The STAGED-PKD 2-Stage Adaptive Study With a Patient Enrichment Strategy and Treatment Effect Modeling for Improved Study Design Efficiency in Patients With ADPKD

RATIONALE & OBJECTIVE: Venglustat, a glucosylceramide synthase inhibitor, inhibits cyst growth and reduces kidney failure in mouse models of autosomal dominant polycystic kidney disease (ADPKD). STAGED-PKD aims to determine the safety and efficacy of venglustat and was designed using patient enr...

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Detalles Bibliográficos
Autores principales: Perrone, Ronald D., Hariri, Ali, Minini, Pascal, Ahn, Curie, Chapman, Arlene B., Horie, Shigeo, Knebelmann, Bertrand, Mrug, Michal, Ong, Albert C.M., Pei, York P.C., Torres, Vicente E., Modur, Vijay, Gansevoort, Ronald T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9529969/
https://www.ncbi.nlm.nih.gov/pubmed/36204243
http://dx.doi.org/10.1016/j.xkme.2022.100538

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