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.

• Authors: 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
• Journal: Kidney international (Kidney Int) Vol. 97 Issue 6 Pg. 1109-1116 (06 2020) ISSN: 1523-1755 [Electronic] United States
• PMID: 32386680 (Publication Type: Journal Article)
• Copyright: Copyright © 2020 International Society of Nephrology. Published by Elsevier Inc. All rights reserved.

Chemical References

• Biomarkers

Topics

• Biomarkers
• Child
• Humans
• Kidney Transplantation
• Nephritis, Hereditary (diagnosis, drug therapy)
• Prospective Studies