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

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.

Timeline

Start

2009-09-21

Primary completion

2015-10-20

Completion

2015-10-20

Publications

• Results Brand-Staufer B, Carcao M, Kerlin BA, Will A, Williams M, Tornoe 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):380-385. doi: 10.1111/hae.12616. Epub 2015 Jan 21.
• Results 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.
• Byrnes JR, Lee T, Sharaby S, Campbell RA, Dobson DA, Holle LA, Luo M, Kangro K, Homeister JW, Aleman MM, Luyendyk JP, Kerlin BA, Dumond JB, Wolberg AS. Reciprocal stabilization of coagulation factor XIII-A and -B subunits is a determinant of plasma FXIII concentration. Blood. 2024 Feb 1;143(5):444-455. doi: 10.1182/blood.2023022042.

Drugs

Indications