A Phase 2 Study of the Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy (RESTOR-1)
Actively Recruiting
About This Study
This Phase 2, randomized, double-blind, placebo-controlled study evaluates the safety, tolerability, and efficacy of oral AV078 in participants with TSC-associated refractory epilepsy. Approximately 42 participants receive once-daily AV078 or matching placebo in a 5:1 ratio, in addition to a stable antiseizure medication regimen, for 12 weeks. AV078 is a selective mTORC1 inhibitor, and efficacy is assessed primarily by change in seizure frequency.
Who Can Participate?
✓ Inclusion Criteria
- •Participants aged 12 years and above.
- •Diagnosis of TSC based on International TSC Consensus Group criteria.
- •History of failure to adequately control seizures despite having been treated by two or more regimens of anti-seizure medications (ASMs).
- •Receiving a stable dose of ASMs for at least 4 weeks at the start of the Screening Period and for the duration of the study. Key
✗ Exclusion Criteria
- •History of any infection requiring use of antibiotics within the last four weeks.
- •Current or history of any clinically significant mental or physical illness or condition other than TSC that the Investigator believes would create significant risk for participation in the study.
- •Recent epilepsy surgery/major surgery or planned surgery during the study.
- •Treatment with medicines which act in a similar way.
- •Treatment with medicines that suppress the immune system.
- •Treatment with medicines that may significantly affect the way the body handles the study drug.