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A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency

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: NCT00883090
Recruitment Status : Completed
First Posted : April 17, 2009
Results First Posted : January 16, 2012
Last Update Posted : January 16, 2012
Information provided by (Responsible Party):
CSL Behring

Brief Summary:
Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding. This study will evaluate the safety and recommended (best) amount or level of Factor XIII in a patient's blood. Factor XIII Concentrate (Human) is given to people whose blood is lacking Factor XIII. Factor XIII Concentrate (Human) works by assisting your blood in the usual clotting process, thereby preventing bleeding.

Condition or disease Intervention/treatment Phase
Factor XIII Deficiency Biological: FXIII Concentrate (Human) Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Official Title: A 12 Week, Multicenter, Pharmacokinetic and Safety Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Study Start Date : May 2009
Actual Primary Completion Date : April 2010
Actual Study Completion Date : April 2010

Arm Intervention/treatment
Experimental: FXIII
All subjects treated with Factor XIII Concentrate (Human) (FXIII)
Biological: FXIII Concentrate (Human)
Subjects will receive approximately 40 U/kg of FXIII every 28 days for 3 doses administered as a bolus intravenous (IV) injection at approximately 250 U/minute.
Other Name: Fibrogammin®-P

Primary Outcome Measures :
  1. Peak FXIII Concentration at Steady State [ Time Frame: 12 weeks ]
  2. Trough FXIII Concentration at Steady State [ Time Frame: 12 weeks ]
  3. Time to Peak Concentration [ Time Frame: 12 weeks ]
  4. Incremental Recovery [ Time Frame: 12 weeks ]
    Incremental recovery (U/mL/U/kg) is defined as the maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of FXIII (U/kg) administered.

  5. Terminal Half-life [ Time Frame: 12 weeks ]
  6. Area Under the Curve at Steady State [ Time Frame: 12 weeks ]
  7. Clearance [ Time Frame: 12 weeks ]
  8. Volume of Distribution at Steady State [ Time Frame: 12 weeks ]
  9. Mean Residence Time [ Time Frame: 12 weeks ]

Secondary Outcome Measures :
  1. Adverse Events [ Time Frame: 16 weeks ]
    Number of participants with an adverse event

  2. Laboratory Safety Parameters [ Time Frame: 16 weeks ]
    Number of participants with clinically significant laboratory safety parameter values. The laboratory safety parameters measured included serum chemistries, hematology and urinalysis.

  3. Vital Signs [ Time Frame: 16 weeks ]
    Number of participants with clinically significant vital signs. The vital signs measured included blood pressure, pulse rate and temperature. Clinically significant changes in vital signs were to be reported as adverse events.

Information from the National Library of Medicine

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

Inclusion Criteria:

  • Written informed consent/assent for study participation obtained before undergoing any study-specific procedures
  • Documented congenital FXIII deficiency that requires prophylactic treatment with a FXIII containing product.
  • Males and females of any age with congenital FXIII deficiency.
  • Received full hepatitis B vaccination and/or is hepatitis B surface antibody positive

Exclusion Criteria:

  • Diagnosis of acquired FXIII deficiency
  • Administration of a FXIII-containing product, including blood transfusions or other blood products within 4 weeks prior to the planned Day 0
  • Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
  • Known or suspected to have antibodies towards FXIII
  • Use of any other investigational medicinal product within 4 weeks prior to the Baseline Visit (Day 0)
  • Positive result at screening for human immunodeficiency virus (HIV)
  • Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) concentration >2.5 times the upper limit of normal
  • Fibrinogen < lower limit of normal
  • Active bleeding
  • Pregnant or breast-feeding
  • Intention to become pregnant during the course of the study
  • Female subjects of childbearing potential not using, or not willing to use, a medically reliable method of contraception for the entire duration of the study
  • Surgical procedure anticipated during the study period
  • Suspected inability (e.g., language problems) or unwillingness to comply with study procedures or history of noncompliance

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): NCT00883090

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United States, Alabama
Study Site
Dothan, Alabama, United States, 36305
United States, California
Study Site
Orange, California, United States, 92868
Study Site
San Francisco, California, United States, 94115
Study Site
Stockton, California, United States, 95204
United States, Massachusetts
Study Site
Boston, Massachusetts, United States, 02115
Study Site
Santa Cruz de Tenerife, Spain
Sponsors and Collaborators
CSL Behring
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Study Director: Program Director, Clinical R&D CSL Behring
Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: CSL Behring Identifier: NCT00883090    
Other Study ID Numbers: BI71023_2002
2009-010387-41 ( EudraCT Number )
1479 ( Other Identifier: CSL Behring )
First Posted: April 17, 2009    Key Record Dates
Results First Posted: January 16, 2012
Last Update Posted: January 16, 2012
Last Verified: December 2011
Keywords provided by CSL Behring:
Factor XIII
Additional relevant MeSH terms:
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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