A Natural History Study in Patients with Duchenne Muscular Dystrophy
Research type
Research Study
Full title
A Natural History Study in Patients with Duchenne Muscular Dystrophy Managed in Routine Clinical Practice.
IRAS ID
260256
Contact name
Rosaline Quinlivan
Contact email
Sponsor organisation
Sarepta Therapeutics, Inc
Duration of Study in the UK
9 years, 7 months, 26 days
Research summary
Duchene Muscular Dystrophy (DMD) is a genetic condition that primarily affects boys, causing symptoms such as progressive muscle weakness.
Diagnosis is usually made between the ages of 3-5 years old where toddlers begin to show mobility problems. By adolescence, boys will mostly become unable to walk and will require a wheelchair. Heart and respiratory muscles will also be affected.
Unfortunately, untreated patients die from respiratory or heart failure in their late teens/early twenties
There is no cure for this condition, but steroids and non-invasive ventilation can ease symptoms and prolong life expectancy.
This study is an observational study where data will be collected every 6 months from patients receiving their normal standard treatment from their doctor. No medication or extra procedures will be performed.
The information collected will be used to look at new ways of treating DMD.
The data of interest will look at how patients are managed and followed in their routine care along with routine assessments.
The routine assessment information collected will be: DMD diagnosis, medical history,heart, kidney and liver function, lung function, physical function, quality of life assessments,current medications,side effects and discomforts, height, weight and age as well as records of visits to hospital or clinics.
The study is sponsored by Sarepta Therapeutics Inc.
Approximately 300 patients will participate in this study worldwide, with 25 in the UK.REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
19/WM/0359
Date of REC Opinion
18 Dec 2019
REC opinion
Further Information Favourable Opinion