NCT05419492

A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 47 (estimated)
• Sponsor: Encoded Therapeutics · Industry
• Sex: All
• Age: 6 Months – 17 Years
• Healthy volunteers: Not accepted

Summary

ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to \<36 months (Part 1A), aged ≥48 months to \<18 years (Part 1B), and aged ≥6 to \<48 months (Part 2). Part 1A follows an open-label, dose-escalation design, Part 1B follows an open-label design, and Part 2 is a randomized, double-blind, sham delayed-treatment control study.

Conditions

• Dravet Syndrome

Interventions

Type	Name	Description
DRUG	ETX101	ETX101 is a non-replicating, recombinant adeno-associated viral vector serotype 9 (rAAV9) comprising a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A). ETX101 is intended as a one-time intracerebroventricular (ICV) administration.

Timeline

• Start date: 2024-05-14
• Primary completion: 2028-01-01
• Completion: 2033-01-01
• First posted: 2022-06-15
• Last updated: 2026-04-16

Locations

10 sites across 3 countries: United States, Australia, United Kingdom

Regulatory

• FDA-regulated drug study