BAX 326 Pediatric Study

Baxalta

Status and phase

Completed

Phase 3

Phase 2

Conditions

Hemophilia B

Treatments

Biological: BAX326

Study type

Interventional

Funder types

Industry

Identifiers

NCT01488994

2011-002437-19 (EudraCT Number)

251101

Details and patient eligibility

About

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.

Full 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.

Enrollment

23 patients

Sex

All

Ages

Under 12 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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