Gene therapy trial aims to tame severe seizures in kids with dravet syndrome
NCT ID NCT06112275
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a gene therapy called ETX101 in 4 children with Dravet syndrome, a severe seizure disorder caused by a gene change. The therapy uses a harmless virus to deliver a working copy of the gene to brain cells. The main goals are to see if it is safe and if it reduces seizures over 52 weeks.
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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
Locations
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The Royal Children's Hospital
Melbourne, Australia
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Other studies related to the condition(s) this trial covers.
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