Overview

A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome

Status:
RECRUITING
Trial end date:
2030-09-01
Target enrollment:
Participant gender:
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.
Phase:
PHASE1
Details
Lead Sponsor:
Encoded Therapeutics