Clinician-to-Clinician Update Clinician-to-Clinician Update

Transplant Services Specialty Updates

July 2017

Open clinical trials for rare kidney diseases

Several clinical trials investigating rare kidney diseases that can lead to organ failure and transplantation are now recruiting patients at University of Minnesota Health locations.

Alport syndrome. University of Minnesota serves as a site in the international phase II-III CARDINAL trial (NCT03019185) testing a new drug in patients with Alport syndrome. Alport syndrome, a rare heritable disease, causes hemorrhagic kidney disease that leads to end-stage renal disease and transplant in patients as early as in childhood. The drug bardoxolone methyl is designed to reduce the inflammation that leads to early-onset kidney failure. The trial is sponsored by Reata Pharmaceuticals, Inc.

An outcomes registry sponsored by the University of Minnesota Clinical and Translational Science Institute and managed in the Department of Pediatrics is helping researchers across the continent coordinate their work on the syndrome. The Alport Syndrome Treatments and Outcomes Registry (NCT00481130) assists investigators at multiple centers in North America in collaborating in the search for new treatments and supports efforts in enrolling patients and supporting clinical trials into this rare condition. For information, contact Sarah Lemmage at lemmage@umn.edu.

Fabry renal disease. A clinical trial intended to shed light on why patients with Fabry disease gradually lose kidney function (NCT01581424) is open to enrolling patients. A rare genetic condition, Fabry disease can lead to death from kidney failure and cardiovascular disease in patients in their 40s. The study will track patients to understand the effects of enzyme replacement therapy for alpha-galactosidase A, the only known treatment for the disorder, and to test whether the therapy works better when started in childhood rather than in adulthood.

Another clinical trial will test if podocytes, cells released in urine, can serve as a natural biomarker for the early onset of kidney failure in patients with Fabry disease (NCT02994303). The trial is currently accepting patients. For information on the trials, contact Michael Mauer, MD, at pinth001@ umn.edu (on NCT02994303) or at mauer002@umn.edu.

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