A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Overview

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.

Full Title of Study: “A Prospective, Multicenter, Open-label, Phase 3b Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Masking: None (Open Label)
  • Study Primary Completion Date: April 2011

Interventions

  • 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.

Arms, Groups and Cohorts

  • Experimental: FXIII
    • All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).

Clinical Trial Outcome Measures

Primary 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
    • The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.

Secondary Measures

  • 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.
  • 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.
  • 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.
  • Trough FXIII Concentration at Steady State
    • Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion.
  • 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.
  • 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.
  • 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.

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: N/A

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • CSL Behring
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Program Director, Clinical R&D, Study Director, CSL Behring

Clinical trials entries are delivered from the US National Institutes of Health and are not reviewed separately by this site. Please see the identifier information above for retrieving further details from the government database.

At TrialBulletin.com, we keep tabs on over 200,000 clinical trials in the US and abroad, using medical data supplied directly by the US National Institutes of Health. Please see the About and Contact page for details.