BAX 326 Pediatric Study

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Baxter Innovations GmbH
Information provided by (Responsible Party):
Baxter Healthcare Corporation
ClinicalTrials.gov Identifier:
NCT01488994
First received: December 6, 2011
Last updated: December 27, 2012
Last verified: December 2012
  Purpose

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.


Condition Intervention Phase
Hemophilia B
Biological: Recombinant Factor IX
Phase 2
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Baxter Healthcare Corporation:

Primary Outcome Measures:
  • Adverse events possibly or probably related to BAX 326 [ Time Frame: 7 months (per subject) ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Area under the plasma concentration versus time curve from 0 to 72 hours post-infusion [ Time Frame: 7 post-infusion time points over a period up to 72 hours ] [ Designated as safety issue: No ]
  • Annualized bleeding rate [ Time Frame: 26 (+/- 1) weeks ] [ Designated as safety issue: No ]
  • Hemostatic efficacy of the acute management of bleeding episodes [ Time Frame: 26 (+/-1) weeks ] [ Designated as safety issue: No ]
    Efficacy assessments: Determination of factor IX level and pharmacokinetic parameters at certain time points, including incremental recovery; rating scale for treatment of bleeding episodes (excellent / good / fair / none)


Estimated Enrollment: 24
Study Start Date: December 2011
Estimated Study Completion Date: September 2013
Estimated Primary Completion Date: May 2013 (Final data collection date for primary outcome measure)
Intervention Details:
    Biological: Recombinant Factor IX
    Prophylactic treatment: twice weekly. Subjects will will undergo a pharmacokinetic evaluation with recombinant Factor IX before starting the prophylactic treatment.
    Other Name: BAX 326
  Eligibility

Ages Eligible for Study:   up to 12 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Main Inclusion Criteria:

  • Subject and/or legal representative has/have voluntarily provided signed informed consent
  • Subject has severe (FIX level < 1%) or moderately severe (FIX level <= 2%) hemophilia B
  • Subject is < 12 years old at the time of screening
  • Subject has no evidence of a history of FIX inhibitors (based on the subject's medical records)
  • Subject is immunocompetent as evidenced by a CD4 count >= 200 cells/mm3

Main Exclusion Criteria:

  • Subject has a detectable FIX inhibitor at screening, with a titer >= 0.6 BU
  • Subject has a history of allergic reaction, e.g. anaphylaxis, following exposure to FIX concentrate(s)
  • Subject has evidence of an ongoing or recent thrombotic disease
  • Subject has an inherited or acquired hemostatic defect other than hemophilia B
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01488994

Locations
Bulgaria
Specialized Hospital For Active Treatment of Oncohematological Diseases in Children
Sofia, Bulgaria, 1527
India
LNJP Maulana Azad Medical College & Associated Hospitals
New Delhi, India, 110002
Sahayadri Speciality Hospital
Pune, India, 411004
Jehangir Hospital and Research Center
Pune, India, 411001
Poland
Antoni Jurasz University Hospital
Bydgoszcz, Poland, 85-094
University Pediatric Hospital
Cracow, Poland, 30-663
Maria Konopnicka Teaching Hospital, Medical University of Lodz
Lodz, Poland, 91-738
Stanislaw Popowski Provincial Specialist Pediatric Hospital
Olsztyn, Poland, 10-561
Professor Tadeusz Sokolowski Independent Public Teaching Hospital of the Pomeranian Medical University in Szczecin
Szczecin, Poland, 71-252
Romania
Prof. Dr. C.T. Nicolau National Institute for Transfusional Hematology
Bucharest, Romania, 11156
Louis Turcanu Emergency Children's Hospital
Timisoara, Romania, 300011
Russian Federation
Regional Clinical Hospital
Ekaterinburg, Russian Federation, 620149
Federal State Institution Kirov, Hematology and Blood Transfusion Research Institute under the Federal Medical and Biological Agency
Kirov, Russian Federation, 610027
Pediatric Regional Clinical Hospital, Hematology Department
Krasnodar, Russian Federation, 350007
Republican Center for Hemophilia Treatment
St. Petersburg, Russian Federation, 195213
Ukraine
State Institution "Institute of Blood Pathology and Transfusion Medicine of the Academy of Medical Sciences of Ukraine"
Lviv, Ukraine, 79044
United Kingdom
Manchester Children´s Hospital
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Baxter Healthcare Corporation
Baxter Innovations GmbH
Investigators
Study Director: Brigitt Abbuehl, MD Baxter Innovations GmbH
  More Information

No publications provided

Responsible Party: Baxter Healthcare Corporation
ClinicalTrials.gov Identifier: NCT01488994     History of Changes
Other Study ID Numbers: 251101, 2011-002437-19
Study First Received: December 6, 2011
Last Updated: December 27, 2012
Health Authority: Bulgaria: Bulgarian Drug Agency
India: Drugs Controller General of India
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Romania: National Medicines Agency
Russia: FSI Scientific Center of Expertise of Medical Application
Ukraine: State Pharmacological Center - Ministry of Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Additional relevant MeSH terms:
Hemophilia B
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on April 15, 2014