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
CSL Behring
500 participants
Jun 15, 2023
OBSERVATIONAL
Conditions
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
Inclusion Criteria5
- 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 Criteria2
- HEMGENIX Cohort:
- \- The patient population that will be observed in this study must not have been treated with etranacogene dezaparvovec in a clinical trial.
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Interventions
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.
FIX prophylaxis therapy
Locations(12)
View Full Details on ClinicalTrials.gov
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NCT06008938