Safety of a Single Intravenous Dose of Recombinant Factor XIII in Children With Congenital FXIII A-subunit Deficiency (mentor™4)

January 10, 2017 updated by: Novo Nordisk A/S

A Phase 3b Trial Investigating the Pharmacokinetics and Safety Profile of a Single Intravenous Dose of rFXIII in Paediatric (1 to Less Than 6 Years Old) Subjects With Congenital FXIII A-subunit Deficiency

This trial is conducted in Europe and United States of America (USA). The aim of this clinical trial is to investigate the pharmacokinetics (at which rate the substance is distributed and eliminated from the body) and the safety profile of catridecacog (recombinant factor XIII (rFXIII)) in children with congenital FXIII A-subunit deficiency. Young children (1 to less than 6 years old) with congenital FXIII deficiency are evaluated.

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
        • Novo Nordisk Investigational Site
  • United Kingdom
      • Birmingham, United Kingdom, B4 6NH
        • Novo Nordisk Investigational Site
      • Leicester, United Kingdom, LE1 5WW
        • Novo Nordisk Investigational Site
      • Manchester, United Kingdom, M13 9WL
        • Novo Nordisk Investigational Site
      • Newcastle upon Tyne, United Kingdom, NE1 4LP
        • Novo Nordisk Investigational Site
      • Reading, United Kingdom, RG1 5AN
        • Novo Nordisk Investigational Site
  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • 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

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:

  • Signed Informed Consent by subject's parents or subject's legally acceptable representative before any trial related activities. Trial related activities are any procedures that would not have been performed during the normal management of the subject
  • Age 1 to less than 6 years old at the time of enrolment
  • Congenital FXIII subunit-A deficiency previously documented by genotyping or evaluated by genotyping through blood sampling at screening visit
  • Body weight at least 10 kg

Exclusion Criteria:

  • Known antibodies to FXIII
  • Hereditary or acquired coagulation disorder other than FXIII A-subunit congenital deficiency
  • Platelet count (thrombocytes) of less than 50 × 10^9/L (at screening visit)
  • 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
  • Known or suspected allergy to trial product or related products
  • Any surgical procedure in the 30 days prior to enrolment and any planned surgery during the trial period
  • Any disease or condition which, judged by the Investigator, could imply a potential hazard to the subject or 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: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: recombinant factor XIII
Drug: catridecacog
Intravenous injection of a single dose of recombinant factor XIII, 35 IU/kg bodyweight

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Area Under the Concentration vs. Time Curve (AUC)
Time Frame: At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing
A measure of the exposure. Blood samples for the PK assessment were drawn pre-dose and up to 30 days after dosing. The PK of FXIII in children was assessed after a single i.v. dose of rFXIII 35 IU/kg.
At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Area Under the Concentration vs. Time Curve (AUC0-∞)
Time Frame: From day 0 to day 30
A measure of exposure.
From day 0 to day 30
Maximum Plasma Concentration (Cmax) for FXIII
Time Frame: At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing
Maximum plasma concentration of the drug reached.
At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing
Terminal Half-life (t½)
Time Frame: From day 0 to day 30
Time point when half of the maximum plasma concentration is reached.
From day 0 to day 30
Mean Residence Time (MRT)
Time Frame: From day 0 to day 30
The mean residence time (MRT) of a drug in the body and related functions are derived for drugs which are intravenously administered.
From day 0 to day 30
Total Plasma Clearance (CL)
Time Frame: From day 0 to day 30
The total plasma clearance is a measure of the elimination of a drug from the body. Drugs are excreted primarily by the kidneys into the urine. Clearance is calculated as 'CL=Dose / AUC0-30 days').
From day 0 to day 30
Volume of Distribution at Steady State (Vss)
Time Frame: At steady state
Volume of distribution at steady state (Vss) is the theoretical volume that the total amount of administered drug would have to occupy (if it were uniformly distributed), to provide the same concentration as it is in blood plasma at steady state. Steady state is achieved when all variables are constant in spite of ongoing processes.
At steady state
Percentage of Subjects With One or More Adverse Events (AEs) Recorded
Time Frame: From day 0 to day 30
From day 0 to day 30
Percentage of Subjects With One or More Serious Adverse Events (SAEs)
Time Frame: From day 0 to day 30
From day 0 to day 30
Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors (Neutralising Antibodies Against Factor XIII)
Time Frame: At screening and day 30
At screening and day 30
Coagulation Related Parameters - Fibrinogen
Time Frame: Day 0 and at day 30
Day 0 and at day 30
Coagulation Related Parameters - Activated Partial Thromboplastin Time (aPTT, Seconds)
Time Frame: Day 0 and day 30
Day 0 and day 30
Coagulation Related Parameters - Prothrombin Time (PT) (Seconds)
Time Frame: Day 0 and day 30
Day 0 and day 30
Clot Solubility Test (Evaluated as Normal/Abnormal)
Time Frame: Day 0 and day 30
Blood samples for clot solubility drawn at each visit (1 hour before and after dose administration). A clot solubility assay was used to screen for FXIII deficiency. The assay is based on the ability of urea to dissolve fibrin clots that have not undergone FXIII-induced stabilization. Normal blood clots generally remain stable for 24 hours or more, while clots in which fibrin molecules have not been cross-linked are soluble within minutes. The outcome of the test is normal (FXIII present; a clot is observed in the test tube) or abnormal (FXIII absent or very low level; no clot in test tube).
Day 0 and day 30
Vital Signs - Pulse
Time Frame: Day 0 and day 30
Day 0 and day 30
Vital Signs - Blood Pressure (Systolic and Diastolic)
Time Frame: Day 0 and day 30
Day 0 and day 30
Physical Examination (Evaluated as Normal/Abnormal)
Time Frame: From day 0 to day 30
From day 0 to day 30

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

November 1, 2010

Primary Completion (Actual)

January 1, 2012

Study Completion (Actual)

January 1, 2012

Study Registration Dates

First Submitted

October 27, 2010

First Submitted That Met QC Criteria

October 27, 2010

First Posted (Estimate)

October 28, 2010

Study Record Updates

Last Update Posted (Actual)

February 24, 2017

Last Update Submitted That Met QC Criteria

January 10, 2017

Last Verified

January 1, 2017

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • F13CD-3760
  • U1111-1116-2533 (Other Identifier: WHO)
  • 2009-016869-28 (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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