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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 two parts: a 48-week randomized, double-blinded, placebo-controlled period (Part 1), and a 96-week, double-blinded extension period (Part 2).
intervention: SRP-9001, Placebo
mechanism of action: Gene therapy to introduce a version of dystrophin
last updated: April 08, 2020
start date: December 22, 2018
estimated completion: October 10, 2022
phase of development: Phase 2
size / enrollment: 41
- Incidence of Serious Adverse Events (SAEs) [ Time Frame: Up to Week 144 ]
- Incidence of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Up to Week 144 ]
- Change From Baseline in Quantity of Microdystrophin Protein Expression Measured by Western Blot [ Time Frame: Baseline up to Week 12 ]
- Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score [ Time Frame: Baseline up to Week 48 ]
- Change From Baseline in Time to Rise From the Floor and Ascend 4 Steps [ Time Frame: Baseline up to Week 48 ]
- Change From Baseline in Time of 10 Meter and 100 Meter Timed Test [ Time Frame: Baseline up to Week 48 ]
• 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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