% 0期刊文章%一个茱莉亚蔡%安德鲁Ligsay % Anke Van Dijck %一个大卫Hessl弗兰克Kooy % %一个Erika Bickel %兰迪哈格曼% Albena Patroneva % T随机双盲,安慰剂对照,交叉试验Ganaxolone与脆性X综合征儿童和青少年(S46.005) % D J神经病学2017% % P S46.005 X % V % 88% N 16补充目的:评估的安全性和有效性Ganaxolone (GNX)治疗的焦虑和关注对象与脆性X综合征(FXS)。首页背景:GNX, allopregnanolone合成模拟,开发各种神经和精神疾病的治疗。GNX是一个积极的变构调制器GABAA受体,抗焦虑和抗惊厥的作用。GNX假设改善FXS症状,如焦虑、多动和注意力。设计/方法:这个探索性研究是一个双盲,安慰剂对照,交叉试验与FXS GNX的孩子。大约60岁儿童6 - 17日年FXS被随机分配在一个1:1比例。每个部门由2治疗时期,持续6周和2周洗掉。安全性和耐受性是由生命体征、心电图、实验室测试、身体检查和不良事件收集。各种临床医师和护理管理工具,包括临床改善的全球印象(CGI-I),异常行为检查表(ABC-CFX)、儿童焦虑评分量表——(PARS-R)、视觉模拟量表(血管),焦虑、抑郁和情绪屏幕(亚当斯),Swanson,诺兰,Pelham问卷是用来衡量有效性。结果:59受试者随机。55(90.2%)完成了第一期和51例(86.4%)完成了第二阶段。虽然这项研究并没有满足CGI-I的主要终点,积极影响焦虑,多动和注意力观察参与者的高基线GNX焦虑(PARS313)。 They showed reduction in anxiety (VAS and ADAMS) and hyperactivity (ADAMS and ABC-CFX), suggesting GNX may improve these symptoms. GNX was generally safe and well tolerated. Six subjects discontinued due to AEs, the most frequently reported being fatigue, somnolence, diarrhea, decreased appetite, rash, and pruritus, no SAEs.Conclusions: Ganaxolone was generally safe and well tolerated in children with FXS and showed improvements in anxiety, hyperactivity and attention in a subset of patients with baseline anxiety.Study Supported by:U.S. Army Medical Research and Material Command (USAMRMC) FUNDING MECHANISM: Contract # W81XWH-11-1-0626Jérôme Lejeune foundation, Marguerite Marie Delacroix foundation, FRAXA Research FoundationDisclosure: Dr. Tsai has received personal compensation for activities with Marinus. Dr. Tsai has received research support from Marinus. Dr. Ligsay has nothing to disclose. Dr. Van Dijck has nothing to disclose. Dr. Kooy has received personal compensation for activities with Marinus as a scientific advisory board member. Dr. Hessl has received personal compensation for activities with Marinus. Dr. Bickel has nothing to disclose. Dr. Hagerman has received personal compensation for activities with Novartis, Roche Diagnostics Corporation, Zynerba, Ovid, and Marinus as a consultant. Dr. Patroneva has received personal compensation for activities with Marinus as the Chief Medical Officer and holds stock options with Marinus. %U