A Phase 3, Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults with Tuberous Sclerosis Complex (TSC)-related Epilepsy (TrustTSC)

Primary Objective • To assess the safety and efficacy of GNX compared to placebo as adjunctive therapy for seizures associated with TSC in children and adults as assessed by the change from baselinea in the frequency of countable major motor and focal seizures (primary endpoint seizuresb) during the double-blind phase. Secondary Objectives • To determine the percentage of change from baselinea in 28-day primary endpoint seizureb frequency during the maintenance period. • To assess the change in focal seizure frequency from baselinea during the double-blind phase. • To assess changes in mood, behavior, and quality of life using the following: o ADAMS o Peds-QL-FIM o SF-36 o ELDQOL • To assess overall clinical outcome using the CGI-I scores by the clinician and the parent/caregiver. • To evaluate the changes in seizure intensity and duration using the CGI-CSID. Exploratory Objectives • To evaluate other seizure outcomes such as change in other seizure types and changes in seizure-free days. • To evaluate changes in sleep using the CSHQ for children or adolescents or SQS for adults. • To evaluate rescue medicine utilization in participants. • To assess PK parameters in participants receiving GNX doses up to 63 mg/kg/day (1800 mg/day maximum). Safety Objectives • To assess the safety and tolerability of GNX compared with placebo as adjunctive therapy.