[Translation] A multicenter, open-label, continuous-dose Phase Ib/II clinical trial: Evaluating the tolerability of CAN106 injection in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who have not received previous complement inhibitor therapy safety, efficacy and safety, pharmacokinetics and pharmacodynamic characteristics
Ib期 主要目的 评价CAN106注射液(CAN106)连续给药在阵发性睡眠性血红蛋白尿症(PNH)受试者中的耐受性和安全性。次要目的1评价CAN106连续给药在PNH受试者中的药代动力学和药效学特征2评价CAN106连续给药在PNH受试者中的免疫原性3评价CAN106连续给药在PNH受试者的暴露-反应关系。探索性目的1初步探索CAN106连续给药在PNH受试者中的有效性2初步探索CAN106连续给药在PNH受试者中补体末端途径激活的状态3初步探索PNH受试者补体受体1(CR1)基因和补体C5基因位点多态性和治疗反应的关系4初步探索经CAN106在PNH受试者给药后红细胞表面C3d沉积变化情况。
II期主要目的评价CAN106在PNH受试者中的有效性。次要目的1评价CAN106在PNH受试者中的安全性2评价CAN106在PNH受试者中的药代动力学和药效学特征3评价CAN106在PNH受试者中的免疫原性4评价CAN106在PNH受试者的暴露-反应关系5评价PNH受试者CR1基因和补体C5基因位点多态性和治疗反应的关系6评价经CAN106在PNH受试者给药后红细胞表面C3d沉积变化情况
[Translation] Phase Ib The main purpose is to evaluate the tolerability and safety of continuous administration of CAN106 injection (CAN106) in subjects with paroxysmal nocturnal hemoglobinuria (PNH). Secondary objectives 1. To evaluate the pharmacokinetics and pharmacodynamics characteristics of CAN106 after continuous administration in PNH subjects. 2. To evaluate the immunogenicity of CAN106 after continuous administration in PNH subjects. 3. To evaluate the effects of continuous administration of CAN106 on PNH subjects. The subject's exposure-response relationship. Exploratory purpose 1. Preliminary exploration of the effectiveness of continuous administration of CAN106 in PNH subjects 2. Preliminary exploration of the activation status of the terminal complement pathway of CAN106 continuous administration in PNH subjects 3. Preliminary exploration of complement receptors in PNH subjects 1 ( The relationship between CR1) gene and complement C5 gene locus polymorphisms and treatment response 4 Preliminary exploration of the changes in C3d deposition on the surface of red blood cells after CAN106 administration in PNH subjects.
The main purpose of Phase II is to evaluate the effectiveness of CAN106 in subjects with PNH. Secondary objectives 1. Evaluate the safety of CAN106 in PNH subjects 2. Evaluate the pharmacokinetic and pharmacodynamic characteristics of CAN106 in PNH subjects 3. Evaluate the immunogenicity of CAN106 in PNH subjects 4. Evaluate CAN106 Exposure-response relationship in PNH subjects 5 Evaluation of the relationship between CR1 gene and complement C5 gene locus polymorphisms and treatment response in PNH subjects 6 Evaluation of changes in C3d deposition on the surface of red blood cells after CAN106 administration in PNH subjects