An Open Enrollment 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.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

61 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-09-30

Study Completion Date

2011-08-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Factor XIII Deficiency

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Hereditary Factor XIII deficiency Factor XIII

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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%.

Group Type EXPERIMENTAL

FXIII Concentrate (Human) (FXIII)

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 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.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

FXIII 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.

Intervention Type BIOLOGICAL

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Fibrogammin-P® Corifact®

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Program Director, Clinical R&D

Role: STUDY_DIRECTOR

CSL Behring

Review additional registry numbers or institutional identifiers associated with this trial.

1488

Identifier Type: OTHER

Identifier Source: secondary_id

BI71023_3002

Identifier Type: -

Identifier Source: org_study_id

An Open Enrollment 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) (FXIII)

Interventions

FXIII Concentrate (Human) (FXIII)

Other Intervention Names

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

CSL Behring

Responsible Party

Principal Investigators

Program Director, Clinical R&D

Locations

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study Site

Study site

Countries

Other Identifiers

1488

BI71023_3002