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

NCT ID NCT06008938

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 500 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2023

Expected to finish

Aug 2043

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Patients with hemophilia B who have been treated with HEMGENIX and are enrolled in either the sponsor's study or the ATHN Transcends study NCT04398628 (Hemophilia Cohort, Gene Therapy Outcomes Arm), and patients who receive routine prophylaxis treatment with FIX replacement therapy that are enrolled in the ATHN Transcends study (Hemophilia Cohort, Natural History Arm), or a similar registry. All patients will provide signed informed consent required for participation.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    12 sites in 7 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

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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