Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants
Status:
Recruiting
Trial end date:
2024-09-01
Target enrollment:
Participant gender:
Summary
This trial will use a two stage, phase I/II clinical trial design. The first stage will
employ an open-label clinical trial design to verify dosing for TAVT-18 (sirolimus) powder
for oral solution in TSC infants (N=5). Results will then be carried forward to inform
appropriate initial dosing, dosing frequency, and dosing adjustments for the second stage, a
randomized, double-blind, placebo-controlled multi-site study to evaluate the safety and
efficacy of early sirolimus to prevent or delay seizure onset in TSC infants.