Skip to main content
OpenTrials
Completed

NCT Number: NCT01253811

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Congenital Bleeding Disorder Congenital FXIII Deficiency

Age range

1 year–6 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Petah Tikva, Israel

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

catridecacog

Drug

Intravenous injection of a single dose of recombinant factor XIII, 35 IU/kg body weight every 4th week

Other names: recombinant factor XIII

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

  8. 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.

  9. 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.

  10. 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.

  11. 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.

  12. 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.

  13. 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.

  14. 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.

  15. 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.

Sponsors and collaborators

Lead sponsor

Novo Nordisk A/S

Industry

Registry information

Official study 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

Acronym: mentor™5

Important dates

Study start
2011
Primary completion
2015
Study completion
2015
First posted
Dec 3, 2010
Registry last updated
Jun 24, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.