An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

A Prospective, Multicenter, Open Enrollment Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00945906

Enrollment

Registered

2009-07-24

Start date

2009-09-30

Completion date

2011-08-31

Last updated

2012-10-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Factor XIII Deficiency

Keywords

Hereditary Factor XIII deficiency, Factor XIII

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.

Interventions

BIOLOGICALFXIII Concentrate (Human) (FXIII)

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 have not received at least 3 doses of FXIII Concentrate in a previous study of this product (i.e., NCT00640289, NCT00885742, or NCT00883090) will initially receive a dose of 40 U/kg by intravenous (IV) infusion.

Study design

Allocation

Intervention model

SINGLE_GROUP

Masking

NONE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
Adverse Events	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.	Number of subjects with any treatment-emergent adverse event (AE), treatment-related AE or serious AE (SAE). Treatment-related AEs are defined as AEs whose relationship to treatment is related, or possibly related and AEs with missing relationship.

Secondary

Measure	Time frame	Description
Hematology and Chemistry Testing	After the first infusion and at the end-of-study (or withdrawal) visit.	Number of participants with treatment-emergent clinically significant hematology and/or chemistry laboratory parameter values.
FXIII Antibody Testing	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.	Number of participants with serum Factor XIII antibodies.
FXIII Concentration	Before the first infusion, at 24 and 48 weeks after the first infusion, and at the end-of-study (or withdrawal) visit.	Trough Factor XIII concentration.
Number of Subjects With at Least One Bleeding Episode	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.	Number of subjects with at least one bleeding episode at any time after the first infusion in the study, and the number of subjects with at least one bleeding episode requiring Factor XIII treatment.
Number of Bleeding Episodes	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.	Number of bleeding episodes at any time after the first infusion in the study.

Countries

United States

Participant flow

Participants by arm

Arm	Count
FXIII Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.	61
Total	61

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Did not want to return for last visit	2
Overall Study	Moved to another country	2
Overall Study	Unable to return for last visit	2
Overall Study	Withdrawal by Subject	1

Baseline characteristics

Characteristic	FXIII
Age Continuous	18.5 years STANDARD_DEVIATION 12.48
Age, Customized 16 to < 65 years	32 participants
Age, Customized < 16 years	29 participants
Age, Customized >=65 years	0 participants
Sex: Female, Male Female	26 Participants
Sex: Female, Male Male	35 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	— / —
other Total, other adverse events	19 / 61
serious Total, serious adverse events	2 / 61

Outcome results

Primary

Adverse Events

Number of subjects with any treatment-emergent adverse event (AE), treatment-related AE or serious AE (SAE). Treatment-related AEs are defined as AEs whose relationship to treatment is related, or possibly related and AEs with missing relationship.

Time frame: 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.

Population: The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study. Treatment related AEs are events whose relationship to study treatment is related, or possibly related, in the opinion of the investigator. AEs with missing relationship are considered related to treatment.

Arm	Measure	Group	Value (NUMBER)
FXIII	Adverse Events	Any treatment-emergent AE	42 participants
FXIII	Adverse Events	Treatment-emergent and related AE	2 participants
FXIII	Adverse Events	Serious AE	2 participants

Secondary

FXIII Antibody Testing

Number of participants with serum Factor XIII antibodies.

Time frame: 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.