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RECRUITING
NCT06008938

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

Sponsor: CSL Behring

View on ClinicalTrials.gov

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.

Official title: An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

OBSERVATIONAL

Enrollment

500

Start Date

2023-06-15

Completion Date

2043-08-01

Last Updated

2026-02-19

Healthy Volunteers

No

Conditions

Interventions

GENETIC

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.

BIOLOGICAL

Factor IX (FIX)

FIX prophylaxis therapy

Locations (12)

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

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