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

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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.

Original language	English
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

Keywords

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

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10.1016/j.kint.2020.02.029

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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

@article{4916423b7d934922b573a8e66073cf2d,

title = "Clinical trial recommendations for potential Alport syndrome therapies",

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.",

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

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

note = "Publisher Copyright: {\textcopyright} 2020 International Society of Nephrology",

year = "2020",

month = jun,

doi = "10.1016/j.kint.2020.02.029",

language = "English",

volume = "97",

pages = "1109--1116",

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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

TY - JOUR

T1 - Clinical trial recommendations for potential Alport syndrome therapies

AU - Weinstock, B. André

AU - Feldman, David L.

AU - Fornoni, Alessia

AU - Gross, Oliver

AU - Kashtan, Clifford E.

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

AU - Pariser, Anne

AU - Feldman, David

AU - Cook, Heather

AU - Chin, Melanie

AU - Goldsberry, Angela

AU - Meyer, Colin

AU - Melia, Lisa Anne

AU - Komers, Radko

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

PY - 2020/6

Y1 - 2020/6

N2 - 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.

AB - 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.

KW - Alport syndrome

KW - CKD

KW - ESKD

KW - ESRD

KW - chronic kidney disease

KW - clinical trial design

KW - end-stage kidney disease

KW - end-stage renal disease

KW - genetic disease

KW - rare disease

UR - http://www.scopus.com/inward/record.url?scp=85084449270&partnerID=8YFLogxK

U2 - 10.1016/j.kint.2020.02.029

DO - 10.1016/j.kint.2020.02.029

M3 - Article

C2 - 32386680

AN - SCOPUS:85084449270

SN - 0085-2538

VL - 97

SP - 1109

EP - 1116

JO - Kidney International

JF - Kidney International

IS - 6

ER -

Clinical trial recommendations for potential Alport syndrome therapies

Abstract

Keywords

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