An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B

Last updated: November 17, 2025
Sponsor: Biocad
Overall Status: Active - Recruiting

Phase

3

Condition

Hemophilia

Treatment

ANB-002

Clinical Study ID

NCT06700096
ANB-002-2
  • Ages > 18
  • Male

Study Summary

The aim of the study is to demonstrate non-inferiority of ANB-002 compared with preventive use of coagulation factor IX (FIX) in adult subjects with hemophilia B with FIX activity ≤2% and without FIX inhibitor. The study will have an open-label single-arm design.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Men diagnosed with hemophilia B aged 18 or older

  • FIX activity ≤2%

  • Absense of FIX inhibitor

  • ≥150 previous exposure days of treatment with FIX concentrates

Exclusion

Exclusion Criteria:

  • Any diseases of blood and hematopoietic organs other than hemophilia B

  • A history of any gene therapy, including ANB-002

  • Diagnosed HIV-infection, not controlled with anti-viral therapy

  • Active HBV or HCV infection

  • Anti-AAV5 antibodies

  • Any active systemic infections or recurrent infections requiring systemic therapy

  • Any other disorders associated with severe immunodeficiency

  • Relevant hepatic disorders or conditions that can be a symptom of existing liverdisorder

  • Malignancies with less than 5 years of remission

Study Design

Total Participants: 24
Treatment Group(s): 1
Primary Treatment: ANB-002
Phase: 3
Study Start date:
November 07, 2024
Estimated Completion Date:
March 31, 2032

Study Description

After signing the ICF and screening examination the subjects are to be included in the Non-interventional lead-in period in which the subject will receive standard FIX prevention. The lead-in period will last for at least 6 month for every subject.

After completiong the lead-in period subjects will enter the main (interventional) period.

At the first visit of the main period subjects will recieve investigational product ANB-002.

The main period ends 18 months after the administration of ANB-002, after which subjects will switch to the follow-up period and will be evaluated up to 5 years after the ANB-002 infusion.

Connect with a study center

  • Chelyabinsk Regional Clinical Hospital

    Chelyabinsk 1508291,
    Russia

    Active - Recruiting

  • Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"

    Kirov 548408, 610027
    Russia

    Active - Recruiting

  • Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"

    Moscow 524901, 125284
    Russia

    Active - Recruiting

  • 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)

    Moscow 524901, 125167
    Russia

    Active - Recruiting

  • 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)

    Moscow 524901, 125167
    Russia

    Active - Recruiting

  • State Novosibirsk Regional Clinical Hospital

    Novosibirsk 1496747, 630087
    Russia

    Active - Recruiting

  • Almazov National Medical Research Centre

    Saint Petersburg 498817, 197341
    Russia

    Active - Recruiting

  • Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency

    Saint Petersburg 498817, 193024
    Russia

    Active - Recruiting

  • State budgetary healthcare institution Leningrad Regional Clinical Hospital

    Saint Petersburg 498817, 188663
    Russia

    Active - Recruiting

  • Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Healthcare of the Russian Federation

    Samara 499099, 443099
    Russia

    Active - Recruiting

  • Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation

    Ufa 479561, 450008
    Russia

    Active - Recruiting

  • Chelyabinsk Regional Clinical Hospital

    Chelyabinsk,
    Russian Federation

    Site Not Available

  • Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"

    Kirov, 610027
    Russian Federation

    Site Not Available

  • Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"

    Moscow, 125284
    Russian Federation

    Active - Recruiting

  • 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)

    Moscow, 125167
    Russian Federation

    Site Not Available

  • 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)

    Moscow, 125167
    Russian Federation

    Active - Recruiting

  • State Novosibirsk Regional Clinical Hospital

    Novosibirsk, 630087
    Russian Federation

    Site Not Available

  • Almazov National Medical Research Centre

    Saint Petersburg, 197341
    Russian Federation

    Site Not Available

  • Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency

    Saint Petersburg, 193024
    Russian Federation

    Active - Recruiting

  • State budgetary healthcare institution Leningrad Regional Clinical Hospital

    Saint Petersburg, 188663
    Russian Federation

    Active - Recruiting

  • Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Healthcare of the Russian Federation

    Samara, 443099
    Russian Federation

    Site Not Available

  • Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian Federation

    Ufa, 450008
    Russian Federation

    Site Not Available

  • King Chulalongkorn Memorial Hospital

    Bangkok 1609350, 10330
    Thailand

    Site Not Available

  • Ramathibodi Hospital

    Bangkok 1609350, 10400
    Thailand

    Site Not Available

  • Siriraj Hospital

    Bangkok 1609350, 10700
    Thailand

    Site Not Available

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