Gene therapy trial aims to tame seizures in kids with rare epilepsy
NCT ID NCT06283212
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Great Ormond Street Hospital
London, WC1N3JH, United Kingdom
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Queen Elizabeth Hospital
Glasgow, G51 4TF, United Kingdom
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Sheffield Children's Hospital
Sheffield, S10 2TH, United Kingdom