A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B

A Phase III Open-label, Multicenter, Pharmacokinetic, Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Previously Treated Children With Hemophilia B

This study will examine the pharmacokinetics, safety and efficacy of rIX-FP for the control and prevention of bleeding episodes in children who have previously received factor replacement therapy for hemophilia B.

Inclusion Criteria:

• Male subjects, younger than 12 years old.
• Severe hemophilia B (Factor IX [FIX] activity of ≤ 2%).
• Body weight ≥ 10 kg.
• Subjects who have received FIX products (plasma-derived and/or recombinant FIX) for > 150 exposure days (EDs) (6 to < 12 years), and > 50 EDs (< 6 years).
• No history of FIX inhibitor formation, no detectable inhibitors at Screening and no family history of inhibitors against FIX.
• Written informed consent for study participation.

Exclusion Criteria:

• Known hypersensitivity to any FIX product or hamster protein.
• Known congenital or acquired coagulation disorder other than congenital FIX deficiency.
• Kidney or liver disease.
• Recent life-threatening bleeding episode.