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)

Last updated: April 11, 2025
Sponsor: Sarepta Therapeutics, Inc.
Overall Status: Active - Recruiting

Phase

3

Condition

Muscular Dystrophy

Treatment

delandistrogene moxeparvovec

placebo

Clinical Study ID

NCT05881408
SRP-9001-303
2024-512626-28-00
2020-002372-13
  • Male

Study Summary

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Definitive diagnosis of DMD based on documented clinical findings and prior genetictesting.

  • Cohort 1 only: Non-ambulatory per protocol-specified criteria.

  • Cohort 2 only: Ambulatory per protocol-specified criteria and ≥8 to <18 years of ageat the time of Screening.

  • Ability to cooperate with motor assessment testing.

  • Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening,and the dose is expected to remain constant throughout the study (except formodifications to accommodate changes in weight).

  • Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are notelevated as per protocol-specified requirements.

  • A pathogenic frameshift mutation or premature stop codon in the DMD gene, except forany deletion mutations in exon 8 and/or 9.

Exclusion

Exclusion Criteria:

  • Exposure to gene therapy, investigational medication, or any treatment designed toincrease dystrophin expression within protocol specified time limits.

  • Abnormality in protocol-specified diagnostic evaluations or laboratory tests.

  • Presence of any other clinically significant illness, medical condition, orrequirement for chronic drug treatment that in the opinion of the Investigatorcreates unnecessary risk for gene transfer.

Other inclusion or exclusion criteria could apply.

Study Design

Total Participants: 148
Treatment Group(s): 2
Primary Treatment: delandistrogene moxeparvovec
Phase: 3
Study Start date:
May 31, 2023
Estimated Completion Date:
June 30, 2028

Connect with a study center

  • The Children's Hospital at Westmead

    Westmead, New South Wales 2145
    Australia

    Active - Recruiting

  • The Royal Children's Hospital

    Parkville, Victoria 3052
    Australia

    Active - Recruiting

  • Universitair Ziekenhuis Gent

    Gent, Oost-Vlaanderen 9000
    Belgium

    Active - Recruiting

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

    Munchen, Bayern
    Germany

    Active - Recruiting

  • Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen

    Essen, Nordrhein-westfalen
    Germany

    Active - Recruiting

  • Universitatsklinikum Hamburg Eppendorf

    Hamburg,
    Germany

    Active - Recruiting

  • Hong Kong Children's Hospital

    Hong Kong,
    Hong Kong

    Active - Recruiting

  • Institute of Neruology, Schneider Children's Medical Center of Israel

    Petach Tikva,
    Israel

    Active - Recruiting

  • Tel Aviv Sourasky Medical Center

    Tel Aviv, 6423906
    Israel

    Active - Recruiting

  • 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

    Active - Recruiting

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

    Milano, 20122
    Italy

    Active - Recruiting

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

    Roma, 00168
    Italy

    Active - Recruiting

  • National Hospital Organization Osaka Toneyama Medical Center

    Toyonaka-shi, Osaka 560-8552
    Japan

    Active - Recruiting

  • National Center of Neurology and Psychiatry

    Kodaira, Tokyo 187-8551
    Japan

    Active - Recruiting

  • Tokyo Women's Medical University Hospital

    Shinjuku-ku, Tokyo 162-866
    Japan

    Active - Recruiting

  • Pusan National University Yangsan Hospital

    Yangsan, Gyeongsangnam-do 50612
    Korea, Republic of

    Active - Recruiting

  • Seoul National University Hospital

    Seoul, NAP 03080
    Korea, Republic of

    Active - Recruiting

  • Kyungpook National University Hospital

    Daegu, 41944
    Korea, Republic of

    Active - Recruiting

  • Gangnam Severance Hospital, Yonsei University Health System

    Seoul, 06273
    Korea, Republic of

    Active - Recruiting

  • Hospital Sant Joan de Deu

    Esplugues de LLobregat, Barcelona 08950
    Spain

    Active - Recruiting

  • Hospital Universitari Politecnic La Fe

    Valencia, 46026
    Spain

    Active - Recruiting

  • Sahlgrenska Universitetssjukhuset

    Goteborg, 416 85
    Sweden

    Active - Recruiting

  • Karolinska Universitetssjukhuset/Astrid Lindgrens Barnsjukhus, Barnneurologen

    Solna, 171 76
    Sweden

    Active - Recruiting

  • Kaohsiung Medical University Chung-Ho Memorial Hospital

    Kaohsiung,
    Taiwan

    Active - Recruiting

  • National Taiwan University Hospital

    Taipei City,
    Taiwan

    Active - Recruiting

  • Great Ormond Street Hospital for Children Foundation Trust

    London, Greater London WC1N 3JH
    United Kingdom

    Active - Recruiting

  • Oxford University Hospitals NHS Foundation Trust

    Oxford, Oxfordshire
    United Kingdom

    Active - Recruiting

  • Institute of Translational and Clinical Research

    Newcastle Upon Tyne, NE13BZ
    United Kingdom

    Active - Recruiting

  • Arkansas Children's

    Little Rock, Arkansas 72202
    United States

    Site Not Available

  • Arkansas Children's Hospital

    Little Rock, Arkansas 72202
    United States

    Site Not Available

  • Lucile Packard Children's Hospital Stanford

    Palo Alto, California 94304
    United States

    Site Not Available

  • University of California at Davis Medical Center

    Sacramento, California 95817
    United States

    Site Not Available

  • Rady Children's Hospital-San Diego

    San Diego, California 92123
    United States

    Site Not Available

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

    Gainesville, Florida 32608
    United States

    Site Not Available

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

    Chicago, Illinois 60611
    United States

    Site Not Available

  • University of Iowa Hospitals and Clinics, Dept of Pediatrics

    Iowa City, Iowa 52242
    United States

    Site Not Available

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

    Baltimore, Maryland 21287
    United States

    Site Not Available

  • Boston Children's Hospital

    Boston, Massachusetts 02115
    United States

    Site Not Available

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

    Saint Louis, Missouri 63110
    United States

    Terminated

  • University of Rochester, Department of Neurology

    Rochester, New York 14642
    United States

    Site Not Available

  • University of Rochester, Dept of Neurology

    Rochester, New York 14642
    United States

    Site Not Available

  • Lenox Baker Children's Hospital (Duke University)

    Durham, North Carolina 27705
    United States

    Site Not Available

  • Nationwide Children's Hospital

    Columbus, Ohio 43205
    United States

    Site Not Available

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • VUMC- Monroe Carell Jr. Children's Hospital at Vanderbilt

    Nashville, Tennessee 37203
    United States

    Site Not Available

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia 23510
    United States

    Site Not Available

  • Children's Wisconsin

    Milwaukee, Wisconsin 53226
    United States

    Site Not Available

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