{ "data": { "asOf": "2026-10-30T22:27:00.231Z", "dedupMethod": "exact cross-registry identifier matching; probabilistic = review only", "byRegistry": { "clinicaltrials_gov": 1 }, "canonicalTrial": { "canonicalId": "NCT03861273", "title": "A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B", "scientificTitle": "PHASE 3, OPEN-LABEL, SINGLE-ARM STUDY TO EVALUATE EFFICACY AND SAFETY OF FIX GENE TRANSFER WITH PF-06838435 (RAAV-SPARK100-HFIX-R338L) IN ADULT MALE PARTICIPANTS WITH MODERATELY SEVERE TO SEVERE HEMOPHILIA B (FIX:C ≤2%) (BENEGENE-2)", "status": "active", "studyType": "interventional", "phase": [ "PHASE3" ], "conditions": [ "Hemophilia B" ], "interventions": [ { "name": "PF-06838435/ fidanacogene elaparvovec", "type": "BIOLOGICAL", "description": "Gene Therapy" } ], "sponsor": { "name": "Pfizer", "type": "INDUSTRY" }, "locations": [ { "city": "San Francisco", "country": "United States", "facility": "UCSF IDS Pharmacy" }, { "city": "San Francisco", "country": "United States", "facility": "University of California, San Francisco - Clinical Research Center" }, { "city": "San Francisco", "country": "United States", "facility": "University of California, San Francisco - Outpatient Hematology Clinic" }, { "city": "San Francisco", "country": "United States", "facility": "The Regents of the University of California, San Francisco campus" }, { "city": "Aurora", "country": "United States", "facility": "Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus" }, { "city": "Aurora", "country": "United States", "facility": "Regents of the University of Colorado University of Colorado Denver, Office of Grants and Contracts" }, { "city": "Indianapolis", "country": "United States", "facility": "Indiana Hemophilia & Thrombosis Center, Inc." }, { "city": "Indianapolis", "country": "United States", "facility": "Indiana Hemophilia and Thrombosis Center, Inc" }, { "city": "Indianapolis", "country": "United States", "facility": "Innovative Hematology, Inc." }, { "city": "Indianapolis", "country": "United States", "facility": "St. Vincent Hospital & Health Care Center, Inc." }, { "city": "Madison", "country": "United States", "facility": "Madison Radiological Group" }, { "city": "Madison", "country": "United States", "facility": "Mississippi Center for Advanced Medicine" }, { "city": "Philadelphia", "country": "United States", "facility": "Center for Human Phenomic Science" }, { "city": "Philadelphia", "country": "United States", "facility": "Investigational Drug Service" }, { "city": "Philadelphia", "country": "United States", "facility": "Penn Blood Disorder Center" }, { "city": "Philadelphia", "country": "United States", "facility": "The Trustees of the University of Pennsylvania, Office of Clinical Research- Legal" }, { "city": "Camperdown", "country": "Australia", "facility": "Royal Prince Alfred Hospital" }, { "city": "Adelaide", "country": "Australia", "facility": "Royal Adelaide Hospital" }, { "city": "Melbourne", "country": "Australia", "facility": "The Alfred Hospital" }, { "city": "Murdoch", "country": "Australia", "facility": "Fiona Stanley Hospital" }, { "city": "Herston", "country": "Australia", "facility": "Royal Brisbane and Women's Hospital" }, { "city": "Vitória", "country": "Brazil", "facility": "Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos - Hemoes" }, { "city": "Campinas", "country": "Brazil", "facility": "Centro de Hematologia e Hemoterapia - Hemocentro de Campinas - UNICAMP" }, { "city": "São Paulo", "country": "Brazil", "facility": "Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo" }, { "city": "Rio de Janeiro", "country": "Brazil", "facility": "Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti- HEMORIO" }, { "city": "Hamilton", "country": "Canada", "facility": "McMaster University Medical Centre - Hamilton Health Sciences" }, { "city": "Hamilton", "country": "Canada", "facility": "Juravinski Hospital - Hamilton Health Sciences" }, { "city": "Toronto", "country": "Canada", "facility": "St. Michael's Hospital" }, { "city": "Bron", "country": "France", "facility": "Hopital Cardiologique Louis Pradel - CRTH" }, { "city": "Paris", "country": "France", "facility": "Hopital Necker" }, { "city": "Berlin", "country": "Germany", "facility": "Vivantes Klinikum Friedrichshain" }, { "city": "Berlin", "country": "Germany", "facility": "Vivantes Klinikum im Friedrichshain" }, { "city": "Bonn", "country": "Germany", "facility": "Universitätsklinikum Bonn Institut für Klinische Chemie und Klinische Pharmakologie -Phase 1-Einheit" }, { "city": "Bonn", "country": "Germany", "facility": "Universitätsklinikum Bonn, Anstalt des öffentlichen Rechts" }, { "city": "Bonn", "country": "Germany", "facility": "Universitätsklinikum Bonn" }, { "city": "Athens", "country": "Greece", "facility": "General Hospital of Athens \"LAIKO\", 2nd Regional Blood Transfusion Center" }, { "city": "Florence", "country": "Italy", "facility": "Azienda Ospedaliero Universitaria Careggi" }, { "city": "Naples", "country": "Italy", "facility": "Azienda Ospedaliera Universitaria Federico II" }, { "city": "Sapporo", "country": "Japan", "facility": "Sapporo Tokushukai Hospital" }, { "city": "Kashihara", "country": "Japan", "facility": "Nara Medical University Hospital" }, { "city": "Iruma-gun", "country": "Japan", "facility": "Saitama Medical University Hospital" }, { "city": "Setagaya-ku", "country": "Japan", "facility": "National Center for Child Health and Development" }, { "city": "Riyadh", "country": "Saudi Arabia", "facility": "King Abdulaziz Medical City-Ministry of national guard" }, { "city": "Riyadh", "country": "Saudi Arabia", "facility": "King Faisal Specialist Hospital and Research Centre" }, { "city": "Riyadh", "country": "Saudi Arabia", "facility": "King Abdullah International Medical Research Center" }, { "city": "Seoul", "country": "South Korea", "facility": "Kyung Hee University Hospital at Gangdong" }, { "city": "El Palmar", "country": "Spain", "facility": "Hospital Universitario Virgen de la Arrixaca" }, { "city": "Barcelona", "country": "Spain", "facility": "Hospital Universitari Vall d´Hebrón" }, { "city": "Malmö", "country": "Sweden", "facility": "Skåne University Hospital, Department of Hematology, Oncology and Radiation Physics" }, { "city": "Malmö", "country": "Sweden", "facility": "ApoEx AB" }, { "city": "Changhua", "country": "Taiwan", "facility": "Changhua Christian Hospital" }, { "city": "Kaohsiung City", "country": "Taiwan", "facility": "Kaohsiung Medical University Chung-Ho Memorial Hospital" }, { "city": "Taichung", "country": "Taiwan", "facility": "Chung Shan Medical University Hospital" }, { "city": "Taichung", "country": "Taiwan", "facility": "Chung Shan Medical University" }, { "city": "Taichung", "country": "Taiwan", "facility": "Taichung Veterans General Hospital" }, { "city": "Taipei", "country": "Taiwan", "facility": "National Taiwan University Hospital" }, { "city": "Adana", "country": "Turkey (Türkiye)", "facility": "Acibadem Adana Hospital" }, { "city": "Gaziantep", "country": "Turkey (Türkiye)", "facility": "Gaziantep University Sahinbey Training and Research Hospital" }, { "city": "Istanbul", "country": "Turkey (Türkiye)", "facility": "Istanbul University Oncology Institute" }, { "city": "Izmir", "country": "Turkey (Türkiye)", "facility": "Ege University Medical Faculty Hospital" }, { "city": "Newcastle upon Tyne", "country": "United Kingdom", "facility": "Newcastle upon Tyne Hospitals NHS FT" }, { "city": "Newcastle upon Tyne", "country": "United Kingdom", "facility": "Newcastle upon Tyne Hospitals NHS FT" }, { "city": "Glasgow", "country": "United Kingdom", "facility": "Glasgow Royal Infirmary" }, { "city": "London", "country": "United Kingdom", "facility": "Guy's and St. Thomas' NHS Foundation Trust" }, { "city": "Newcastle upon Tyne", "country": "United Kingdom", "facility": "Newcastle upon Tyne Hospitals NHS Foundation Trust" } ], "outcomes": { "other": [], "primary": [ "Annualized Bleeding Rate (ABR) for Total Bleeds (Treated and Untreated) From Week 12 to Month 15" ], "secondary": [ "ABR for Treated Bleeds From Week 12 to Month 15", "Annualized Infusion Rate (AIR) of Exogenous FIX From Week 12 to Month 15", "Steady State Circulating Factor IX (FIX:C) From Week 12 to Month 15", "Circulating Factor IX (FIX:C) at Week 12, Week 24, Week 65", "Annualized Factor IX (FIX) Consumption From Week 12 to Month 15", "ABR for Spontaneous Bleeds From Week 12 to Month 15", "ABR for Traumatic Bleeds From Week 12 to Month 15", "ABR for Untreated Bleeds From Week 12 to Month 15", "Number of Target Joint Bleeds From Week 12 to Month 15", "Percentage of the Participants Without Bleeds From Week 12 to Month 15", "Change From Baseline in Joint Health as Measured by the Hemophilia Joint Health Score (HJHS) Instrument at Month 12", "Change From Baseline in Hemophilia Quality of Life (Haem A QoL) Physical Health Domain at Month 12", "Change From Baseline in Hemophilia Activities List (HAL) Complex Lower Extremity Activities Component Score at Month 12", "Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs", "Number of Participants With Adverse Events of Special Interest", "Number of Participants With Positive Neutralizing Antibody (nAb) to Adeno-associated Virus Vector (AAV) and Anti-Drug Antibody (ADA)", "ABR for Total Bleeds (Treated and Untreated) Through the Study", "ABR for Treated Bleeds Through the Study", "AIR of Exogenous Factor IX Through the Study", "FIX: C Level Through the Study", "Annualized Factor IX Consumption Through the Study", "ABR for Spontaneous and Traumatic, and Untreated Bleeds Through the Study", "Change From Baseline in Joint Health as Measured by the HJHS Instrument Through the Study", "Number of Target Joint Bleeds Through the Study", "Change From Baseline in Haem A QoL Physical Health Domain Through the Study", "Change From Baseline in HAL Complex Lower Extremity Activities Component Score Through the Study" ] }, "eligibility": { "sex": "MALE", "criteria": "Inclusion Criteria\n\n* Males who completed 6 months of Factor IX prophylaxis therapy during the lead-in study (C0371004) prior to providing consent at the screening visit for this study.\n* Documented moderately severe to severe hemophilia B (Factor IX activity \\< =2%)\n* Previous experience with FIX therapy (=\\>50 documented exposure days to a FIX protein product)\n* Suspension of prophylaxis therapy for hemophilia B after administration of the study drug\n* Laboratory values (hemoglobin, platelets and creatinine) within study specified limits\n* Agree to contraception until components of the drug are eliminated from their body\n* Capable of giving signed informed consent\n\nExclusion Criteria\n\n* Anti-AAVRh74var neutralizing antibodies (nAb) titer above the established threshold (ie, positive for nAb).\n* History of inhibitor to Factor IX or inhibitor detected during screening. Clinical signs or symptoms of decreased response to Factor IX\n* Hypersensitivity to Factor IX replacement product or IV immunoglobulin administration\n* History of chronic infection or other chronic disease\n* Any conditions associated with increased thromboembolic risk\n* Concurrent clinically significant major disease or condition unsuitable for participation and/or may interfere with the interpretation of study results\n* Laboratory values at screening visit that are abnormal or outside acceptable study limits\n* Current unstable liver or biliary disease\n* Currently on antiviral therapy for hepatitis B or C\n* Planned surgical procedure requiring Factor IX surgical prophylactic factor treatment 15 months from screening visit\n* Use of restricted therapies (e.g., blood products, acetylsalicylic acid \\[aspirin\\] or ibuprofen, other investigational therapy, and by-passing agents)\n* Previously dosed in a gene therapy research trial at any time or in an interventional clinical study within 12 weeks of screening visit\n* Active hepatitis B or C; hepatitis B surface antigen, hepatitis B virus deoxyribonucleic acid positivity, or hepatitis C virus ribonucleic acid positivity\n* Significant liver disease\n* Serological evidence of HIV1 or HIV2 infection with either CD4+ cell count \\<=200 mm3 and/or a viral load \\>20 copies/mL\n* Study and sponsor staff involved in the conduct of the study and their families\n* Unable to comply with study procedures\n* Sensitivity to heparin or heparin induced thrombocytopenia\n* Sensitivity to any of the study interventions, or components thereof, or drug or other allergy", "maximumAge": "65 Years", "minimumAge": "18 Years", "healthyVolunteers": false }, "enrollment": { "type": "ACTUAL", "count": 51 }, "startDate": "2019-07-29", "completionDate": "2028-02-25", "lastUpdated": "2026-10-22T00:00:00.000Z", "firstPosted": "2019-03-04T00:00:00.000Z", "resultsAvailable": true, "sourceRegistries": [ "clinicaltrials_gov" ], "sourceUrls": [ "https://clinicaltrials.gov/study/NCT03861273" ] }, "identifiers": [ { "idType": "nct", "idValue": "NCT03861273", "declaredBy": "clinicaltrials_gov" }, { "idType": "euct", "idValue": "2022-502844-11-00", "declaredBy": "clinicaltrials_gov" } ], "sourceRecords": [ { "registry": "clinicaltrials_gov", "registryName": "ClinicalTrials.gov", "license": "public-domain", "sourceId": "NCT03861273", "sourceUrl": "https://clinicaltrials.gov/study/NCT03861273", "sourceStatus": "ACTIVE_NOT_RECRUITING", "retrievedAt": "2026-10-28T04:32:06.526Z", "payloadSha256": "195ad135803fd8fddd3710f142d53a24752004ac783547a7c050bb156a0b0848", "piiRedacted": true } ], "citation": { "canonicalId": "NCT03861273", "sources": [ { "registry": "clinicaltrials_gov", "registryName": "ClinicalTrials.gov", "license": "public-domain", "sourceId": "NCT03861273", "verifyAt": "https://clinicaltrials.gov/study/NCT03861273", "retrievedAt": "2026-10-28T04:32:06.526Z", "payloadSha256": "195ad135803fd8fddd3710f142d53a24752004ac783547a7c050bb156a0b0848", "text": "ClinicalTrials.gov, record NCT03861273 (https://clinicaltrials.gov/study/NCT03861273), retrieved 2026-10-28, licence: public-domain." } ], "crossRegistry": false, "dedupMethod": "exact cross-registry identifier matching; probabilistic = review only", "text": "Trial NCT03861273, registered in ClinicalTrials.gov, retrieved 2026-10-28. 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