The purpose of this study is to evaluate whether rAHF-PFM is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII before.

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

Further Study Details: 

Primary Outcomes: - To assess the immunogenicity of rAHF PFM by determining the risk of factor VIII inhibitor development; - To assess in vivo incremental recovery of rAHF PFM at the first study visit, at every other interval study visit, and at the study termination visit; - To evaluate the hemostatic efficacy of rAHF PFM in the management of acute bleeding events; - To evaluate the hemostatic efficacy of rAHF PFM during surgical prophylaxis, if surgery is required; - To assess the safety of rAHF PFM; - To assess the incidence of the development of antibodies to CHO protein, murine IgG, and human VWF
Expected Total Enrollment:  50

Ages Eligible for Study:  up to  6 Years,  Genders Eligible for Study:  Male

Criteria

Inclusion Criteria:

• The subject has severe or moderately severe hemophilia A as defined by a baseline factor VIII level <= 2% of normal, as documented at screening
• The subject is < 6 years of age
• The subject’s legally authorized representative has provided written informed consent

Exclusion Criteria:

• The subject has a history of exposure to factor VIII other than rAHF PFM or more than 3 infusions of commercially available rAHF PFM (i.e., ADVATE) within 28 days prior to screening, as determined by the subject’s medical history. Any infusion of factor VIII replacement products prior to the 28-day period excludes the subject from participation
• The subject has received more than 3 infusions of rAHF PFM (commercially available and/or study product) between screening and prior to the initial recovery infusion
• The subject has a detectable inhibitor to factor VIII, as measured in the screening sample by the Nijmegen assay in the central laboratory
• The subject has a history of inhibitor to factor VIII at any time prior to screening
• The subject has a known hypersensitivity to rAHF PFM

• The subject has any 1 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 upper limit of normal (ULN) for age
  4. Total bilirubin > 2 times the ULN for age
• The subject has an inherited or acquired hemostatic defect other than hemophilia A (e.g., qualitative platelet defect or von Willebrand’s disease)
• The subject is known to be seropositive for human immunodeficiency virus (HIV), hepatitis C virus (HCV), or hepatitis B virus (HBV), as determined by the subject’s medical history
• At the time of enrollment, the subject has a clinically significant chronic disease other than hemophilia A
• The subject is currently participating in another investigational drug study, or has participated in any clinical study involving an investigational drug within 120 days of the screening visit
• The subject (or the subject’s legally authorized representative) is identified by the investigator as being unable or unwilling to cooperate with study procedures
• The subject has received any blood product, including packed red blood cells (RBC), platelets, plasma, or cryoprecipitate

Please refer to this study by ClinicalTrials.gov identifier  NCT00157157

Illinois
      Children´s Memorial Hospital, Chicago,  Illinois,  60614,  United States; Recruiting
Pennsylvania
      St. Christopher´s Hospital for Children, Section of Hematology/Oncology, Philadelphia,  Pennsylvania,  19134-1095,  United States; Recruiting
Texas
      University of Texas Health Science Center, Gulf States Hemophilia & Thrombophilia Center, Houston,  Texas,  77030,  United States; Recruiting

Study chairs or principal investigators

Michael Recht, MD,  Principal Investigator,  Phoenix Children´s Hospital, The Hemophilia Center, Phoenix, AZ, USA   

Study ID Numbers:  060103
Last Updated:  September 10, 2005
Record first received:  September 9, 2005
ClinicalTrials.gov Identifier:  NCT00157157
Health Authority: United States: Food and Drug Administration; Germany: Paul-Ehrlich-Institut; Austria: Federal Ministry for Health and Women; France: Afssaps - French Health Products Safety Agency; Italy: Ministry of Health; Spain: Spanish Agency of Medicines; Sweden: Medical Products Agency; United Kingdom: Medicines and Healthcare Products Regulatory Agency
ClinicalTrials.gov processed this record on 2005-09-13