Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)

Last updated: November 28, 2024
Sponsor: Biocad
Overall Status: Active - Recruiting

Phase

1/2

Condition

Hemophilia

Treatment

ANB-002, dose 2

ANB-002, dose 1

ANB-002, dose 3

Clinical Study ID

NCT06120582
ANB-002-1
  • Ages > 18
  • Male

Study Summary

The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-002 in subjects with hemophilia В. The study will have a dose-escalation design with elements of phase I/II seamless adaptive design.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Male with hemophilia B.

  2. Age ≥18 years.

  3. FIX activity at screening ≤2% without FIX inhibitor.

  4. ≥150 previous exposure days of treatment with FIX concentrates.

Exclusion

Exclusion Criteria:

  1. Previous gene therapy.

  2. Other blood or hematopoietic disorders.

  3. Positive Anti-AAV5 antibodies (for Cohorts 1-3).

  4. Diagnosed HIV-infection, not controlled with anti-viral therapy.

  5. Hepatitis B (for Cohorts 1-3), acute or chronic hepatitis C.

  6. Any active systemic infections or recurrent infections requiring systemic therapy atscreening.

  7. Any other disorders associated with severe immunodeficiency.

  8. Significant hepatic disorders (liver cirrhosis, liver fibrosis, etc).

  9. Malignancies with remission <5 years.

Study Design

Total Participants: 28
Treatment Group(s): 3
Primary Treatment: ANB-002, dose 2
Phase: 1/2
Study Start date:
May 02, 2023
Estimated Completion Date:
November 30, 2029

Study Description

The study design includes ANB-002 dose escalation in at three cohorts. In Cohort 1, the subject is treated with a single dose of ANB-002 (dose 1) administered as an intravenous infusion. Follow-up for the assessment of dose-limiting toxicity (DLT) will be carried out for 28 days. If no DLT events are observed in the subject of Cohort 1, the following subjects will be included in Cohort 1.

In Cohort 2, the subjects are treated with a single dose of ANB-002 (dose 2). In Cohort 3, the subject are treated with a single dose of ANB-002 (dose 3).

The decision to continue enrolling in cohorts will be made at the Independent Data Monitoring Committee (IDMC) meeting. After the IDMC makes a decision regarding the dosing subjects, further enrolment to the cohort will be carried out.

Based on the data from the follow-up period of subjects included in Сohorts 1-3, a potential therapeutic dose for further study will be determined and additional patients will be included to recieve this dose.

In exploratory Cohort 4 patients with anti-AAV5 antibodies and/or hepatitis B in anamnesis will be included. These subjects will recieve the dose 3 of ANB-002.

The total duration of participation of one subjects in the study will be 5 years.

Connect with a study center

  • Republican Scientific and Practical Center for Radiation Medicine and Human Ecology

    Gomel, 246040
    Belarus

    Active - Recruiting

  • Minsk Scientific and Practical Center for Surgery, Transplantology and Hematology

    Minsk, 220089
    Belarus

    Active - Recruiting

  • State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital"

    Chelyabinsk, 454048
    Russian Federation

    Active - Recruiting

  • State budgetary healthcare institution Leningrad Regional Clinical Hospital

    Gatchina, 188300
    Russian Federation

    Active - Recruiting

  • Kuzbass Clinical Hospital named after S.V. Belyaev

    Kemerovo, 650066
    Russian Federation

    Active - Recruiting

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

    Kirov, 610027
    Russian Federation

    Active - Recruiting

  • 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

    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, 125167
    Russian Federation

    Active - Recruiting

  • LLC "Medis"

    Nizhny Novgorod, 603137
    Russian Federation

    Active - Recruiting

  • State Novosibirsk Regional Clinical Hospital

    Novosibirsk, 630087
    Russian Federation

    Active - Recruiting

  • Almazov National Medical Research Centre

    Saint Petersburg, 197341
    Russian Federation

    Active - Recruiting

  • City Polyclinic №37

    Saint Petersburg, 191186
    Russian Federation

    Active - Recruiting

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

    Saint Petersburg, 191024
    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

    Active - Recruiting

  • State Institution "Komi Republican Oncological Dispensary"

    Syktyvkar, 167904
    Russian Federation

    Active - Recruiting

  • 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

    Active - Recruiting

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