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Nonacog beta pegol (N9-GP) in haemophilia B: A multinational phase III safety and efficacy extension trial (paradigm™4)

Young, Guy, Collins, Peter, Colberg, Torben, Chuansumrit, Ampaiwan, Hanabusa, Hideji, Lentz, Steven R., Mahlangu, Johnny, Mauser-Bunschoten, Evelien P., Négrier, Claude, Oldenburg, Johannes, Patiroglu, Turkan, Santagostino, Elena, Tehranchi, Ramin, Zak, Marek and Karim, Faraizah Abdul 2016. Nonacog beta pegol (N9-GP) in haemophilia B: A multinational phase III safety and efficacy extension trial (paradigm™4). Thrombosis Research 141 , pp. 69-76. 10.1016/j.thromres.2016.02.030

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Abstract

Introduction Paradigm™4 was an international extension trial investigating the safety and efficacy of nonacog beta pegol, a recombinant glycoPEGylated factor IX (FIX) with extended half-life, in haemophilia B patients (FIX activity ≤ 2%; aged 13–70 years) who had previously participated in phase III pivotal (paradigm™2) or surgery (paradigm™3) trials. Methods Patients chose to continue treatment with nonacog beta pegol in either one of two once-weekly prophylaxis arms (10 IU/kg or 40 IU/kg), or an on-demand arm (40 IU/kg for mild/moderate bleeds; 80 IU/kg for severe bleeds). The primary objective was to evaluate immunogenicity; key secondary objectives included assessing safety and haemostatic efficacy in the treatment and prevention of bleeds. Results Seventy-one patients received prophylaxis or on-demand treatment. No patient developed an inhibitor and no safety concerns were identified. The success rate for the treatment of reported bleeds was 94.6%; most (87.9%) resolved with one injection. The median annualised bleeding rate for patients on prophylaxis was 1.36 (interquartile range [IQR] 0.00–2.23) and 1.00 (IQR 0.00–2.03) for the 10 and 40 IU/kg treatment arms, respectively. The mean FIX activity trough achieved for 10 and 40 IU once weekly was 9.8% and 21.3%, respectively. Fourteen patients on prophylaxis underwent 23 minor surgical procedures; haemostatic perioperative outcomes for all of those evaluated were ‘excellent’ or ‘good’. Conclusions Nonacog beta pegol showed a favourable tolerability profile (with no safety issues identified) with good prophylactic protection and control of bleeding in previously treated adult and adolescent haemophilia B patients.

Item Type: Article
Date Type: Published Online
Status: Published
Schools: Medicine
Uncontrolled Keywords: Nonacog beta pegol; Half-life; Haemophilia BFactor IX; Prophylaxis
Publisher: Elsevier
ISSN: 0049-3848
Date of Acceptance: 27 February 2016
Last Modified: 30 Jun 2017 08:01
URI: http://orca.cf.ac.uk/id/eprint/101948

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