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CRAD001M2304: A three-arm, randomized, double-blind, placebo-controlled study of the efficacy and safety of two trough-ranges of everolimus as adjunctive therapy in patients with tuberous sclerosis complex (TSC) who have refractory partial-onset seizures
This study will investigate the efficacy and safety of everolimus, as adjunctive treatment, in patients ages 2-65 years with Tuberous Sclerosis Complex (TSC) that have refractory partial-onset seizures. The primary objective is to compare the reduction in frequency of partial-onset seizures between treatment groups. Treatment includes any drug or combination of drugs (required stable treatment with 1 to 3 anti-epileptic drugs to be eligible for this study) as well as placebo or evirolimus. Evirolimus has been shown to counteract the increased mTOR pathway activation caused by genetic mutations in TSC that lead to epileptic seizures. Participants in this study will be randomly assigned to one of three treatment groups: 1) high dose of evirolimus (whole blood trough concentration 9-15 ng/mL), 2) low dose of evirolimus (whole blood trough concentration 3-7 ng/mL), or 3) placebo. Participants will be required to visit KKI for 11 study visits during the core phase (including Screening visit), after which they will be invited to participate in an extension phase lasting up to 48 weeks after the last participant completes the core phase. Although all participants in the extension phase will be receiving evirolimus, blinding of the treatment groups will continue. Participants receiving evirolimus in the core phase will continue at the same target dose throughout the extension, while those on placebo during the core phase will receive evirolimus titrated to a trough level of 3 to 15 ng/mL. Participants entering the extension phase will be asked to visit KKI 6 times during the first 12 weeks, then once every 12 weeks thereafter.