Abstract: INFO19-SA
C-Path's PKD Outcomes Consortium (PKDOC): Advancing Tools to Accelerate PKD Treatment Development
Session Information
- Informational Posters - 3
November 08, 2025 | Location: Exhibit Hall, 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 by the Critical Path Institute (C-Path), a global nonprofit that accelerates medical innovation through data sharing and regulatory science. PKDOC unites C-Path, the PKD Foundation, academic centers, pharmaceutical companies, patient groups, and international regulatory agencies including the U.S. FDA, the EMA, and Health Canada. The consortium aims to develop regulatory-grade tools to support the evaluation and approval of new therapies 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 to variable 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 CDISC Study Data Tabulation Model (SDTM), enabling cross-study analyses. Its data repository includes three ADPKD longitudinal registries and multiple randomized controlled trials. These resources support the development 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, as a 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. Ongoing efforts focus on expanding biomarkers and outcome assessments to further support model-informed drug development (MIDD) in PKD, ultimately improving outcomes for patients across the disease spectrum.
Since 2010, PKDOC has advanced the development and regulatory qualification of tools to support clinical trials for new PKD therapies.
Funding
- Critical Path Institute is supported by the Food and Drug Administration (FDA) of the Department of Health and Human Services (HHS) and is 56% funded by the FDA/HHS, totaling $23,740,424, and 44% funded by non-government source(s), totaling $18,881,611. 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.