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
Katarzyna Kotulska
Lead sponsor
Children's Memorial Health Institute, Poland
Sponsor institution