Gene therapy for hard-to-treat temporal lobe seizures

Plain-English translation of NCT06063850 on ClinicalTrials.gov ↗ · Source last updated July 2026 · Translation generated June 2026 · How we translate trials

Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.

This is one of the first times this treatment has been tested in people.

What is this trial?

This is the first human study of AMT-260, a new gene therapy for a specific type of hard-to-treat epilepsy called mesial temporal lobe epilepsy. The treatment is delivered directly to the brain during a single surgical procedure using advanced imaging guidance, without removing any brain tissue. Researchers are testing two different dose levels to find the safest and most effective dose.

Many people with mesial temporal lobe epilepsy continue to have frequent, disabling seizures even after trying multiple anti-seizure medications. This study exists to see whether a one-time gene therapy approach can help control seizures in people for whom current medications are not working well enough.

Do you qualify?

You likely qualify if…

you have been diagnosed with unilateral mesial temporal lobe epilepsy that hasn't responded well to medications
you have had at least 2 documented focal seizures or seizures that spread to both sides of your brain per month over the past 3 months
you are currently taking up to 4 anti-seizure medications at stable doses
imaging tests (MRI and/or PET scans) confirm that your seizures originate from a single, specific location in your hippocampus
you are willing to use reliable birth control throughout the study (if you are a woman who could become pregnant or a man who could father a child)

You likely don't qualify if…

you have implanted medical devices that are not compatible with MRI scans (or if you have compatible devices, they were implanted less than 3 months ago)
you cannot safely undergo general anesthesia or brain surgery
you are taking medications that could interfere with the study or make surgery unsafe
you have seizures that originate from both sides of your brain or from multiple locations
you have had previous brain surgery for epilepsy treatment (except for diagnostic electrode placement), or you have had a serious seizure lasting 30 minutes or longer in the past year

What participation looks like

If you join this study, you will receive the gene therapy treatment during one surgical procedure performed with real-time MRI guidance. You will then be monitored closely through regular study visits, phone calls, blood tests, and questionnaires about how your seizures affect your daily life. You will also record your seizures in an electronic diary to help researchers track how the treatment is working over time.

AI-generated summary from trial data · Jun 3, 2026 · Not medical advice

Trial locations(18 sites)

United States

University of Alabama at Birmingham, Birmingham, Alabama

Mayo Clinic Arizona, Phoenix, Arizona

Stanford University, Palo Alto, California

Mayo Clinic Florida, Jacksonville, Florida

Kansas University Medical Center, Kansas City, Kansas