Clinical Trial of Factor XIII (FXIII) Concentrate

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00640289
Recruitment Status : Completed
First Posted : March 21, 2008
Last Update Posted : February 11, 2015
CSL Behring
Information provided by (Responsible Party):
Diane J. Nugent, MD, Children’s Hospital of Orange County

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.

Condition or disease Intervention/treatment Phase
Hemophilia Factor XIII Deficiency Drug: Fibrogammin P Not Applicable

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 73 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Clinical Research Study of Factor XIII Concentrate From Human Plasma Fibrogammin P in Patients With Factor XIII Deficiency
Study Start Date : January 2000
Actual Primary Completion Date : December 2011
Actual Study Completion Date : December 2011

Arm Intervention/treatment
Experimental: A
Drug: Fibrogammin P
Prophylaxis treatment

Primary Outcome Measures :
  1. Response to treatment. Active bleeding is controlled. [ Time Frame: Within 12 hours ]

Secondary Outcome Measures :
  1. Minimal to no bleeding with surgery following prophylactic treatment. [ Time Frame: During surgery. ]

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00640289

United States, California
Children's Hospital of Orange Co.
Orange, California, United States, 92868
Sponsors and Collaborators
Children’s Hospital of Orange County
CSL Behring
Principal Investigator: Diane J. Nugent, MD Children's Hospital of Orange Co.

Responsible Party: Diane J. Nugent, MD, Director Hemostasis/Thrombosis Research, Children’s Hospital of Orange County Identifier: NCT00640289     History of Changes
Other Study ID Numbers: BB-IND5986
First Posted: March 21, 2008    Key Record Dates
Last Update Posted: February 11, 2015
Last Verified: March 2008

Keywords provided by Diane J. Nugent, MD, Children’s Hospital of Orange County:
Factor XIII Deficiency
Rare Bleeding Disorder
Fibrogammin P

Additional relevant MeSH terms:
Hemophilia A
Factor XIII Deficiency
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Fibrinolytic Agents
Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action