e-Article
Clinical trial recommendations for potential Alport syndrome therapies
Document Type
Article
Author
Weinstock, B. André; Feldman, David L.; Fornoni, Alessia; Gross, Oliver; Kashtan, Clifford E.; Lagas, Sharon; Lennon, Rachel; Miner, Jeffrey H.; Rheault, Michelle N.; Simon, James F.; Bonebrake, Lisa; Dunleavy, Marty; Kumnick, Phil; Lagas, Sharon; Parziale, Gina; Reed, Janine; Weinstock, André; Gear, Susie; Binaso, Kristen; Manuel, Raymond; Simon, James; Appel, Gerald; Blank, Melanie; Tang, Winson; Thompson, Aliza; Torra, Roser; Lieberman, Kenneth; Licht, Christoph; Dahan, Karin; Nozu, Kandai; Kai, Hirofumi; Ricardo, Sharon; Pariser, Anne; Feldman, David; Cook, Heather; Chin, Melanie; Goldsberry, Angela; Meyer, Colin; Melia, Lisa Anne; Komers, Radko; Markels, Michael; Mercer, Alex; Prunotto, Marco; Morgenstern, Bruce; Hariri, Ali; Modur, Vijay; Turner, Neil; Gross, Oliver; Lennon, Rachel; Fornoni, Alessia; Kashtan, Clifford; Rheault, Michelle; Baigent, Colin; DeSacco, Stephano; Perin, Laura; Barua, Moumita; Nakanishi, Koichi; Jarad, George; Miner, Jeffrey
Source
Kidney International; June 2020, Vol. 97 Issue: 6 p1109-1116, 8p
Subject
Language
ISSN
00852538; 15231755
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.