An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Factor XIII Deficiency Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00945906
• Other study ID #: BI71023_3002
• Secondary ID: 1488
• Status: Completed
• Phase: Phase 3
• First received: July 23, 2009
• Last updated: September 12, 2012
• Start date: September 2009
• Est. completion date: August 2011

Study information

• Verified date: September 2012
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 61
• Est. completion date: August 2011
• Est. primary completion date: August 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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 Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label

Related Conditions & MeSH terms

• Factor XIII Deficiency

Intervention

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.

Locations

Country	Name	City	State
United States	Study Site	Albany	New York
United States	Study Site	Ann Arbor	Michigan
United States	Study Site	Boston	Massachusetts
United States	Study Site	Chapel Hill	North Carolina
United States	Study Site	Columbus	Ohio
United States	Study Site	Dallas	Texas
United States	Study Site	Detroit	Michigan
United States	Study Site	Dothan	Alabama
United States	Study Site	Fort Myers	Florida
United States	Study Site	Hershey	Pennsylvania
United States	Study Site	Houston	Texas
United States	Study Site	Kansas City	Missouri
United States	Study Site	Las Vegas	Nevada
United States	Study Site	Lebanon	New Hampshire
United States	Study Site	Miami	Florida
United States	Study site	Milwaukee	Wisconsin
United States	Study Site	Nashville	Tennessee
United States	Study Site	New Orleans	Louisiana
United States	Study Site	New York	New York
United States	Study Site	Oakland	California
United States	Study Site	Orange	California
United States	Study Site	San Francisco	California
United States	Study Site	Sioux Falls	South Dakota
United States	Study Site	St. Petersburg	Florida
United States	Study Site	Stockton	California

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Yes
Secondary	Hematology and Chemistry Testing	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.	Yes
Secondary	FXIII Antibody Testing	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.	Yes
Secondary	FXIII Concentration	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.	Yes
Secondary	Number of Subjects With at Least One Bleeding Episode	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.	Yes
Secondary	Number of Bleeding Episodes	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.	Yes

External Links

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