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)

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

148

Participants

Timeline

Start Date

May 31, 2023

Primary Completion Date

May 31, 2027

Study Completion Date

June 30, 2028

Conditions
Duchenne Muscular Dystrophy
Interventions
GENETIC

delandistrogene moxeparvovec

Single IV infusion of delandistrogene moxeparvovec

GENETIC

placebo

Single IV infusion of matching placebo

Trial Locations (46)

2145

The Children's Hospital at Westmead, Westmead

3052

The Royal Children's Hospital, Parkville

9000

Universitair Ziekenhuis Gent, Ghent

14642

University of Rochester, Department of Neurology, Rochester

16147

U.O.S.D Centro Traslazionale di Miologia e Patologie Neurodegenerative, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico, Genova

19104

Children's Hospital of Philadelphia, Philadelphia

20122

UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan

20133

IRCCS Istituto Neurologico Carlo Besta Neurepsichiatria Infantile 2 - Epilettologia e Neurologia dello Sviluppo, Milan

21287

The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit, Baltimore

23510

Children's Hospital of the King's Daughters, Norfolk

27705

Lenox Baker Children's Hospital (Duke University), Durham

32608

University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases, Gainesville

41944

Kyungpook National University Hospital, Daegu

43205

Nationwide Children's Hospital, Columbus

46026

Hospital Universitari Politecnic La Fe, Valencia

50612

Pusan National University Yangsan Hospital, Yangsan

60611

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

63110

Washington University of St. Louis, St. Louis Children's Hospital, St Louis

72202

Arkansas Children's Hospital, Little Rock

92123

Rady Children's Hospital-San Diego, San Diego

94304

Lucile Packard Children's Hospital Stanford, Palo Alto

95817

University of California at Davis Medical Center, Sacramento

6423906

Tel Aviv Sourasky Medical Center, Tel Aviv

02115

Boston Children's Hospital, Boston

K1H8L1

The Children's Hospital of Eastern Ontario, Ottawa

H4A3J1

Research Institute McGill University Health Centre, Montreal

G1V 4G2

Centre Hospitalier Universitaire de Québec - Université Laval (pavillon Centre Hospitalier Universitaire Laval), Québec

Unknown

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

00168

UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS, Roma

560-8552

National Hospital Organization Osaka Toneyama Medical Center, Toyonaka-shi

187-8551

National Center of Neurology and Psychiatry, Kodaira

162-866

Tokyo Women's Medical University Hospital, Shinjuku-ku

03080

Seoul National University Hospital, Seoul

06273

Gangnam Severance Hospital, Yonsei University Health System, Seoul

08950

Hospital Sant Joan de Deu, Esplugues de Llobregat

416 85

Sahlgrenska Universitetssjukhuset, Gothenburg

171 76

Karolinska Universitetssjukhuset/Astrid Lindgrens Barnsjukhus, Barnneurologen, Solna

WC1N 3JH

Great Ormond Street Hospital for Children Foundation Trust, London

NE13BZ

Institute of Translational and Clinical Research, Newcastle upon Tyne

All Listed Sponsors
collaborator

Hoffmann-La Roche

INDUSTRY

lead

Sarepta Therapeutics, Inc.

INDUSTRY