RECRUITINGOBSERVATIONAL
An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B
An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B
About This Trial
This observational, post-authorization, long-term follow-up study aims to investigate the short and long-term effectiveness and safety of HEMGENIX in patients with hemophilia B. The study will also include a cohort of patients with hemophilia B treated with FIX prophylaxis to enable interpretation of relevant efficacy and safety findings of HEMGENIX.
Who May Be Eligible (Plain English)
Who May Qualify:
- HEMGENIX Cohort:
- \- Treatment with commercial HEMGENIX.
- \- Have provided signed written willing to sign a consent form within 3 months before or within 6 months after HEMGENIX treatment, or within 6 months of when the study is initiated at the participating site.
- FIX Prophylaxis Cohort:
- \- Adult patients (≥ 18 years) with hemophilia B who have consented and enrolled in ATHN Transcends Hemophilia Cohort (or a similar registry) and are receiving FIX prophylaxis therapy.
Who Should NOT Join This Trial:
- HEMGENIX Cohort:
- \- The patient population that will be observed in this study must not have been treated with etranacogene dezaparvovec in a clinical trial.
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
Inclusion Criteria:
* HEMGENIX Cohort:
* \- Treatment with commercial HEMGENIX.
* \- Have provided signed written informed consent within 3 months before or within 6 months after HEMGENIX treatment, or within 6 months of when the study is initiated at the participating site.
* FIX Prophylaxis Cohort:
* \- Adult patients (≥ 18 years) with hemophilia B who have consented and enrolled in ATHN Transcends Hemophilia Cohort (or a similar registry) and are receiving FIX prophylaxis therapy.
Exclusion Criteria:
* HEMGENIX Cohort:
* \- The patient population that will be observed in this study must not have been treated with etranacogene dezaparvovec in a clinical trial.
Treatments Being Tested
GENETIC
HEMGENIX
HEMGENIX is a gene therapy medicinal product that aims to deliver a factor IX (FIX) gene expression cassette to the liver of patients with hemophilia B.
BIOLOGICAL
Factor IX (FIX)
FIX prophylaxis therapy
Locations (12)
American Thrombosis and Hemostasis Network
Rochester, New York, United States
Medical University Vienna
Vienna, Austria
Aarhus Universitetshospital
Århus N, Denmark
Centre Hospitalier Universitaire de Brest / CHU Morvan
Brest, France
Centre Régional de Traitement de l'Hémophilie
Nantes, France
CHU Nancy - Hôpital Brabois
Vandœuvre-lès-Nancy, France
Klinik für Angiologie/ Hämostaseologie
Berlin, Germany
University of Clinic Bonn
Bonn, Germany
Klinikum der Johann-Wolfgang Goethe Universitaet
Frankfurt, Germany
Hannover Medical School
Hanover, Germany
Hospital Alvaro Cunqueiro Dr. Manuel Rodriquez-Lopez
Vigo, Spain
University Hospital Bern Inselspital
Bern, Switzerland