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Research summary · AI summary · not yet reviewedHuman studyPhase 2Trial registry
Study of Oral AV078 in Humans with Refractory Epilepsy from Tuberous Sclerosis Complex
This Phase 2 study enrolled 42 human participants with refractory epilepsy due to TSC. They were randomized to receive either AV078 or a placebo for 12 weeks, aimed at assessing safety and seizure frequency changes. No results have been posted yet.
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This plain-English summary was written by AI from a published abstract and may contain errors. It is not medical advice. Read the original study and talk to your MS team before making decisions about treatment.
Why it matters
This research could provide insights into the effectiveness of AV078 for managing seizures in TSC, a condition often resistant to standard treatments.
What this does not prove
As no results are available, it is uncertain if the study met its endpoints or determined AV078's efficacy or safety.
Next milestone
No next milestone was established from the available source.
Study facts
- Study design
- Interventional
- Participants / samples
- 42 · basis not reported
- Randomised
- Yes
- Controlled
- Yes
- Primary endpoint met
- Not reported
- Relevant MS type
- Not reported
- Publication date
- Not reported
- Evidence reviewed
- Trial registry record
- Regulatory approval
- Not reported
- Research areas
- Not classified
Original sources
Supporting passages (12)
study phaseThis 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).
study designA 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
subjectsAV078, the \"study drug,\" is a unique medication that can decrease the activity of mTORC1.\n\nApproximately 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.
sample sizeFive 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.\n\nThe study consists of:\n\n* A screening period of approximately 4 to 6 weeks\n* A 12-week treatment period during which participants receive once-daily dosing of AV078 or placebo\n* A follow-up visit approximately 2 weeks after the end of treatment\n\nDose adjustments of AV078 may be performed based on measured drug concentrations to achieve target exposure levels.
randomized"interventionModelDescription": "Participants will be randomized in a 5:1 ratio to receive oral AV078 or matching placebo in parallel treatment arms.",
controlled"maskingDescription": "This is a double-blind study in which participants, investigators, care providers, and outcome assessors are blinded to treatment assignment.
peer reviewedNo results are posted.
interventionParticipants will receive oral AV078 once daily in addition to their stable background anti-seizure medication regimen for 12 weeks.
comparatorParticipants will receive matching oral placebo once daily in addition to their stable background anti-seizure medication regimen for 12 weeks.
primary endpointChange from Baseline in the number of seizures experienced in participants on active treatment.
findingsThe 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.
limitationsNo results are posted. Planned enrolment and endpoints are not findings.
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