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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01230021
Recruitment Status : Completed
First Posted : October 28, 2010
Results First Posted : June 12, 2014
Last Update Posted : February 24, 2017
Information provided by (Responsible Party):
Novo Nordisk A/S

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Congenital Bleeding Disorder Congenital FXIII Deficiency Drug: catridecacog Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Study Start Date : November 2010
Actual Primary Completion Date : January 2012
Actual Study Completion Date : January 2012

Resource links provided by the National Library of Medicine

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

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. Area Under the Concentration vs. Time Curve (AUC0-∞) [ Time Frame: From day 0 to day 30 ]
    A measure of exposure.

  2. 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.

  3. Terminal Half-life (t½) [ Time Frame: From day 0 to day 30 ]
    Time point when half of the maximum plasma concentration is reached.

  4. 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.

  5. 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').

  6. 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.

  7. Percentage of Subjects With One or More Adverse Events (AEs) Recorded [ Time Frame: From day 0 to day 30 ]
  8. Percentage of Subjects With One or More Serious Adverse Events (SAEs) [ Time Frame: From day 0 to day 30 ]
  9. Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors (Neutralising Antibodies Against Factor XIII) [ Time Frame: At screening and day 30 ]
  10. Coagulation Related Parameters - Fibrinogen [ Time Frame: Day 0 and at day 30 ]
  11. Coagulation Related Parameters - Activated Partial Thromboplastin Time (aPTT, Seconds) [ Time Frame: Day 0 and day 30 ]
  12. Coagulation Related Parameters - Prothrombin Time (PT) (Seconds) [ Time Frame: Day 0 and day 30 ]
  13. 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).

  14. Vital Signs - Pulse [ Time Frame: Day 0 and day 30 ]
  15. Vital Signs - Blood Pressure (Systolic and Diastolic) [ Time Frame: Day 0 and day 30 ]
  16. Physical Examination (Evaluated as Normal/Abnormal) [ Time Frame: From day 0 to day 30 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 6 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01230021

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United States, Massachusetts
Novo Nordisk Investigational Site
Boston, Massachusetts, United States, 02115
United States, Michigan
Novo Nordisk Investigational Site
Detroit, Michigan, United States, 48201
United States, Minnesota
Novo Nordisk Investigational Site
Minneapolis, Minnesota, United States, 55404
United States, Ohio
Novo Nordisk Investigational Site
Columbus, Ohio, United States, 43205
Novo Nordisk Investigational Site
Petach Tikva, Israel, 49100
United Kingdom
Novo Nordisk Investigational Site
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
Sponsors and Collaborators
Novo Nordisk A/S
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Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
Additional Information:
Publications of Results:
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Responsible Party: Novo Nordisk A/S Identifier: NCT01230021    
Other Study ID Numbers: F13CD-3760
U1111-1116-2533 ( Other Identifier: WHO )
2009-016869-28 ( EudraCT Number )
First Posted: October 28, 2010    Key Record Dates
Results First Posted: June 12, 2014
Last Update Posted: February 24, 2017
Last Verified: January 2017
Additional relevant MeSH terms:
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Hemostatic Disorders
Blood Coagulation Disorders
Hematologic Diseases
Vascular Diseases
Cardiovascular Diseases
Hemorrhagic Disorders