clinical trial · NCT06283212
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome
Dravet SyndromeETX101
brief summary
EXPEDITION is a Phase 1/2 study in the UK to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet Syndrome aged 6 to \< 48 months. The study follows and open-label, dose-escalation design.
started
May 9, 2024
primary completion
Oct 1, 2030
completion
Oct 1, 2030
last updated
Dec 12, 2025
official title
EXPEDITION: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol