A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency
A 12 Week, Multicenter, Pharmacokinetic and Safety Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
3 other identifiers
interventional
15
2 countries
6
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started May 2009
Shorter than P25 for phase_2
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 16, 2009
CompletedFirst Posted
Study publicly available on registry
April 17, 2009
CompletedStudy Start
First participant enrolled
May 1, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2010
CompletedStudy Completion
Last participant's last visit for all outcomes
April 1, 2010
CompletedResults Posted
Study results publicly available
January 16, 2012
CompletedJanuary 16, 2012
December 1, 2011
11 months
April 16, 2009
December 7, 2011
December 7, 2011
Conditions
Keywords
Outcome Measures
Primary Outcomes (9)
Peak FXIII Concentration at Steady State
12 weeks
Trough FXIII Concentration at Steady State
12 weeks
Time to Peak Concentration
12 weeks
Incremental Recovery
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.
12 weeks
Terminal Half-life
12 weeks
Area Under the Curve at Steady State
12 weeks
Clearance
12 weeks
Volume of Distribution at Steady State
12 weeks
Mean Residence Time
12 weeks
Secondary Outcomes (3)
Adverse Events
16 weeks
Laboratory Safety Parameters
16 weeks
Vital Signs
16 weeks
Study Arms (1)
FXIII
EXPERIMENTALAll subjects treated with Factor XIII Concentrate (Human) (FXIII)
Interventions
Subjects will receive approximately 40 U/kg of FXIII every 28 days for 3 doses administered as a bolus intravenous (IV) injection at approximately 250 U/minute.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- CSL Behringlead
Study Sites (6)
Study Site
Dothan, Alabama, 36305, United States
Study Site
Orange, California, 92868, United States
Study Site
San Francisco, California, 94115, United States
Study Site
Stockton, California, 95204, United States
Study Site
Boston, Massachusetts, 02115, United States
Study Site
Santa Cruz de Tenerife, Spain
Related Publications (1)
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.
PMID: 25458735DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Clinical Trial Disclosure Manager
- Organization
- CSL Behring
Study Officials
- STUDY DIRECTOR
Program Director, Clinical R&D
CSL Behring
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 16, 2009
First Posted
April 17, 2009
Study Start
May 1, 2009
Primary Completion
April 1, 2010
Study Completion
April 1, 2010
Last Updated
January 16, 2012
Results First Posted
January 16, 2012
Record last verified: 2011-12