A Study Investigating the Anti-epileptic Efficacy of Afinitor (Everolimus) in Patients With Refractory Seizures Who Have Focal Cortical Dysplasia Type II (FCD II)

• Status: Completed
• Trial end date: 2020-09-11
• Target enrollment: 0
• Participant gender: All

Summary

This is a prospective, randomized, double-blind, placebo-controlled cross over study designed to evaluate the efficacy and safety of everolimus (trough 5-15 ng/mL) given as adjunctive therapy in patients with focal cortical dysplasia type II who already failed more than two antiepileptic drugs and surgery.

This study will assess the impact of everolimus to placebo on seizure frequency in focal cortical dysplasia type II. The number of patients who experience seizure reduction of 50% or more will be counted during last 4 weeks of each core phase.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Yonsei University
• Treatments: Anticonvulsants; Everolimus; Sirolimus

Criteria

Inclusion Criteria:

• Male or female between the ages of 4 and 40

• Pathologically confirmed Focal Cortical Dysplasia type II (FCDII)

• Refractory seizure in spite of at least 2 antiepileptic drugs (AEDs) and surgery

• Subjects must have experienced at least 3 seizure events per month for two months retrospectively among 3 months prior to the baseline period in spite of using 1 AED or more.

• Must be at least one antiepileptic drug at a stable dose for at least 4 weeks at the start of the 4-week prospective baseline phase, remain on the same regimen throughout the baseline phase.

• VNS and ketogenic diet are allowed. If the patient is using VNS, device stimulator parameters must remain constant throughout the baseline phase. If the patient is on ketogenic diet, the ratio of the diet must remain constant throughout the baseline phase.

• At least 3 seizures throughout the baseline phase.

• Subjects and their legal guardians must have the ability to comprehend the informed consent form and be willing to provide informed consent. For subjects who are too young or unable to comprehend the written consent, a legal guardian who is able to describe and provide an understanding of the informed consent to the subject must sign the consent form on behalf of the subject.

Exclusion Criteria:

• Patients who need hospitalization due to causes not related to FCDII or epilepsy

• Patients who are pregnant or planning on becoming pregnant

• Patients with seizures secondary to causes other than focal cortical dysplasia

• Immunocompromised patients

• Patients who have received prior treatment with a systemic mTOR inhibitor

• Patients who do not follow up last one year

• Patients who do not show EEG abnormalities

• Patients with a history of non-compliance to medical regimens or who are considered potentially unreliable

• Patients with positive HBV Ag

• Patients who receive live vaccination during baseline study

• Patients with a known hypersensitivity to everolimus or other rapamycin-analogues(sirolimus, temsirolimus) or to its excipients

• Patients who have galactose intolerance, Lapp lactase deficiency, glucose-galactose malabsorption, or other genetic problems related to lactose digestion.