Blocking the complement system as a therapeutic strategy has been proposed for numerous glomerular diseases but presents myriad questions and challenges, not the least of which is demonstrating efficacy and safety. In light of these potential issues and because there are an increasing number of anticomplement therapy trials either planned or under way, the National Kidney Foundation facilitated an all-virtual scientific workshop entitled “Improving Clinical Trials for Anti-Complement Therapies in Complement-Mediated Glomerulopathies.” Attended by patient representatives and experts in glomerular diseases, complement physiology, and clinical trial design, the aim of this workshop was to develop standards applicable for designing and conducting clinical trials for anticomplement therapies across a wide spectrum of complement-mediated glomerulopathies. Discussions focused on study design, participant risk assessment and mitigation, laboratory measurements and biomarkers to support these studies, and identification of optimal outcome measures to detect benefit, specifically for trials in complement-mediated diseases. This report summarizes the discussions from this workshop and outlines consensus recommendations.

中文翻译：

---

改进补体介导的肾小球病的抗补体疗法的临床试验：国家肾脏基金会赞助的科学研讨会报告


阻断补体系统作为一种治疗策略已被提议用于治疗多种肾小球疾病，但提出了无数问题和挑战，其中最重要的是证明有效性和安全性。鉴于这些潜在问题，并且由于计划或正在进行的抗补体治疗试验数量不断增加，国家肾脏基金会举办了一次全虚拟科学研讨会，题为“改进补体介导的肾小球病的抗补体疗法的临床试验”。 ”本次研讨会由患者代表和肾小球疾病、补体生理学和临床试验设计方面的专家参加，其目的是制定适用于设计和开展各种补体介导的肾小球疾病的抗补体疗法临床试验的标准。讨论的重点是研究设计、参与者风险评估和缓解、支持这些研究的实验室测量和生物标志物，以及确定最佳结果测量以检测益处，特别是针对补体介导疾病的试验。本报告总结了本次研讨会的讨论并概述了共识建议。