Gene therapy trial aims to tame severe seizures in kids

NCT ID NCT06112275

First seen Nov 16, 2025 · Last updated May 15, 2026 · Updated 23 times

Summary

This study tests a gene therapy called ETX101 in children aged 6 months to under 7 years with Dravet syndrome, a severe epilepsy. The treatment uses a harmless virus to deliver a working gene to brain cells. The main goals are to check safety and see if it reduces seizures. Only 4 children are enrolled in this early-stage trial.

Disclaimer Read more

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for DRAVET SYNDROME are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Contacts and locations

Locations

  • The Royal Children's Hospital

    Melbourne, Australia

Conditions

Explore the condition pages connected to this study.