CLINICAL TRIAL: DAILY VS WEEKLY PREDNISONE THERAPY IN DUCHENNE MUSCULAR DYSTROP

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 该方案的目的是进行一项前瞻性随机研究，以比较强的松两种剂量方案(每日0.75 mg/kg，每周10 mg/kg，连续两天)治疗DMD的疗效和安全性。 目的1：比较强的松每日剂量0.75 mg/kg和每周剂量10 mg/kg分两天给药的疗效。主要的疗效衡量标准将是上肢和上肢的定量肌肉测试(QMT)分数 四肢，由成对的屈/伸肌群组成。次要疗效测量将包括肘、膝屈伸肌和手握力的个人QMT评分、手动肌肉测试、计时功能测试、肺功能测试和功能评估。 假设：间歇性给予大剂量强的松(每日2倍剂量)在延缓DMD患者肌肉无力的进展方面与每日剂量一样有效。 目的：比较两种方案的不良反应情况，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、血压和行为改变。 假设：间歇性给予大剂量强的松(每日2次剂量)与每日剂量治疗相比，毒性较小，因此副作用较少。 CINRG临床评估仪可靠性培训 目的：这项培训是为了确保临床评估员对多中心CINRG组进行手动肌肉测试(MMT)、QMT和功能测试的评定者之间的可靠性。 具体目标和假设：对于DMD受试者的MMT、QMT和功能测试，德克萨斯儿童医院CINRG站点的临床评估者(相对于这个多中心研究小组的“金标准”临床评估者)的评价者之间的可靠性是什么？这是当前随机研究的基础。

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. The purpose of this protocol is to conduct a prospective, randomized study to compare efficacy and safety of two dose schedules of prednisone (0.75 mg/kg/day given daily and 10 mg/kg/week given in equal doses over two days) in boys with DMD. AIM 1: To compare efficacy of a daily prednisone dose of .75mg/kg with a weekly dose of 10 mg/kg split over two consecutive days. The primary efficacy measures will be quantitative muscle testing (QMT) scores of the upper and lower extremities, consisting of paired flexor/extensor groups. Secondary efficacy measures will include individual QMT scores of elbow and knee flexors and extensors and hand grip, manual muscle testing, timed function tests, pulmonary function tests and functional evaluation. Hypothesis: High dose prednisone (2X daily dose) given intermittently will be as effective as daily dose in slowing down the progress of muscle weakness in DMD. AIM 2: 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. Hypothesis: High dose prednisone (2X daily dose) given intermittently will be less toxic and hence cause fewer side effects than daily dose therapy in DMD. CINRG Clinical Evaluator Reliability Training Objective: This training is being conducted to ensure inter-rater reliability of clinical evaluators performing manual muscle testing (MMT), QMT, and functional tests for the multicenter CINRG group. Specific Aims and Hypothesis: What is the inter-rater reliability of clinical evaluators at the Texas Children's Hospital CINRG site (relative to the "gold standard" clinical evaluators of this multicenter research group) for MMT, QMT, and functional testing of DMD subjects?base for the current randomized study.