Clinical Trial of Factor XIII (FXIII) Concentrate

This study has been completed.
Sponsor:
Collaborator:
CSL Behring
Information provided by (Responsible Party):
Diane J. Nugent, MD, Children's Hospital of Orange County
ClinicalTrials.gov Identifier:
NCT00640289
First received: March 17, 2008
Last updated: February 9, 2015
Last verified: March 2008
  Purpose
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 Intervention
Hemophilia
Factor XIII Deficiency
Drug: Fibrogammin P

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Children’s Hospital of Orange County:

Primary Outcome Measures:
  • Response to treatment. Active bleeding is controlled. [ Time Frame: Within 12 hours ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Minimal to no bleeding with surgery following prophylactic treatment. [ Time Frame: During surgery. ] [ Designated as safety issue: No ]

Enrollment: 73
Study Start Date: January 2000
Study Completion Date: December 2011
Primary Completion Date: December 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: A
Treatment
Drug: Fibrogammin P
Prophylaxis treatment

  Eligibility

Ages Eligible for Study:   Child, Adult, Senior
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00640289

Locations
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
Investigators
Principal Investigator: Diane J. Nugent, MD Children's Hospital of Orange Co.
  More Information

Responsible Party: Diane J. Nugent, MD, Director Hemostasis/Thrombosis Research, Children's Hospital of Orange County
ClinicalTrials.gov Identifier: NCT00640289     History of Changes
Other Study ID Numbers: BB-IND5986 
Study First Received: March 17, 2008
Last Updated: February 9, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Children’s Hospital of Orange County:
Factor XIII Deficiency
Rare Bleeding Disorder
Hemophilia
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
Fibrinolysin
Fibrinolytic Agents
Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on July 28, 2016