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An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT00945906
First received: July 23, 2009
Last updated: September 12, 2012
Last verified: September 2012
History of Changes
  Purpose

Congenital deficiency of factor XIII 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 of FXIII Concentrate (Human) that will best minimize the chance of bruising and bleeding. The purpose of the study is to provide FXIII Concentrate (Human) to patients until the product becomes commercially available in the United States.


Condition Intervention Phase
Factor XIII Deficiency
 Biological: FXIII Concentrate (Human) (FXIII)
 Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Official Title: A Prospective, Multicenter, Open Enrollment Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by CSL Behring:

Primary Outcome Measures:
  • Adverse Events [ Time Frame: After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA. ] [ Designated as safety issue: Yes ]
    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 treatment is related, or possibly related and AEs with missing relationship.


Secondary Outcome Measures:
  • Hematology and Chemistry Testing [ Time Frame: After the first infusion and at the end-of-study (or withdrawal) visit. ] [ Designated as safety issue: Yes ]
    Number of participants with treatment-emergent clinically significant hematology and/or chemistry laboratory parameter values.

  • FXIII Antibody Testing [ Time Frame: Before the first infusion, then every 48 weeks, at the end-of-study (or withdrawal) visit and after a bleeding episode requiring treatment with a Factor XIII -containing product. ] [ Designated as safety issue: Yes ]
    Number of participants with serum Factor XIII antibodies.

  • FXIII Concentration [ Time Frame: Before the first infusion, at 24 and 48 weeks after the first infusion, and at the end-of-study (or withdrawal) visit. ] [ Designated as safety issue: Yes ]
    Trough Factor XIII concentration.

  • Number of Subjects With at Least One Bleeding Episode [ Time Frame: After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA. ] [ Designated as safety issue: Yes ]
    Number of subjects with at least one bleeding episode at any time after the first infusion in the study, and the number of subjects with at least one bleeding episode requiring Factor XIII treatment.

  • Number of Bleeding Episodes [ Time Frame: After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA. ] [ Designated as safety issue: Yes ]
    Number of bleeding episodes at any time after the first infusion in the study.


Enrollment: 61
Study Start Date: September 2009
Study Completion Date: August 2011
Primary Completion Date: August 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: FXIII
Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.
 Biological: FXIII Concentrate (Human) (FXIII)

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 have not received at least 3 doses of FXIII Concentrate in a previous study of this product (i.e., NCT00640289, NCT00885742, or NCT00883090) will initially receive a dose of 40 U/kg by intravenous (IV) infusion.

Other Names:
  • Fibrogammin-P®
  • Corifact®

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Written informed consent/assent for study participation obtained before undergoing any study specific procedures
  • Diagnosed with congenital FXIII deficiency requiring prophylactic treatment
  • Males and females of any age

Exclusion Criteria:

  • Diagnosis of acquired FXIII deficiency
  • Administration of a FXIII-containing product, including blood transfusions or other blood products, within 3 weeks prior to the Baseline/Day 0 Visit
  • Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
  • Use of any other IMP within 4 weeks prior to Baseline/Day 0 Visit
  • 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
  • Any laboratory finding or medical condition which, in the opinion of the Investigator, would put the subject or subject's disease management at risk
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00945906

  Show 25 Study Locations
Sponsors and Collaborators
CSL Behring
Investigators
Study Director: Program Director, Clinical R&D CSL Behring
  More Information

Additional Information:
Click here to request more information about this study  This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT00945906     History of Changes
Other Study ID Numbers: BI71023_3002, 1488
Study First Received: July 23, 2009
Results First Received: September 12, 2012
Last Updated: September 12, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by CSL Behring:
Hereditary Factor XIII deficiency
Factor XIII

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

ClinicalTrials.gov processed this record on May 16, 2013