International Alport Syndrome Therapy Registry Observing Early and Late Treatments to Delay Kidney Failure and Improve Life Expectancy

What this Trial Is About

Alport syndrome is a hereditary type IV collagen disease that typically causes kidney failure early in life. This observational study investigates whether various medications can delay disease progression, postpone kidney failure, and improve life expectancy compared to untreated relatives. The study began as a European registry in 2006 and has expanded globally as Alport XXL through the International Alport Alliance, focusing on early therapy outcomes in young patients using ACE-inhibitors, Angiotensin-receptor blockers, or their combination. The study collects data on patients at different disease stages, from early microscopic hematuria to kidney failure, to assess the effectiveness of early medication initiation. Patients are grouped by treatment status and disease severity, including untreated relatives and those receiving therapy at various stages. The registry records detailed clinical data, including genetic information, urine albumin levels, kidney function, and medication doses, in a strictly observational manner without intervening in treatment decisions. Participants provide retrospective and prospective data through standardized questionnaires at baseline and follow-up visits. Data collected include kidney function tests, blood pressure, hearing loss, eye involvement, and adverse events. Researchers measure outcomes such as age at kidney failure, life expectancy, kidney function decline, and changes in urinary albumin. The study aims to observe long-term effects of therapies until 2037, supporting improved understanding of treatment timing and effectiveness in Alport syndrome.

Alport Therapy Registry - European Initiative Towards Delaying Renal Failure in Alport Syndrome