An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B
NCT ID: NCT06008938
Last Updated: 2025-12-26
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
500 participants
OBSERVATIONAL
2023-06-15
2043-08-01
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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HEMGENIX
Patients with hemophilia B treated with HEMGENIX in countries where HEMGENIX is approved for commercial use.
HEMGENIX
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
Patients with hemophilia B on FIX prophylaxis and enrolled in American Thrombosis and Hemostasis Network (ATHN) Transcends (A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment in People with Non-Neoplastic Hematologic Disorders) Hemophilia Cohort, or a similar registry.
Factor IX (FIX)
FIX prophylaxis therapy
Interventions
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HEMGENIX
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.
Factor IX (FIX)
FIX prophylaxis therapy
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* \- 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
* \- The patient population that will be observed in this study must not have been treated with etranacogene dezaparvovec in a clinical trial.
18 Years
ALL
No
Sponsors
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CSL Behring
INDUSTRY
Responsible Party
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Principal Investigators
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Global Program Director
Role: STUDY_DIRECTOR
CSL Behring
Locations
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American Thrombosis and Hemostasis Network
Rochester, New York, United States
Medical University Vienna
Vienna, , Austria
Aarhus Universitetshospital
Århus N, , Denmark
Centre Hospitalier Universitaire de Brest / CHU Morvan
Brest, , France
Centre Régional de Traitement de l'Hémophilie
Nantes, , France
CHU Nancy - Hôpital Brabois
Vandœuvre-lès-Nancy, , France
Klinik für Angiologie/ Hämostaseologie
Berlin, , Germany
Hannover Medical School
Hanover, , Germany
Hospital Alvaro Cunqueiro Dr. Manuel Rodriquez-Lopez
Vigo, , Spain
University Hospital Bern Inselspital
Bern, , Switzerland
Countries
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Central Contacts
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Other Identifiers
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EUPAS106066
Identifier Type: REGISTRY
Identifier Source: secondary_id
CSL222_4001
Identifier Type: -
Identifier Source: org_study_id