MN-166（伊布地特）在肌萎缩侧索硬化症的 IIb/III 期研究中：COMBAT-ALS 研究设计。
MN-166 (ibudilast) in amyotrophic lateral sclerosis in a Phase IIb/III study: COMBAT-ALS study design.
机构信息
Department of Neurology, Mayo Clinic, Jacksonville, FL, USA.
Department of Neurology, John Hopkins University, Baltimore, MD, USA.
出版信息
Neurodegener Dis Manag. 2021 Dec;11(6):431-443. doi: 10.2217/nmt-2021-0042. Epub 2021 Nov 24.
Amyotrophic 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.
肌萎缩侧索硬化症（ALS）是一种神经退行性疾病，以运动神经元丧失为特征。尽管付出了巨大努力，但治疗上的突破仍然有限。MN-166（伊布地尔）通过多种机制显示出神经保护作用：抑制促炎细胞因子和巨噬细胞移动抑制因子、磷酸二酯酶抑制作用，以及减轻 ALS 模型中的神经胶质细胞激活。早期研究表明，MN-166 可能改善 ALS 患者的生存结局并减缓疾病进展。本文描述了 COMBAT-ALS 的原理和设计，这是一项正在进行的、随机、双盲、安慰剂对照、多中心的 ALS Ⅱb/Ⅲ期研究。该研究旨在评估 MN-166 的药代动力学、安全性和耐受性，并评估 MN-166 在 ALS 患者的功能、肌肉力量、生活质量和生存方面的疗效。
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