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

Phase 3

Completed

Brief Summary: 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.

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
FXIII	FXIII Concentrate (Human) (FXIII)	Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.

Primary Outcome Measures

Name	Time	Method
Adverse Events	After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.	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 treatment is related, or possibly related and AEs with missing relationship.

Secondary Outcome Measures

Name	Time	Method
Hematology and Chemistry Testing	After the first infusion and at the end-of-study (or withdrawal) visit.	Number of participants with treatment-emergent clinically significant hematology and/or chemistry laboratory parameter values.
FXIII Antibody Testing	Before the first infusion, then every 48 weeks, at the end-of-study (or withdrawal) visit and after a bleeding episode requiring treatment with a Factor XIII -containing product.	Number of participants with serum Factor XIII antibodies.
FXIII Concentration	Before the first infusion, at 24 and 48 weeks after the first infusion, and at the end-of-study (or withdrawal) visit.	Trough Factor XIII concentration.
Number of Subjects With at Least One Bleeding Episode	After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.	Number of subjects with at least one bleeding episode at any time after the first infusion in the study, and the number of subjects with at least one bleeding episode requiring Factor XIII treatment.
Number of Bleeding Episodes	After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.	Number of bleeding episodes at any time after the first infusion in the study.