[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT05962398":3,"trial-entities:NCT05962398":321,"trial-summary:NCT05962398":17},{"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":17,"phases":22,"enrollment_info":23,"interventions":26,"primary_outcomes":35,"secondary_outcomes":43,"sex":87,"minimum_age":88,"maximum_age":17,"healthy_volunteers":15,"eligibility_criteria":89,"std_ages":93,"locations":96,"central_contacts":310,"overall_officials":311,"references":315,"see_also_links":320},"NCT05962398","CSL222_3003","Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)","An Extension Study Assessing the Long-term Safety and Efficacy of Etranacogene Dezaparvovec (CSL222) Previously Administered to Adult Male Subjects With Hemophilia B","ENROLLING_BY_INVITATION","2035-03-23","2025-07","2025-07-18","2023-08-30","CSL Behring","INDUSTRY",false,"The primary purpose of this study is to assess the long-term safety and efficacy in male adults with hemophilia B who were treated with CSL222 (CSL222) in parent studies CSL222\\_2001 (NCT03489291) or CSL222\\_3001 (NCT03569891).",null,[19],"Hemophilia B",[],"OBSERVATIONAL",[],{"count":24,"type":25},56,"ESTIMATED",[27],{"type":28,"name":29,"description":30,"armGroupLabels":31,"otherNames":33},"GENETIC","AAV5-hFIXco-Padua","Single intravenous infusion of AAV5-hFIXco-Padua in the study CSL222\\_2001 (NCT03489291) or CSL222\\_3001 (NCT03569891).",[32],"CSL222",[34],"Etranacogene dezaparvovec",[36,39,41],{"measure":37,"timeFrame":38},"Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs)","From Year 5 after administration of CSL222 in parent study up to Year 15",{"measure":40,"timeFrame":38},"Percentage of Participants With SAEs and AESIs",{"measure":42,"timeFrame":38},"Number of SAEs and AESIs",[44,47,49,52,56,59,62,64,66,68,70,73,77,80,82,85],{"measure":45,"description":46,"timeFrame":38},"Annualized Bleeding Rate (ABR)","The total, spontaneous, joint, and traumatic bleeds will be analyzed.",{"measure":48,"timeFrame":38},"Number of Participants With Zero Total, Spontaneous, Joint, and Traumatic Bleeding Episodes",{"measure":50,"description":51,"timeFrame":38},"Endogenous Factor IX (FIX) Activity","Endogenous FIX activity (expressed as percentage \\[%\\]) will be assessed using the one-stage activated partial thromboplastin time assay and a chromogenic assay.",{"measure":53,"description":54,"timeFrame":55},"Change from Baseline by Visit in Endogenous FIX Activity","Endogenous FIX activity (expressed as %) will be assessed using the one-stage activated partial thromboplastin time assay and a chromogenic assay.","At Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15",{"measure":57,"description":58,"timeFrame":38},"Annualized Consumption of FIX Replacement Therapy","The annualized consumption of FIX replacement therapy (excluding FIX replacement for invasive procedures) will be provided.",{"measure":60,"description":61,"timeFrame":38},"Annualized Infusion Rate of FIX Replacement Therapy","Annualized infusion rate of FIX replacement therapy (excluding FIX replacement for invasive procedures) will be provided.",{"measure":63,"timeFrame":38},"Number of Participants Remaining Free of Continuous FIX Prophylaxis",{"measure":65,"timeFrame":38},"Percentage of Participants Remaining Free of Continuous FIX Prophylaxis",{"measure":67,"timeFrame":38},"Number of New Target Joints",{"measure":69,"timeFrame":38},"Percentage Resolution of Pre-existing Target Joints",{"measure":71,"description":72,"timeFrame":38},"EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Index Value","The EQ-5D-5L questionnaire consists of the EQ-5D-5L descriptive system and the EQ-5D visual analogue scale (VAS). The EQ-5D-5L descriptive system of health-related quality of life (QoL) consists of 5 dimensions (mobility, self-care, usual activities, pain\u002Fdiscomfort, anxiety\u002Fdepression) for which responses will be recorded on 5 levels of severity (no problems, slight problems, moderate problems, severe problems, and extreme problems). The responses will be converted into a single overall health state utility value. This utility value will be derived using the EQ-5D-5L Crosswalk Index Value Calculator v2.xls \\[Hernández Alava et al, 2023\\], using the United Kingdom value set.",{"measure":74,"description":75,"timeFrame":76},"Change From Baseline in EQ-5D-5L Index Value","The EQ-5D-5L questionnaire consists of the EQ-5D-5L descriptive system and the EQ-5D VAS. The EQ-5D-5L descriptive system of health-related QoL consists of 5 dimensions (mobility, self-care, usual activities, pain\u002Fdiscomfort, anxiety\u002Fdepression) for which responses will be recorded on 5 levels of severity (no problems, slight problems, moderate problems, severe problems, and extreme problems). The responses will be converted into a single overall health state utility value. This utility value will be derived using the EQ-5D-5L Crosswalk Index Value Calculator v2.xls \\[Hernández Alava et al, 2023\\], using the United Kingdom value set.","Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15",{"measure":78,"description":79,"timeFrame":38},"EQ-5D VAS Score","The EQ-5D VAS, which measures overall health status on a vertical VAS, ranges from 0 to 100. A higher score indicates better QoL.",{"measure":81,"description":79,"timeFrame":76},"Change From Baseline in EQ-5D VAS Score",{"measure":83,"description":84,"timeFrame":38},"Hemophilia Quality of Life Questionnaire (Hem-A-QoL) Total Score","The Hem-A-QoL consists of 46 items comprising 10 domains: physical health, feelings, treatment, work and school, dealing with hemophilia, family planning, future, partnerships and sexuality, sports and leisure, and view of yourself for which responses will be recorded from 5 options (never, seldom, sometimes, often, and always; for some items, there is also a \"not applicable\" option). The total score is based on the scores for each domain and ranges from 0 to 100, with lower scores reflecting a better QoL.",{"measure":86,"description":84,"timeFrame":76},"Change From Baseline in Hem-A-QoL Total Score","MALE","18 Years",{"inclusion":90,"exclusion":91,"raw_text":92},[],[],"Inclusion Criteria:\n\nReceived treatment with CSL222 in Study CSL222\\_2001 or Study CSL222\\_3001, and completed participation in Study CSL222\\_2001 or Study CSL222\\_3001 or at least 5 years have passed since receiving CSL222.\n\nExclusion Criteria:\n\nNot Applicable",[94,95],"ADULT","OLDER_ADULT",[97,106,114,122,126,133,140,148,156,164,172,180,188,196,204,212,220,227,235,243,251,259,266,272,279,287,296,303],{"facility":98,"city":99,"state":100,"zip":101,"country":102,"geoPoint":103},"10-15 Phoenix Childrens Hospital","Phoenix","Arizona","85016","United States",{"lat":104,"lon":105},33.44838,-112.07404,{"facility":107,"city":108,"state":109,"zip":110,"country":102,"geoPoint":111},"10-14 Arkansas Children's Hospital - Pharmacology","Little Rock","Arkansas","72202",{"lat":112,"lon":113},34.74648,-92.28959,{"facility":115,"city":116,"state":117,"zip":118,"country":102,"geoPoint":119},"10-22 Orthopaedic Institute for Children","Los Angeles","California","90007",{"lat":120,"lon":121},34.05223,-118.24368,{"facility":123,"city":116,"state":117,"zip":124,"country":102,"geoPoint":125},"10-21 Children's Hospital of Los Angeles","90027",{"lat":120,"lon":121},{"facility":127,"city":128,"state":117,"zip":129,"country":102,"geoPoint":130},"10-63 UC Davis Medical Center","Sacramento","95817",{"lat":131,"lon":132},38.58157,-121.4944,{"facility":134,"city":135,"state":117,"zip":136,"country":102,"geoPoint":137},"10-12 University of California, San Diego (UCSD)","San Diego","92121",{"lat":138,"lon":139},32.71571,-117.16472,{"facility":141,"city":142,"state":143,"zip":144,"country":102,"geoPoint":145},"10-25 University of Colorado Denver","Aurora","Colorado","80045",{"lat":146,"lon":147},39.72943,-104.83192,{"facility":149,"city":150,"state":151,"zip":152,"country":102,"geoPoint":153},"10-19 University of South Florida","Tampa","Florida","33612",{"lat":154,"lon":155},27.94752,-82.45843,{"facility":157,"city":158,"state":159,"zip":160,"country":102,"geoPoint":161},"10-10 University of Michigan Medical Center","Ann Arbor","Michigan","48109",{"lat":162,"lon":163},42.27756,-83.74088,{"facility":165,"city":166,"state":167,"zip":168,"country":102,"geoPoint":169},"10-16 University of North Carolina at Chapel Hill","Chapel Hill","North Carolina","27599",{"lat":170,"lon":171},35.9132,-79.05584,{"facility":173,"city":174,"state":175,"zip":176,"country":102,"geoPoint":177},"10-18 Oregon Health & Science University","Portland","Oregon","97239",{"lat":178,"lon":179},45.52345,-122.67621,{"facility":181,"city":182,"state":183,"zip":184,"country":102,"geoPoint":185},"10-20 Vanderbilt University Medical Center","Nashville","Tennessee","37232",{"lat":186,"lon":187},36.16589,-86.78444,{"facility":189,"city":190,"state":191,"zip":192,"country":102,"geoPoint":193},"10-28 University of Texas Health Science Center","Houston","Texas","77030",{"lat":194,"lon":195},29.76328,-95.36327,{"facility":197,"city":198,"state":199,"zip":200,"country":102,"geoPoint":201},"10-26 University of Utah","Salt Lake City","Utah","84108",{"lat":202,"lon":203},40.76078,-111.89105,{"facility":205,"city":206,"state":207,"zip":208,"country":102,"geoPoint":209},"10-62 Washington Institute for Coagulation","Seattle","Washington","98104",{"lat":210,"lon":211},47.60621,-122.33207,{"facility":213,"city":214,"zip":215,"country":216,"geoPoint":217},"11-31 Cliniques Universitaires Saint-Luc","Brussels","1200","Belgium",{"lat":218,"lon":219},50.85045,4.34878,{"facility":221,"city":222,"zip":223,"country":216,"geoPoint":224},"11-30 Universitair Ziekenhuis Leuven","Leuven","3000",{"lat":225,"lon":226},50.87959,4.70093,{"facility":228,"city":229,"zip":230,"country":231,"geoPoint":232},"12-32 Rigshospitalet","Copenhagen","2100","Denmark",{"lat":233,"lon":234},55.67594,12.56553,{"facility":236,"city":237,"zip":238,"country":239,"geoPoint":240},"15-42 Vivantes Klinikum im Friedrichshain - Landsberger Allee","Berlin","10249","Germany",{"lat":241,"lon":242},52.52437,13.41053,{"facility":244,"city":245,"zip":246,"country":247,"geoPoint":248},"19-56 St. James Hospital","Dublin","D08 A978","Ireland",{"lat":249,"lon":250},53.33306,-6.24889,{"facility":252,"city":253,"zip":254,"country":255,"geoPoint":256},"13-33 Amsterdam Universitair Medische Centra (UMC)","Amsterdam-Zuidoost","1105 AZ","Netherlands",{"lat":257,"lon":258},52.3075,4.97222,{"facility":260,"city":261,"zip":262,"country":255,"geoPoint":263},"13-35 University Medical Center Groningen","Groningen","9713 GZ",{"lat":264,"lon":265},53.21917,6.56667,{"facility":267,"city":268,"country":255,"geoPoint":269},"13-34 Erasmus University Medical Center","Rotterdam",{"lat":270,"lon":271},51.9225,4.47917,{"facility":273,"city":274,"zip":275,"country":255,"geoPoint":276},"13-36 Universitair Medisch Centrum Utrecht","Utrecht","3584 CX",{"lat":277,"lon":278},52.09083,5.12222,{"facility":280,"city":281,"zip":282,"country":283,"geoPoint":284},"16-43 Skane University Hospital","Malmö","SE-205 02","Sweden",{"lat":285,"lon":286},55.60587,13.00073,{"facility":288,"city":289,"state":290,"zip":291,"country":292,"geoPoint":293},"18-52 Barts Health NHS Trust","London","England","E1 1FR.","United Kingdom",{"lat":294,"lon":295},51.50853,-0.12574,{"facility":297,"city":298,"zip":299,"country":292,"geoPoint":300},"18-54 Addenbrooke's Hospital","Cambridge","CB2 0QQ",{"lat":301,"lon":302},52.2,0.11667,{"facility":304,"city":305,"zip":306,"country":292,"geoPoint":307},"18-53 University Hospital Southampton NHS Foundation Trust","Southampton","SO16 6YD",{"lat":308,"lon":309},50.90395,-1.40428,[],[312],{"name":313,"affiliation":13,"role":314},"Study Director","STUDY_DIRECTOR",[316],{"pmid":317,"type":318,"citation":319},"40188458","DERIVED","von Drygalski A, Gomez E, Giermasz A, Castaman G, Key NS, Lattimore SU, Leebeek FWG, Miesbach WA, Recht M, Monahan PE, Le Quellec S, Pipe SW. Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years. Blood Adv. 2025 Jul 22;9(14):3543-3552. doi: 10.1182\u002Fbloodadvances.2024015291.",[],{"nct_id":4,"conditions":322,"biomarkers":323},[19],[]]