Tests gene therapy ETX101 in young children with Dravet syndrome
Tests ETX101 gene therapy in children with SCN1A-positive Dravet syndrome.
Plain-language title generated from the registry record
Registry title: A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
Key facts
- Where
- Ann Arbor, Aurora, Chicago, Columbus, Durham, Fort Worth, Miami, New York, Portland, Rochester, and San Francisco, United StatesSee the 11 sites
- Phase
- Phase 1/2
Where in United States
- Mott Children's HospitalAnn Arbor, United StatesTaking people
- Colorado Children's HospitalAurora, United StatesTaking people
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, United StatesTaking people
- Nationwide Children's HospitalColumbus, United StatesTaking people
- Duke Children's Hospital & Health CenterDurham, United StatesTaking people
- Cook Children's Medical CenterFort Worth, United StatesTaking people
- Nicklaus Children's HospitalMiami, United StatesTaking people
- NYU Langone HealthNew York, United StatesNot yet recruiting
- Oregon Health and Science university (OHSU)Portland, United StatesTaking people
- Mayo ClinicRochester, United StatesTaking people
- UCSF Benioff Children's HospitalsSan Francisco, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Encoded Therapeutics
- Trial code
- NCT05419492
- Record updated
- 23 October 2026
- Original description
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.Read full original descriptionShow less
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
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.
Inclusion: 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 1 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: 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; Participant has received sodium channel blockers during the Pre-Dosing Seizure Period; 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.