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 recruiting

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

results: https://clinicaltrials.gov/ct2/show/results/NCT03769116

last updated: October 06, 2020

start date: December 22, 2018

estimated completion: December 31, 2020

phase of development: Phase 2

size / enrollment: 41

primary outcomes:

• 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) ]

secondary outcomes:

• 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) ]

inclusion criteria:

exclusion criteria:
• 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

sponsor: Sarepta Therapeutics, Inc.

• United States, California
  David Geffen School of Medicine at UCLA
  
  
• United States, Ohio
  Nationwide Children’s Hospital