Adjunctive Everolimus Treatment of Refractory Epilepsy

NCT ID: NCT05613166

Last Updated: 2022-11-14

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 108 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-11-30
• Study Completion Date: 2023-03-31

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.

Conditions

Epilepsy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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.

Group Type: EXPERIMENTAL

Everolimus

Intervention Type: DRUG

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.

Placebo

Intervention Type: DRUG

Vitamin C

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.

Group Type: EXPERIMENTAL

Everolimus

Intervention Type: DRUG

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.

Placebo

Intervention Type: DRUG

Vitamin C

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.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Vitamin C

Interventions

Everolimus

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.

Intervention Type: DRUG

Placebo

Vitamin C

Intervention Type: DRUG

Other Intervention Names

Afinitor

Eligibility Criteria

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.

• Minimum Eligible Age: 19 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Peking University

OTHER

Sponsor Role: collaborator

Shengjing Hospital

OTHER

Sponsor Role: collaborator

National Institute on Drug Dependence, China

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Shengjing Hospital of China Medical University

Shenyang, Liaoning, China

Site Status: RECRUITING

Countries

China

Central Contacts

Weining Ma, MD.

Role: CONTACT

maweining1985@163.com

86-024-96615-36316

Facility Contacts

Weining Ma, MD.

Role: primary

maweining1985@163.com

86-024-96615-36316

Other Identifiers

PKU-SJ-01-2021-V1

Identifier Type: -

Identifier Source: org_study_id