Kim Sungha, Kim Jae Kyoun, Son Mi Ju, Kim Dongwoung, Song Bongkeun, Son Ilhong, Kang Hyung Won, Lee Jongdeok, Kim Sungchul
Clinical Research Division, Korea Institute of Oriental Medicine, 1672 Yuseong-daero, Yuseong-gu, Daejeon, 34054, Republic of Korea.
Department of Global Public Health and Korean Medicine Management, Graduate School, Kyung Hee University, 26 Kyungheedae-ro, Dongdaemun-gu, Seoul, 02447, Republic of Korea.
Trials. 2018 Apr 13;19(1):225. doi: 10.1186/s13063-018-2557-z.
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that causes paralysis of limb, swallowing, and breathing muscles. Riluzole, the Food and Drug Administration-approved drug for ALS, provides minimal benefit, prolonging patient life by only 2-3 months. Previous studies have found a neuro-protective and anti-neuroinflammatory effect of Mecasin, with retrospective studies providing suggestive evidence for a beneficial effect of Mecasin. The aim of this study was to develop a protocol to determine the proper dosage of Mecasin.
This is a phase II-A, multi-center, randomized study with three arms. Thirty-six patients with ALS will be randomly assigned to one of three groups, each receiving the standard treatment with 100 mg of riluzole in addition to one of 1.6 g of Mecasin, 2.4 g of Mecasin, or a placebo. The Primary outcome is the Korean version of the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised result after 12 weeks of treatment. Secondary outcomes include results of the Short Form Health Survey-8, Medical Research Council Scale, Visual Analogue Scale for Pain, Hamilton Rating Scale for Depression, Fatigue Severity Scale, Patient Global Impression of Change, pulmonary function test, forced expiratory volume in 1 s and its ratio to forced vital capacity, creatine kinase, and body weight. The frequencies of total adverse events and serious adverse events will be described and documented. The trial protocol has been approved by the Institutional Review Board of the Wonkwang University Gwangju and Sanbon Hospital (2016-5-4 and 2016-34-01, respectively). An Investigational New Drug status (30731) was granted by the Korea Food and Drug Administration.
This trial will aim to identify the optimal dosage of Mecasin. Additionally, it will test the efficacy and safety of Mecasin in conjunction with standard treatment, riluzole, for alleviating the functional decline in patients with ALS.
Korean National Clinical Trial Registry CRIS; KCT0001984 . Registered on 28 July 2016.
肌萎缩侧索硬化症（ALS）是一种致命的神经退行性疾病，可导致肢体、吞咽和呼吸肌肉麻痹。利鲁唑是美国食品药品监督管理局批准用于治疗ALS的药物，疗效甚微，仅能将患者生命延长2至3个月。先前的研究发现了美卡西林的神经保护和抗神经炎症作用，回顾性研究为美卡西林的有益作用提供了暗示性证据。本研究的目的是制定一个方案来确定美卡西林的合适剂量。
这是一项II-A期、多中心、随机研究，分为三个组。36例ALS患者将被随机分配到三个组中的一组，每组除接受100毫克利鲁唑的标准治疗外，还分别接受1.6克美卡西林、2.4克美卡西林或安慰剂。主要结局是治疗12周后修订版肌萎缩侧索硬化症功能评定量表的韩语版结果。次要结局包括简短健康调查问卷-8、医学研究委员会量表、疼痛视觉模拟量表、汉密尔顿抑郁评定量表、疲劳严重程度量表、患者整体变化印象、肺功能测试、第1秒用力呼气量及其与用力肺活量的比值、肌酸激酶和体重的结果。将描述并记录总不良事件和严重不良事件的发生频率。该试验方案已分别获得光州圆光大学和三峰医院机构审查委员会的批准（分别为2016-5-4和2016-34-01）。韩国食品药品管理局授予了研究性新药状态（30731）。
本试验旨在确定美卡西林的最佳剂量。此外，它将测试美卡西林与标准治疗药物利鲁唑联合使用对缓解ALS患者功能衰退的疗效和安全性。
韩国国家临床试验注册中心CRIS；KCT0001984。于2016年7月28日注册。
