Tuberous Sclerosis Complex-Sirolimus TSC Epilepsy Prevention Study

What is the TSC-Steps study about?

Epilepsy is a common symptom present in infants diagnosed with tuberous sclerosis complex (TSC) and it can be difficult to treat. TSC-Steps aims to evaluate the safety and efficacy of early treatment with sirolimus for epilepsy prevention in infants with TSC. Sirolimus is a medication that is often used in older children and adults with TSC. The hope of this study is to test if sirolimus treatment can aid in the prevention of epilepsy before seizure onset.

Infants are seen starting at 6-months of age. Behavioral assessments, EEG, and MRI recordings are conducted across 7-8 study visits spanning over 2 years. Clinical outcomes are assessed, and EEG recordings are evaluated by our lab to further understand neural connectivity in infants treated with sirolimus compared to placebo.

Who can participate?

Infants under 6 months of age with a TSC diagnosis and no history of seizures are eligible for this study. Infants also cannot have been treated with anti-seizure medications prior to enrolling in TSC-Steps.

There is currently no compensation for participating in this clinical trial; however, there are funds available to help pay for travel expenses.