[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07157254":3,"trial-entities:NCT07157254":262,"trial-summary:NCT07157254":266},{"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":46,"secondary_outcomes":59,"sex":78,"minimum_age":79,"maximum_age":80,"healthy_volunteers":81,"eligibility_criteria":82,"std_ages":86,"locations":89,"central_contacts":241,"overall_officials":250,"references":254,"see_also_links":255},"NCT07157254","GTX-102-CL210","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","RECRUITING","2030-01","2026-08","2026-08-07","2025-10-13","Ultragenyx Pharmaceutical Inc","INDUSTRY",true,"The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.","This basket study is designed to evaluate safety and efficacy of GTX-102 in participants with Angelman syndrome across genotypes and age groups. The study consists of subprotocols A, B, C and D. All subprotocols are open-label and follow the same design which includes a Screening, Loading and Maintenance period. Subprotocols A, B and C are single arm only. In subprotocol D, participants are randomized 2:1 to a GTX-102 group or a No Treatment group. The No Treatment group follows the same schedule of events as all other groups after completion of the No Treatment period. Participants from all the subprotocols have the option to continue treatment in a long-term extension study following their end of study visit.",[19],"Angelman Syndrome",[19],"INTERVENTIONAL","TREATMENT",[24],"PHASE2",{"count":26,"type":27},60,"ESTIMATED",[29,35],{"type":30,"name":31,"description":32,"armGroupLabels":33},"OTHER","No intervention","During the no treatment period participants do not receive any study drug",[34],"Subprotocol D No Intervention then GTX-102",{"type":36,"name":37,"description":38,"armGroupLabels":39,"otherNames":44},"DRUG","GTX-102","antisense oligonucleotide",[40,41,42,43,34],"Subprotocol A GTX-102","Subprotocol B GTX-102","Subprotocol C GTX-102","Subprotocol D GTX-102",[45],"apazunersen",[47,50,53,56],{"measure":48,"timeFrame":49},"Subprotocol A\u002FB\u002FC\u002FD: Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs), Severe Events, and Events Related to Investigational Product, Procedure, and Premedication","Up to Day 506",{"measure":51,"timeFrame":52},"Subprotocol A Only: Bayley-4 Cognitive Without Caregiver Input Raw Score Change from Baseline at Day 338","Baseline, Day 338",{"measure":54,"description":55,"timeFrame":52},"Subprotocol B\u002FD Only: Multidomain Responder Index (MDRI) Net Response at Day 338","The following assessments will be included to calculate the MDRI net response: Bayley-4 Cognitive and Receptive Communication, Aberrant Behavior Checklist- Community (ABC-C) Hyperactivity\u002FNoncompliance (H\u002FN), Angelman Severity Assessment (ASA) Sleep, ASA Gross Motor. For each assessment a meaningful score difference (MSD) is defined. A single net response score per participant will be derived accordingly, and a summary measure of net response will then be calculated across all participants.",{"measure":57,"description":58,"timeFrame":52},"Subprotocol C Only: MDRI Net Response at Day 338","The following assessments will be included to calculate the MDRI net response: Vineland-3 Expressive and Receptive Communication, ABC-C Irritability, ASA Gross Motor. For each assessment a meaningful score difference (MSD) is defined. A single net response score per participant will be derived accordingly, and a summary measure of net response will then be calculated across all participants.",[60,62,64,66,68,70,72,74,76],{"measure":61,"timeFrame":52},"Subprotocol A\u002FB\u002FD Only: Change From Baseline at Day 338 in Bayley-4 Receptive Communication Raw Score",{"measure":63,"timeFrame":52},"Subprotocol A\u002FB\u002FD Only: Change From Baseline at Day 338 in Bayley-4 Gross Motor Raw Score",{"measure":65,"timeFrame":52},"Subprotocol B\u002FD Only: Change From Baseline at Day 338 in Bayley-4 Cognitive Raw Score",{"measure":67,"timeFrame":52},"Subprotocol B\u002FC\u002FD Only: Change From Baseline at Day 338 in Vineland-3 Receptive Communication Raw Score",{"measure":69,"timeFrame":52},"Subprotocol B\u002FC\u002FD Only: Change From Baseline at Day 338 in Vineland-3 Expressive Communication Raw Score",{"measure":71,"timeFrame":52},"Subprotocol B\u002FD: Change From Baseline at Day 338 in Aberrant Behavior Checklist-Community (ABC-C) Hyperactivity\u002FNoncompliance Raw Score",{"measure":73,"timeFrame":52},"Subprotocol B\u002FD Only: Change From Baseline at Day 338 in Angelman Severity Assessment (ASA) Sleep Rating",{"measure":75,"timeFrame":52},"Subprotocol B\u002FC\u002FD Only: Change From Baseline at Day 338 in ASA Gross Motor Rating",{"measure":77,"timeFrame":52},"Subprotocol C Only: Change From Baseline at Day 338 in ABC-C Irritability Raw Score","ALL","1 Year","64 Years",false,{"inclusion":83,"exclusion":84,"raw_text":85},[],[],"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",[87,88],"CHILD","ADULT",[90,99,107,115,124,132,140,148,155,163,171,178,185,191,198,205,211,216,223,229,235],{"facility":91,"status":8,"city":92,"state":93,"zip":94,"country":95,"geoPoint":96},"Cedars Sinai Medical Center","Los Angeles","California","90048","United States",{"lat":97,"lon":98},34.05223,-118.24368,{"facility":100,"status":8,"city":101,"state":102,"zip":103,"country":95,"geoPoint":104},"Rush University Medical Center","Chicago","Illinois","60612",{"lat":105,"lon":106},41.85003,-87.65005,{"facility":108,"status":8,"city":109,"state":110,"zip":111,"country":95,"geoPoint":112},"Kennedy Kreiger Institute","Baltimore","Maryland","21205",{"lat":113,"lon":114},39.29038,-76.61219,{"facility":116,"status":117,"city":118,"state":119,"zip":120,"country":95,"geoPoint":121},"Clinical Trial Site","NOT_YET_RECRUITING","Kansas City","Missouri","64108",{"lat":122,"lon":123},39.09973,-94.57857,{"facility":125,"status":8,"city":126,"state":127,"zip":128,"country":95,"geoPoint":129},"Rare Disease Research","Hillsborough","North Carolina","27278",{"lat":130,"lon":131},36.07542,-79.09973,{"facility":133,"status":8,"city":134,"state":135,"zip":136,"country":95,"geoPoint":137},"Akron Children's Hospital","Akron","Ohio","44308",{"lat":138,"lon":139},41.08144,-81.51901,{"facility":141,"status":8,"city":142,"state":143,"zip":144,"country":95,"geoPoint":145},"UT Health Austin","Austin","Texas","78723",{"lat":146,"lon":147},30.26715,-97.74306,{"facility":149,"status":8,"city":150,"state":143,"zip":151,"country":95,"geoPoint":152},"Carum Research Inc.","Dallas","75243",{"lat":153,"lon":154},32.78306,-96.80667,{"facility":156,"status":8,"city":157,"state":158,"country":159,"geoPoint":160},"Hospital Universitario Austral","Pilar","Buenos Aires","Argentina",{"lat":161,"lon":162},-34.45867,-58.91398,{"facility":164,"status":8,"city":165,"state":166,"country":167,"geoPoint":168},"Hospital de Crianças César Pernetta e Hospital Pequeno Príncipe","Curitiba","Paraná","Brazil",{"lat":169,"lon":170},-25.42778,-49.27306,{"facility":172,"status":8,"city":173,"state":174,"country":167,"geoPoint":175},"Casa dos Raros","Santa Cecília","Porto Alegre",{"lat":176,"lon":177},-26.96083,-50.42694,{"facility":179,"status":8,"city":180,"country":181,"geoPoint":182},"Hopital de la Timone","Marseille","France",{"lat":183,"lon":184},43.29695,5.38107,{"facility":186,"status":8,"city":187,"country":181,"geoPoint":188},"Necker-Enfants Malades Hospital","Paris",{"lat":189,"lon":190},48.85341,2.3488,{"facility":192,"status":8,"city":193,"country":194,"geoPoint":195},"Sheba Medical Center","Ramat Gan","Israel",{"lat":196,"lon":197},32.08227,34.81065,{"facility":199,"status":8,"city":200,"country":201,"geoPoint":202},"Azienda Ospedaliera Universitaria Meyer IRCCS","Florence","Italy",{"lat":203,"lon":204},43.77925,11.24626,{"facility":206,"status":8,"city":207,"country":201,"geoPoint":208},"Fondazione IRCCS Istituto Neurologico C. Besta","Milan",{"lat":209,"lon":210},45.46427,9.18951,{"facility":116,"status":117,"city":212,"country":201,"geoPoint":213},"Rome",{"lat":214,"lon":215},41.89193,12.51133,{"facility":217,"status":8,"city":218,"country":219,"geoPoint":220},"Hospital de Santa Maria","Lisbon","Portugal",{"lat":221,"lon":222},38.72509,-9.1498,{"facility":224,"status":8,"city":225,"country":219,"geoPoint":226},"Hospital Santa Joao","Porto",{"lat":227,"lon":228},41.1485,-8.61097,{"facility":116,"status":117,"city":230,"country":231,"geoPoint":232},"London","United Kingdom",{"lat":233,"lon":234},51.50853,-0.12574,{"facility":236,"status":8,"city":237,"country":231,"geoPoint":238},"University of Oxford","Oxford",{"lat":239,"lon":240},51.75222,-1.25596,[242,247],{"name":243,"role":244,"phone":245,"email":246},"Patients Contact Trial Recruitment","CONTACT","1-888-756-8657","Trialrecruitment@ultragenyx.com",{"name":248,"role":244,"phone":245,"email":249},"HCPs Contact: Medical Information","medinfo@ultragenyx.com",[251],{"name":252,"affiliation":13,"role":253},"Medical Director","STUDY_DIRECTOR",[],[256,259],{"label":257,"url":258},"Ultragenyx Patient Advocacy Website","https:\u002F\u002Fwww.ultrarareadvocacy.com\u002Fconditions-we-study\u002Fangelman-syndrome-as\u002F",{"label":260,"url":261},"Ultragenyx Transparency Commitment","https:\u002F\u002Fwww.ultragenyx.com\u002Four-research\u002Four-clinical-trial-transparency-commitment\u002F",{"nct_id":4,"conditions":263,"biomarkers":264},[19],[265],"Ubiquitin-Protein Ligase E3A",{"nct_id":4,"found":15,"summary":267,"prompt_version":277},{"design":268,"status":269,"heading":270,"summary":271,"follow_up":272,"word_count":273,"commitments":274,"compensation":275,"drugs_mentioned":276},"This study is an interventional basket study with several subprotocols. Most subprotocols are open-label (everyone knows what treatment is being given) and single-arm, while one subprotocol randomizes participants 2:1 to receive GTX-102 or no treatment.","completed","GTX-102 for Angelman Syndrome","This study is testing a drug called GTX-102 for people with Angelman syndrome. Researchers want to see how safe and effective GTX-102 is. Participants can be between 1 and 64 years old and have a confirmed diagnosis of Angelman syndrome, specifically the deletion-type for some groups. The study will measure side effects and look at how GTX-102 affects cognitive (thinking and learning) abilities and overall response to treatment. The study is currently unclear on its recruitment status, but plans to enroll 60 participants. Some participants will receive GTX-102, while others in one group will not receive treatment for a period.","Participants will be followed for safety up to Day 506. Cognitive and multidomain responses will be measured at Day 338.",100,"Not specified in the trial record.","Not stated in the trial record.",[37],"v2"]