ENVISION
Research type
Research Study
Full title
A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)
IRAS ID
1004707
Contact name
Patrick O´Malley
Contact email
Sponsor organisation
Sarepta Therapeutics, Inc.
Eudract number
2020-002372-13
Clinicaltrials.gov Identifier
Research summary
Duchenne muscular dystrophy (DMD) is an X-linked degenerative neuromuscular disease caused by mutations in the dystrophin gene. Duchenne muscular dystrophy occurs in approximately 1 in 5000 males worldwide (Mendell 2012). 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 ENVISION 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 works on physical function and respiratory function in ambulatory and non-ambulatory participants. The ENVISION study will have two parts. In Part 1, half of the study participants will be given dummy drug (placebo) and half of the study participants will be given SRP-9001. In Part 2, patients will cross-over and those that received SRP-9001 in Part 1 will receive placebo and patients who received placebo in Part 1 will have the opportunity to receive SRP-9001.
This study plans on including approximately 116 study participants from over 40 study sites across Europe and North America and APAC.REC name
South Central - Oxford A Research Ethics Committee
REC reference
22/SC/0389
Date of REC Opinion
1 Mar 2023
REC opinion
Further Information Favourable Opinion