Gene therapy breakthrough? new trial targets duchenne muscular dystrophy

NCT ID NCT05881408

Ongoing Disease control Sponsor: Sarepta Therapeutics, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This Phase 3 trial tests a gene therapy called SRP-9001 for Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. It includes 148 males who can and cannot walk. Participants receive a one-time IV infusion of the therapy or a placebo, and are followed for about 128 weeks to see if it improves arm function and breathing.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: delandistrogene moxeparvovec (gene therapy)
What this could lead to: If successful, this gene therapy could slow muscle decline and improve upper limb function in people with Duchenne muscular dystrophy.
What could go wrong: This is an early Phase 3 trial with 148 participants, so results may not apply to everyone. Gene therapy carries risks like immune reactions or liver issues.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DUCHENNE MUSCULAR DYSTROPHY

Contacts and locations

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Locations

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

Chicago, Illinois, 60611, United States
Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States
Boston Children's Hospital

Boston, Massachusetts, 02115, United States
Centre Hospitalier Universitaire de Québec - Université Laval (pavillon Centre Hospitalier Universitaire Laval)

Québec, G1V 4G2, Canada
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of the King's Daughters

Norfolk, Virginia, 23510, United States
Gangnam Severance Hospital, Yonsei University Health System

Seoul, 06273, South Korea
Great Ormond Street Hospital for Children Foundation Trust

London, Greater London, WC1N 3JH, United Kingdom
Hong Kong Children's Hospital

Hong Kong, Hong Kong
Hospital Sant Joan de Deu

Esplugues de Llobregat, Barcelona, 08950, Spain
Hospital Universitari Politecnic La Fe

Valencia, 46026, Spain
IRCCS Istituto Neurologico Carlo Besta Neurepsichiatria Infantile 2 - Epilettologia e Neurologia dello Sviluppo

Milan, 20133, Italy
Institute of Neruology, Schneider Children's Medical Center of Israel

Petah Tikva, Israel
Institute of Translational and Clinical Research

Newcastle upon Tyne, NE13BZ, United Kingdom
Kaohsiung Medical University Chung-Ho Memorial Hospital

Kaohsiung City, Taiwan
Karolinska Universitetssjukhuset/Astrid Lindgrens Barnsjukhus, Barnneurologen

Solna, 171 76, Sweden
Kyungpook National University Hospital

Daegu, 41944, South Korea
LMU- Klinikum der Universitat Munchen, Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital, Abeteilung Neuropadiatrie, Campus Innenstadt

München, Bavaria, Germany
Lenox Baker Children's Hospital (Duke University)

Durham, North Carolina, 27705, United States
Lucile Packard Children's Hospital Stanford

Palo Alto, California, 94304, United States
National Center of Neurology and Psychiatry

Kodaira, Tokyo, 187-8551, Japan
National Hospital Organization Osaka Toneyama Medical Center

Toyonaka-shi, Osaka, 560-8552, Japan
National Taiwan University Hospital

Taipei, Taiwan
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Oxford University Hospitals NHS Foundation Trust

Oxford, Oxfordshire, United Kingdom
Pusan National University Yangsan Hospital

Yangsan, Gyeongsangnam-do, 50612, South Korea
Rady Children's Hospital-San Diego

San Diego, California, 92123, United States
Research Institute McGill University Health Centre

Montreal, Quebec, H4A3J1, Canada
Sahlgrenska Universitetssjukhuset

Gothenburg, 416 85, Sweden
Seoul National University Hospital

Seoul, NAP, 03080, South Korea
Tel Aviv Sourasky Medical Center

Tel Aviv, 6423906, Israel
The Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia
The Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H8L1, Canada
The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit

Baltimore, Maryland, 21287, United States
The Royal Children's Hospital

Parkville, Victoria, 3052, Australia
Tokyo Women's Medical University Hospital

Shinjuku-ku, Tokyo, 162-866, Japan
U.O.S.D Centro Traslazionale di Miologia e Patologie Neurodegenerative, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico

Genova, 16147, Italy
UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy
UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS

Roma, 00168, Italy
Universitair Ziekenhuis Gent

Ghent, Oost-Vlaanderen, 9000, Belgium
Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen

Essen, North Rhine-Westphalia, Germany
Universitatsklinikum Hamburg Eppendorf

Hamburg, Germany
University of California at Davis Medical Center

Sacramento, California, 95817, United States
University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases

Gainesville, Florida, 32608, United States
University of Rochester, Department of Neurology

Rochester, New York, 14642, United States
Washington University of St. Louis, St. Louis Children's Hospital

St Louis, Missouri, 63110, United States

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