[Translation] A single-arm, open-label, multicenter clinical trial to evaluate the efficacy, safety, and pharmacokinetic characteristics of recombinant human coagulation factor VIII in the prevention and treatment of severe hemophilia A in children who have been treated
主要目的:评价12岁以下经治疗的重型血友病A患儿给予注射用重组人凝血因子Ⅷ制剂进行预防治疗的有效性和安全性。
次要目的:观察注射用重组人凝血因子Ⅷ在12岁以下经治疗的重型血友病A患儿中单次给药后的药代动力学特征,并计算药代动力学参数。
探索性目的:1)探索注射用重组人凝血因子Ⅷ个体化用药方法。2)探索性观察围手术期的重型血友病A患儿使用注射用重组人凝血因子Ⅷ的疗效。
[Translation] Primary objective: To evaluate the efficacy and safety of recombinant human coagulation factor VIII preparations for prophylaxis in children under 12 years of age with severe hemophilia A who have been treated.
Secondary objective: To observe the pharmacokinetic characteristics of recombinant human coagulation factor VIII after a single dose in children under 12 years of age with severe hemophilia A who have been treated, and to calculate the pharmacokinetic parameters.
Exploratory objectives: 1) To explore the individualized medication method of recombinant human coagulation factor VIII for injection. 2) To explore the efficacy of recombinant human coagulation factor VIII for injection in children with severe hemophilia A in the perioperative period.