ClinicalTrials.gov record NCT07680322
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
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase 2, randomized, placebo-controlled trial tests once-daily oral AV078 for 12 weeks in people with refractory epilepsy due to tuberous sclerosis complex. About 42 participants will be randomized 5:1 to AV078 or matching placebo, after a 4-week baseline period of seizure recording. There is an in-person follow-up visit about 2 weeks after the last dose.
What it is studying. The study is testing whether 12 weeks of once-daily oral AV078 (a selective inhibitor of mTORC1) is safe, tolerable, and affects seizure frequency compared with placebo in people with TSC-related refractory epilepsy.
What the researchers hope to learn. To evaluate the safety and tolerability of AV078, and to measure its effect on seizure frequency and overall clinical change (Clinical Global Impression of Change) over a 12-week treatment period.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- AV078
- Placebo
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Show the detailed description from the registry
This study is a Phase 2, randomized, placebo controlled, double-blind, study that will evaluate the safety, tolerability, and efficacy of 12 weeks of treatment with AV078 (a selective inhibitor of mammalian target of rapamycin complex 1 (mTORC1) in participants with refractory epilepsy due to TSC. Tuberous Sclerosis Complex (TSC) is a genetic disorder where mTOR1 complex 1 (or mTORC1) becomes more active than normal. This is an important cause of TSC symptoms, including epilepsy, which can be very difficult to treat. Developing a drug to act directly on the mTORC1 complex, reducing its activity, may be an effective way to treat the unmet medical needs of patients with TSC epilepsy, potentially with fewer side effects than existing medications. The purpose of this study is to determine, over a 12-week treatment period, if the investigational drug AV078 is safe and can reduce seizures in people with TSC. AV078, the "study drug," is a unique medication that can decrease the activity of mTORC1. Approximately 42 participants will be enrolled and randomized in a 5:1 ratio to receive oral AV078 or matching placebo in addition to their existing stable anti-seizure medication regimen. Five participants aged 18 years and above will enrolled and dosed for at least four weeks before participants aged 12 years and above can be eligible to enroll. The study consists of:
- A screening period of approximately 4 to 6 weeks
- A 12-week treatment period during which participants receive once-daily dosing of AV078 or placebo
- A follow-up visit approximately 2 weeks after the end of treatment
Dose adjustments of AV078 may be performed based on measured drug concentrations to achieve target exposure levels. Safety assessments will include monitoring of adverse events, laboratory tests, vital signs, electrocardiograms, and other clinical evaluations. Efficacy will be assessed primarily through changes in seizure frequency.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
You would complete a 4–6 week screening that includes 4 weeks of baseline seizure recording, then be randomized to take once-daily oral AV078 or matching placebo for 12 weeks while continuing your stable anti-seizure medications. You and/or your caregiver will keep a seizure diary throughout baseline and treatment. Dosing may be adjusted during the study based on measured drug concentrations to reach target exposure. Safety monitoring during the study will include laboratory tests, vital-sign checks, electrocardiograms, other clinical evaluations, and an in-person follow-up visit about two weeks after the last dose.
How the study is structured. Randomized, parallel-group, placebo-controlled trial with masking so participants and key study staff do not know treatment assignments. There is a screening period including a 4-week baseline seizure diary, a 12-week treatment period with once-daily dosing (dose adjustments may be made based on measured drug concentrations), and a single in-person follow-up visit about 2 weeks after treatment ends. Participants continue their stable background anti-seizure medications while receiving study drug or placebo.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- The listed sites are in Canada. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on tuberous sclerosis complex
Important dates
- Study start
- Sep 1, 2026 (estimated)
- Primary completion
- Sep 1, 2027 (estimated)
- Study completion
- Dec 21, 2027 (estimated)
- Target enrollment
- 42 participants (estimated)
- Last updated on registry
- Sep 29, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Aeovian Pharmaceuticals, Inc.
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Davis Ryman, MD, PhD 📞 510-961-1148 ✉ info@aeovian.com
Participating sites (1)
- London Health Sciences Centre
London, Ontario, N6A 5A5 · Canada
- Cadence Baker · 519-685-8500 ext. x 34769 · cadence.baker@lhsc.on.ca
- Andrew House, MD
Terms used on this page · explanations by MedicalUpdates.ai
- randomized
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- double-blind
- Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
- placebo-controlled
- A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
- refractory
- A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
- placebo
- A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
- Screening Period
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- Baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- stable dose
- A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
- study drug
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- Key Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Key Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- adverse events
- Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this website; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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