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 II Phase III

Further Study Details: 

Expected Total Enrollment:  50

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

Criteria

Inclusion Criteria:

• Subject is < 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

Georgia
      Emory University, Department of Pediatrics, Atlanta,  Georgia,  30322,  United States
New York
      Schneider Children´s Hospital, New York,  New York,  11040,  United States
      The New York Presbyterian Hospital, Division of Pediatric Hematology/Oncology, New York,  New York,  10021,  United States
Texas
      University of Texas Health Science Center, Houston,  Texas,  77030,  United States
Canada, Ontario
      Hospital for Sick Children, Division of Hematology/Oncology, Toronto,  Ontario,  M5G 1X8,  Canada

Study chairs or principal investigators

Victor Blanchette, MD,  Principal Investigator,  Hospital for Sick Children, Division of Hematology/Oncology, Toronto, Canada   

Study ID Numbers:  060101
Last Updated:  September 10, 2005
Record first received:  September 8, 2005
ClinicalTrials.gov Identifier:  NCT00157040
Health Authority: United States: Food and Drug Administration; Canada: Health Canada; Finland: National Agency for Medicines; France: Afssaps - French Health Products Safety Agency; 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
ClinicalTrials.gov processed this record on 2005-09-13