Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)
Trial Parameters
Brief 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 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 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 at screening. 7. Any other disorders associated with severe immunodeficiency. 8. Significant hepatic disorders (liver cirrhosis, liver fibrosis, etc). 9. Malignancies with remission \<5 years.