Clinical Trial of Factor XIII (FXIII) Concentrate

Hemophilia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00640289
• Other study ID #: BB-IND5986
• Status: Completed
• Phase: N/A
• Start date: January 2000
• Est. completion date: December 2011

Study information

• Verified date: March 2008
• Source: Children’s Hospital of Orange County
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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 information / eligibility

• Status: Completed
• Enrollment: 72
• Est. completion date: December 2011
• Est. primary completion date: December 2011
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

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:

• Patient has acquired Factor XIII deficiency

Related Conditions & MeSH terms

• Factor XIII Deficiency
• Hemophilia
• Hemophilia A

Intervention

Drug:
• Fibrogammin P
Prophylaxis treatment

Locations

Country	Name	City	State
United States	Children's Hospital of Orange Co.	Orange	California

Sponsors (2)

Lead Sponsor	Collaborator
Children’s Hospital of Orange County	CSL Behring

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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