A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)

study id #: NCT03769116

condition: Muscular Dystrophy, Duchenne

status: 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 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

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

last updated: December 04, 2019

start date: December 22, 2018

estimated completion: October 10, 2022

phase of development: Phase 2

size / enrollment: 40

primary outcomes:

• 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 ]

secondary outcomes:

• 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 ]

inclusion criteria:
• 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.

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

contacts: 1-888-727-3782, clinicaltrials@sarepta.com

• United States, California
  David Geffen School of Medicine at UCLA
  Howard Mao, 310-825-3264, hcmao@mednet.ucla.edu
  
  
• United States, Ohio
  Nationwide Children’s Hospital
  Jordan Vaiea, 614-722-2650, Jordan.Vaiea@nationwidechildrens.org