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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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Boston Children's Hospital
NOT_YET_RECRUITINGBoston, Massachusetts, 02115, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Colorado Children's Hospital
RECRUITINGAurora, Colorado, 80045, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Cook Children's Medical Center
RECRUITINGFort Worth, Texas, 76104, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Great Ormond Street Hospital
NOT_YET_RECRUITINGLondon, WC1N3JH, United Kingdom
Contact Email: •••••@•••••
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Mayo Clinic
RECRUITINGRochester, Minnesota, 55905, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Mott Children's Hospital
RECRUITINGAnn Arbor, Michigan, 48109, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Nationwide Children's Hospital
NOT_YET_RECRUITINGColumbus, Ohio, 43205, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Nicklaus Children's Hospital
RECRUITINGMiami, Florida, 33155, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Oregon Health and Science University (OSHU)
RECRUITINGPortland, Oregon, 97239, United States
Contact Email: •••••@•••••
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Queen Elizabeth Hospital
RECRUITINGGlasgow, G51 4TF, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
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The Royal Children's Hospital
RECRUITINGMelbourne, Australia
Contact
Contact
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UCSF Benioff Children's Hospitals
RECRUITINGSan Francisco, California, 94158, United States
Contact
Contact Email: •••••@•••••