A study of vesleteplirsen (SRP-5051) in ambulatory males with DMD amenable to exon 51 skipping
Research type
Research Study
Full title
A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study with a Treatment and Observation Period to Evaluate the Safety and Efficacy of Vesleteplirsen (SRP-5051) in Ambulatory Subjects with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Treatment (MOTIVATE)
IRAS ID
1008258
Contact name
Lorrie Lucht
Contact email
Sponsor organisation
Sarepta Therapeutics Inc.
ISRCTN Number
Not applicable
Clinicaltrials.gov Identifier
Pending
Research summary
Sarepta Therapeutics, Inc. (the trial sponsor) is doing this trial to learn more about a possible treatment for Duchenne muscular dystrophy called vesleteplirsen.
People with Duchenne have a genetic mutation that limits their ability to make a protein called dystrophin. Lack of dystrophin causes muscle weakness that gets worse with time. Duchenne can be life-threatening and typically leads to premature death.
This trial will include boys and young men (6 to 15 years old) with Duchenne caused by certain genetic mutations.
The trial will be done in 2 parts. In Part 1, about 2/3 of the participants will receive vesleteplirsen. Others will receive a placebo (an identical treatment with no active study drug). A computer program will randomly assign the treatment each participant will receive. During this part, none of the participants, doctors, or other staff will know which treatment each participant is receiving. Participants will be in Part 1 for 76 weeks.In Part 2, all participants will receive vesleteplirsen for 80 weeks.
Treatments will be given intravenously (through a vein) once every 4 weeks.
The trial will be done at about 85 sites (hospitals and medical centers) in multiple countries, including the United Kingdom. Participants will visit a trial site about 85 times during the trial. They will be asked to perform physical activities, which will let doctors measure their muscle strength and physical function. Some participants will be asked to provide small samples of muscle tissue to measure levels of dystrophin production.
The main goal is to learn whether participants who receive vesleteplirsen in Part 1 have better outcomes than those who receive a placebo. Another goal is to learn more about possible side effects of vesleteplirsen.
REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
23/WM/0187
Date of REC Opinion
1 Nov 2023
REC opinion
Further Information Favourable Opinion