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