Newcastle Clinical Trials Unit



Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid Regimen

Study Stage: Closed
Sponsor: University of Rochester (NY, USA)
Funder: NIH
Therapeutic area: Musculoskeletal
Type of study: CTIMP

Aim: To establish the optimum corticosteroid regimen, of three under investigation, to address the pragmatic hypothesis that daily corticosteroids (prednisone or deflazacort) will be of greater benefit in terms of function and subject/parent satisfaction than intermittent corticosteroids (prednisone).

Primary outcome:  The primary outcome variable is a three-dimensional (multivariate) outcome consisting of the following three components (each averaged over all post-baseline follow-up visits through Month 36):

(1) time to stand from lying (log-transformed)
(2) forced vital capacity
(3) subject/parent global satisfaction with treatment, as measured by the Treatment Satisfaction Questionnaire for Medication

Population: Paediatric
Clinical Phase: III
Design: RCT
Setting: Secondary Care
Planned Sample Size: 225