Abstract: INFO10-SA
Proteinuria and GFR as Clinical Trial Endpoints in FSGS (PARASOL)
Session Information
- Informational Posters - 3
October 26, 2024 | Location: Exhibit Hall, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- No subcategory defined
Authors
- Mariani, Laura H., University of Michigan, Ann Arbor, Michigan, United States
- Smith, Abigail R., Northwestern University Feinberg School of Medicine, Chicago, Illinois, United States
Group or Team Name
- PARASOL.
Description
Focal segmental glomerulosclerosis (FSGS) is a rare glomerular disease that affects both adults and pediatric patients and has a high risk of progression to kidney failure. Current treatment options have significant side effects and limited efficacy; there are currently no FDA-approved therapies to treat this disease. The FDA currently accepts complete remission as an endpoint for traditional approval in FSGS, but this is a challenging endpoint to achieve, given the scarring nature of the disease and diverse patient population. Lesser changes in proteinuria have been proposed as a basis for accelerated approval, but further investigation of the relationship between short-term changes in key biomarkers and long-term clinical outcomes is needed. Proteinuria and GFR as Clinical Trial Endpoints in Focal Segmental Glomerulosclerosis (PARASOL) is an international collaborative effort that aims to integrate observational, registry, and clinical trial data to identify reasonably likely surrogate and traditional endpoints that will inform feasible trial design and regulatory pathways for FSGS.
PARASOL is sponsored by NephCure, the International Society of Glomerular Disease, the Kidney Health Initiative, and the National Kidney Foundation. Experts convened at a kickoff meeting in December 2023, where key data sources, initial eligibility criteria and outcomes were defined. Preliminary results were reviewed at an interim follow-up meeting in June 2024 and feedback was received from key stakeholders for the next stage of the statistical analysis.
To date, 26 datasets from around the world have been identified and are in various stages of regulatory approval for data sharing. Datasets that can be integrated at a patient level will be combined for analysis; those that cannot will serve as independent validation cohorts. The limited clinical trial data available for this population will be used to test proposed endpoints.
A public scientific workshop will be held in Washington, DC in October, 2024 to present key findings, which will inform clinical trial design and regulatory pathways for the development of new therapies for FSGS. This project will also serve as a model for data sharing and the use of observational and registry data to support clinical trial endpoint development in other rare diseases with limited clinical trial data.
Funding
- NephCure