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 is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT01253811
First received: December 1, 2010
Last updated: March 17, 2014
Last verified: March 2014
  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: recombinant factor XIII
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
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: every 4th week, from 0 to week 56 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Frequency of development of anti-rFXIII antibodies, including inhibitors [ Time Frame: every 4th week, from week 0 to week 56 ] [ Designated as safety issue: No ]
  • Clinical laboratory assessments: Biochemistry, haematology [ Time Frame: every 6th month, from 0 to week 56 ] [ Designated as safety issue: No ]
  • Physical examinations [ Time Frame: every 4th week, from 0 to week 52 ] [ Designated as safety issue: No ]
  • Vital signs (Blood Pressure) [ Time Frame: every 4th week, from week 0 to week 56 ] [ Designated as safety issue: No ]
  • Vital signs (Pulse) [ Time Frame: every 4th week, from week 0 to week 56 ] [ Designated as safety issue: No ]
  • Rate (number per subject year) of all bleeding episodes requiring treatment with a FXIII containing product other than recombinant factor XIII [ Time Frame: weeks 0-52 ] [ Designated as safety issue: No ]

Estimated Enrollment: 6
Study Start Date: January 2011
Estimated Study Completion Date: June 2015
Estimated Primary Completion Date: June 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: recombinant factor XIII Drug: recombinant factor XIII
Intravenous injection of a single dose of recombinant factor XIII, 35 IU/kg body weight every 4th week

  Eligibility

Ages Eligible for Study:   1 Year to 6 Years
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01253811

Locations
United States, Minnesota
Novo Nordisk Clinical Trial Call Center
Minneapolis, Minnesota, United States, 55404
United States, Ohio
Novo Nordisk Clinical Trial Call Center
Columbus, Ohio, United States, 43205
Israel
Petach Tikva, Israel, 49100
United Kingdom
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
Study Director: Gita Ohlsson Novo Nordisk A/S
  More Information

Additional Information:
No publications provided

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
Last Updated: March 17, 2014
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Regulatory Authority (MHRA)
Israel: Ministry of Health

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

ClinicalTrials.gov processed this record on April 17, 2014