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

Study Overview
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Brief Summary
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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.

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

Conditions
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Factor XIII Deficiency

Intervention / Treatment
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* Biological: FXIII Concentrate (Human) (FXIII)


Participation Criteria
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Eligibility Criteria
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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
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: FXIII<br>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%. | Biological: FXIII Concentrate (Human) (FXIII)<br>* Doses will be guided by the individual subject's most recent FXIII activity levels, with the objective of dosing every 28 days to maintain a trough FXIII activity level of approximately 5 to 20%. Subjects enrolled in this study who have not received at least 3 doses of FXIII Concentrate in a previous study of this product (i.e., NCT00640289, NCT00885742, or NCT00883090) will initially receive a dose of 40 U/kg by intravenous (IV) infusion.<br>* Other names: Corifact®;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse Events | 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. | 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. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hematology and Chemistry Testing | Number of participants with treatment-emergent clinically significant hematology and/or chemistry laboratory parameter values. | After the first infusion and at the end-of-study (or withdrawal) visit. | 
| FXIII Antibody Testing | Number of participants with serum Factor XIII antibodies. | 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. | 
| FXIII Concentration | Trough Factor XIII concentration. | Before the first infusion, at 24 and 48 weeks after the first infusion, and at the end-of-study (or withdrawal) visit. | 
| Number of Subjects With at Least One Bleeding Episode | 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. | 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 | Number of bleeding episodes at any time after the first infusion in the study. | 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. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hereditary Factor XIII deficiency, Factor XIII