[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100471485":3},{"organization":4,"armGroups":7,"interventions":19,"overallOfficials":26,"centralContacts":30,"locations":36,"responsibleParty":243,"collaborators":25,"id":245,"slug":25,"hasResults":246,"nctId":247,"briefTitle":248,"officialTitle":249,"acronym":250,"eligibilityCriteria":251,"healthyVolunteers":246,"sex":252,"minAge":253,"maxAge":254,"enrollmentInfo":255,"targetDuration":25,"studyType":258,"phases":259,"briefSummary":262,"conditions":263,"keywords":265,"overallStatus":39,"whyStopped":25,"lastUpdateSubmitDate":272,"lastUpdatePostDateStruct":273,"startDateStruct":276,"completionDateStruct":278,"leadSponsor":280,"locationsCount":281},{"fullName":5,"class":6},"Encoded Therapeutics","INDUSTRY",[8,14],{"label":9,"type":10,"description":11,"interventionNames":12},"Part 1 (US Only)","EXPERIMENTAL","Part 1A will follow an open-label, rule-based, dose-escalation design and will evaluate up to 4 dose levels of ETX101. Part 1B will follow an open-label design and will evaluate 1 dose level of ETX101.",[13],"Drug: ETX101",{"label":15,"type":16,"description":17,"interventionNames":18},"Part 2","SHAM_COMPARATOR","Part 2 will follow a double-blind (up through Week 52), randomized, sham delayed-treatment control design.\n\nThere will be 2 cohorts in Part 2. A single dose level of ETX101 will be evaluated in Part 2 and participants will be randomized 2:1 to study treatment or sham procedure with delayed treatment.",[13],[20],{"type":21,"name":22,"description":23,"armGroupLabels":24,"otherNames":25},"DRUG","ETX101","ETX101 is a non-replicating, recombinant adeno-associated viral vector serotype 9 (rAAV9) comprising a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A). ETX101 is intended as a one-time intracerebroventricular (ICV) administration.",[9,15],null,[27],{"name":28,"affiliation":5,"role":29},"Salvador Rico, M.D., Ph.D","STUDY_DIRECTOR",[31],{"name":32,"role":33,"phone":34,"phoneExt":25,"email":35},"Encoded Patient Advocacy","CONTACT","+1 (650) 398-4301","patientadvocacy@encoded.com",[37,57,72,91,108,124,139,154,169,184,201,215,230],{"facility":38,"status":39,"city":40,"state":41,"zip":42,"country":43,"cosmosGeoPoint":44,"geoPoint":49,"contacts":50},"UCSF Benioff Children's Hospitals","RECRUITING","San Francisco","California","94158","United States",{"type":45,"coordinates":46},"Point",[47,48],-122.41942,37.77493,{"lat":48,"lon":47},[51,54],{"name":52,"role":33,"phone":25,"phoneExt":25,"email":53},"Adam Numis, MD","Adam.numis@ucsf.edu",{"name":55,"role":56,"phone":25,"phoneExt":25,"email":25},"Adam Numis","PRINCIPAL_INVESTIGATOR",{"facility":58,"status":39,"city":59,"state":60,"zip":61,"country":43,"cosmosGeoPoint":62,"geoPoint":66,"contacts":67},"Colorado Children's Hospital","Aurora","Colorado","80045",{"type":45,"coordinates":63},[64,65],-104.83192,39.72943,{"lat":65,"lon":64},[68],{"name":69,"role":33,"phone":70,"phoneExt":25,"email":71},"Kallie Dock","720-777-5322","Kallie.dock@childrenscolorado.org",{"facility":73,"status":39,"city":74,"state":75,"zip":76,"country":43,"cosmosGeoPoint":77,"geoPoint":81,"contacts":82},"Nicklaus Children's Hospital","Miami","Florida","33155",{"type":45,"coordinates":78},[79,80],-80.19366,25.77427,{"lat":80,"lon":79},[83,87],{"name":84,"role":33,"phone":85,"phoneExt":25,"email":86},"Matt Lallas","305-662-8330","Matt.Lallas@nicklaushealth.org",{"name":88,"role":33,"phone":89,"phoneExt":25,"email":90},"Dainelys Pena Rodriguez","786-624-3547","Dainelys.PenaRodriguez@nicklaushealth.org",{"facility":92,"status":39,"city":93,"state":94,"zip":95,"country":43,"cosmosGeoPoint":96,"geoPoint":100,"contacts":101},"Ann & Robert H. Lurie Children's Hospital of Chicago","Chicago","Illinois","60611",{"type":45,"coordinates":97},[98,99],-87.65005,41.85003,{"lat":99,"lon":98},[102,106],{"name":103,"role":33,"phone":104,"phoneExt":25,"email":105},"Enrique Rojas","312-227-2532","erojas@luriechildrens.org",{"name":107,"role":56,"phone":25,"phoneExt":25,"email":25},"Linda Laux",{"facility":109,"status":110,"city":111,"state":112,"zip":113,"country":43,"cosmosGeoPoint":114,"geoPoint":118,"contacts":119},"Boston Children's Hospital","NOT_YET_RECRUITING","Boston","Massachusetts","02115",{"type":45,"coordinates":115},[116,117],-71.05977,42.35843,{"lat":117,"lon":116},[120],{"name":121,"role":33,"phone":122,"phoneExt":25,"email":123},"Melissa DiBacco","617-919-4617","Melissa.DiBacco@childrens.harvard.edu",{"facility":125,"status":39,"city":126,"state":127,"zip":128,"country":43,"cosmosGeoPoint":129,"geoPoint":133,"contacts":134},"Mott Children's Hospital","Ann Arbor","Michigan","48109",{"type":45,"coordinates":130},[131,132],-83.74088,42.27756,{"lat":132,"lon":131},[135],{"name":136,"role":33,"phone":137,"phoneExt":25,"email":138},"Stephanie Rau","734-232-8474","shatchew@med.umich.edu",{"facility":140,"status":39,"city":141,"state":142,"zip":143,"country":43,"cosmosGeoPoint":144,"geoPoint":148,"contacts":149},"Mayo Clinic","Rochester","Minnesota","55905",{"type":45,"coordinates":145},[146,147],-92.4699,44.02163,{"lat":147,"lon":146},[150],{"name":151,"role":33,"phone":152,"phoneExt":25,"email":153},"Bridget Neja","507-266-9150","neja.bridget@mayo.edu",{"facility":155,"status":110,"city":156,"state":157,"zip":158,"country":43,"cosmosGeoPoint":159,"geoPoint":163,"contacts":164},"Nationwide Children's Hospital","Columbus","Ohio","43205",{"type":45,"coordinates":160},[161,162],-82.99879,39.96118,{"lat":162,"lon":161},[165],{"name":166,"role":33,"phone":167,"phoneExt":25,"email":168},"Kaitlyn Brown","614-722-2286","Kaitlyn.brown2@nationwidechildrens.org",{"facility":170,"status":39,"city":171,"state":172,"zip":173,"country":43,"cosmosGeoPoint":174,"geoPoint":178,"contacts":179},"Oregon Health and Science University (OSHU)","Portland","Oregon","97239",{"type":45,"coordinates":175},[176,177],-122.67621,45.52345,{"lat":177,"lon":176},[180],{"name":181,"role":33,"phone":25,"phoneExt":182,"email":183},"Emily Stein","503-418-4349","steine@ohsu.edu",{"facility":185,"status":39,"city":186,"state":187,"zip":188,"country":43,"cosmosGeoPoint":189,"geoPoint":193,"contacts":194},"Cook Children's Medical Center","Fort Worth","Texas","76104",{"type":45,"coordinates":190},[191,192],-97.32085,32.72541,{"lat":192,"lon":191},[195,199],{"name":196,"role":33,"phone":197,"phoneExt":25,"email":198},"Dianna Grado, RN, CCRC","(682) 885-2844","Dianna.Grado@cookchildrens.org",{"name":200,"role":56,"phone":25,"phoneExt":25,"email":25},"Scott Perry",{"facility":202,"status":39,"city":203,"state":25,"zip":25,"country":204,"cosmosGeoPoint":205,"geoPoint":209,"contacts":210},"The Royal Children's Hospital","Melbourne","Australia",{"type":45,"coordinates":206},[207,208],144.96332,-37.814,{"lat":208,"lon":207},[211,213],{"name":212,"role":33,"phone":25,"phoneExt":25,"email":25},"Prof. Ingrid Scheffer",{"name":214,"role":33,"phone":25,"phoneExt":25,"email":25},"Dr. Katherine Howell",{"facility":216,"status":39,"city":217,"state":25,"zip":218,"country":219,"cosmosGeoPoint":220,"geoPoint":224,"contacts":225},"Queen Elizabeth Hospital","Glasgow","G51 4TF","United Kingdom",{"type":45,"coordinates":221},[222,223],-4.25763,55.86515,{"lat":223,"lon":222},[226],{"name":227,"role":33,"phone":228,"phoneExt":25,"email":229},"Patricia Clark","0141 232 7600","Patricia.Clark2@ggc.scot.nhs.uk",{"facility":231,"status":110,"city":232,"state":25,"zip":233,"country":219,"cosmosGeoPoint":234,"geoPoint":238,"contacts":239},"Great Ormond Street Hospital","London","WC1N3JH",{"type":45,"coordinates":235},[236,237],-0.12574,51.50853,{"lat":237,"lon":236},[240],{"name":241,"role":33,"phone":25,"phoneExt":25,"email":242},"Helen Cross, MD","helen.cross10@nhs.net",{"type":244,"investigatorFullName":25,"investigatorTitle":25,"investigatorAffiliation":25,"oldNameTitle":25,"oldOrganization":25},"SPONSOR","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":256,"type":257},47,"ESTIMATED","INTERVENTIONAL",[260,261],"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.",[264],"Dravet Syndrome",[266,267,268,269,264,270,271],"Dravet","SCN1A","DEE","developmental and epileptic encephalopathy","SCN1A-positive","SCN1A+","2026-06-26",{"date":274,"type":275},"2026-06-29","ACTUAL",{"date":277,"type":275},"2024-05-14",{"date":279,"type":257},"2033-01",{"name":5,"class":6},13]