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

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

  • Muscle And Peripheral Nerve Disease
  • Duchenne Muscular Dystrophy (DMD)
Please note that the recruitment status of the study at your site may differ from the overall study status because some study sites may recruit earlier than others.
Study Status:

Completed

This study runs in
City
  • Columbus
Trial Identifier:

NCT03375164 IRB17-00512 2021-000077-83 SRP-9001-101

  • Nationwide Children's Hospital

    700 Children's Dr43205ColumbusUnited States
    +−
    Show study locations

    The source of the below information is public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc.. It has been summarised and edited into simpler language. For more information about this clinical study see the For Expert tab on the specific ForPatients page or follow these links to https://clinicaltrials.gov and/or https://euclinicaltrials.eu and/or https://www.isrctn.com.

    The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc., and has not been edited.

    Results Disclaimer

    Study Summary

    This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study was originally designed to consist of 12 patients across 2 Cohorts. Cohort A would have included participants ages 3 months to 3 years, and Cohort B included participants ages 4 to 7 years old. No participants were enrolled in Cohort A.

    Sarepta Therapeutics, Inc. Sponsor
    Phase 1/Phase 2 Phase
    NCT03375164, SRP-9001-101, IRB17-00512, 2021-000077-83 Trial Identifier
    delandistrogene moxeparvovec Treatments
    Duchenne Muscular Dystrophy Condition
    Official Title

    Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin (microDys-IV-001)

    Eligibility Criteria

    Male Gender
    ≥3 Months & ≤ 7 Years Age
    No Healthy Volunteers
    Inclusion Criteria
    • Cohort A participants: 3 months to 3 years of age, inclusive
    • Cohort B participants: 4 to 7 years of age, inclusive
    • Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
    • Ability to cooperate with motor assessment testing.
    • Cohort A participants: No previous treatment with corticosteroids.
    • Cohort B participants: Stable dose equivalent of oral corticosteroids for at least 12 weeks prior to screening and the dose is expected to remain constant (except for potential modifications to accommodate changes in weight) throughout the first year of the study.
    • Cohorts A & B: A frameshift mutation contained between exons 18 and 58 (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.

    For the latest version of this information please go to www.forpatients.roche.com

     

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