Nonacog beta pegol in previously treated children with hemophilia B: results from an international open-label phase 3 trial.
Nonacog beta pegol in previously treated children with hemophilia B: results from an international open-label phase 3 trial
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.