Current Research Studies

Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study (TSC-STEPS)


What is the goal of the study?

This trial will use a phase IIb, randomized, double-blind, placebo-controlled, multicenter clinical trial design to evaluate the safety and efficacy of early sirolimus treatment to prevent epilepsy in TSC infants 0-12 months of age (N=64). Subjects will be between the ages of 0-6 months at the time of treatment initiation and meet clinical and/or genetic diagnostic criteria for TSC and have no prior history of seizures (clinical or electrographic) at the time of enrollment. Treatment will be continued until subjects reach 12 months of age.

Who can participate in the study?

Please contact the study team listed below to learn more.

Study Team: