Clinical trial NCT00945906: An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Official title: A Prospective, Multicenter, Open Enrollment 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: 2009-07-24
Enrollment: 61
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 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.


Eligibility criteria: 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

Primary outcomes:
measure: Adverse Events
timeFrame: 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.
Secondary outcomes:
measure: Hematology and Chemistry Testing
timeFrame: After the first infusion and at the end-of-study (or withdrawal) visit.
measure: FXIII Antibody Testing
timeFrame: 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.
measure: FXIII Concentration
timeFrame: Before the first infusion, at 24 and 48 weeks after the first infusion, and at the end-of-study (or withdrawal) visit.
measure: Number of Subjects With at Least One Bleeding Episode
timeFrame: 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.
measure: Number of Bleeding Episodes
timeFrame: 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.
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: Fort Myers
country: United States
facility: Study Site
state: Florida
city: Miami
country: United States
facility: Study Site
state: Florida
city: St. Petersburg
country: United States
facility: Study Site
state: Florida
city: New Orleans
country: United States
facility: Study Site
state: Louisiana
city: Boston
country: United States
facility: Study Site
state: Massachusetts
city: Ann Arbor
country: United States
facility: Study Site
state: Michigan
city: Detroit
country: United States
facility: Study Site
state: Michigan
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: 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: Columbus
country: United States
facility: Study Site
state: Ohio
city: Hershey
country: United States
facility: Study Site
state: Pennsylvania
city: Sioux Falls
country: United States
facility: Study Site
state: South Dakota
city: Nashville
country: United States
facility: Study Site
state: Tennessee
city: Dallas
country: United States
facility: Study Site
state: Texas
city: Houston
country: United States
facility: Study Site
state: Texas
city: Milwaukee
country: United States
facility: Study site
state: Wisconsin
Study officials:
affiliation: CSL Behring
name: Program Director, Clinical R&D
role: STUDY_DIRECTOR
References: