Can an early medication prevent seizures in babies with tuberous sclerosis?

Plain-English translation of NCT05104983 on ClinicalTrials.gov ↗ · Source last updated January 2026 · Translation generated June 2026 · How we translate trials

Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.

What is this trial?

Tuberous sclerosis is a genetic condition that often causes seizures in infants, sometimes affecting their long-term brain development. This study is testing whether the medication , given very early to babies with this diagnosis, can help prevent or delay those first seizures. Researchers hope that stopping seizures before they start might improve outcomes for these infants.

When babies with tuberous sclerosis have their first seizure early in life, it can impact their brain development and long-term learning and development. This medication has already been used to treat seizures in people with this condition, but this will be the first study to see whether giving it before seizures happen can actually prevent them from occurring in the first place.

Do you qualify?

You likely qualify if…

you are between newborn and 6 months old at enrollment (less than 7 months at the time of treatment start)
you have been diagnosed with tuberous sclerosis through genetic testing or clinical criteria
you have never had a seizure (clinical or on brain wave monitoring)
your corrected age is at least 39 weeks (accounting for prematurity)

You likely don't qualify if…

you have ever had a seizure or seizure-like brain activity, even if very brief
you are currently taking or have recently taken anti-seizure medications or other mTOR inhibitor drugs
you were born very prematurely (before 30 weeks) or had serious complications at birth that could increase seizure risk
you have a TSC-related tumor that already needs mTOR treatment
you have significant liver, kidney, or bone marrow problems on recent blood tests

What participation looks like

If your baby is enrolled, you will be randomly assigned to give either the medication or a placebo (inactive treatment) regularly. You will attend study visits to monitor your baby's health, have blood tests done, and complete brain wave recordings (EEGs) to watch for any seizure activity. The study will track your baby's development and seizure status over time to see whether the early treatment makes a difference.

AI-generated summary from trial data · Jun 1, 2026 · Not medical advice

Trial locations(11 sites)

United States

University of Alabama at Birmingham, Birmingham, Alabama

University of California at Los Angeles, Los Angeles, California

Stanford University, Palo Alto, California

Children's Hospital Colorado, Aurora, Colorado

Lurie Children's Hospital of Chicago, Chicago, Illinois