126
Participants
Start Date
October 27, 2021
Primary Completion Date
October 4, 2023
Study Completion Date
October 25, 2024
delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec.
placebo
Single IV infusion of matching placebo.
Kaohsiung Medical University Chung-Ho Memorial Hospital, Kaohsiung City
University Hospital Ghent, Ghent
Columbia University/NYPH, New York
University of Rochester, Rochester
IRCCS Istituto G.Gaslini, U.O., Genoa
The Children's Hospital of Philadelphia, Philadelphia
UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan
University Hospital Hamburg- Eppendorf, Hamburg
The Johns Hopkins Hospital, Baltimore
Children's Hospital of the King's Daughters, Norfolk
Duke University Medical Center, Lenox Baker Children's Hospital, Durham
University of Florida, Gainesville
Vanderbilt University Medical Center, Nashville
Nationwide Children's Hospital, Columbus
Universitätsklinikum Essen - Klinik für Kinderheilkunde I, Essen
University of Iowa Stead Family Children's Hospital, Iowa City
Children's Wisconsin, Milwaukee
Lurie Children's Hospital of Chicago, Chicago
Washington University of St. Louis, St Louis
Arkansas Children's, Little Rock
University of Texas Southwestern, Dallas
Children's Hospital Colorado, Aurora
LMU - Klinikum der Universitaet Muenchen - Kinderklinik und, Bayern
University of Utah Hospital, Salt Lake City
UCLA Medical Center, Los Angeles
UC San Diego Altman Clinical and Translational Research Institute, La Jolla
Lucile Packard Children's Hospital at Stanford, Palo Alto
University of California, Davis, Sacramento
Oregon Health & Science University, Portland
National Taiwan University Hospital, Taipei
Boston Children's Hospital, Boston
Hong Kong Children's Hospital, Kowloon
Hospital Universitari i Politécnico La Fe, Valencia
UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gamelli IRCCS, Roma
Kobe University Hospital, Kobe
National Center for Child Health and Development, Tokyo
Tokyo Women's Medical University Hospital - Pediatrics, Tokyo
National Center of Neurology and Psychiatry, Tokyo
Hospital Sant Joan de Déu, Barcelona
Oxford University Hospitals NHS Foundation Trust, Headington
Great Ormond Street Hospital for Children NHS Foundation Trust, London
The Newcastle Upon Tyne NHS Hospital NHS Foundation Trust, Royal Victoria Infirmary, Newcastle upon Tyne
Hoffmann-La Roche
INDUSTRY
Sarepta Therapeutics, Inc.
INDUSTRY