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An Open Enrollment 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: NCT00945906
Recruitment Status : Completed
First Posted : July 24, 2009
Results First Posted : October 12, 2012
Last Update Posted : October 12, 2012
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Tracking Information
First Submitted Date  ICMJE July 23, 2009
First Posted Date  ICMJE July 24, 2009
Results First Submitted Date  ICMJE September 12, 2012
Results First Posted Date  ICMJE October 12, 2012
Last Update Posted Date October 12, 2012
Study Start Date  ICMJE September 2009
Actual Primary Completion Date August 2011   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 12, 2012)
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. ]
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.
Original Primary Outcome Measures  ICMJE
 (submitted: July 23, 2009)
Adverse Events [ Time Frame: For the duration of the study ]
Change History Complete list of historical versions of study NCT00945906 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: September 12, 2012)
  • Hematology and Chemistry Testing [ Time Frame: After the first infusion and at the end-of-study (or withdrawal) visit. ]
    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. ]
    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. ]
    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. ]
    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. ]
    Number of bleeding episodes at any time after the first infusion in the study.
Original Secondary Outcome Measures  ICMJE
 (submitted: July 23, 2009)
  • Hematology and Chemistry Testing [ Time Frame: For the duration of the study ]
  • FXIII Antibody Testing [ Time Frame: For the duration of the study ]
  • FXIII activity level [ Time Frame: Every 24 weeks for the duration of the study ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Official Title  ICMJE A Prospective, Multicenter, Open Enrollment Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Brief Summary

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.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Condition  ICMJE Factor XIII Deficiency
Intervention  ICMJE 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®
Study Arms  ICMJE 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%.
Intervention: Biological: FXIII Concentrate (Human) (FXIII)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 21, 2011)
61
Original Estimated Enrollment  ICMJE
 (submitted: July 23, 2009)
80
Actual Study Completion Date  ICMJE August 2011
Actual Primary Completion Date August 2011   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00945906
Other Study ID Numbers  ICMJE BI71023_3002
1488 ( Other Identifier: CSL Behring )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party CSL Behring
Study Sponsor  ICMJE CSL Behring
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Program Director, Clinical R&D CSL Behring
PRS Account CSL Behring
Verification Date September 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP