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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
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: December 04, 2019
start date: December 22, 2018
estimated completion: October 10, 2022
phase of development: Phase 2
size / enrollment: 40
- Incidence of Serious Adverse Events (SAEs) [ Time Frame: Up to 144 weeks ]
- Incidence of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Up to 144 weeks ]
- Incidence of Adverse Events of Special Interest (AESIs) [ Time Frame: Up to 144 weeks ]
- 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 ]
• 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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