Placebo controlled study to assess the efficacy and safety of rapamycin in drug resistant epilepsy associated with tuberous sclerosis complex

Prior treatment

• history of treatment with mTOR inhibitor in the three months prior to screening
• recent surgery within 2 weeks prior to the screening
• severe infection within 2 weeks prior to the screening

Other illness

• Allergy to peanuts or soy
• history of pseudo-epileptic seizures
• history of progressive CNS disease other than TSC
• hypersensitivity to the active substance or to any of the excipients of IMP / placebo

Other

• 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

• Parents/caregivers are willing to and able to give informed consent form for the participation in the study
• 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
• male or female aged from 3 months up to 55 years at the day of randomization
• body weight of at least 6 kg and proper nutritional status (assessed by investigator)