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 trial at your site may differ.
Trial Status:


This trial runs in
  • United States
Trial Identifier:

NCT03375164 IRB17-00512 SRP-9001-101

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      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 trial 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.

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      Trial Summary

      This is an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study will consist of 2 Cohorts. Cohort A will include participants ages 3 months to 3 years, and Cohort B will include participants ages 4 to 7 years old. All participants in the study will receive IV delandistrogene moxeparvovec.

      Sarepta Therapeutics, Inc. Sponsor
      Phase 1/Phase 2 Phase
      NCT03375164,SRP-9001-101,IRB17-00512 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.

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