Clinical Trial: A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

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

Official Title: A Prospective, Multicenter, Open-label, Phase 3b Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Brief Summary:

Congenital deficiency of factor XIII (FXIII) is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII. Factor XIII Concentrate works by assisting blood in the usual clotting process, thereby preventing bleeding.

In this study, patients will be treated with FXIII Concentrate (Human) and followed closely to determine that they receive the dose that will best minimize the chance of bruising and bleeding.


Detailed Summary:
Sponsor: CSL Behring

Current Primary Outcome: The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII‑Containing Product to Treat the Bleeding Event) [ Time Frame: Up to week 52 ]

The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.


Original Primary Outcome: The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII‑Containing Product to Treat the Bleeding Event) [ Time Frame: Up to week 52 ]

Current Secondary Outcome:

  • Association of the Incidence of Spontaneous Bleeding Events Requiring Treatment and FXIII Activity Trough Levels [ Time Frame: 12 months ]
    P-value determined from Generalized Estimating Equation (GEE) model parameter estimates with bleeding as the response variable and FXIII activity trough level as the explanatory variable.
  • Adverse Events [ Time Frame: 12 months ]
    Number of subjects with any treatment-emergent adverse event (AE), treatment-related AE or serious AE (SAE). Treatment related AEs are defined as AEs whose relationship to study treatment is related, or possibly related, and AEs with missing relationship.
  • Peak FXIII Concentration at Steady State [ Time Frame: At 12, 24, 36 and 48 weeks: at 30 and 60 minutes after the end of the infusion. ]
  • Trough FXIII Concentration at Steady State [ Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion. ]
  • Time to Peak Concentration [ Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion. ]
  • Incremental Recovery [ Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion. ]
    Incremental recovery (U/mL/U/kg) is defined as maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of (U/kg) infusion.
  • Achievement of Trough Factor XIII Levels of 5% or Higher. [ Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion. ]
    Number of subjects with Factor XIII level ≥ 5% before infusion at Week 12, Week 24, Week 36 and Week 48.


Original Secondary Outcome:

  • Association of the Incidence of Spontaneous Bleeding Events Requiring Treatment and FXIII Activity Trough Levels [ Time Frame: 12 months ]
  • Adverse events [ Time Frame: 12 months ]
  • Long-term pharmacokinetic data [ Time Frame: 12 months ]


Information By: CSL Behring

Dates:
Date Received: April 21, 2009
Date Started: August 2009
Date Completion:
Last Updated: July 10, 2012
Last Verified: June 2012