BAY81-8973 Pediatric Safety and Efficacy Trial
Condition: Haemophilia A
MeSH terms: Hemophilia A
Intervention: Recombinant Factor VIII (Kovaltry, BAY81-8973); Recombinant Factor VIII (Kovaltry, BAY81-8973)
Intervention description: Main study: 25-50 IU/kg at least 2x/week for 6 months and at least 50 EDs, IV infusion; Extension study: 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part A and extension study), IV infusion. Exposure day (ED): An ED is a unit of time (1 day) in which replacement treatment of Hemophilia is given to a patient.; Main study: 15-50 IU/kg at least 1x/week for at least 50 EDs or until inhibitor development, IV infusion; Extension study: For participants having reached at least 50 EDs in main study - Part B: 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part B and extension study), IV infusion. For participants who developed an inhibitor in main study - Part B: up to 200 IU/kg per day or 100 IU/kg twice a day at the discretion of the investigator and coordinating investigator until successful eradication of the inhibitor, or until failure, for up to18 months (treatment beyond 18 months required an agreement with the sponsor and coordinating investigator), IV 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: 94.0
Sex: Male
Minimum age: 0 Years
Maximum age: 12 Years
Healthy volunteers: No
Keywords: Recombinant factor VIII; Pediatric use

Summary:

      The primary objective was to evaluate the safety and efficacy of the treatment with
      BAY81-8973 for prophylaxis and treatment of breakthrough bleeds in children with severe
      hemophilia A.

      The secondary objectives were

        -  To assess the safety and efficacy of BAY81-8973 during surgeries.

        -  To assess incremental recovery of BAY81-8973.

        -  To assess pharmacokinetic (PK) parameters in a subset of children (Previously treated
           patients [PTPs] and previously untreated patients [PUPs] / minimally treated patients
           [MTPs] - participation in PK sampling was voluntary and required consent).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male

          -  PTPs (previously treated patients): aged <= 12 years

          -  PUPs (previously untreated patients) / MTPs (minimally treated patients): aged < 6
             years

          -  Severe hemophilia A defined as < 1% FVIII concentration (FVIII:C)

          -  PTPs: >= 50 exposure days (EDs) with any FVIII concentrate, no current evidence of
             inhibitory antibody, and no history of FVIII inhibitor formation

          -  PUPs: no prior exposure to any FVIII product

          -  MTPs: having no more than 3 EDs with any FVIII product, no current evidence of
             inhibitory antibody and no history of FVIII inhibitor formation

        Exclusion Criteria:

          -  With another bleeding disorder that is different from Hemophilia A

          -  With thrombocytopenia (platelet count < 100 000/mm^3)

          -  Creatinine > 2x upper limit of normal or Aspartate aminotransferase (AST)/Alanine
             aminotransferase (ALT) > 5x upper limit of normal

          -  Without a negative inhibitor testing at screening (except for PUPs)

          -  Receiving chemotherapy, immune modulatory drugs, has received another investigational
             FVIII product within the last month, or received another experimental drug within the
             last 3 months

          -  Requires any pre-medication to tolerate FVIII treatment

          -  Known hypersensitivity to active substance, mouse, or hamster protein