Kidney Week

Abstract: SA-OR074

Empagliflozin Therapy in Patients with Autosomal Dominant Polycystic Kidney Disease: A Feasibility Randomized Controlled Trial

Session Information

• Late-Breaking Research Orals: Pediatrics, Genetics, and Glomerular Diseases
  October 24, 2026 | Location: Four Seasons Ballroom 2, Convention Center
  Abstract Time: 04:42 PM - 04:54 PM

Category: Genetic Diseases of the Kidneys

• 1201 Genetic Diseases of the Kidneys: Cystic (Monogenic)

Authors

• Seliger, Stephen L., University of Maryland School of Medicine, Baltimore, Maryland, United States

Stephen L. Seliger, MD, MS, FASN

• Gitomer, Berenice Y., University of Colorado Anschutz Medical Campus School of Medicine, Aurora, Colorado, United States

Berenice Y. Gitomer, PhD

• You, Zhiying, University of Colorado Anschutz Medical Campus School of Medicine, Aurora, Colorado, United States

Zhiying You, MD, PhD

• Wang, Wei, University of Colorado Anschutz Medical Campus School of Medicine, Aurora, Colorado, United States

Wei Wang, MD

• Ostrow, Anna, University of Colorado Anschutz Medical Campus School of Medicine, Aurora, Colorado, United States

Anna Ostrow, MPH

• Andrews, Emily, University of Colorado Anschutz Medical Campus School of Medicine, Aurora, Colorado, United States

Emily Andrews, MS

• Diggs, Charalett E., University of Maryland School of Medicine, Baltimore, Maryland, United States

Charalett E. Diggs, RN

• George, Diana, University of Colorado Anschutz Medical Campus School of Medicine, Aurora, Colorado, United States

Diana George

• West, Miranda, University of Colorado Anschutz Medical Campus School of Medicine, Aurora, Colorado, United States

Miranda West, MBA

• Nowak, Kristen L., University of Colorado Anschutz Medical Campus School of Medicine, Aurora, Colorado, United States

Kristen L. Nowak, PhD, MPH

• Watnick, Terry J., University of Maryland School of Medicine, Baltimore, Maryland, United States

Terry J. Watnick, MD

• Chonchol, Michel, University of Colorado Anschutz Medical Campus School of Medicine, Aurora, Colorado, United States

Michel Chonchol, MD, FASN

Background

Sodium glucose co-transporter 2 inhibitors (SGLT2i) reduce the risk of diabetic and non-diabetic kidney disease progression; however, their safety and efficacy in patients with autosomal dominant polycystic kidney disease (ADPKD) are undetermined.

Methods

In this pilot and feasibility randomized, controlled trial, we assigned 50 participants, 18-55 years of age, with ADPKD, at high risk of progression, not treated with tolvaptan, to receive empagliflozin (25mg once daily) or placebo in a 1:1 ratio for 12 months. Primary outcomes were medication safety and tolerability. Secondary outcomes were (a) change in height-adjusted-total kidney volume; (b) acute and chronic slope of eGFR, and (c) changes in aortic pulse wave velocity (aPWV), plasma copeptin levels, urine kidney injury molecule-1 (KIM-1), and patient-reported outcomes.

Results

Mean age was 42 ± 7 years, and eGFR was 70 ± 17 mL/min/1.73m². Frequency of adverse events was similar between the two groups. Urinary tract infection occurred in 2 empagliflozin-treated patients vs. none in placebo, and acute kidney injury in one placebo-treated patient. 96% of participants in each group tolerated the full dose of the study drug. Among secondary outcomes, the rate of change in htTKV was significantly greater in empagliflozin-treated participants (10.5%/year, 95% CI: 8.2%-12.7%) compared to placebo (5.1%/year, 95% CI: 3.0%-7.3%; p=0.001). Additional adjustment for between-group differences in Mayo Imaging Class did not change results. There was a faster acute decline in eGFR at 1 month among the empagliflozin (-6.2 ml/min/1.73m²) vs. placebo groups (0.1 ml/min/1.73m2; p=0.01), but no differences in eGFR change from 1 to 12 months (empagliflozin group: -0.77 ± 1.44 ml/min/1.73m²/year vs placebo group: -2.87 ± 1.96 ml/mi/1.73m²/year; p=0.4). No between-group differences were observed in changes in aPWV, copeptin, urine KIM-1, and ADPKD-Impact Score.

Conclusion

Empagliflozin at 25mg once daily was well-tolerated by ADPKD patients. Empagliflozin induced an expected acute decline in eGFR by 1 month, followed by stable eGFR. However, those treated with empagliflozin had a greater increase in kidney volume.

Funding

• NIDDK Support