Department of Neurology, University of Kansas Medical Center, 3901 Rainbow Boulevard, MS 2012, Kansas City, Kansas, 66160, USA.
The Forbes Norris MDA/ALS Research Center, California Pacific Medical Center, San Francisco, California, USA.
Muscle Nerve. 2019 Feb;59(2):201-207. doi: 10.1002/mus.26335. Epub 2018 Nov 26.
Rasagiline is a monoamine oxidase B (MAO-B) inhibitor with possible neuroprotective effects in patients with amyotrophic lateral sclerosis (ALS).
We performed a randomized, double-blind, placebo-controlled trial of 80 ALS participants with enrichment of the placebo group with historical controls (n = 177) at 10 centers in the United States. Participants were randomized in a 3:1 ratio to 2 mg/day rasagiline or placebo. The primary outcome was average slope of decline on the ALS Functional Rating Scale-Revised (ALSFRS-R). Secondary measures included slow vital capacity, survival, mitochondrial and molecular biomarkers, and adverse-event reporting.
There was no difference in the average 12-month ALSFRS-R slope between rasagiline and the mixed placebo and historical control cohorts. Rasagiline did not show signs of drug-target engagement in urine and blood biomarkers. Rasagiline was well tolerated with no serious adverse events.
Rasagiline did not alter disease progression compared with controls over 12 months of treatment. Muscle Nerve 59:201-207, 2019.
雷沙吉兰是一种单胺氧化酶 B（MAO-B）抑制剂，在肌萎缩侧索硬化症（ALS）患者中可能具有神经保护作用。
我们在美国的 10 个中心进行了一项随机、双盲、安慰剂对照试验，共有 80 名 ALS 参与者，其中安慰剂组通过历史对照进行了富集（n=177）。参与者以 3:1 的比例随机分配到 2mg/天雷沙吉兰或安慰剂组。主要结局是 ALS 功能评定量表修订版（ALSFRS-R）的平均下降斜率。次要测量指标包括肺活量、存活率、线粒体和分子生物标志物以及不良事件报告。
雷沙吉兰与混合安慰剂和历史对照队列在 12 个月的平均 ALSFRS-R 斜率上没有差异。雷沙吉兰在尿液和血液生物标志物中没有显示出药物靶点结合的迹象。雷沙吉兰耐受性良好，无严重不良事件。
与对照组相比，雷沙吉兰在 12 个月的治疗期间并未改变疾病进展。肌肉神经 59:201-207,2019。
