Current Members

Critical Path Institute

PKD Foundation

CDISC

Emory University

Mayo Clinic

Tufts Medical Center

University of Colorado - Denver

Polycystic Kidney Disease Outcomes Consortium

The Polycystic Kidney Disease (PKD) Outcomes Consortium is a successful collaboration between Critical Path Institute (C-Path), the PKD Foundation, Clinical Data Interchange Standards Consortium (CDISC), and four leading academic medical centers (Tufts University, University of Colorado - Denver, Emory University, and Mayo Clinic). Its mission is to promote research that will lead to the discovery of treatments for PKD and improve the lives of all it affects. The Consortium is led by C-Path and funded through a grant from the PKD Foundation and philanthropic donations. Additionally, a representative from the U.S. Food and Drug Administration (FDA) serves as an active advisor to the Consortium.

Autosomal Dominant PKD (ADPKD) is a debilitating genetic disease affecting more than 600,000 Americans and 12 million people worldwide and for which there is currently no known cure or effective treatment. Traditional endpoints of renal function only show changes very late in the course of the disease, making it difficult to assess the effectiveness of new medications. There is critical need for a biomarker that will assess disease progression at an earlier stage when patients may be more likely to respond to new therapies.

The primary goals of the PKD Consortium are to develop CDISC research data standards for PKD and to use clinical data from ADPKD patients collected over many years through patient registries and clinical trials to support the FDA-qualification of an imaging biomarker, Total Kidney Volume (TKV), as a biomarker in drug development trials. For additional details on the qualification plans, please click HERE.

Scientists will use the data collected to develop a disease progression model that will evaluate the relationship between TKV and the known complications of ADPKD, including rate of loss of kidney function, hypertension, gross hematuria, kidney stones, urinary tract infections, development of end-stage renal disease, and mortality. These analyses will be used to support the regulatory qualification of TKV as an accepted measure for assessing the progression of ADPKD in clinical trials in which new therapies are tested.

 

Executive Director: Dr. Eslie Dennis (Critical Path Institute)
Co-Director: Dr. Ronald Perrone (Tufts Medical Center)