Safety Extension Study of Edaravone Oral Suspension in Patients With Amyotrophic Lateral Sclerosis for up to an Additional 96 Weeks of Treatment.

IF 3.1 3区医学 Q2 CLINICAL NEUROLOGY

Muscle & Nerve Pub Date : 2025-09-01 Epub Date: 2025-06-09 DOI:10.1002/mus.28451

Angela Genge, Gary L Pattee, Gen Sobue, Masashi Aoki, Hiide Yoshino, Philippe Couratier, Christian Lunetta, Susanne Petri, Daniel Selness, Vesna Todorovic, Nissim Sasson, Manabu Hirai, Fumihiro Takahashi, Alejandro Salah, Stephen Apple, Art Wamil, Alexander Kalin, Carlayne E Jackson

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引用次数: 0

Abstract

Introduction/aims: Edaravone intravenous (IV) and oral suspension have been shown to have similar pharmacokinetics, safety, and slowing of functional decline in patients with amyotrophic lateral sclerosis (ALS). Study MT-1186-A01 indicated that edaravone oral suspension was well-tolerated over 48 weeks, with no new safety concerns identified relative to existing safety data of IV edaravone, including Study MCI186-19. The aim of this study was to assess the long-term safety and tolerability of edaravone oral suspension in patients with ALS.

Methods: Study MT-1186-A03 (NCT04577404) was a phase 3, open-label, multi-center, extension study that evaluated the long-term safety of edaravone oral suspension over an additional 96 weeks in patients with ALS who have completed the initial 48 weeks of Study MT-1186-A01, for a total of up to 144 weeks of treatment. Patients received a 105-mg dose of edaravone administered in treatment cycles identical to the approved edaravone on/off dosing schedule. Patients had definite, probable, probable-laboratory-supported, or possible ALS.

Results: In Study MT-1186-A03, edaravone oral suspension was well tolerated with no new safety concerns. The most common treatment-emergent adverse events (TEAEs) were fall, muscular weakness, dyspnea, constipation, and dysphagia. These TEAEs were consistent with the safety profile for edaravone from previous clinical trials.

Discussion: These results help establish the long-term safety and tolerability profile of edaravone oral suspension.

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依达拉奉口服悬液在肌萎缩性侧索硬化症患者治疗96周的安全性扩展研究

介绍/目的：依达拉奉静脉（IV）和口服悬液在肌萎缩性侧索硬化症（ALS）患者中具有相似的药代动力学、安全性和减缓功能衰退的能力。研究MT-1186-A01表明，在48周内，依达拉奉口服混悬液耐受性良好，与现有IV依达拉奉安全性数据（包括研究MCI186-19）相比，未发现新的安全性问题。本研究的目的是评估依达拉奉口服混悬液在ALS患者中的长期安全性和耐受性。研究MT-1186-A03 （NCT04577404）是一项开放标签、多中心、扩展的3期研究，在完成研究MT-1186-A01最初48周治疗的ALS患者中，评估依达拉奉口服悬剂额外96周的长期安全性，总共长达144周的治疗。患者接受105毫克剂量的依达拉奉，治疗周期与批准的依达拉奉开/关给药计划相同。患者有明确的、可能的、可能的实验室支持的或可能的ALS。结果：在研究MT-1186-A03中，依达拉奉口服混悬液耐受性良好，无新的安全性问题。最常见的治疗不良事件（teae）是跌倒、肌肉无力、呼吸困难、便秘和吞咽困难。这些teae与先前临床试验中依达拉奉的安全性一致。讨论：这些结果有助于建立依达拉奉口服混悬液的长期安全性和耐受性。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Muscle & Nerve 医学-临床神经学

CiteScore

6.40

自引率

5.90%

发文量

287

审稿时长

3-6 weeks

期刊介绍： Muscle & Nerve is an international and interdisciplinary publication of original contributions, in both health and disease, concerning studies of the muscle, the neuromuscular junction, the peripheral motor, sensory and autonomic neurons, and the central nervous system where the behavior of the peripheral nervous system is clarified. Appearing monthly, Muscle & Nerve publishes clinical studies and clinically relevant research reports in the fields of anatomy, biochemistry, cell biology, electrophysiology and electrodiagnosis, epidemiology, genetics, immunology, pathology, pharmacology, physiology, toxicology, and virology. The Journal welcomes articles and reports on basic clinical electrophysiology and electrodiagnosis. We expedite some papers dealing with timely topics to keep up with the fast-moving pace of science, based on the referees'' recommendation.