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A randomized phase 1b cross-over study of safety of low-dose pioglitazone for treatment of autosomal dominant polycystic kidney disease

Clinical Kidney Journal Jan 31, 2021

Blazer-Yost BL, Bacallao RL, Erickson BJ, et al. - Researchers performed this small pilot trial in individuals with autosomal dominant polycystic kidney disease (ADPKD) but without diabetes, to assess whether it is safe to treat PKD with a low dose (15 mg) of the peroxisome proliferator-activated receptor-γ (PPAR-γ) agonist pioglitazone vs placebo, with each treatment administered for 1 year. In this Phase 1b cross-over study, recognized side effects of PPAR-γ agonist therapy, including fluid retention and edema, were monitored. Treatment with pioglitazone, vs placebo, led to a significant reduction in total body water as evaluated by bioimpedance analysis and no disparities in episodes of heart failure, clinical edema or alteration in echocardiography. Findings showed that pioglitazone 15 mg was as safe as placebo as a treatment choice for these patients.

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