# Phase 2 Study of Avacopan for Treatment of C3G, IND 132321, Orphan Designation 16-5590 > **NIH FDA R01** · CHEMOCENTRYX, INC. · 2020 · $500,000 ## Abstract Project Summary Complement 3 Glomerulopathy (C3G) is an orphan disease which includes C3 glomerulonephritis (C3GN) and the closely related dense deposit disease (DDD), forms of glomerulonephritis. This disease results from abnormal regulation of the alternative complement pathway resulting in unrestrained complement activation. The clinical presentation varies from mild proteinuria to rapid renal deterioration and about half of the individuals with C3G move the End Stage Renal Disease within in 10 years of diagnosis. There are no approved drugs for C3G currently The current treatment recommendations include control of hypertension with angiotensin-converting enzyme (ACE)/angiotensin receptor blocker (ARBs) to empirical use of plasma exchange or infusion. None of these treatments address the dysregulation of the complement system. Hence, there is a significant unmet medical need for novel drugs to treat C3G. Avacopan is an orally active drug that specific inhibitor of the C5a receptor (part of the complement system) and has been shown to be safe and efficacious in Phase 1 and Phase 2 human clinical trials in AAV, and is currently being tested in a Phase 3 AAV trial. More importantly, compelling clinical, laboratory, and kidney biopsy responses have been observed in C3G patients treated with avacopan under a compassionate use protocol. Taken together, these findings provide a strong justification for a human clinical trial to test the safety and efficacy of avacopan as a novel treatment of C3G. This study will enroll 44 patients with C3G at 40 clinical sites in 11 countries. The primary goal of the study is to evaluate the efficacy of avacopan compared to placebo based on histologic changes in kidney biopsies taken before and during treatment. Secondary goals of this study include the evaluation of: 1 The safety of avacopan compared to placebo based on the incidence of adverse events, changes in clinical laboratory measurements, and vital signs; 2 Changes in laboratory parameters of renal disease including estimated glomerular filtration rate (eGFR), proteinuria, and urinary excretion of monocyte chemoattractant protein-1 (MCP- 1) with avacopan compared to placebo; 3 Health-related quality-of-life changes based on Short Form-36 version 2 (SF-36 v2) and EuroQOL-5D-5L (EQ-5D-5L) with avacopan compared to placebo; 4 The pharmacokinetic profile of avacopan in patients with C3G. Additionally, exploratory evaluations studying changes from baseline in markers of alternative complement pathway involvement and other markers of inflammation may be assessed in plasma/serum or urine over the course of the treatment period. ## Key facts - **NIH application ID:** 10015262 - **Project number:** 5R01FD006342-02 - **Recipient organization:** CHEMOCENTRYX, INC. - **Principal Investigator:** Peter M Staehr - **Activity code:** R01 (R01, R21, SBIR, etc.) - **Funding institute:** FDA - **Fiscal year:** 2020 - **Award amount:** $500,000 - **Award type:** 5 - **Project period:** 2019-09-10 → 2022-01-31 ## Primary source NIH RePORTER: https://reporter.nih.gov/project-details/10015262 ## Citation > US National Institutes of Health, RePORTER application 10015262, Phase 2 Study of Avacopan for Treatment of C3G, IND 132321, Orphan Designation 16-5590 (5R01FD006342-02). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10015262. Licensed CC0. --- *[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*