1 Department of Neurosurgery, Faculty of Medicine and Graduate School of Medicine, Hokkaido University, Japan.
2 International University of Health and Welfare, Center for Brain and Cerebral Vessels, Sanno Hospital and Sanno Medical Center, Japan.
Int J Stroke. 2018 Jun;13(4):444-448. doi: 10.1177/1747493017743057. Epub 2017 Nov 14.
Rationale MultiStem® (HLM051) is one of the promising allogenic cell products for acute ischemic stroke with strong evidence. A previous phase 2 randomized, double-blind, placebo-controlled, multicenter dose-escalation trial showed the safety of MultiStem® for acute ischemic stroke, with a time window beyond that of rt-PA and endovascular thrombectomy. We aim to obtain stronger evidence and to show the efficacy of the MultiStem® for treatment of ischemic stroke. Sample size Estimated sample size is 220 (110 patients per group), which has 90% power at 5% significance level. Methods and design TREASURE is a randomized, double-blind, placebo-controlled, multicenter phase 2/3 trial. The trial will be done at 31 medical centers in Japan. Patients with acute ischemic stroke including motor or speech deficit defined by a National Institution of Health Stroke Scale (NIHSS) score of 8-20 at baseline will be randomized 1:1 to receive a single intravenous infusion of MultiStem® or placebo within 18-36 h of stroke onset. Study outcomes Primary outcome in this study is the proportion of patients with an excellent outcome at day 90 defined by the functional assessment. Trial registration ClinicalTrials.gov (NCT02961504). Conclusion The TREASURE trial will provide a novel treatment option and expand the therapeutic window for patients with stroke if the results are positive.
多能干细胞（HLM051）是一种有前景的同种异体细胞产品，用于急性缺血性脑卒中，有充分的证据支持。先前的 2 期随机、双盲、安慰剂对照、多中心剂量递增试验表明，多能干细胞用于急性缺血性脑卒中是安全的，其时间窗超过了 rt-PA 和血管内血栓切除术。我们旨在获得更强有力的证据，并展示多能干细胞治疗缺血性脑卒中的疗效。样本量 预计样本量为 220 例（每组 110 例），在 5%显著性水平下具有 90%的功效。方法和设计 TREASURE 是一项随机、双盲、安慰剂对照、多中心 2/3 期试验。该试验将在日本的 31 个医疗中心进行。入选患者为基线 NIHSS 评分 8-20 分的急性缺血性脑卒中患者，包括运动或言语功能障碍，在发病后 18-36 小时内按 1:1 随机接受单次静脉输注多能干细胞或安慰剂。主要研究终点 本研究的主要终点为第 90 天功能评估的优良结局比例。试验注册号 ClinicalTrials.gov（NCT02961504）。结论 如果结果为阳性，TREASURE 试验将为脑卒中患者提供一种新的治疗选择，并扩大治疗窗口。
