Status: Recruiting
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
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.
Participants needed: 47
Trial details
Phase: Phase 1, Phase 2Age: 6-17Biological sex: AllType: InterventionalSponsor: Encoded TherapeuticsUpdated: Jun 29, 2026Locations: 13
Eligibility criteria
Participant must be aged between ≥6 months and <36 months in Part 1A, ≥48 months... [+4]
Participant has another genetic mutation or clinical comorbidity which could pot... [+7]