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

Dravet Syndrome Clinical Trial

Official title:
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

Clinical Trial 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 1 follows an open-label, dose-escalation design, and Part 2 is a randomized, double-blind, sham delayed-treatment control, dose-selection study.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05419492
Study type Interventional
Source Encoded Therapeutics
Contact Encoded Patient Advocacy
Phone +1 (650) 398-4301
Email patientadvocacy@encoded.com
Status Recruiting
Phase Phase 1/Phase 2
Start date April 2024
Completion date April 2031
View Details
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