ISGD is collaborating in a multidisciplinary effort to tackle the urgent problem of developing quantitative models to support clinical trial design and surrogate endpoint evaluation in FSGS. On December 9 and 10, we met with an international group of researchers, clinicians, biostatisticians, regulators, and patients in Washington, DC to kick off the PARASOL project (Proteinuria and GFR as Clinical Trial Endpoints in Focal Segmental Glomerulosclerosis).
As nephrologists know well, FSGS is an inherently heterogeneous condition with variable disease mechanisms and trajectories, no FDA-approved treatments, and a significant unmet need for therapeutic options. There is an urgent need to conduct randomized clinical trials and to develop safe and effective therapies for FSGS. A key component of successful trials is defining feasible endpoints that enable accelerated and full approval of test therapies. Surrogate endpoints in other glomerular diseases like IgA nephropathy and membranous nephropathy have been successfully established in recent years. However, FSGS poses unique challenges due to a lack of large randomized controlled trials and greater variability in disease measures like proteinuria and GFR over time.
PARASOL is a collaborative project that aims to define the quantitative relationships between short-term changes in biomarkers (proteinuria, GFR) and long-term outcomes in order to identify reasonably likely surrogate and traditional endpoints that will inform feasible trial designs and future regulatory pathways for FSGS. The PARASOL workgroup will achieve this in 2024 by conducting a large-scale analysis of existing data from observational cohort studies, regional or national registries, randomized controlled trials, and real-world data sets. So far, it includes over a dozen different datasets; if you have FSGS cohort data that could potentially be shared with this effort, please reach out to us.
Some of the PARASOL participants at the kickoff meeting in Washington, DC.
The project is sponsored by NephCure, the International Society of Glomerular Disease, ASN's Kidney Health Initiative (KHI), and the National Kidney Foundation. The data analysis is being led by a team from the Michigan Kidney Translational Medicine Center at the University of Michigan, where Dr. Laura Mariani and Dr. Matthias Kretzler are co-chairing the PARASOL project. The PARASOL team will hold another in-person interim meeting in June 2024 in Reykjavik, Iceland, and will ultimately present its findings in October 2024 at a public scientific workshop in Washington, DC.
With PARASOL, the hope of new FSGS therapies is on the horizon. Please contact us if you have any questions about the project or are interested in participating.