadiatrie, Campus Innenstadt München Bavaria
Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen Essen North Rhine-Westphalia
Universitatsklinikum Hamburg Eppendorf Hamburg Germany
Hong Kong Children's Hospital Hong Kong Hong Kong
Institute of Neruology, Schneider Children's Medical Center of Israel Petah Tikva Israel
Tel Aviv Sourasky Medical Center Tel Aviv Israel
U.O.S.D Centro Traslazionale di Miologia e Patologie Neurodegenerative, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico Genova Italy
UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan Italy
IRCCS Istituto Neurologico Carlo Besta Neurepsichiatria Infantile 2 - Epilettologia e Neurologia dello Sviluppo Milan Italy
UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS Roma Italy
National Hospital Organization Osaka Toneyama Medical Center Toyonaka-shi Osaka
National Center of Neurology and Psychiatry Kodaira Tokyo
Tokyo Women's Medical University Hospital Shinjuku-ku Tokyo
Pusan National University Yangsan Hospital Yangsan Gyeongsangnam-do
Seoul National University Hospital Seoul NAP
Kyungpook National University Hospital Daegu South Korea
Gangnam Severance Hospital, Yonsei University Health System Seoul South Korea
Hospital Sant Joan de Deu Esplugues de Llobregat Barcelona
Hospital Universitari Politecnic La Fe Valencia Spain
Sahlgrenska Universitetssjukhuset Gothenburg Sweden

+ 6 more sites — see the full list on the official registry below.

More Sarepta Therapeutics, Inc. trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05881408 on ClinicalTrials.gov ↗ ← All trials in the USA