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)

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.

Interventions

Study Locations

Facility	City	State	Country
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

Official Trial Information

View on ClinicalTrials.gov