Gene therapy trial aims to tame severe childhood epilepsy

NCT ID NCT05419492

First seen Jun 27, 2026 · Last updated Jun 30, 2026 · Updated 2 times

Summary

This study tests a gene therapy called ETX101 for children with Dravet syndrome, a severe form of epilepsy caused by a genetic change. The treatment uses a harmless virus to deliver a working copy of the gene to help control seizures. The trial includes infants and children up to 18 years old and will measure how well the therapy reduces seizure frequency and improves development over time.

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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.

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Conditions

The condition(s) this trial relates to.

Dravet syndrome Epilepsies, Myoclonic generalized epilepsy with febrile seizures plus, type 2

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Boston Children's Hospital

    NOT_YET_RECRUITING

    Boston, Massachusetts, 02115, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Colorado Children's Hospital

    RECRUITING

    Aurora, Colorado, 80045, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Cook Children's Medical Center

    RECRUITING

    Fort Worth, Texas, 76104, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Great Ormond Street Hospital

    NOT_YET_RECRUITING

    London, WC1N3JH, United Kingdom

    Contact Email: •••••@•••••

  • Mayo Clinic

    RECRUITING

    Rochester, Minnesota, 55905, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Mott Children's Hospital

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Nationwide Children's Hospital

    NOT_YET_RECRUITING

    Columbus, Ohio, 43205, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Nicklaus Children's Hospital

    RECRUITING

    Miami, Florida, 33155, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Oregon Health and Science University (OSHU)

    RECRUITING

    Portland, Oregon, 97239, United States

    Contact Email: •••••@•••••

  • Queen Elizabeth Hospital

    RECRUITING

    Glasgow, G51 4TF, United Kingdom

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • The Royal Children's Hospital

    RECRUITING

    Melbourne, Australia

    Contact

    Contact

  • UCSF Benioff Children's Hospitals

    RECRUITING

    San Francisco, California, 94158, United States

    Contact

    Contact Email: •••••@•••••