RecruitingPhase 3NCT05534672

Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex

Multicenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With TSC

Sponsor

Katarzyna Kotulska

Enrollment

200 participants

Start Date

Jan 23, 2023

Study Type

INTERVENTIONAL

Conditions

Tuberous Sclerosis Complex

Summary

The purpose of the RaRE-TS study is to determine safety, tolerability and efficacy of rapamycin versus placebo in a drug resistant epilepsy associated with tuberous sclerosis complex (TSC).

Eligibility

Min Age: 3 MonthsMax Age: 50 Years

Inclusion Criteria5

male or female aged from 3 months up to 50 years at the day of randomization
patients/parents/caregivers are willing to and able to give informed consent form for the participation in the study
patients/parents/caregivers are willing to and able to comply with all study requirements
definite diagnosis of TSC according to the Consensus criteria (Northrup, 2013)
drug-resistant epilepsy associated with TSC with at least 8 seizures during 4 weeks

Exclusion Criteria9

history of treatment with mTOR inhibitor in the three months prior to screening,
history of pseudo-epileptic seizures,
history of progressive CNS disease other than TSC
recent surgery within 2 weeks prior to the screening
severe infection within 2 weeks prior to the screening
use of the cannabis derivatives
contraindications for MRI or general anesthesia
occurrence of the serious comorbidities which, in the opinion of the investigator, may either put a patient at significant risk associated with the participation in the study or may influence the results of the study the investigator
pregnancy