Health Trials

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

This trial does not accept healthy volunteers

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

Design model

Sequential

Treatments tested in this trial

  • ANB-002, dose 1

    Genetic

    Adeno-associated viral vector carrying the FIX gene single infusion at dose 1.

  • ANB-002, dose 2

    Genetic

    Adeno-associated viral vector carrying the FIX gene single infusion at dose 2.

  • ANB-002, dose 3

    Genetic

    Adeno-associated viral vector carrying the FIX gene single infusion at dose 3.

Treatment groups

28 Participants
are divided into 4 treatment groups
Group A: Cohort 1Experimental treatment 1 intervention
Group B: Cohort 2Experimental treatment 1 intervention
Group C: Cohort 3Experimental treatment 1 intervention
Group D: Cohort 4Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Change in FIX activity from baseline

Time frame
12 months; final assessment - 5 years
2

Proportion of subjects with adverse reactions

Time frame
12 months; final assessment - 5 years

Secondary outcomes

1

Changes in FIX activity

Time frame
5 years
2

Annualized use of FIX concentrates

Time frame
5 years
3

Annualized bleeding rate

Time frame
5 years
4

Annualized rate of bleedings requiring therapy with FIX concentrates

Time frame
5 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Biocad

Lead sponsor

Source ClinicalTrials.gov
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