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

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。Duchenne肌营养不良症(DMD)是最常见和最具破坏性的肌营养不良症类型。这是一项前瞻性随机研究，旨在比较强的松两种剂量方案(每天0.75 mg/kg和每周10 mg/kg，分两天服用)对患有DMD的男孩的疗效和安全性。这项研究的一个目标是比较强的松每日剂量0.75毫克/公斤和每周剂量10毫克/公斤分连续两天服用的疗效。主要的疗效测量将是肘关节和膝关节屈肌、伸肌和手柄的定量肌肉测试。二级疗效测量将包括手动肌肉测试、计时功能测试和功能评估。这项研究的第二个目标是比较两种方案的副作用，包括身高、体重、体重/身高比、体重指数、骨密度、白内障形成、血糖、血压和行为变化。

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.