[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100605033":3},{"organization":4,"armGroups":7,"interventions":31,"overallOfficials":45,"centralContacts":49,"locations":58,"responsibleParty":260,"collaborators":37,"id":262,"slug":263,"hasResults":264,"nctId":265,"briefTitle":266,"officialTitle":267,"acronym":268,"eligibilityCriteria":269,"healthyVolunteers":264,"sex":270,"minAge":271,"maxAge":272,"enrollmentInfo":273,"targetDuration":37,"studyType":276,"phases":277,"briefSummary":279,"conditions":280,"keywords":282,"overallStatus":61,"whyStopped":37,"lastUpdateSubmitDate":283,"lastUpdatePostDateStruct":284,"startDateStruct":287,"completionDateStruct":289,"leadSponsor":291,"locationsCount":292},{"fullName":5,"class":6},"Ultragenyx Pharmaceutical Inc","INDUSTRY",[8,14,18,22,26],{"label":9,"type":10,"description":11,"interventionNames":12},"Subprotocol A GTX-102","EXPERIMENTAL","Participants with deletion-type Angelman syndrome, ≥1 to \\\u003C4 years of age will receive increasing doses of GTX-102 via intrathecal (IT) injection until the target dose is achieved. Dosing occurs every 3 months (Q3M) thereafter.",[13],"Drug: GTX-102",{"label":15,"type":10,"description":16,"interventionNames":17},"Subprotocol B GTX-102","Participants with paternal uniparental disomy (UPD)\u002Fimprinting center defect (ICD) Angelman syndrome, ≥4 to \\\u003C18 years of age will receive increasing doses of GTX-102 via IT injection until the target dose is achieved. Dosing occurs Q3M thereafter.",[13],{"label":19,"type":10,"description":20,"interventionNames":21},"Subprotocol C GTX-102","Participants with all genotypes of Angelman syndrome, ≥18 to \\\u003C65 years of age will receive increasing doses of GTX-102 via IT injection until the target dose is achieved. Dosing occurs Q3M thereafter.",[13],{"label":23,"type":10,"description":24,"interventionNames":25},"Subprotocol D GTX-102","Participants with mutation-type Angelman syndrome, ≥4 to \\\u003C18 years of age will receive increasing doses of GTX-102 via IT injection until the target dose is achieved. Dosing occurs Q3M thereafter.",[13],{"label":27,"type":10,"description":28,"interventionNames":29},"Subprotocol D No Intervention then GTX-102","Participants with mutation-type Angelman syndrome, ≥4 to \\\u003C18 years of age will receive no treatment during the initial period. At the end of the no treatment period, participants will receive increasing doses of GTX-102 via IT injection until the target dose is achieved. Dosing occurs Q3M thereafter.",[30,13],"Other: No intervention",[32,38],{"type":33,"name":34,"description":35,"armGroupLabels":36,"otherNames":37},"OTHER","No intervention","During the no treatment period participants do not receive any study drug",[27],null,{"type":39,"name":40,"description":41,"armGroupLabels":42,"otherNames":43},"DRUG","GTX-102","antisense oligonucleotide",[9,15,19,23,27],[44],"apazunersen",[46],{"name":47,"affiliation":5,"role":48},"Medical Director","STUDY_DIRECTOR",[50,55],{"name":51,"role":52,"phone":53,"phoneExt":37,"email":54},"Patients Contact Trial Recruitment","CONTACT","1-888-756-8657","Trialrecruitment@ultragenyx.com",{"name":56,"role":52,"phone":53,"phoneExt":37,"email":57},"HCPs Contact: Medical Information","medinfo@ultragenyx.com",[59,73,83,94,103,113,123,133,142,153,164,172,182,190,200,210,218,225,235,243,252],{"facility":60,"status":61,"city":62,"state":63,"zip":64,"country":65,"countryCode":66,"cosmosGeoPoint":67,"geoPoint":72,"contacts":37},"Cedars Sinai Medical Center","RECRUITING","Los Angeles","California","90048","United States","US",{"type":68,"coordinates":69},"Point",[70,71],-118.24368,34.05223,{"lat":71,"lon":70},{"facility":74,"status":61,"city":75,"state":76,"zip":77,"country":65,"countryCode":66,"cosmosGeoPoint":78,"geoPoint":82,"contacts":37},"Rush University Medical Center","Chicago","Illinois","60612",{"type":68,"coordinates":79},[80,81],-87.65005,41.85003,{"lat":81,"lon":80},{"facility":84,"status":85,"city":86,"state":87,"zip":88,"country":65,"countryCode":66,"cosmosGeoPoint":89,"geoPoint":93,"contacts":37},"Clinical Trial Site","NOT_YET_RECRUITING","Baltimore","Maryland","21205",{"type":68,"coordinates":90},[91,92],-76.61219,39.29038,{"lat":92,"lon":91},{"facility":84,"status":85,"city":95,"state":96,"zip":97,"country":65,"countryCode":66,"cosmosGeoPoint":98,"geoPoint":102,"contacts":37},"Kansas City","Missouri","64108",{"type":68,"coordinates":99},[100,101],-94.57857,39.09973,{"lat":101,"lon":100},{"facility":104,"status":61,"city":105,"state":106,"zip":107,"country":65,"countryCode":66,"cosmosGeoPoint":108,"geoPoint":112,"contacts":37},"Rare Disease Research","Hillsborough","North Carolina","27278",{"type":68,"coordinates":109},[110,111],-79.09973,36.07542,{"lat":111,"lon":110},{"facility":114,"status":61,"city":115,"state":116,"zip":117,"country":65,"countryCode":66,"cosmosGeoPoint":118,"geoPoint":122,"contacts":37},"Akron Children's Hospital","Akron","Ohio","44308",{"type":68,"coordinates":119},[120,121],-81.51901,41.08144,{"lat":121,"lon":120},{"facility":124,"status":61,"city":125,"state":126,"zip":127,"country":65,"countryCode":66,"cosmosGeoPoint":128,"geoPoint":132,"contacts":37},"UT Health Austin","Austin","Texas","78723",{"type":68,"coordinates":129},[130,131],-97.74306,30.26715,{"lat":131,"lon":130},{"facility":134,"status":61,"city":135,"state":126,"zip":136,"country":65,"countryCode":66,"cosmosGeoPoint":137,"geoPoint":141,"contacts":37},"Carum Research Inc.","Dallas","75243",{"type":68,"coordinates":138},[139,140],-96.80667,32.78306,{"lat":140,"lon":139},{"facility":143,"status":61,"city":144,"state":145,"zip":37,"country":146,"countryCode":147,"cosmosGeoPoint":148,"geoPoint":152,"contacts":37},"Hospital Universitario Austral","Pilar","Buenos Aires","Argentina","AR",{"type":68,"coordinates":149},[150,151],-58.91398,-34.45867,{"lat":151,"lon":150},{"facility":154,"status":61,"city":155,"state":156,"zip":37,"country":157,"countryCode":158,"cosmosGeoPoint":159,"geoPoint":163,"contacts":37},"Hospital de Crianças César Pernetta e Hospital Pequeno Príncipe","Curitiba","Paraná","Brazil","BR",{"type":68,"coordinates":160},[161,162],-49.27306,-25.42778,{"lat":162,"lon":161},{"facility":84,"status":85,"city":165,"state":166,"zip":37,"country":157,"countryCode":158,"cosmosGeoPoint":167,"geoPoint":171,"contacts":37},"Santa Cecília","Porto Alegre",{"type":68,"coordinates":168},[169,170],-50.42694,-26.96083,{"lat":170,"lon":169},{"facility":173,"status":61,"city":174,"state":37,"zip":37,"country":175,"countryCode":176,"cosmosGeoPoint":177,"geoPoint":181,"contacts":37},"Hopital de la Timone","Marseille","France","FR",{"type":68,"coordinates":178},[179,180],5.38107,43.29695,{"lat":180,"lon":179},{"facility":183,"status":61,"city":184,"state":37,"zip":37,"country":175,"countryCode":176,"cosmosGeoPoint":185,"geoPoint":189,"contacts":37},"Necker-Enfants Malades Hospital","Paris",{"type":68,"coordinates":186},[187,188],2.3488,48.85341,{"lat":188,"lon":187},{"facility":191,"status":61,"city":192,"state":37,"zip":37,"country":193,"countryCode":194,"cosmosGeoPoint":195,"geoPoint":199,"contacts":37},"Sheba Medical Center","Ramat Gan","Israel","IL",{"type":68,"coordinates":196},[197,198],34.81065,32.08227,{"lat":198,"lon":197},{"facility":201,"status":61,"city":202,"state":37,"zip":37,"country":203,"countryCode":204,"cosmosGeoPoint":205,"geoPoint":209,"contacts":37},"Azienda Ospedaliera Universitaria Meyer IRCCS","Florence","Italy","IT",{"type":68,"coordinates":206},[207,208],11.24626,43.77925,{"lat":208,"lon":207},{"facility":211,"status":61,"city":212,"state":37,"zip":37,"country":203,"countryCode":204,"cosmosGeoPoint":213,"geoPoint":217,"contacts":37},"Fondazione IRCCS Istituto Neurologico C. Besta","Milan",{"type":68,"coordinates":214},[215,216],9.18951,45.46427,{"lat":216,"lon":215},{"facility":84,"status":85,"city":219,"state":37,"zip":37,"country":203,"countryCode":204,"cosmosGeoPoint":220,"geoPoint":224,"contacts":37},"Rome",{"type":68,"coordinates":221},[222,223],12.51133,41.89193,{"lat":223,"lon":222},{"facility":226,"status":61,"city":227,"state":37,"zip":37,"country":228,"countryCode":229,"cosmosGeoPoint":230,"geoPoint":234,"contacts":37},"Hospital de Santa Maria","Lisbon","Portugal","PT",{"type":68,"coordinates":231},[232,233],-9.1498,38.72509,{"lat":233,"lon":232},{"facility":236,"status":61,"city":237,"state":37,"zip":37,"country":228,"countryCode":229,"cosmosGeoPoint":238,"geoPoint":242,"contacts":37},"Hospital Santa Joao","Porto",{"type":68,"coordinates":239},[240,241],-8.61097,41.1485,{"lat":241,"lon":240},{"facility":84,"status":85,"city":244,"state":37,"zip":37,"country":245,"countryCode":246,"cosmosGeoPoint":247,"geoPoint":251,"contacts":37},"London","United Kingdom","UK",{"type":68,"coordinates":248},[249,250],-0.12574,51.50853,{"lat":250,"lon":249},{"facility":253,"status":61,"city":254,"state":37,"zip":37,"country":245,"countryCode":246,"cosmosGeoPoint":255,"geoPoint":259,"contacts":37},"University of Oxford","Oxford",{"type":68,"coordinates":256},[257,258],-1.25596,51.75222,{"lat":258,"lon":257},{"type":261,"investigatorFullName":37,"investigatorTitle":37,"investigatorAffiliation":37,"oldNameTitle":37,"oldOrganization":37},"SPONSOR","100605033","phase-2-a-safety-and-efficacy-study-of-gtx-102-in-subjects-with-deletion--or-nondeletion-type-angelman-syndrome-as-100605033",false,"NCT07157254","A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)","A Phase 2, Open-label, Basket Study Investigating the Safety and Efficacy of GTX-102 in Adult and Pediatric Subjects With Deletion- or Nondeletion-type Angelman Syndrome","Aurora","Inclusion Criteria:\n\n1. Signed informed consent from parent(s) or legal guardian(s)\n2. Males and females of the following ages and genotypes at time of informed consent:\n\n   1. Subprotocol A: ≥ 1 to \\\u003C 4 years of age with a genetically confirmed diagnosis of deletion-type Angelman syndrome\n   2. Subprotocol B: ≥ 4 to \\\u003C 18 years of age with a genetically confirmed diagnosis of UPD\u002FICD Angelman syndrome\n   3. Subprotocol C: ≥ 18 to \\\u003C 65 years of age with a genetically confirmed diagnosis of Angelman syndrome, any genotype\n   4. Subprotocol D: ≥ 4 to \\\u003C 18 years of age with a genetically confirmed diagnosis of mutation-type Angelman syndrome\n3. Weight ≥ 8 kg at Screening Visit\n4. Platelet count, prothrombin time \u002F international normalized ratio, and partial thromboplastin time \\\u003C 1.5x the upper limit of normal and platelets \\> 75,000 cells\u002Fmm3 at the Screening Visit\n5. Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, and all study procedures, including lumbar puncture (LP) procedure, magnetic resonance imaging (MRI) and tolerating anesthesia without intubation\n6. From the time of informed consent through to at least 6 months after the final dose of GTX-102, females of childbearing potential who are sexually active must use highly effective contraception or abstinence. Males are able to participate if they agree to remain abstinent (refrain from heterosexual intercourse) or use acceptable contraceptive methods during the study and for at least 3 months after the final dose of GTX-102\n\nExclusion Criteria:\n\n1. Any change in medications or diet\u002Fsupplements intended to treat symptoms of Angelman Syndrome (eg, sleeping aids, antiseizure medications, supplements, dietary change including ketogenic or low-glycemic index diet, other) within the month prior to the Screening Visit (excluding weight-based adjustments)\n2. Any condition that creates an increased risk of unsuccessful lumbar puncture\n3. Current or expected concomitant use of drugs that increase the risk of bleeding (eg, heparin, low molecular weight heparin, platelet inhibitors)\n4. Known hypersensitivity to GTX-102 or its excipients or required premedication that, in the judgment of the Investigator, places the subject at increased risk for adverse effects\n5. Presence or history of any condition, lab abnormality, or infection that, in the judgment of the Investigator, would interfere with study participation, pose undue safety risk, or would confound interpretation of results\n6. Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study\n7. Use of any investigational product or investigational medical device within 6 months or 5 half-lives prior to the Screening Visit, or any prior use of gene therapy or an ASO regardless of length of time since last use\n8. Concurrent participation in any interventional study","ALL","1 Year","64 Years",{"count":274,"type":275},60,"ESTIMATED","INTERVENTIONAL",[278],"PHASE2","The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.",[281],"Angelman Syndrome",[281],"2026-06-22",{"date":285,"type":286},"2026-06-23","ACTUAL",{"date":288,"type":286},"2025-10-13",{"date":290,"type":275},"2030-01",{"name":5,"class":6},21]