Gene therapy trial aims to tame seizures in kids with rare epilepsy

NCT ID NCT06283212

Ongoing Disease control Sponsor: Encoded Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage study tests a gene therapy called ETX101 in 5 children with Dravet syndrome, a severe form of epilepsy. The therapy delivers a gene that helps calm overactive brain cells. Researchers will check if it safely reduces seizure frequency and improves thinking and daily skills over a year.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: ETX101 (a gene therapy that boosts SCN1A gene activity in the brain)
What this could lead to: If it works, this could point toward a treatment that reduces seizures and improves developmental outcomes in children with Dravet syndrome.
What could go wrong: This is a very early, small trial (only 5 children) testing safety and dosing. Gene therapies can have side effects like immune reactions, and it may not work for everyone.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Dravet syndrome Epilepsies, Myoclonic

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DRAVET SYNDROME

Contacts and locations

Locations

Great Ormond Street Hospital

London, WC1N3JH, United Kingdom
Queen Elizabeth Hospital

Glasgow, G51 4TF, United Kingdom
Sheffield Children's Hospital

Sheffield, S10 2TH, United Kingdom

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