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

Launched by BIOCAD · Nov 19, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called ANB-002 for men with hemophilia B, a condition where the blood doesn’t clot properly. The goal is to see if a single dose of ANB-002 is as effective as the standard treatment with factor IX, which is used to help prevent bleeding. The study is currently looking for adult men aged 18 and older who have low levels of factor IX activity, specifically 2% or less, and who have not developed inhibitors that would make standard treatments less effective.

Participants in this trial can expect to receive ANB-002 and will be monitored for its effectiveness and safety. To be eligible, men must have had at least 150 previous treatments with factor IX concentrates and should not have other serious blood diseases or infections. This study is important because it could offer new insights into treatment options for people with hemophilia B, helping to improve their quality of life.

Gender

MALE

Eligibility criteria

• 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