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