BAX 326 Pediatric Study
Information source: Baxter Healthcare Corporation
ClinicalTrials.gov processed this data on August 20, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Hemophilia B
Intervention: Recombinant Factor IX (Biological)
Phase: Phase 2/Phase 3
Status: Completed
Sponsored by: Baxter Healthcare Corporation Official(s) and/or principal investigator(s): Srilatha Tangada, PhD, Study Director, Affiliation: Baxter Healthcare Corporation
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.
Clinical Details
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 <= 2%) Hemophilia B
Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Adverse events possibly or probably related to BAX 326
Secondary outcome: Area under the plasma concentration versus time curve from 0 to 72 hours post-infusionTotal Area under the plasma concentration versus time curve per dose (Total AUC/dose) Mean residence time (MRT) Factor IX (FIX) Clearance(CL) Incremental recovery (IR) Elimination phase half-life (T 1/2) Volume of distribution at steady state (Vss) Incremental recovery (IR) over time Hemostatic efficacy of the acute management of bleeding episodes Treatment of bleeding episodes: number of infusions per bleeding episode Treatment of bleeding episodes (BEs): overall hemostatic efficacy rating at resolution of bleed Prophylaxis: Annualized bleeding rate Consumption of BAX326: number of infusions Consumption of BAX326: weight-adjusted consumption Consumption of BAX326: weight-adjusted consumption per event Development of inhibitory antibodies to FIX Development of total binding antibodies to FIX Occurrence of severe allergic reactions, e.g. anaphylaxis Occurrence of thrombotic events Clinically significant changes in routine laboratory parameters (hematology and clinical chemistry), and vital signs Development of antibodies to Chinese hamster ovary (CHO) proteins and recombinant furin (rFurin) Health-related Quality of Life Outcome Measure: Generic: PedsQL™ (Parent-proxy versions: age group 2-4 years and age group 5-7 years) Health-related Quality of Life Outcome Measure: Hospitalizations Health-related Quality of Life Outcome Measure: Length of Hospitalizations Health-related Quality of Life Outcome Measure: Emergency Room Visits Health-related Quality of Life Outcome Measure: Unscheduled Office Visits Health-related Quality of Life Outcome Measure: Days Lost from School Health-related Quality of Life Outcome Measure: Disease-specific: Haemo-QoL, short version: age group 8 to 11 years of age Health-related Quality of Life Outcome Measure: Generic- PedsQL™ Child version: age group 8 to 11 years of age Health-related Quality of Life Outcome Measure: Hospitalizations Health-related Quality of Life Outcome Measure: Length of Hospitalizations Health-related Quality of Life Outcome Measure: Emergency Room Visits Health-related Quality of Life Outcome Measure: Unscheduled Office Visits Health-related Quality of Life Outcome Measure: Days Lost from School
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.
Eligibility
Minimum age: N/A.
Maximum age: 12 Years.
Gender(s): Both.
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
Locations and Contacts
LNJP Maulana Azad Medical College & Associated Hospitals, New Delhi 110002, India
University Pediatric Hospital, Cracow 30-663, Poland
Stanislaw Popowski Provincial Specialist Pediatric Hospital, Olsztyn 10-561, Poland
Professor Tadeusz Sokolowski Independent Public Teaching Hospital of the Pomeranian Medical University in Szczecin, Szczecin 71-252, Poland
S.C. Sanador SRL, Bucharest 11156, Romania
Louis Turcanu Emergency Children's Hospital, Timisoara 300011, Romania
Regional Clinical Hospital, Ekaterinburg 620149, Russian Federation
Pediatric Regional Clinical Hospital, Hematology Department, Krasnodar 350007, Russian Federation
Republican Center for Hemophilia Treatment, St. Petersburg 195213, Russian Federation
State Institution "Institute of Blood Pathology and Transfusion Medicine of the Academy of Medical Sciences of Ukraine", Lviv 79044, Ukraine
Manchester Children´s Hospital, Manchester M13 9WL, United Kingdom
Additional Information
Starting date: December 2011
Last updated: May 29, 2014
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