Tests ANB-002 gene therapy in adults with hemophilia B
Tests the ANB-002 gene therapy in adults with hemophilia B.
Plain-language title generated from the registry record
Registry title: Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)
Key facts
- Where
- Chelyabinsk, Gatchina, Kemerovo, Kirov, Moscow, Nizhny Novgorod, Novosibirsk, Saint Petersburg, Samara, Syktyvkar, and Ufa, RussiaSee the 15 sites
- Phase
- Phase 1/2
Where in Russia
- State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital"Chelyabinsk, RussiaTaking people
- State budgetary healthcare institution Leningrad Regional Clinical HospitalGatchina, RussiaTaking people
- Kuzbass Clinical Hospital named after S.V. BelyaevKemerovo, RussiaTaking people
- Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency"Kirov, RussiaTaking people
- Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin"Moscow, RussiaTaking people
- 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, RussiaTaking people
- 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, RussiaTaking people
- LLC "Medis"Nizhny Novgorod, RussiaTaking people
- State Novosibirsk Regional Clinical HospitalNovosibirsk, RussiaTaking people
- Almazov National Medical Research CentreSaint Petersburg, RussiaTaking people
- City Polyclinic №37Saint Petersburg, RussiaTaking people
- Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological AgencySaint Petersburg, RussiaTaking people
- Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Healthcare of the Russian FederationSamara, RussiaTaking people
- State Institution "Komi Republican Oncological Dispensary"Syktyvkar, RussiaTaking people
- Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Healthcare of the Russian FederationUfa, RussiaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Biocad
- Trial code
- NCT06120582
- Record updated
- 13 June 2025
- Original description
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.Read full original descriptionShow less
An Open-label, Non-comparative, Single Dose-Escalation Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B
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.
Inclusion: Male with hemophilia B; Age ≥18 years; FIX activity at screening ≤2% without FIX inhibitor; ≥150 previous exposure days of treatment with FIX concentrates.
Exclusion: Previous gene therapy; Positive Anti-AAV5 antibodies (for Cohorts 1-3); Other blood or hematopoietic disorders; Diagnosed HIV-infection, not controlled with anti-viral therapy; Hepatitis B (for Cohorts 1-3), acute or chronic hepatitis C; Any active systemic infections or recurrent infections requiring systemic therapy at screening; Any other disorders associated with severe immunodeficiency; Significant hepatic disorders (liver cirrhosis, liver fibrosis, etc); Malignancies with remission <5 years.