A Phase 2 interventional study of AV078 and Placebo in Tuberous Sclerosis Complex and Epilepsy, sponsored by Aeovian Pharmaceuticals, Inc.. Not yet recruiting at 1 site in Canada. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2026-09-29.
Sponsored by Aeovian Pharmaceuticals, Inc. · Phase 2, Interventional, and Treatment
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.
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:
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.
115 studies on the registry are indexed under Tuberous Sclerosis; 27 are open to participants now.
This study's planned enrollment of 42 is below the median of 52 across 73 interventional studies indexed under Tuberous Sclerosis.
Browse Tuberous Sclerosis studies →Aeovian Pharmaceuticals, Inc. is the lead sponsor of 2 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Drug: AV078
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.
Drug: Placebo
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.
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.
Also known as: Matching Placebo
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.
Time frame: 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.
Time frame: Week 12
Plan to share: No
No publications or documents are linked to this record.
Start is estimated for Sep 2026; primary completion for Sep 2027, 12 months later.
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From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is not yet recruiting, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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Aeovian Pharmaceuticals, Inc.