Gene therapy for hemophilia b: does it last? new study tracks 500 patients over years.

NCT ID NCT06008938

Recruiting now Disease control Sponsor: CSL Behring Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows 500 people with hemophilia B who received HEMGENIX gene therapy, plus a comparison group on standard factor IX treatment. Researchers will track bleeding rates and safety over several years to see if the gene therapy provides lasting control. The goal is to understand how well HEMGENIX works in the real world.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: HEMGENIX (etranacogene dezaparvovec) gene therapy
What this could lead to: If successful, this could confirm that a single dose of HEMGENIX provides long-lasting control of bleeding in hemophilia B, reducing or eliminating the need for regular factor IX infusions.
What could go wrong: This is an observational study, not a controlled trial, so results may be less definitive. Long-term safety and effectiveness are still being tracked, and some patients may not respond as well or may need ongoing treatment.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

hemophilia B

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HEMOPHILIA B

Contacts and locations

Locations

Aarhus Universitetshospital
RECRUITING

Århus N, Denmark
American Thrombosis and Hemostasis Network
RECRUITING

Rochester, New York, 14604, United States
CHU Nancy - Hôpital Brabois
RECRUITING

Vandœuvre-lès-Nancy, France
Centre Hospitalier Universitaire de Brest / CHU Morvan
RECRUITING

Brest, France
Centre Régional de Traitement de l'Hémophilie
RECRUITING

Nantes, France
Hannover Medical School
RECRUITING

Hanover, Germany
Hospital Alvaro Cunqueiro Dr. Manuel Rodriquez-Lopez
RECRUITING

Vigo, Spain
Klinik für Angiologie/ Hämostaseologie
RECRUITING

Berlin, Germany
Klinikum der Johann-Wolfgang Goethe Universitaet
RECRUITING

Frankfurt, Germany
Medical University Vienna
RECRUITING

Vienna, Austria
University Hospital Bern Inselspital
RECRUITING

Bern, Switzerland
University of Clinic Bonn
RECRUITING

Bonn, Germany

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