Oskarsson, BjörnMaragakis, NicholasBedlack, Richard S.Goyal, NamitaMeyer, Jenny A.Genge, AngelaBodkin, CynthiaMaiser, SamuelStaff, NathanZinman, LorneOlney, NicholasTurnbull, JohnBrooks, Benjamin RixKlonowski, EmeliaMakhay, MalathYasui, SeiichiMatsuda, Kazuko2024-04-242024-04-242021Oskarsson B, Maragakis N, Bedlack RS, et al. MN-166 (ibudilast) in amyotrophic lateral sclerosis in a Phase IIb/III study: COMBAT-ALS study design. Neurodegener Dis Manag. 2021;11(6):431-443. doi:10.2217/nmt-2021-0042https://hdl.handle.net/1805/40176Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease with motor neuron loss as a defining feature. Despite significant effort, therapeutic breakthroughs have been modest. MN-166 (ibudilast) has demonstrated neuroprotective action by various mechanisms: inhibition of proinflammatory cytokines and macrophage migration inhibitory factor, phosphodiesterase inhibition, and attenuation of glial cell activation in models of ALS. Early-phase studies suggest that MN-166 may improve survival outcomes and slow disease progression in patients with ALS. This article describes the rationale and design of COMBAT-ALS, an ongoing randomized, double-blind, placebo-controlled, multicenter Phase IIb/III study in ALS. This study is designed to evaluate the pharmacokinetics, safety and tolerability and assess the efficacy of MN-166 on function, muscle strength, quality of life and survival in ALS.en-USAttribution-NonCommercial-NoDerivatives 4.0 InternationalALSAmyotrophic lateral sclerosisGlial cell attenuationIbudilastNeurodegenerative diseaseNeuroprotectionSurvivalArticle