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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
61

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 1, 2009

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 1, 2011

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 1, 2011

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 23, 2009

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 23, 2009

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 24, 2009

Study Record Updates

Last Update Posted (Estimate)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 12, 2012

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 12, 2012

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2012

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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