Rasagiline for amyotrophic lateral sclerosis: A randomized, controlled trial.

Abstract

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.

• Authors: Jeffrey M Statland, Dan Moore, Yunxia Wang, Maureen Walsh, Tahseen Mozaffar, Lauren Elman, Sharon P Nations, Hiroshi Mitsumoto, J Americo Fernandes, David Saperstein, Ghazala Hayat, Laura Herbelin, Chafic Karam, Jonathan Katz, Heather M Wilkins, Abdulbaki Agbas, Russell H Swerdlow, Regina M Santella, Mazen M Dimachkie, Richard J Barohn, Rasagiline Investigators of the Muscle Study Group and Western ALS Consortium
• Journal: Muscle & nerve (Muscle Nerve) Vol. 59 Issue 2 Pg. 201-207 (02 2019) ISSN: 1097-4598 [Electronic] United States
• PMID: 30192007 (Publication Type: Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, N.I.H., Extramural)
• Copyright: © 2018 Wiley Periodicals, Inc.

Chemical References

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

Topics

• Adult
• Aged
• Aged, 80 and over
• Amyotrophic Lateral Sclerosis (drug therapy, 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