Clinical trial NCT00883090: A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency
Official title: A 12 Week, Multicenter, Pharmacokinetic and Safety Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Factor XIII Deficiency
Keywords: Factor XIII
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2009-04-17
Enrollment: 15
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP

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.


Eligibility criteria: 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

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

Primary outcomes:
measure: Peak FXIII Concentration at Steady State
timeFrame: 12 weeks
measure: Trough FXIII Concentration at Steady State
timeFrame: 12 weeks
measure: Time to Peak Concentration
timeFrame: 12 weeks
measure: Incremental Recovery
timeFrame: 12 weeks
measure: Terminal Half-life
timeFrame: 12 weeks
measure: Area Under the Curve at Steady State
timeFrame: 12 weeks
measure: Clearance
timeFrame: 12 weeks
measure: Volume of Distribution at Steady State
timeFrame: 12 weeks
measure: Mean Residence Time
timeFrame: 12 weeks
Secondary outcomes:
measure: Adverse Events
timeFrame: 16 weeks
measure: Laboratory Safety Parameters
timeFrame: 16 weeks
measure: Vital Signs
timeFrame: 16 weeks
Other outcomes:
Locations:
city: Dothan
country: United States
facility: Study Site
state: Alabama
city: Orange
country: United States
facility: Study Site
state: California
city: San Francisco
country: United States
facility: Study Site
state: California
city: Stockton
country: United States
facility: Study Site
state: California
city: Boston
country: United States
facility: Study Site
state: Massachusetts
city: Santa Cruz de Tenerife
country: Spain
facility: Study Site
Study officials:
affiliation: CSL Behring
name: Program Director, Clinical R&D
role: STUDY_DIRECTOR
References:
citation: Nugent DJ, Ashley C, Garcia-Talavera J, Lo LC, Mehdi AS, Mangione A. Pharmacokinetics and safety of plasma-derived factor XIII concentrate (human) in patients with congenital factor XIII deficiency. Haemophilia. 2015 Jan;21(1):95-101. doi: 10.1111/hae.12505. Epub 2014 Dec 2.