A Clinical Trial of Bempedoic Acid for Polycystic Kidney Disease (BEAT-PKD)
What is Autosomal Dominant Polycystic Kidney Disease?
Autosomal dominant polycystic kidney disease, the most commonly inherited kidney disease, is characterized by the development of cysts in the kidney that impair function. Of those affected, half will progress to end-stage kidney disease by age 60, requiring dialysis or kidney transplant. Tolvaptan is effective but not tolerated by all patients. The investigators’ goal is to develop new strategies to treat autosomal dominant polycystic kidney disease that are safe and tolerable.
Quick Facts:
- The most common genetic disorder affecting 1 in 500-1000
- Characterized by slow, continuous development of cysts derived from tubular epithelial cells
- Cysts compromise renal function
- Approximately ½ of ADPKD patients reach end stage kidney disease by age 60
- Cause by mutations in polycystin-1 and -2 (PKD1 and PKD2)
- Current therapies (e.g. tolvaptan) have limitations and do not treat the root cause
About the Bempedoic Acid Therapy for Polycystic Kidney Disease Clinical Trial
Why is the Study Being Done?
To investigate whether bempedoic acid is safe and tolerable in adults with autosomal dominant polycystic kidney disease who are at high risk of worsening disease,whether or not you are already taking tolvaptan.
Clinical Impact
Successful completion of this study could have a significant clinical impact, as it will provide the necessary foundation for a larger, phase 3 study to test bempedoic acid efficacy in autosomal dominant polycystic kidney disease patients. Given the established safety record and preclinical data showing the effects of bempedoic acid in the disease process, it is expected that bempedoic acid could alter clinical practice. Moreover, identification of biomarkers for disease severity and therapeutic response would significantly improve the management of autosomal dominant polycystic kidney disease patients.
What is Bempedoic Acid?
Bempedoic acid is a medicine approved by the FDA to lower cholesterol levels. Based on pre-clinical studies, it is thought to potentially improve autosomal dominant polycystic kidney disease. It is not approved by the FDA to treat autosomal dominant polycystic kidney disease.