Brief Title
BAX 326 Pediatric Study
Official Title
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
Brief Summary
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.
Detailed Description
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.
Study Phase
Phase 2/Phase 3
Study Type
Interventional
Primary Outcome
Adverse Events (AEs) Possibly or Probably Related to BAX326
Secondary Outcome
Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Post-infusion Per Dose (AUC 0-72h/Dose)
Condition
Hemophilia B
Intervention
BAX326
Study Arms / Comparison Groups
BAX326 < 6 years of age
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
23
Start Date
December 20, 2011
Completion Date
May 14, 2013
Primary Completion Date
May 14, 2013
Eligibility Criteria
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
Gender
All
Ages
N/A - 12 Years
Accepts Healthy Volunteers
No
Contacts
Study Director, ,
Location Countries
India
Location Countries
India
Administrative Informations
NCT ID
NCT01488994
Organization ID
251101
Secondary IDs
2011-002437-19
Responsible Party
Sponsor
Study Sponsor
Baxalta now part of Shire
Study Sponsor
Study Director, Study Director, Takeda
Verification Date
April 2021