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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)
Sponsor: Sarepta Therapeutics, Inc.
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.
Official title: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP- 9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)
Key Details
Gender
MALE
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
148
Start Date
2023-05-31
Completion Date
2028-06-30
Last Updated
2025-06-22
Healthy Volunteers
No
Conditions
Interventions
delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec
placebo
Single IV infusion of matching placebo
Locations (46)
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Lucile Packard Children's Hospital Stanford
Palo Alto, California, United States
University of California at Davis Medical Center
Sacramento, California, United States
Rady Children's Hospital-San Diego
San Diego, California, United States
University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases
Gainesville, Florida, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Washington University of St. Louis, St. Louis Children's Hospital
St Louis, Missouri, United States
University of Rochester, Department of Neurology
Rochester, New York, United States
Lenox Baker Children's Hospital (Duke University)
Durham, North Carolina, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States
The Children's Hospital at Westmead
Westmead, New South Wales, Australia
The Royal Children's Hospital
Parkville, Victoria, Australia
Universitair Ziekenhuis Gent
Ghent, Oost-Vlaanderen, Belgium
The Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada
Research Institute McGill University Health Centre
Montreal, Quebec, Canada
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, Germany
Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen
Essen, North Rhine-Westphalia, Germany
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, Japan
National Center of Neurology and Psychiatry
Kodaira, Tokyo, Japan
Tokyo Women's Medical University Hospital
Shinjuku-ku, Tokyo, Japan
Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do, South Korea
Seoul National University Hospital
Seoul, NAP, South Korea
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, Spain
Hospital Universitari Politecnic La Fe
Valencia, Spain
Sahlgrenska Universitetssjukhuset
Gothenburg, Sweden
Karolinska Universitetssjukhuset/Astrid Lindgrens Barnsjukhus, Barnneurologen
Solna, Sweden
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, Taiwan
National Taiwan University Hospital
Taipei, Taiwan
Great Ormond Street Hospital for Children Foundation Trust
London, Greater London, United Kingdom
Oxford University Hospitals NHS Foundation Trust
Oxford, Oxfordshire, United Kingdom
Institute of Translational and Clinical Research
Newcastle upon Tyne, United Kingdom