NCT05534672 Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex
| NCT ID | NCT05534672 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Katarzyna Kotulska |
| Condition | Tuberous Sclerosis Complex |
| Study Type | INTERVENTIONAL |
| Enrollment | 200 participants |
| Start Date | 2023-01-23 |
| Primary Completion | 2027-06 |
Trial Parameters
Eligibility Fast-Check
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Brief 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 Criteria
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 as