In July 2026, the third Alport Research Hub Symposium took place at the Institute of Child Health in London. Organized by Kidney Research UK, this symposium brought together preclinical researchers, medical doctors, representatives from patient advocacy associations and pharmaceutical companies to discuss the latest advances in Alport syndrome research. Medpace was represented by Dr. Priscilla Smith (Medical Director), Pippa Goddard and Amelie Borie (Clinical Trial Managers), who attended the symposium to further strengthen Medpace’s expertise in this indication.
Alport syndrome is a rare genetic condition characterized by kidney impairment, hearing loss, and eye abnormalities. It is caused by mutations in the genes encoding type IV collagen. However, disease penetrance is extremely variable, with symptoms ranging from very mild hematuria to kidney failure, with some patients requiring dialysis or multiple kidney transplants throughout their lifetime.
Advances in Alport Syndrome Management
The symposium highlighted significant progress in advancing the understanding of Alport syndrome, from improving disease characterization – including distinguishing Alport risk from Alport syndrome – to the development of potential new treatments.
While angiotensin-converting enzyme (ACE) inhibitors and angiotensin receptor blockers (ARBs) remain the standard of care for these patients, it is now evident that sodium-glucose cotransporter-2 (SGLT2) inhibitors and mineralocorticoid antagonists are becoming more broadly used. However, some challenges remain, including whether these treatments should be administered to younger populations.
Shaping the Future of Clinical Trials
The need for validated surrogate endpoints in clinical trials was another key topic of discussion. One proposed approach is the introduction of more renal biopsies as endpoints for clinical trials. While biopsies are not typically required to establish a diagnosis of Alport syndrome, histologic staining can provide valuable insights into disease progression and treatment response. Discussions have occurred with the FDA, which is now supportive of including more biopsies in clinical trials.
Although often left out of clinical trial protocols, hearing loss is a major concern for patients as it drastically impacts their quality of life. Future clinical trials should include hearing assessments to not only monitor whether investigational therapies influence hearing decline, but also to evaluate a potential beneficial impact on these outcomes. Standard-of-care tests such as pure-tone audiometry, may be used to assess patients’ hearing, while newer measures, such as patient-reported outcomes, are also being developed.
The symposium reinforced the value of collaboration among researchers, clinicians, industry, and patient organizations in accelerating progress for individuals living with Alport syndrome. Medpace attendees valued the collaborative, patient-centered discussions and believe the insights gained will further strengthen the company’s ability to support current and future sponsors developing therapies for this rare disease.
An Industry Leader in Nephrology Clinical Research
With decades of experience conducting phase I-IV nephrology clinical trials, Medpace is a globally recognized leader in nephrology clinical research. Our seasoned medical in-house nephrologists, operational, and regulatory teams are fully embedded throughout your project – providing unmatched support for your program. Combined with our strong site network and KOL relationships to accelerate development and enhance patient recruitment, we’ve successfully led Phase III studies (several to regulatory approval) in key indications such as acute kidney injury, chronic kidney disease, glomerulonephritis, genetic and rare kidney diseases, and renal transplantation.
