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NCT06008938Recruiting

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

CSL Behring

Start Date

6/15/2023

Completion Date

8/1/2043

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 Criteria

Age Range: 18 years to No maximum

Inclusion Criteria: * 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 Criteria: * HEMGENIX Cohort: * \- The patient population that will be observed in this study must not have been treated with etranacogene dezaparvovec in a clinical trial.

Interventions

GENETIC

HEMGENIX

BIOLOGICAL

Factor IX (FIX)

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Conditions

Hemophilia B

Locations

American Thrombosis and Hemostasis Network

Rochester, New York 14604

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

University of Clinic Bonn

Bonn,

Germany

Klinikum der Johann-Wolfgang Goethe Universitaet

Frankfurt,

Germany

Hannover Medical School

Hanover,

Germany

Hospital Alvaro Cunqueiro Dr. Manuel Rodriquez-Lopez

Vigo,

Spain

University Hospital Bern Inselspital

Bern,

Switzerland