MCID DMD study
Research type
Research Study
Full title
Minimal Clinically Important Difference (MCID) in Duchenne Muscular Dystrophy (DMD) outcome measures
IRAS ID
232121
Contact name
Francesco Muntoni
Contact email
Sponsor organisation
University College London
Duration of Study in the UK
1 years, 6 months, 27 days
Research summary
Duchenne Muscular Dystrophy (DMD) is the most common form of muscular disease in childhood, affecting 1 in 5000 live male births. It classically presents in the first decade of life with proximal muscle weakness that is progressive and leads to loss of independent ambulation between 6 and 16 years old. \nIn recent years, there have been promising advances for new potential treatments for DMD with an increasing number of clinical trials. Several outcome measures have been used in those studies. It is essential to have a good understanding of the natural history of the disease evaluated with these measures as the design and interpretation of clinical trials will intimately depend on it.\nAlthough clinical trials may report statistically significant differences in outcome measures, these may not translate into clinically relevant or meaningful differences for patients, their families and clinicians. On the other hand, the changes found in clinical trials might be statistically significant but not represent the minimal difference. \nThis study aims to determine the minimal clinically important difference (MCID) for two commonly used clinical trial outcome measures in DMD: the North Star Ambulatory Assessment (NSAA) and the Performance of Upper Limb (PUL). The NSAA is focused on ambulant patients and the PUL is particularly useful for non-ambulant patients. \nThe MCID can be defined as the smallest change in an outcome measure that a patient/parent/clinician would identify as meaningful. \nA qualitative questionnaire exploring patients’, their families’ and clinician’s views on the MCID for these scales and currently used quality of life measures will be undertaken.\nWe hope to determine the MCID for both NSAA and PUL and consequently aid in the design and interpretation of the results of current and future trials. The results will also be of increasing relevance to regulatory authorities, advocacy groups, families and patients.\n
REC name
South West - Cornwall & Plymouth Research Ethics Committee
REC reference
17/SW/0213
Date of REC Opinion
1 Nov 2017
REC opinion
Further Information Favourable Opinion