Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)

This is a signal-seeking Phase II randomized, placebo-controlled trial of RAD001 in children and young adults with TSC with neurocognition as the primary outcome and autism spectrum disorder as a secondary outcome. Specific Aims /Objectives Primary objective * To evaluate the efficacy of RAD001 on neurocognition in patients with TSC compared to placebo as measured by well-validated, standardized, direct and indirect neurocognitive tools. * To evaluate the safety of RAD001 compared with placebo in patients with TSC focusing on NCI CTCAE Grade 3 and 4 adverse events, serious adverse events, and Grade 3 and 4 laboratory toxicities. Secondary objectives * Comparison of absolute change from baseline in frequency of epileptiform events as recorded on seizure diaries between patients taking RAD001 vs. placebo * Comparison of sleep disturbances between patients taking RAD001 vs. placebo, measured by the Pediatric Sleep Questionnaire (PSQ) and sleep logs * Comparison of autism spectrum disorders features between patients taking RAD001 vs. placebo, measured by ADOS and SRS * Comparison of academic skills between patients taking RAD001 vs. placebo, measured by WRAT4 * Comparison of behavioral problems between patients taking RAD001 vs placebo, measured by behavioral rating scales (BRIEF, BASC, SDQ, CHQ and SRS)