An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00945906|
Recruitment Status : Completed
First Posted : July 24, 2009
Results First Posted : October 12, 2012
Last Update Posted : October 12, 2012
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.
|Condition or disease||Intervention/treatment||Phase|
|Factor XIII Deficiency||Biological: FXIII Concentrate (Human) (FXIII)||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||61 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Prospective, Multicenter, Open Enrollment Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency|
|Study Start Date :||September 2009|
|Actual Primary Completion Date :||August 2011|
|Actual Study Completion Date :||August 2011|
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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00945906
|United States, Alabama|
|Dothan, Alabama, United States, 36301|
|United States, California|
|Oakland, California, United States, 94609|
|Orange, California, United States, 92868|
|San Francisco, California, United States, 94118|
|Stockton, California, United States, 95204|
|United States, Florida|
|Fort Myers, Florida, United States, 33908|
|Miami, Florida, United States, 33136|
|St. Petersburg, Florida, United States, 33701|
|United States, Louisiana|
|New Orleans, Louisiana, United States, 70121|
|United States, Massachusetts|
|Boston, Massachusetts, United States, 02115|
|United States, Michigan|
|Ann Arbor, Michigan, United States, 48109|
|Detroit, Michigan, United States, 48201|
|United States, Missouri|
|Kansas City, Missouri, United States, 64108|
|United States, Nevada|
|Las Vegas, Nevada, United States, 89109|
|United States, New Hampshire|
|Lebanon, New Hampshire, United States, 03756|
|United States, New York|
|Albany, New York, United States, 12208|
|New York, New York, United States, 10065|
|United States, North Carolina|
|Chapel Hill, North Carolina, United States, 27599|
|United States, Ohio|
|Columbus, Ohio, United States, 43205|
|United States, Pennsylvania|
|Hershey, Pennsylvania, United States, 17033|
|United States, South Dakota|
|Sioux Falls, South Dakota, United States, 57105|
|United States, Tennessee|
|Nashville, Tennessee, United States, 37232|
|United States, Texas|
|Dallas, Texas, United States, 75390|
|Houston, Texas, United States, 77030|
|United States, Wisconsin|
|Milwaukee, Wisconsin, United States, 53223|
|Study Director:||Program Director, Clinical R&D||CSL Behring|