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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at below P25 for phase_3

Timeline

Completed

Started Aug 2009

Geographic Reach

2 countries

23 active sites

Status

completed

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

1.7 years study duration

First Submitted

Initial submission to the registry

April 21, 2009

Completed

1 day until next milestone

First Posted

Study publicly available on registry

April 22, 2009

Completed

3 months until next milestone

Study Start

First participant enrolled

August 1, 2009

Completed

1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2011

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2011

Completed

1.3 years until next milestone

Results Posted

Study results publicly available

July 4, 2012

Completed

Last Updated

July 16, 2012

Status Verified

June 1, 2012

Enrollment Period

1.7 years

First QC Date

April 21, 2009

Results QC Date

April 30, 2012

Last Update Submit

July 10, 2012

Conditions

Factor XIII Deficiency

Keywords

Hereditary Factor XIII deficiencyFactor XIII

Outcome Measures

Primary Outcomes (1)

The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event)
The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.
Up to week 52

Secondary Outcomes (7)

Association of the Incidence of Spontaneous Bleeding Events Requiring Treatment and FXIII Activity Trough Levels
12 months
Adverse Events
12 months
Peak FXIII Concentration at Steady State
At 12, 24, 36 and 48 weeks: at 30 and 60 minutes after the end of the infusion.
Trough FXIII Concentration at Steady State
At 12, 24, 36 and 48 weeks: immediately before infusion.
Time to Peak Concentration
At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.
+2 more secondary outcomes

Study Arms (1)

FXIII

EXPERIMENTAL

All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).

Biological: FXIII Concentrate (Human)

Interventions

FXIII Concentrate (Human)BIOLOGICAL

Doses will be guided by the individual subject's most recent FXIII activity levels, with the objective of dosing every 28 days to maintain a trough FXIII activity level of approximately 5 to 20%. Subjects enrolled in this study who did not complete the pharmacokinetic study (Factor XIII Study BI71023\_2002 \[NCT00883090\]) will initially receive FXIII Concentrate (Human) at a dose of 40 U/kg by intravenous (IV) infusion.

Also known as: Fibrogammin-P

FXIII

Eligibility Criteria

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

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)
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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

CSL Behringlead

Study Sites (23)

Study Site

Dothan, Alabama, 36305, United States

Location

Study Site

Oakland, California, 94610, United States

Location

Study Site

Orange, California, 92868, United States

Location

Study Site

San Francisco, California, 94115, United States

Location

Study Site

Stockton, California, 95204, United States

Location

Study Site

Hartford, Connecticut, 06106, United States

Location

Study Site

Fort Meyers, Florida, 33908, United States

Location

Study

Miami, Florida, 33136, United States

Location

Study Site

Boise, Idaho, 83712, United States

Location

Study Site

South Bend, Indiana, 46601, United States

Location

Study Site

Boston, Massachusetts, 02115, United States

Location

Study Site

Saint Paul, Minnesota, 55102, United States

Location

Study Site

Kansas City, Missouri, 64108, United States

Location

Study Site

Las Vegas, Nevada, 89015, United States

Location

Study Site

Lebanon, New Hampshire, 03756, United States

Location

Study Site

Newark, New Jersey, 07102, United States

Location

Study Site

Albany, New York, 12208, United States

Location

Study Site

New York, New York, 10021, United States

Location

Study Site

Chapel Hill, North Carolina, 27599, United States

Location

Study Site

Hershey, Pennsylvania, 17033, United States

Location

Study Site

Dallas, Texas, 75390, United States

Location

Study Site

Milwaukee, Wisconsin, 53233, United States

Location

Study Site

Santa Cruz de Tenerife, 38009, Spain

Location

Related Publications (1)

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.
PMID: 25377187DERIVED

MeSH Terms

Conditions

Factor XIII Deficiency

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

Title: Clinical Trial Disclosure Manager
Organization: CSL Behring

Study Officials

Program Director, Clinical R&D
CSL Behring
STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee: No
Restriction Type: OTHER
Restrictive Agreement: Yes

Study Design

Study Type: interventional
Phase: phase 3
Allocation: NA
Masking: NONE
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

April 21, 2009

First Posted

April 22, 2009

Study Start

August 1, 2009

Primary Completion

April 1, 2011

Study Completion

April 1, 2011

Last Updated

July 16, 2012

Results First Posted

July 4, 2012

Record last verified: 2012-06

Locations

US(22)ES(1)

A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Brief Summary

Trial Health

Trial Health Score

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Results Posted

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (7)

Study Arms (1)

FXIII

Interventions

Eligibility Criteria

You may qualify if:

You may not qualify if:

Sponsors & Collaborators

Study Sites (23)

Related Publications (1)

Related Links

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Results Point of Contact

Study Officials

Publication Agreements

Study Design

Study Record Dates

Locations

Brief Summary

Trial Health

Trial Health Score

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Results Posted

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (7)

Study Arms (1)

FXIII

Interventions

Eligibility Criteria

You may qualify if:

You may not qualify if:

Sponsors & Collaborators

Study Sites (23)

Related Publications (1)

Related Links

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Results Point of Contact

Study Officials

Publication Agreements

Study Design

Study Record Dates

Locations