Clinical Trial of Factor XIII (FXIII) Concentrate

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

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.

Study Overview

• Status: Completed
• Conditions: Hemophilia; Factor XIII Deficiency
• Intervention / Treatment: Drug: Fibrogammin P

• Study Type: Interventional
• Enrollment (Actual): 72
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • California
    • Orange, California, United States, 92868
      • Children's Hospital of Orange Co.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: A; Treatment	Drug: Fibrogammin P; Prophylaxis treatment

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Children's Hospital of Orange County
• Collaborators: CSL Behring
• Investigators: Principal Investigator: Diane J. Nugent, MD, Children's Hospital of Orange Co.

Study record dates

Study Major Dates

• Study Start: January 1, 2000
• Primary Completion (Actual): December 1, 2011
• Study Completion (Actual): December 1, 2011

Study Registration Dates

• First Submitted: March 17, 2008
• First Submitted That Met QC Criteria: March 17, 2008
• First Posted (Estimate): March 21, 2008

Study Record Updates

• Last Update Posted (Actual): November 7, 2018
• Last Update Submitted That Met QC Criteria: October 11, 2018
• Last Verified: March 1, 2008

More Information

Terms related to this study

• Keywords: Hemophilia; Factor XIII Deficiency; Rare Bleeding Disorder; Fibrogammin P
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A; Factor XIII Deficiency; Molecular Mechanisms of Pharmacological Action; Fibrinolytic Agents; Fibrin Modulating Agents; Fibrinolysin
• Other Study ID Numbers: BB-IND5986