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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT01253811
First received: December 1, 2010
Last updated: May 18, 2016
Last verified: May 2016
  Purpose

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.


Condition Intervention Phase
Congenital Bleeding Disorder
Congenital FXIII Deficiency
Drug: catridecacog
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Novo Nordisk A/S:

Primary Outcome Measures:
  • 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. ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors. [ Time Frame: Week 0 to end of trial visit (week 173). ] [ Designated as safety issue: No ]
    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.

  • Clinical Laboratory Assessments: Biochemistry: Creatinine [ Time Frame: Every 6th month, from week 24 to end of trial visit (week 173). ] [ Designated as safety issue: No ]
    Clinical laboratory assessments for creatinine at week 24 to end of trial visit.

  • Clinical Laboratory Assessments: Biochemistry: Urea [ Time Frame: Every 6th month, week 24 to end of trial visit (week 173). ] [ Designated as safety issue: No ]
    Clinical laboratory assessments for urea at week 24 to end ot trial visit.

  • Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT) [ Time Frame: Every 6th month, from week 24 to end of trial visit (week 173). ] [ Designated as safety issue: No ]
    Clinical laboratory assessments for ALAT at week 24 to end of trial visit.

  • Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT) [ Time Frame: Every 6th month, from week 24 to end of trial visit (week 173). ] [ Designated as safety issue: No ]
    Clinical laboratory assessments for ASAT at week 24 to end of trial visit.

  • Clinical Laboratory Assessments: Haematology: Haemoglobin [ Time Frame: Every 6th month, from week 0 to end of trial visit (week 173). ] [ Designated as safety issue: No ]
    Clinical values for haemoglobin collected from week 0 to end of trial visit.

  • Clinical Laboratory Assessments: Haematology: Leucocytes [ Time Frame: Every 6th month, from week 0 to end of trial visit (week 173). ] [ Designated as safety issue: No ]
    Clinical laboratory values for leucocytes collected from week 0 to end of trial visit.

  • Clinical Laboratory Assessments: Haematology: Thrombocytes [ Time Frame: Every 6th month, from week 0 to end of trial visit (week 173). ] [ Designated as safety issue: No ]
    Clinical laboratory values for thrombocytes collected from week 0 to end of trial visit.

  • Clinical Laboratory Assessments: Haematology: Erythrocytes [ Time Frame: Every 6th month, from week 0 to end of trial visit (week 173). ] [ Designated as safety issue: No ]
    Clinical laboratory values for erythrocytes collected from week 0 to end of trial visit.

  • Clinical Laboratory Assessments: Haematology: Haematocrit [ Time Frame: Every 6th month, from week 0 to end of trial visit (week 173). ] [ Designated as safety issue: No ]
    Clinical laboratory values for haematocrit collected from week 0 to end of trial visit.

  • Physical Examinations [ Time Frame: Week 0 to end of trial visit (week 173). ] [ Designated as safety issue: No ]
    Number of subjects in percentage with changes in values of physical examinations from week 0 to end of trial visit were collected.

  • Vital Signs: Systolic BP (Blood Pressure) [ Time Frame: Week 0 to end of trial visit (week 173). ] [ Designated as safety issue: No ]
    Values collected for systolic BP from week 0 to end of trial visit.

  • Vital Signs: Diastolic BP (Blood Pressure) [ Time Frame: Week 0 to end of trial visit (week 173). ] [ Designated as safety issue: No ]
    Values collected for diastolic BP from week 0 to end of trial visit.

  • Vital Signs: Pulse [ Time Frame: Week 0 to end of trial visit (week 173). ] [ Designated as safety issue: No ]
    Values collected for pulse from week 0 to end of trial visit.

  • 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). ] [ Designated as safety issue: No ]
    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.


Enrollment: 6
Study Start Date: January 2011
Study Completion Date: March 2015
Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Name: recombinant factor XIII

  Eligibility

Ages Eligible for Study:   1 Year to 6 Years   (Child)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01253811

Locations
United States, Massachusetts
Novo Nordisk Clinical Trial Call Center
Boston, Massachusetts, United States, 02115
United States, Ohio
Novo Nordisk Clinical Trial Call Center
Columbus, Ohio, United States, 43205
Israel
Petach Tikva, Israel, 49100
United Kingdom
Leicester, United Kingdom, LE1 5WW
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
  More Information

Additional Information:
Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT01253811     History of Changes
Other Study ID Numbers: F13CD-3835  U1111-1117-1063  2010-020192-23 
Study First Received: December 1, 2010
Results First Received: March 29, 2016
Last Updated: May 18, 2016
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Israel: Ministry of Health

Additional relevant MeSH terms:
Blood Coagulation Disorders
Hemostatic Disorders
Hematologic Diseases
Vascular Diseases
Cardiovascular Diseases
Hemorrhagic Disorders

ClinicalTrials.gov processed this record on December 09, 2016