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
Condition(s) studied
Investigational drug(s) / intervention(s)
PF-06838435/ fidanacogene elaparvovec: Gene Therapy
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).
Eligibility
Primary outcome measure(s)
- Annualized Bleeding Rate (ABR) for Total Bleeds (Treated and Untreated) From Week 12 to Month 15 — Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
ABR = number of total bleeding episodes on study during the given time period) \*365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Treated Bleed: An event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding (protocol definition, unless specifically referring to untreated bleed). Untreated Bleed: A bleeding event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
Trial sites (65)
| 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 | |
| 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 | |
| Nara Medical University Hospital | Kashihara | Nara |
+ 25 more sites — see the full list on the official registry below.
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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 the USA