Survey about urinary symptoms and renal function in adults with DMD v1
Research type
Research Study
Full title
Survey about the prevalence of urinary symptoms and abnormal renal function in adults with Duchenne muscular dystrophy (DMD) who are on and off glucocorticoids (CSs) treatment.
IRAS ID
310078
Contact name
Ros Quinlivan
Contact email
Sponsor organisation
UCLH/UCL Joint Research Office
Duration of Study in the UK
0 years, 6 months, 1 days
Research summary
Duchenne Muscular Dystrophy (DMD) is a genetic, childhood-onset condition, which shortens life expectancy. The disease causes progressive muscle damage resulting in muscular degeneration, weakness, and wasting, associated functional delay, loss of independent walking by early teens, heart and lungs muscles failure, and premature death. In the past two decades introduction of corticosteroid (CS) treatment and non-invasive ventilation, timely management of cardiomyopathy, surgical correction of scoliosis and proactive, coordinated multidisciplinary care have increased the life expectancy. DMD has evolved from a children disease to a severe, chronic, and complex adult condition. However, during the disease advanced stage patients are at risk of developing renal dysfunction, which can be fatal.
The project aims to explore the occurrence of bladder symptoms and to assess kidney function in adults with DMD to help clinicians recognise signs and symptoms of kidney dysfunction sooner to prevent severe complications like kidney failure. We also want to explore whether CS treatment influences kidney function. The overall purpose is to learn more about the disease in adulthood to improve Standards of Care for people living with DMD.
This study is funded by the Muscular Dystrophy UK. It will include a self-reported survey and a collection of patients’ blood results and current medications related to kidney function.
A group of approximately 30 males (age 16 years and above) with confirmed diagnosis of DMD attending UCLH will be recruited. Participants will need to sign informed consent and complete the questionnaire independently. Therefore, individuals with severe behavioural or cognitive problems will not be eligible.
The recruitment will start at the beginning of 2022 for approximately 3 months. Results will be presented to clinicians, at scientific meetings and to patients via the patient support groups and published in a scientific journal.REC name
North of Scotland Research Ethics Committee 2
REC reference
22/NS/0041
Date of REC Opinion
22 Mar 2022
REC opinion
Favourable Opinion