Long-term follow-up of a gene therapy for hemophilia B
Follows adults with hemophilia B after treatment with the gene therapy Hemgenix.
Plain-language title generated from the registry record
Registry title: An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B
Key facts
- Where
- Berlin, Bonn, Frankfurt, and Hanover, GermanySee the 4 sites
Where in Germany
- Klinik für Angiologie/ HämostaseologieBerlin, GermanyTaking people
- University of Clinic BonnBonn, GermanyTaking people
- Klinikum der Johann Wolfgang Goethe-UniversitaetFrankfurt, GermanyTaking people
- Hannover Medical SchoolHanover, GermanyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- CSL Behring
- Trial code
- NCT06008938
- Record updated
- 19 February 2026
- Original description
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.Read full original descriptionShow less
An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B
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.
Inclusion: 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: - The patient population that will be observed in this study must not have been treated with etranacogene dezaparvovec in a clinical trial; HEMGENIX Cohort:.