EMBARK
Research type
Research Study
Full title
A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK)
IRAS ID
1003556
Contact name
Patrick O´Malley
Contact email
Sponsor organisation
Sarepta Therapeutics, Inc.
Eudract number
2019-003374-91
Clinicaltrials.gov Identifier
Research summary
Duchenne muscular dystrophy (DMD) is an X-linked degenerative neuromuscular disease caused by mutations in the dystrophin gene. DMD occurs in approximately 1 in 5000 males worldwide. The mutations that cause DMD typically disrupt the dystrophin messenger ribonucleic acid reading frame and prevent production of the corresponding protein. Duchenne muscular dystrophy is usually diagnosed between the ages of 3 to 5 years.
To date, there are limited treatment options for patients suffering from DMD, none of which reverse the course of this debilitating and ultimately fatal disease. Management of DMD requires a multidisciplinary approach that includes both preventive and therapeutic measures
SRP-9001 is a gene therapy designed to treat the underlying biological cause of DMD by replacing dysfunctional or missing dystrophin protein with a functional truncated dystrophin, called micro-dystrophin, in cardiac and skeletal muscle; the key tissues affected in this lethal degenerative disease. Thus, micro-dystrophin may address the root cause of DMD, alter the course of the disease, and address a significant unmet medical need.
The purpose of EMBARK study is to continue checking the safety of SRP-9001 and how well it works. This study will also help us understand how SRP-9001 acts in your body and how your body reacts to it. In EMBARK, half of the study participants will be given dummy drug (placebo) and half of the study participants will be given SRP-9001.
This study study plans on including approximately 120 study participants from over 43 study sites across Europe and North America and APAC.REC name
South Central - Oxford A Research Ethics Committee
REC reference
21/SC/0352
Date of REC Opinion
10 May 2022
REC opinion
Further Information Favourable Opinion