STEROID THERAPY IN DUCHENNE MUSCULAR DYSTROPHY (DMD)

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。 预期/随机化 多机构：13个CINRG中心 研究对象：4-10岁的流动男童，确诊为DMD，不接受糖皮质激素治疗。 受试人数：140人 治疗组(每组35名患者) 4&lt；7岁，每日服用强的松 7岁，每周大剂量强的松 7岁以上至10岁儿童每日服用泼尼松 7岁以上至10岁儿童每周大剂量强的松 主端点： 力量：上肢和下肢QMT评分变化百分比。 副作用：治疗前后身高和体重/身高比变化的绝对平均数差异。 研究时间：十二(12)个28天治疗周期。(大约12个月)。 随访频率：在第1个月和第3个月访问，然后每3个月访问一次。

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. Prospective/Randomized Multi-Institution: 13 CINRG centers Subject Population: Ambulant boys aged 4-10 years with established DMD diagnosis, naive to treatment with glucocorticoids. Number of Subjects: 140 Treatment groups (35 patients per group) 4-<7 years of age daily prednisone 4-<7 years of age high-dose weekly prednisone 7+ to 10 years of age daily prednisone 7+ to 10 years of age high-dose weekly prednisone Primary Endpoints: Strength: Percent change in QMT scores of upper and lower extremities. Side-Effects: Difference in absolute means of the pre and post treatment change in height and weight/height ratio. Duration of Study: twelve (12) 28-day treatment cycles. (Approximately 12 months). Frequency of Follow-Ups: Visits at month 1 and month 3, then every 3 months.