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

What this Trial Is About

Researchers are evaluating the safety, tolerability, and effectiveness of rapamycin compared to a placebo in people with drug-resistant epilepsy linked to tuberous sclerosis complex (TSC). This is a phase 3, randomized, double-blind, placebo-controlled study aimed at understanding how rapamycin performs in this specific condition. The study is sponsored by Katarzyna Kotulska and focuses on patients aged from 3 months up to 50 years old who have a confirmed diagnosis of TSC and frequent seizures despite treatment. Participants will be randomly assigned to receive either rapamycin or a placebo in liquid form, with dosing tailored to each person's body surface area. The study includes three main phases: screening, a dose adjustment blinded phase, and a core blinded phase. After these phases, there is an open-label observation period where all participants may be monitored further. Randomization considers age, sex, and history of antiepileptic drug use to balance the groups evenly. Throughout the study, participants will be closely monitored for seizure frequency and safety outcomes. Researchers will compare the number of patients who experience at least a 50% reduction in seizures and track adverse events during the blinded core phase. Data collection includes seizure counts, days without seizures, and severity of side effects. The study aims to complete analysis within one month after the last participant's final visit, with ongoing follow-up to ensure participant safety and gather comprehensive results over the study duration.

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