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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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 Facto

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.

Detailed Summary:
Sponsor: Novo Nordisk A/S

Current Primary Outcome: 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.


Original Primary Outcome: Adverse Events (serious and non-serious) [ Time Frame: from week 0 to week 52 ]

Current Secondary Outcome: 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.


Original Secondary Outcome: Antibody and Inhibitor Development [ Time Frame: From week 0 to week 52 ]

Information By: Novo Nordisk A/S

Dates:
Date Received: September 15, 2009
Date Started: September 21, 2009
Date Completion:
Last Updated: February 28, 2017
Last Verified: February 2017