Clinical trial NCT00885742: A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Official title: A Prospective, Multicenter, Open-label, Phase 3b Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Factor XIII Deficiency
Keywords: Hereditary Factor XIII deficiency
Factor XIII
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2009-04-22
Enrollment: 41
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 (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.


Eligibility criteria: 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

Primary outcomes:
measure: The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event)
timeFrame: Up to week 52
Secondary outcomes:
measure: Association of the Incidence of Spontaneous Bleeding Events Requiring Treatment and FXIII Activity Trough Levels
timeFrame: 12 months
measure: Adverse Events
timeFrame: 12 months
measure: Peak FXIII Concentration at Steady State
timeFrame: At 12, 24, 36 and 48 weeks: at 30 and 60 minutes after the end of the infusion.
measure: Trough FXIII Concentration at Steady State
timeFrame: At 12, 24, 36 and 48 weeks: immediately before infusion.
measure: Time to Peak Concentration
timeFrame: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.
measure: Incremental Recovery
timeFrame: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.
measure: Achievement of Trough Factor XIII Levels of 5% or Higher.
timeFrame: At 12, 24, 36 and 48 weeks: immediately before infusion.
Other outcomes:
Locations:
city: Dothan
country: United States
facility: Study Site
state: Alabama
city: Oakland
country: United States
facility: Study Site
state: California
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: Hartford
country: United States
facility: Study Site
state: Connecticut
city: Fort Meyers
country: United States
facility: Study Site
state: Florida
city: Miami
country: United States
facility: Study
state: Florida
city: Boise
country: United States
facility: Study Site
state: Idaho
city: South Bend
country: United States
facility: Study Site
state: Indiana
city: Boston
country: United States
facility: Study Site
state: Massachusetts
city: St. Paul
country: United States
facility: Study Site
state: Minnesota
city: Kansas City
country: United States
facility: Study Site
state: Missouri
city: Las Vegas
country: United States
facility: Study Site
state: Nevada
city: Lebanon
country: United States
facility: Study Site
state: New Hampshire
city: Newark
country: United States
facility: Study Site
state: New Jersey
city: Albany
country: United States
facility: Study Site
state: New York
city: New York
country: United States
facility: Study Site
state: New York
city: Chapel Hill
country: United States
facility: Study Site
state: North Carolina
city: Hershey
country: United States
facility: Study Site
state: Pennsylvania
city: Dallas
country: United States
facility: Study Site
state: Texas
city: Milwaukee
country: United States
facility: Study Site
state: Wisconsin
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: Ashley C, Chang E, Davis J, Mangione A, Frame V, Nugent DJ. Efficacy and safety of prophylactic treatment with plasma-derived factor XIII concentrate (human) in patients with congenital factor XIII deficiency. Haemophilia. 2015 Jan;21(1):102-8. doi: 10.1111/hae.12524. Epub 2014 Nov 7.