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ENVOL: A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)

Sponsor
Hoffmann-La Roche (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT06128564
Collaborator
Sarepta Therapeutics, Inc. (Industry)
21
Enrollment
1
Arm
108.5
Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.

Condition or Disease Intervention/Treatment Phase
  • Genetic: delandistrogene moxeparvovec
 Phase 2

Study Design

Study Type:
Interventional
Anticipated Enrollment :
21 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Two-Part, Open-Label Systemic Gene Delivery Study to Evaluate the Safety and Expression of RO7494222 (SRP-9001) in Subjects Under the Age of Four With Duchenne Muscular Dystrophy
Anticipated Study Start Date :
Nov 16, 2023
Anticipated Primary Completion Date :
Nov 30, 2032
Anticipated Study Completion Date :
Nov 30, 2032

Arms and Interventions

Arm Intervention/Treatment
Experimental: Delandistrogene Moxeparvovec

Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.

Genetic: delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec
Other Names:
  • SRP-9001
  • ELEVIDYS
  • RO7494222
  • delandistrogene moxeparvovec-rokl

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Percentage of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) [Baseline up to Week 260]

    Secondary Outcome Measures

    1. Change in Quantity of Delandistrogene Moxeparvovec Dystrophin as Measured by Western Blot [Baseline, Week 12]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A to 3 Years
    Sexes Eligible for Study:
    Male
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Cohort A: >=3 years of age to <4 years of age

    • Cohort B: >=2 years of age to <3 years of age

    • Cohort C: >6 months to <2 years of age

    • Cohort D: <=6 months of age

    • Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test

    • Able to cooperate with age-appropriate motor assessment testing

    • 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

    • Recombinant Adeno-Associated Virus Serotype rh74 (rAArh74) antibody titers are elevated, as per protocol-specified criteria

    • Receiving regular oral corticosteroids as a treatment for DMD or planning to receive oral corticosteroids as a treatment for DMD within 1 year of baseline

    • 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

    • Medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the participant's ability to comply with the protocol required testing or procedures, or compromise the participant's well-being or safety, or clinical interpretability

    Other inclusion or exclusion criteria could apply

    Contacts and Locations

    Locations

    No locations specified.

    Sponsors and Collaborators

    • Hoffmann-La Roche
    • Sarepta Therapeutics, Inc.

    Investigators

    • Study Director: Clinical Trials, Hoffmann-La Roche

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Hoffmann-La Roche
    ClinicalTrials.gov Identifier:
    NCT06128564
    Other Study ID Numbers:
    • BN43881
    • 2022-000691-19
    First Posted:
    Nov 13, 2023
    Last Update Posted:
    Nov 13, 2023
    Last Verified:
    Nov 1, 2023
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Product Manufactured in and Exported from the U.S.:
    Yes
    Keywords provided by Hoffmann-La Roche
    DMD
    Duchenne
    Dystrophin
    Gene-Delivery
    Pediatric
    Additional relevant MeSH terms:
    Muscular Dystrophies
    Muscular Dystrophy, Duchenne

    Study Results

    No Results Posted as of Nov 13, 2023