KHI Current Project

Surrogate Endpoints in IgA Nephropathy

Abstract

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.

Deliverables

Publication.

Timeline

Spring 2019.

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.

Role Name Organization
Co-ChairPatrick Nachman, MDUniversity of North Carolina Kidney Center
Co-ChairAliza Thompson, MDU.S. Food and Drug Administration
MemberJonathan Barratt, PhD, FRCPUniversity of Leicester, United Kingdom
MemberKevin J. Carroll, PhDKJC Statistics Limited
MemberDaniel Cattran, MDUniversity of Toronto, Canada
MemberJürgen Floege, MDUniversity of Aachen, Germany
MemberBarbara S. Gillespie, MD, MMS, FASNCovance Global CRO
MemberAnnamaria T. Kausz, MD, MSAllena Pharmaceuticals
MemberAlex Mercer, PhDThe George Institute for Global Health
Co-ChairVlado Perkovic, MBBS, PhD, FASNU.S. Food and Drug Administration
MemberHeather Reich, MD, CM, PhD, FRCPCUniversity of Toronto, Canada
MemberBrad H. Rovin, MDThe Ohio State University
Board of Directors LiaisonRonald J. Falk, MD, FASNUniversity of North Carolina Chapel Hill
Additional SupportSonia Boyer, MDUniversity of North Carolina
Additional SupportRupert Major, PhDUniversity of Leicester, United Kingdom
Additional SupportJudith SchimpfUniversity of Aachen, Germany
Staff LiaisonMeaghan AllainKidney Health Initiative