One-Shot gene therapy could free hemophilia b patients from frequent injections

NCT ID NCT06700096

Recruiting now Disease control Sponsor: Biocad Source: ClinicalTrials.gov ↗

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.

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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

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Locations

Almazov National Medical Research Centre
RECRUITING

Saint Petersburg, 197341, Russia
Chelyabinsk Regional Clinical Hospital
RECRUITING

Chelyabinsk, Russia
Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"
RECRUITING

Moscow, 125284, Russia
Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation
RECRUITING

Ufa, 450008, Russia
Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Healthcare of the Russian Federation
RECRUITING

Samara, 443099, Russia

Contact Email: •••••@•••••
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)
RECRUITING

Moscow, 125167, Russia
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)
RECRUITING

Moscow, 125167, Russia
Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"
RECRUITING

Kirov, 610027, Russia
King Chulalongkorn Memorial Hospital
NOT_YET_RECRUITING

Bangkok, 10330, Thailand
Ramathibodi Hospital
NOT_YET_RECRUITING

Bangkok, 10400, Thailand
Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency
RECRUITING

Saint Petersburg, 193024, Russia
Siriraj Hospital
NOT_YET_RECRUITING

Bangkok, 10700, Thailand
State Novosibirsk Regional Clinical Hospital
RECRUITING

Novosibirsk, 630087, Russia
State budgetary healthcare institution Leningrad Regional Clinical Hospital
RECRUITING

Saint Petersburg, 188663, Russia

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