Prognostic Enrichment Design in Clinical Trials for Autosomal Dominant Polycystic Kidney Disease

Clinical trials on autosomal dominant polycystic kidney disease (ADPKD) prove to be a challenging endeavor because of recruitment issues, the need for a large sample size to evaluate efficacy, and the long duration of study to prove safety, efficacy, and cost.1 Adaptive trial design has been proposed as a means to increase the efficiency of randomized clinical trials.2 Inclusion criteria for entry into a study seems easy to handle for established investigators until one encounters the many patients who prove to be ineligible because they are “just below the margins” of potential inclusion.

Abstract licence: CC BY-NC-ND

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   Modelling kidney disease with CRISPR-mutant kidney organoids derived from human pluripotent epiblast spheroids · 2015

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