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

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

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.

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.

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 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 liver disorder
• Malignancies with less than 5 years of remission