Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)
An Open-label, Non-comparative, Single Dose-Escalation Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B
Biocad
28 participants
May 2, 2023
INTERVENTIONAL
Conditions
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
Inclusion Criteria4
- Male with hemophilia B.
- Age ≥18 years.
- FIX activity at screening ≤2% without FIX inhibitor.
- ≥150 previous exposure days of treatment with FIX concentrates.
Exclusion Criteria9
- Previous gene therapy.
- Other blood or hematopoietic disorders.
- Positive Anti-AAV5 antibodies (for Cohorts 1-3).
- Diagnosed HIV-infection, not controlled with anti-viral therapy.
- Hepatitis B (for Cohorts 1-3), acute or chronic hepatitis C.
- Any active systemic infections or recurrent infections requiring systemic therapy at screening.
- Any other disorders associated with severe immunodeficiency.
- Significant hepatic disorders (liver cirrhosis, liver fibrosis, etc).
- Malignancies with remission <5 years.
Interested in this trial?
Get notified about updates and connect with the research team.
Interventions
Adeno-associated viral vector carrying the FIX gene single infusion at dose 1.
Adeno-associated viral vector carrying the FIX gene single infusion at dose 2.
Adeno-associated viral vector carrying the FIX gene single infusion at dose 3.
Locations(17)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06120582