Epilepsy
Conditions
Keywords
everolimus
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.
Interventions
DRUGEverolimus
Everolimus will be administrated orally based on seizure events, with an administration interval longer than 24 hours. Participates with a body surface area (BSA) of \<= 1.2 m\^2, the dosage was 2.5 mg/time; for BSA 1.3-2.1 m\^2, the dosage was 5 mg/time; and for BSA \>=2.2 m\^2, the dosage was 7.5 mg/time.
DRUGPlacebo
Vitamin C
Sponsors
Peking University
Shengjing Hospital
National Institute on Drug Dependence, China
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
19 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline frequency of epileptic discharge | 1 week | Comparing frequency of epileptic discharge during video-EEG monitoring after versus before treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline seizure types | 6 months | Comparing types of seizures in 3 months after treatment versus baseline |
| 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 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 |
| Quality of life questionnaire (QOLIE-31-Chinese version) scores | 3 months | Comparing the scores at 3 months after treatment versus before treatment |
| Seizure-free rate | 3 months | Patients remaining seizure free in 3 months after treatment |
Countries
China
Contacts
Primary ContactWeining Ma, MD.
Outcome results
None listed