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Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725 (mentor™2)

Primary Purpose

Congenital Bleeding Disorder, Congenital FXIII Deficiency

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
catridecacog
Sponsored by
Novo Nordisk A/S
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Congenital Bleeding Disorder

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • For subjects who participated in F13CD-1725:
  • Previous participation (means up to and inclusive Visit 16, (End of Trial)) in F13CD-1725
  • For all other subjects:
  • Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit or documented results from previously performed genotyping)
  • Body weight at least 20 kg

Exclusion Criteria:

  • Known neutralizing antibodies (inhibitors) towards FXIII
  • Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
  • Platelet count (thrombocytes) of less than 50 × 109/L. For subjects who participated in F13CD-1725 platelet count from visit 15 in F13CD-1725 must be used for evaluation.
  • Females of childbearing potential who are pregnant, breastfeeding or are not using adequate contraceptive methods

Sites / Locations

  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

A

Arm Description

Outcomes

Primary Outcome Measures

Adverse Events (AEs)(Serious and Non-serious)
An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Trial AEs (serious) included any event such as death, life-threatening experience, in-subject hospitalisation, significant disability/ congential anomaly experienced from the trial product.

Secondary Outcome Measures

Antibody and Inhibitor Development
All subjects who received rFXIII were monitored for anti-rFXIII antibodies and inhibitor development. 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. Percentage of subjects with antibody and inhibitor development were reported.

Full Information

First Posted
September 15, 2009
Last Updated
January 18, 2018
Sponsor
Novo Nordisk A/S
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1. Study Identification

Unique Protocol Identification Number
NCT00978380
Brief Title
Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725
Acronym
mentor™2
Official Title
A Multi-Centre, Open-Label, Single-Arm, and Multiple Dosing Trial on Safety of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Subjects With Congenital Factor XIII Deficiency
Study Type
Interventional

2. Study Status

Record Verification Date
January 2018
Overall Recruitment Status
Completed
Study Start Date
September 21, 2009 (Actual)
Primary Completion Date
October 20, 2015 (Actual)
Study Completion Date
October 20, 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 is conducted in Asia, Europe and North America. The aim of the trial is to investigate the safety of monthly replacement therapy of recombinant factor XIII in patients with congenital FXIII deficiency. The trial continues until the product is commercially available, but an interim assessment will take place when all subjects have completed 52 weeks in the trial.

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
Prevention
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
63 (Actual)

8. Arms, Groups, and Interventions

Arm Title
A
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
catridecacog
Other Intervention Name(s)
recombinant factor XIII
Intervention Description
Monthly administration of recombinant factor XIII as preventative treatment of bleeding episodes. Dose: 35 IU/kg body weight intravenous (into the vein)
Primary Outcome Measure Information:
Title
Adverse Events (AEs)(Serious and Non-serious)
Description
An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Trial AEs (serious) included any event such as death, life-threatening experience, in-subject hospitalisation, significant disability/ congential anomaly experienced from the trial product.
Time Frame
All AEs were collected and reported from screening (week 0) for a minimum period of 52 weeks or until the end of trial visit.
Secondary Outcome Measure Information:
Title
Antibody and Inhibitor Development
Description
All subjects who received rFXIII were monitored for anti-rFXIII antibodies and inhibitor development. 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. Percentage of subjects with antibody and inhibitor development were reported.
Time Frame
From week 0 to week 52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: For subjects who participated in F13CD-1725: Previous participation (means up to and inclusive Visit 16, (End of Trial)) in F13CD-1725 For all other subjects: Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit or documented results from previously performed genotyping) Body weight at least 20 kg Exclusion Criteria: Known neutralizing antibodies (inhibitors) towards FXIII Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency Platelet count (thrombocytes) of less than 50 × 109/L. For subjects who participated in F13CD-1725 platelet count from visit 15 in F13CD-1725 must be used for evaluation. Females of childbearing potential who are pregnant, breastfeeding or are not using adequate contraceptive methods
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 Investigational Site
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Tampa
State/Province
Florida
ZIP/Postal Code
33607
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Boise
State/Province
Idaho
ZIP/Postal Code
83712
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55404
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23219
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Seattle
State/Province
Washington
ZIP/Postal Code
98104
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Klagenfurt
ZIP/Postal Code
A-9020
Country
Austria
Facility Name
Novo Nordisk Investigational Site
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Facility Name
Novo Nordisk Investigational Site
City
Helsinki
ZIP/Postal Code
00290
Country
Finland
Facility Name
Novo Nordisk Investigational Site
City
Le Kremlin Bicetre
ZIP/Postal Code
94270
Country
France
Facility Name
Novo Nordisk Investigational Site
City
Paris
ZIP/Postal Code
75015
Country
France
Facility Name
Novo Nordisk Investigational Site
City
Rouen
ZIP/Postal Code
76031
Country
France
Facility Name
Novo Nordisk Investigational Site
City
Valence Cedex 9
ZIP/Postal Code
26953
Country
France
Facility Name
Novo Nordisk Investigational Site
City
Bonn
ZIP/Postal Code
53127
Country
Germany
Facility Name
Novo Nordisk Investigational Site
City
Braunschweig
ZIP/Postal Code
38118
Country
Germany
Facility Name
Novo Nordisk Investigational Site
City
Duisburg
ZIP/Postal Code
47051
Country
Germany
Facility Name
Novo Nordisk Investigational Site
City
Petach Tikva
ZIP/Postal Code
49100
Country
Israel
Facility Name
Novo Nordisk Investigational Site
City
Vicenza
ZIP/Postal Code
36100
Country
Italy
Facility Name
Novo Nordisk Investigational Site
City
Hiroshima-shi, Hiroshima
ZIP/Postal Code
734 8551
Country
Japan
Facility Name
Novo Nordisk Investigational Site
City
Shinjuku-ku, Tokyo
ZIP/Postal Code
160 0023
Country
Japan
Facility Name
Novo Nordisk Investigational Site
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Novo Nordisk Investigational Site
City
Málaga
ZIP/Postal Code
29011
Country
Spain
Facility Name
Novo Nordisk Investigational Site
City
Tortosa
ZIP/Postal Code
43500
Country
Spain
Facility Name
Novo Nordisk Investigational Site
City
Zürich
ZIP/Postal Code
8091
Country
Switzerland
Facility Name
Novo Nordisk Investigational Site
City
Aberdeen
ZIP/Postal Code
AB25 2ZN
Country
United Kingdom
Facility Name
Novo Nordisk Investigational Site
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
Facility Name
Novo Nordisk Investigational Site
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom
Facility Name
Novo Nordisk Investigational Site
City
Newcastle upon Tyne
ZIP/Postal Code
NE1 4LP
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
25643920
Citation
Brand-Staufer B, Carcao M, Kerlin BA, Will A, Williams M, Tornoe CW, Sandberg Lundblad M, Nugent D. Pharmacokinetic characterization of recombinant factor XIII (FXIII)-A2 across age groups in patients with FXIII A-subunit congenital deficiency. Haemophilia. 2015 May;21(3):380-385. doi: 10.1111/hae.12616. Epub 2015 Jan 21.
Results Reference
result
PubMed Identifier
25263390
Citation
Kerlin B, Brand B, Inbal A, Halimeh S, Nugent D, Lundblad M, Tehranchi R. Pharmacokinetics of recombinant factor XIII at steady state in patients with congenital factor XIII A-subunit deficiency. J Thromb Haemost. 2014 Dec;12(12):2038-43. doi: 10.1111/jth.12739. Epub 2014 Oct 25.
Results Reference
result
Links:
URL
http://novonordisk-trials.com
Description
Clinical Trials at Novo Nordisk

Learn more about this trial

Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725

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