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Duchenne Muscular Dystrophy: Double-Blind Randomized Trial to Find The Optimum Steroid Regimen (FOR-DMD)
This Phase III multi-center, double-blind, parallel group, 36 - 60 month study funded by the NIH will compare three corticosteroid regimens in wide use in DMD: 1) daily prednisone (0.75 mg/kg/day); 2) intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off); and 3) daily deflazacort (0.9 mg/kg/day). The randomized controlled trial will 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). Male participants must be >4 - <8 years of age with a genetically confirmed diagnosis of DMD and have never taken oral steroids. Participants will receive study medication for a minimum of three years and a maximum of five years (depending on how early the participant was recruited into the study). Participation involves visits to the study hospital every three months for the first six months and every six months thereafter. Travel funds provided by the MDA are available.