Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Baxter Healthcare Corporation
ClinicalTrials.gov Identifier:
NCT00157040
First received: September 8, 2005
Last updated: September 26, 2011
Last verified: September 2011
  Purpose

The purpose of this study is to evaluate whether rAHF-PFM is effective and safe in the treatment of children with hemophilia A. The study consists of 2 parts. Part 1 of the study is a pharmacokinetic evaluation, and Part 2 is an evaluation of efficacy and safety.


Condition Intervention Phase
Hemophilia A
Drug: Antihemophilic factor, recombinant, manufactured protein-free
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Recombinant Antihemophilic Factor (rAHF-PFM) Manufactured and Formulated Without Added Human or Animal Proteins: Evaluation of Pharmacokinetics, Immunogenicity, Efficacy and Safety in Previously Treated Pediatric Patients With Hemophilia A

Resource links provided by NLM:


Further study details as provided by Baxter Healthcare Corporation:

Estimated Enrollment: 50
Study Start Date: June 2002
Study Completion Date: January 2005
Primary Completion Date: January 2005 (Final data collection date for primary outcome measure)
  Eligibility

Ages Eligible for Study:   up to 6 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subject is less than 6 years of age
  • Severe or moderately-severe hemophilia A as defined by a baseline factor VIII level <= 2%, documented at screening or on the basis of historical data (e.g., at hemophilia diagnosis)
  • Documented medical history of at least 50 exposure days for treatment with all other factor VIII products
  • Subject's parent or legally authorized representative has provided informed consent

Exclusion Criteria:

  • Detectable inhibitor to factor VIII measured in the screening sample by the local or central hemostasis laboratory
  • History of inhibitor to factor VIII at any time prior to screening
  • Subject has any one of the following laboratory abnormalities at the time of screening:

    1. platelet count < 100,000/mm3
    2. hemoglobin concentration < 10 g/dL (100 g/L)
    3. serum creatinine > 1.5 times the ULN for age
    4. total bilirubin > 2 times the ULN for age
  • Subject has an inherited or acquired hemostatic defect other than hemophilia A (e.g., platelet dysfunction secondary to uremia, liver failure, von Willebrand's Disease)
  • Subject has known hypersensitivity to RECOMBINATE rAHF
  • Subject is currently participating in another investigational drug study or has participated in any clinical study involving an investigational drug within 30 days of study entry
  • Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00157040

Locations
United States, California
Children´s Hospital Los Angeles
Los Angeles, California, United States, 90027
United States, Georgia
Emory University, Department of Pediatrics
Atlanta, Georgia, United States, 30322
United States, Illinois
Children´s Memorial Hospital
Chicago, Illinois, United States, 60614
Comprehensive Bleeding Disorders Center
Peoria, Illinois, United States, 61614
United States, Indiana
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, United States, 46260
United States, Iowa
University of Iowa Hospitals and Clinics, Iowa Regional Hemophilia Center, Department of Pediatrics
Iowa City, Iowa, United States, 52242
United States, Michigan
University of Michigan Hemophilia Treatment Center
Ann Arbor, Michigan, United States, 48109
Children´s Hospital of Michigan
Detroit, Michigan, United States, 48201
United States, Pennsylvania
Children´s Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
University of Texas Health Science Center
Houston, Texas, United States, 77030
Canada, Ontario
Hospital for Sick Children, Division of Hematology/Oncology
Toronto, Ontario, Canada, M5G 1X8
Puerto Rico
University Pediatric Hospital
San Juan, Puerto Rico, 00927
Sponsors and Collaborators
Baxter Healthcare Corporation
Investigators
Principal Investigator: Victor Blanchette, MD Hospital for Sick Children, Division of Hematology/Oncology, Toronto, Canada
  More Information

No publications provided by Baxter Healthcare Corporation

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Baxter Healthcare Corporation
ClinicalTrials.gov Identifier: NCT00157040     History of Changes
Other Study ID Numbers: 060101
Study First Received: September 8, 2005
Last Updated: September 26, 2011
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Finland: Finnish Medicines Agency
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Paul-Ehrlich-Institut
Italy: Ministry of Health
Netherlands: Medicines Evaluation Board (MEB)
Spain: Spanish Agency of Medicines
Sweden: Medical Products Agency
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Baxter Healthcare Corporation:
Factor VIII Deficiency

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 18, 2014