[Translation] A multicenter, single-arm, open-label phase III clinical study to evaluate the efficacy and safety of HSK39297 tablets in patients with paroxysmal nocturnal hemoglobinuria who still have anemia after stable use of C5 monoclonal antibody
主要目的:评价HSK39297片在C5单抗治疗后仍贫血的阵发性睡眠性血红蛋白尿症(PNH)患者中的有效性。 次要目的:
评价HSK39297片在C5单抗治疗后仍贫血PNH患者中对输血需求的影响;
评价HSK39297片在C5单抗治疗后仍贫血PNH患者中对贫血纠正及溶血控制的影响;
评价HSK39297片在C5单抗治疗后仍贫血PNH患者中对疲劳症状的改善作用;
评价HSK39297片在C5单抗治疗后仍贫血PNH患者中突破性溶血(BTH)与主要不良血管事件(MAVE)发生情况;
评价HSK39297片在C5单抗治疗后仍贫血PNH患者中的安全性;
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 in patients with paroxysmal nocturnal hemoglobinuria (PNH) who are still anemic after C5 monoclonal antibody treatment. Secondary objectives:
To evaluate the effect of HSK39297 tablets on the need for blood transfusion in PNH patients who are still anemic after C5 monoclonal antibody treatment;
To evaluate the effect of HSK39297 tablets on anemia correction and hemolysis control in PNH patients who are still anemic after C5 monoclonal antibody treatment;
To evaluate the improvement of fatigue symptoms in PNH patients who are still anemic after C5 monoclonal antibody treatment with HSK39297 tablets;
To evaluate the occurrence of breakthrough hemolysis (BTH) and major adverse vascular events (MAVE) in PNH patients who are still anemic after C5 monoclonal antibody treatment with HSK39297 tablets;
To evaluate the safety of HSK39297 tablets in PNH patients who are still anemic after C5 monoclonal antibody treatment;
To evaluate the pharmacokinetic (PK) and pharmacodynamic (PD) characteristics of HSK39297 tablets in PNH patients