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

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

63

Phase

  • 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

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
Intervention / Treatment
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

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

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

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)

September 21, 2009

Primary Completion (Actual)

October 20, 2015

Study Completion (Actual)

October 20, 2015

Study Registration Dates

First Submitted

September 15, 2009

First Submitted That Met QC Criteria

September 15, 2009

First Posted (Estimate)

September 16, 2009

Study Record Updates

Last Update Posted (Actual)

January 24, 2018

Last Update Submitted That Met QC Criteria

January 18, 2018

Last Verified

January 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID 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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