[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT06415344":3,"trial-entities:NCT06415344":261,"trial-summary:NCT06415344":264},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":20,"study_type":21,"primary_purpose":22,"phases":23,"enrollment_info":25,"interventions":28,"primary_outcomes":36,"secondary_outcomes":40,"sex":47,"minimum_age":48,"maximum_age":48,"healthy_volunteers":49,"eligibility_criteria":50,"std_ages":61,"locations":65,"central_contacts":244,"overall_officials":245,"references":250,"see_also_links":251},"NCT06415344","GTX-102-CL302","Long-term Extension of GTX-102 in Angelman Syndrome","A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome","ENROLLING_BY_INVITATION","2029-02","2026-07","2026-07-27","2024-07-31","Ultragenyx Pharmaceutical Inc","INDUSTRY",true,"The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)","This study is a phase 3, long term extension (LTE) which is open to participants rolling over from a prior GTX-102 clinical study. The LTE study will evaluate the long-term safety and efficacy of GTX-102 in participants with AS.",[19],"Angelman Syndrome",[],"INTERVENTIONAL","TREATMENT",[24],"PHASE3",{"count":26,"type":27},255,"ESTIMATED",[29],{"type":30,"name":31,"description":32,"armGroupLabels":33,"otherNames":34},"DRUG","GTX-102","Antisense Oligonucleotide",[31],[35],"apazunersen",[37],{"measure":38,"timeFrame":39},"Incidence of Treatment Emergent Adverse Events (TEAEs)","Up to 5 Years",[41,44],{"measure":42,"timeFrame":43},"Change from Baseline in the Bayley-4 Raw Score","Baseline, Up to 5 Years",{"measure":45,"timeFrame":46},"Net Response in Multidomain Responder Index (MDRI)","Month 12, Month 24","ALL",null,false,{"inclusion":51,"exclusion":55,"raw_text":60},[52,53,54],"Signed informed consent from parent(s) or legal guardian(s).","Completed a final study visit in a prior clinical trial with GTX-102. If the subject has an ongoing adverse event, medical monitor approval is required to rollover. The Screening visit must occur within 6 months of the last visit in the prior GTX-102 trial.","From the time of informed consent through the end of the study and for 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.",[56,57,58,59],"Discontinued from study or withdrew from study treatment in a prior GTX-102 trial.","History or evidence of any other medical, neurological, or psychological condition that would expose the subject to an undue risk of a significant AE or interfere with study assessments during the course of the trial as determined by the clinical judgment of the Investigator.","Known hypersensitivity to GTX-102 or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects.","Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study","Inclusion Criteria:\n\n* Signed informed consent from parent(s) or legal guardian(s).\n* Completed a final study visit in a prior clinical trial with GTX-102. If the subject has an ongoing adverse event, medical monitor approval is required to rollover. The Screening visit must occur within 6 months of the last visit in the prior GTX-102 trial.\n* From the time of informed consent through the end of the study and for 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\n* Discontinued from study or withdrew from study treatment in a prior GTX-102 trial.\n* History or evidence of any other medical, neurological, or psychological condition that would expose the subject to an undue risk of a significant AE or interfere with study assessments during the course of the trial as determined by the clinical judgment of the Investigator.\n* Known hypersensitivity to GTX-102 or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects.\n* Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study",[62,63,64],"CHILD","ADULT","OLDER_ADULT",[66,75,82,90,98,106,113,122,130,139,147,155,162,170,178,185,193,200,208,216,223,231,237],{"facility":67,"city":68,"state":69,"zip":70,"country":71,"geoPoint":72},"University of California, Los Angeles (UCLA)","Los Angeles","California","90095","United States",{"lat":73,"lon":74},34.05223,-118.24368,{"facility":76,"city":77,"state":69,"zip":78,"country":71,"geoPoint":79},"University of California, San Diego - Rady Children's Hospital","San Diego","92123",{"lat":80,"lon":81},32.71571,-117.16472,{"facility":83,"city":84,"state":85,"zip":86,"country":71,"geoPoint":87},"Rare Disease Research, LLC","Atlanta","Georgia","30329",{"lat":88,"lon":89},33.749,-84.38798,{"facility":91,"city":92,"state":93,"zip":94,"country":71,"geoPoint":95},"Rush University Medical Center","Chicago","Illinois","60612",{"lat":96,"lon":97},41.85003,-87.65005,{"facility":99,"city":100,"state":101,"zip":102,"country":71,"geoPoint":103},"Boston Children's Hospital","Boston","Massachusetts","02115",{"lat":104,"lon":105},42.35843,-71.05977,{"facility":107,"city":108,"state":108,"zip":109,"country":71,"geoPoint":110},"Weill Cornell Medicine","New York","10021",{"lat":111,"lon":112},40.71427,-74.00597,{"facility":114,"city":115,"state":116,"zip":117,"country":118,"geoPoint":119},"Queensland Children's Hospital","South Brisbane","Queensland","4101","Australia",{"lat":120,"lon":121},-27.48034,153.02049,{"facility":123,"city":124,"state":125,"zip":126,"country":118,"geoPoint":127},"The Royal Children's Hospital","Melbourne","Victoria","3052",{"lat":128,"lon":129},-37.814,144.96332,{"facility":131,"city":132,"state":133,"zip":134,"country":135,"geoPoint":136},"MAGIC Clinic Ltd","Calgary","Alberta","T2E 7Z4","Canada",{"lat":137,"lon":138},51.05011,-114.08529,{"facility":140,"city":141,"state":142,"zip":143,"country":135,"geoPoint":144},"British Columbia Children's Hospital","Vancouver","British Columbia","V6H 3V4",{"lat":145,"lon":146},49.24966,-123.11934,{"facility":148,"city":149,"state":150,"zip":151,"country":135,"geoPoint":152},"Childrens Hospital London Health Sciences Centre","London","Ontario","N6A 5W9",{"lat":153,"lon":154},42.98339,-81.23304,{"facility":156,"city":157,"state":150,"zip":158,"country":135,"geoPoint":159},"Childrens Hospital of Eastern Ontario","Ottawa","K1H 8L1",{"lat":160,"lon":161},45.41117,-75.69812,{"facility":163,"city":164,"state":165,"zip":166,"country":135,"geoPoint":167},"The Research Institute of the McGill University Health Centre","Montreal","Quebec","H3G 1A4",{"lat":168,"lon":169},45.50884,-73.58781,{"facility":171,"city":172,"zip":173,"country":174,"geoPoint":175},"AP-HM - Hopital de la Timone","Marseille","13385","France",{"lat":176,"lon":177},43.29695,5.38107,{"facility":179,"city":180,"zip":181,"country":174,"geoPoint":182},"AP-HP Hopital Necker-Enfants Malades","Paris","75015",{"lat":183,"lon":184},48.85341,2.3488,{"facility":186,"city":187,"zip":188,"country":189,"geoPoint":190},"Universitaetsklinikum Hamburg-Eppendorf","Hamburg","20246","Germany",{"lat":191,"lon":192},53.55073,9.99302,{"facility":194,"city":195,"zip":196,"country":189,"geoPoint":197},"Universitaetsklinikum Leipzig","Leipzig","04103",{"lat":198,"lon":199},51.33962,12.37129,{"facility":201,"city":202,"zip":203,"country":204,"geoPoint":205},"Sheba Medical Center","Ramat Gan","5265601","Israel",{"lat":206,"lon":207},32.08227,34.81065,{"facility":209,"city":210,"zip":211,"country":212,"geoPoint":213},"Hospital Universitario Puerta de Hierro","Majadahonda","28222","Spain",{"lat":214,"lon":215},40.47353,-3.87182,{"facility":217,"city":218,"zip":219,"country":212,"geoPoint":220},"Corporacio Sanitaria Parc Tauli - Hospital de Sabadell","Sabadell","08208",{"lat":221,"lon":222},41.54329,2.10942,{"facility":224,"city":225,"zip":226,"country":227,"geoPoint":228},"University of Cambridge","Cambridge","CB2 0QQ","United Kingdom",{"lat":229,"lon":230},52.2,0.11667,{"facility":232,"city":149,"zip":233,"country":227,"geoPoint":234},"Great Ormond Street Hospital for Children","WC1N 3JH",{"lat":235,"lon":236},51.50853,-0.12574,{"facility":238,"city":239,"zip":240,"country":227,"geoPoint":241},"Oxford University Hospitals NHS Foundation Trust","Oxford","OX3 9DU",{"lat":242,"lon":243},51.75222,-1.25596,[],[246],{"name":247,"affiliation":248,"role":249},"Medical Director","Ultragenyx Pharmaceuticals Inc.","STUDY_DIRECTOR",[],[252,255,258],{"label":253,"url":254},"Ultragenyx Patient Advocacy Website","https:\u002F\u002Fwww.ultrarareadvocacy.com\u002Fconditions-we-study\u002Fangelman-syndrome-as\u002F",{"label":256,"url":257},"Ultragenyx Transparency Commitment","https:\u002F\u002Fwww.ultragenyx.com\u002Four-research\u002Four-clinical-trial-transparency-commitment\u002F",{"label":259,"url":260},"Ultragenyx Angelman Syndrome (AS) Research Study Opportunity","https:\u002F\u002Fwww.ultraclinicaltrials.com\u002Fangelman-syndrome-as\u002F",{"nct_id":4,"conditions":262,"biomarkers":263},[19],[],{"nct_id":4,"found":15,"summary":265,"prompt_version":275},{"design":266,"status":267,"heading":268,"summary":269,"follow_up":270,"word_count":271,"commitments":272,"compensation":273,"drugs_mentioned":274},"This is a Phase 3, long-term extension study. It is open to participants who have already been in a previous GTX-102 clinical study, with a planned enrollment of 255 people.","completed","Long-term Study of GTX-102 for Angelman Syndrome","This study is looking at the long-term safety and effectiveness of a medicine called GTX-102 for people with Angelman Syndrome (a genetic disorder affecting development). You can join this study if you have already participated in a previous clinical trial involving GTX-102 and completed your final visit. The main goal is to see how safe GTX-102 is over a long period, up to five years. This study is currently open to participants who have been in a prior GTX-102 study.","The study will measure side effects for up to 5 years.",80,"Not specified in the trial record.","Not stated in the trial record.",[31],"v2"]