Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII
Condition: Hemophilia A
MeSH terms: Hemophilia A
Intervention: Moroctocog alfa (AF-CC); Moroctocog alfa (AF-CC)
Intervention description: On-demand therapy for 6 months, followed by routine prophylaxis 25 IU/kg, administered every other day for 1 year.; Routine prophylaxis crossover:
45 IU/kg, administered 2 times a week for 1 year followed by 25 IU/kg administered every other day for 1 year, or, 25 IU/kg, administered every other day for 1 year, followed by 45 IU/kg, administered 2 times a week for 1 year.
Intervention MeSH terms: Factor VIII
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: None (Open Label)
Enrollment: 66.0
Sex: Male
Minimum age: 6 Months
Maximum age: 15 Years
Healthy volunteers: No

Summary:

      The purpose of this research study is to determine the effectiveness, safety, and
      pharmacokinetics (PK) of moroctocog alfa (AF-CC) in previously treated subjects, who are
      younger than 6 years of age, with severe or moderately severe hemophilia A.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male subjects, aged less than 6 years, with moderately severe to severe hemophilia A.

          -  A negative FVIII inhibitor titer at screening, and a medical history negative for a
             past FVIII inhibitor.

          -  At least 20 exposure days to any FVIII replacement product.

          -  Adequate hepatic and renal function

          -  CD4 count > 400 cells/uL, and if receiving antiviral therapy must be on a stable
             regimen

        Additional criteria for subjects participating in the PK assessment:

          -  Male subjects as described immediately above except they must have a FVIII Activity of
             less than or equal to 1% confirmed by the central laboratory screening test

          -  Age < 6 years at time of PK assessment.

          -  The subject's size is sufficient to permit PK-related phlebotomy.

          -  The subject is able to comply with the procedures conducted during the PK assessment,
             including a mandatory 72-hour washout period preceding the PK assessment.

        Exclusion Criteria:

          -  A history of FVIII inhibitor.

          -  Presence of a bleeding disorder in addition to hemophilia A.

          -  Treatment with any investigational drug or device within 30 days before the time of
             signing the informed consent form.

          -  Major or orthopedic surgery planned to occur during the course of the study.

          -  Regular (e.g., daily, every other day) use of antifibrinolytic agents or medications
             known to influence platelet function such as aspirin or certain nonsteroidal
             anti-inflammatory drugs (NSAIDs), or regular, concomitant therapy with
             immunomodulating drugs (e.g., intravenous immunoglobulin [IVIG], routine systemic
             corticosteroids).

          -  Known hypersensitivity to hamster protein.