Porteinuria

On December 9, 2023, researchers, doctors, regulators, and patients from across the globe met in Washington, D.C. at the Proteinuria and GFR as Clinical Trial Endpoints in Focal Segmental Glomerulosclerosis (PARASOL) Project to discuss developing biostatistical models that could help with the design of clinical trials and aid subsequent regulatory approval of new treatments for FSGS.

FSGS is a challenging disease to study because of the diverse patient population who experience different symptoms, response to treatments and rates of progression.

Two important things doctors measure are protein in the urine (proteinuria) and kidney function (eGFR). But these can fluctuate a lot in FSGS. At this meeting, the group discussed how to account for this variability in their statistical models of disease course.

PARASOL project attendees also considered what outcomes the models should predict, like kidney failure. Linking changes in proteinuria or GFR to these outcomes could help identify new treatments more quickly.

“We recognize the urgent need for innovative additonal approaches to study FSGS. As we navigate the complexities of this challenging disease, the collaboration among global experts is key. By developing precise mathematical models, we aim to not only categorize patient groups more effectively, but also better predict outcomes like kidney failure. The entire PARASOL team is committed to providing regulatory agencies and drug companies data driven and feasible tools for better clinical trials and testing new FSGS therapies,” Josh Tarnoff, NephCure’s CEO, said.

Participants agreed that to achieve these goals, they would need to bring together research datasets from all over the world to make sure that the models work for all patients with FSGS. By reviewing data, they hope to make decisions to start building initial models.

Their models aim to provide a practical tool for drug companies to design clinical trials that could test new FSGS therapies and improve options for patients.

The PARASOL project is sponsored by NephCure, International Society of Glomerular Diseases, National Kidney Foundation, and Kidney Health Initiative.

There are plans for researchers and doctors to attend a follow up PARASOL project in June 2024, and to present project outcomes at the American Society of Nephrology project in late 2024.

If you would like to get involved further with the PARASOL project, or have additional questions, please direct all inquires to info@nephcure.org.