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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01233440
• Date of registration: 02/11/2010
• Prospective Registration: No
• Primary sponsor: CSL Behring
• Public title: Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B
• Scientific 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
• Date of first enrolment: October 2010
• Target sample size: 25
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01233440
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

Austria, France, Germany, Israel, Italy, Spain

Contacts

• Name: Iris Jacobs, MD
• Affiliation: CSL Behring

Key inclusion & exclusion 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



Age minimum: 12 Years
Age maximum: 65 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Hemophilia B

Intervention(s)

Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein

Biological: Plasma derived FIX [pdFIX]

Primary Outcome(s)

Frequency of serious adverse events (SAEs) [Time Frame: up to 28 days after drug administration]

Frequency of adverse events (AEs) [Time Frame: up to 14 days after drug administration]

Occurrence of antibodies against rIX-FP [Time Frame: up to 28 days after drug administration]

Occurrence of inhibitor against FIX [Time Frame: up to 28 days after drug administration]

Secondary Outcome(s)

Half-life (t1/2) [Time Frame: From time of dosing up to 7 days after the dose]

AUC extrapolated to infinity (AUCt-8) [Time Frame: From time of dosing up to 7 days after the dose]

AUC to the last sample with quantifiable drug concentration (AUC0-t) [Time Frame: From time of dosing up to 7 days after the dose]

Incremental recovery (IU/mL/IU/kg) [Time Frame: From time of dosing up to 7 days after the dose]

Clearance [Time Frame: From time of dosing up to 7 days after the dose]

Secondary ID(s)

CSL654_2001

1508

2010-018477-38

Source(s) of Monetary Support

Please refer to primary and secondary sponsors