Abstract: INFO12-FR
C-Path's Polycystic Kidney Disease Outcomes Consortium (PKDOC): Advancing Tools to Accelerate PKD Treatment Development
Session Information
- Informational Posters - 2
October 23, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Genetic Diseases of the Kidneys
- No subcategory defined
Author
- Ruyle, Wendy Therese, Critical Path Institute, Tucson, Arizona, United States
Group or Team Name
- Polycystic Kidney Disease Outcomes Consortium
Description
The Polycystic Kidney Disease Outcomes Consortium (PKDOC) is a collaborative initiative led bythe Critical Path Institute (C-Path), a global nonprofit that accelerates medical innovation through data sharingand regulatory science. PKDOC unites C-Path, the PKD Foundation, academic centers, pharmaceuticalcompanies, patient groups, and international regulatory agencies including the U.S. FDA, the EMA, and HealthCanada. The consortium aims to develop regulatory-grade tools to support the evaluation and approval of newtherapies to deliver safer, more effective treatments for polycystic kidney disease.
ADPKD, a common inherited disorder, is characterized by progressive kidney cyst growth, renal enlargement,and eventual kidney failure. ARPKD is a rarer, pediatric-onset disease involving dilated collecting ducts,congenital hepatic fibrosis, and early-onset kidney dysfunction. Both diseases are difficult to treat due tovariable disease progression and a lack of validated biomarkers to guide early intervention.
To address this gap, PKDOC integrates and standardizes clinical trial and observational data using the CDISCStudy Data Tabulation Model (SDTM), enabling cross-study analyses. Its data repository includes threeADPKD longitudinal registries and multiple randomized controlled trials. These resources support thedevelopment of quantitative drug-disease trial models that inform clinical trial design and regulatory decisions.
PKDOC's work led to the regulatory qualification of total kidney volume (TKV), with or without age inclusion, asa prognostic enrichment biomarker for ADPKD, predicting a 30% decline in estimated glomerular filtration rate(eGFR). This milestone reflects PKDOC's impact in enabling more targeted, efficient clinical trials. Ongoingefforts focus on expanding biomarkers and outcome assessments to further support model-informed drugdevelopment (MIDD) in PKD, ultimately improving outcomes for patients across the disease spectrum.
Acknowledgment
Critical Path Institute is supported by the Food and Drug Administration (FDA) of the Department of Health and Human Services (HHS) and is 43% funded by the FDA/HHS, totaling $20,724,703, and 57% funded by non-government source(s), totaling $27,346,613. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.
Since 2010, PKDOC has advanced the development and regulatory qualification of tools to supportclinical trials for new PKD therapies.
Funding
- Critical Path Institute Polycystic Kidney Disease Outcomes Consortium