THE MILES TRIAL

这个子项目是许多利用 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可在其他CRISP条目中表示。所列机构为 中心，但不一定是研究者所在的机构。 本试验将测试雷帕霉素西罗莫司在淋巴管平滑肌瘤病- LAM患者中的安全性和有效性。240例患者将随机分配至安慰剂组或西罗莫司组，治疗一年，然后再停用药物或安慰剂一年。 主要结果是12个月内一秒用力呼气量斜率-FEV 1斜率的变化。 分析将基于“意向治疗”设计。当100例患者达到1年时，将进行中期分析。将在24个月时进行最终分析，以评估缓解的持久性。次要结局指标将包括用力肺活量、残气量、弥散量、6分钟步行、全因死亡率和基于问卷的呼吸困难、疲劳和抑郁评估。

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 trial will test the safety and efficacy of sirolimus Rapamycin in patients with lymphangioleiomyomatosis- LAM. Two hundred and forty patients will be randomized to placebo or sirolimus groups, treated for one year and followed off of drug or placebo for one additional year. The primary outcome will be change in forced expiratory volume in one second slope-FEV1 slope over twelve months. The analysis will be based on an 'intention to treat' design. Interim analyses will occur when 100 patients reach the one year point. The final analysis, which will assess the durability of response, will occur at 24 months. Secondary outcome measures will include forced vital capacity, residual volume, diffusing capacity, six minute walk, all cause mortality and questionnaire based assessments of dyspnea, fatigue and depression.