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Gene therapy breakthrough? new trial targets duchenne muscular dystrophy

NCT ID NCT05881408

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 148 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2023

Expected to finish

Jun 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing. * Cohort 1 only: Non-ambulatory per protocol-specified criteria. * Cohort 2 only: Ambulatory per protocol-specified criteria and ≥8 to \<18 years of age at 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 for modifications to accommodate changes in weight). * Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are not elevated as per protocol-specified requirements. * A pathogenic frameshift mutation or premature stop codon in the DMD gene, except for any deletion mutations in exon 8 and/or 9. Exclusion Criteria: * Exposure to gene therapy, investigational medication, or any treatment designed to increase 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, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer. Other inclusion or exclusion criteria could apply.

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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

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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