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NCT Number: NCT00945906

An Open Enrollment Study of 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.

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Key information

Conditions

Factor XIII Deficiency Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Study Site, Dothan, Alabama, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

FXIII Concentrate (Human) (FXIII)

Biological

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®

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

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

Important dates

Study start
2009
Primary completion
2011
Study completion
2011
First posted
Jul 24, 2009
Registry last updated
Oct 12, 2012

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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