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
- Vigo, SpainSee the site
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The study follows adults with hemophilia B who were treated with HEMGENIX, and adults with hemophilia B who are on FIX prophylaxis (regular factor IX treatment to prevent bleeding).
- Age
- 18 years or older (the age is given for the FIX prophylaxis group)
- Sex
- All sexes can join.
- Disease stage
- Hemophilia B.
- Earlier treatment
- HEMGENIX group: treated with commercial HEMGENIX. FIX group: receiving FIX prophylaxis therapy.
Main reasons for not taking part
- HEMGENIX group: treated with etranacogene dezaparvovec in a clinical trial
Read by machine on 9 November 2026.
Where in Spain
- Hospital Alvaro Cunqueiro Dr. Manuel Rodriquez-LopezVigo, SpainTaking 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?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial 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:.