Clinical Trial: A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency

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

Official Title: A 12 Week, Multicenter, Pharmacokinetic and Safety Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Brief Summary: Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding. This study will evaluate the safety and recommended (best) amount or level of Factor XIII in a patient's blood. Factor XIII Concentrate (Human) is given to people whose blood is lacking Factor XIII. Factor XIII Concentrate (Human) works by assisting your blood in the usual clotting process, thereby preventing bleeding.

Detailed Summary:
Sponsor: CSL Behring

Current Primary Outcome:

  • Peak FXIII Concentration at Steady State [ Time Frame: 12 weeks ]
  • Trough FXIII Concentration at Steady State [ Time Frame: 12 weeks ]
  • Time to Peak Concentration [ Time Frame: 12 weeks ]
  • Incremental Recovery [ Time Frame: 12 weeks ]
    Incremental recovery (U/mL/U/kg) is defined as the maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of FXIII (U/kg) administered.
  • Terminal Half-life [ Time Frame: 12 weeks ]
  • Area Under the Curve at Steady State [ Time Frame: 12 weeks ]
  • Clearance [ Time Frame: 12 weeks ]
  • Volume of Distribution at Steady State [ Time Frame: 12 weeks ]
  • Mean Residence Time [ Time Frame: 12 weeks ]


Original Primary Outcome:

  • Peak FXIII Concentration at Steady State [ Time Frame: 12 weeks ]
  • Trough FXIII Concentration at Steady State [ Time Frame: 12 weeks ]
  • Time to Peak Concentration [ Time Frame: 12 weeks ]
  • Incremental Recovery [ Time Frame: 12 weeks ]
  • Terminal Half-life [ Time Frame: 12 weeks ]
  • Area Under the Curve at Steady State [ Time Frame: 12 weeks ]
  • Clearance [ Time Frame: 12 weeks ]
  • Volume of Distribution at Steady State [ Time Frame: 12 weeks ]
  • Mean Residence Time [ Time Frame: 12 weeks ]


Current Secondary Outcome:

  • Adverse Events [ Time Frame: 16 weeks ]
    Number of participants with an adverse event
  • Laboratory Safety Parameters [ Time Frame: 16 weeks ]
    Number of participants with clinically significant laboratory safety parameter values. The laboratory safety parameters measured included serum chemistries, hematology and urinalysis.
  • Vital Signs [ Time Frame: 16 weeks ]
    Number of participants with clinically significant vital signs. The vital signs measured included blood pressure, pulse rate and temperature. Clinically significant changes in vital signs were to be reported as adverse events.


Original Secondary Outcome:

  • Adverse Events [ Time Frame: 16 weeks ]
  • Laboratory Safety Parameters [ Time Frame: 16 weeks ]
  • Vital Signs [ Time Frame: 16 weeks ]


Information By: CSL Behring

Dates:
Date Received: April 16, 2009
Date Started: May 2009
Date Completion:
Last Updated: December 7, 2011
Last Verified: December 2011