THE MILES TRIAL

里程试验

• 批准号: 7950735
• 负责人: MARK Louis BRANTLY
• 金额: $ 0.28万
• 依托单位: UNIVERSITY OF FLORIDA
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-12-01 至 2009-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7950735
• 关键词: Clinical Research; Computer Retrieval of Information on Scientific Projects Database; Diffuse; Dyspnea; Fatigue; Forced expiratory volume function; Funding; Grant; Institution; Intention; Lymphangioleiomyomatosis; Outcome Measure; Patients; Pharmaceutical Preparations; Placebos; Questionnaires; Randomized; Research; Research Personnel; Residual volume; Resources; Sirolimus; Source; United States National Institutes of Health; Vital capacity; Walking; base; depression; design; mortality; primary outcome; response; safety testing; secondary outcome

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.

该副本是利用众多研究子项目之一 由NIH/NCRR资助的中心赠款提供的资源。子弹和 调查员（PI）可能已经从其他NIH来源获得了主要资金， 因此可以在其他清晰的条目中代表。列出的机构是 对于中心，这不一定是调查员的机构。 该试验将测试西罗木霉素对淋巴血管瘤瘤病患者的安全性和功效。 240名患者将被随机分为安慰剂或西罗莫司组，接受了一年的治疗，然后再服用药物或安慰剂一年。 主要结果将是在十二个月内在一秒钟的斜率 - 频率-FEV1坡度内变化。 该分析将基于“治疗”设计的意图。当100名患者达到一年时，将进行临时分析。最终分析将在24个月内评估响应的持久性。次要结果指标将包括强迫生命力，剩余体积，散布能力，步行六分钟，所有这些都导致死亡率和基于问卷调查的呼吸困难，疲劳和抑郁症的评估。