Adjunctive Everolimus Treatment of Refractory Epilepsy
- Registration Number
- NCT05613166
- Lead Sponsor
- National Institute on Drug Dependence, China
- Brief Summary
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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 108
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description everolimus 1h Placebo 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. placebo 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. everolimus 8-9h Placebo 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. everolimus 1h Everolimus 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. everolimus 8-9h Everolimus 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.
- Primary Outcome Measures
Name Time Method Change from baseline frequency of epileptic discharge 1 week Comparing frequency of epileptic discharge during video-EEG monitoring after versus before treatment
- Secondary Outcome Measures
Name Time Method Change from baseline frequency of seizure-free days 6 months Comparing seizure-free days in 3 months after treatment versus baseline
Change from baseline seizure frequency 6 months Comparing number of seizures in 3 months after treatment versus baseline
Change from baseline seizure types 6 months Comparing types of seizures in 3 months after treatment versus baseline
Change from baseline occurrence of secondary generalized seizure and status epilepticus 6 months Comparing number of occurrence of secondary generalized seizure and status epilepticus in 3 months after treatment versus baseline
Seizure-free rate 3 months Patients remaining seizure free in 3 months after treatment
Quality of life questionnaire (QOLIE-31-Chinese version) scores 3 months Comparing the scores at 3 months after treatment versus before treatment
Trial Locations
- Locations (1)
Shengjing Hospital of China Medical University
🇨🇳Shenyang, Liaoning, China