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

Recruitment & Eligibility

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

Study & Design

Arm && Interventions

Group	Intervention	Description
FXIII	FXIII Concentrate (Human)	All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).

Primary Outcome Measures

Name	Time	Method
The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event)	Up to week 52	The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.

Secondary Outcome Measures

Name	Time	Method
Association of the Incidence of Spontaneous Bleeding Events Requiring Treatment and FXIII Activity Trough Levels	12 months	P-value determined from Generalized Estimating Equation (GEE) model parameter estimates with bleeding as the response variable and FXIII activity trough level as the explanatory variable.
Adverse Events	12 months	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 study treatment is related, or possibly related, and AEs with missing relationship.
Peak FXIII Concentration at Steady State	At 12, 24, 36 and 48 weeks: at 30 and 60 minutes after the end of the infusion.
Trough FXIII Concentration at Steady State	At 12, 24, 36 and 48 weeks: immediately before infusion.
Time to Peak Concentration	At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.
Incremental Recovery	At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.	Incremental recovery (U/mL/U/kg) is defined as maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of (U/kg) infusion.
Achievement of Trough Factor XIII Levels of 5% or Higher.	At 12, 24, 36 and 48 weeks: immediately before infusion.	Number of subjects with Factor XIII level ≥ 5% before infusion at Week 12, Week 24, Week 36 and Week 48.

Recruitment & Eligibility