Clinical Trial: Study of Biostate® in Children With Hemophilia A

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase III, Open-Label, Multicentre Study to Evaluate Efficacy, Pharmacokinetics, and Safety of Biostate® in Paediatric Subjects With Haemophilia A

Brief Summary: The objective of this study is to assess the efficacy and safety of a Von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, and to investigate the pharmacokinetics of Biostate in children with haemophilia A.

Detailed Summary:
Sponsor: CSL Behring

Current Primary Outcome:

  • Subjective assessment of Haemostatic efficacy [ Time Frame: Over minimum of 50 exposure days ]
  • Incremental recovery of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Half-life of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Area under the concentration curve (AUC) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Mean residence time (MRT) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Volume of distribution at steady state (Vss) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Maximum Plasma Concentration (Cmax) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Minimum Plasma Concentration (Cmin) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Time the maximum concentration occurs (tmax) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Total clearance of the drug from the body (CL=dose/AUC) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Number of infusions p

    Original Primary Outcome: Same as current

    Current Secondary Outcome:

    • Frequency of adverse events (AEs) [ Time Frame: 6 months ]
    • Severity of AEs per subject [ Time Frame: 6 months ]
    • Severity of AEs per infusion [ Time Frame: 6 months ]
    • Relatedness of AEs per subject [ Time Frame: 6 months ]
    • Relatedness of AEs per infusion [ Time Frame: 6 months ]
    • Development of FVIII inhibitors [ Time Frame: Samples taken at screening visit, on day 2, on months 1 and 3, and at final visit ]


    Original Secondary Outcome: Same as current

    Information By: CSL Behring

    Dates:
    Date Received: October 1, 2010
    Date Started: August 2010
    Date Completion:
    Last Updated: July 8, 2014
    Last Verified: July 2014