STEROID THERAPY IN DUCHENNE MUSCULAR DYSTROPHY (DMD)

杜氏肌营养不良症 (DMD) 的类固醇治疗

• 批准号: 7377264
• 负责人: ALAN PESTRONK
• 金额: $ 0.25万
• 依托单位: WASHINGTON UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-04-01 至 2007-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7377264
• 关键词: STEROID; THERAPY; DUCHENNE; MUSCULAR; DYSTROPHY

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.

该子项目是利用 NIH/NCRR 资助的中心拨款提供的资源的众多研究子项目之一。子项目和研究者 (PI) 可能已从另一个 NIH 来源获得主要资金，因此可以在其他 CRISP 条目中得到体现。列出的机构是中心的机构，不一定是研究者的机构。前瞻性/随机多机构：13 个 CINRG 中心 受试者人群：4-10 岁、已确诊 DMD、未接受过糖皮质激素治疗的流动男孩。 受试者数量：140 名治疗组（每组 35 名患者） 4-<7 岁，每日泼尼松 4-<7 岁，每周高剂量泼尼松 7+ 至 10 岁，每日泼尼松 7+ 至 10 岁，每周高剂量泼尼松 主要终点： 强度：上肢和下肢 QMT 评分的百分比变化。副作用：治疗前后身高和体重/身高比变化的绝对平均值差异。研究持续时间：十二 (12) 个 28 天的治疗周期。 （大约12个月）。 随访频率：第 1 个月和第 3 个月进行一次随访，然后每 3 个月进行一次。