Prophylaxis and treatment of bleeding episodes in subjects with congenital Factor IX (FIX) deficiency (Hemophilia B) MedDRA version: 12.1 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX)
Conditions
Interventions
Product Name: Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP)
Product Code: CSL654 or rIX-FP
Pharmaceutical Form: Powder for concentrate for solution for infusion
Current Sponsor cod
Sponsors
CSL Behring GmbH
Eligibility
Sex/Gender
Male
Inclusion criteria
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 =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Primary Outcome Measures: 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] ;Secondary Objective: Secondary Objective: 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. ;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]. | — |
Countries
Austria, Germany, Italy, Spain, United Kingdom
Outcome results
None listed