A 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:
NCT00885742
First received: April 21, 2009
Last updated: July 10, 2012
Last verified: June 2012
  Purpose

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 Intervention Phase
Factor XIII Deficiency
Biological: FXIII Concentrate (Human)
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-label, Phase 3b 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:
  • 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 ] [ Designated as safety issue: No ]
    The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.


Secondary Outcome Measures:
  • Association of the Incidence of Spontaneous Bleeding Events Requiring Treatment and FXIII Activity Trough Levels [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    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.

  • Adverse Events [ Time Frame: 12 months ] [ 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 study treatment is related, or possibly related, and AEs with missing relationship.

  • 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. ] [ Designated as safety issue: No ]
  • Trough FXIII Concentration at Steady State [ Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion. ] [ Designated as safety issue: No ]
  • 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. ] [ Designated as safety issue: No ]
  • 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. ] [ Designated as safety issue: No ]
    Incremental recovery (U/mL/U/kg) is defined as maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of (U/kg) infusion.

  • Achievement of Trough Factor XIII Levels of 5% or Higher. [ Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion. ] [ Designated as safety issue: No ]
    Number of subjects with Factor XIII level ≥ 5% before infusion at Week 12, Week 24, Week 36 and Week 48.


Enrollment: 41
Study Start Date: August 2009
Study Completion Date: April 2011
Primary Completion Date: April 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  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
  • 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00885742

  Show 23 Study Locations
Sponsors and Collaborators
CSL Behring
Investigators
Study Director: Program Director, Clinical R&D CSL Behring
  More Information

Additional Information:
No publications provided

Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT00885742     History of Changes
Other Study ID Numbers: BI71023_3001, 1482, 2009-010722-19
Study First Received: April 21, 2009
Results First Received: April 30, 2012
Last Updated: July 10, 2012
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Spain: Spanish Agency of Medicines

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 April 14, 2014