[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT06431893":3,"trial-entities:NCT06431893":422,"trial-summary:NCT06431893":427},{"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":24,"primary_purpose":25,"phases":26,"enrollment_info":28,"interventions":31,"primary_outcomes":41,"secondary_outcomes":55,"sex":66,"minimum_age":67,"maximum_age":68,"healthy_volunteers":69,"eligibility_criteria":70,"std_ages":76,"locations":80,"central_contacts":415,"overall_officials":416,"references":420,"see_also_links":421},"NCT06431893","TVTX-TVT058-302","A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)","An Open-Label, Phase 3 Long-Term Extension (LTE) Study To Assess The Safety, Tolerability, And Efficacy Of Treatment With Pegtibatinase In Participants With Classical Homocystinuria (HCU) Due To Cystathionine Beta Synthase Deficiency (ENSEMBLE)","ENROLLING_BY_INVITATION","2030-08","2026-05","2026-05-28","2024-04-30","Travere Therapeutics, Inc.","INDUSTRY",true,"The goal of this long-term extension (LTE) study is to evaluate the safety and efficacy of pegtibatinase in patients with classical homocystinuria (HCU). Patients who are active in the Phase 1\u002F2 COMPOSE study or those who complete the 24 weeks of blinded treatment in the Phase 3 HARMONY are eligible to participate.\n\nParticipants will be in this clinical study for a minimum of 108 weeks including:\n\n* a treatment period of up to 104 weeks\n* a 4-week safety follow-up period","Overall Design:\n\nThis is a global, multicenter, single-arm, open-label study. Participants who meet all eligibility criteria may transition from other pegtibatinase studies and enroll into this LTE study for long-term safety, efficacy, and clinical assessments of pegtibatinase treatment.\n\nAll participants will follow a protocol of self-administration of pegtibatinase unless exempted. During the study, pegtibatinase pharmacokinetic assessments will be conducted. An optional protein tolerance modification (PTM) sub-study will also be conducted for eligible participants.",[19],"Homocystinuria",[21,22,23],"Classical homocystinuria","HCU","Cystathionine Beta Synthase Deficiency","INTERVENTIONAL","TREATMENT",[27],"PHASE3",{"count":29,"type":30},100,"ESTIMATED",[32],{"type":33,"name":34,"description":35,"armGroupLabels":36,"otherNames":38},"DRUG","Pegtibatinase","Pegtibatinase BIW",[37],"pegtibatinase",[39,40],"OT-58","TVT-058",[42,46,49,52],{"measure":43,"description":44,"timeFrame":45},"Treatment-emergent AEs","Incidence of treatment-emergent AEs","Week 1 - Week 108",{"measure":47,"description":48,"timeFrame":45},"Hypermethioninemia","Incidence of hypermethioninemia",{"measure":50,"description":51,"timeFrame":45},"Hypomethioninemia","Incidence of hypomethioninemia",{"measure":53,"description":54,"timeFrame":45},"Dietary Protein Rescue","Proportion of participants requiring dietary protein rescue for participants with hypomethioninemia",[56,60,63],{"measure":57,"description":58,"timeFrame":59},"Maximum Drug Concentration","Pegtibatinase pharmacokinetic parameters at steady state - maximum drug concentration \\[Cmax\\]","Pharmacokinetic Assessment Period - 4 weeks & 8 weeks",{"measure":61,"description":62,"timeFrame":59},"Area under the Curve within the Dosing Interval","Pegtibatinase pharmacokinetic parameters at steady state within the - area under the curve within the dosing interval \\[AUC0-tau\\]",{"measure":64,"description":65,"timeFrame":45},"Total Homocysteine (tHcy) Levels","Relative and absolute changes from baseline in tHcy levels","ALL","5 Years","65 Years",false,{"inclusion":71,"exclusion":73,"raw_text":75},[72],"Participants active in the COMPOSE study or who completed the 24-week blinded period on study intervention (pegtibatinase or placebo) in the Phase 3 HARMONY study",[74],"Participant permanently discontinued from study intervention treatment in a previous pegtibatinase study due to serious AE (SAE) or intolerance","Inclusion Criteria:\n\n* Participants active in the COMPOSE study or who completed the 24-week blinded period on study intervention (pegtibatinase or placebo) in the Phase 3 HARMONY study\n\nExclusion Criteria:\n\n* Participant permanently discontinued from study intervention treatment in a previous pegtibatinase study due to serious AE (SAE) or intolerance",[77,78,79],"CHILD","ADULT","OLDER_ADULT",[81,90,98,106,114,122,130,137,145,152,160,167,175,183,190,196,202,209,215,222,228,234,241,247,253,260,267,273,279,285,291,298,304,307,314,320,326,333,336,343,346,353,359,362,368,374,381,387,393,400,403,409],{"facility":82,"city":83,"state":84,"zip":85,"country":86,"geoPoint":87},"Phoenix Children's Hospital","Phoenix","Arizona","85006","United States",{"lat":88,"lon":89},33.44838,-112.07404,{"facility":91,"city":92,"state":93,"zip":94,"country":86,"geoPoint":95},"Yale University School of Medicine","New Haven","Connecticut","06519",{"lat":96,"lon":97},41.30815,-72.92816,{"facility":99,"city":100,"state":101,"zip":102,"country":86,"geoPoint":103},"Emory University","Atlanta","Georgia","30322",{"lat":104,"lon":105},33.749,-84.38798,{"facility":107,"city":108,"state":109,"zip":110,"country":86,"geoPoint":111},"Ann & Robert H. Lurie Children's Hospital of Chicago","Chicago","Illinois","60611",{"lat":112,"lon":113},41.85003,-87.65005,{"facility":115,"city":116,"state":117,"zip":118,"country":86,"geoPoint":119},"Maine Health Pediatric Specialty Care","Portland","Maine","04012",{"lat":120,"lon":121},43.65737,-70.2589,{"facility":123,"city":124,"state":125,"zip":126,"country":86,"geoPoint":127},"Uncommon Cures","Chevy Chase","Maryland","20815",{"lat":128,"lon":129},39.00287,-77.07115,{"facility":131,"city":132,"state":132,"zip":133,"country":86,"geoPoint":134},"Icahn School of Medicine at Mount Sinai","New York","10029",{"lat":135,"lon":136},40.71427,-74.00597,{"facility":138,"city":139,"state":140,"zip":141,"country":86,"geoPoint":142},"Science 37 - Virtual Site","Morrisville","North Carolina","27560",{"lat":143,"lon":144},35.82348,-78.82556,{"facility":146,"city":116,"state":147,"zip":148,"country":86,"geoPoint":149},"Oregon Health and Science University","Oregon","97239",{"lat":150,"lon":151},45.52345,-122.67621,{"facility":153,"city":154,"state":155,"zip":156,"country":86,"geoPoint":157},"The Children's Hospital of Philadelphia","Philadelphia","Pennsylvania","19104",{"lat":158,"lon":159},39.95238,-75.16362,{"facility":161,"city":162,"state":155,"zip":163,"country":86,"geoPoint":164},"University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh","Pittsburgh","15224",{"lat":165,"lon":166},40.44062,-79.99589,{"facility":168,"city":169,"state":170,"zip":171,"country":86,"geoPoint":172},"University of Texas Southwestern Medical Center","Dallas","Texas","75390",{"lat":173,"lon":174},32.78306,-96.80667,{"facility":176,"city":177,"state":178,"zip":179,"country":86,"geoPoint":180},"Utah Health - The University of Utah Primary Children's Hospital","Salt Lake City","Utah","84113",{"lat":181,"lon":182},40.76078,-111.89105,{"facility":184,"city":185,"country":186,"geoPoint":187},"Royal Adelaide Hospital","Adelaide","Australia",{"lat":188,"lon":189},-34.92866,138.59863,{"facility":191,"city":192,"country":186,"geoPoint":193},"Royal Children's Hospital Melbourne","Parkville",{"lat":194,"lon":195},-37.78333,144.95,{"facility":197,"city":198,"country":186,"geoPoint":199},"Westmead Hospital","Westmead",{"lat":200,"lon":201},-33.80383,150.98768,{"facility":203,"city":204,"country":205,"geoPoint":206},"Cliniques Universitaires Saint-Luc","Brussels","Belgium",{"lat":207,"lon":208},50.85045,4.34878,{"facility":210,"city":211,"country":205,"geoPoint":212},"Universitair Ziekenhuis Antwerpen (UZA)","Edegem",{"lat":213,"lon":214},51.15662,4.44504,{"facility":216,"city":217,"country":218,"geoPoint":219},"Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades","Paris","France",{"lat":220,"lon":221},48.85341,2.3488,{"facility":223,"city":224,"country":218,"geoPoint":225},"CHRU Hôpitaux de Tours - Hôpital Bretonneau","Tours",{"lat":226,"lon":227},47.39484,0.70398,{"facility":229,"city":230,"country":218,"geoPoint":231},"Centre Hospitalier Régional Universitaire de Nancy (CHRU) - Hopitaux de Brabois - Hôpital d'enfants","Vandœuvre-lès-Nancy",{"lat":232,"lon":233},48.66115,6.17114,{"facility":235,"city":236,"country":237,"geoPoint":238},"Universitätsklinikum Hamburg-Eppendorf","Hamburg","Germany",{"lat":239,"lon":240},53.55073,9.99302,{"facility":242,"city":243,"country":237,"geoPoint":244},"Universitätsklinikum Leipzig","Leipzig",{"lat":245,"lon":246},51.33962,12.37129,{"facility":248,"city":249,"country":237,"geoPoint":250},"Universitätsklinikum Münster","Münster",{"lat":251,"lon":252},51.96236,7.62571,{"facility":254,"city":255,"country":256,"geoPoint":257},"Children's Health Ireland (CHI) at Temple Street","Dublin","Ireland",{"lat":258,"lon":259},53.33306,-6.24889,{"facility":261,"city":262,"country":263,"geoPoint":264},"Ospedale Pediatrico Giovanni XXIII","Bari","Italy",{"lat":265,"lon":266},41.12066,16.86982,{"facility":268,"city":269,"country":263,"geoPoint":270},"IRCCS Istituto Giannina Gaslini","Genova",{"lat":271,"lon":272},45.21604,11.87211,{"facility":274,"city":275,"country":263,"geoPoint":276},"Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (Policlinico di Milano)","Milan",{"lat":277,"lon":278},42.78235,12.59836,{"facility":280,"city":281,"country":263,"geoPoint":282},"Fondazione IRCCS San Gerardo Dei Tintori - Ospedale San Gerardo","Monza",{"lat":283,"lon":284},45.58005,9.27246,{"facility":286,"city":287,"country":263,"geoPoint":288},"Azienda Ospedaliera di Padova","Padova",{"lat":289,"lon":290},44.38225,11.14261,{"facility":292,"city":293,"country":294,"geoPoint":295},"SP ZOZ Szpital Uniwersytecki w Krakowie","Krakow","Poland",{"lat":296,"lon":297},50.06143,19.93658,{"facility":299,"city":300,"country":294,"geoPoint":301},"Instytut Matki i Dziecka","Warsaw",{"lat":302,"lon":303},52.22977,21.01178,{"facility":305,"city":300,"country":294,"geoPoint":306},"Instytut Pomnik-Centrum Zdrowia Dziecka",{"lat":302,"lon":303},{"facility":308,"city":309,"country":310,"geoPoint":311},"Unidade Local de Saúde de Coimbra, EPE - Hospitais da Universidade de Coimbra","Coimbra","Portugal",{"lat":312,"lon":313},40.20686,-8.41996,{"facility":315,"city":316,"country":310,"geoPoint":317},"Unidade Local de Saúde de Santa Maria, EPE - Hospital de Santa Maria","Lisbon",{"lat":318,"lon":319},38.72509,-9.1498,{"facility":321,"city":322,"country":310,"geoPoint":323},"Unidade Local de Saúde de São João, EPE - Hospital São João","Porto",{"lat":324,"lon":325},41.1485,-8.61097,{"facility":327,"city":328,"country":329,"geoPoint":330},"Hamad General Hospital","Doha","Qatar",{"lat":331,"lon":332},25.28545,51.53096,{"facility":334,"city":328,"country":329,"geoPoint":335},"Sidra Hospital",{"lat":331,"lon":332},{"facility":337,"city":338,"country":339,"geoPoint":340},"King Abdullah International Medical Research Center","Riyadh","Saudi Arabia",{"lat":341,"lon":342},24.68773,46.72185,{"facility":344,"city":338,"country":339,"geoPoint":345},"King Faisal Specialist Hospital and Research Centre (KFSHRC) - Riyadh",{"lat":341,"lon":342},{"facility":347,"city":348,"country":349,"geoPoint":350},"Hospital Sant Joan de Déu","Barcelona","Spain",{"lat":351,"lon":352},41.38879,2.15899,{"facility":354,"city":355,"country":349,"geoPoint":356},"Hospital Universitario 12 de Octubre","Madrid",{"lat":357,"lon":358},40.4165,-3.70256,{"facility":360,"city":355,"country":349,"geoPoint":361},"Hospital Universitario Ramón y Cajal",{"lat":357,"lon":358},{"facility":363,"city":364,"country":349,"geoPoint":365},"Complejo Hospitalario Universitario de Santiago (CHUS) - Hospital Clínico Universitario","Santiago de Compostela",{"lat":366,"lon":367},42.88052,-8.54569,{"facility":369,"city":370,"country":349,"geoPoint":371},"Hospital Universitario Virgen del Rocío","Seville",{"lat":372,"lon":373},37.38283,-5.97317,{"facility":375,"city":376,"country":377,"geoPoint":378},"Gazi University Hospital","Ankara","Turkey (Türkiye)",{"lat":379,"lon":380},39.91987,32.85427,{"facility":382,"city":383,"country":377,"geoPoint":384},"Ege Üniversitesi Hastanesi Bornova","Bornova",{"lat":385,"lon":386},38.47921,27.2399,{"facility":388,"city":389,"country":377,"geoPoint":390},"Istanbul University Cerrahpasa Hospital","Istanbul",{"lat":391,"lon":392},41.01384,28.94966,{"facility":394,"city":395,"country":396,"geoPoint":397},"Birmingham Women's and Children's NHS Foundation Trust","Birmingham","United Kingdom",{"lat":398,"lon":399},52.48142,-1.89983,{"facility":401,"city":395,"country":396,"geoPoint":402},"University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital Birmingham",{"lat":398,"lon":399},{"facility":404,"city":405,"country":396,"geoPoint":406},"University College London Hospitals NHS Foundation Trust - National Hospital for Neurology and Neurosurgery","London",{"lat":407,"lon":408},51.50853,-0.12574,{"facility":410,"city":411,"country":396,"geoPoint":412},"Salford Royal NHS Foundation Trust - Salford Royal Hospital","Salford",{"lat":413,"lon":414},53.48771,-2.29042,[],[417],{"name":418,"affiliation":13,"role":419},"Michael Imperiale, MD","STUDY_DIRECTOR",[],[],{"nct_id":4,"conditions":423,"biomarkers":425},[424],"Classical Homocystinuria",[426],"Cystathionine beta-synthase",{"nct_id":4,"found":69,"summary":428,"prompt_version":428},null]