PF-06838435/ fidanacogene elaparvovec: Gene Therapy
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).
| Facility | City | Region | Status |
|---|---|---|---|
| UCSF IDS Pharmacy | San Francisco | California | |
| University of California, San Francisco - Clinical Research Center | San Francisco | California | |
| University of California, San Francisco - Outpatient Hematology Clinic | San Francisco | California | |
| The Regents of the University of California, San Francisco campus | San Francisco | California | |
| Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus | Aurora | Colorado | |
| Regents of the University of Colorado University of Colorado Denver, Office of Grants and Contracts | Aurora | Colorado | |
| Indiana Hemophilia & Thrombosis Center, Inc. | Indianapolis | Indiana | |
| Indiana Hemophilia and Thrombosis Center, Inc | Indianapolis | Indiana | |
| Innovative Hematology, Inc. | Indianapolis | Indiana | |
| St. Vincent Hospital & Health Care Center, Inc. | Indianapolis | Indiana | |
| Madison Radiological Group | Madison | Mississippi | |
| Mississippi Center for Advanced Medicine | Madison | Mississippi | |
| The Trustees of the University of Pennsylvania, Office of Clinical Research- Legal | Hamilton | Pennsylvania | |
| Center for Human Phenomic Science | Philadelphia | Pennsylvania | |
| Investigational Drug Service | Philadelphia | Pennsylvania | |
| Penn Blood Disorder Center | Philadelphia | Pennsylvania | |
| The Trustees of the University of Pennsylvania, Office of Clinical Research- Legal | Philadelphia | Pennsylvania | |
| Royal Prince Alfred Hospital | Camperdown | New South Wales | |
| Royal Adelaide Hospital | Adelaide | South Australia | |
| The Alfred Hospital | Melbourne | Victoria | |
| Fiona Stanley Hospital | Murdoch | Western Australia | |
| Royal Brisbane and Women's Hospital | Herston | Australia | |
| Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos - Hemoes | Vitória | Espírito Santo | |
| Centro de Hematologia e Hemoterapia - Hemocentro de Campinas - UNICAMP | Campinas | São Paulo | |
| Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo | São Paulo | São Paulo | |
| Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti- HEMORIO | Rio de Janeiro | Brazil | |
| McMaster University Medical Centre - Hamilton Health Sciences | Hamilton | Ontario | |
| Juravinski Hospital - Hamilton Health Sciences | Hamilton | Ontario | |
| St. Michael's Hospital | Toronto | Ontario | |
| 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 |
+ 27 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT03861273 on ClinicalTrials.gov ↗ ← All trials in Canada