[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100471485":3},{"organization":4,"armGroups":7,"interventions":19,"overallOfficials":26,"centralContacts":30,"locations":36,"responsibleParty":246,"collaborators":25,"id":248,"slug":249,"hasResults":250,"nctId":251,"briefTitle":252,"officialTitle":253,"acronym":254,"eligibilityCriteria":255,"healthyVolunteers":250,"sex":256,"minAge":257,"maxAge":258,"enrollmentInfo":259,"targetDuration":25,"studyType":262,"phases":263,"briefSummary":266,"conditions":267,"keywords":269,"overallStatus":39,"whyStopped":25,"lastUpdateSubmitDate":276,"lastUpdatePostDateStruct":277,"startDateStruct":280,"completionDateStruct":282,"leadSponsor":284,"locationsCount":285},{"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",[37,58,73,92,109,125,140,155,170,185,202,217,233],{"facility":38,"status":39,"city":40,"state":41,"zip":42,"country":43,"countryCode":44,"cosmosGeoPoint":45,"geoPoint":50,"contacts":51},"UCSF Benioff Children's Hospitals","RECRUITING","San Francisco","California","94158","United States","US",{"type":46,"coordinates":47},"Point",[48,49],-122.41942,37.77493,{"lat":49,"lon":48},[52,55],{"name":53,"role":33,"phone":25,"phoneExt":25,"email":54},"Adam Numis, MD","Adam.numis@ucsf.edu",{"name":56,"role":57,"phone":25,"phoneExt":25,"email":25},"Adam Numis","PRINCIPAL_INVESTIGATOR",{"facility":59,"status":39,"city":60,"state":61,"zip":62,"country":43,"countryCode":44,"cosmosGeoPoint":63,"geoPoint":67,"contacts":68},"Colorado Children's Hospital","Aurora","Colorado","80045",{"type":46,"coordinates":64},[65,66],-104.83192,39.72943,{"lat":66,"lon":65},[69],{"name":70,"role":33,"phone":71,"phoneExt":25,"email":72},"Kallie Dock","720-777-5322","Kallie.dock@childrenscolorado.org",{"facility":74,"status":39,"city":75,"state":76,"zip":77,"country":43,"countryCode":44,"cosmosGeoPoint":78,"geoPoint":82,"contacts":83},"Nicklaus Children's Hospital","Miami","Florida","33155",{"type":46,"coordinates":79},[80,81],-80.19366,25.77427,{"lat":81,"lon":80},[84,88],{"name":85,"role":33,"phone":86,"phoneExt":25,"email":87},"Matt Lallas","305-662-8330","Matt.Lallas@nicklaushealth.org",{"name":89,"role":33,"phone":90,"phoneExt":25,"email":91},"Dainelys Pena Rodriguez","786-624-3547","Dainelys.PenaRodriguez@nicklaushealth.org",{"facility":93,"status":39,"city":94,"state":95,"zip":96,"country":43,"countryCode":44,"cosmosGeoPoint":97,"geoPoint":101,"contacts":102},"Ann & Robert H. Lurie Children's Hospital of Chicago","Chicago","Illinois","60611",{"type":46,"coordinates":98},[99,100],-87.65005,41.85003,{"lat":100,"lon":99},[103,107],{"name":104,"role":33,"phone":105,"phoneExt":25,"email":106},"Enrique Rojas","312-227-2532","erojas@luriechildrens.org",{"name":108,"role":57,"phone":25,"phoneExt":25,"email":25},"Linda Laux",{"facility":110,"status":111,"city":112,"state":113,"zip":114,"country":43,"countryCode":44,"cosmosGeoPoint":115,"geoPoint":119,"contacts":120},"Boston Children's Hospital","NOT_YET_RECRUITING","Boston","Massachusetts","02115",{"type":46,"coordinates":116},[117,118],-71.05977,42.35843,{"lat":118,"lon":117},[121],{"name":122,"role":33,"phone":123,"phoneExt":25,"email":124},"Melissa DiBacco","617-919-4617","Melissa.DiBacco@childrens.harvard.edu",{"facility":126,"status":39,"city":127,"state":128,"zip":129,"country":43,"countryCode":44,"cosmosGeoPoint":130,"geoPoint":134,"contacts":135},"Mott Children's Hospital","Ann Arbor","Michigan","48109",{"type":46,"coordinates":131},[132,133],-83.74088,42.27756,{"lat":133,"lon":132},[136],{"name":137,"role":33,"phone":138,"phoneExt":25,"email":139},"Stephanie Rau","734-232-8474","shatchew@med.umich.edu",{"facility":141,"status":39,"city":142,"state":143,"zip":144,"country":43,"countryCode":44,"cosmosGeoPoint":145,"geoPoint":149,"contacts":150},"Mayo Clinic","Rochester","Minnesota","55905",{"type":46,"coordinates":146},[147,148],-92.4699,44.02163,{"lat":148,"lon":147},[151],{"name":152,"role":33,"phone":153,"phoneExt":25,"email":154},"Bridget Neja","507-266-9150","neja.bridget@mayo.edu",{"facility":156,"status":111,"city":157,"state":158,"zip":159,"country":43,"countryCode":44,"cosmosGeoPoint":160,"geoPoint":164,"contacts":165},"Nationwide Children's Hospital","Columbus","Ohio","43205",{"type":46,"coordinates":161},[162,163],-82.99879,39.96118,{"lat":163,"lon":162},[166],{"name":167,"role":33,"phone":168,"phoneExt":25,"email":169},"Kaitlyn Brown","614-722-2286","Kaitlyn.brown2@nationwidechildrens.org",{"facility":171,"status":39,"city":172,"state":173,"zip":174,"country":43,"countryCode":44,"cosmosGeoPoint":175,"geoPoint":179,"contacts":180},"Oregon Health and Science University (OSHU)","Portland","Oregon","97239",{"type":46,"coordinates":176},[177,178],-122.67621,45.52345,{"lat":178,"lon":177},[181],{"name":182,"role":33,"phone":25,"phoneExt":183,"email":184},"Emily Stein","503-418-4349","steine@ohsu.edu",{"facility":186,"status":39,"city":187,"state":188,"zip":189,"country":43,"countryCode":44,"cosmosGeoPoint":190,"geoPoint":194,"contacts":195},"Cook Children's Medical Center","Fort Worth","Texas","76104",{"type":46,"coordinates":191},[192,193],-97.32085,32.72541,{"lat":193,"lon":192},[196,200],{"name":197,"role":33,"phone":198,"phoneExt":25,"email":199},"Dianna Grado, RN, CCRC","(682) 885-2844","Dianna.Grado@cookchildrens.org",{"name":201,"role":57,"phone":25,"phoneExt":25,"email":25},"Scott Perry",{"facility":203,"status":39,"city":204,"state":25,"zip":25,"country":205,"countryCode":206,"cosmosGeoPoint":207,"geoPoint":211,"contacts":212},"The Royal Children's Hospital","Melbourne","Australia","AU",{"type":46,"coordinates":208},[209,210],144.96332,-37.814,{"lat":210,"lon":209},[213,215],{"name":214,"role":33,"phone":25,"phoneExt":25,"email":25},"Prof. Ingrid Scheffer",{"name":216,"role":33,"phone":25,"phoneExt":25,"email":25},"Dr. Katherine Howell",{"facility":218,"status":39,"city":219,"state":25,"zip":220,"country":221,"countryCode":222,"cosmosGeoPoint":223,"geoPoint":227,"contacts":228},"Queen Elizabeth Hospital","Glasgow","G51 4TF","United Kingdom","UK",{"type":46,"coordinates":224},[225,226],-4.25763,55.86515,{"lat":226,"lon":225},[229],{"name":230,"role":33,"phone":231,"phoneExt":25,"email":232},"Patricia Clark","0141 232 7600","Patricia.Clark2@ggc.scot.nhs.uk",{"facility":234,"status":111,"city":235,"state":25,"zip":236,"country":221,"countryCode":222,"cosmosGeoPoint":237,"geoPoint":241,"contacts":242},"Great Ormond Street Hospital","London","WC1N3JH",{"type":46,"coordinates":238},[239,240],-0.12574,51.50853,{"lat":240,"lon":239},[243],{"name":244,"role":33,"phone":25,"phoneExt":25,"email":245},"Helen Cross, MD","helen.cross10@nhs.net",{"type":247,"investigatorFullName":25,"investigatorTitle":25,"investigatorAffiliation":25,"oldNameTitle":25,"oldOrganization":25},"SPONSOR","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":260,"type":261},47,"ESTIMATED","INTERVENTIONAL",[264,265],"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.",[268],"Dravet Syndrome",[270,271,272,273,268,274,275],"Dravet","SCN1A","DEE","developmental and epileptic encephalopathy","SCN1A-positive","SCN1A+","2026-06-26",{"date":278,"type":279},"2026-06-29","ACTUAL",{"date":281,"type":279},"2024-05-14",{"date":283,"type":261},"2033-01",{"name":5,"class":6},13]