Complement-mediated Kidney Disease

This Phase 2 study is designed to assess the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-038 in adults with complement-mediated kidney diseases.

This study is a first-in-human (FIH) trial of CMS-D017 conducted in healthy Chinese adult participants, consisting of two parts: Part 1-a single ascending dose (SAD) study (referred to as Part 1 SAD), and Part 2-a multiple ascending dose (MAD) study (referred to as Part 2 MAD). The study aims to evaluate the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) characteristics of CMS-D017 capsules following single and multiple oral administrations in healthy Chinese adult participants. Both parts of the study are designed as randomized, double-blind, placebo-controlled, sequential cohort trials. Part 1 SAD plans to include 6 dose cohorts, with 8 participants per cohort (6 receiving CMS-D017 and 2 receiving placebo), for a total of 48 participants. Part 2 MAD plans to include 4 dose cohorts, with 10 participants per cohort (8 receiving CMS-D017 and 2 receiving placebo), for a total of 40 participants.

This is a non-interventional chart abstraction cohort study with longitudinal follow up. Patients with C3G treated with iptacopan will be enrolled and characterized (i.e., systematically describe and summarize) regarding their medical history and iptacopan use and evaluated for clinical events, outcomes, and laboratory measurements upon and after iptacopan treatment initiation. Medical charts will be used to obtain secondary pseudonymized patient-level data with reference to 2 time anchors: at index date (date of iptacopan treatment initiation) with baseline covering 12 months prior to index date, and at 12-month follow-up (twelve months after the index date).The observation period includes baseline plus follow-up. Iptacopan will be used as prescribed by the clinician in accordance with the terms of the marketing authorization. This Novartis-sponsored study, mainly executed by a contract research organization (CRO), will use secondary data from EHR obtained through reference centers/ centers of excellence in glomerular diseases in Germany. The primary objective of this study is to characterize the demographic and clinical profiles of adult patients diagnosed with C3G upon iptacopan treatment initiation.