Multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of rapamycin in drug-resistant epilepsy associated with tuberous sclerosis

Acronym

Rare Ts

Project Title

Wielośrodkowe, randomizowane, podwójnie zaślepione, kontrolowane placebo badanie oceniające skuteczność i bezpieczeństwo rapamycyny w lekoopornej padaczce związanej ze stwardnieniem guzowatym.

Financing Institution

Medical Research Agency

Lead

prof. dr hab. n. med. Sergiusz Jóźwiak

Project Objective

The aim of the RaRE-TS study is to establish the effectiveness and safety of rapamycin treatment of drug-resistant epilepsy in tuberous sclerosis (TSC).
The drug will be administered off-label; rapamycin is currently registered in transplantology; in TSC, under therapeutic experiments, it was administered in cases of organ tumours, skin lesions and in few centres as a drug added to antiepileptic drugs.