Evaluation of renal end points in nephrology trials

Chronic kidney disease (CKD) is a public health problem and is associated with a high risk of dialysis, renal transplantation, and cardiovascular disease. To date, very few interventions have been proven to be effective for slowing the progression of kidney function decline. Novel therapeutic strategies are thus desired to address the high unmet medical need of CKD.
Clinical outcome trials are required to ultimately establish a drug’s efficacy and safety. The current established trial end points in CKD are late events in the progression of CKD which lead to large and complex trials to ultimately assess the efficacy of new interventions for slowing the progression of CKD. This thesis has explored new definitions for clinical trial end points in CKD.
The findings described in this thesis indicate that the optimal end point in clinical trials of CKD progression should be a composite end point consisting of a decline in kidney function together with a clinically meaningful end point such as dialysis or kidney transplantation. With respect to the decline in renal function, this thesis helped to establish a new end point namely a 30% and 40% estimated glomerular filtration rate (eGFR) decline which is currently used in new clinical trials of CKD progression.