Gene therapy for hemophilia b: does it last? new study tracks 500 patients over years.
NCT ID NCT06008938
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Aarhus Universitetshospital
RECRUITINGÅrhus N, Denmark
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American Thrombosis and Hemostasis Network
RECRUITINGRochester, New York, 14604, United States
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CHU Nancy - Hôpital Brabois
RECRUITINGVandœuvre-lès-Nancy, France
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Centre Hospitalier Universitaire de Brest / CHU Morvan
RECRUITINGBrest, France
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Centre Régional de Traitement de l'Hémophilie
RECRUITINGNantes, France
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Hannover Medical School
RECRUITINGHanover, Germany
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Hospital Alvaro Cunqueiro Dr. Manuel Rodriquez-Lopez
RECRUITINGVigo, Spain
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Klinik für Angiologie/ Hämostaseologie
RECRUITINGBerlin, Germany
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Klinikum der Johann-Wolfgang Goethe Universitaet
RECRUITINGFrankfurt, Germany
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Medical University Vienna
RECRUITINGVienna, Austria
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University Hospital Bern Inselspital
RECRUITINGBern, Switzerland
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University of Clinic Bonn
RECRUITINGBonn, Germany