Clinical Trial of Factor XIII (FXIII) Concentrate

Not Applicable

Completed

Brief Summary: 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.

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
A	Fibrogammin P	Treatment

Primary Outcome Measures

Name	Time	Method
Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions	Within 12 hours of FXIII infusion	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.

Secondary Outcome Measures

Name	Time	Method
Surgical Efficacy Assessments With Factor XIII	During surgical procedure	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.