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.
Phase 2/Phase 3
Adverse Events (AEs) Possibly or Probably Related to BAX326
Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Post-infusion Per Dose (AUC 0-72h/Dose)
Study Arms / Comparison Groups
BAX326 < 6 years of age
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
December 20, 2011
May 14, 2013
Primary Completion Date
May 14, 2013
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
N/A - 12 Years
Accepts Healthy Volunteers
Study Director, ,
Baxalta now part of Shire
Study Director, Study Director, Takeda