Rasagiline for amyotrophic lateral sclerosis: A randomized, controlled trial

Muscle Nerve. 2019 Feb;59(2):201-207. doi: 10.1002/mus.26335. Epub 2018 Nov 26.


Introduction: Rasagiline is a monoamine oxidase B (MAO-B) inhibitor with possible neuroprotective effects in patients with amyotrophic lateral sclerosis (ALS).

Methods: 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.

Results: 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.

Discussion: Rasagiline did not alter disease progression compared with controls over 12 months of treatment. Muscle Nerve 59:201-207, 2019.

Keywords: MAO-B inhibitor; amyotrophic lateral sclerosis; biomarker; controlled clinical trial; motor neuron disease; randomized; rasagiline.

Publication types

  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, N.I.H., Extramural

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Amyotrophic Lateral Sclerosis / drug therapy*
  • Amyotrophic Lateral Sclerosis / psychology
  • DNA-Binding Proteins / metabolism
  • Double-Blind Method
  • Female
  • Humans
  • Indans / therapeutic use*
  • Male
  • Middle Aged
  • Neuroprotective Agents / therapeutic use*
  • Outcome Assessment, Health Care
  • Quality of Life
  • Retrospective Studies
  • Severity of Illness Index
  • Treatment Outcome
  • United States
  • Young Adult


  • DNA-Binding Proteins
  • Indans
  • Neuroprotective Agents
  • TARDBP protein, human
  • rasagiline