One-Shot gene therapy could free hemophilia b patients from frequent injections
NCT ID NCT06700096
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 3 trial is testing a single dose of ANB-002, a gene therapy that delivers a working copy of the FIX gene, in 24 adults with hemophilia B. The goal is to see if it can reduce bleeding episodes compared to regular preventive factor IX infusions. Participants will be followed for up to 18 months after the infusion.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
ANB-002 (arvenacogene sanparvovec) - a gene therapy that delivers a working FIX gene via a single infusion
What this could lead to
If successful, this could offer a long-term reduction in bleeding episodes and decrease the need for regular factor IX injections for people with hemophilia B.
What could go wrong
This is a small, early-phase trial with only 24 participants, so results may not apply to everyone. Gene therapy carries risks like immune reactions or the treatment not lasting long-term.
Disclaimer
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This is a summary of
the original study
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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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Almazov National Medical Research Centre
RECRUITINGSaint Petersburg, 197341, Russia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Chelyabinsk Regional Clinical Hospital
RECRUITINGChelyabinsk, Russia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"
RECRUITINGMoscow, 125284, Russia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation
RECRUITINGUfa, 450008, Russia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Healthcare of the Russian Federation
RECRUITINGSamara, 443099, Russia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Federal State Budgetary Institution "National Medical Research Centre for Hematology" of the Ministry of Health of Russian Federation (Department of Clinical Diagnostics for Hematology and Hemostasis Disorders)
RECRUITINGMoscow, 125167, Russia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Federal State Budgetary Institution "National Medical Research Centre for Hematology" of the Ministry of Health of Russian Federation (Department of Traumatology and Reconstructive and Restorative Orthopedics for Patients with Hemophilia)
RECRUITINGMoscow, 125167, Russia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"
RECRUITINGKirov, 610027, Russia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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King Chulalongkorn Memorial Hospital
NOT_YET_RECRUITINGBangkok, 10330, Thailand
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Ramathibodi Hospital
NOT_YET_RECRUITINGBangkok, 10400, Thailand
Contact Email: •••••@•••••
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Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency
RECRUITINGSaint Petersburg, 193024, Russia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Siriraj Hospital
NOT_YET_RECRUITINGBangkok, 10700, Thailand
Contact Phone: •••-•••-•••• Email: •••••@•••••
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State Novosibirsk Regional Clinical Hospital
RECRUITINGNovosibirsk, 630087, Russia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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State budgetary healthcare institution Leningrad Regional Clinical Hospital
RECRUITINGSaint Petersburg, 188663, Russia
Contact Phone: •••-•••-•••• Email: •••••@•••••