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One-Time gene therapy could free hemophilia b patients from frequent infusions

NCT ID NCT03569891

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 trial tested a one-time gene therapy called AMT-061 in 67 men with severe or moderately severe hemophilia B. The therapy uses a harmless virus to deliver a working copy of the gene for clotting factor IX, so the body can produce its own. The main goal was to see if it reduces bleeding episodes and the need for factor IX infusions.

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 (AMT-061, etranacogene dezaparvovec)
What this could lead to
If successful, this gene therapy could allow people with hemophilia B to produce their own clotting factor, greatly reducing or eliminating the need for regular factor IX infusions.
What could go wrong
This is a single-dose, open-label trial without a placebo group, so results may be less definitive. Long-term safety and durability of the effect are still being studied, and there is a risk of liver inflammation or immune reactions.

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.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

67 people

The number who actually took part.

Started

Jun 2018

Finished

Mar 2025

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.

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: 1. Male 2. Age ≥18 years 3. Subjects with congenital hemophilia B, classified as severe or moderately severe, and are currently on factor IX prophylaxis 4. \>150 previous exposure days of treatment with factor IX protein Exclusion Criteria: 1. History of factor IX inhibitors 2. Positive factor IX inhibitor test at screening 3. Select screening laboratory value \>2 times upper limit of normal 4. Positive human immunodeficiency virus (HIV) test at screening, not controlled with anti-viral therapy 5. Active infection with hepatitis B or C virus at screening 6. History of Hepatitis B or C exposure, currently controlled by antiviral therapy at the end of the lead-in phase 7. Previous gene therapy treatment 8. Receipt of an experimental agent within 60 days prior to screening 9. Current participation or anticipated participation within one year after study drug administration in this trial in any other interventional clinical trial involving drugs or devices

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Conditions

The condition(s) this trial relates to.

hemophilia A hemophilia B Hemorrhage

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, 1105 AZ, Netherlands

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Bloodworks Northwest

    Seattle, Washington, 98104, United States

  • Center for Thrombosis and Hemostasis Skåne University Hospital Malmö

    Malmö, SE-205 02, Sweden

  • Children's Hospital of Los Angeles

    Los Angeles, California, 90027, United States

  • Children's National Medical Center Hematology and Oncology

    Washington D.C., District of Columbia, 20010, United States

  • Cliniques Universitaires Saint-Luc

    Brussels, 1200, Belgium

  • Erasmus MC

    Rotterdam, 3015 CE, Netherlands

  • Hemophilia Center of Western New York

    Buffalo, New York, 14209, United States

  • Klinikum der Johann Wolfgang Goethe Universitat

    Frankfurt am Main, 60590, Germany

  • Los Angeles Orthopedic Hospital

    Los Angeles, California, 90007, United States

  • National Coagulation Centre, St James's Hospital

    Dublin, D08 A978, Ireland

  • Oregon Health & Science University

    Portland, Oregon, 97239, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • Righospitalet

    Copenhagen, 2100, Denmark

  • The Cambridge Haemophilia and Thrombophilia Centre Camridge University Hospitals NHS Foundation Trust - Box 217 Addenbrooke's Hospital

    Cambridge, CB2 0QQ, United Kingdom

  • The Royal London Hospital (Barts Health NHS Trust)

    London, E1 2ES, United Kingdom

  • UMC Utrecht, Van Creveldkliniek

    Utrecht, 3508 GA, Netherlands

  • Universitair Medisch Centrum Groningen

    Groningen, 9713 GZ, Netherlands

  • University Hospital Leuven

    Leuven, 3000, Belgium

  • University Hospital Southampton NHS Foundation Trust

    Southampton, SO16 6YD, United Kingdom

  • University of California, Davis

    Sacramento, California, 95817, United States

  • University of California, San Diego

    San Diego, California, 92161, United States

  • University of Colorado Denver

    Aurora, Colorado, 80045, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109, United States

  • University of North Carolina, Chapel Hill

    Chapel Hill, North Carolina, 27599, United States

  • University of South Florida

    Tampa, Florida, 33612, United States

  • University of Tennessee Health Science Center

    Memphis, Tennessee, 38163, United States

  • University of Texas Health Science Center & Medical School

    Houston, Texas, 77030, United States

  • University of Utah

    Salt Lake City, Utah, 84108, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

  • Vivantes Klinikum im Friedrichshain

    Berlin, 10249, Germany

  • Washington Institute for Coagulation

    Seattle, Washington, 98101, United States

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