148
Participants
Start Date
May 31, 2023
Primary Completion Date
May 31, 2027
Study Completion Date
June 30, 2028
delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec
placebo
Single IV infusion of matching placebo
The Children's Hospital at Westmead, Westmead
The Royal Children's Hospital, Parkville
Universitair Ziekenhuis Gent, Ghent
University of Rochester, Department of Neurology, Rochester
U.O.S.D Centro Traslazionale di Miologia e Patologie Neurodegenerative, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico, Genova
Children's Hospital of Philadelphia, Philadelphia
UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan
IRCCS Istituto Neurologico Carlo Besta Neurepsichiatria Infantile 2 - Epilettologia e Neurologia dello Sviluppo, Milan
The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit, Baltimore
Children's Hospital of the King's Daughters, Norfolk
Lenox Baker Children's Hospital (Duke University), Durham
University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases, Gainesville
Kyungpook National University Hospital, Daegu
Nationwide Children's Hospital, Columbus
Hospital Universitari Politecnic La Fe, Valencia
Pusan National University Yangsan Hospital, Yangsan
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago
Washington University of St. Louis, St. Louis Children's Hospital, St Louis
Arkansas Children's Hospital, Little Rock
Rady Children's Hospital-San Diego, San Diego
Lucile Packard Children's Hospital Stanford, Palo Alto
University of California at Davis Medical Center, Sacramento
Tel Aviv Sourasky Medical Center, Tel Aviv
Boston Children's Hospital, Boston
The Children's Hospital of Eastern Ontario, Ottawa
Research Institute McGill University Health Centre, Montreal
Centre Hospitalier Universitaire de Québec - Université Laval (pavillon Centre Hospitalier Universitaire Laval), Québec
LMU- Klinikum der Universitat Munchen, Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital, Abeteilung Neuropadiatrie, Campus Innenstadt, München
Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen, Essen
Universitatsklinikum Hamburg Eppendorf, Hamburg
Hong Kong Children's Hospital, Hong Kong
Institute of Neruology, Schneider Children's Medical Center of Israel, Petah Tikva
Kaohsiung Medical University Chung-Ho Memorial Hospital, Kaohsiung City
National Taiwan University Hospital, Taipei
Oxford University Hospitals NHS Foundation Trust, Oxford
UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS, Roma
National Hospital Organization Osaka Toneyama Medical Center, Toyonaka-shi
National Center of Neurology and Psychiatry, Kodaira
Tokyo Women's Medical University Hospital, Shinjuku-ku
Seoul National University Hospital, Seoul
Gangnam Severance Hospital, Yonsei University Health System, Seoul
Hospital Sant Joan de Deu, Esplugues de Llobregat
Sahlgrenska Universitetssjukhuset, Gothenburg
Karolinska Universitetssjukhuset/Astrid Lindgrens Barnsjukhus, Barnneurologen, Solna
Great Ormond Street Hospital for Children Foundation Trust, London
Institute of Translational and Clinical Research, Newcastle upon Tyne
Hoffmann-La Roche
INDUSTRY
Sarepta Therapeutics, Inc.
INDUSTRY