Tuberous Sclerosis ComplexPhase 2RESTOR-1NCT07680322
Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy (RESTOR-1)
A Phase 2 study testing AV078 in people with Tuberous Sclerosis Complex and Epilepsy, run by Aeovian Pharmaceuticals, Inc. at 1 site in Canada.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Tuberous Sclerosis Complex, Epilepsy
- What is being tested?
- AV078
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 42 participants
- Where
- 1 siteCanada
- Study dates
- 1 Sep 2026to 21 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Canada1 site
London Health Sciences Centre
London, Ontario, N6A 5A5
Cadence Baker, 519-685-8500 ext. x 34769, cadence.baker@lhsc.on.ca
Andrew House, MD
About this study
Official title: 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
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.
Read the detailed description
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.
- Conditions
- Tuberous Sclerosis Complex, Epilepsy
- Being tested
- AV078, Placebo
- Keywords
- Tuberous Sclerosis Complex (TSC), Refractory Epilepsy, Drug-Resistant Epilepsy, Seizures, mTORC1 Inhibitor, AV078, mTOR Pathway
- Medical subject headings
- Tuberous Sclerosis, Epilepsy, Drug Resistant Epilepsy, Seizures
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AV078Drug
AV078 is a selective inhibitor of mammalian target of rapamycin complex 1 (mTORC1) administered orally once daily. Dosing may be adjusted based on measured drug concentrations to achieve target exposure levels.
PlaceboDrug
Also called Matching Placebo
Matching oral placebo administered once daily. The placebo is formulated to match AV078 and may undergo dose adjustments similar to active treatment to maintain blinding.
| Study group | Role | What they receive |
|---|---|---|
| AV078 | Experimental | 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. Receives: Drug: AV078 |
| Placebo | Placebo comparator | 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. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Seizure Frequency
Change from Baseline in the number of seizures experienced in participants on active treatment. The number of seizures will be recorded by participants or their caregivers using a seizure diary.
Measured: Baseline (28-day period prior to treatment) to end of treatment (Week 12)
Clinical Global Impression of Change (CGI-C)
Change from Baseline in symptoms of TSC, as evaluated by the study doctor. The Clinical Global Impression of Change (CGI-C) is a 7-point scale that evaluates overall change in a participant's clinical condition relative to baseline, with scores ranging from 1 to 7. A score of 1 indicates "very much improved," 2 "much improved," 3 "minimally improved," 4 "no change," 5 "minimally worse," 6 "much worse," and 7 "very much worse." Lower scores indicate greater improvement, and higher scores indicate worsening of clinical status.
Measured: Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jul 2026
- Study start1 Sep 2026 (estimated)
- Primary completion1 Sep 2027 (estimated)
- Study completion21 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Tuberous Sclerosis ComplexStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Participants will be randomized in a 5:1 ratio to receive oral AV078 or matching placebo in parallel treatment arms.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 42 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Aeovian Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07680322
- Sponsor's protocol ID
- CL-078-201
- Other IDs
- 2025-522330-31, 2025
- Acronym
- RESTOR-1
- Status last verified
- Jun 2026
- First submitted
- 25 Jun 2026
- Last updated
- 29 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07680322
- Acronym / sponsor ID
- RESTOR-1 / CL-078-201
- Conditions
- Tuberous Sclerosis Complex, Epilepsy
- Keywords
- Tuberous Sclerosis Complex (TSC), Refractory Epilepsy, Drug-Resistant Epilepsy, Seizures, mTORC1 Inhibitor, AV078, mTOR Pathway
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 42 participants (estimated)
- Lead sponsor
- Aeovian Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Sep 2026 (estimated)
- Primary completion
- 1 Sep 2027 (estimated)
- Study completion
- 21 Dec 2027 (estimated)
- First posted
- 2 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Participants will be randomized in a 5:1 ratio to receive oral AV078 or matching placebo in parallel treatment arms.
| Arm | Type | Receives |
|---|---|---|
| AV078 | Experimental | Drug: AV078Participants 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. |
| Placebo | Placebo comparator | Drug: PlaceboParticipants 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. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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 (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AV078AV078 is a selective inhibitor of mammalian target of rapamycin complex 1 (mTORC1) administered orally once daily. Dosing may be adjusted based on measured drug concentrations to achieve target exposure levels. | Drug | AV078 |
| PlaceboAlso: Matching PlaceboMatching oral placebo administered once daily. The placebo is formulated to match AV078 and may undergo dose adjustments similar to active treatment to maintain blinding. | Drug | Placebo |
Primary outcome
Change From Baseline in Seizure Frequency
Time frame: Baseline (28-day period prior to treatment) to end of treatment (Week 12)
Change from Baseline in the number of seizures experienced in participants on active treatment. The number of seizures will be recorded by participants or their caregivers using a seizure diary.
Clinical Global Impression of Change (CGI-C)
Time frame: Week 12
Change from Baseline in symptoms of TSC, as evaluated by the study doctor. The Clinical Global Impression of Change (CGI-C) is a 7-point scale that evaluates overall change in a participant's clinical condition relative to baseline, with scores ranging from 1 to 7. A score of 1 indicates "very much improved," 2 "much improved," 3 "minimally improved," 4 "no change," 5 "minimally worse," 6 "much worse," and 7 "very much worse." Lower scores indicate greater improvement, and higher scores indicate worsening of clinical status.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2026.
- Individual participant dataSharing planned: No
Related trials for Tuberous Sclerosis Complex
RecruitingObservational study
Study of the Disease Process of Lymphangioleiomyomatosis
Lung Disease, Pneumothorax
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
Active, not recruitingPhase 2
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Tuberous Sclerosis Complex, Epilepsy
RecruitingObservational study
Study of Skin Tumors in Tuberous Sclerosis
Tuberous Sclerosis
RecruitingPhase 2/3
Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
Tuberous Sclerosis Complex
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AV078 in people with Tuberous Sclerosis Complex and Epilepsy, run by Aeovian Pharmaceuticals, Inc. at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 29 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Davis Ryman, MD, PhD (510-961-1148, info@aeovian.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Seizure Frequency (time frame: Baseline (28-day period prior to treatment) to end of treatment (Week 12)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2026.