The Safety and Efficacy Evaluation of Everolimus as an Adjunctive Treatment for Focal Refractory Epilepsy
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18-60
SponsorXuanwu Hospital, Beijing
About this trial
The aim of this study is to evaluate the clinical efficacy of everolimus as an adjunctive therapy for refractory epilepsy. The significance lies in addressing whether the mTOR inhibitor sirolimus has antiepileptic adjunctive effects for a broader range of patients with refractory epilepsy, with the hope of providing a new mTOR-targeted antiepileptic adjunctive medication regimen that is administered only during epileptic events and can be widely used for various types of refractory epilepsy.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age between 18 and 60 years (inclusive), regardless of sex.
Diagnosis of epilepsy, meeting the criteria for focal seizures or focal to bilateral tonic-clonic seizures as defined by the 2017 International League Against Epilepsy (ILAE) classification.
History of epileptic seizures lasting ≥30 seconds, accompanied by impaired awareness.
At least 16 focal seizures during the 8-week baseline period prior to enrollment, with no seizure-free interval of 21 consecutive days.
Disqualifiers
Diagnosis of primary generalized epilepsy.
History of non-epileptic events (e.g., psychogenic non-epileptic seizures).
Patients with only non-motor focal seizures according to the 2017 ILAE classification.
Epilepsy with identifiable and treatable causes (e.g., metabolic disorders, intoxication, infection, space-occupying lesions, or confirmed genetic abnormalities).
Trial design
Design model
Single group
Treatments tested in this trial
Everolimus
DrugOral administration is given solely for the epilepsy seizure event. Dosage: The dosage is determined based on body surface area (BSA), with 2.5 mg per dose for BSA \<1.2 m², 5 mg per dose for BSA 1.3-2.1 m², and 7.5 mg per dose for BSA \>2.2 m².
Treatment groups
5 Participants
are divided into 1 treatment groupGroup A: EverolimusExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Percent Change From Baseline in Seizure Frequency Per 28 Days
Percent change in 28-day frequency of seizures during the 12 week treatment and follow-up period relative to baseline
Time frame
assessed per 28 days during the treatment until the end of 12-week treatment.Secondary outcomes
1
50% Seizure Responder Rate
The proportion of patients with a ≥ 50% reduction from Baseline in seizure frequency.
Time frame
Baseline observation period, and 12-week treatment period (ending at 12 weeks).2
Seizure Severity
The percent change from baseline in seizure severity evaluated by Liverpool seizure severity scale (LSSS) .
Time frame
Baseline observation period, and 12-week treatment period (ending at 12 weeks).3
Life quality evaluation
Percentage change from baseline in Quality of Life in Epilepsy-31 inventory (QOLIE-31) score.
Time frame
Baseline observation period, and 12-week treatment period (ending at 12 weeks).4
Adverse Events
Rate of adverse events which were judged to be study-related throughout the study.
Time frame
through study completion, 12 weeks.Sponsors and contacts
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Source ClinicalTrials.gov