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A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)
study id #: NCT03769116
condition: Muscular Dystrophy, Duchenne
status: active, not recruitingpurpose:
The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD patients 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). Patients who are randomized to placebo in Part 1 will have the opportunity for treatment with SRP-9001 in Part 2.
intervention: SRP-9001, Placebo
mechanism of action: Gene therapy to introduce a version of dystrophin
last updated: October 06, 2020
start date: December 22, 2018
estimated completion: December 31, 2020
phase of development: Phase 2
size / enrollment: 41
- Change From Baseline in Quantity of Micro-dystrophin Protein Expression as Measured by Western Blot [ Time Frame: Baseline up to Week 12 (Part 1) ]
- Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score [ Time Frame: Baseline up to Week 48 (Part 1) ]
- Change From Baseline in Time to Rise From the Floor [ Time Frame: Baseline up to Week 48 (Part 1) ]
- Change From Baseline in Time to Ascend 4 Steps [ Time Frame: Baseline up to Week 48 (Part 1) ]
- Change From Baseline in Time of 10 Meter Timed Test [ Time Frame: Baseline up to Week 48 (Part 1) ]
- Change From Baseline in Time of 100 Meter Timed Test [ Time Frame: Baseline up to Week 48 (Part 1) ]
- Change From Baseline in Quantity of Micro-dystrophin Expression Measured by Immunofluorescence (IF) Fiber Intensity [ Time Frame: Baseline up to Week 12 (Part 1) ]
- Change From Baseline in Quantity of Micro-dystrophin Expression Measured by IF Percent Dystrophin Positive Fibers (PDPF) [ Time Frame: Baseline up to Week 12 (Part 1) ]
• Eligible Sexes: male
• Established clinical diagnosis of DMD and documented dystrophin gene mutation of DMD phenotype.
• Indication of symptomatic muscular dystrophy by protocol-specified criteria.
• Ability to cooperate with motor assessment testing.
• Stable dose equivalent of oral corticosteroids for at least 12 weeks.
• Impaired cardiovascular function on ECHO.
• Prior or ongoing medical condition on physical examination, ECG, or laboratory findings that could adversely affect subject safety, compromise completion of follow-up, or impair assessment of study results.
• Exposure to another investigational drug or exon skipping medication within months.
• Exposure to an investigational or commercial gene therapy product.
• Abnormal liver or renal function by protocol-specified criteria
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