A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency

Overview

Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding. This study will evaluate the safety and recommended (best) amount or level of Factor XIII in a patient's blood. Factor XIII Concentrate (Human) is given to people whose blood is lacking Factor XIII. Factor XIII Concentrate (Human) works by assisting your blood in the usual clotting process, thereby preventing bleeding.

Full Title of Study: “A 12 Week, Multicenter, Pharmacokinetic and Safety 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 2010

Interventions

  • Biological: FXIII Concentrate (Human)
    • Subjects will receive approximately 40 U/kg of FXIII every 28 days for 3 doses administered as a bolus intravenous (IV) injection at approximately 250 U/minute.

Arms, Groups and Cohorts

  • Experimental: FXIII
    • All subjects treated with Factor XIII Concentrate (Human) (FXIII)

Clinical Trial Outcome Measures

Primary Measures

  • Peak FXIII Concentration at Steady State
    • Time Frame: 12 weeks
  • Trough FXIII Concentration at Steady State
    • Time Frame: 12 weeks
  • Time to Peak Concentration
    • Time Frame: 12 weeks
  • Incremental Recovery
    • Time Frame: 12 weeks
    • Incremental recovery (U/mL/U/kg) is defined as the maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of FXIII (U/kg) administered.
  • Terminal Half-life
    • Time Frame: 12 weeks
  • Area Under the Curve at Steady State
    • Time Frame: 12 weeks
  • Clearance
    • Time Frame: 12 weeks
  • Volume of Distribution at Steady State
    • Time Frame: 12 weeks
  • Mean Residence Time
    • Time Frame: 12 weeks

Secondary Measures

  • Adverse Events
    • Time Frame: 16 weeks
    • Number of participants with an adverse event
  • Laboratory Safety Parameters
    • Time Frame: 16 weeks
    • Number of participants with clinically significant laboratory safety parameter values. The laboratory safety parameters measured included serum chemistries, hematology and urinalysis.
  • Vital Signs
    • Time Frame: 16 weeks
    • Number of participants with clinically significant vital signs. The vital signs measured included blood pressure, pulse rate and temperature. Clinically significant changes in vital signs were to be reported as adverse events.

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 that 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)
  • Positive result at screening for human immunodeficiency virus (HIV)
  • Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) concentration >2.5 times the upper limit of normal
  • Fibrinogen < lower limit of normal
  • Active bleeding
  • 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
  • Surgical procedure anticipated during the study period
  • 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

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