A 40-week phase 2B randomized, multicenter, double-blind, placebo-controlled study evaluating the safety and efficacy of memantine in amyotrophic lateral sclerosis.

Autor: Bhai S; University of Texas Southwestern Medical Center, Dallas, Texas, USA.; Neuromuscular Center, Institute for Exercise and Environmental Medicine, Texas Health Dallas, Dallas, Texas, USA., Levine T; Honor Health, Scottsdale, Arizona, USA., Moore D; Calico Consulting, Livermore, California, USA., Bowser R; Barrow Neurological Institute, Phoenix, Arizona, USA., Heim AJ; University of Kansas Medical Center, Kansas City, Kansas, USA., Walsh M; University of Kansas Medical Center, Kansas City, Kansas, USA., Shibani A; Nerve and Muscle Center of Texas, Houston, Texas, USA., Simmons Z; Penn State Hershey Medical Center, Hershey, Pennsylvania, USA., Grogan J; Penn State Hershey Medical Center, Hershey, Pennsylvania, USA., Goyal NA; University of California, Irvine, California, USA., Govindarajan R; University of Missouri, Columbia, Missouri, USA., Hussain Y; Austin Neuromuscular Center, Austin, Texas, USA.; University of Texas Dell Medical School, Austin, Texas, USA., Papsdorf T; Access TeleCare, Dallas, Texas, USA., Schwasinger-Schmidt T; University of Kansas School of Medicine-Wichita, Wichita, Kansas, USA., Olney N; Providence Brain and Spine Institute, Portland, Oregon, USA., Goslin K; Providence Brain and Spine Institute, Portland, Oregon, USA., Pulley M; University of Florida College of Medicine Jacksonville, Jacksonville, Florida, USA., Kasarskis E; University of Kentucky, Lexington, Kentucky, USA., Weiss M; University of Washington, Seattle, Washington, USA., Katz SW; Syneos Health, Morrisville, North Carolina, USA., Moser S; University of Missouri, Columbia, Missouri, USA., Jabari D; Cedars-Sinai Medical Center, Los Angeles, California, USA., Jawdat O; University of Kansas Medical Center, Kansas City, Kansas, USA., Statland J; University of Kansas Medical Center, Kansas City, Kansas, USA., Dimachkie MM; University of Kansas Medical Center, Kansas City, Kansas, USA., Barohn R; University of Missouri, Columbia, Missouri, USA.
Jazyk: angličtina
Zdroj: Muscle & nerve [Muscle Nerve] 2025 Jan; Vol. 71 (1), pp. 63-72. Date of Electronic Publication: 2024 Nov 07.
DOI: 10.1002/mus.28287
Abstrakt: Introduction: Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disease with no known cure, limited treatment options with minimal benefits, and significant unmet need for disease modifying therapies.
Aims: This study investigated memantine's impact on ALS progression, with an additional focus on the effects of memantine on cognitive and behavioral changes associated with the disease.
Methods: A randomized, double-blind, placebo-controlled clinical trial was conducted from December 2018 to September 2020. ALS patients were enrolled in-person and remotely across 13 sites in the United States. Participants were randomized to memantine (20 mg twice daily) or placebo in a 2:1 ratio and completed 36 weeks of treatment. The primary outcome of disease progression was assessed by the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), and blood was collected for biomarker analysis.
Results: Of the 99 participants enrolled in the study, 89 were randomized to memantine or placebo (ages 24-83 years, male-to-female ratio ~3:2). Fifty-two participants completed the study treatment with no significant differences in disease progression, biomarker changes (including neurofilament light chain [NfL]), or neuropsychiatric testing noted between the groups. Initial NfL values correlated with the rate of ALSFRS-R decline.
Discussion: In this study, memantine did not impact ALS disease progression or neuropsychiatric symptoms. Trials with remote enrollment may help trial participation and success.
(© 2024 The Author(s). Muscle & Nerve published by Wiley Periodicals LLC.)
Databáze: MEDLINE
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