An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
A Prospective, Multicenter, Open Enrollment Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
2 other identifiers
interventional
61
1 country
25
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Sep 2009
25 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 23, 2009
CompletedFirst Posted
Study publicly available on registry
July 24, 2009
CompletedStudy Start
First participant enrolled
September 1, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2011
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2011
CompletedResults Posted
Study results publicly available
October 12, 2012
CompletedOctober 12, 2012
September 1, 2012
1.9 years
July 23, 2009
September 12, 2012
September 12, 2012
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Adverse Events
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 Outcomes (5)
Hematology and Chemistry Testing
After the first infusion and at the end-of-study (or withdrawal) visit.
FXIII Antibody Testing
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
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
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
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.
Study Arms (1)
FXIII
EXPERIMENTALSubjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.
Interventions
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.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- CSL Behringlead
Study Sites (25)
Study Site
Dothan, Alabama, 36301, United States
Study Site
Oakland, California, 94609, United States
Study Site
Orange, California, 92868, United States
Study Site
San Francisco, California, 94118, United States
Study Site
Stockton, California, 95204, United States
Study Site
Fort Myers, Florida, 33908, United States
Study Site
Miami, Florida, 33136, United States
Study Site
St. Petersburg, Florida, 33701, United States
Study Site
New Orleans, Louisiana, 70121, United States
Study Site
Boston, Massachusetts, 02115, United States
Study Site
Ann Arbor, Michigan, 48109, United States
Study Site
Detroit, Michigan, 48201, United States
Study Site
Kansas City, Missouri, 64108, United States
Study Site
Las Vegas, Nevada, 89109, United States
Study Site
Lebanon, New Hampshire, 03756, United States
Study Site
Albany, New York, 12208, United States
Study Site
New York, New York, 10065, United States
Study Site
Chapel Hill, North Carolina, 27599, United States
Study Site
Columbus, Ohio, 43205, United States
Study Site
Hershey, Pennsylvania, 17033, United States
Study Site
Sioux Falls, South Dakota, 57105, United States
Study Site
Nashville, Tennessee, 37232, United States
Study Site
Dallas, Texas, 75390, United States
Study Site
Houston, Texas, 77030, United States
Study site
Milwaukee, Wisconsin, 53223, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Clinical Trial Disclosure Manager
- Organization
- CSL Behring
Study Officials
- STUDY DIRECTOR
Program Director, Clinical R&D
CSL Behring
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 23, 2009
First Posted
July 24, 2009
Study Start
September 1, 2009
Primary Completion
August 1, 2011
Study Completion
August 1, 2011
Last Updated
October 12, 2012
Results First Posted
October 12, 2012
Record last verified: 2012-09