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Clinical Trials in Canada / NCT03861273
Active, not recruiting Phase 3

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

NCT03861273 · tracked via the Priya Life Science Canada tracker
Sponsor
Phase
Phase 3
Started
2019-07-29
Last updated
2026-06-01

Condition(s) studied

Hemophilia B

Investigational drug(s) / intervention(s)

PF-06838435/ fidanacogene elaparvovec

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

Sex
MALE
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
Inclusion Criteria * 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. * Documented moderately severe to severe hemophilia B (Factor IX activity \< =2%) * Previous experience with FIX therapy (=\>50 documented exposure days to a FIX protein product) * Suspension of prophylaxis therapy for hemophilia B after administration of the study drug * Laboratory values (hemoglobin, platelets and creatinine) within study specified limits * Agree to contraception until components of the drug are eliminated from their body * Capable of giving signed informed consent Exclusion Criteria * Anti-AAVRh74var neutralizing antibodies (nAb) titer above the established threshold (ie, positive for nAb). * History of inhibitor to Factor IX or inhibitor detected during screening. Clinical signs or symptoms of decreased response to Factor IX * Hypersensitivity to Factor IX replacement product or IV immunoglobulin administration * History of chronic infection or other chronic disease * Any conditions associated with increased thromboembolic risk * Concurrent clinically significant major disease or condition unsuitable for participation and/or may interfere with the interpretation of study results * Laboratory values at screening visit that are abnormal or outside acceptable study limits * Current unstable liver or biliary disease * Currently on antiviral therapy for hepatitis B or C * Planned surgical procedure requiring Factor IX surgical prophylactic factor treatment 15 months from screening visit * Use of restricted therapies (e.g., blood products, acetylsalicylic acid \[aspirin\] or ibuprofen, other investigational therapy, and by-passing agents) * Previously dosed in a gene therapy research trial at any time or in an interventional clinical study within 12 weeks of screening visit * Active hepatitis B or C; hepatitis B surface antigen, hepatitis B virus deoxyribonucleic acid positivity, or hepatitis C virus ribonucleic acid positivity * Significant liver disease * Serological evidence of HIV1 or HIV2 infection with either CD4+ cell count \<=200 mm3 and/or a viral load \>20 copies/mL * Study and sponsor staff involved in the conduct of the study and their families * Unable to comply with study procedures * Sensitivity to heparin or heparin induced thrombocytopenia * Sensitivity to any of the study interventions, or components thereof, or drug or other allergy

Primary outcome measure(s)

Trial sites (67)

FacilityCityRegionStatus
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.

More Pfizer trials in Canada

Other trials for the same condition

Official registry record

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