Design of the APPEAR-C3G study: a study to learn how well iptacopan works in people with complement 3 glomerulopathy

Abstract

This is a summary of the article describing the design of the APPEAR-C3G clinical study, which was published in Kidney International Reports in August 2022. The APPEAR-C3G study is an ongoing study that includes participants with complement 3 glomerulopathy, known as C3G. Results of the study will be published in the future. C3G is an extremely rare disease that causes damage to the kidneys, and usually starts in children or young adults. There are currently no medicines approved by the United States Food and Drug Administration (FDA) or European Medicines Agency (EMA) to treat the cause of C3G. APPEAR-C3G is a study to learn more about a new medicine called iptacopan. The main purpose of the study is to help researchers understand if iptacopan can stop or slow down the worsening of kidney function (how well the kidneys work) in participants with C3G who have not had a kidney transplant. The study will also help researchers learn if participants experience any side effects or other health problems during the study, known as adverse events. APPEAR-C3G will enroll around 68 participants who will take part in the study for 12 months. The study will have two parts. In Part 1, half of the participants will take iptacopan and the other half will take a placebo (a pill that looks the same but with no medicine in it) for 6 months. The effects of iptacopan will be compared with the effects of the placebo. In Part 2, all participants will take iptacopan for an additional 6 months. At the end of the study, all participants will have the option to continue taking iptacopan as part of a long-term extension study. The APPEAR-C3G study is registered with the study identifier NCT04817618 The extension study of APPEAR-C3G is registered with the study identifier NCT03955445