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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

41 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-08-31

Study Completion Date

2011-04-30

Brief Summary

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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.

Detailed Description

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Conditions

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Factor XIII Deficiency

Keywords

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Hereditary Factor XIII deficiency Factor XIII

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Blinding Strategy

NONE

Study Groups

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FXIII

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

Group Type EXPERIMENTAL

FXIII Concentrate (Human)

Intervention Type 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.

Interventions

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FXIII Concentrate (Human)

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.

Intervention Type BIOLOGICAL

Other Intervention Names

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Fibrogammin-P

Eligibility Criteria

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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

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Program Director, Clinical R&D

Role: STUDY_DIRECTOR

CSL Behring

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United States Spain

References

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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.

Reference Type DERIVED

PMID: 25377187 (View on PubMed)

Other Identifiers

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1482

Identifier Type: OTHER

Identifier Source: secondary_id

2009-010722-19

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

BI71023_3001

Identifier Type: -

Identifier Source: org_study_id

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

Study Results

Basic Information

Brief Summary

Detailed Description

Conditions

Keywords

Study Design

Study Groups

FXIII

FXIII Concentrate (Human)

Interventions

FXIII Concentrate (Human)

Other Intervention Names

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

CSL Behring

Responsible Party

Principal Investigators

Program Director, Clinical R&D

Locations

Study Site

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Study Site

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Study

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Study Site

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Study Site

Study Site

Countries

References

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.

Related Links

Other Identifiers

1482

2009-010722-19

BI71023_3001