A MULTI-CENTER, PHASE III, RANDOMIZED, DOUBLE BLIND, PLACEBO-CONTROLLED, CLINIC

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 目前研究将检验的假设是米诺环素减缓肌萎缩侧索硬化症(ALS)患者整体功能的进行性恶化。主要的结果衡量标准是服用米诺环素的患者与服用安慰剂的患者相比，肌萎缩侧索硬化功能评定量表(ALSFRS-R)检测到的功能变化。这项研究有80%的能力来检测随着时间的推移，患者ALSFRS-R评分的平均斜率下降18%或更多。次要结果指标是手动肌肉测试(MMT)、用力肺活量(FVC，预测百分比)、生活质量(QOL)和存活率的变化。 这是一项由研究人员发起的、多中心、III期、随机(1：1)、双盲、安慰剂对照试验。研究总时间为48个月：24个月的患者招募，4个月的连续月度评估，以确定每个患者的基线进展斜率，然后是9个月的干预(米诺环素或安慰剂)，以及另外11个月的生存跟踪、数据分析和准备出版物。受试者在13个月的参与期间每月接受评估。随机化将在第4个月的访问中进行。在干预阶段的前3周(第5个月)，受试者在耐受性范围内每天接受最高8片(400毫克)的递增剂量，并每周进行电话联系。

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. The hypothesis that the current study will test is that minocycline slows the progressive deterioration of global function in patients with amyotrophic lateral sclerosis (ALS). The primary outcome measure is change in function as detected by the ALS Functional Rating Scale (ALSFRS-R) in patients taking minocycline compared to those taking placebo. The study is 80 % powered to detect an 18% or greater reduction in the average slope of patients' ALSFRS-R scores over time. The secondary outcome measures are changes in manual muscle testing (MMT), forced vital capacity (FVC, percent predicted), quality of life (QOL) and survival. This is an investigator-initiated, multi-center, phase III, randomized (1:1), double blind, placebo-controlled trial. The total study length is 48 months: Twenty-four months for patient recruitment, 4 months of serial monthly evaluations to determine baseline slopes of progression for each patient followed by 9 months of intervention (minocycline or placebo), and 11 additional months of survival follow-up, data analysis and preparation of publications. Subjects receive monthly evaluations during their 13 months of participation. Randomization will occur at the month 4 visit. During the first 3 weeks of the intervention phase (month 5) subjects receive an escalating dose of up to 8 pills (400 mg) per day as tolerated and have weekly phone contact.