EXPEDITION
Research type
Research Study
Full title
A Phase 3, Multinational, Long-Term Follow-Up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study
IRAS ID
1008633
Contact name
Patrick O'Malley
Contact email
Sponsor organisation
Sarepta Therapeutics, Inc.
Eudract number
2023-505043-39
Clinicaltrials.gov Identifier
Research summary
Sarepta Therapeutics, Inc. (the trial sponsor) is doing this trial to learn more about a possible treatment for Duchenne muscular dystrophy called delandistrogene moxeparvovec.
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 people with Duchenne who received treatment with delandistrogene moxeparvovec (also known as SRP-9001) in a previous clinical trial. After participants finish their previous trial, they will be invited to visit a trial site to see if they are eligible to take part in this trial. If they qualify, they will be asked to return to the trial site once every 6 months until 5 years have passed since they were treated with delandistrogene moxeparvovec in their previous clinical trial.
No additional treatments will be given in this trial. Trial doctors will monitor all new or worsening medical events that occur during the trial and any changes seen during physical exams (check-ups); any changes in vital signs, such as blood pressure and heart rate; and any changes in heart health. They will also collect blood and urine samples. Participants will be asked to perform physical activities so that trial doctors can measure their muscle strength and physical function.
The trial will be done at about 50 sites (hospitals and medical centres) in multiple countries, including the United Kingdom.
The main goal is to learn about the long-term safety of delandistrogene moxeparvovec. Other goals are to learn about possible long-term effects of delandistrogene moxeparvovec on the heart and other muscles, and on physical, respiratory, and heart function.REC name
South Central - Oxford A Research Ethics Committee
REC reference
24/SC/0015
Date of REC Opinion
2 May 2024
REC opinion
Further Information Favourable Opinion