An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX (Etranacogene Dezaparvovec) in Patients With Hemophilia B

What this Trial Is About

Researchers are investigating the short- and long-term safety and effectiveness of HEMGENIX, a gene therapy for patients with hemophilia B. This observational, post-authorization, long-term follow-up study also includes a comparison group of patients receiving Factor IX (FIX) prophylaxis to better understand the results related to HEMGENIX treatment. The study is sponsored by CSL Behring and aims to provide important real-world data on these treatments. The study follows two groups: patients treated with commercial HEMGENIX in countries where it is approved, and patients with hemophilia B receiving FIX prophylaxis who are enrolled in a natural history cohort or similar registry. There is no intervention assigned by the study; instead, participants continue their usual treatment while their health and bleeding outcomes are observed over time. Participants will be monitored for up to 15 years, with regular assessments of bleeding rates, FIX activity levels, joint health, and use of replacement therapy. Researchers will collect data on adverse events and serious adverse events related to treatment. Regular follow-up visits will take place to evaluate long-term outcomes and safety, helping to characterize the effects of HEMGENIX compared to standard FIX prophylaxis.

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B