A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

PHASE3CompletedINTERVENTIONAL
Enrollment

126

Participants

Timeline

Start Date

October 27, 2021

Primary Completion Date

October 4, 2023

Study Completion Date

October 25, 2024

Conditions
Duchenne Muscular Dystrophy
Interventions
GENETIC

delandistrogene moxeparvovec

Single IV infusion of delandistrogene moxeparvovec.

GENETIC

placebo

Single IV infusion of matching placebo.

Trial Locations (42)

807

Kaohsiung Medical University Chung-Ho Memorial Hospital, Kaohsiung City

9000

University Hospital Ghent, Ghent

10032

Columbia University/NYPH, New York

14642

University of Rochester, Rochester

16147

IRCCS Istituto G.Gaslini, U.O., Genoa

19104

The Children's Hospital of Philadelphia, Philadelphia

20122

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

20251

University Hospital Hamburg- Eppendorf, Hamburg

21287

The Johns Hopkins Hospital, Baltimore

23510

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

27705

Duke University Medical Center, Lenox Baker Children's Hospital, Durham

32608

University of Florida, Gainesville

37232

Vanderbilt University Medical Center, Nashville

43210

Nationwide Children's Hospital, Columbus

45147

Universitätsklinikum Essen - Klinik für Kinderheilkunde I, Essen

52242

University of Iowa Stead Family Children's Hospital, Iowa City

53226

Children's Wisconsin, Milwaukee

60611

Lurie Children's Hospital of Chicago, Chicago

63110

Washington University of St. Louis, St Louis

72202

Arkansas Children's, Little Rock

75390

University of Texas Southwestern, Dallas

80045

Children's Hospital Colorado, Aurora

80337

LMU - Klinikum der Universitaet Muenchen - Kinderklinik und, Bayern

84132

University of Utah Hospital, Salt Lake City

90095

UCLA Medical Center, Los Angeles

92037

UC San Diego Altman Clinical and Translational Research Institute, La Jolla

94304

Lucile Packard Children's Hospital at Stanford, Palo Alto

95817

University of California, Davis, Sacramento

97239

Oregon Health & Science University, Portland

100225

National Taiwan University Hospital, Taipei

02115

Boston Children's Hospital, Boston

Unknown

Hong Kong Children's Hospital, Kowloon

Hospital Universitari i Politécnico La Fe, Valencia

00168

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

650-0017

Kobe University Hospital, Kobe

157-8535

National Center for Child Health and Development, Tokyo

162-8666

Tokyo Women's Medical University Hospital - Pediatrics, Tokyo

187-8551

National Center of Neurology and Psychiatry, Tokyo

08950

Hospital Sant Joan de Déu, Barcelona

OX3 9DU

Oxford University Hospitals NHS Foundation Trust, Headington

WC1N3JK

Great Ormond Street Hospital for Children NHS Foundation Trust, London

NE1 4LP

The Newcastle Upon Tyne NHS Hospital NHS Foundation Trust, Royal Victoria Infirmary, Newcastle upon Tyne

All Listed Sponsors
collaborator

Hoffmann-La Roche

INDUSTRY

lead

Sarepta Therapeutics, Inc.

INDUSTRY

NCT05096221 - A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) | Biotech Hunter | Biotech Hunter