A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)
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ClinicalTrials.gov Identifier: NCT03769116 |
Recruitment Status :
Completed
First Posted : December 7, 2018
Results First Posted : September 15, 2023
Last Update Posted : October 25, 2023
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Tracking Information | |||||
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First Submitted Date ICMJE | December 6, 2018 | ||||
First Posted Date ICMJE | December 7, 2018 | ||||
Results First Submitted Date ICMJE | July 5, 2023 | ||||
Results First Posted Date ICMJE | September 15, 2023 | ||||
Last Update Posted Date | October 25, 2023 | ||||
Actual Study Start Date ICMJE | December 5, 2018 | ||||
Actual Primary Completion Date | December 8, 2020 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
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Change History | |||||
Current Secondary Outcome Measures ICMJE | Not Provided | ||||
Original Secondary Outcome Measures ICMJE | Not Provided | ||||
Current Other Pre-specified Outcome Measures |
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Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD) | ||||
Official Title ICMJE | A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial for Duchenne Muscular Dystrophy Using SRP-9001 | ||||
Brief Summary | The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD participants by measuring biological and clinical endpoints in three parts: two 48-week randomized, double-blinded, placebo-controlled periods (Part 1 and Part 2), and an open-label follow-up period (Part 3). Participants who are randomized to placebo in Part 1 will have the opportunity for treatment with delandistrogene moxeparvovec in Part 2. In order to provide a uniform approach to monitoring long-term safety and efficacy in participants who received SRP-9001 in a clinical trial, the Sponsor has amended Study Completion for this study to occur at Week 130. Therefore, participants have transitioned and will complete the remainder of the Part 3 follow up visits in a long-term extension study, SRP-9001-305 (NCT05967351). |
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Detailed Description | Not Provided | ||||
Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 1 Phase 2 |
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Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Intervention Model Description: Parallel up to the measurement of the primary outcome at Week 48. At the beginning of Part 2, participants who were originally assigned to placebo will have the opportunity to receive delandistrogene moxeparvovec. All participants will be followed for 5 years following treatment with delandistrogene moxeparvovec. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)Primary Purpose: Treatment |
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Condition ICMJE | Muscular Dystrophy, Duchenne | ||||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Completed | ||||
Actual Enrollment ICMJE |
41 | ||||
Original Estimated Enrollment ICMJE |
24 | ||||
Actual Study Completion Date ICMJE | August 16, 2023 | ||||
Actual Primary Completion Date | December 8, 2020 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
Other inclusion/exclusion criteria apply. |
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Sex/Gender ICMJE |
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Ages ICMJE | 4 Years to 7 Years (Child) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT03769116 | ||||
Other Study ID Numbers ICMJE | SRP-9001-102 2021-000078-27 ( EudraCT Number ) |
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Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE | Not Provided | ||||
Current Responsible Party | Sarepta Therapeutics, Inc. | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor ICMJE | Sarepta Therapeutics, Inc. | ||||
Original Study Sponsor ICMJE | Same as current | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE |
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PRS Account | Sarepta Therapeutics, Inc. | ||||
Verification Date | October 2023 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |