NCT06283212

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

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

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Encoded Therapeutics · Industry
• Sex: All
• Age: 6 Months – 47 Months
• Healthy volunteers: Not accepted

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.

Conditions

• Dravet Syndrome

Interventions

Type	Name	Description
DRUG	ETX101	ETX101 is composed of a non-replicating, recombinant adeno-associated viral serotype 9 (rAAV9) vector used to deliver a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A)

Timeline

• Start date: 2024-05-09
• Primary completion: 2030-10-01
• Completion: 2030-10-01
• First posted: 2024-02-28
• Last updated: 2025-12-12

Locations

3 sites across 1 country: United Kingdom