The purpose of this study is to determine the safety of increasing doses of rFXIII in subjects with Congenital Factor XIII Deficiency.

This is a Phase 1, single-site, open-label study to evaluate the safety and pharmacokinetics of escalating doses of rFXIII in subjects with Congenital Factor XIII Deficiency.

Ages Eligible for Study:  18 Years and above,  Genders Eligible for Study:  Both

Criteria

Inclusion Criteria

• Subject is at least 18 years of age at the time of enrollment.
• Subject has documented congenital FXIII deficiency.
• Subject has normal platelet count and clotting parameters defined as platelet count, PT, PTT, INR, thrombin time and fibrinogen within 2.5% of normal limits.
• Subject has adequate renal and hepatic function defined as: creatinine is less or equal to 2.0 mg%, bilirubin is less or equal 1.5 times upper limit of normal; alkaline phosphatase is less or equal to 3 times upper limit of normal and AST is less or equal to 3 times upper limit of normal.
• If female and of child-bearing potential, subject has a negative serum pregnancy test within seven (7) days of enrollment.
• If subject is a sexually active male or a sexually active female of child-bearing potential, subject agrees to use a medically accepted form of contraception from the time of enrollment to completion of all follow-up study visits.
• Subject has a negative drug and alcohol screen.
• Subject agrees to abstain from alcohol intake for 24 hours prior to admittance to the test facility and during the confinement period.
• Patient has signed an IRB-approved informed consent form.

Exclusion Criteria

• Subject has received blood products or FXIII concentrates within four weeks of study enrollment.
• Subject has known antibodies to FXIII.
• Subject has a hereditary or acquired coagulation disorder other than FXIII deficiency.
• Subject has a previous history of autoimmune disorder involving autoantibodies, e.g., systemic lupus erythematosus.
• Subject has a previous history of thromboembolic events, e.g., cerebrovascular accident or deep vein thrombosis or who has received any antithrombotic or anti-platelet drugs within 7 days of study enrollment.
• Subject has received treatment with any experimental agent within 30 days of study enrollment.
• Subject is pregnant or lactating.
• Subject has any concurrent serious chronic or acute illness or infection.
• Subject has had any surgical procedure in the 30 days prior to enrollment.
• Subject has donated blood within 30 days prior to enrollment.
• Subject has medical, social or psychosocial factors expected to impact compliance or safety.