AV078: 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.
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 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.
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.
Eligibility
Sex
ALL
Min age
12 Years
Max age
—
Healthy volunteers
No
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.
Primary outcome measure(s)
Change From Baseline in Seizure Frequency — 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) — 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.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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