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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 publication

DOI

10.1016/j.kint.2020.02.029

Type

Journal article

Journal

Kidney International

Publication Date

06/2020

Volume

97

Pages

1109 - 1116

Addresses

Alport Syndrome Foundation, Phoenix, Arizona, USA. Electronic address: aweinstock@alportsyndrome.org.

Keywords

Workshop Participants