This clinical trial is focused on studying the effects of a treatment for tuberous sclerosis complex (TSC), a rare genetic disorder that causes non-cancerous tumors to grow in the brain and other vital organs. The treatment being tested is an mTOR inhibitor called sirolimus, which is administered as an oral solution. The purpose of the study is to evaluate the long-term neuropsychological outcomes of this treatment in children under 4 months of age who have been diagnosed with TSC.

Participants in the study will receive either the sirolimus treatment or a placebo, in addition to the standard care they would normally receive. The study will monitor the children’s development over a period of 24 months, assessing their cognitive abilities using a tool called the Bayley Scales of Infant and Toddler Development III (BSID-III). This will help determine if the treatment has a positive impact on their neuropsychological development compared to standard care alone.

Throughout the study, various aspects of the children’s health will be closely monitored, including their behavior, signs of autism spectrum disorder, and any changes in seizure frequency. The study will also look at the size and number of tumors in the brain and heart, as well as any potential side effects of the treatment. The goal is to gather comprehensive data on the safety and effectiveness of sirolimus in improving the quality of life for children with tuberous sclerosis complex.