Clinical trial recommendations for potential Alport syndrome therapies

B. André Weinstock; David L. Feldman; Alessia Fornoni; Oliver Gross; Clifford E. Kashtan; Sharon Lagas; Rachel Lennon; Jeffrey H. Miner; Michelle N. Rheault; James F. Simon; Lisa Bonebrake; Marty Dunleavy; Phil Kumnick; Gina Parziale; Janine Reed; André Weinstock; Susie Gear; Kristen Binaso; Raymond Manuel; James Simon; Gerald Appel; Melanie Blank; Winson Tang; Aliza Thompson; Roser Torra; Kenneth Lieberman; Christoph Licht; Karin Dahan; Kandai Nozu; Hirofumi Kai; Sharon Ricardo; Anne Pariser; David Feldman; Heather Cook; Melanie Chin; Angela Goldsberry; Colin Meyer; Lisa Anne Melia; Radko Komers; Michael Markels; Alex Mercer; Marco Prunotto; Bruce Morgenstern; Ali Hariri; Vijay Modur; Neil Turner; Clifford Kashtan; Michelle Rheault; Colin Baigent; Stephano DeSacco; Laura Perin; Moumita Barua; Koichi Nakanishi; George Jarad; Jeffrey Miner

doi:10.1016/j.kint.2020.02.029

Clinical trial recommendations for potential Alport syndrome therapies

B. André Weinstock, David L. Feldman, Alessia Fornoni, Oliver Gross, Clifford E. Kashtan, Sharon Lagas, Rachel Lennon, Jeffrey H. Miner, Michelle N. Rheault, James F. Simon, Lisa Bonebrake, Marty Dunleavy, Phil Kumnick, Gina Parziale, Janine Reed, André Weinstock, Susie Gear, Kristen Binaso, Raymond Manuel, James SimonGerald Appel, Melanie Blank, Winson Tang, Aliza Thompson, Roser Torra, Kenneth Lieberman, Christoph Licht, Karin Dahan, Kandai Nozu, Hirofumi Kai, Sharon Ricardo, Anne Pariser, David Feldman, Heather Cook, Melanie Chin, Angela Goldsberry, Colin Meyer, Lisa Anne Melia, Radko Komers, Michael Markels, Alex Mercer, Marco Prunotto, Bruce Morgenstern, Ali Hariri, Vijay Modur, Neil Turner, Clifford Kashtan, Michelle Rheault, Colin Baigent, Stephano DeSacco, Laura Perin, Moumita Barua, Koichi Nakanishi, George Jarad, Jeffrey Miner

Pediatrics - Nephrology

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

6 Scopus citations

Abstract

Alport syndrome is experiencing a remarkable increase in preclinical investigations. To proactively address the needs of the Alport syndrome community, as well as offer clarity for future clinical research sponsors, the Alport Syndrome Foundation hosted a workshop to generate consensus recommendations for prospective trials for conventional drugs. Opinions of key stakeholders were carefully considered, including those of the biopharmaceutical industry representatives, academic researchers, clinicians, regulatory agency representatives, and—most critically—patients with Alport syndrome. Recommendations were established for preclinical researchers, the use and selection of biomarkers, standards of care, clinical trial designs, trial eligibility criteria and outcomes, pediatric trial considerations, and considerations for patient engagement, recruitment, and treatment. This paper outlines their recommendations.

Original language	English (US)
Pages (from-to)	1109-1116
Number of pages	8
Journal	Kidney international
Volume	97
Issue number	6
DOIs	https://doi.org/10.1016/j.kint.2020.02.029
State	Published - Jun 2020

Bibliographical note

Publisher Copyright:
© 2020 International Society of Nephrology

Keywords

Alport syndrome
CKD
ESKD
ESRD
chronic kidney disease
clinical trial design
end-stage kidney disease
end-stage renal disease
genetic disease
rare disease

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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Weinstock, B. A., Feldman, D. L., Fornoni, A., Gross, O., Kashtan, C. E., Lagas, S., Lennon, R., Miner, J. H., Rheault, M. N., Simon, J. F., Bonebrake, L., Dunleavy, M., Kumnick, P., Parziale, G., Reed, J., Weinstock, A., Gear, S., Binaso, K., Manuel, R., ... Miner, J. (2020). Clinical trial recommendations for potential Alport syndrome therapies. Kidney international, 97(6), 1109-1116. https://doi.org/10.1016/j.kint.2020.02.029

Weinstock, BA, Feldman, DL, Fornoni, A, Gross, O, Kashtan, CE, Lagas, S, Lennon, R, Miner, JH, Rheault, MN, Simon, JF, Bonebrake, L, Dunleavy, M, Kumnick, P, Parziale, G, Reed, J, Weinstock, A, Gear, S, Binaso, K, Manuel, R, Simon, J, Appel, G, Blank, M, Tang, W, Thompson, A, Torra, R, Lieberman, K, Licht, C, Dahan, K, Nozu, K, Kai, H, Ricardo, S, Pariser, A, Feldman, D, Cook, H, Chin, M, Goldsberry, A, Meyer, C, Melia, LA, Komers, R, Markels, M, Mercer, A, Prunotto, M, Morgenstern, B, Hariri, A, Modur, V, Turner, N, Kashtan, C, Rheault, M, Baigent, C, DeSacco, S, Perin, L, Barua, M, Nakanishi, K, Jarad, G & Miner, J 2020, 'Clinical trial recommendations for potential Alport syndrome therapies', Kidney international, vol. 97, no. 6, pp. 1109-1116. https://doi.org/10.1016/j.kint.2020.02.029

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abstract = "Alport syndrome is experiencing a remarkable increase in preclinical investigations. To proactively address the needs of the Alport syndrome community, as well as offer clarity for future clinical research sponsors, the Alport Syndrome Foundation hosted a workshop to generate consensus recommendations for prospective trials for conventional drugs. Opinions of key stakeholders were carefully considered, including those of the biopharmaceutical industry representatives, academic researchers, clinicians, regulatory agency representatives, and—most critically—patients with Alport syndrome. Recommendations were established for preclinical researchers, the use and selection of biomarkers, standards of care, clinical trial designs, trial eligibility criteria and outcomes, pediatric trial considerations, and considerations for patient engagement, recruitment, and treatment. This paper outlines their recommendations.",

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AU - Lagas, Sharon

AU - Lennon, Rachel

AU - Miner, Jeffrey H.

AU - Rheault, Michelle N.

AU - Simon, James F.

AU - Bonebrake, Lisa

AU - Dunleavy, Marty

AU - Kumnick, Phil

AU - Parziale, Gina

AU - Reed, Janine

AU - Weinstock, André

AU - Gear, Susie

AU - Binaso, Kristen

AU - Manuel, Raymond

AU - Simon, James

AU - Appel, Gerald

AU - Blank, Melanie

AU - Tang, Winson

AU - Thompson, Aliza

AU - Torra, Roser

AU - Lieberman, Kenneth

AU - Licht, Christoph

AU - Dahan, Karin

AU - Nozu, Kandai

AU - Kai, Hirofumi

AU - Ricardo, Sharon

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AU - Feldman, David

AU - Cook, Heather

AU - Chin, Melanie

AU - Goldsberry, Angela

AU - Meyer, Colin

AU - Melia, Lisa Anne

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AU - Markels, Michael

AU - Mercer, Alex

AU - Prunotto, Marco

AU - Morgenstern, Bruce

AU - Hariri, Ali

AU - Modur, Vijay

AU - Turner, Neil

AU - Kashtan, Clifford

AU - Rheault, Michelle

AU - Baigent, Colin

AU - DeSacco, Stephano

AU - Perin, Laura

AU - Barua, Moumita

AU - Nakanishi, Koichi

AU - Jarad, George

AU - Miner, Jeffrey

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KW - chronic kidney disease

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KW - end-stage renal disease

KW - genetic disease

KW - rare disease

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SN - 0085-2538

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EP - 1116

JO - Kidney international

JF - Kidney international

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ER -

Clinical trial recommendations for potential Alport syndrome therapies

Abstract

Bibliographical note

Keywords

UN SDGs

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