Abstract: INFO05-FR
Alport Syndrome Surrogate End Point NeTwork (ASSENT)
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
Authors
- Brubaker, Lauren, Geisinger Medical Center, Danville, Pennsylvania, United States
- Chang, Alexander R., Geisinger Medical Center, Danville, Pennsylvania, United States
- Gale, Daniel P., University of London, London, England, United Kingdom
- Bonebrake, Lisa, Alport Syndrome Foundation, Phoenix, Arizona, United States
- Inker, Lesley, Tufts University, Medford, Massachusetts, United States
- Lennon, Rachel, The University of Manchester, Manchester, England, United Kingdom
- Mercer, Alex, JAMCO Pharma Consulting, Stockholm, Sweden
- Rheault, Michelle N., University of Minnesota Twin Cities, Minneapolis, Minnesota, United States
- Simon, James F., Cleveland Clinic, Cleveland, Ohio, United States
- Mariani, Laura H., University of Michigan, Ann Arbor, Michigan, United States
- Miner, Jeffrey H., Washington University in St Louis School of Medicine, St. Louis, Missouri, United States
- Warady, Bradley A., Children's Mercy Kansas City, Kansas City, Missouri, United States
- Kirchner, H. Lester, Geisinger Medical Center, Danville, Pennsylvania, United States
- Smith, Abigail R., Northwestern University, Evanston, Illinois, United States
- Wongboonsin, Janewit, Mahidol University Faculty of Medicine Siriraj Hospital, Bangkok, Thailand
- Pitcher, David, UK Renal Registry, Bristol, England, United Kingdom
- Aberle, Makabe, Alport Syndrome Foundation, Phoenix, Arizona, United States
- Fornoni, Alessia, University of Miami, Coral Gables, Florida, United States
- Gillespie, Barbara S., Division of Nephrology, University of North Carolina and Covance, Chapel Hill, North Carolina, United States
- Diva, Ulysses, U Diva Statistical Consulting, San Diego, California, United States
Description
Alport syndrome is a rare genetic disorder of basement membranes caused by variants in the type IV collagen genes (COL4A5, COL4A3, COL4A4) that can lead to microscopic hematuria, proteinuria, glomerulopathy, kidney failure, hearing loss and eye abnormalities. Currently, there are no Food & Drug Administration (FDA)-approved therapies for Alport syndrome. A major challenge for clinical trials is that Alport syndrome is a rare disease and progression to kidney failure is extremely variable.
ASSENT is an international initiative with the goal of identifying surrogate endpoints that can be used in clinical trials to accelerate drug development. Overall objectives include evaluating associations between changes in estimated glomerular filtration rate (eGFR) and clinical events (e.g., kidney failure), and assessing proteinuria as a candidate surrogate endpoint for potential application to accelerated and traditional approval pathways.
The Alport Syndrome Foundation (ASF), a patient-led, US nonprofit organization, serves as the convener and organizer of this initiative. The ASF held an initial Research and Regulatory workshop May 8-9, 2025, followed by an ASSENT roadmap alignment meeting with the FDA on December 5, 2025 and established an ASF-FDA Public Private Partnership to advance this effort. Steering, strategic advisory, and analysis committees were convened, and Alport syndrome registries around the world have been contacted and invited to participate. A follow-up ASF-FDA meeting was held June 15, 2026 discussing preliminary analyses and confirming continued alignment with the approach.
As of June 2026, at least 18 Alport syndrome cohorts have expressed interest in participating, and the Data Coordinating Center (DCC) is working with potential partners to assess data availability and quality prior to incorporation into ASSENT. The ASSENT team continues to seek Alport syndrome cohorts that can either: share deidentified or anonymized data with the DCC to contribute to the primary discovery analytic dataset; or contribute as an independent validation dataset through local federated analysis. Aggregated results will be compared to the discovery dataset analysis and contribute to pre-specified meta-analyses.
The ASSENT framework will clarify whether short- and intermediate-term changes in proteinuria and eGFR can serve as viable surrogate endpoints in Alport syndrome, thereby enabling more timely clinical trials.
Funding
- Alport Syndrome Foundation Boheringer-Ingelheim Eloxx Pharmaceuticals Pursespring Therapeutics