A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Factor XIII Deficiency Clinical Trial

Official title:

A Prospective, Multicenter, Open-label, Phase 3b Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00885742
• Other study ID #: BI71023_3001
• Secondary ID: 14822009-010722-
• Status: Completed
• Phase: Phase 3
• First received: April 21, 2009
• Last updated: July 10, 2012
• Start date: August 2009
• Est. completion date: April 2011

Study information

• Verified date: June 2012
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationCanada: Health CanadaSpain: Spanish Agency of Medicines
• Study type: Interventional

Clinical Trial Summary

Congenital deficiency of factor XIII (FXIII) 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 that will best minimize the chance of bruising and bleeding.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 41
• Est. completion date: April 2011
• Est. primary completion date: April 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

• Documented congenital FXIII deficiency which requires prophylactic treatment with a FXIII containing product.

• Males and females of any age with congenital FXIII deficiency

• Received full hepatitis B vaccination and/or is hepatitis B surface antibody positive

Exclusion Criteria:

• Diagnosis of acquired FXIII deficiency

• Administration of a FXIII-containing product, including blood transfusions or other blood products within 4 weeks prior to the planned Day 0

• Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency

• Known or suspected to have antibodies towards FXIII

• Use of any other investigational medicinal product within 4 weeks prior to the Baseline Visit (Day 0)

• Known Positivity for human immunodeficiency virus (HIV) or a positive result for HIV at the Screening Visit of this study or the FXIII study 2002 (NCT00883090).

• Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) concentration >2.5 times the upper limit of normal at the Screening Visit of this study or at the Day 56 Visit of Factor XIII Study BI71023_2002 (NCT00883090)

• Fibrinogen level less than 85% of the lower limit of normal at the Screening Visit of this study or the Factor XIII Study BI71023_2002 (NCT00883090)

• Active bleeding = Common Terminology Criteria for Adverse Events (CTCAE) Grade 2 and/or = moderate between the Screening and Baseline Visits

• Pregnant or breast-feeding

• Intention to become pregnant during the course of the study

• 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

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)
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 did not complete the pharmacokinetic study (Factor XIII Study BI71023_2002 [NCT00883090]) will initially receive FXIII Concentrate (Human) at a dose of 40 U/kg by intravenous (IV) infusion.

Locations

Country	Name	City	State
Spain	Study Site	Santa Cruz de Tenerife
United States	Study Site	Albany	New York
United States	Study Site	Boise	Idaho
United States	Study Site	Boston	Massachusetts
United States	Study Site	Chapel Hill	North Carolina
United States	Study Site	Dallas	Texas
United States	Study Site	Dothan	Alabama
United States	Study Site	Fort Meyers	Florida
United States	Study Site	Hartford	Connecticut
United States	Study Site	Hershey	Pennsylvania
United States	Study Site	Kansas City	Missouri
United States	Study Site	Las Vegas	Nevada
United States	Study Site	Lebanon	New Hampshire
United States	Study	Miami	Florida
United States	Study Site	Milwaukee	Wisconsin
United States	Study Site	New York	New York
United States	Study Site	Newark	New Jersey
United States	Study Site	Oakland	California
United States	Study Site	Orange	California
United States	Study Site	San Francisco	California
United States	Study Site	South Bend	Indiana
United States	Study Site	St. Paul	Minnesota
United States	Study Site	Stockton	California

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Countries where clinical trial is conducted

United States,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event)	The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.	Up to week 52	No
Secondary	Association of the Incidence of Spontaneous Bleeding Events Requiring Treatment and FXIII Activity Trough Levels	P-value determined from Generalized Estimating Equation (GEE) model parameter estimates with bleeding as the response variable and FXIII activity trough level as the explanatory variable.	12 months	No
Secondary	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 study treatment is related, or possibly related, and AEs with missing relationship.	12 months	Yes
Secondary	Peak FXIII Concentration at Steady State		At 12, 24, 36 and 48 weeks: at 30 and 60 minutes after the end of the infusion.	No
Secondary	Trough FXIII Concentration at Steady State		At 12, 24, 36 and 48 weeks: immediately before infusion.	No
Secondary	Time to Peak Concentration		At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.	No
Secondary	Incremental Recovery	Incremental recovery (U/mL/U/kg) is defined as maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of (U/kg) infusion.	At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.	No
Secondary	Achievement of Trough Factor XIII Levels of 5% or Higher.	Number of subjects with Factor XIII level = 5% before infusion at Week 12, Week 24, Week 36 and Week 48.	At 12, 24, 36 and 48 weeks: immediately before infusion.	No

External Links

•   Click here to request more information about this study
•   Factor XIII Study BI71023_2002 (NCT00883090)
•   Factor XIII Study BI71023_3002 (NCT00945906)