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).
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)
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
Parallel
Rapamycin
Other interventionRapamycin in liquid administered orally
Placebo
Other interventionPlacebo in liquid administered orally
comparison of the number of patients with at least 50% reduction of seizures per week in the last month of the core blinded phase in comparison to screening phase in the rapamycin vs placebo group
number of adverse events (according to CTCAE classification) in the rapamycin vs placebo group during the double-blind core phase
comparison of the number of seizures per week and the number of days free of seizures in the rapamycin vs placebo group, during 12-week treatment in double-blind core phase
severity of adverse events (according to CTCAE) and the number of patients withdrawn from the study due to adverse events in the rapamycin vs placebo group
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Katarzyna Kotulska
Lead sponsor
Children's Memorial Health Institute, Poland
Sponsor institution
