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 groupsSponsors and collaborators
Source ClinicalTrials.gov