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)

NCT05881408 | Active Not Recruiting Phase 3 DMC FDA Drug

• 3 other identifiers: SRP-9001-3032020-002372-132024-512626-28-00
• Study Type: interventional
• Target: 148
• Locations: 14 countries
• Sites: 46

Brief 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.

Conditions

Duchenne Muscular Dystrophy

Keywords

DMD; Gene-Delivery; Pediatric; North Star Ambulatory Assessment (NSAA); Performance of Upper Limb (PUL); Duchenne

Outcome Measures

Primary Outcomes (1)

• Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72

  Baseline, Week 72

Secondary Outcomes (8)

• Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72

  Baseline, Week 72

• Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72

  Baseline, Week 72

• Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot

  Week 12

• Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72

  Baseline, Week 72

• Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)

  Baseline up to Week 124

Study Arms (2)

Delandistrogene Moxeparvovec followed by Placebo

EXPERIMENTAL

Participants will receive single IV infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at approximately 72 weeks.

Genetic: delandistrogene moxeparvovec; Genetic: placebo

Placebo followed by Delandistrogene Moxeparvovec

PLACEBO COMPARATOR

Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of delandistrogene moxeparvovec at approximately 72 weeks.

Genetic: delandistrogene moxeparvovec; Genetic: placebo

Interventions

delandistrogene moxeparvovec GENETIC

Single IV infusion of delandistrogene moxeparvovec

Also known as: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS

Delandistrogene Moxeparvovec followed by Placebo; Placebo followed by Delandistrogene Moxeparvovec

placebo GENETIC

Single IV infusion of matching placebo

Delandistrogene Moxeparvovec followed by Placebo; Placebo followed by Delandistrogene Moxeparvovec

Eligibility Criteria

• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sarepta Therapeutics, Inc.: lead
• Hoffmann-La Roche: collaborator

Study Sites (46)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

Lucile Packard Children's Hospital Stanford

Palo Alto, California, 94304, United States

Location

University of California at Davis Medical Center

Sacramento, California, 95817, United States

Location

Rady Children's Hospital-San Diego

San Diego, California, 92123, United States

Location

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

Gainesville, Florida, 32608, United States

Location

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

Chicago, Illinois, 60611, United States

Location

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

Baltimore, Maryland, 21287, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

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

St Louis, Missouri, 63110, United States

Location

University of Rochester, Department of Neurology

Rochester, New York, 14642, United States

Location

Lenox Baker Children's Hospital (Duke University)

Durham, North Carolina, 27705, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of the King's Daughters

Norfolk, Virginia, 23510, United States

Location

The Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia

Location

The Royal Children's Hospital

Parkville, Victoria, 3052, Australia

Location

Universitair Ziekenhuis Gent

Ghent, Oost-Vlaanderen, 9000, Belgium

Location

The Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H8L1, Canada

Location

Research Institute McGill University Health Centre

Montreal, Quebec, H4A3J1, Canada

Location

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

Québec, G1V 4G2, Canada

Location

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

München, Bavaria, Germany

Location

Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen

Essen, North Rhine-Westphalia, Germany

Location

Universitatsklinikum Hamburg Eppendorf

Hamburg, Germany

Location

Hong Kong Children's Hospital

Hong Kong, Hong Kong

Location

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

Petah Tikva, Israel

Location

Tel Aviv Sourasky Medical Center

Tel Aviv, 6423906, Israel

Location

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

Location

UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

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

Milan, 20133, Italy

Location

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

Roma, 00168, Italy

Location

National Hospital Organization Osaka Toneyama Medical Center

Toyonaka-shi, Osaka, 560-8552, Japan

Location

National Center of Neurology and Psychiatry

Kodaira, Tokyo, 187-8551, Japan

Location

Tokyo Women's Medical University Hospital

Shinjuku-ku, Tokyo, 162-866, Japan

Location

Pusan National University Yangsan Hospital

Yangsan, Gyeongsangnam-do, 50612, South Korea

Location

Seoul National University Hospital

Seoul, NAP, 03080, South Korea

Location

Kyungpook National University Hospital

Daegu, 41944, South Korea

Location

Gangnam Severance Hospital, Yonsei University Health System

Seoul, 06273, South Korea

Location

Hospital Sant Joan de Deu

Esplugues de Llobregat, Barcelona, 08950, Spain

Location

Hospital Universitari Politecnic La Fe

Valencia, 46026, Spain

Location

Sahlgrenska Universitetssjukhuset

Gothenburg, 416 85, Sweden

Location

Karolinska Universitetssjukhuset/Astrid Lindgrens Barnsjukhus, Barnneurologen

Solna, 171 76, Sweden

Location

Kaohsiung Medical University Chung-Ho Memorial Hospital

Kaohsiung City, Taiwan

Location

National Taiwan University Hospital

Taipei, Taiwan

Location

Great Ormond Street Hospital for Children Foundation Trust

London, Greater London, WC1N 3JH, United Kingdom

Location

Oxford University Hospitals NHS Foundation Trust

Oxford, Oxfordshire, United Kingdom

Location

Institute of Translational and Clinical Research

Newcastle upon Tyne, NE13BZ, United Kingdom

Location

Related Links

• Click here to access the website, clinicaltrials.sarepta.com/ENVISION, for additional information for the study.

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Medical Director

  Sarepta Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 19, 2023
• First Posted: May 31, 2023
• Study Start: May 31, 2023
• Primary Completion (Estimated): May 31, 2027
• Study Completion (Estimated): June 30, 2028
• Last Updated: June 22, 2025

Record last verified: 2025-06

Locations

HK (1); IL (2); BE (1); AU (2); DE (3); ES (2); SE (2); IT (4); CA (3); JP (3); GB (3); TW (2); KR (4); US (14)