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) (ENVISION)

May 21, 2026 updated by: Sarepta Therapeutics, Inc.

A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP- 9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

148

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Westmead, New South Wales, Australia, 2145
        • The Children's Hospital at Westmead
    • Victoria
      • Parkville, Victoria, Australia, 3052
        • The Royal Children's Hospital
  • Belgium
    • Oost-Vlaanderen
      • Ghent, Oost-Vlaanderen, Belgium, 9000
        • Universitair Ziekenhuis Gent
  • Canada
      • Québec, Canada, G1V 4G2
        • Centre Hospitalier Universitaire de Québec - Université Laval (pavillon Centre Hospitalier Universitaire Laval)
    • Ontario
      • Ottawa, Ontario, Canada, K1H8L1
        • The Children's Hospital of Eastern Ontario
    • Quebec
      • Montreal, Quebec, Canada, H4A3J1
        • Research Institute McGill University Health Centre
  • Germany
      • Hamburg, Germany
        • Universitatsklinikum Hamburg Eppendorf
    • Bavaria
      • München, Bavaria, Germany
        • LMU- Klinikum der Universitat Munchen, Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital, Abeteilung Neuropadiatrie, Campus Innenstadt
    • North Rhine-Westphalia
      • Essen, North Rhine-Westphalia, Germany
        • Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen
  • Hong Kong
      • Hong Kong, Hong Kong
        • Hong Kong Children's Hospital
  • Israel
      • Petah Tikva, Israel
        • Institute of Neruology, Schneider Children's Medical Center of Israel
      • Tel Aviv, Israel, 6423906
        • Tel Aviv Sourasky Medical Center
  • Italy
      • Genova, Italy, 16147
        • U.O.S.D Centro Traslazionale di Miologia e Patologie Neurodegenerative, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico
      • Milan, Italy, 20122
        • UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
      • Milan, Italy, 20133
        • IRCCS Istituto Neurologico Carlo Besta Neurepsichiatria Infantile 2 - Epilettologia e Neurologia dello Sviluppo
      • Roma, Italy, 00168
        • UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS
  • Japan
    • Osaka
      • Toyonaka-shi, Osaka, Japan, 560-8552
        • National Hospital Organization Osaka Toneyama Medical Center
    • Tokyo
      • Kodaira, Tokyo, Japan, 187-8551
        • National Center of Neurology and Psychiatry
      • Shinjuku-ku, Tokyo, Japan, 162-866
        • Tokyo Women's Medical University Hospital
  • South Korea
      • Daegu, South Korea, 41944
        • Kyungpook National University Hospital
      • Seoul, South Korea, 06273
        • Gangnam Severance Hospital, Yonsei University Health System
    • Gyeongsangnam-do
      • Yangsan, Gyeongsangnam-do, South Korea, 50612
        • Pusan National University Yangsan Hospital
    • NAP
      • Seoul, NAP, South Korea, 03080
        • Seoul National University Hospital
  • Spain
      • Valencia, Spain, 46026
        • Hospital Universitari Politecnic La Fe
    • Barcelona
      • Esplugues de Llobregat, Barcelona, Spain, 08950
        • Hospital Sant Joan de Déu
  • Sweden
      • Gothenburg, Sweden, 416 85
        • Sahlgrenska Universitetssjukhuset
      • Solna, Sweden, 171 76
        • Karolinska Universitetssjukhuset/Astrid Lindgrens Barnsjukhus, Barnneurologen
  • Taiwan
      • Kaohsiung City, Taiwan
        • Kaohsiung Medical University Chung-Ho Memorial Hospital
      • Taipei, Taiwan
        • National Taiwan University Hospital
  • United Kingdom
      • Newcastle upon Tyne, United Kingdom, NE13BZ
        • Institute of Translational and Clinical Research
    • Greater London
      • London, Greater London, United Kingdom, WC1N 3JH
        • Great Ormond Street Hospital for Children Foundation Trust
    • Oxfordshire
      • Oxford, Oxfordshire, United Kingdom
        • Oxford University Hospitals NHS Foundation Trust
  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital
    • California
      • Palo Alto, California, United States, 94304
        • Lucile Packard Children's Hospital Stanford
      • Sacramento, California, United States, 95817
        • University of California at Davis Medical Center
      • San Diego, California, United States, 92123
        • Rady Children's Hospital-San Diego
    • Florida
      • Gainesville, Florida, United States, 32608
        • University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Washington University of St. Louis, St. Louis Children's Hospital
    • New York
      • Rochester, New York, United States, 14642
        • University of Rochester, Department of Neurology
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Lenox Baker Children's Hospital (Duke University)
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
    • Virginia
      • Norfolk, Virginia, United States, 23510
        • Children's Hospital of The King's Daughters

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Delandistrogene Moxeparvovec followed by Placebo
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
Single IV infusion of delandistrogene moxeparvovec
Other Names:
  • SRP-9001
  • delandistrogene moxeparvovec-rokl
  • ELEVIDYS
Genetic: placebo
Single IV infusion of matching placebo
Placebo Comparator: Placebo followed by Delandistrogene Moxeparvovec
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
Single IV infusion of delandistrogene moxeparvovec
Other Names:
  • SRP-9001
  • delandistrogene moxeparvovec-rokl
  • ELEVIDYS
Genetic: placebo
Single IV infusion of matching placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72
Time Frame: Baseline, Week 72
Baseline, Week 72

Secondary Outcome Measures

Outcome Measure
Time Frame
Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72
Time Frame: Baseline, Week 72
Baseline, Week 72
Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72
Time Frame: Baseline, Week 72
Baseline, Week 72
Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72
Time Frame: Baseline, 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)
Time Frame: Baseline up to Week 124
Baseline up to Week 124
Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72
Time Frame: Baseline, Week 72
Baseline, Week 72
Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72
Time Frame: Baseline, Week 72
Baseline, Week 72
Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
Time Frame: Week 12
Week 12
Part 1: Change From Baseline in the Middle Domain Score of PUL (Version 2.0) at Week 72
Time Frame: Baseline, Week 72
Baseline, Week 72

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sarepta Therapeutics, Inc.

Collaborators

Hoffmann-La Roche

Investigators

  • Study Director: Medical Director, Sarepta Therapeutics, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 31, 2023

Primary Completion (Estimated)

May 31, 2027

Study Completion (Estimated)

June 30, 2028

Study Registration Dates

First Submitted

May 19, 2023

First Submitted That Met QC Criteria

May 19, 2023

First Posted (Actual)

May 31, 2023

Study Record Updates

Last Update Posted (Actual)

May 22, 2026

Last Update Submitted That Met QC Criteria

May 21, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SRP-9001-303
  • 2020-002372-13 (EudraCT Number)
  • 2024-512626-28-00 (Other Identifier: EU Clinical Trial Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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