The Results of This Phase 3 Alport Syndrome Trial Look Promising

According to a story from Globe Newswire, the biopharmaceutical company Reata Pharmaceuticals, Inc., recently announced the results of a phase 3 clinical trial that was testing its experimental product candidate bardoxolone methyl as a treatment for patients with chronic kidney disease caused by Alport syndrome, a rare genetic disorder. The results of this trial were positive and the drug was able to satisfy all primary and secondary study endpoints.

About Alport Syndrome

Alport syndrome is a genetic disorder which is characterized by end stage kidney disease, hearing loss, and glomerulonephritis, a type of kidney disease in which the glomeruli or small blood vessels in the kidneys become inflamed. Alport syndrome is a heritable disease, and is caused by mutations of the COL4A3, COL4A4, and COL4A5 genes. The symptoms of the disorder include blood and protein in the urine, leiomyomatosis (coughing, vomiting, bronchitis, stomach pain, dysphagia), various eye conditions, and, rarely, aortic dissection. Since kidney failure inevitably develops eventually, treatment is similar to that of chronic kidney disease, and patients will eventually require dialysis or a kidney transplant. ACE inhibitors appear to slow the decline of kidney function. There is a serious need for more effective treatments for this disorder. To learn more about Alport syndrome, click here.

Trial Results

The phase 3 trial included a total of 157 patients; approximately 15 percent were children. The primary endpoint in this study was estimated glomerular filtration rate (eGFR). This endpoint is an important measurement of kidney function. The secondary endpoint used in this study was the change of retained eGFR after four weeks of withdrawal of bardoxolone methyl after the 48 week treatment period. Following the conclusion of treatment, the drug was able to demonstrate statistically meaningful improvements in these endpoints when compared to treatment with a placebo. 

Nine of the patients receiving the drug during the trial ultimately were forced to stop treatment as a result of adverse effects. Primary side effects of bardoxolone methyl in the trial included an increase in aminotransferases and muscle spasms. These side effects were previously documented in earlier trials.

The results from this trial suggest that the treatment could be a useful therapy for this patient population.

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