EMERGENE
Research type
Research Study
Full title
A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects with Limb Girdle Muscular Dystrophy 2E/R4
IRAS ID
1009294
Contact name
Sandra Rivas
Contact email
Sponsor organisation
Sarepta Therapeutics, Inc.
Research summary
The limb-girdle muscular dystrophies (LGMDs) are a group of rare, genetic disorders involving progressive weakness and wasting of the shoulder and pelvic girdle muscles. Currently, there is no cure available for the treatment of LGMD. Current palliative management is only focused on symptomatic and supportive treatments. Each type of LGMD is named after the specific gene and protein which are affected. This study is testing a new medication for LGMD2E/R4.
The sponsor is studying SRP-9003 (the ‘study drug’) for the treatment of LGMD2E. The purpose of this research study to find out if it is safe to add a new, functional gene to the body. This can be done by using a method called “gene transfer.” Doctors will transfer a version of the gene that is made in the laboratory and put it into the participant`s body.
At the screening visit, the participant will be given an activity monitor device to wear which continuously records their movements during everyday activities. The goal of the wearable device is to measure their daily movement and activity levels.
In parallel with the clinical research study involving the study drug, one of the blood tests is also under investigation. This is considered an investigational device study and is being sponsored by Quest Diagnostics, Inc. We call this test the “In Vitro Device Study” or IVD Study.
Globally this study will include approximately 15 participants, minimum of 6 paediatric (minimum 4 years old), from 12-14 research sites in total.
The involvement of each participant will last approximately 5 and a half years, including a 31-day Screening Period as well as a 6-month Lead-In Period and a second Screening period prior to receiving the study drug.REC name
North of Scotland Research Ethics Committee 1
REC reference
24/NS/0004
Date of REC Opinion
13 Feb 2024
REC opinion
Further Information Favourable Opinion