[Translation] A randomized, open-label, C5 inhibitor-controlled study to evaluate the efficacy and safety of Pozelimab combined with Cemdisiran in patients with paroxysmal nocturnal hemoglobinuria who are complement inhibitor-naive or have not recently received complement inhibitor therapy
本研究正在评价一种涉及两种研究药物Pozelimab和Cemdisiran联合用药的临床治疗方案。本研究的目标人群为阵发性夜间血红蛋白尿(PNH)患者。本研究的目的是了解Pozelimab+Cemdisiran联合用药治疗PNH患者的安全性和有效性,并与2种现有治疗(一种称为Ravulizumab,另一种称为依库珠单抗)进行比较。可将Pozelimab+Cemdisiran联合用药称为“研究药物”,也可将Ravulizumab和依库珠单抗称为“对照药”。
本研究主要关注以下几个研究问题:
Pozelimab+Cemdisiran联合用药相较Ravulizumab的有效性?
Pozelimab+Cemdisiran联合用药相较依库珠单抗的有效性?
使用研究药物可能会引发哪些副作用?
在不同时间点您血液中的研究药物含量?
机体是否会产生抗研究药物的抗体(可能使研究药物疗效降低或可能导致副作用)
[Translation] This study is evaluating a clinical treatment regimen that involves two investigational drugs, Pozelimab and Cemdisiran, given in combination. The study population is people with paroxysmal nocturnal hemoglobinuria (PNH). The purpose of this study is to learn about the safety and effectiveness of the combination of Pozelimab and Cemdisiran in treating people with PNH and to compare it with 2 existing treatments, one called Ravulizumab and the other called Eculizumab. The combination of Pozelimab and Cemdisiran may be called the "study drug," and Ravulizumab and Eculizumab may be called the "control drugs."
This study focuses on the following research questions:
How effective is the combination of Pozelimab and Cemdisiran compared to Ravulizumab?
How effective is the combination of Pozelimab and Cemdisiran compared to Eculizumab?
What side effects may occur with the study drug?
How much study drug is in your blood at different times?
Will your body develop antibodies against the study drug (which may make the study drug less effective or cause side effects)