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ENVISION: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 (Delandistrogene Moxeparvovec) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

Sponsor
Sarepta Therapeutics, Inc. (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05881408
Collaborator
Hoffmann-La Roche (Industry)
148
Enrollment
14
Locations
2
Arms
43.1
Anticipated Duration (Months)
10.6
Patients Per Site
0.2
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The study will evaluate the safety and efficacy of SRP-9001 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) SRP-9001 (delandistrogene moxeparvovec) in either Part 1 or Part 2.

Condition or Disease Intervention/Treatment Phase
  • Genetic: SRP-9001
  • Genetic: Placebo
 Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
148 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
Official 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)
Anticipated Study Start Date :
Jun 30, 2023
Anticipated Primary Completion Date :
Jan 31, 2026
Anticipated Study Completion Date :
Jan 31, 2027

Arms and Interventions

Arm Intervention/Treatment
Experimental: SRP-9001 followed by Placebo

Participants will receive single IV infusion of SRP-9001 on Day 1. Then, participants will receive a single IV infusion of matching placebo at approximately 72 weeks.

Genetic: SRP-9001
Single IV infusion of SRP-9001
Other Names:
  • delandistrogene moxeparvovec

    • Genetic: Placebo
    Single IV infusion of matching placebo
    Placebo Comparator: Placebo followed by SRP-9001

    Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of SRP-9001 at approximately 72 weeks.

    Genetic: SRP-9001
    Single IV infusion of SRP-9001
    Other Names:
  • delandistrogene moxeparvovec

    • Genetic: Placebo
    Single IV infusion of matching placebo

    Outcome Measures

    Primary Outcome Measures

    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 Outcome Measures

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

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

    3. Part 1: Quantity of SRP-9001 Protein Expression at Week 12 as Measured by Western Blot [Week 12]

    4. Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72 [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) [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 [Baseline, Week 72]

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

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    Male
    Accepts Healthy Volunteers:
    No
    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 contained between exons 18 and 79 (inclusive).

    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Arkansas Children's Little Rock Arkansas United States 72202
    2 Rady Children's Hospital-San Diego San Diego California United States 92123
    3 University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases Gainesville Florida United States 32608
    4 Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois United States 60611
    5 University of Iowa Hospitals and Clinics, Dept of Pediatrics Iowa City Iowa United States 52242
    6 The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit Baltimore Maryland United States 21287
    7 Boston Children's Hospital Boston Massachusetts United States 02115
    8 Washington University of St. Louis, St. Louis Children's Hospital Saint Louis Missouri United States 63110
    9 University of Rochester, Dept of Neurology Rochester New York United States 14642
    10 Nationwide Children's Hospital Columbus Ohio United States 43205
    11 Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
    12 VUMC- Monroe Carell Jr. Children's Hospital at Vanderbilt Nashville Tennessee United States 37203
    13 Children's Hospital of the King's Daughters Norfolk Virginia United States 23510
    14 Children's Wisconsin Milwaukee Wisconsin United States 53226

    Sponsors and Collaborators

    • Sarepta Therapeutics, Inc.
    • Hoffmann-La Roche

    Investigators

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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Sarepta Therapeutics, Inc.
    ClinicalTrials.gov Identifier:
    NCT05881408
    Other Study ID Numbers:
    • SRP-9001-303
    • 2020-002372-13
    First Posted:
    May 31, 2023
    Last Update Posted:
    May 31, 2023
    Last Verified:
    May 1, 2023
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Sarepta Therapeutics, Inc.
    Ambulatory
    Non-ambulatory
    DMD
    Gene-Delivery
    Pediatric
    North Star Ambulatory Assessment (NSAA)
    Performance of Upper Limb (PUL)
    Duchenne
    Additional relevant MeSH terms:
    Muscular Dystrophies
    Muscular Dystrophy, Duchenne

    Study Results

    No Results Posted as of May 31, 2023