Gene therapy trial offers hope for children with devastating seizure disorder
NCT ID NCT06283212
Summary
This early-stage study is testing a one-time gene therapy called ETX101 in young children (6 months to 4 years old) with a severe form of genetic epilepsy called Dravet Syndrome. The goal is to see if delivering a corrected gene directly to the brain is safe and can help reduce seizures and improve development. The study is small, enrolling only 5 children in the UK, and will carefully monitor them for a year.
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Contacts and locations
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Locations
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Great Ormond Street Hospital
London, WC1N3JH, United Kingdom
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Queen Elizabeth Hospital
Glasgow, G51 4TF, United Kingdom
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Sheffield Children's Hospital
Sheffield, S10 2TH, United Kingdom
Conditions
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