Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients
Condition: Hemophilia B
MeSH terms: Hemophilia A; Hemophilia B
Intervention: BAX 326; BeneFIX
Intervention description: Study Part 1: Pharmacokinetic (PK) Crossover with BAX326 and BeneFIX
Study Part 2: Open-label evaluation of prophylaxis and on-demand BAX326 only
Study Part 3: Open-label repeat of PK evaluation (repeat Study Part 1) with BAX326 only and same study participants as Study Part 1; Study Part 1: Pharmacokinetic (PK) Crossover with BAX326 and BeneFIX.
BeneFIX only used in Part 1 of this study.
Study Part 2 and 3 only utilized BAX326
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Double (Participant, Investigator)
Enrollment: 86.0
Sex: All
Minimum age: 12 Years
Maximum age: 65 Years
Healthy volunteers: No

Summary:

      The purpose of this pivotal Phase 1/3 study is to determine the pharmacokinetic (PK)
      parameters, the hemostatic efficacy, and the safety of BAX 326, a recombinant factor IX, in
      previously treated patients (PTPs) with severe and moderately severe hemophilia B.
    

Eligibility criteria:

        Main Inclusion Criteria:

          -  Participant is 12 to 65 years old at the time of screening

          -  Participant and/or legal representative has/have provided signed informed consent

          -  Participant has severe (factor IX (FIX) level < 1%) or moderately severe (FIX level
             1-2%) hemophilia B (based on the one stage activated partial thromboplastin time
             (aPTT) assay), as tested at screening at the central laboratory

          -  Participant is previously treated with plasma-derived or recombinant FIX
             concentrate(s) for a minimum of 150 exposure days (EDs) (based on the participant's
             medical records); if a verifiable, documented history is unavailable, the participant
             can be enrolled if s/he has 100-150 EDs to any FIX product that are not fully
             documented and has participated in Study 050901 for at least 50 EDs to Immunine prior
             to enrollment (not valid for US and Japan).

          -  Participant has no evidence of a history of FIX inhibitors

          -  If the participant is to receive prophylactic treatment, the participant is willing to
             receive prophylactic treatment over a period of 6 months.

          -  If the participant is to receive on-demand treatment, the participant has ≥12
             documented bleeding episodes requiring treatment within 12 months prior to enrollment
             and is willing to receive on-demand treatment for the duration of this study.

        Main Exclusion Criteria:

          -  The participant has a history of FIX inhibitors with a titer ≥0.6 Bethesda Units (BU)
             (as determined by the Nijmegen modification of the Bethesda assay or the assay
             employed in the respective local laboratory) at any time prior to screening

          -  The participant has a detectable FIX inhibitor at screening, with a titer ≥0.6 BU as
             determined by the Nijmegen modification of the Bethesda assay in the central
             laboratory

          -  The participant's weight is < 35 kg or > 120 kg

          -  The participant has a history of allergic reaction, eg, anaphylaxis, following
             exposure to FIX concentrate(s)

          -  The participant has a known hypersensitivity to hamster proteins or recombinant furin
             (rFurin)

          -  The participant has ongoing or recent evidence of a thrombotic disease, fibrinolysis
             or disseminated intravascular coagulation (DIC)