Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B

Launched by CANGENE CORPORATION · Jan 27, 2014

Keywords

ClinConnect Summary

Primary Objectives:

* to evaluate safety of IB1001 within the first 50 exposure days,
* to determine IB1001 pharmacokinetics (PK), and
* to assess efficacy of IB1001 prophylaxis with respect to breakthrough bleeding and with respect to control of hemorrhaging in subjects with severe hemophilia B within the first 50 exposure days

Secondary Objectives:

* to evaluate long-term safety of IB1001; and
* to evaluate long term efficacy of IB1001.

Exploratory Objectives:

* to evaluate markers of thrombogenicity during the first 24 hours post-infusion \[thrombogenicity markers will include at a ...

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Age of at least 12 years
• • 2. Body Mass Index of ≤ 29, with a minimum body weight of 40 kg
• • 3. Written Institutional Review Board (IRB)/ Ethics Committee (EC)-approved informed consent form (ICF)
• • 4. Willingness to make the required study visits, and follow instructions while enrolled in the study (up to 12 months)
• • 5. Severe (factor IX activity ≤2 U/dL) hemophilia B patients with a minimum of 3 bleeding episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12 months or in the event the subject is on prophylaxis, a minimum of 3 bleeding episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12 months prior to being placed on prophylaxis
• • 6. Subjects must be on prophylaxis or switch to a prophylaxis regimen for the duration of the PK and Treatment/Continuation Phase of the study
• • 7. Previously treated patients with a minimum of 150 exposure days to a factor IX preparation
• • 8. Willingness to adhere to the 5-day washout of any factor IX replacement therapy prior to PK evaluations
• • 9. Immunocompetent (CD4 count \>400/mm3) and not receiving immune modulating or chemotherapeutic agents
• • 10. Platelet count at least 150,000/mm3
• • 11. Liver function: alanine transaminase (ALT) and aspartate transaminase (AST) ≤2 times the upper limit of the normal range
• • 12. Total bilirubin ≤1.5 times the upper limit of the normal range
• • 13. Renal function: serum creatinine ≤1.25 times the upper limit of the normal range
• • 14. Hemoglobin ≥7 g/dL at the time of the blood draw
• Exclusion Criteria:
• • 1. History of factor IX inhibitor ≥0.6 BU (Bethesda units)
• • 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. Previous use of IB1001
• • 6. Use of medications that could impact hemostasis, such as aspirin
• • 7. Hypersensitivity to the active substance or to any of the excipients in the investigational products
• • 8. Known allergic reaction to hamster proteins
• • 9. 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
• • 10. 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