Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B
Condition: Hemophilia B
MeSH terms: Hemophilia A; Hemophilia B
Intervention: IB1001
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 9.0
Sex: All
Maximum age: 12 Years
Healthy volunteers: No
Keywords: Recombinant Factor IX

Summary:

      The Study's Primary Objective is to evaluate the pharmacokinetics, safety (acute effects
      associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and
      control of hemorrhaging during prophylaxis) of IB1001 in previously treated pediatric
      subjects with hemophilia B.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Subject's parent or legal guardian must give written Institutional Review Board
             (IRB)/Independent Ethics Committee (IEC)-approved informed consent and be willing to
             make the required study visits and follow instructions while enrolled in the study.
             For subjects ≥7 years of age, assent will be obtained if required by the institution.
             For subjects < 7 years of age, legal assent is not reasonable to obtain.

          2. Severe (factor IX activity ≤2 IU/dL) hemophilia B subjects currently on-demand therapy
             with a minimum of 2 bleeding episodes requiring factor IX therapy over the preceding 6
             months or 4 bleeding episodes over the preceding 12 months; subjects on prophylaxis
             with a bleeding pattern similar to that above demonstrated prior to starting
             prophylaxis

          3. Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or
             chemotherapeutic agents

          4. Previously treated patients with a minimum of 50 exposure days to a factor IX
             preparation

          5. Platelet count at least 150,000/mm3

          6. Liver function: alanine transaminase [ALT] and aspartate transaminase [AST] ≤2 times
             the upper limit of the normal range

          7. Total bilirubin ≤1.5 times the upper limit of the normal range

          8. Renal function: serum creatinine ≤1.25 times the upper limit of the normal range

          9. Willingness to participate in the trial for approximately 6 months (50 exposures)

         10. Age ≤12 years

         11. Hemoglobin ≥7 g/dL at the time of the blood draw

        Exclusion Criteria:

          1. History of factor IX inhibitor ≥0.6 Bethesda units (BU)

          2. Existence of another coagulation disorder

          3. Evidence of thrombotic disease, fibrinolysis, or disseminated intravascular
             coagulation (DIC)

          4. Use of an investigational drug within 30 days prior to study entry

          5. On medications that could impact hemostasis, such as aspirin

          6. History of poor compliance, a serious medical or social condition, or any other
             circumstance that, in the opinion of the investigator, would interfere with
             participation or compliance with the study protocol

          7. History of adverse reaction to either plasma-derived factor IX or recombinant factor
             IX that interfered with the subject's ability to treat bleeding episodes with a factor
             IX product