An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B
CSL Behring
Summary
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.
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
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…
Interventions
- GeneticHEMGENIX
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.
- BiologicalFactor IX (FIX)
FIX prophylaxis therapy
Locations (12)
- American Thrombosis and Hemostasis NetworkRochester, New York
- Medical University ViennaVienna
- Aarhus UniversitetshospitalÅrhus N
- Centre Hospitalier Universitaire de Brest / CHU MorvanBrest
- Centre Régional de Traitement de l'HémophilieNantes
- CHU Nancy - Hôpital BraboisVandœuvre-lès-Nancy