First Patient Enrolled in Phase 3 Trial for Autosomal Dominant Polycystic Kidney Disease

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According to a story from, the biopharmaceutical company Reata Pharmaceuticals, Inc. has recently announced that the company has officially enrolled its first patient in its phase 3 clinical trial. This trial will test the drub bardoxolone as a treatment for the rare illness autosomal dominant polycystic kidney disease (ADPKD). Reata is focused on the development of novel treatments for life-threatening, serious diseases. Bardoxolone is an experimental drug and is currently not approved to treat any disease.

About Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Autosomal dominant polycystic kidney disease, though generally rare, is the most prevalent, potentially fatal, monogenic disorder in humans. It is characterized by the appearance of cysts in the kidneys and other internal organs. The disease is caused by a mutation affecting either the PKD1 or PKD2 gene. The disease can inflict a variety of symptoms and can worsen over time; these symptoms include acute loin pain, cysts in the liver or other organs, berry aneurysm, hematuria (bloody urine), and high blood pressure. Anemia and uremia often appear in the later stages as kidney function begins to decline. About half of patients will experience kidney failure, requiring dialysis or a transplant. There are some treatments that can help slow disease progression, such as aquaretic drugs, cyst aspiration, cyst decortication, and surgical removal of the kidney. Pain management is also an important part of disease treatment. Common causes of death include cardiovascular disease, infections, or a ruptured aneurysm. To learn more about autosomal dominant polycystic kidney disease, click here.

About The Clinical Trial

This clinical trial will include around 300 patients with the disease that will range in age from 18-70 years. The phase 3 trial will include a treatment period of 48 weeks followed by a 4 week withdrawal period. The primary endpoint of the study will be changes to the estimated glomerular filtration rate (eGFR). This trial follows encouraging findings in the phase 2 study in which bardoxolone was able to cause significant improvements in kidney function for autosomal dominant polycystic kidney disease patients. 

Bardoxolone is an experimental drug designed to activate transcription factor Nrf2. It has earned orphan status in the US and EU for Alport syndrome and in the US for pulmonary arterial hypertension. 

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