clinical trial · NCT06112275
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (Australia Only)
Dravet SyndromeETX101
brief summary
WAYFINDER is a Phase 1/2 study in Australia to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged 6 to \<84 months. The study follows an open-label, dose-escalation design.
started
Feb 28, 2024
primary completion
Sep 1, 2030
completion
Sep 1, 2030
last updated
Nov 14, 2025
official title
WAYFINDER: 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