Herminia D .玫瑰花% % 0期刊文章%一个Sona Gevorkian乔戈doro % %一个马丁路透社基斯Malarick % %一个泰勒让-菲利普•Coutu % D区格%一个韦恩Matson Paul j .主教练威尔肯斯% % Steven m . Hersch David h .礼拜% % T PRECREST:一二期高危亨廷顿疾病预防和肌酸的生物标志物试验% D R 10.1212 / WNL 2014%。0000000000000187 % J首页神经病学% P 850 - 857 X % V 82% N 10%目的:评估大剂量肌酸的安全性和耐受性,招收premanifest的可行性和50%高危预防试验科目,和潜在的认知,成像,和血液标记。方法:六十四合格的参与者被随机分配(1:1)15克每天两次的一水肌酸或安慰剂6个双盲阶段,后跟一个12个月的非盲扩展。主题包括premanifest(测试)和高危个体(未测试)没有临床症状和亨廷顿病(HD)的迹象。主要结果的安全性和耐受性。探索性端点包括精细动作、视觉空间的内存性能;结构和扩散磁共振成像;和选择血液标记。结果:47个高清运营商和17 non-HD控制被录取。15停止治疗(2分配给安慰剂);都是跟着整个研究期间。主要是意图治疗分析。 The most common adverse events were gastrointestinal. Neuroimaging demonstrated treatment-related slowing of cortical and striatal atrophy at 6 and 18 months.Conclusion: We describe a design that preserves the autonomy of subjects not wanting genetic testing while including controls for assessing the specificity of treatment effects. Our results demonstrate the feasibility of prevention trials for HD and the safety of high-dose creatine, provide possible evidence of disease modification, support future studies of creatine, and illustrate the value of prodromal biomarkers.Classification of evidence: This study provides Class I evidence that high-dose creatine is safe and tolerable.AD=axial diffusivity; HC=healthy control; HD=Huntington disease; PHD=premanifest Huntington disease; PRECREST=Creatine Safety and Tolerability in Premanifest HD %U //www.ez-admanager.com/content/neurology/82/10/850.full.pdf