A Prospective, Randomized, Double-blind, Placebo-Controlled Phase Ⅱ Study to Evaluate the Efficacy of Adjunctive Everolimus Treatment in Patients With Refractory Epilepsy
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 108
- 试验地点
- 1
- 主要终点
- Change from baseline frequency of epileptic discharge
研究概览
简要总结
This project is a prospective, randomized, placebo-controlled, double-blind study that will evaluate the clinical efficacy of everolimus as an adjunctive treatment in adult patients diagnosed with refractory epilepsy.
详细描述
The project consists of a screening and baseline monitoring period of 1-2 weeks, and a treatment period of 1 week, followed by a 3-month follow-up period. Approximately 108 participants will be randomized in a blinded manner to one of three arms in a 1:1:1 fashion (everolimus 1h : everolimus 8-9h : Placebo). After screening, participants will have the first video-EEG monitoring for up to 24 hours to assess baseline levels, followed by 1 week of treatment, the second video-EEG monitoring, and a 3-month post treatment follow-up period. During the treatment period, participants will be given everolimus or placebo directed to seizure events. In the "everolimus 1h" group, everolimus will be administrated immediately after seizure events (within 1 hour); while in the "everolimus 8-9h" group, everolimus administration will be delayed (at 8-9 hours after seizure events). We conduct this study to assess the efficacy of everolimus in adult refractory epilepsy patients under an administration strategy in a limited time window immediately after seizure events.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 19 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of drug resistant epilepsy, with treatment of at least two approved anti-epileptic drugs (AEDs), and having at least one reported seizure per month during the 3-month baseline phase and no continuous 3-month seizure-free period.
- •Diagnosis of focal epilepsy without secondary generalization.
- •Treatment with a stable dose of AEDs that must have no drug interactions with everolimus (eg, valproic acid, topiramate, oxazepine, phenobarbital, phenytoin, and primidone) for at least 12 weeks before enrollment.
排除标准
- •History of non-drug treatment for epilepsy, eg, vagus nerve stimulation (VNS), ketogenic diet, and epilepsy surgery.
- •Severe dysfunction in kidney.
- •With significant infectious, immunologic, or oncologic comorbidity at the time of enrollment.
- •Currently taking or previously treated systemically with an mammilian target of rapamycin (mTOR) inhibitor.
- •History of seizures secondary to drug abuse, psychogenic nonepileptic seizures, or an episode of status epilepticus within 1 year before enrollment.
研究组 & 干预措施
everolimus 1h
The study participants will orally receive everolimus within 1 hour and placebo at 8-9 hours after each seizure event, but with intervals longer than 24 hours.
干预措施: Everolimus (Drug)
everolimus 1h
The study participants will orally receive everolimus within 1 hour and placebo at 8-9 hours after each seizure event, but with intervals longer than 24 hours.
干预措施: Placebo (Drug)
everolimus 8-9h
The study participants will orally receive placebo within 1 hour and everolimus at 8-9 hours after each seizure event, but with intervals longer than 24 hours.
干预措施: Everolimus (Drug)
everolimus 8-9h
The study participants will orally receive placebo within 1 hour and everolimus at 8-9 hours after each seizure event, but with intervals longer than 24 hours.
干预措施: Placebo (Drug)
placebo
The study participants will orally receive placebo both within 1 hour and at 8-9 hours after each seizure event, but with intervals longer than 24 hours.
干预措施: Placebo (Drug)
结局指标
主要结局
Change from baseline frequency of epileptic discharge
时间窗: 1 week
Comparing frequency of epileptic discharge during video-EEG monitoring after versus before treatment
次要结局
- Change from baseline frequency of seizure-free days(6 months)
- Change from baseline seizure frequency(6 months)
- Change from baseline seizure types(6 months)
- Change from baseline occurrence of secondary generalized seizure and status epilepticus(6 months)
- Seizure-free rate(3 months)
- Quality of life questionnaire (QOLIE-31-Chinese version) scores(3 months)