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NCT Number: NCT05881408

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)

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

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

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

The Children's Hospital at Westmead, Westmead, New South Wales, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

delandistrogene moxeparvovec

Genetic

Single IV infusion of delandistrogene moxeparvovec

Other names: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS

placebo

Genetic

Single IV infusion of matching placebo

Primary outcomes

  1. 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

Secondary outcomes

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

    Time frame: Baseline, Week 72

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

    Time frame: Baseline, Week 72

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

    Time frame: Week 12

  4. 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

  5. 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

  6. 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

  7. Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72

    Time frame: Baseline, Week 72

  8. Part 1: Change From Baseline in the Middle Domain Score of PUL (Version 2.0) at Week 72

    Time frame: Baseline, Week 72

Sponsors and collaborators

Lead sponsor

Sarepta Therapeutics, Inc.

Industry

Collaborators

  • Hoffmann-La Roche

Registry information

Official study title

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)

Acronym: ENVISION

Important dates

Study start
2023
Primary completion
2027
Study completion
2028
First posted
May 31, 2023
Registry last updated
May 22, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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