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 Cravedi; Elif 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

doi:10.1053/j.ajkd.2021.07.025

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 journal › Article › peer-review

Abstract

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 language	English (US)
Pages (from-to)	570-581
Number of pages	12
Journal	American Journal of Kidney Diseases
Volume	79
Issue number	4
DOIs	https://doi.org/10.1053/j.ajkd.2021.07.025
State	Published - Apr 2022

Keywords

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

ASJC Scopus subject areas

Nephrology

Access to Document

10.1053/j.ajkd.2021.07.025

Cite this

Bomback, A. S., Appel, G. B., Gipson, D. S., Hladunewich, M. A., Lafayette, R., Nester, C. M., Parikh, S. V., Smith, R. J. H., Trachtman, H., Heeger, P. S., Ram, S., Rovin, B. H., Ali, S., Arceneaux, N., Ashoor, I., Bailey-Wickins, L., Barratt, J., Beck, L., Cattran, D. C., ... Thurman, J. M. (2022). Improving Clinical Trials for Anticomplement Therapies in Complement-Mediated Glomerulopathies: Report of a Scientific Workshop Sponsored by the National Kidney Foundation. American Journal of Kidney Diseases, 79(4), 570-581. https://doi.org/10.1053/j.ajkd.2021.07.025

Improving Clinical Trials for Anticomplement Therapies in Complement-Mediated Glomerulopathies: Report of a Scientific Workshop Sponsored by the National Kidney Foundation. / Bomback, Andrew S.; Appel, Gerald B.; Gipson, Debbie S. et al.
In: American Journal of Kidney Diseases, Vol. 79, No. 4, 04.2022, p. 570-581.

Research output: Contribution to journal › Article › peer-review

Bomback, AS, Appel, GB, Gipson, DS, Hladunewich, MA, Lafayette, R, Nester, CM, Parikh, SV, Smith, RJH, Trachtman, H, Heeger, PS, Ram, S, Rovin, BH, Ali, S, Arceneaux, N, Ashoor, I, Bailey-Wickins, L, Barratt, J, Beck, L, Cattran, DC, Cravedi, P, Erkan, E, Fervenza, F, Frazer-Abel, AA, Fremeaux-Bacchi, V, Fuller, L, Gbadegesin, R, Hogan, JJ, Kiryluk, K, le Quintrec-Donnette, M, Licht, C, Mahan, JD, Pickering, MC, Quigg, R, Rheault, M, Ronco, P, Sarwal, MM, Sethna, C, Spino, C, Stegall, M, Vivarelli, M, Feldman, DL & Thurman, JM 2022, 'Improving Clinical Trials for Anticomplement Therapies in Complement-Mediated Glomerulopathies: Report of a Scientific Workshop Sponsored by the National Kidney Foundation', American Journal of Kidney Diseases, vol. 79, no. 4, pp. 570-581. https://doi.org/10.1053/j.ajkd.2021.07.025

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

abstract = "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.",

keywords = "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",

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

note = "Publisher Copyright: {\textcopyright} 2021 National Kidney Foundation, Inc.",

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doi = "10.1053/j.ajkd.2021.07.025",

language = "English (US)",

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AU - Gipson, Debbie S.

AU - Hladunewich, Michelle A.

AU - Lafayette, Richard

AU - Nester, Carla M.

AU - Parikh, Samir V.

AU - Smith, Richard J.H.

AU - Trachtman, Howard

AU - Heeger, Peter S.

AU - Ram, Sanjay

AU - Rovin, Brad H.

AU - Ali, Shadab

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AU - Ashoor, Isa

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AU - Barratt, Jonathan

AU - Beck, Laurence

AU - Cattran, Daniel C.

AU - Cravedi, Paolo

AU - Erkan, Elif

AU - Fervenza, Fernando

AU - Frazer-Abel, Ashley A.

AU - Fremeaux-Bacchi, Veronique

AU - Fuller, Lindsey

AU - Gbadegesin, Rasheed

AU - Hogan, Jonathan J.

AU - Kiryluk, Krzysztof

AU - le Quintrec-Donnette, Moglie

AU - Licht, Christoph

AU - Mahan, John D.

AU - Pickering, Matthew C.

AU - Quigg, Richard

AU - Rheault, Michelle

AU - Ronco, Pierre

AU - Sarwal, Minnie M.

AU - Sethna, Christine

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AB - 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.

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KW - glomerulonephritides

KW - infectious risk

KW - patient-reported outcome (PRO)

KW - rare diseases

KW - research priorities

KW - surrogate outcome

KW - trial end points

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

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Keywords

ASJC Scopus subject areas

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