An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency - Full Text View

Sponsor:	CSL Behring
Information provided by:	CSL Behring
ClinicalTrials.gov Identifier:	NCT00945906

Purpose

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 and followed closely to determine that they receive the dose of FXIII Concentrate that will best minimize the chance of bruising and bleeding. The purpose of the study is to provide FXIII Concentrate to patients until the product becomes commercially available in the United States.

Condition	Intervention	Phase
Factor XIII Deficiency	Biological: FXIII Concentrate Human	Phase III

Study Type:	Interventional
Study Design:	Open Label, Single Group Assignment, Safety/Efficacy Study
Official Title:	A Prospective, Multicenter, Open Enrollment Study of Human Plasma Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Resource links provided by NLM:

Genetics Home Reference related topics: hemophilia Help Me Understand Genetics

U.S. FDA Resources

Further study details as provided by CSL Behring:

Primary Outcome Measures:

Adverse events [ Time Frame: For the duration of the study ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Hematology and chemistry testing [ Time Frame: For the duration of the study ] [ Designated as safety issue: Yes ]
FXIII antibody testing [ Time Frame: For the duration of the study ] [ Designated as safety issue: Yes ]
FXIII activity level [ Time Frame: Every 24 weeks for the duration of the study ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	80
Study Start Date:	September 2009
Estimated Study Completion Date:	January 2011
Estimated Primary Completion Date:	January 2011 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
FXIII: Experimental	Biological: FXIII Concentrate Human 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 10%. 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.

Arms

Assigned Interventions

FXIII: Experimental

Biological: FXIII Concentrate Human

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 10%.

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.

Eligibility

Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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 investigational medicinal product (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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00945906

Contacts

Contact: Central Contact: Clinical Trial Registration Coordinator

clinicaltrials@cslbehring.com

Locations

United States, South Dakota
Study Site	Recruiting
Sioux Falls, South Dakota, United States, 57105
Contact: Use Central Contact

Sponsors and Collaborators

CSL Behring

Investigators

Study Director:

Program Director, Clinical R&D

CSL Behring

More Information

No publications provided

Responsible Party:	CSL Behring ( Global Head Clinical Research & Development )
Study ID Numbers:	1488, BI71023_3002
Study First Received:	July 23, 2009
Last Updated:	October 19, 2009
ClinicalTrials.gov Identifier:	NCT00945906 History of Changes
Health Authority:	United States: Food and Drug Administration

Keywords provided by CSL Behring:

Hereditary Factor XIII deficiency
Factor XIII

Additional relevant MeSH terms:

Hemorrhagic Disorders
Blood Coagulation Disorders, Inherited
Genetic Diseases, Inborn
Coagulation Protein Disorders

Hematologic Diseases
Blood Coagulation Disorders
Factor XIII Deficiency

ClinicalTrials.gov processed this record on February 09, 2010