Back to resultsSafety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency (mentor™5)
Primary Purpose
Congenital Bleeding Disorder, Congenital FXIII Deficiency
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
catridecacog
Sponsored by
About this trial
This is an interventional treatment trial for Congenital Bleeding Disorder
Eligibility 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
Sites / Locations
- Novo Nordisk Clinical Trial Call Center
- Novo Nordisk Clinical Trial Call Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
rFXIII 35 IU/kg
Arm Description
Outcomes
Primary Outcome Measures
Number of Treatment Emergent (Serious and Non-serious) Adverse Events
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.
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
Clinical laboratory assessments for creatinine at week 24 to end of trial visit.
Clinical Laboratory Assessments: Biochemistry: Urea
Clinical laboratory assessments for urea at week 24 to end ot trial visit.
Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)
Clinical laboratory assessments for ALAT at week 24 to end of trial visit.
Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)
Clinical laboratory assessments for ASAT at week 24 to end of trial visit.
Clinical Laboratory Assessments: Haematology: Haemoglobin
Clinical values for haemoglobin collected from week 0 to end of trial visit.
Clinical Laboratory Assessments: Haematology: Leucocytes
Clinical laboratory values for leucocytes collected from week 0 to end of trial visit.
Clinical Laboratory Assessments: Haematology: Thrombocytes
Clinical laboratory values for thrombocytes collected from week 0 to end of trial visit.
Clinical Laboratory Assessments: Haematology: Erythrocytes
Clinical laboratory values for erythrocytes collected from week 0 to end of trial visit.
Clinical Laboratory Assessments: Haematology: Haematocrit
Clinical laboratory values for haematocrit collected from week 0 to end of trial visit.
Physical Examinations
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)
Values collected for systolic BP from week 0 to end of trial visit.
Vital Signs: Diastolic BP (Blood Pressure)
Values collected for diastolic BP from week 0 to end of trial visit.
Vital Signs: Pulse
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.
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01253811
Brief Title
Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency
Acronym
mentor™5
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
Study Type
Interventional
2. Study Status
Record Verification Date
May 2016
Overall Recruitment Status
Completed
Study Start Date
January 2011 (undefined)
Primary Completion Date
March 2015 (Actual)
Study Completion Date
March 2015 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novo Nordisk A/S
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Bleeding Disorder, Congenital FXIII Deficiency
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)
8. Arms, Groups, and Interventions
Arm Title
rFXIII 35 IU/kg
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
catridecacog
Other Intervention Name(s)
recombinant factor XIII
Intervention Description
Intravenous injection of a single dose of recombinant factor XIII, 35 IU/kg body weight every 4th week
Primary Outcome Measure Information:
Title
Number of Treatment Emergent (Serious and Non-serious) Adverse Events
Description
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.
Time Frame
Week 0 to end of trial visit (week 173) for a minimum period of 52 weeks.
Secondary Outcome Measure Information:
Title
Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors.
Description
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.
Time Frame
Week 0 to end of trial visit (week 173).
Title
Clinical Laboratory Assessments: Biochemistry: Creatinine
Description
Clinical laboratory assessments for creatinine at week 24 to end of trial visit.
Time Frame
Every 6th month, from week 24 to end of trial visit (week 173).
Title
Clinical Laboratory Assessments: Biochemistry: Urea
Description
Clinical laboratory assessments for urea at week 24 to end ot trial visit.
Time Frame
Every 6th month, week 24 to end of trial visit (week 173).
Title
Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)
Description
Clinical laboratory assessments for ALAT at week 24 to end of trial visit.
Time Frame
Every 6th month, from week 24 to end of trial visit (week 173).
Title
Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)
Description
Clinical laboratory assessments for ASAT at week 24 to end of trial visit.
Time Frame
Every 6th month, from week 24 to end of trial visit (week 173).
Title
Clinical Laboratory Assessments: Haematology: Haemoglobin
Description
Clinical values for haemoglobin collected from week 0 to end of trial visit.
Time Frame
Every 6th month, from week 0 to end of trial visit (week 173).
Title
Clinical Laboratory Assessments: Haematology: Leucocytes
Description
Clinical laboratory values for leucocytes collected from week 0 to end of trial visit.
Time Frame
Every 6th month, from week 0 to end of trial visit (week 173).
Title
Clinical Laboratory Assessments: Haematology: Thrombocytes
Description
Clinical laboratory values for thrombocytes collected from week 0 to end of trial visit.
Time Frame
Every 6th month, from week 0 to end of trial visit (week 173).
Title
Clinical Laboratory Assessments: Haematology: Erythrocytes
Description
Clinical laboratory values for erythrocytes collected from week 0 to end of trial visit.
Time Frame
Every 6th month, from week 0 to end of trial visit (week 173).
Title
Clinical Laboratory Assessments: Haematology: Haematocrit
Description
Clinical laboratory values for haematocrit collected from week 0 to end of trial visit.
Time Frame
Every 6th month, from week 0 to end of trial visit (week 173).
Title
Physical Examinations
Description
Number of subjects in percentage with changes in values of physical examinations from week 0 to end of trial visit were collected.
Time Frame
Week 0 to end of trial visit (week 173).
Title
Vital Signs: Systolic BP (Blood Pressure)
Description
Values collected for systolic BP from week 0 to end of trial visit.
Time Frame
Week 0 to end of trial visit (week 173).
Title
Vital Signs: Diastolic BP (Blood Pressure)
Description
Values collected for diastolic BP from week 0 to end of trial visit.
Time Frame
Week 0 to end of trial visit (week 173).
Title
Vital Signs: Pulse
Description
Values collected for pulse from week 0 to end of trial visit.
Time Frame
Week 0 to end of trial visit (week 173).
Title
Rate (Number Per Subject Year) of All Bleeding Episodes Requiring Treatment With a FXIII Containing Product Other Than Recombinant Factor XIII.
Description
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.
Time Frame
Weeks 0 to end of trial visit (week 173).
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Global Clinical Registry (GCR, 1452)
Organizational Affiliation
Novo Nordisk A/S
Official's Role
Study Director
Facility Information:
Facility Name
Novo Nordisk Clinical Trial Call Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Novo Nordisk Clinical Trial Call Center
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
City
Petach Tikva
ZIP/Postal Code
49100
Country
Israel
City
Leicester
ZIP/Postal Code
LE1 5WW
Country
United Kingdom
12. IPD Sharing Statement
Citations:
PubMed Identifier
28581691
Citation
Kerlin BA, Inbal A, Will A, Williams M, Garly ML, Jacobsen L, Kearney SL. Recombinant factor XIII prophylaxis is safe and effective in young children with congenital factor XIII-A deficiency: international phase 3b trial results. J Thromb Haemost. 2017 Aug;15(8):1601-1606. doi: 10.1111/jth.13748. Epub 2017 Jul 10.
Results Reference
derived
Links:
URL
http://novonordisk-trials.com
Description
Clinical Trials at Novo Nordisk
Learn more about this trial
Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency
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