Clinical Trial of Factor XIII (FXIII) Concentrate

Study Overview
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Brief Summary
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Congenital deficiency of Factor XIII is a rare but potentially life threatening disorder. It is inherited in an autosomal recessive fashion. Infusion of Factor XIII has proved to be useful for prevention and treatment of bleeding episodes, especially of spontaneous intracranial bleedings. In this study, Fibrogammin P will be given to patients with congenital Factor XIII deficiency and congenital/acquired FXIII deficiency to prevent bleeding and to treat established bleeding episodes. For Factor XIII prophylaxis to prevent hemorrhages, the dosage will depend on the weight of the subject. The frequency of Factor XIII administration will be determined by the factor's circulating half-life. During the first month only, a Factor XIII pharmacokinetic study will be determined over a 4-week period. Safety data will include accrual of information on viral safety, liver function, complete blood counts and adverse events. Historical data concerning spontaneous bleeds will be collected whenever possible two years prior to treatment with Fibrogammin P.

Official Title
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Clinical Research Study of Factor XIII Concentrate From Human Plasma Fibrogammin P in Patients With Factor XIII Deficiency

Conditions
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Hemophilia, Factor XIII Deficiency

Intervention / Treatment
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* Drug: Fibrogammin P


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients may be of either sex or age. Children and newborn infants are specifically included in this study. Patient must have documented congenital Factor XIII deficiency Patient or legal guardian must sign informed consent Patients who have negative serology for hepatitis B should receive Hepatitis B vaccination. Exclusion Criteria: Patient has acquired Factor XIII deficiency
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

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: A<br>Treatment | Drug: Fibrogammin P<br>* Prophylaxis treatment<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions | Response is defined as: Excellent/Good = adequate hemostasis, similar to that expected for subjects without known bleeding disorders; Fair/Poor = hemostasis less than expected; None = severe bleeding, judged due to disease despite Factor XIII (FXIII) therapy. Only the subjects who needed additional FXIII infusions (apart from the prophylactic treatment) to control a bleed and who had investigator assessment of efficacy were counted in this outcome. | Within 12 hours of FXIII infusion | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Surgical Efficacy Assessments With Factor XIII | Surgical efficacy is defined as: Excellent/Good = adequate hemostasis, similar to that expected for subjects without known bleeding disorders; Fair/Poor = hemostasis less than expected; None = severe bleeding, judged due to disease despite Factor XIII (FXIII) therapy. Only the subjects who underwent a surgical procedure were counted in this outcome measure. | During surgical procedure | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Factor XIII Deficiency, Rare Bleeding Disorder, Hemophilia, Fibrogammin P