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

Congenital Bleeding Disorder Clinical Trial

— 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01253811
• Other study ID #: F13CD-3835
• Secondary ID: U1111-1117-10632
• Status: Completed
• Phase: Phase 3
• First received: December 1, 2010
• Last updated: April 7, 2015
• Start date: January 2011
• Est. completion date: March 2015

Study information

• Verified date: April 2015
• Source: Novo Nordisk A/S
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyIsrael: Ministry of Health
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 6
• Est. completion date: March 2015
• Est. primary completion date: March 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year to 6 Years

Eligibility

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Blood Coagulation Disorders
• Congenital Bleeding Disorder
• Congenital FXIII Deficiency
• Hemostatic Disorders

Intervention

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

Locations

Country	Name	City	State
United States	Novo Nordisk Clinical Trial Call Center	Columbus	Ohio
United States	Novo Nordisk Clinical Trial Call Center	Minneapolis	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Countries where clinical trial is conducted

United States,  Israel,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of treatment emergent (serious and non-serious) adverse events		every 4th week, from 0 to week 56	No
Secondary	Frequency of development of anti-rFXIII antibodies, including inhibitors		every 4th week, from week 0 to week 56	No
Secondary	Clinical laboratory assessments: Biochemistry, haematology		every 6th month, from 0 to week 56	No
Secondary	Physical examinations		every 4th week, from 0 to week 52	No
Secondary	Vital signs (Blood Pressure)		every 4th week, from week 0 to week 56	No
Secondary	Vital signs (Pulse)		every 4th week, from week 0 to week 56	No
Secondary	Rate (number per subject year) of all bleeding episodes requiring treatment with a FXIII containing product other than recombinant factor XIII		weeks 0-52	No

External Links

•   Clinical Trials at Novo Nordisk