KHI Current Project
Surrogate Endpoints in IgA Nephropathy
IgA nephropathy, the most common glomerular disease worldwide, may progress to end stage kidney disease (ESKD) over many years. There are no approved treatments and no accepted surrogate endpoints for registration of investigational products for this disease. The goals of this project are to evaluate and identify surrogate endpoint(s) for use in clinical trials in patients with different risks of progression to ESKD. The workgroup, representing all KHI stakeholders, will critically analyze data from the literature, available patient registries and clinical trials and critically evaluate candidate surrogate endpoints according to the general criteria considered by the Food and Drug Administration.
IgA is an antibody––a protein made by the immune system to protect the body from foreign substances such as bacteria or viruses. IgA nephropathy is a kidney disease that occurs when IgA deposits build up in the kidneys, causing inflammation that damages kidney tissues and can lead to end-stage kidney disease (ESKD) or the need for dialysis.
Clinical trial endpoints measure success or failure of therapies being tested. Unfortunately, no well-defined endpoints exist for IgA nephropathy. Without clear endpoints, researchers and industry face challenges developing new therapies for the disease. This is an important barrier to innovative drug development in this field.
A KHI workgroup has been assembled to discuss clinical trials in IgA nephropathy and to outline the challenges currently facing pharmaceutical companies. The first step will be to published a white paper on drug development in IgA nephropathy. If deemed necessary, a second step of data collection and analysis will be completed to confirm any recommendations. At the conclusion, the workgroup will recommend endpoints that should be incorporated into all future IgA nephropathy clinical trials. Developing these measures will make it much more feasible for companies to invest in the research necessary to improve treatments for patients with this serious disease.
|Co-Chair||Patrick Nachman, MD||University of North Carolina Kidney Center|
|Co-Chair||Aliza Thompson, MD||U.S. Food and Drug Administration|
|Member||Jonathan Barratt, PhD, FRCP||University of Leicester, United Kingdom|
|Member||Kevin J. Carroll, PhD||KJC Statistics Limited|
|Member||Daniel Cattran, MD||University of Toronto, Canada|
|Member||Jürgen Floege, MD||University of Aachen, Germany|
|Member||Barbara S. Gillespie, MD, MMS, FASN||Covance Global CRO|
|Member||Annamaria T. Kausz, MD, MS||Allena Pharmaceuticals|
|Member||Alex Mercer, PhD||The George Institute for Global Health|
|Co-Chair||Vlado Perkovic, MBBS, PhD, FASN||U.S. Food and Drug Administration|
|Member||Heather Reich, MD, CM, PhD, FRCPC||University of Toronto, Canada|
|Member||Brad H. Rovin, MD||The Ohio State University|
|Board of Directors Liaison||Ronald J. Falk, MD, FASN||University of North Carolina Chapel Hill|
|Additional Support||Sonia Boyer, MD||University of North Carolina|
|Additional Support||Rupert Major, PhD||University of Leicester, United Kingdom|
|Additional Support||Judith Schimpf||University of Aachen, Germany|
|Staff Liaison||Meaghan Allain||Kidney Health Initiative|