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)
What is the goal of the study?
This is a Phase 3, multi-site, double-blind, randomized, placebo-controlled study of adjunctive Ganaxolone (GNX) treatment in children and adults with Tuberous Sclerosis Complex (TSC)-related epilepsy. TSC is one of the most common genetic causes of epilepsy. , Seizure types and presentations vary by age. The primary goal is to evaluate the safety and effectiveness of GNX compared to placebo as adjunctive therapy for seizures associated with TSC in children and adults. This will be evaluated by the change from baseline in the frequency of countable major motor and focal seizures (primary endpoint seizures) during the double-blind phase. The study consists of a 4-week prospective baseline phase, defined as the first 28 days following screening, followed by a double-blind phase consisting of a 4-week titration period and a 12-week maintenance period. Participants eligible for inclusion in this double-blind study must have inadequately controlled seizures after exposure to at least 2 AEDs administered at adequate doses and for adequate durations, with approximately 8 seizures per month in the 2 months prior to screening and seizure-free periods of not more than 1 week. Participants who complete the study may be eligible to take part in the OLE. About 200 participants with TSC will be screened. The aim is to randomize about 162 participants (81 per arm), aged 1 to 65 years (inclusive). Participants will be randomized (1:1) to either GNX or placebo as adjunctive therapy with their standard Antiepileptic drugs.
Who can participate in the study?
Please contact the study team listed below to learn more.