Deutetrabenazine影响电动机总分亨廷顿疾病患者(第一)(S27.008)

文摘

目的:量化deutetrabenazine整体运动措施的变化,评估的统一亨廷顿疾病评定量表(UHDRS)电动机总得分(TMS)。背景:亨廷顿病(HD)是一个世袭的,进步的,神经退行性疾病,其特征是舞蹈病、认知功能障碍和精神症状。Deutetrabenazine (sd - 809)是一种新型的分子目前正在开发的治疗相关的高清舞蹈病。设计/方法:将90例患者随机(1:1)deutetrabenazine或安慰剂治疗在这个双盲,安慰剂对照,与这些相应平行的组织学习。受试者走动的成年人患有运动体现高清,考试的电动机特性和计画扩大CAG重复(≥37),谁有一个基线总最大舞蹈病(TMC)得分≥8。TMC由7 TMS的31项。台湾记忆体公司是主要终点和经颅磁刺激是一个预先确定端点。结果:从基线deutetrabenazine组,经颅磁刺激提高7.4分(标准错误(SE) 0.9)和3.4分(0.9 SE)安慰剂组(P = 0.002)。这个结果是由显著改善舞蹈病(主要终点;提高了4.4和1.9分,P < 0.0001),肌张力障碍(提高了0.9和0.1分,P = 0.02)。 Changes in other UHDRS motor components did not differ significantly between treatment groups, and there was no worsening in gait or parkinsonism subscores. Rates of depression, irritability, insomnia were low and similar to or less than those observed with placebo. CONCLUSION: While deutetrabenazine clearly improved chorea, there was also significant improvement in TMS, with substantial contributions from improved dystonia. Combined with its favorable safety profile, the TMS findings suggest deutetrabenazine can provide clinically meaningful overall motor benefit to patients with HD. Study Supported by: Auspex Pharmaceuticals - a wholly owned subsidiary of Teva Pharmaceuticals, Inc.

披露:弗兰克博士获得了研究支持通过亨廷顿研究小组,由药品占卜者。斯塔姆勒博士已经收到个人活动与占卜者制药作为雇员补偿。唱博士已经收到个人活动与占卜者补偿制药和Lundbeck公司,它是一家公司担任顾问。Claassen博士接到占卜者制药研究支持。Janicki博士没有披露。阿来博士没有披露。Furr-Stimming博士没有披露。奥克斯博士已经收到了个人在一篇社论中补偿的弹跳能力。甲壳博士获得了研究亨廷顿研究集团有限公司的支持。