Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
Condition: Hemophilia A
MeSH terms: Hemophilia A
Intervention: Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
Intervention description: Treatment regimens were determined by the investigator, and may have been any combination of standard prophylaxis (25 to 50 IU/kg body weight, 3 to 4 times per week), investigator-determined prophylaxis, and/or on-demand treatment (dose selected by investigator).
The treatment of bleeding episodes and perioperative management was at the discretion of the investigator and consistent with the institution's standard of care.
For incremental recovery assessments, a single infusion at 50 +/- 5 IU/kg was to be given.
Immune tolerance induction (ITI) therapy for subjects who developed factor VIII inhibitors was at the discretion of the investigator, based on the institution's guidelines or described in peer-reviewed literature, and was to be approved by the sponsor's medical director.
rAHF-PFM was to be administered intravenously via bolus infusion, except for perioperative management when it may have been given either by continuous or bolus infusion.
Intervention MeSH terms: Factor VIII
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 66.0
Sex: All
Maximum age: 6 Years
Healthy volunteers: No
Keywords: Factor VIII Deficiency

Summary:

      The purpose of this study is to evaluate whether Antihemophilic factor, recombinant,
      manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A
      patients who have not been treated with factor VIII (FVIII) before.
    

Eligibility 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/mm^3

               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