Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency (mentor™5)

May 18, 2016 updated by: Novo Nordisk A/S

A Multi-Centre, Multinational, Open-Label, Single-Arm and Multiple Dosing Trial on Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency. Safety Extension Trial to F13CD-3760

This trial will be conducted in Asia, Europe and the United States of America (USA).

The aim of this clinical trial is to investigate long-term safety of rFXIII when administered for prevention of bleeding episodes in children aged between 1 and 6 years with congenital FXIII A-subunit deficiency. This trial is an extension to trial F13CD-3760 (mentor™4, NCT01230021). If applicable the trial will be extended up to maximum 3 years dependent on when recombinant factor XIII will be commercially available in subject's respective country for use in children of 1-6 years of age.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

6

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

  • Israel
      • Petach Tikva, Israel, 49100
  • United Kingdom
      • Leicester, United Kingdom, LE1 5WW
  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Novo Nordisk Clinical Trial Call Center
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Novo Nordisk Clinical Trial Call Center

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

1 year to 6 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Completed participation in trial F13CD-3760 (NCT01230021)

Exclusion Criteria:

  • Known or suspected hypersensitivity to trial product or related products
  • Known history of development of inhibitors against FXIII (factor XIII)
  • Hereditary or acquired coagulation disorder other than FXIII congenital deficiency
  • Platelet count (thrombocytes) less than 50X10e9 / L
  • Previous history of autoimmune disorder involving autoantibodies e.g., systemic lupus erythematosus
  • Previous history of arterial or venous thromboembolic events e.g., cerebrovascular accident or deep vein thrombosis
  • Any disease or condition which, judged by the trial physician, could imply a potential hazard to the subject, interfere with the trial participation or trial outcome including renal and/or liver dysfunction

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: rFXIII 35 IU/kg
Drug: catridecacog
Intravenous injection of a single dose of recombinant factor XIII, 35 IU/kg body weight every 4th week
Other Names:
  • recombinant factor XIII

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Treatment Emergent (Serious and Non-serious) Adverse Events
Time Frame: Week 0 to end of trial visit (week 173) for a minimum period of 52 weeks.
An adverse event was described as any untoward medical occurrence in a subject or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with this treatment. Treatment emergent adverse events (serious and non-serious), defined as adverse events occurring from first trial product administration to the end of the subject's participation in the trial.
Week 0 to end of trial visit (week 173) for a minimum period of 52 weeks.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors.
Time Frame: Week 0 to end of trial visit (week 173).
All subjects who received rFXIII were monitored for the frequency of development of anti-rFXIII antibodies. 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.
Week 0 to end of trial visit (week 173).
Clinical Laboratory Assessments: Biochemistry: Creatinine
Time Frame: Every 6th month, from week 24 to end of trial visit (week 173).
Clinical laboratory assessments for creatinine at week 24 to end of trial visit.
Every 6th month, from week 24 to end of trial visit (week 173).
Clinical Laboratory Assessments: Biochemistry: Urea
Time Frame: Every 6th month, week 24 to end of trial visit (week 173).
Clinical laboratory assessments for urea at week 24 to end ot trial visit.
Every 6th month, week 24 to end of trial visit (week 173).
Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)
Time Frame: Every 6th month, from week 24 to end of trial visit (week 173).
Clinical laboratory assessments for ALAT at week 24 to end of trial visit.
Every 6th month, from week 24 to end of trial visit (week 173).
Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)
Time Frame: Every 6th month, from week 24 to end of trial visit (week 173).
Clinical laboratory assessments for ASAT at week 24 to end of trial visit.
Every 6th month, from week 24 to end of trial visit (week 173).
Clinical Laboratory Assessments: Haematology: Haemoglobin
Time Frame: Every 6th month, from week 0 to end of trial visit (week 173).
Clinical values for haemoglobin collected from week 0 to end of trial visit.
Every 6th month, from week 0 to end of trial visit (week 173).
Clinical Laboratory Assessments: Haematology: Leucocytes
Time Frame: Every 6th month, from week 0 to end of trial visit (week 173).
Clinical laboratory values for leucocytes collected from week 0 to end of trial visit.
Every 6th month, from week 0 to end of trial visit (week 173).
Clinical Laboratory Assessments: Haematology: Thrombocytes
Time Frame: Every 6th month, from week 0 to end of trial visit (week 173).
Clinical laboratory values for thrombocytes collected from week 0 to end of trial visit.
Every 6th month, from week 0 to end of trial visit (week 173).
Clinical Laboratory Assessments: Haematology: Erythrocytes
Time Frame: Every 6th month, from week 0 to end of trial visit (week 173).
Clinical laboratory values for erythrocytes collected from week 0 to end of trial visit.
Every 6th month, from week 0 to end of trial visit (week 173).
Clinical Laboratory Assessments: Haematology: Haematocrit
Time Frame: Every 6th month, from week 0 to end of trial visit (week 173).
Clinical laboratory values for haematocrit collected from week 0 to end of trial visit.
Every 6th month, from week 0 to end of trial visit (week 173).
Physical Examinations
Time Frame: Week 0 to end of trial visit (week 173).
Number of subjects in percentage with changes in values of physical examinations from week 0 to end of trial visit were collected.
Week 0 to end of trial visit (week 173).
Vital Signs: Systolic BP (Blood Pressure)
Time Frame: Week 0 to end of trial visit (week 173).
Values collected for systolic BP from week 0 to end of trial visit.
Week 0 to end of trial visit (week 173).
Vital Signs: Diastolic BP (Blood Pressure)
Time Frame: Week 0 to end of trial visit (week 173).
Values collected for diastolic BP from week 0 to end of trial visit.
Week 0 to end of trial visit (week 173).
Vital Signs: Pulse
Time Frame: Week 0 to end of trial visit (week 173).
Values collected for pulse from week 0 to end of trial visit.
Week 0 to end of trial visit (week 173).
Rate (Number Per Subject Year) of All Bleeding Episodes Requiring Treatment With a FXIII Containing Product Other Than Recombinant Factor XIII.
Time Frame: Weeks 0 to end of trial visit (week 173).
The rate (number per subject year) of all spontaneous, traumatic and intracranial bleeding episodes requiring treatment with FXIII-containing products during the rFXIII treatment period was assessed for treatment period.
Weeks 0 to end of trial visit (week 173).

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

January 1, 2011

Primary Completion (Actual)

March 1, 2015

Study Completion (Actual)

March 1, 2015

Study Registration Dates

First Submitted

December 1, 2010

First Submitted That Met QC Criteria

December 2, 2010

First Posted (Estimate)

December 3, 2010

Study Record Updates

Last Update Posted (Estimate)

June 24, 2016

Last Update Submitted That Met QC Criteria

May 18, 2016

Last Verified

May 1, 2016

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • F13CD-3835
  • U1111-1117-1063 (Other Identifier: WHO)
  • 2010-020192-23 (EudraCT Number)

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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