RecruitingNCT06008938

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

Sponsor

CSL Behring

Enrollment

500 participants

Start Date

Jun 15, 2023

Study Type

OBSERVATIONAL

Conditions

Hemophilia B

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

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a biological treatment called Factor IX (FIX) and HEMGENIX for people with hemophilia b. The study is currently recruiting participants at 12 locations. People eligible for this study include aged 18 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICHEMGENIX

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.

BIOLOGICALFactor 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

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NCT06008938

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