Surrogate Endpoints in IgA Nephropathy

Overview

IgA nephropathy, the most common glomerular disease worldwide, may progress to kidney failure over many years. In the past, there were no approved treatments or accepted surrogate endpoints for registration of investigational products for this disease. Recently, there has been a dramatic increase in drug development for IgAN; however, clinical trials are largely being conducted in adults. At this time, it is not clear whether the efficacy and safety of these treatments should also be assessed in children with IgAN, and, if so, how best to design studies to obtain the data needed to support use of novel therapies in this population.

In 2016, the Kidney Health Initiative convened a project to address surrogate endpoints in adults, followed later by a second project focused on clinical trials in pediatric patients.

• Surrogate Endpoints in IgA Nephropathy

  The goals of this project were to evaluate and identify surrogate endpoints for use in clinical trials in patients with different risks of progression to kidney failure. The Kidney Health Initiative working group analyzed data from literature, available patient registries, and clinical trials to critically evaluate candidate surrogate endpoints according to the general criteria considered by the U.S. Food and Drug Administration.

  Deliverable

  Proteinuria Reduction as a Surrogate End Point in Trials of IgA Nephropathy

• Clinical Trials in Pediatric Patients with IgA Nephropathy (IgAN)

  A Kidney Health Initiative working group engaged subject matter experts to assess the current literature and trial landscape to formulate a community consensus on a path forward for developing therapies for children with IgAN. Additionally, the working group engaged stakeholders who have clinical trial or registry data that can be used to inform natural history of disease research or identify biomarkers candidates and outcome measurements that advance therapies for children living with IgAN. A summary of findings and recommendations is currently in development.