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Clinical Trial of Factor XIII Concentrate

This study is currently recruiting participants.
Verified by Children’s Hospital of Orange County, March 2008

Sponsors and Collaborators: Children’s Hospital of Orange County
CSL Behring
Information provided by: Children’s Hospital of Orange County
ClinicalTrials.gov Identifier: NCT00640289
  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

Genetics Home Reference related topics:   hemophilia   

ChemIDplus related topics:   Fibrinolysin, human   

U.S. FDA Resources

Study Type:   Interventional
Study Design:   Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:   Clinical Research Study of Factor XIII Concentrate From Human Plasma Fibrogammin P in Patients With Factor XIII Deficiency

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 ]

Estimated Enrollment:   80
Study Start Date:   January 2000
Estimated Study Completion Date:   December 2010
Estimated Primary Completion Date:   December 2010 (Final data collection date for primary outcome measure)

Arms Assigned Interventions
A: Experimental
Treatment
Drug: Fibrogammin P
Prophylaxis treatment

  Eligibility
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

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

Contacts
Contact: Diane J. Nugent, MD     714-532-8744     djn0@choc.org    
Contact: Kathie J. Birschbach     714-532-8777     kjb0@choc.org    

Locations
United States, California
Children's Hospital of Orange Co.     Recruiting
      Orange, California, United States, 92868
      Contact: Diane J. Nugent, MD     714-532-8744     djn0@choc.org    
      Contact: Kathie J. Birschbach, MD     714-532-8744     kjb0@choc.org    

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:   Director of Hematology at Childrens Hospitl of Orange County ( Diane Nugent, MD )
Study ID Numbers:   BB-IND5986
First Received:   March 17, 2008
Last Updated:   March 17, 2008
ClinicalTrials.gov Identifier:   NCT00640289
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  

Study placed in the following topic categories:
Plasmin
Hemorrhagic Disorders
Genetic Diseases, Inborn
Hematologic Diseases
Blood Coagulation Disorders
Hemophilia A
Factor XIII Deficiency
Hemostatic Disorders
Hemorrhage

Additional relevant MeSH terms:
Fibrin Modulating Agents
Blood Coagulation Disorders, Inherited
Molecular Mechanisms of Pharmacological Action
Coagulation Protein Disorders
Therapeutic Uses
Hematologic Agents
Fibrinolytic Agents
Cardiovascular Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on October 17, 2008




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