Gene therapy trial aims to tame severe seizures in young children
NCT ID NCT05419492
First seen Feb 28, 2026 · Last updated May 10, 2026 · Updated 10 times
Summary
This study tests a gene therapy called ETX101 in infants and 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 will include up to 47 participants 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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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
NOT_YET_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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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
NOT_YET_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: •••••@•••••
Conditions
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