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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-infusion

Total 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

Page last updated: August 20, 2015

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