Changes in muscle mass in DMD using the D3-creatine dilution method
Research type
Research Study
Full title
Changes in muscle mass using the D3-creatine dilution method and function in DMD
IRAS ID
297878
Contact name
Michela Guglieri
Contact email
Sponsor organisation
University of California, Berkeley
Duration of Study in the UK
3 years, 0 months, 0 days
Research summary
Duchenne Muscular Dystrophy (DMD) is a severe disease of the muscles caused by a mutation in the gene responsible for producing a protein called dystrophin. This leads to muscle degeneration over time.
This study will use a new and non-invasive method to determine the muscle mass in patients with Duchenne's Muscular Dystrophy. This will be done by the participant drinking a small amount of D3 Creatine. By collecting urine samples from the participant, we can then measure the amount of D3 creatine in the urine, which can then be used to measure changes in muscle mass. Participants will attend 3 study visits over the course of a year that will coincide with their regular clinic visits. Boys with DMD over the age of 5 will be recruited to participate in the study.
The study will be conducted in the UK and USA. Approximately 30 patients between the ages of 5 and 25 will be recruited in the UK. The duration for each participant will be 12 months.
Participants will be assessed at on-site clinical visits at baseline, 6 months and 12 months.
REC name
East Midlands - Nottingham 1 Research Ethics Committee
REC reference
21/EM/0194
Date of REC Opinion
14 Oct 2021
REC opinion
Further Information Favourable Opinion