PILOT TRIAL OF PENTOXIFYLLINE IN DUCHENNE MUSCULAR DYSTROPHY (DMD)

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。这将是一项开放标签的试点研究，将己酮茶碱用于未使用类固醇的DMD患者。患者人群将包括4至7岁的男孩，确诊为DMD，在入组研究一年内未接受糖皮质激素治疗。主要终点是5个肌肉群（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、功能评估和定时功能测试相比的变化。探索性终点将包括MRI、TGF - β水平和TNF - α水平，这些将被用于评估MRI在DMD中的基线特征、MRI疾病随时间的进展模式和治疗效果

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