Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)
ConditionHemophilia B
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexMale
Age18+
SponsorBiocad
About this trial
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 criteria
Qualifiers
Male with hemophilia B.
Age ≥18 years.
FIX activity at screening ≤2% without FIX inhibitor.
≥150 previous exposure days of treatment with FIX concentrates.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- ANB-002, dose 1
- ANB-002, dose 2
- ANB-002, dose 3
Treatment groups
28 Participants
are divided into 4 treatment groupsLocations
17Belarus
Republican Scientific and Practical Center for Radiation Medicine and Human Ecology246040, Homyel Belarus
Minsk Scientific and Practical Center for Surgery, Transplantology and Hematology220089, Minsk Belarus
Russia
State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital"454048, Chelyabinsk Russia
State budgetary healthcare institution Leningrad Regional Clinical Hospital188300, Gatchina Russia
Sponsors and collaborators
Biocad
Lead sponsor
Source ClinicalTrials.gov