Clinical trial · Interventional
Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy (RESTOR-1)
A Parallel-Group, Phase 2, Double-Blind, Placebo-Controlled, Randomized Study of the Safety and Efficacy of Oral AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 30, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260930-000001
Summary
Brief summary (as posted)
This Phase 2, randomized, double-blind, placebo-controlled study will evaluate the safety, tolerability, and efficacy of oral AV078 in participants with refractory epilepsy due to tuberous sclerosis complex (TSC). Approximately 42 participants will be randomized in a 5:1 ratio to receive AV078 or placebo. The study will include a Screening Period collecting 4 weeks of pre-treatment Baseline data on seizure frequency, and progress to a 12-week Treatment Period, followed by an in person final follow-up visit approximately 2 weeks after the last dose.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Epilepsy | — | UNRESOLVED | — |
| Tuberous Sclerosis Complex | — | UNRESOLVED | — |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| AV078 | Drug | — | UNRESOLVED |
| Placebo | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- AV078
- description
- Participants will receive oral AV078 once daily in addition to their stable background anti-seizure medication regimen for 12 weeks. Dosing will be titrated based on measured drug concentrations to achieve target exposure levels.
- interventionNames
- Drug: AV078
- type
- PLACEBO_COMPARATOR
- label
- Placebo
- description
- Participants will receive matching oral placebo once daily in addition to their stable background anti-seizure medication regimen for 12 weeks. Dose adjustments may be performed in a manner similar to active treatment to maintain blinding.
- interventionNames
- Drug: Placebo
Primary outcomes (2)
- measure
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 12 Years
Show eligibility criteria text
Key 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.
References
Publications (0)
Data not yet available