Clinical Trial: An Open Enrollment 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 Enrollment 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 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 of FXIII Concentrate (Human) that will best minimize the chance of bruising and bleeding. The purpose of the study is to provide FXIII Concentrate (Human) to patients until the product becomes commercially available in the United States.


Detailed Summary:
Sponsor: CSL Behring

Current Primary Outcome: Adverse Events [ Time Frame: After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA. ]

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 treatment is related, or possibly related and AEs with missing relationship.


Original Primary Outcome: Adverse Events [ Time Frame: For the duration of the study ]

Current Secondary Outcome:

  • Hematology and Chemistry Testing [ Time Frame: After the first infusion and at the end-of-study (or withdrawal) visit. ]
    Number of participants with treatment-emergent clinically significant hematology and/or chemistry laboratory parameter values.
  • FXIII Antibody Testing [ Time Frame: Before the first infusion, then every 48 weeks, at the end-of-study (or withdrawal) visit and after a bleeding episode requiring treatment with a Factor XIII -containing product. ]
    Number of participants with serum Factor XIII antibodies.
  • FXIII Concentration [ Time Frame: Before the first infusion, at 24 and 48 weeks after the first infusion, and at the end-of-study (or withdrawal) visit. ]
    Trough Factor XIII concentration.
  • Number of Subjects With at Least One Bleeding Episode [ Time Frame: After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA. ]
    Number of subjects with at least one bleeding episode at any time after the first infusion in the study, and the number of subjects with at least one bleeding episode requiring Factor XIII treatment.
  • Number of Bleeding Episodes [ Time Frame: After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA. ]
    Number of bleeding episodes at any time after the first infusion in the study.


Original Secondary Outcome:

  • Hematology and Chemistry Testing [ Time Frame: For the duration of the study ]
  • FXIII Antibody Testing [ Time Frame: For the duration of the study ]
  • FXIII activity level [ Time Frame: Every 24 weeks for the duration of the study ]


Information By: CSL Behring

Dates:
Date Received: July 23, 2009
Date Started: September 2009
Date Completion:
Last Updated: September 12, 2012
Last Verified: September 2012