Nonacog beta pegol in previously treated children with hemophilia B: results from an international open‐label phase 3 trial. (22nd June 2016)

• Record Type: Journal Article
• Authors: Carcao, M.; Zak, M.; Abdul Karim, F.; Hanabusa, H.; Kearney, S.; Lu, M.‐Y.; Persson, P.; Rangarajan, S.; Santagostino, E.
• Is Part Of: Journal of thrombosis and haemostasis. Volume 14:Number 8(2016:Aug.)
• Page Start: 1521
• Page End: 1529
• Publication Date: 2016-06-22
• Subjects: factor IX -- hemophilia B -- nonacog beta pegol -- pediatrics -- prophylaxis; Thrombosis -- Periodicals; Hemostasis -- Periodicals; Blood coagulation disorders -- Periodicals; 616.1
• DOI: 10.1111/jth.13360
• Languages: English
• ISSNs: 1538-7933

Abstract

Abstract : Essentials Nonacog beta pegol is a recombinant glycoPEGylated factor IX with an extended half‐life. This phase 3 trial investigated its safety/efficacy in previously treated hemophilia B boys ≤ 12 years. A 40 IU kg −1 dose provided effective once‐weekly prophylaxis and hemostasis when used to treat bleeds. Nonacog beta pegol was well tolerated in previously treated boys ≤ 12 years with hemophilia B. Summary: Background: Nonacog beta pegol is a recombinant glycoPEGylated factor IX with an extended half‐life, developed to improve care for patients with hemophilia B. Objectives: To investigate the safety, efficacy and pharmacokinetics of nonacog beta pegol for the prophylaxis and treatment of bleeds in previously treated children with hemophilia B. Patients/Methods: This phase 3 trial, paradigm ™ 5, enrolled and treated 25 children (aged ≤ 12 years) with hemophilia B (FIX ≤ 2%). Patients were stratified by age (0–6 years and 7–12 years), and received once‐weekly prophylaxis with 40 IU kg −1 nonacog beta pegol for 50 exposure days. Results: No patient developed inhibitors, and no safety concerns were identified. Forty‐two bleeds in 15 patients were reported to have been treated; the overall success rate was 92.9%, and most bleeds (85.7%) resolved after one dose. The median annualized bleeding rates (ABRs; bleeds per patient per year) were 1.0 in the total population, 0.0 in the 0–6‐year group, and 2.0 in the 7–12‐year group; the estimated mean ABRs were 1.44 in the total population, 0.87 in the 0–6‐year group, and 1.88 in the 7–12‐year group. For 22 patients who had previously been receiving prophylaxis, the estimated mean ABR was 1.38 versus a historical ABR of 2.51. Estimated mean steady‐state FIX trough levels were 0.153 IU mL −1 (0–6 years) and 0.190 IU mL −1 (7–12 years). Conclusion: Nonacog beta pegol was well tolerated in previously treated children with hemophilia B; a 40 IU kg −1 dose provided effective once‐weekly prophylaxis and hemostasis when bleeds were treated.