Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725 (mentor™2)

January 18, 2018 updated by: Novo Nordisk A/S

A Multi-Centre, Open-Label, Single-Arm, and Multiple Dosing Trial on Safety of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Subjects With Congenital Factor XIII Deficiency

This trial is conducted in Asia, Europe and North America. The aim of the trial is to investigate the safety of monthly replacement therapy of recombinant factor XIII in patients with congenital FXIII deficiency. The trial continues until the product is commercially available, but an interim assessment will take place when all subjects have completed 52 weeks in the trial.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
63

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Klagenfurt, Austria, A-9020
        • Novo Nordisk Investigational Site
  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • Novo Nordisk Investigational Site
  • Finland
      • Helsinki, Finland, 00290
        • Novo Nordisk Investigational Site
  • France
      • Le Kremlin Bicetre, France, 94270
        • Novo Nordisk Investigational Site
      • Paris, France, 75015
        • Novo Nordisk Investigational Site
      • Rouen, France, 76031
        • Novo Nordisk Investigational Site
      • Valence Cedex 9, France, 26953
        • Novo Nordisk Investigational Site
  • Germany
      • Bonn, Germany, 53127
        • Novo Nordisk Investigational Site
      • Braunschweig, Germany, 38118
        • Novo Nordisk Investigational Site
      • Duisburg, Germany, 47051
        • Novo Nordisk Investigational Site
  • Israel
      • Petach Tikva, Israel, 49100
        • Novo Nordisk Investigational Site
  • Italy
      • Vicenza, Italy, 36100
        • Novo Nordisk Investigational Site
  • Japan
      • Hiroshima-shi, Hiroshima, Japan, 734 8551
        • Novo Nordisk Investigational Site
      • Shinjuku-ku, Tokyo, Japan, 160 0023
        • Novo Nordisk Investigational Site
  • Spain
      • Barcelona, Spain, 08035
        • Novo Nordisk Investigational Site
      • Málaga, Spain, 29011
        • Novo Nordisk Investigational Site
      • Tortosa, Spain, 43500
        • Novo Nordisk Investigational Site
  • Switzerland
      • Zürich, Switzerland, 8091
        • Novo Nordisk Investigational Site
  • United Kingdom
      • Aberdeen, United Kingdom, AB25 2ZN
        • Novo Nordisk Investigational Site
      • London, United Kingdom, WC1N 3JH
        • Novo Nordisk Investigational Site
      • Manchester, United Kingdom, M13 9WL
        • Novo Nordisk Investigational Site
      • Newcastle upon Tyne, United Kingdom, NE1 4LP
        • Novo Nordisk Investigational Site
  • United States
    • California
      • Orange, California, United States, 92868
        • Novo Nordisk Investigational Site
    • District of Columbia
      • Washington, District of Columbia, United States, 20007
        • Novo Nordisk Investigational Site
    • Florida
      • Tampa, Florida, United States, 33607
        • Novo Nordisk Investigational Site
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Novo Nordisk Investigational Site
    • Idaho
      • Boise, Idaho, United States, 83712
        • Novo Nordisk Investigational Site
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Novo Nordisk Investigational Site
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Novo Nordisk Investigational Site
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Novo Nordisk Investigational Site
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Novo Nordisk Investigational Site
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Novo Nordisk Investigational Site
    • Virginia
      • Richmond, Virginia, United States, 23219
        • Novo Nordisk Investigational Site
    • Washington
      • Seattle, Washington, United States, 98104
        • Novo Nordisk Investigational Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

6 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • For subjects who participated in F13CD-1725:
  • Previous participation (means up to and inclusive Visit 16, (End of Trial)) in F13CD-1725
  • For all other subjects:
  • Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit or documented results from previously performed genotyping)
  • Body weight at least 20 kg

Exclusion Criteria:

  • Known neutralizing antibodies (inhibitors) towards FXIII
  • Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
  • Platelet count (thrombocytes) of less than 50 × 109/L. For subjects who participated in F13CD-1725 platelet count from visit 15 in F13CD-1725 must be used for evaluation.
  • Females of childbearing potential who are pregnant, breastfeeding or are not using adequate contraceptive methods

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: A
Drug: catridecacog
Monthly administration of recombinant factor XIII as preventative treatment of bleeding episodes. Dose: 35 IU/kg body weight intravenous (into the vein)
Other Names:
  • recombinant factor XIII

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Adverse Events (AEs)(Serious and Non-serious)
Time Frame: All AEs were collected and reported from screening (week 0) for a minimum period of 52 weeks or until the end of trial visit.
An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Trial AEs (serious) included any event such as death, life-threatening experience, in-subject hospitalisation, significant disability/ congential anomaly experienced from the trial product.
All AEs were collected and reported from screening (week 0) for a minimum period of 52 weeks or until the end of trial visit.

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Antibody and Inhibitor Development
Time Frame: From week 0 to week 52
All subjects who received rFXIII were monitored for anti-rFXIII antibodies and inhibitor development. Samples passed through 2 tiers of ELISA testing: an initial screen with a specific cut-off point (including ~5% false positives) and a second confirmatory assay for samples yielding a result above the screening cut-off point. If samples were confirmed as antibody positive in the confirmation assay, an inhibitor assay was also carried out to detect functional inhibitors. Percentage of subjects with antibody and inhibitor development were reported.
From week 0 to week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novo Nordisk A/S

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 21, 2009

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 20, 2015

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 20, 2015

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 15, 2009

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 15, 2009

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 16, 2009

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 24, 2018

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 18, 2018

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • F13CD-3720
  • 2008-007883-41 (EudraCT Number)
  • U1111-1111-9289 (Other Identifier: WHO)
  • JapicCTI-121958 (Registry Identifier: JAPIC)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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