Optimizing clinical trial design: in chronic kidney disease

Patients with chronic kidney disease (CKD) are at increased risk of kidney failure and cardiovascular complications. There is still a large unmet need for novel treatment options that reduce the risk of kidney disease progression. Clinical trials are necessary to assess the efficacy and safety of new treatment options for these patients. However, the conduct of clinical trials in CKD has proven to be challenging. End stage kidney disease is used as a clinical endpoint in clinical trials of CKD progression. However, this is a late manifestation of CKD which requires large trials of long duration to assess drug efficacy and safety. Subsequently, clinical trials are becoming more and more complex and expensive. Optimization and innovations in clinical trial design are thus needed to bring new therapies to clinical practice.
This aim of this thesis was therefore to identify and develop strategies to improve clinical trial design in CKD. We have primarily focused on the efficient use of albuminuria and estimated glomerular filtration rate (eGFR) as inclusion and enrichment criteria, and as surrogate endpoints for clinical trials. Finally, we focused on the use of novel biomarkers as enrichment criteria to identify high risk populations for new clinical trials.