A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

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

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.

Inclusion Criteria:

• Written informed consent/assent for study participation obtained before undergoing any study-specific procedures
• Documented congenital FXIII deficiency which requires prophylactic treatment with a FXIII containing product.
• Males and females of any age with congenital FXIII deficiency
• Received full hepatitis B vaccination and/or is hepatitis B surface antibody positive

Exclusion Criteria:

• Diagnosis of acquired FXIII deficiency
• Administration of a FXIII-containing product, including blood transfusions or other blood products within 4 weeks prior to the planned Day 0
• Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
• Known or suspected to have antibodies towards FXIII
• Use of any other investigational medicinal product within 4 weeks prior to the Baseline Visit (Day 0)
• Known Positivity for human immunodeficiency virus (HIV) or a positive result for HIV at the Screening Visit of this study or the FXIII study 2002 (NCT00883090).
• Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) concentration >2.5 times the upper limit of normal at the Screening Visit of this study or at the Day 56 Visit of Factor XIII Study BI71023_2002 (NCT00883090)
• Fibrinogen level less than 85% of the lower limit of normal at the Screening Visit of this study or the Factor XIII Study BI71023_2002 (NCT00883090)
• Active bleeding ≥ Common Terminology Criteria for Adverse Events (CTCAE) Grade 2 and/or ≥ moderate between the Screening and Baseline Visits
• Pregnant or breast-feeding
• Intention to become pregnant during the course of the study
• Female subjects of childbearing potential not using, or not willing to use, a medically reliable method of contraception for the entire duration of the study
• Suspected inability (e.g., language problems) or unwillingness to comply with study procedures or history of noncompliance