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

Research output: Contribution to journalArticle

1 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 languageEnglish (US)
Pages (from-to)1109-1116
Number of pages8
JournalKidney international
Volume97
Issue number6
DOIs
StatePublished - 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

ASJC Scopus subject areas

  • Nephrology

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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