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 journalArticlepeer-review

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

Bibliographical note

Funding Information:
BAW is a volunteer and unpaid board member of the Alport Syndrome Foundation (ASF, Scottsdale, AZ) and an employee of Teleflex Medical (Wayne, PA). DLF has served as a consultant for Ironwood Pharmaceuticals (Cambridge, MA), but the consultancy fees were donated to the National Kidney Foundation (New York, NY). AF is an inventor on pending or issued patents intended to diagnose or treat proteinuric kidney diseases, including Alport syndrome. She stands to gain royalties from their future commercialization. Relevant to this topic, AF is Chief Scientific Officer of L&F Health LLC (Miami, FL) and founder of Liponext LLC (Miami, FL). In the past 2 years, she has served as consultant for several pharmaceutical companies working in this space, including Variant Pharmaceuticals (now ZyVersa Therapeutics; Weston, FL), ONO Pharmaceuticals (Osaka, Japan), Reata Pharmaceuticals (Plano, TX), and Novartis (Basel, Switzerland). OG is the initiator and principal investigator of the EARLY PRO-TECT Alport trial, which was sponsored by the German Ministry of Education and Research (01KG1104; Bonn, Germany). He is national principal investigator for Germany for the CARDINAL trial (Reata; Plano, TX) and is the initiator and principal investigator of the European Alport registry (Göttingen, Germany). His employer, The University of Göttingen (Göttingen, Germany), has had or still has consulting relationships with Regulus Therapeutics (San Diego, CA), Reata Pharmaceuticals (Plano, TX), Retrophin (San Diego, CA), Roche (Basel, Switzerland), Novartis (Basel, Switzerland), Boehringer-Ingelheim (Ingelheim am Rhein, Germany), and Ono Pharmaceuticals (Osaka, Japan). CEK has had consulting relationships with Regulus Therapeutics (San Diego, CA), Reata Pharmaceuticals (Plano, TX), Retrophin (San Diego, CA), Boehringer-Ingelheim (Ingelheim am Rhein, Germany), Ono Pharmaceuticals (Osaka, Japan), and Daiichi Sankyo (Tokyo, Japan). He is a site investigator for the CARDINAL trial (Reata; Plano, TX) and the HERA trial (Sanofi-Genzyme; Cambridge, MA), has had research support from the Novartis Institute for Biomedical Research (Cambridge, MA) and National Institutes of Health (1R21DC017019-01A1, P. Santi, principal investigator; Bethesda, MD), and is the Executive Director of the Alport Syndrome Treatments and Outcomes Registry (ASTOR; Minneapolis, MN), which is supported by the ASF (Scottsdale, AZ) and private philanthropy. SL is cofounder, volunteer, and unpaid board member (past president) of the ASF (Phoenix, AZ). RL is supported by a Wellcome Trust Senior Fellowship award (202860/Z/16/Z; London, UK) and has consulted for Retrophin (San Diego, CA) and ONO Pharmaceuticals (Osaka, Japan) regarding AS therapies in the past 12 months. JHM has received grant support from Reneo Pharmaceuticals (San Diego, CA) and Reata Pharmaceuticals (Plano, TX) and has served as a consultant to Regulus Therapeutics (San Diego, CA) and Retrophin (San Diego, CA). MNR is a site primary investigator for clinical trials for Retrophin (San Diego, CA), Reata Pharmaceuticals (Plano, TX), and Genentech (South San Francisco, CA), and has received research funding from Goldfinch Bio (Cambridge, MA), Novartis (Basel, Switzerland), Advicenne (Nimes, France), the National Institute of Diabetes and Digestive and Kidney Diseases (Bethesda, MD), and the US Department of Defense (Alexandria, VA). JFS is a member of the speakers’ bureau for Alexion Pharmaceuticals (Boston, MA).

Keywords

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

PubMed: MeSH publication types

  • Journal Article

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