Gene therapy trial aims to tame severe childhood epilepsy

NCT ID NCT05419492

Recruiting now Disease control Sponsor: Encoded Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 30, 2026 · Updated 6 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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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.

DRAVET SYNDROME

Contacts and locations

Locations

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

Chicago, Illinois, 60611, United States
Boston Children's Hospital
NOT_YET_RECRUITING

Boston, Massachusetts, 02115, United States
Colorado Children's Hospital
RECRUITING

Aurora, Colorado, 80045, United States
Cook Children's Medical Center
RECRUITING

Fort Worth, Texas, 76104, United States
Duke Children's Hospital & Health Center
NOT_YET_RECRUITING

Durham, North Carolina, 27705, United States
Great Ormond Street Hospital
NOT_YET_RECRUITING

London, WC1N3JH, United Kingdom
Mayo Clinic
RECRUITING

Rochester, Minnesota, 55905, United States
Mott Children's Hospital
RECRUITING

Ann Arbor, Michigan, 48109, United States
Nationwide Children's Hospital
NOT_YET_RECRUITING

Columbus, Ohio, 43205, United States
Nicklaus Children's Hospital
RECRUITING

Miami, Florida, 33155, United States
Oregon Health and Science University (OSHU)
RECRUITING

Portland, Oregon, 97239, United States
Queen Elizabeth Hospital
RECRUITING

Glasgow, G51 4TF, United Kingdom
The Royal Children's Hospital
RECRUITING

Melbourne, Australia
UCSF Benioff Children's Hospitals
RECRUITING

San Francisco, California, 94158, United States

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