[Translation] A single-arm, open-label, single-center clinical trial to evaluate the safety and pharmacokinetic characteristics of human coagulation factor IX after a single dose in patients with moderate to severe hemophilia B (factor IX deficiency)
主要目的:
评价人凝血因子Ⅸ在血友病B(FⅨ活性水平≤2%)患者中单次给药后PK/PD特征。
次要目的:
评价人凝血因子Ⅸ在血友病B(FⅨ活性水平≤2%)患者中单次给药后的安全性;
评价人凝血因子Ⅸ在血友病B(FⅨ活性水平≤2%)患者出血事件按需治疗的安全性和有效性。
[Translation] Primary objective:
To evaluate the PK/PD characteristics of human coagulation factor IX after a single dose in patients with hemophilia B (FIX activity level ≤2%).
Secondary objective:
To evaluate the safety of human coagulation factor IX after a single dose in patients with hemophilia B (FIX activity level ≤2%);
To evaluate the safety and efficacy of human coagulation factor IX in the on-demand treatment of bleeding events in patients with hemophilia B (FIX activity level ≤2%).