BAX 326 Pediatric Study

BAX 326 (Recombinant Factor IX): A Phase 2/3 Prospective, Uncontrolled, Multicenter Study Evaluating Pharmacokinetics, Efficacy, Safety, and Immunogenicity in Previously Treated Pediatric Patients With Severe (FIX Level < 1%) or Moderately Severe (FIX Level 1-2%) Hemophilia B

The purpose of this study is to assess BAX 326 pharmacokinetic parameters, to evaluate its hemostatic efficacy, safety, immunogenicity, and changes in health-related quality of life in pediatric patients.

The secondary outcome measure: Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours (h) Post-infusion analysis was not done due to the different time-points for the last PK blood sample, AUC0-72 h was redundant and only total AUC was included in the PK analysis.

Main Inclusion Criteria:

• Participant and/or legal representative has/have voluntarily provided signed informed consent
• Participant has severe (FIX level < 1%) or moderately severe (FIX level ≤ 2%) hemophilia B
• Participant is < 12 years old at the time of screening
• Participant has no evidence of a history of FIX inhibitors (based on the participant's medical records)
• Participant is immunocompetent as evidenced by a CD4 count ≥ 200 cells/mm^3

Main Exclusion Criteria:

• Participant has a detectable FIX inhibitor at screening, with a titer ≥ 0.6 Bethesda Unit (BU)
• Participant has a history of allergic reaction, e.g. anaphylaxis, following exposure to FIX concentrate(s)
• Participant has evidence of an ongoing or recent thrombotic disease
• Participant has an inherited or acquired hemostatic defect other than hemophilia B