PT -期刊文章盟-布鲁斯·科恩盟阿梅尔Karaa AU -理查德·哈斯AU -艾米·戈尔茨坦盟-杰拉德Vockley TI -影响Elamipretide成人原发性线粒体肌病:第二阶段的一项双盲,随机,安慰剂对照交叉试验(MMPOWER-2) (S5.003) DP - 2018年4月10 TA -神经病学PG - S5.003 VI - 90 IP - 15补充4099 - //www.ez-admanager.com/content/90/15_Supplement/S5.003.short 4100 - //www.ez-admanager.com/content/90/15_Suppl首页ement/S5.003.full所以Neurology2018 4月10;90年MMPOWER AB -目标:患者基因的研究证实主要线粒体肌病(PMM),静脉elamipretide(以拦)5天增加步行的距离和安慰剂在6分钟步行试验(6 mwt)以剂量依赖的方式。当前的研究(MMPOWER-2)评估4周皮下拦在几个功能的影响和patient-reported评估。背景:常见症状的疲劳,运动不耐受,肌肉无力和PMM成年人身体功能和生活质量产生负面影响。埃兰是一个aromatic-cationic四肽很容易穿透细胞膜和暂时性的本地化内线粒体膜,它的同事心磷脂,提高ATP生产和运动能力。设计/方法MMPOWER科目(N = 30)被随机分配(1:1),4周的每日以拦40毫克(治疗期1),然后4周匹配的安慰剂治疗期2(四周冲刷中间),反之亦然,评估期间走的距离6 mwt(主要终点),和其他功能评估,patient-reported问卷,安全评估(二次端点)。结果:6 mwt走进的距离与安慰剂相比提高了19.8米每天以拦40毫克的治疗期(P = 0.0833)。在治疗结束,ELAM-treated受试者& lt;走450米在基线有更大的进步在安慰剂和8.5米(24.3米)改进对于那些走在基线相比安慰剂≥450米。PMMSA总疲劳和疲劳的活动中成绩表明ELAM-treated主题报道更少的症状在治疗的最后一周(分别为P = 0.0006, P = 0.0018)。ELAM-treated科目也有改善Neuro-QoL疲劳评分在治疗与安慰剂。注射部位反应是最常报道的AE与拦(80%)。Conclusions: Subjects who received daily ELAM for 4 weeks had an improvement in their 6MWT and patient reported outcomes; therefore, a phase 3 trial will be initiated.Study Supported by: Stealth BioTherapeuticsDisclosure: Dr. Cohen has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Stealth Biotheraputics. Dr. Karra has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Stealth BioTherapeutics. Dr. Karra has received research support from Stealth BioTherapeutics. Dr. Haas has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Stealth BioTherapeutics. Dr. Haas has received research support from Stealth BioTherapeutics. Dr. Goldstein has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Stealth BioTherapeutics. Dr. Goldstein has received research support from Stealth BioTherapeutics. Dr. Vockley has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Stealth BioTherapeutics. Dr. Vockley has received research support from Stealth BioTherapeiutics.