CNMC0601: A RANDOMIZED STUDY OF DAILY VS HIGH-DOSE WEEKLY PREDNISONE IN DUCHEN

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Duchenne muscular dystrophy (DMD) is the most common and devastating type of muscular dystrophy. This is a prospective, randomized study to compare efficacy and safety of two dose schedules of prednisone (.75 mg/kg/day given daily and 10mg/kg/week given in equal doses over two days) in boys with DMD. One objective of this study to compare the efficacy of a daily prednisone dose of .75mg/kg with a weekly dose of 10mg/kg split over two consecutive days. The primary efficacy measure will be quantitative muscle testing of elbow and knee flexors and extensors and handgrip. Secondary efficacy measures will include manual muscle testing, timed function tests and functional evaluation. The second objective of this study is to compare side effect profiles between the two regimens, to include height, weight, weight/height ratio, body mass index, bone density, cataract formation, blood glucose, blood pressure and behavioral changes.