Study 4045-301: Phase 3 of SRP-4045 and SRP-4053 in DMD Patients
Research type
Research Study
Full title
A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
IRAS ID
214578
Contact name
Meaghan Whalen
Contact email
Sponsor organisation
Sarepta Therapeutics, Inc.
Eudract number
2015-002069-52
Clinicaltrials.gov Identifier
Duration of Study in the UK
5 years, 0 months, 1 days
Research summary
Duchenne muscular dystrophy (DMD) is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births (around 2500 people have DMD in the UK).
Because the Duchenne gene is found on the X-chromosome, it primarily affects boys with less than 1% of those with Duchenne being female. However, it occurs across all races and cultures.
Duchenne results in progressive loss of strength and is caused by a mutation in the gene that encodes for dystrophin. Because dystrophin is absent, the muscle cells are easily damaged. The progressive muscle weakness leads to serious medical problems, particularly issues relating to the heart and lungs.
Although there are medical treatments that may help slow its progression, there is currently no cure for DMD.
Some DMD patients are missing certain parts or “exons” of the dystrophin gene so their bodies can’t make full-length dystrophin protein. For these patients with so-called deletion mutations, “skipping” over the missing area may allow the body to produce a shortened, but still working, form of the dystrophin protein. SRP-4045 is designed to skip exon 45 and SRP-4053 is designed to skip exon 53. This research study will test the effects of these drugs on muscle function in people with DMD who have deletions that may be treated by skipping exon 45 or 53.
The study will be conducted in NHS hospitals in the United Kingdom. This study will also be conducted in Belgium, France, Germany, Italy, The Netherlands and Sweden as well as in the United States of America and Canada.
REC name
London - Dulwich Research Ethics Committee
REC reference
16/LO/2002
Date of REC Opinion
23 Jan 2017
REC opinion
Further Information Favourable Opinion