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

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

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.

Inclusion Criteria:

• Written informed consent/assent for study participation obtained before undergoing any study-specific procedures
• Documented congenital FXIII deficiency that 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)
• Positive result at screening for human immunodeficiency virus (HIV)
• Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) concentration >2.5 times the upper limit of normal
• Fibrinogen < lower limit of normal
• Active bleeding
• 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
• Surgical procedure anticipated during the study period
• Suspected inability (e.g., language problems) or unwillingness to comply with study procedures or history of noncompliance