Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)

Launched by BIOCAD · Nov 4, 2023

Keywords

ClinConnect Summary

The SAFRAN clinical trial is studying a new treatment called ANB-002 for men with Hemophilia B, a genetic condition that affects blood clotting. This research aims to understand how safe the treatment is, how well it works, and whether it causes the body to develop any unwanted immune responses. The trial is currently looking for male participants aged 18 and older who have low levels of a specific protein needed for blood clotting and have received certain types of treatment in the past.

Eligible participants will be closely monitored throughout the study. They will receive ANB-002 and will be checked for any side effects and how their body responds to the treatment. It’s important to note that individuals with certain medical conditions, such as previous gene therapy or active infections, will not be eligible to participate. This trial represents a hopeful step towards better treatment options for those living with Hemophilia B.

Gender

MALE

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.