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A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII 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.
 
ClinicalTrials.gov Identifier: NCT00885742
Recruitment Status : Completed
First Posted : April 22, 2009
Results First Posted : July 4, 2012
Last Update Posted : July 16, 2012
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Brief Summary:

Congenital deficiency of factor XIII (FXIII) is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII. Factor XIII Concentrate works by assisting blood in the usual clotting process, thereby preventing bleeding.

In this study, patients will be treated with FXIII Concentrate (Human) and followed closely to determine that they receive the dose that will best minimize the chance of bruising and bleeding.


Condition or disease Intervention/treatment Phase
Factor XIII Deficiency Biological: FXIII Concentrate (Human) Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 41 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Official Title: A Prospective, Multicenter, Open-label, Phase 3b Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Study Start Date : August 2009
Actual Primary Completion Date : April 2011
Actual Study Completion Date : April 2011


Arm Intervention/treatment
Experimental: FXIII
All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Biological: FXIII Concentrate (Human)

Doses will be guided by the individual subject's most recent FXIII activity levels, with the objective of dosing every 28 days to maintain a trough FXIII activity level of approximately 5 to 20%.

Subjects enrolled in this study who did not complete the pharmacokinetic study (Factor XIII Study BI71023_2002 [NCT00883090]) will initially receive FXIII Concentrate (Human) at a dose of 40 U/kg by intravenous (IV) infusion.

Other Name: Fibrogammin-P




Primary Outcome Measures :
  1. The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event) [ Time Frame: Up to week 52 ]
    The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.


Secondary Outcome Measures :
  1. Association of the Incidence of Spontaneous Bleeding Events Requiring Treatment and FXIII Activity Trough Levels [ Time Frame: 12 months ]
    P-value determined from Generalized Estimating Equation (GEE) model parameter estimates with bleeding as the response variable and FXIII activity trough level as the explanatory variable.

  2. Adverse Events [ Time Frame: 12 months ]
    Number of subjects with any treatment-emergent adverse event (AE), treatment-related AE or serious AE (SAE). Treatment related AEs are defined as AEs whose relationship to study treatment is related, or possibly related, and AEs with missing relationship.

  3. Peak FXIII Concentration at Steady State [ Time Frame: At 12, 24, 36 and 48 weeks: at 30 and 60 minutes after the end of the infusion. ]
  4. Trough FXIII Concentration at Steady State [ Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion. ]
  5. Time to Peak Concentration [ Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion. ]
  6. Incremental Recovery [ Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion. ]
    Incremental recovery (U/mL/U/kg) is defined as maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of (U/kg) infusion.

  7. Achievement of Trough Factor XIII Levels of 5% or Higher. [ Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion. ]
    Number of subjects with Factor XIII level ≥ 5% before infusion at Week 12, Week 24, Week 36 and Week 48.



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

Inclusion Criteria:

  • Written informed consent/assent for study participation obtained before undergoing any study-specific procedures
  • Documented congenital FXIII deficiency which 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)
  • Known Positivity for human immunodeficiency virus (HIV) or a positive result for HIV at the Screening Visit of this study or the FXIII study 2002 (NCT00883090).
  • Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) concentration >2.5 times the upper limit of normal at the Screening Visit of this study or at the Day 56 Visit of Factor XIII Study BI71023_2002 (NCT00883090)
  • Fibrinogen level less than 85% of the lower limit of normal at the Screening Visit of this study or the Factor XIII Study BI71023_2002 (NCT00883090)
  • Active bleeding ≥ Common Terminology Criteria for Adverse Events (CTCAE) Grade 2 and/or ≥ moderate between the Screening and Baseline Visits
  • 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
  • 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 ClinicalTrials.gov identifier (NCT number): NCT00885742


Locations
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United States, Alabama
Study Site
Dothan, Alabama, United States, 36305
United States, California
Study Site
Oakland, California, United States, 94610
Study Site
Orange, California, United States, 92868
Study Site
San Francisco, California, United States, 94115
Study Site
Stockton, California, United States, 95204
United States, Connecticut
Study Site
Hartford, Connecticut, United States, 06106
United States, Florida
Study Site
Fort Meyers, Florida, United States, 33908
Study
Miami, Florida, United States, 33136
United States, Idaho
Study Site
Boise, Idaho, United States, 83712
United States, Indiana
Study Site
South Bend, Indiana, United States, 46601
United States, Massachusetts
Study Site
Boston, Massachusetts, United States, 02115
United States, Minnesota
Study Site
St. Paul, Minnesota, United States, 55102
United States, Missouri
Study Site
Kansas City, Missouri, United States, 64108
United States, Nevada
Study Site
Las Vegas, Nevada, United States, 89015
United States, New Hampshire
Study Site
Lebanon, New Hampshire, United States, 03756
United States, New Jersey
Study Site
Newark, New Jersey, United States, 07102
United States, New York
Study Site
Albany, New York, United States, 12208
Study Site
New York, New York, United States, 10021
United States, North Carolina
Study Site
Chapel Hill, North Carolina, United States, 27599
United States, Pennsylvania
Study Site
Hershey, Pennsylvania, United States, 17033
United States, Texas
Study Site
Dallas, Texas, United States, 75390
United States, Wisconsin
Study Site
Milwaukee, Wisconsin, United States, 53233
Spain
Study Site
Santa Cruz de Tenerife, Spain, 38009
Sponsors and Collaborators
CSL Behring
Investigators
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Study Director: Program Director, Clinical R&D CSL Behring
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT00885742    
Other Study ID Numbers: BI71023_3001
1482 ( Other Identifier: CSL Behring )
2009-010722-19 ( EudraCT Number )
First Posted: April 22, 2009    Key Record Dates
Results First Posted: July 4, 2012
Last Update Posted: July 16, 2012
Last Verified: June 2012
Keywords provided by CSL Behring:
Hereditary Factor XIII deficiency
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
Fibrinolysin
Fibrinolytic Agents
Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action