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

Study Description

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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.

Condition or disease	Intervention/treatment	Phase
Congenital Bleeding Disorder; Congenital FXIII Deficiency	Drug: catridecacog	Phase 3

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	63 participants
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Prevention
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
Actual Study Start Date :	September 21, 2009
Actual Primary Completion Date :	October 20, 2015
Actual Study Completion Date :	October 20, 2015

Arms and Interventions

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Arm	Intervention/treatment
Experimental: A	Drug: catridecacog; Monthly administration of recombinant factor XIII as preventative treatment of bleeding episodes. Dose: 35 IU/kg body weight intravenous (into the vein); Other Name: recombinant factor XIII

Outcome Measures

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Primary Outcome Measures :

1. Adverse Events (AEs)(Serious and Non-serious) [ 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. ]
   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 :

1. Antibody and Inhibitor Development [ Time Frame: From week 0 to week 52 ]
   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.

Eligibility Criteria

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Ages Eligible for Study:	6 Years and older (Child, Adult, Senior)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

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

Contacts and Locations

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  Show 34 Study Locations

Sponsors and Collaborators

Novo Nordisk A/S

Investigators

Study Director:	Global Clinical Registry (GCR, 1452)	Novo Nordisk A/S

More Information

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Additional Information:

Clinical Trials at Novo Nordisk 

Publications of Results:

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. Haemophilia. 2015 May;21(3):380-5. doi: 10.1111/hae.12616. Epub 2015 Jan 21.

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.

Responsible Party:	Novo Nordisk A/S
ClinicalTrials.gov Identifier:	NCT00978380 History of Changes
Other Study ID Numbers:	F13CD-3720; 2008-007883-41 ( EudraCT Number ); U1111-1111-9289 ( Other Identifier: WHO ); JapicCTI-121958 ( Registry Identifier: JAPIC )
First Posted:	September 16, 2009
Results First Posted:	December 13, 2016
Last Update Posted:	January 24, 2018
Last Verified:	January 2018

Additional relevant MeSH terms:

Blood Coagulation Disorders; Hemostatic Disorders; Factor XIII Deficiency; Hematologic Diseases; Vascular Diseases	Cardiovascular Diseases; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Genetic Diseases, Inborn