[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Encoded Therapeutics\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":50},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":27,"conditions":28,"keywords":30,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":38,"lastUpdatePostDateStruct":39,"startDateStruct":42,"completionDateStruct":44,"leadSponsor":46,"locationsCount":49},"100471485","phase-1-a-clinical-study-to-evaluate-the-safety-and-efficacy-of-etx101-in-infants-and-children-with-scn1a-positive-dravet-syndrome-100471485",false,"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","ENDEAVOR","Inclusion Criteria:\n\n* Participant must be aged between ≥6 months and \\\u003C36 months in Part 1A, ≥48 months and \\\u003C18 years in Part 1B, ≥6 months and \\\u003C48 months in Part 2.\n* Participant must have a predicted loss of function pathogenic or likely pathogenic SCN1A variant.\n* Participant must have experienced their first seizure between the ages of 3 and 15 months.\n* 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.\n* Participant is receiving at least one prophylactic antiseizure medication.\n\nExclusion Criteria:\n\n* Participant has another genetic mutation or clinical comorbidity which could potentially confound the typical Dravet phenotype.\n* Participant has a known central nervous system structural and\u002For vascular abnormality (indicated by an MRI or CT scan of the brain).\n* Participant has an abnormality that may interfere with CSF distribution and\u002For has an existing ventriculoperitoneal shunt.\n* Participant has received sodium channel blockers during the Pre-Dosing Seizure Period.\n* Participant has experienced seizure freedom for a period of 4 consecutive weeks within the 90-day period prior to informed consent.\n* Participant has previously received gene or cell therapy.\n* Participant is currently enrolled in a clinical trial or receiving an investigational therapy.\n* Participant has clinically significant underlying liver disease.","ALL","6 Months","17 Years",{"count":21,"type":22},47,"ESTIMATED","INTERVENTIONAL",[25,26],"PHASE1","PHASE2","ENDEAVOR is a Phase 1\u002F2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to \\\u003C36 months (Part 1A), aged ≥48 months to \\\u003C18 years (Part 1B), and aged ≥6 to \\\u003C48 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.",[29],"Dravet Syndrome",[31,32,33,34,29,35,36],"Dravet","SCN1A","DEE","developmental and epileptic encephalopathy","SCN1A-positive","SCN1A+","RECRUITING","2026-06-26",{"date":40,"type":41},"2026-06-29","ACTUAL",{"date":43,"type":41},"2024-05-14",{"date":45,"type":22},"2033-01",{"name":47,"class":48},"Encoded Therapeutics","INDUSTRY",13,""]