TY - T1的功效Soticlestat治疗Dravet综合症患者的癫痫类型或Lennox-Gastaut综合症在阶段2中,随机、安慰剂对照研究(艾丽卡)(赛0 - 1.002)摩根富林明-神经学乔-神经学做- 10.1212 / WNL。首页0000000000203534六世- 100 - 17补充2 SP - 3800 AU - Venkatesha没吃非盟-丹尼斯Dlugos盟亚希尔汗AU -塞缪尔·萧盟Arturo贝尼特斯盟Mahnaz Asgharnejad Y1 - 2023/04/25 UR - //www.ez-admanager.com/content/100/17_Supplement_2/3800.abstract N2 -目首页标:开发一个soticlestat治疗功效的事后分析Dravet综合症患者的癫痫类型(DS)或Lennox-Gastaut综合症(LGS)厄勒克特拉(NCT03650452),第二阶段,随机、安慰剂对照的研究辅助soticlestat(≤300毫克,体重调整一下给)与DS 2-17岁儿童展示≥3抽搐发作/ 28天,或与LGS展示在基线≥4降发作/ 28天。背景:DS和LGS涉及发育迟缓和频繁的癫痫活动。厄勒克特拉发表研究调查soticlestat的疗效和安全性,胆固醇24-hydroxylase抑制剂,DS儿童或LGS。主要终点是剧烈的变化(DS)和(LGS)发作频率下降。设计/方法:事后功效分析确定癫痫发作频率变化百分比从基线,发作类型。记录病人的护理人员扣押数量和类型在基线和完整(提供)治疗周期,包括广义tonic-clonic、双边tonic-clonic焦,焦与运动迹象和清音的发作。结果:141年招收患者中,126(89%)完成了艾丽卡。修改后的intent-to-treat人口收到≥1剂研究药物和≥1效果评估(DS, n = 51;LGS, n = 88)。发作频率变化中值基线DS患者接受soticlestat(安慰剂):广义tonic-clonic癫痫,−27.2% (n = 14) (19.5%, n = 21);焦双边tonic-clonic癫痫,−73.9% (n = 9) (8.4%, n = 4)。 Median seizure frequency changes from baseline in patients with LGS receiving soticlestat (placebo): generalized tonic–clonic seizures, −33.4% [n=17] (−20.0%, n=11); focal seizures with motor signs, −64.3% [n=10] (−21.1%, n=7); atonic seizures, −33.9% [n=16] (−17.9%; n=13); focal to bilateral tonic–clonic seizures, −41.3% [n=6] (none observed).Conclusions: Reduced median frequency of specific seizure types was observed in patients receiving soticlestat as adjunctive therapy. Investigation of soticlestat adjunctive therapy for these seizure types in other epilepsies is warranted (phase 3 studies ongoing for DS and LGS).Study funded by Takeda Pharmaceutical Company Limited.Disclosure: Dr. Pathi Jagannatham has received personal compensation for serving as an employee of Takeda Pharmaceuticals. Dr. Pathi Jagannatham has received personal compensation for serving as an employee of GlaxoSmithKline. Dr. Pathi Jagannatham has stock in Takeda Pharmaceuticals. Dr. Pathi Jagannatham has stock in GlaxoSmithKline. Dr. Dlugos has received research support from NIH. The institution of Dr. Dlugos has received research support from The Epilepsy Study Consortium. Yasir Khan has nothing to disclose. Dr. Hsiao has received personal compensation for serving as an employee of Takeda Pharmaceutical Company. Dr. Hsiao has stock in Takeda Pharmaceutical Company. Dr. Benitez has received personal compensation for serving as an employee of Takeda . Mahnaz Asgharnejad, PharmD has received personal compensation for serving as an employee of Takeda Pharmaceuticals. Mahnaz Asgharnejad, PharmD has stock in GlaxoSmithKline. Mahnaz Asgharnejad, PharmD has stock in Takeda Pharmaceuticals. ER -