TY -的T1 PRECREST:第二阶段预防和高危亨廷顿疾病生物标志物审判的肌酸JF -神经学乔-神经病学SP - 850 LP - 857 - 10.1212 / WNL。首页0000000000000187六世Herminia d - 82 - 10盟-罗萨斯盟-乔戈doro盟Sona Gevorkian AU -基斯Malarick AU -马丁路透社AU -让-菲利普•Coutu AU -泰勒d区格盟- Paul j .主教练威尔肯斯盟韦恩Matson AU - David h .礼拜盟Steven m . Hersch Y1 - 2014/03/11 UR - //www.ez-admanager.com/content/82/10/850.abstract N2 -目的:评估大剂量肌酸的安全性和耐受性,招收的可行性premanifest预防首页试验和50%高危对象,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。 Primary analysis was by intent to treat. 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 ER -