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)

Phase 1/2 Active not recruiting 4 enrolled Encoded Therapeutics

Non-randomizedSequentialOpen-labelTreatment

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.

Timeline

Start: 2024-02-28
Primary completion: 2030-09
Completion: 2030-09

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	ETX101	Gene therapy (AAV / viral vector)	—	—

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)

Summary

Timeline

Drugs

Indications