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, Workshop Participants, Lisa Bonebrake, Marty Dunleavy, Phil Kumnick, Sharon Lagas, 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, Oliver Gross, Rachel Lennon, Alessia Fornoni, Clifford Kashtan, Michelle Rheault, Colin Baigent, Stephano DeSacco, Laura Perin, Moumita Barua, Koichi Nakanishi, George Jarad, Jeffrey Miner, 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, Workshop Participants, Lisa Bonebrake, Marty Dunleavy, Phil Kumnick, Sharon Lagas, 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, Oliver Gross, Rachel Lennon, Alessia Fornoni, Clifford Kashtan, Michelle Rheault, Colin Baigent, Stephano DeSacco, Laura Perin, Moumita Barua, Koichi Nakanishi, George Jarad, Jeffrey Miner

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.

Copyright © 2020 International Society of Nephrology. Published by Elsevier Inc. All rights reserved.

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