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Gene therapy for hemophilia b shows promise in Long-Term study

NCT ID NCT05360706

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 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This study follows 9 adults with severe or moderately severe hemophilia B who received a one-time gene therapy (AAV5-hFIX) to help their bodies produce clotting factor IX. Researchers are checking if the treatment remains safe and effective for up to 10 years after the initial dose. The goal is to see if this gene therapy can reduce or prevent bleeding episodes over the long term.

What this could mean

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

Active substance
gene therapy (AAV5-hFIX) to produce clotting factor IX
What this could lead to
If successful, this could show that a single gene therapy treatment provides long-lasting control of bleeding episodes, reducing or eliminating the need for regular factor IX infusions.
What could go wrong
This is a small extension study with only 9 participants, so results may not apply to everyone. Long-term safety concerns, such as liver effects or loss of effectiveness over time, are still being monitored.

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

9 people

The number who actually took part.

Started

Mar 2021

Finished

Mar 2026

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

Male subjects with severe or moderately severe haemophilia B, who previously received an infusion of AMT-060 and completed all assessments in Study CTAMT-060-01

Ages

18 years and older

Sex

Male participants only

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: * \- Subjects with congenital hemophilia B who completed Study CTAMT-060-01 * \- Able to provide informed consent following receipt of verbal and written * information about the trial. Exclusion Criteria: * \- Enrolled subjects will have already been assessed based on the exclusion * criteria for Study CT-AMT-060-01.

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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

  • Amsterdam UMC - Locatie AMC

    Amsterdam, Netherlands

  • Erasmus MC

    Rotterdam, Netherlands

  • Groningen UMC

    Groningen, Netherlands

  • Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitae

    Frankfurt, Germany

  • Universitair Medisch Centrum Utrecht

    Utrecht, Netherlands

  • Vivantes Klinikum im Friedrichshain

    Berlin, Germany

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