Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B

Brief Title

Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B

Official Title

An Open-label, Multicenter, Dose-Escalation Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects With Hemophilia B

Brief Summary

      The primary objective of the study is to assess the safety of IV administration of rIX-FP.
      Safety will be evaluated by adverse events and laboratory changes over time. The secondary
      objective of the study is to evaluate the pharmacokinetics parameters, following a single
      intravenous dose of rIX-FP.

Detailed Description

      This study is comprised of both a rIX-FP dose-escalation safety segment (25, 50 and 75 IU/kg
      of rIX-FP), and PK evaluation of rIX-FP after a single dose of 50 IU/kg, as well as PK
      evaluation after a single dose of 50 IU/kg of the previously given Factor IX (FIX) product
      (recombinant FIX [rFIX] or plasma derived FIX [pdFIX]) which is used as the reference
      product.

Study Phase

Phase 1

Study Type

Interventional

Primary Outcome

Frequency of adverse events (AEs)

Secondary Outcome

 AUC to the last sample with quantifiable drug concentration (AUC0-t)

Condition

Hemophilia B

Intervention

Recombinant Coagulation Factor IX Albumin Fusion Protein

Study Arms / Comparison Groups

 Cohort 1

Description:  25 IU/kg dose

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Biological

Estimated Enrollment

Start Date

October 2010

Completion Date

July 2011

Primary Completion Date

July 2011

Eligibility Criteria

        Inclusion Criteria:

          -  Male, 12 - 65 years, with body weight ≥ 30 kg and ≤ 120 kg

          -  Documented severe Hemophilia B (FIX activity of ≤ 2%) or tested by the central
             laboratory at screening

          -  Subjects who have received FIX products for > 150 exposure days (EDs) (estimated)

          -  No confirmed prior history of FIX inhibitor (history of positive FIX inhibitor defined
             as two consecutive positive tests - a confirmatory test on a second, separately drawn
             sample shortly after the previous positive test) and confirmed no detectable FIX
             inhibitors (negative FIX inhibitor defined as < 0.6 Bethesda Units [BU] by the central
             laboratory at screening

          -  Subjects can be treated on-demand or under prophylactic therapy

          -  Signed Informed Consent/Assent

        Exclusion Criteria:

          -  Known hypersensitivity (allergic reaction or anaphylaxis) to any FIX product or
             hamster protein

          -  Any known congenital or acquired coagulation disorder other than congenital FIX
             deficiency

          -  Platelet count < 100,000/µL

          -  Immunocompromised (CD4 count < 200/mm3), (HIV positive subjects may participate in the
             study and protease inhibitors and antiviral therapy are permitted, at the discretion
             of the Investigator)

          -  Currently receiving IV immunomodulating agents such as immunoglobulin or chronic
             systemic corticosteroid treatment

          -  Serum aspartate aminotransferase (AST) or serum alanine aminotransferase (ALT)
             concentration > 5 times (x) the upper limit of normal (ULN)

          -  Serum creatinine > 2 x ULN

          -  Evidence of thrombosis, including deep vein thrombosis, stroke, pulmonary embolism,
             myocardial infarction and arterial embolus within 3 months prior to enrollment

          -  Use of an Investigational Medicinal Product (IMP) within 30 days prior to the first
             rIX-FP administration

          -  Experienced life-threatening bleeding episode or had major surgery or an orthopedic
             surgical procedure during the 3 months prior to study entry

          -  Subject currently on a dose and/or regimen of FIX that would preclude participation in
             the study due to possible increased risk of bleeding because of the requirement to
             withhold treatment during the PK sampling period

          -  Suspected inability (e.g., language problem or mental condition) or unwillingness to
             comply with study procedures or history of noncompliance

Gender

Male

Ages

12 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

Iris Jacobs, MD, ,

Location Countries

Austria

Location Countries

Austria

Administrative Informations

NCT ID

NCT01233440

Organization ID

CSL654_2001

Secondary IDs

Responsible Party

Sponsor

Study Sponsor

CSL Behring

Study Sponsor

Iris Jacobs, MD, Study Director, CSL Behring

Verification Date

January 2012