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

December 7, 2011 updated by: CSL Behring

A 12 Week, Multicenter, Pharmacokinetic and Safety Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Spain
      • Santa Cruz de Tenerife, Spain
        • Study Site
  • United States
    • Alabama
      • Dothan, Alabama, United States, 36305
        • Study Site
    • California
      • Orange, California, United States, 92868
        • Study Site
      • San Francisco, California, United States, 94115
        • Study Site
      • Stockton, California, United States, 95204
        • Study Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Study Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • ADULT
  • OLDER_ADULT
  • CHILD

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: FXIII
All subjects treated with Factor XIII Concentrate (Human) (FXIII)
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.
Other Names:
  • Fibrogammin®-P

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Peak FXIII Concentration at Steady State
Time Frame: 12 weeks
12 weeks
Trough FXIII Concentration at Steady State
Time Frame: 12 weeks
12 weeks
Time to Peak Concentration
Time Frame: 12 weeks
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.
12 weeks
Terminal Half-life
Time Frame: 12 weeks
12 weeks
Area Under the Curve at Steady State
Time Frame: 12 weeks
12 weeks
Clearance
Time Frame: 12 weeks
12 weeks
Volume of Distribution at Steady State
Time Frame: 12 weeks
12 weeks
Mean Residence Time
Time Frame: 12 weeks
12 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adverse Events
Time Frame: 16 weeks
Number of participants with an adverse event
16 weeks
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.
16 weeks
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.
16 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CSL Behring

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2009

Primary Completion (ACTUAL)

April 1, 2010

Study Completion (ACTUAL)

April 1, 2010

Study Registration Dates

First Submitted

April 16, 2009

First Submitted That Met QC Criteria

April 16, 2009

First Posted (ESTIMATE)

April 17, 2009

Study Record Updates

Last Update Posted (ESTIMATE)

January 16, 2012

Last Update Submitted That Met QC Criteria

December 7, 2011

Last Verified

December 1, 2011

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BI71023_2002
  • 2009-010387-41 (EUDRACT_NUMBER)
  • 1479 (CSL Behring)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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