An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

September 12, 2012 updated by: CSL Behring

A Prospective, Multicenter, Open Enrollment Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

61

Phase

  • Phase 3

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

  • United States
    • Alabama
      • Dothan, Alabama, United States, 36301
        • Study Site
    • California
      • Oakland, California, United States, 94609
        • Study Site
      • Orange, California, United States, 92868
        • Study Site
      • San Francisco, California, United States, 94118
        • Study Site
      • Stockton, California, United States, 95204
        • Study Site
    • Florida
      • Fort Myers, Florida, United States, 33908
        • Study Site
      • Miami, Florida, United States, 33136
        • Study Site
      • St. Petersburg, Florida, United States, 33701
        • Study Site
    • Louisiana
      • New Orleans, Louisiana, United States, 70121
        • Study Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Study Site
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Study Site
      • Detroit, Michigan, United States, 48201
        • Study Site
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Study Site
    • Nevada
      • Las Vegas, Nevada, United States, 89109
        • Study Site
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
        • Study Site
    • New York
      • Albany, New York, United States, 12208
        • Study Site
      • New York, New York, United States, 10065
        • Study Site
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • Study Site
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Study Site
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Study Site
    • South Dakota
      • Sioux Falls, South Dakota, United States, 57105
        • Study Site
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Study Site
    • Texas
      • Dallas, Texas, United States, 75390
        • Study Site
      • Houston, Texas, United States, 77030
        • Study Site
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53223
        • 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

  • Child
  • Adult
  • Older Adult

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
  • 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

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: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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%.
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®

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
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.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
After the first infusion and at the end-of-study (or withdrawal) visit.
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.
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.
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.
Before the first infusion, at 24 and 48 weeks after the first infusion, and at the end-of-study (or withdrawal) visit.
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.
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
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.
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.

Collaborators and Investigators

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

Sponsor

CSL Behring

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

September 1, 2009

Primary Completion (Actual)

August 1, 2011

Study Completion (Actual)

August 1, 2011

Study Registration Dates

First Submitted

July 23, 2009

First Submitted That Met QC Criteria

July 23, 2009

First Posted (Estimate)

July 24, 2009

Study Record Updates

Last Update Posted (Estimate)

October 12, 2012

Last Update Submitted That Met QC Criteria

September 12, 2012

Last Verified

September 1, 2012

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BI71023_3002
  • 1488 (Other Identifier: 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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