NCT05534672

Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex

Multicenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With TSC

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 200 (estimated)
• Sponsor: Katarzyna Kotulska · Academic / Other
• Sex: All
• Age: 3 Months – 50 Years
• Healthy volunteers: Not accepted

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).

Detailed description

This is a two-arm, randomized, double-blind, placebo controlled study to evaluate the efficacy, tolerability, and safety of rapamycin versus placebo in a drug resistant epilepsy associated with TSC. The study consists of 3 phases for each patient: screening, dose adjustment blinded phase, core blinded phase, followed by open-label observation. Patients who meet the eligibility criteria will be randomized to receive rapamycin or placebo. The randomization ratio is 1:1. Randomization will be stratified by age, sex and and the number of antiepileptic drugs ever used in the patient's history (up to 3 drugs / more than 3 drugs).

Conditions

• Tuberous Sclerosis Complex

Interventions

Type	Name	Description
OTHER	Rapamycin	Rapamycin in liquid administered orally
OTHER	Placebo	Placebo in liquid administered orally

Timeline

• Start date: 2023-01-23
• Primary completion: 2027-06-01
• Completion: 2027-06-01
• First posted: 2022-09-09
• Last updated: 2023-08-08

Locations

2 sites across 1 country: Poland