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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: 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
Who can join
6 Months – 17 Years · All sexes
Full eligibility criteria
Inclusion Criteria: * Participant must be aged between ≥6 months and \<36 months in Part 1A, ≥48 months and \<18 years in Part 1B, ≥6 months and \<48 months in Part 2. * Participant must have a predicted loss of function pathogenic or likely pathogenic SCN1A variant. * Participant must have experienced their first seizure between the ages of 3 and 15 months. * Participant must have a clinical diagnosis of Dravet syndrome or the treating clinician must have a high clinical suspicion of a diagnosis of Dravet syndrome. * Participant is receiving at least one prophylactic antiseizure medication. Exclusion Criteria: * Participant has another genetic mutation or clinical comorbidity which could potentially confound the typical Dravet phenotype. * Participant has a known central nervous system structural and/or vascular abnormality (indicated by an MRI or CT scan of the brain). * Participant has an abnormality that may interfere with CSF distribution and/or has an existing ventriculoperitoneal shunt. * Participant has received sodium channel blockers during the Pre-Dosing Seizure Period. * Participant has experienced seizure freedom for a period of 4 consecutive weeks within the 90-day period prior to informed consent. * Participant has previously received gene or cell therapy. * Participant is currently enrolled in a clinical trial or receiving an investigational therapy. * Participant has clinically significant underlying liver disease.
About the study
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.
What is being tested
- ETX101 (drug)
Sponsor: Encoded Therapeutics · Participants: 47 · Started: May 14, 2024
Contact the study team
- Encoded Patient Advocacy · Phone: +1 (650) 398-4301
Official record on ClinicalTrials.gov — NCT05419492
Locations in the U.S.
Conditions
From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.