Official Title: A Phase 2/3 Trial of the Efficacy and Safety of Bardoxolone Methyl in Patients With Alport Syndrome - CARDINAL (CARDINAL)

Study Sponsor: This Study is funded by Reata Pharmaceuticals, Irving, Texas.

Study Status: Active, not recruiting

Purpose & Summary: 
CARDINAL is a Phase 2 and Phase 3 clinical study evaluating the safety and effectiveness of an oral investigational drug, bardoxolone methyl for the treatment of Alport syndrome. The phase 3 portion of CARDINAL is double-blind, placebo-controlled, and is fully enrolled with 157 patients randomized on a 1:1 basis to once-daily, oral bardoxolone methyl or placebo. A dose-titration scheme will be used to reach a goal dose of 20 mg or 30 mg. All patients in the study will have the same visit and assessment schedule and will be followed for two years.

ATHENA Natural History Study

Official Title: A Natural History Study to Observe Disease Progression, Standard of Care and Investigate Biomarkers in Alport Syndrome Patients” also known as the“Athena Study”.

Study Sponsor: This study is funded by Regulus Therapeutics, San Diego, CA.

Study Status:  Recruitment ended in June 2017

Purpose & Summary:
The purpose of this study is to collect data to characterize or measure the progress of kidney disease in Alport syndrome patients over the course of 6 to 24 months.  We hope to establish the rate of change of certain renal biomarkers in subjects whose renal function is steadily declining. No treatment for Alport syndrome will be provided in this study and you may remain on any medications prescribed by your physician.

Novartis Study:

Official Title: Urine, DNA and retrospective clinical information collection from patients with Alport nephropathy.

Study Sponsor: This Study was funded by Novartis Pharmaceuticals Corporation, Cambridge, MA. 

Study Status: Recruitment ended & Completed 

Purpose & Summary: 
This biomarker study is a follow-up to CPLATFRM2201. The goal of CBASICHR0005 is to collect another urine sample, interval clinical information, and an optional DNA sample from as many of the original 80 patients as possible. This new information will transform the data obtained in PLATFRM2201 from a cross-section to a temporal profile, which will (a) further enable the identification of biomarkers predictive of faster progression, and (b) satisfy the FDA's recommendation to perform "natural history studies" in rare diseases.

Microalbuminuria Study:

Official Title: A Prospective Study of Microalbuminuria in Untreated Boys with Alport Syndrome

Study Sponsor: University of Minnesota - Clinical and Translational Science Institute and collaborated with The University of Utah.

Study Status: Recruitment ended & Completed

Purpose & Summary: 
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. Information needs to be collected 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 microalbuminuria 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.

Urinary Biomarkers Study:

Official Title: Urinary Biomarkers of the Progression of Alport Kidney Disease

Study Sponsor: University of Minnesota - Clinical and Translational Science Institute

Study Status: Recruitment ended & Completed

Purpose & Summary: 
The purpose of the study is to determine if there are certain laboratory tests that can be performed to detect substances or features in a child's urine that can be used to measure the progress of Alport kidney disease and the effects of treatment. These tests and their results could be of use to measure responses to new treatments in future clinical trials.