Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (IGNITE DMD)
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ClinicalTrials.gov Identifier: NCT03368742 |
Recruitment Status :
Recruiting
First Posted : December 11, 2017
Last Update Posted : February 17, 2021
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Tracking Information | |||||
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First Submitted Date ICMJE | December 5, 2017 | ||||
First Posted Date ICMJE | December 11, 2017 | ||||
Last Update Posted Date | February 17, 2021 | ||||
Actual Study Start Date ICMJE | December 6, 2017 | ||||
Estimated Primary Completion Date | April 2022 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE | Same as current | ||||
Change History | |||||
Current Secondary Outcome Measures ICMJE | Not Provided | ||||
Original Secondary Outcome Measures ICMJE | Not Provided | ||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Microdystrophin Gene Transfer Study in Adolescents and Children With DMD | ||||
Official Title ICMJE | A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy | ||||
Brief Summary | This is a controlled, open-label, single-ascending dose study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Patients will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years. The protocol was amended to drop the control arm after 4 subjects were dosed. Subjects currently enrolling are assigned to active treatment. Control subjects enrolled under original protocol will continue through the study per the original protocol. |
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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: Non-Randomized Intervention Model: Single Group Assignment Intervention Model Description: Active treatment group for all patients enrolled after June 2019. Total of approximately 16 to 32 patients. Masking: None (Open Label)Primary Purpose: Treatment |
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Condition ICMJE | Duchenne Muscular Dystrophy | ||||
Intervention ICMJE | Genetic: SGT-001
AAV9 vector containing muscle-specific promoter and microdystrophin construct
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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 | Recruiting | ||||
Estimated Enrollment ICMJE |
16 | ||||
Original Estimated Enrollment ICMJE | Same as current | ||||
Estimated Study Completion Date ICMJE | April 2027 | ||||
Estimated Primary Completion Date | April 2022 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
Additional inclusion/exclusion criteria may apply. Patients over 30 kg will not be eligible for treatment at this time. A weight limit of ≤ 18 kg will be implemented for the next two patients to be dosed. |
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Sex/Gender ICMJE |
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Ages ICMJE | 4 Years to 17 Years (Child) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | |||||
Listed Location Countries ICMJE | United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT03368742 | ||||
Other Study ID Numbers ICMJE | GX1001 | ||||
Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Responsible Party | Solid Biosciences, LLC | ||||
Study Sponsor ICMJE | Solid Biosciences, LLC | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE |
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PRS Account | Solid Biosciences, LLC | ||||
Verification Date | October 2020 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |