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....

中文翻译：

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开发 C3 肾小球病的治疗方法：肾脏健康倡议报告 C3 肾小球病试验终点工作组


使用预先设定的试验终点作为临床益处的衡量标准，以及 （2） 对他们认为作为天然肾脏 C3G 治疗的疗效发现发表意见。C3G 试验终点工作组的两个小组审查了三个预先设定的终点（（1） 蛋白尿、（2） eGFR 和 （3） 组织病理学——与预期结局之间关联的现有证据和不确定性。整个工作组就小组提供的摘要以及他们认为对拟议终点的潜在治疗效果提供了反馈，以支持研究产品治疗 C3G 的有效性证据。整个工作组的成员同意数据、证据和不确定性的定性，支持终点。鉴于可用数据的局限性，工作组无法定义任何可能被认为具有临床意义的终点变化的最低阈值。工作组得出结论，在所有三个终点上的良好治疗效果将为针对补体途径的治疗提供令人信服的疗效证据。如果蛋白尿有意义降低并且 eGFR 稳定或改善，则在所有三个终点均未完全对齐的情况下，可能认为一种疗法有效。该小组一致支持促进学术和行业合作伙伴之间的数据共享的努力，以解决通过审查上述试验的终点所确定的当前知识差距。