This Phase 3 study compares a single dose of ANB-002 (arvenacogene sanparvovec) with preventive coagulation factor IX (FIX) treatment in adults with hemophilia B, aiming to demonstrate non-inferiority. The study uses an open-label, single-arm design. Eligible participants are men aged 18 or older with FIX activity of 2% or less, no FIX inhibitor, and at least 150 previous exposure days to FIX concentrates; a history of prior gene therapy is an exclusion.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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