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

This study has been completed.
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
First received: April 16, 2009
Last updated: December 7, 2011
Last verified: December 2011
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 Intervention Phase
Factor XIII Deficiency
Biological: FXIII Concentrate (Human)
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:

Further study details as provided by CSL Behring:

Primary Outcome Measures:
  • Peak FXIII Concentration at Steady State [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Trough FXIII Concentration at Steady State [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Time to Peak Concentration [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Incremental Recovery [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
    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.

  • Terminal Half-life [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Area Under the Curve at Steady State [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Clearance [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Volume of Distribution at Steady State [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Mean Residence Time [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Adverse Events [ Time Frame: 16 weeks ] [ Designated as safety issue: Yes ]
    Number of participants with an adverse event

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

  • Vital Signs [ Time Frame: 16 weeks ] [ Designated as safety issue: Yes ]
    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.

Enrollment: 15
Study Start Date: May 2009
Study Completion Date: April 2010
Primary Completion Date: April 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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


Genders Eligible for Study:   Both
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
  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00883090

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
Study Director: Program Director, Clinical R&D CSL Behring
  More Information

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT00883090     History of Changes
Other Study ID Numbers: BI71023_2002  2009-010387-41  1479 
Study First Received: April 16, 2009
Results First Received: December 7, 2011
Last Updated: December 7, 2011
Health Authority: United States: Food and Drug Administration
Spain: Comité Ético de Investigación Clínica

Keywords provided by CSL Behring:
Factor XIII

Additional relevant MeSH terms:
Factor XIII Deficiency
Blood Coagulation Disorders
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Genetic Diseases, Inborn
Hematologic Diseases
Hemorrhagic Disorders

ClinicalTrials.gov processed this record on May 23, 2016