Researchers want to find out if bardoxolone methyl, a drug being developed by Reata Pharmaceuticals, can improve Alport Syndrome, with its disease-modifying effects in kidney function. Bardoxolone methyl is an investigational (experimental) drug that is being tested, and is not currently approved by the U.S. Food and Drug Administration (FDA), or any other regulatory agency for sale in the United States or any other country. In this study, bardoxolone methyl will be given to approximately 210 patients with Alport Syndrome from approximately 60 study centers who are between the ages of 12 and 60. The trial will consist of two cohorts (i.e. groups). Cohort 1 is considered a Phase 2 study, and will enroll up to 30 subjects. Cohort 1 will be open-label where everyone involved in the study (including you) know that the study drug being used will ONLY be bardoxolone methyl capsules. CHOP will not be participating in Cohort 1.
Cohort 2 will be a double-blind, randomized, placebo-controlled, Phase 3 study, and will enroll up to 180 subjects. This means that you will be randomly assigned (like flipping a coin) to either bardoxolone methyl, or a placebo (i.e. inactive substance/sugar pill), and that both you AND your doctor will not know which drug you are assigned to. IRB 17-013989