An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

A Prospective, Multicenter, Open Enrollment Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

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.

Inclusion Criteria:

• Written informed consent/assent for study participation obtained before undergoing any study specific procedures
• Diagnosed with congenital FXIII deficiency requiring prophylactic treatment
• Males and females of any age

Exclusion Criteria:

• Diagnosis of acquired FXIII deficiency
• Administration of a FXIII-containing product, including blood transfusions or other blood products, within 3 weeks prior to the Baseline/Day 0 Visit
• Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
• Use of any other IMP within 4 weeks prior to Baseline/Day 0 Visit
• 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
• Any laboratory finding or medical condition which, in the opinion of the Investigator, would put the subject or subject's disease management at risk