PILOT TRIAL OF PENTOXIFYLLINE IN DUCHENNE MUSCULAR DYSTROPHY (DMD)

己酮可可碱治疗杜氏肌营养不良症 (DMD) 的试点试验

• 批准号: 7377261
• 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/7377261
• 关键词: PILOT; TRIAL; PENTOXIFYLLINE; DUCHENNE; MUSCULAR

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. This will be an open-label pilot study of pentoxifylline in steroid-naive patients with DMD. The patient population will include boys, aged 4 to 7 years old, with an established DMD diagnosis, that have not received glucocorticosteroids within one year of their enrollment in the study. The primary endpoint is the change in QMT of 5 muscle groups (elbow flexion and extension, knee flexion and extension and grip). The secondary endpoint is the change on MMT score at 12 months compared to baseline, functional evaluation and timed function tests. Exploratory endpoints will include MRI, TGF beta levels and TNF alpha levels, which will be explored to assess baseline characteristics of MRI in DMD, MRI disease progression patterns over time and effect of therapy

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得主要资金，因此可以在其他CRISP条目中表示。所列机构为中心机构，不一定为研究者机构。这将是一项在未接受过类固醇治疗的DMD患者中进行的戊氨酰茶碱开放标签初探性研究。患者人群将包括4至7岁的男孩，具有确定的DMD诊断，在他们入组研究的一年内未接受糖皮质激素。主要终点为5个肌群（肘关节屈伸、膝关节屈伸和握力）的QMT变化。次要终点是12个月时MMT评分与基线相比的变化、功能评价和定时功能测试。探索性终点将包括MRI、TGF β水平和TNF α水平，将对其进行探索以评估DMD患者的MRI基线特征、随时间推移的MRI疾病进展模式和治疗效果