[Translation] A multicenter, randomized, open-label, positive-controlled phase III clinical study to evaluate the efficacy and safety of HSK39297 tablets in patients with paroxysmal nocturnal hemoglobinuria who had not previously received complement inhibitor treatment
主要目的:
评价HSK39297片相较于依库珠单抗治疗阵发性睡眠性血红蛋白尿症(PNH)患者的有效性。 次要目的: 评价HSK39297片相较于依库珠单抗对输血需求的影响;
评价HSK39297片相较于依库珠单抗对贫血纠正及溶血控制的影响;
评价HSK39297片相较于依库珠单抗对疲劳症状的改善作用;
评价HSK39297片相较于依库珠单抗的突破性溶血(BTH)与主要不良血管事件(MAVE)发生情况;
评价HSK39297片相较于依库珠单抗治疗PNH患者的安全性;
评价HSK39297片用于PNH患者的药代动力学(PK)和药效动力学(PD)特征。
[Translation] Primary objective:
To evaluate the effectiveness of HSK39297 tablets compared with eculizumab in the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). Secondary objectives: To evaluate the effect of HSK39297 tablets on the need for blood transfusion compared with eculizumab;
To evaluate the effect of HSK39297 tablets on anemia correction and hemolysis control compared with eculizumab;
To evaluate the improvement of fatigue symptoms by HSK39297 tablets compared with eculizumab;
To evaluate the occurrence of breakthrough hemolysis (BTH) and major adverse vascular events (MAVE) of HSK39297 tablets compared with eculizumab;
To evaluate the safety of HSK39297 tablets compared with eculizumab in the treatment of PNH patients;
To evaluate the pharmacokinetic (PK) and pharmacodynamic (PD) characteristics of HSK39297 tablets in patients with PNH.