Cargando…

Clinical trial recommendations for potential Alport syndrome therapies

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 recommendatio...

Descripción completa

Detalles Bibliográficos
Autores principales: Weinstock, B. André, Feldman, David L., Fornoni, Alessia, Gross, Oliver, Kashtan, Clifford E., Lagas, Sharon, Lennon, Rachel, Miner, Jeffrey H., Rheault, Michelle N., Simon, James F.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7614298/
https://www.ncbi.nlm.nih.gov/pubmed/32386680
http://dx.doi.org/10.1016/j.kint.2020.02.029
_version_ 1783605588798734336
author Weinstock, B. André
Feldman, David L.
Fornoni, Alessia
Gross, Oliver
Kashtan, Clifford E.
Lagas, Sharon
Lennon, Rachel
Miner, Jeffrey H.
Rheault, Michelle N.
Simon, James F.
author_facet Weinstock, B. André
Feldman, David L.
Fornoni, Alessia
Gross, Oliver
Kashtan, Clifford E.
Lagas, Sharon
Lennon, Rachel
Miner, Jeffrey H.
Rheault, Michelle N.
Simon, James F.
author_sort Weinstock, B. André
collection PubMed
description 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.
format Online
Article
Text
id pubmed-7614298
institution National Center for Biotechnology Information
language English
publishDate 2020
record_format MEDLINE/PubMed
spelling pubmed-76142982023-03-09 Clinical trial recommendations for potential Alport syndrome therapies Weinstock, B. André Feldman, David L. Fornoni, Alessia Gross, Oliver Kashtan, Clifford E. Lagas, Sharon Lennon, Rachel Miner, Jeffrey H. Rheault, Michelle N. Simon, James F. Kidney Int Article 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. 2020-06-01 2020-04-06 /pmc/articles/PMC7614298/ /pubmed/32386680 http://dx.doi.org/10.1016/j.kint.2020.02.029 Text en https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the CC BY-NC-ND license (https://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle Article
Weinstock, B. André
Feldman, David L.
Fornoni, Alessia
Gross, Oliver
Kashtan, Clifford E.
Lagas, Sharon
Lennon, Rachel
Miner, Jeffrey H.
Rheault, Michelle N.
Simon, James F.
Clinical trial recommendations for potential Alport syndrome therapies
title Clinical trial recommendations for potential Alport syndrome therapies
title_full Clinical trial recommendations for potential Alport syndrome therapies
title_fullStr Clinical trial recommendations for potential Alport syndrome therapies
title_full_unstemmed Clinical trial recommendations for potential Alport syndrome therapies
title_short Clinical trial recommendations for potential Alport syndrome therapies
title_sort clinical trial recommendations for potential alport syndrome therapies
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7614298/
https://www.ncbi.nlm.nih.gov/pubmed/32386680
http://dx.doi.org/10.1016/j.kint.2020.02.029
work_keys_str_mv AT weinstockbandre clinicaltrialrecommendationsforpotentialalportsyndrometherapies
AT feldmandavidl clinicaltrialrecommendationsforpotentialalportsyndrometherapies
AT fornonialessia clinicaltrialrecommendationsforpotentialalportsyndrometherapies
AT grossoliver clinicaltrialrecommendationsforpotentialalportsyndrometherapies
AT kashtanclifforde clinicaltrialrecommendationsforpotentialalportsyndrometherapies
AT lagassharon clinicaltrialrecommendationsforpotentialalportsyndrometherapies
AT lennonrachel clinicaltrialrecommendationsforpotentialalportsyndrometherapies
AT minerjeffreyh clinicaltrialrecommendationsforpotentialalportsyndrometherapies
AT rheaultmichellen clinicaltrialrecommendationsforpotentialalportsyndrometherapies
AT simonjamesf clinicaltrialrecommendationsforpotentialalportsyndrometherapies