A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency

Study Overview
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Brief Summary
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Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding. This study will evaluate the safety and recommended (best) amount or level of Factor XIII in a patient's blood. Factor XIII Concentrate (Human) is given to people whose blood is lacking Factor XIII. Factor XIII Concentrate (Human) works by assisting your blood in the usual clotting process, thereby preventing bleeding.

Official Title
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A 12 Week, Multicenter, Pharmacokinetic and Safety 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)


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 Documented congenital FXIII deficiency that 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) Positive result at screening for human immunodeficiency virus (HIV) Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) concentration >2.5 times the upper limit of normal Fibrinogen < lower limit of normal Active bleeding 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 Surgical procedure anticipated during the study period Suspected inability (e.g., language problems) or unwillingness to comply with study procedures or history of noncompliance
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>All subjects treated with Factor XIII Concentrate (Human) (FXIII) | Biological: FXIII Concentrate (Human)<br>* Subjects will receive approximately 40 U/kg of FXIII every 28 days for 3 doses administered as a bolus intravenous (IV) injection at approximately 250 U/minute.<br>* Other names: Fibrogammin®-P;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Peak FXIII Concentration at Steady State |  | 12 weeks | 
| Trough FXIII Concentration at Steady State |  | 12 weeks | 
| Time to Peak Concentration |  | 12 weeks | 
| Incremental Recovery | Incremental recovery (U/mL/U/kg) is defined as the maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of FXIII (U/kg) administered. | 12 weeks | 
| Terminal Half-life |  | 12 weeks | 
| Area Under the Curve at Steady State |  | 12 weeks | 
| Clearance |  | 12 weeks | 
| Volume of Distribution at Steady State |  | 12 weeks | 
| Mean Residence Time |  | 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse Events | Number of participants with an adverse event | 16 weeks | 
| Laboratory Safety Parameters | Number of participants with clinically significant laboratory safety parameter values. The laboratory safety parameters measured included serum chemistries, hematology and urinalysis. | 16 weeks | 
| Vital Signs | Number of participants with clinically significant vital signs. The vital signs measured included blood pressure, pulse rate and temperature. Clinically significant changes in vital signs were to be reported as adverse events. | 16 weeks | 

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