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.

• Conditions: Prophylaxis and treatment of bleeding episodes in subjects with congenital Factor IX (FIX) deficiency (Hemophilia B); MedDRA version: 12.1Level: LLTClassification code 10060614Term: Hemophilia B (Factor IX)

• Registration Number: EUCTR2010-018477-38-DE
• Lead Sponsor: CSL Behring GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-04-01
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 24

Inclusion Criteria

•Male, with body weight = 30 kg and = 120 kg
For sites in France, Germany, and UK: aged 18-65 years
For sites in Germany and UK: aged 12-17 years can be enrolled after the safety
data from the first 2 cohorts have been reviewed with no safety concerns
identified by Data Review Committee
•Documented history of severe Hemophilia B (FIX activity of = 2%)
•Subjects who have received FIX products for > 150 exposure days (EDs)
•No confirmed prior history of FIX inhibitor (history of positive FIX inhibitor defined as two consecutive positive tests) and no detectable FIX inhibitors (negative FIX inhibitor defined as < 0.6 Bethesda Units [BU] by the local lab) at screening
•Subjects can be treated on-demand or under prophylactic therapy
•Signed Informed Consent/Assent

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Primary Outcome Measures:<br>Safety of intravenous (IV) infusion of rIX-FP. Safety will be evaluated by adverse events and laboratory changes over time. [Time Frame: assessed up to 28 days after rIX-FP administration]<br>;Secondary Objective: Secondary Objective:<br>To evaluate the pharmacokinetic (PK) parameters following a single IV dose of 50 international units (IU) per kilogram (kg) of body weight of rIX-FP. <br>;Primary end point(s): •Frequency of Adverse Events (AEs), Serious Adverse Events (SAEs) and occurrence of neutralizing antibodies against FIX, and rIX-FP [Time Frame: assessed up to 28 days after rIX-FP administration ] [Designated as safety issue: Yes].