One-Time gene therapy could free hemophilia b patients from frequent infusions
NCT ID NCT03569891
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.
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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Locations
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Amsterdam UMC - Locatie AMC
Amsterdam, 1105 AZ, Netherlands
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Bloodworks Northwest
Seattle, Washington, 98104, United States
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Center for Thrombosis and Hemostasis Skåne University Hospital Malmö
Malmö, SE-205 02, Sweden
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Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
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Children's National Medical Center Hematology and Oncology
Washington D.C., District of Columbia, 20010, United States
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Cliniques Universitaires Saint-Luc
Brussels, 1200, Belgium
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Erasmus MC
Rotterdam, 3015 CE, Netherlands
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Hemophilia Center of Western New York
Buffalo, New York, 14209, United States
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Klinikum der Johann Wolfgang Goethe Universitat
Frankfurt am Main, 60590, Germany
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Los Angeles Orthopedic Hospital
Los Angeles, California, 90007, United States
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National Coagulation Centre, St James's Hospital
Dublin, D08 A978, Ireland
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Oregon Health & Science University
Portland, Oregon, 97239, United States
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Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
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Righospitalet
Copenhagen, 2100, Denmark
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The Cambridge Haemophilia and Thrombophilia Centre Camridge University Hospitals NHS Foundation Trust - Box 217 Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
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The Royal London Hospital (Barts Health NHS Trust)
London, E1 2ES, United Kingdom
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UMC Utrecht, Van Creveldkliniek
Utrecht, 3508 GA, Netherlands
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Universitair Medisch Centrum Groningen
Groningen, 9713 GZ, Netherlands
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University Hospital Leuven
Leuven, 3000, Belgium
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University Hospital Southampton NHS Foundation Trust
Southampton, SO16 6YD, United Kingdom
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University of California, Davis
Sacramento, California, 95817, United States
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University of California, San Diego
San Diego, California, 92161, United States
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University of Colorado Denver
Aurora, Colorado, 80045, United States
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University of Michigan
Ann Arbor, Michigan, 48109, United States
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University of North Carolina, Chapel Hill
Chapel Hill, North Carolina, 27599, United States
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University of South Florida
Tampa, Florida, 33612, United States
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University of Tennessee Health Science Center
Memphis, Tennessee, 38163, United States
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University of Texas Health Science Center & Medical School
Houston, Texas, 77030, United States
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University of Utah
Salt Lake City, Utah, 84108, United States
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Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
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Vivantes Klinikum im Friedrichshain
Berlin, 10249, Germany
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Washington Institute for Coagulation
Seattle, Washington, 98101, United States