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

Congenital Bleeding Disorder Clinical Trial

— 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00978380
• Other study ID #: F13CD-3720
• Secondary ID: 2008-007883-41U1
• Status: Completed
• Phase: Phase 3
• First received: September 15, 2009
• Last updated: January 18, 2018
• Start date: September 21, 2009
• Est. completion date: October 20, 2015

Study information

• Verified date: January 2018
• Source: Novo Nordisk A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 63
• Est. completion date: October 20, 2015
• Est. primary completion date: October 20, 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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

Related Conditions & MeSH terms

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

Intervention

Drug:
• catridecacog
Monthly administration of recombinant factor XIII as preventative treatment of bleeding episodes. Dose: 35 IU/kg body weight intravenous (into the vein)

Locations

Country	Name	City	State
Austria	Novo Nordisk Investigational Site	Klagenfurt
Canada	Novo Nordisk Investigational Site	Toronto	Ontario
Finland	Novo Nordisk Investigational Site	Helsinki
France	Novo Nordisk Investigational Site	Le Kremlin Bicetre
France	Novo Nordisk Investigational Site	Paris
France	Novo Nordisk Investigational Site	Rouen
France	Novo Nordisk Investigational Site	Valence Cedex 9
Germany	Novo Nordisk Investigational Site	Bonn
Germany	Novo Nordisk Investigational Site	Braunschweig
Germany	Novo Nordisk Investigational Site	Duisburg
Israel	Novo Nordisk Investigational Site	Petach Tikva
Italy	Novo Nordisk Investigational Site	Vicenza
Japan	Novo Nordisk Investigational Site	Hiroshima-shi, Hiroshima
Japan	Novo Nordisk Investigational Site	Shinjuku-ku, Tokyo
Spain	Novo Nordisk Investigational Site	Barcelona
Spain	Novo Nordisk Investigational Site	Málaga
Spain	Novo Nordisk Investigational Site	Tortosa
Switzerland	Novo Nordisk Investigational Site	Zürich
United Kingdom	Novo Nordisk Investigational Site	Aberdeen
United Kingdom	Novo Nordisk Investigational Site	London
United Kingdom	Novo Nordisk Investigational Site	Manchester
United Kingdom	Novo Nordisk Investigational Site	Newcastle upon Tyne
United States	Novo Nordisk Investigational Site	Atlanta	Georgia
United States	Novo Nordisk Investigational Site	Boise	Idaho
United States	Novo Nordisk Investigational Site	Columbus	Ohio
United States	Novo Nordisk Investigational Site	Detroit	Michigan
United States	Novo Nordisk Investigational Site	Hershey	Pennsylvania
United States	Novo Nordisk Investigational Site	Minneapolis	Minnesota
United States	Novo Nordisk Investigational Site	Oklahoma City	Oklahoma
United States	Novo Nordisk Investigational Site	Orange	California
United States	Novo Nordisk Investigational Site	Richmond	Virginia
United States	Novo Nordisk Investigational Site	Seattle	Washington
United States	Novo Nordisk Investigational Site	Tampa	Florida
United States	Novo Nordisk Investigational Site	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Countries where clinical trial is conducted

United States,  Austria,  Canada,  Finland,  France,  Germany,  Israel,  Italy,  Japan,  Spain,  Switzerland,  United Kingdom, 

References & Publications (2)

Brand-Staufer B, Carcao M, Kerlin BA, Will A, Williams M, Tornøe 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. Haemophili — View 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 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	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	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.	From week 0 to week 52

External Links

•   Clinical Trials at Novo Nordisk