Clinical Trial of Factor XIII (FXIII) Concentrate

Children's Health of Orange County

Status: Completed

Conditions: Factor XIII Deficiency; Hemophilia

Treatments: Drug: Fibrogammin P

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT00640289

BB-IND5986

Details and patient eligibility

About

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.

Enrollment: 72 patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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72 participants in 1 patient group

A

Experimental group

Description:

Treatment

Treatment:

Drug: Fibrogammin P