Improving Clinical Trials for Anticomplement Therapies in Complement-Mediated Glomerulopathies: Report of a Scientific Workshop Sponsored by the National Kidney Foundation

Andrew S. Bomback, Gerald B. Appel, Debbie S. Gipson, Michelle A. Hladunewich, Richard Lafayette, Carla M. Nester, Samir V. Parikh, Richard J.H. Smith, Howard Trachtman, Peter S. Heeger, Sanjay Ram, Brad H. Rovin, Shadab Ali, Nicole Arceneaux, Isa Ashoor, Laura Bailey-Wickins, Jonathan Barratt, Laurence Beck, Daniel C. Cattran, Paolo CravediElif Erkan, Fernando Fervenza, Ashley A. Frazer-Abel, Veronique Fremeaux-Bacchi, Lindsey Fuller, Rasheed Gbadegesin, Jonathan J. Hogan, Krzysztof Kiryluk, Moglie le Quintrec-Donnette, Christoph Licht, John D. Mahan, Matthew C. Pickering, Richard Quigg, Michelle Rheault, Pierre Ronco, Minnie M. Sarwal, Christine Sethna, Cathie Spino, Mark Stegall, Marina Vivarelli, David L. Feldman, Joshua M. Thurman

Research output: Contribution to journalArticlepeer-review

4 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)570-581
Number of pages12
JournalAmerican Journal of Kidney Diseases
Issue number4
StatePublished - Apr 2022

Bibliographical note

Funding Information:
Funding for the workshop was provided by Alexion, Apellis, Biocryst, ChemoCentryx, Omeros, Alnylam, Novartis, and Q32 Bio. The funders had no role in defining the content of the report.

Funding Information:
Andrew S. Bomback, MD, MPH, Gerald B. Appel, MD, Debbie S. Gipson, MD, MS, Michelle A. Hladunewich, MD, FRCPC, MSc, Richard Lafayette, MD, Carla M. Nester, MD, MSA, Samir V. Parikh, MD, Richard J.H. Smith, MD, Howard Trachtman, MD, Peter S. Heeger, MD, Sanjay Ram, MD, Brad H. Rovin, MD, Shadab Ali, BS, MBA, Nicole Arceneaux, BA, MS, Isa Ashoor, MD, Laura Bailey-Wickins, BA, MBA, Jonathan Barratt, PhD, FRCP, Laurence Beck, MD, PhD, Daniel C. Cattran, MD, FRCPC, Paolo Cravedi, MD, PhD, Elif Erkan, MD, MS, Fernando Fervenza, MD, PhD, Ashley A. Frazer-Abel, PhD, Veronique Fremeaux-Bacchi, MD, PhD, Lindsey Fuller, BS, MS, Rasheed Gbadegesin, MD, MBBS, Jonathan J. Hogan, MD, Krzysztof Kiryluk, MD, MS, Moglie Le Quintrec, MD, PhD, Christoph Licht, MD, John D. Mahan, MD, Matthew C. Pickering, PhD, Richard Quigg, MD, Michelle Rheault, MD, Pierre Ronco, MD, PhD, Minnie M. Sarwal, MD, PhD, FRCP, MRCP, DCh, Christine Sethna, MD, EdM, Cathie Spino, ScD, Mark Stegall, MD, Marina Vivarelli, MD, David L. Feldman, PhD, and Joshua M. Thurman, MD. Funding for the workshop was provided by Alexion, Apellis, Biocryst, ChemoCentryx, Omeros, Alnylam, Novartis, and Q32 Bio. The funders had no role in defining the content of the report. Dr Bomback reports consulting honoraria from Achillion, Alexion, Catalyst, ChemoCentryx, Novartis, Silence, and Visterra. Dr Appel reports consulting honoraria from Apellis and Alexion. Dr Barratt reports receiving honoraria for consulting and/or advisory boards from Alnylam, argenx, BioCryst, Ionis, Novartis, and Omeros. Dr Beck reports consulting honoraria from Alexion, Ionis, and Visterra; in addition, he is coinventor on the patent ?Diagnostics for Membranous Nephropathy.? Dr Cattran is a member of the Data Monitoring Committee for Novartis's study of LNP023 for IgA nephropathy. Dr Fervenza reports consulting honoraria from Genentech, Roche, Takeda, Novartis, ChemoCentryx, Alnylam, and Morphosys. Dr Fremeaux-Bacchi reports consulting honoraria from Alexion, Biocryst, Roche, and Apellis. Ms Fuller has participated in an advisory board for Apellis and a research interview with Biocryst. Dr Gipson reports research funding from Novartis. Dr Heeger reports consulting honoraria from Mallinckrodt. Dr Hladunewich reports consulting honoraria from Alnylam and research support from Ionis. Dr Hogan reports consulting honoraria and research support from Alexion. Dr Kiryluk reports serving on an advisory board for Goldfinch and Gilead. Dr Lafayette reports consulting honoraria from Omeros, Novartis, and Alexion. Dr Le Quintrec reports consulting honoraria from Novartis, Alexion, Sanofi, and Astellas. Dr Licht reports consulting honoraria from Alexion, Novartis, Apelli, and Catalyst. Dr Nester reports consulting honorarium from Biocryst and research funding from Novartis. Dr Parikh reports consulting honoraria from Alexion, Aurinia, Bristol Myers Squibb, GlaxoSmithKline, and Kezar Life, as well as research funding from EMD-Serono and Aurinia. Dr Pickering reports consulting honoraria from Gyroscope, Apellis, Alexion, Sobi, Silence, and Gemini Pharma. Dr Ram reports consulting honoraria from Ionis and serves on the data safety monitoring board for Apellis. Dr Ronco reports consulting honoraria from Alexion and Morphosys. Dr Rovin reports consulting honoraria from Omeros, Biocryst, Novartis, and Alexion. Dr Sarwa reports consulting honoraria from Roche, Genentech, Jazz, Ionis, Astella, Bristol Myers Squibb, Organ-I, and NephroSant. Dr Smith reports consulting honoraria from Novartis. Dr Stegall reports consulting honoraria from Genentech, Roche, Takeda, Novartis, Hansa, eGenesis, and Sanofi and research contracts with Transplant Genomic and Veloxis. Dr Vivarelli reports consulting honoraria from Travere, Novartis, Roche, Apellis, and Achillion. Dr Trachtman reports consulting for Travere, Goldfinch, Otsuka, and ChemoCentryx and receipt of research support from Natera. Dr Thurman reports royalties from Alexion and consulting honorarium from Q32 Bio, for which he holds stock and will receive royalties. The remaining authors declare that they have no relevant financial interests. The authors are incredibly grateful to Sarah Kim at the National Kidney Foundation for her behind-the-scenes work in planning and organizing the workshops, including trouble-shooting all aspects of the virtual format. Received March 23, 2021. Evaluated by 3 external peer reviewers, with direct editorial input from an Associate Editor, who served as Acting Editor-in-Chief. Accepted in revised form July 13, 2021. The involvement of an Acting Editor-in-Chief was to comply with AJKD's procedures for potential conflicts of interest for editors, described in the Information for Authors & Journal Policies.

Publisher Copyright:
© 2021 National Kidney Foundation, Inc.


  • Glomerular disease
  • clinical trial design
  • complement biomarkers
  • complement blockade
  • complement inhibition
  • complement-mediated glomerulopathy
  • drug approval pathway
  • drug safety
  • glomerulonephritides
  • infectious risk
  • patient-reported outcome (PRO)
  • rare diseases
  • research priorities
  • surrogate outcome
  • trial end points

PubMed: MeSH publication types

  • Congress
  • Research Support, Non-U.S. Gov't


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