PT -期刊文章盟Vissia Viglietta AU -奥格尔曼(John AU -莱斯利·威廉姆斯盟-英田AU -阿尔弗雷德沙岩盟蕾切尔杜迪AU -斯蒂芬Salloway盟弗雷德里克Barkhof AU -布鲁诺维拉拉盟玛丽左盟——保罗·爱森盟——杰弗里·科洛TI -随机、双盲、安慰剂对照研究,以评估治疗Aducanumab (BIIB037)患者早期阿尔茨海默氏症:第三阶段研究设计(S1.003) DP - 2016年4月05 TA -神经病学PG - S1.003 VI - 86 IP - 16补充4099 - //www.ez-admanager.com/content/86/16_Supplement/S1.003.short 4100 - //www.ez-admanager.com/content/86/16_Supplement/S1.003.full所以Neurology2016 4月05;首页86 AB -目的:描述两个阶段3的设计研究评价疗效和安全性aducanumab (BIIB037)患者早期阿尔茨海默病(AD)。背景:Aducanumab是人类单克隆抗体对聚合β淀粉样蛋白(Aβ)被调查作为一个疾病修饰治疗广告。临时结果进行第一阶段b在前驱的学习/轻微的广告展示目标订婚,药效学Aβ减少的影响,和对探索性临床影响结果;主要安全/耐受性结果amyloid-related成像异常(咏叹调)。设计/方法:两个随机、安慰剂对照,3期研究(参与;出现),相同的设计,是招收患者(N≍1350,≍150网站/研究)50 - 85岁,与早期的广告,临床痴呆评定(CDR)全球得分0.5,电池可重复的神经心理状态的评估分数≤85,细微精神状态检查(MMSE)得分能力和大脑淀粉样蛋白(以PET)的存在。为期18个月的安慰剂对照段,患者将随机比每月第四低收入(3或6毫克/公斤)或高剂量(6到10毫克/公斤)aducanumab(基于ApoE4载体状态;剂量滴定限制咏叹调)或安慰剂。主要终点是改变CDR-Sum基线的盒子分数(78周)。 Secondary endpoints are change from baseline in MMSE, AD Assessment Scale-Cognitive Subscale, and AD Cooperative Study-Activities of Daily Living Inventory (MCI version) scores (Week 78). Tertiary endpoints include safety, pharmacokinetics, and clinical, radiologic, and patient/informant-reported outcomes. Following the placebo-controlled period, individuals may enter the 24-month dose-blind extension in which those randomized to placebo in placebo-controlled period will be re-randomized 1:1 (based on ApoE ε4 carrier status) to low- or high-dose aducanumab, to evaluate long-term safety, tolerability and efficacy of aducanumab. RESULTS: N/A. CONCLUSION: ENGAGE and EMERGE are Phase 3 clinical trials designed to confirm the clinical efficacy and safety profile of aducanumab in individuals with Early AD. Study support: funded by Biogen Disclosure: Dr. Viglietta holds stock and/or stock options in Biogen Idec. Dr. O'Gorman holds stock and/or stock options in Biogen Idec. Dr. Williams holds stock and/or stock options in Biogen Idec. Dr. Tian has received personal compensation for activities with Biogen as an employee. Dr. Tian holds stock and/or stock options in Biogen which sponsored research in which Dr. Tian was involved as an investigator. Dr. Sandrock holds stock and/or stock options in Biogen as an employee of the company. Dr. Doody has received research support from Accera, Genentech, Merck Serono, and Takeda. Dr. Salloway has receiveed research support for the conduct of clinical trials sponsored by Lilly, Merck, Roche, Functional Neuromodulation, Biogen, Genentech, and Avid. Dr. Barkhof has received personal compensation for activities with Bayer Schering Pharma, Biogen Idec, Genzyme, Janssen Research, Merck Serono, Novartis, Roche, Sanofi, Synthon BV, and Teva as a consultant. Dr. Vellas has received personal compensation for activities with Biogen Idec, GlaxoSmithKline, Lilly, Lundbeck, Medivation, MSD, Nestlé, Nutricia, Pfizer, Roche, Sanofi, Servier, and TauRx Therapeutics. Dr. Sano has received personal compensation for activities with Biogen, Eli Lilly, F Hoffman-La Roche Ltd, Forum Pharmaceutical, Genentech, Neurocog Trials, Otsuka, Takeda, Targaset, and VTV Therapeutics. Dr. Aisen receives research support from Eli Lilly, the Alzheimers Association & the NIH and NIA. Dr. Sevigny holds stock and/or stock in Biogen Idec.Saturday, April 16 2016, 6:30 am-8:30 am