Status:
COMPLETED
Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B
Lead Sponsor:
CSL Behring
Conditions:
Hemophilia B
Eligibility:
MALE
12-65 years
Phase:
PHASE1
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 s...
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 s...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
October 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2011
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT01233440
Start Date
October 1 2010
End Date
July 1 2011
Last Update
January 31 2012
Active Locations (20)
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1
Study site
Vienna, Austria
2
Study site
Le Kremlin-Bicêtre, France
3
Study Site
Lyon, France
4
Study site
Nantes, France