Phase 3
Completed N=23
BAX 326 Pediatric Study
Source: ClinicalTrials.gov NCT01488994 ↗Enrolled (actual)
23
Serious AEs
13.0%
Results posted
Sep 2016
Primary outcomePrimary: Adverse Events (AEs) Possibly or Probably Related to BAX326 — 0; 0; 0 AEs considered related to BAX326
◆ Published Evidence
Emerging
9citations · ~1 / year
Recombinant factor IX (BAX326) in previously treated paediatric patients with haemophilia B: a prospective clinical trial.
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.
Linked Publications
-
Recombinant factor IX (BAX326) in previously treated paediatric patients with haemophilia B: a prospective clinical trial.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Adverse Events (AEs) Possibly or Probably Related to BAX326 |
0; 0; 0 | — |
| SECONDARY Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Post-infusion Per Dose (AUC 0-72h/Dose) |
— | — |
| SECONDARY Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to Infinity Post-infusion Per Dose (Total AUC/Dose) |
723.7; 886; 808.4 | — |
| SECONDARY Pharmacokinetics (PK): Mean Residence Time (MRT) |
30.62; 25.31; 27.85 | — |
| SECONDARY Pharmacokinetics (PK): Factor IX (FIX) Clearance (CL) |
0.1058; 0.0874; 0.0962 | — |
| SECONDARY Pharmacokinetics (PK): Incremental Recovery (IR) |
0.586; 0.731; 0.665 | — |
| SECONDARY Pharmacokinetics (PK): Elimination Phase Half-life (T 1/2) |
27.67; 23.15; 25.31 | — |
| SECONDARY Pharmacokinetics (PK): Volume of Distribution at Steady State (Vss) |
3.225; 2.209; 2.695 | — |
| SECONDARY Pharmacokinetics (PK): Incremental Recovery (IR) Over Time |
0.586; 0.731; 0.665; 0.630; 0.726; 0.680 | — |
| SECONDARY Hemostatic Efficacy: Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode |
9; 6; 15; 1; 7; 8 | — |
| SECONDARY Hemostatic Efficacy: Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed |
9; 4; 13; 2; 10; 12 | — |
| SECONDARY Hemostatic Efficacy: Prophylaxis: Annualized Bleeding Rate (ABR) |
1.9; 3.4; 2.7 | — |
| SECONDARY Consumption of BAX326: Number of Infusions Per Month |
6.8; 7.2; 7.0 | — |
| SECONDARY Consumption of BAX326: Number of Infusions Per Year |
82.1; 85.9; 84.1 | — |
| SECONDARY Consumption of BAX326: Weight-adjusted Consumption Per Month |
393.4; 414.8; 404.6 | — |
| SECONDARY Consumption of BAX326: Weight-adjusted Consumption Per Year (Annualized) |
4720.9; 4978.2; 4855.1 | — |
| SECONDARY Consumption of BAX326: Weight-adjusted Consumption Per Event |
56.3; 56.2; 56.2; 57.6; 62.1; 59.9 | — |
| SECONDARY Safety and Immunogenicity: Number of Participants Who Developed Inhibitory Antibodies to Factor IX (FIX) |
0; 0; 0 | — |
| SECONDARY Safety and Immunogenicity: Number of Participants Who Developed Total Binding Antibodies to Factor IX (FIX) |
0; 0; 0 | — |
| SECONDARY Safety: Number of Participants With Severe Allergic Reactions, e.g. Anaphylaxis |
0; 0; 0 | — |
| SECONDARY Safety: Number of Participants With Thrombotic Events |
0; 0; 0 | — |
| SECONDARY Safety: Number of Participants With Clinically Significant Changes in Routine Laboratory Parameters (Haematology and Clinical Chemistry), and Vital Signs |
0; 2; 2; 0; 0; 0 | — |
| SECONDARY Safety: Number of Participants Who Developed Antibodies to Chinese Hamster Ovary (CHO) Proteins and Recombinant Furin (rFurin) |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Health-related Quality of Life (HRQoL): PedsQL™ Change From Baseline in Total Score |
3.27; -7.07; 4.02 | — |
| SECONDARY Health-related Quality of Life (HRQoL): Haemo-QoL, Change From Baseline in Total Score |
-0.18 | — |
| SECONDARY Health Resource Use: Number of Hospitalizations |
0.0; 0.0; 0.0; 0.0; 0.0; 0.0 | — |
| SECONDARY Health Resource Use: Length of Hospitalization |
NA; NA; NA; NA; NA; NA | — |
| SECONDARY Health Resource Use: Unscheduled Doctor's Office Visits |
0.0; 0.0; 0.0; 0.0; 0.0; 0.0 | — |
| SECONDARY Health Resource Use: Emergency Room Visits |
0.0; 0.0; 0.0; 0.0; 0.0; 0.0 | — |
| SECONDARY Health Resource Use: Days Lost From School |
0.0; 0.0; 0.0; 0.0; 0.0; 0.0 | — |
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
Data sourced from ClinicalTrials.gov (NCT01488994) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.