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

Inclusion Criteria:

* 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 Criteria:

* 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

Conditions3

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