文摘
摘要目的:报告设计/方法的动力,第三阶段,随机研究探索如果辅助lorcaserin DS能降低惊厥的发作频率。
背景:DS是一个严重的难治性癫痫儿童。非盲研究5-HT2C受体激动剂lorcaserin支持DS的功效在控制抽搐发作。
设计/方法:动力由一个核心研究(Pre-randomization筛选/基线(4周);(分层随机化的历史使用氟苯丙胺和重量;治疗;四周后续])和开放的扩展(OLEx)阶段(12周的治疗;四周后续])。合格标准:DS诊断;年龄≥2年;在Pre-randomization≥4抽搐发作;接收≥1抗癫痫药物。排除标准:存在的另一个进步的神经系统疾病; received lorcaserin (4-weeks) and/or fenfluramine (2-months) before Screening; previous lack of efficacy with lorcaserin/fenfluramine; recent or concomitant use of serotonergic drugs or monoamine oxidase inhibitors; enrolled in another clinical study. Patients will receive lorcaserin (oral suspension)/placebo twice daily. Target dose for weights 10–<20, 20–<40, and ≥40 kg will be 5, 10, and 20 mg/day, respectively. Based on tolerability, lorcaserin can be increased to 10/20 mg/day for patients weighing 10–<20/20–<40 kg, respectively during the first 2 weeks of treatment. Patients completing the Core Study can enter the OLEx and long-term Expanded Access Program (Study 405;NCT04457687)。核心研究主要终点:抽搐发作频率/ 28天的变化。二次端点:抽搐发作应答/发作率50%;lorcaserin药物动力学;治疗诱发的不良事件。探索性端点:临床全球变化的印象;生活质量;变化总发作的频率/ non-convulsive发作/每发作类型。epilepsy-related基因突变将收集的历史。安全评估:生命体征; weight; ECG; clinical laboratory tests; echocardiography.
结果:研究预计在2021年完工。受限制的随机化方法将随机~ 58病人1:1辅助lorcaserin /安慰剂在20 ~我们网站。
结论:势头将提供数据辅助lorcaserin作为一个潜在的治疗难治性人口。
资金:卫材公司。
披露:Devinsky博士已经收到Tilray的个人作为一个雇员补偿。Devinsky博士已经收到Empatica的个人作为一个雇员补偿。Devinsky博士已经收到个人赔偿作为受体生命科学的员工。Devinsky博士已经收到个人赔偿作为加州大麻企业的一名员工。Devinsky博士已经收到个人赔偿作为Tevard生物科学的员工。Devinsky博士已经收到个人赔偿作为脚本生物科学的员工。Devinsky博士已经收到个人赔偿作为Regel生物科学的员工。Devinsky博士已经收到个人赔偿作为Qstate生物科学的员工。Devinsky博士已经收到个人薪酬在500 - 4999美元的范围为卫材担任顾问。Devinsky博士已经收到个人薪酬在100000 - 499999美元的范围作为一个军官或董事会成员参与。 Dr. Devinsky has received personal compensation in the range of $50,000-$99,999 for serving as an officer or member of the Board of Directors for Tevard Biosciences. Dr. Devinsky has received stock or an ownership interest from Engage. Dr. Devinsky has received stock or an ownership interest from Empatica. Dr. Devinsky has received stock or an ownership interest from California Cannabis Enterprises. Dr. Devinsky has received stock or an ownership interest from Empatica. Dr. Devinsky has received stock or an ownership interest from Silver Spike. The institution of Dr. Devinsky has received research support from GW Pharma. The institution of Dr. Devinsky has received research support from Zogenix. The institution of Dr. Devinsky has received research support from PTC Pharma. Dr. Devinsky has received intellectual property interests from a discovery or technology relating to health care. Dr. Devinsky has received intellectual property interests from a discovery or technology relating to health care. Dr. Devinsky has received publishing royalties from a publication relating to health care. Gleb Filippov has received personal compensation for serving as an employee of Eisai Inc. Dr. Cheng has received personal compensation for serving as an employee of Eisai Inc. Amanda Goodwin has received personal compensation for serving as an employee of Eisai, Inc. Carlos Perdomo has nothing to disclose. Larisa Reyderman has nothing to disclose. Martin Rabe, M.Sc. has received personal compensation for serving as an employee of Eisai Inc. Dr. Irizarry has received personal compensation for serving as an employee of Eisai, Inc.. Dr. Kramer has received personal compensation for serving as an employee of Eisai Inc.
