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.

journal_name

Kidney Int

journal_title

Kidney international

authors

Weinstock BA,Feldman DL,Fornoni A,Gross O,Kashtan CE,Lagas S,Lennon R,Miner JH,Rheault MN,Simon JF,Workshop Participants.

doi

10.1016/j.kint.2020.02.029

subject

Has Abstract

pub_date

2020-06-01 00:00:00

pages

1109-1116

issue

6

eissn

0085-2538

issn

1523-1755

pii

S0085-2538(20)30352-5

journal_volume

97

pub_type

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