Research

A Prospective Study of Microalbuminuria in Untreated Boys with Alport Syndrome

 Reasons to conduct the study

In recent years, we have learned a great deal about Alport syndrome. However, there is no proven treatment. Some research studies in animals have suggested several promising potential therapies. Potential drug treatments that might delay or prevent the development of kidney failure exist, but need to be studied carefully through clinical trials.

 The goal of the Microalbuminuria in Untreated Boys with Alport Syndrome study is to gather information about critical clinical time points such as when patients with small amounts of protein (microalbuminuria) in their urine progress to larger amounts (overt proteinuria). Large amounts of protein in the urine is often an early sign of kidney disease. 

 We need to collect this information in boys who are not taking medications known as angiotensin converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB) in order to obtain accurate data about the length of time between the onset of microalbumuria and the start of overt proteinuria. This new information will give physicians a better understanding of how to treat patients with Alport syndrome.  

 The information we gather by conducting this study will aid in planning future clinical trials because the identification of time points in disease progression, such as microalbuminuria and overt proteinuria, could reduce the time necessary to show a clinical benefit of a new treatment option.

 The study has been approved by the University of Minnesota’s Institutional Review Board.

 Study Aims

 1.       To determine the average ages of onset of microalbuminuria and overt proteinuria in untreated boys with Alport syndrome

2.       To determine the average duration of microalbuminuria before transition to overt proteinuria in untreated boys with Alport syndrome

This study does not involve treatment and is anticipated to last 3-5 years. 

 Inclusion criteria:

• Diagnosis of Alport syndrome, confirmed by skin biopsy, kidney biopsy, or molecular genetic analysis
• Diagnosis of Alport syndrome, based on presence of hematuria and confirmed diagnosis of Alport syndrome in a first-degree relative
• Male gender
• Absence of overt proteinuria
• Subject is not currently receiving treatment with an angiotensin converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB)

Exclusion Criteria:

• Female gender
• Presence of overt proteinuria
• Current treatment with ACEI or ARB
• End-stage kidney disease (on dialysis or kidney transplant recipient)

What’s involved

 Subjects recruited into the study will be asked to consent to a periodic medical record review and to submit a urine sample to the ASTOR central office every four months. As the study progresses, subjects will be asked to submit urine samples more frequently (monthly or bi-monthly).

 Enrollment

 If you would like to receive more information about the study or know of a child who meets the inclusion criteria please contact Theresa Cassidy at (612) 626 - 7632 or cassi044@umn.edu or simply refer to the Enroll Now tab and complete the form.