Status:
RECRUITING
Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)
Lead Sponsor:
Biocad
Conditions:
Hemophilia B
Eligibility:
MALE
18+ years
Phase:
PHASE1
PHASE2
Brief 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 des...
Detailed Description
The study design includes ANB-002 dose escalation in at three cohorts. In Cohort 1, the subject is treated with a single dose of ANB-002 (dose 1) administered as an intravenous infusion. Follow-up for...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
May 2 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
November 1 2029
Estimated Enrollment :
28 Patients enrolled
Trial Details
Trial ID
NCT06120582
Start Date
May 2 2023
End Date
November 1 2029
Last Update
June 13 2025
Active Locations (17)
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1
Republican Scientific and Practical Center for Radiation Medicine and Human Ecology
Homyel, Belarus, 246040
2
Minsk Scientific and Practical Center for Surgery, Transplantology and Hematology
Minsk, Belarus, 220089
3
State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital"
Chelyabinsk, Russia, 454048
4
State budgetary healthcare institution Leningrad Regional Clinical Hospital
Gatchina, Russia, 188300