2024-515937-13-00
Recruiting
Phase 3
Multicenter, randomized, double-blind, placebo controlled study to assess the efficacy and safety of RApamycin in drug Resistant Epilepsy associated with TSC
Instytut Pomnik Centrum Zdrowia Dziecka4 sites in 1 country200 target enrollmentStarted: December 20, 2024Last updated:
Overview
- Phase
- Phase 3
- Status
- Recruiting
- Sponsor
- Instytut Pomnik Centrum Zdrowia Dziecka
- Enrollment
- 200
- Locations
- 4
- Primary Endpoint
- 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 (
Overview
Brief Summary
The primary objective 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
Eligibility Criteria
- Ages
- 0 years to 64 years (0-17 Years, 18-64 Years)
- Accepts Healthy Volunteers
- Yes
Inclusion Criteria
- •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)
Exclusion Criteria
- •history of treatment with mTOR inhibitor in the three months prior to screening
- •Allergy to peanuts or soy
- •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
- •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
- •hypersensitivity to the active substance or to any of the excipients of IMP / placebo
Outcomes
Primary Outcomes
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 (
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
number of adverse events (according to CTCAE classification) in the rapamycin vs placebo group during the double-blind core phase
Secondary Outcomes
- 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
Investigators
Katarzyna Kotulska-Jóźwiak
Scientific
Instytut Pomnik Centrum Zdrowia Dziecka
Study Sites (4)
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