SAFRAN is a multicenter, two-stage, open-label Phase 1/2 study investigating the safety, immunogenicity and efficacy of ANB-002 (arvenacogene sanparvovec) in people with hemophilia B, using a dose-escalation design across three dose levels with seamless adaptive elements. Eligible participants are men aged 18 or older with FIX activity of 2% or below, no FIX inhibitor, and at least 150 previous exposure days to FIX concentrates. Prior gene therapy, other blood or hematopoietic disorders, and positive anti-AAV5 antibodies are exclusions.
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: 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.