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Lookup NU author(s): Dr Sally Johnson, Dr Edwin Wong
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
Copyright © 2024 The Author(s). Published by Wolters Kluwer Health, Inc.Randomized clinical trials are underway to evaluate the efficacy of novel agents targeting the alternative complement pathway in patients with C3 glomerulopathy (C3G), a rare glomerular disease. The Kidney Health Initiative convened a panel of experts in C3G to (1) assess the data supporting the use of the prespecified trial end points as measures of clinical benefit and (2) opine on efficacy findings they would consider compelling as treatment(s) of C3G in native kidneys. Two subpanels of the C3G Trial Endpoints Work Group reviewed the available evidence and uncertainties for the association between the three prespecified end points - (1) proteinuria, (2) eGFR, and (3) histopathology - and anticipated outcomes. The full work group provided feedback on the summaries provided by the subpanels and on what potential treatment effects on the proposed end points they would consider compelling to support evidence of an investigational product's effectiveness for treating C3G. Members of the full work group agreed with the characterization of the data, evidence, and uncertainties, supporting the end points. Given the limitations of the available data, the work group was unable to define a minimum threshold for change in any of the end points that might be considered clinically meaningful. The work group concluded that a favorable treatment effect on all three end points would provide convincing evidence of efficacy in the setting of a therapy that targeted the complement pathway. A therapy might be considered effective in the absence of complete alignment in all three end points if there was meaningful lowering of proteinuria and stabilization or improvement in eGFR. The panel unanimously supported efforts to foster data sharing between academic and industry partners to address the gaps in the current knowledge identified by the review of the end points in the aforementioned trials.
Author(s): Nester C, Decker DA, Meier M, Aslam S, Bomback AS, Caravaca-Fontan F, Cook TH, Feldman DL, Fremeaux-Bacchi V, Gale DP, Gooch A, Johnson S, Licht C, Mathur M, Pickering MC, Praga M, Remuzzi G, Selvarajah V, Smith RJ, Tabriziani H, Van De Kar N, Wang Y, Wong E, Mistry K, Lim M, Portillo C, Balogun S, Trachtman H, Thompson A
Publication type: Article
Publication status: Published
Journal: Clinical Journal of the American Society of Nephrology
Year: 2024
Pages: epub ahead of print
Online publication date: 03/06/2024
Acceptance date: 02/04/2024
Date deposited: 27/08/2024
ISSN (print): 1555-9041
ISSN (electronic): 1555-905X
Publisher: Lippincott Williams and Wilkins
URL: https://doi.org/10.2215/CJN.0000000000000505
DOI: 10.2215/CJN.0000000000000505
PubMed id: 38829708
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