A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency

Phase 2

Completed

• Conditions: Factor XIII Deficiency
• Interventions: Biological: FXIII Concentrate (Human)

• Registration Number: NCT00883090
• Lead Sponsor: CSL Behring

Brief Summary

Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding. This study will evaluate the safety and recommended (best) amount or level of Factor XIII in a patient's blood. Factor XIII Concentrate (Human) is given to people whose blood is lacking Factor XIII. Factor XIII Concentrate (Human) works by assisting your blood in the usual clotting process, thereby preventing bleeding.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2009-04-16
• Study First Submitted QC: 2009-04-16
• Study First Posted: 2009-04-17
• Study Start: 2009-05
• Primary Completion: 2010-04
• Study Completion: 2010-04
• Status Verified: 2011-12
• Results First Submitted: 2011-12-07
• Results First Submitted QC: 2011-12-07
• Last Update Submitted: 2011-12-07
• Results First Posted: 2012-01-16
• Last Update Posted: 2012-01-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

• Written informed consent/assent for study participation obtained before undergoing any study-specific procedures
• Documented congenital FXIII deficiency that 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

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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)
• Positive result at screening for human immunodeficiency virus (HIV)
• Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) concentration >2.5 times the upper limit of normal
• Fibrinogen < lower limit of normal
• Active bleeding
• 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
• Surgical procedure anticipated during the study period
• Suspected inability (e.g., language problems) or unwillingness to comply with study procedures or history of noncompliance

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
FXIII	FXIII Concentrate (Human)	All subjects treated with Factor XIII Concentrate (Human) (FXIII)

Primary Outcome Measures

Name	Time	Method
Peak FXIII Concentration at Steady State	12 weeks
Trough FXIII Concentration at Steady State	12 weeks
Time to Peak Concentration	12 weeks
Volume of Distribution at Steady State	12 weeks
Terminal Half-life	12 weeks
Clearance	12 weeks
Incremental Recovery	12 weeks	Incremental recovery (U/mL/U/kg) is defined as the maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of FXIII (U/kg) administered.
Area Under the Curve at Steady State	12 weeks
Mean Residence Time	12 weeks

Secondary Outcome Measures

Name	Time	Method
Laboratory Safety Parameters	16 weeks	Number of participants with clinically significant laboratory safety parameter values. The laboratory safety parameters measured included serum chemistries, hematology and urinalysis.
Adverse Events	16 weeks	Number of participants with an adverse event
Vital Signs	16 weeks	Number of participants with clinically significant vital signs. The vital signs measured included blood pressure, pulse rate and temperature. Clinically significant changes in vital signs were to be reported as adverse events.

Trial Locations

Locations (1)

Study Site; 🇪🇸 Santa Cruz de Tenerife, Spain