Study Summary
This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or less). The gene therapy is designed to introduce genetic material into cells to compensate for missing or non-functioning Factor IX. Eligible study participants will have completed a minimum 6 months of routine Factor IX prophylaxis therapy during the lead in study (C0371004). Participants will be dosed once (intravenously) and will be evaluated over the course of 6 years. The main objective of the study will evaluate the annualized bleeding rate \[ABR\] for participants treated with gene therapy versus standard of care (SOC) therapy (FIX prophylaxis replacement regimen).
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PF-06838435/ fidanacogene elaparvovecBIOLOGICAL
Gene Therapy
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| UCSF IDS Pharmacy | San Francisco | California | United States |
| University of California, San Francisco - Clinical Research Center | San Francisco | California | United States |
| University of California, San Francisco - Outpatient Hematology Clinic | San Francisco | California | United States |
| The Regents of the University of California, San Francisco campus | San Francisco | California | United States |
| Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus | Aurora | Colorado | United States |
| Regents of the University of Colorado University of Colorado Denver, Office of Grants and Contracts | Aurora | Colorado | United States |
| Indiana Hemophilia & Thrombosis Center, Inc. | Indianapolis | Indiana | United States |
| Indiana Hemophilia and Thrombosis Center, Inc | Indianapolis | Indiana | United States |
| Innovative Hematology, Inc. | Indianapolis | Indiana | United States |
| St. Vincent Hospital & Health Care Center, Inc. | Indianapolis | Indiana | United States |
| Madison Radiological Group | Madison | Mississippi | United States |
| Mississippi Center for Advanced Medicine | Madison | Mississippi | United States |
| Center for Human Phenomic Science | Philadelphia | Pennsylvania | United States |
| Investigational Drug Service | Philadelphia | Pennsylvania | United States |
| Penn Blood Disorder Center | Philadelphia | Pennsylvania | United States |
| The Trustees of the University of Pennsylvania, Office of Clinical Research- Legal | Philadelphia | Pennsylvania | United States |
| Royal Prince Alfred Hospital | Camperdown | New South Wales | Australia |
| Royal Adelaide Hospital | Adelaide | South Australia | Australia |
| The Alfred Hospital | Melbourne | Victoria | Australia |
| Fiona Stanley Hospital | Murdoch | Western Australia | Australia |
| Royal Brisbane and Women's Hospital | Herston | Australia | |
| Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos - Hemoes | Vitória | Espírito Santo | Brazil |
| Centro de Hematologia e Hemoterapia - Hemocentro de Campinas - UNICAMP | Campinas | São Paulo | Brazil |
| Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo | São Paulo | São Paulo | Brazil |
| Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti- HEMORIO | Rio de Janeiro | Brazil | |
| McMaster University Medical Centre - Hamilton Health Sciences | Hamilton | Ontario | Canada |
| Juravinski Hospital - Hamilton Health Sciences | Hamilton | Ontario | Canada |
| St. Michael's Hospital | Toronto | Ontario | Canada |
| Hopital Cardiologique Louis Pradel - CRTH | Bron | France | |
| Hopital Necker | Paris | France | |
| Vivantes Klinikum Friedrichshain | Berlin | Germany | |
| Vivantes Klinikum im Friedrichshain | Berlin | Germany | |
| Universitätsklinikum Bonn Institut für Klinische Chemie und Klinische Pharmakologie -Phase 1-Einheit | Bonn | Germany | |
| Universitätsklinikum Bonn, Anstalt des öffentlichen Rechts | Bonn | Germany | |
| Universitätsklinikum Bonn | Bonn | Germany | |
| General Hospital of Athens "LAIKO", 2nd Regional Blood Transfusion Center | Athens | Greece | |
| Azienda Ospedaliero Universitaria Careggi | Florence | Italy | |
| Azienda Ospedaliera Universitaria Federico II | Naples | Italy | |
| Sapporo Tokushukai Hospital | Sapporo | Hokkaido | Japan |
| Nara Medical University Hospital | Kashihara | Nara | Japan |
| Saitama Medical University Hospital | Iruma-gun | Saitama | Japan |
| National Center for Child Health and Development | Setagaya-ku | Tokyo | Japan |
| King Abdulaziz Medical City-Ministry of national guard | Riyadh | Kingdom of Saudi Arabia | Saudi Arabia |
| King Faisal Specialist Hospital and Research Centre | Riyadh | Saudi Arabia | |
| King Abdullah International Medical Research Center | Riyadh | Saudi Arabia | |
| Kyung Hee University Hospital at Gangdong | Seoul | South Korea | |
| Hospital Universitario Virgen de la Arrixaca | El Palmar | Murcia | Spain |
| Hospital Universitari Vall d´Hebrón | Barcelona | Spain | |
| Skåne University Hospital, Department of Hematology, Oncology and Radiation Physics | Malmo | Sweden | |
| ApoEx AB | Malmo | Sweden | |
| Changhua Christian Hospital | Changhua | Taiwan | |
| Kaohsiung Medical University Chung-Ho Memorial Hospital | Kaohsiung City | Taiwan | |
| Chung Shan Medical University Hospital | Taichung | Taiwan | |
| Chung Shan Medical University | Taichung | Taiwan | |
| Taichung Veterans General Hospital | Taichung | Taiwan | |
| National Taiwan University Hospital | Taipei | Taiwan | |
| Acibadem Adana Hospital | Adana | Turkey (Türkiye) | |
| Gaziantep University Sahinbey Training and Research Hospital | Gaziantep | Turkey (Türkiye) | |
| Istanbul University Oncology Institute | Istanbul | Turkey (Türkiye) | |
| Ege University Medical Faculty Hospital | Izmir | Turkey (Türkiye) | |
| Newcastle upon Tyne Hospitals NHS FT | Newcastle upon Tyne | TYNE & WEAR | United Kingdom |
| Newcastle upon Tyne Hospitals NHS FT | Newcastle upon Tyne | TYNE & WEAR | United Kingdom |
| Glasgow Royal Infirmary | Glasgow | United Kingdom | |
| Guy's and St. Thomas' NHS Foundation Trust | London | United Kingdom | |
| Newcastle upon Tyne Hospitals NHS Foundation Trust | Newcastle upon Tyne | United Kingdom |