Study of Empagliflozin in Patients With Autosomal Dominant Polycystic Kidney Disease (EMPA-PKD) (EMPA-PKD)

The EMPA-PKD trial is assessing the safety of empagliflozin in patients with rapid progressive ADPKD with and without concomitant tolvaptan use by monitoring kidney growth and the rate of loss of kidney function.

Study Overview

• Status: Active, not recruiting
• Conditions: Autosomal Dominant Polycystic Kidney
• Intervention / Treatment: Drug: Placebo; Drug: Empagliflozin 10 MG

Detailed Description

In autosomal dominant polycystic kidney disease (ADPKD) formation of cysts in the kidneys causes destruction of functional parenchyma and loss of kidney function, which may progress to end-stage kidney disease. Tolvaptan is the only drug specifically approved for slowing down the progression of ADPKD. Sodium glucose cotransporter 2 inhibitors (SGLT2i) might provide additional benefits but there is currently no information on safety and outcome effects of SGLT2i in patients with ADPKD, as these patients were excluded in the landmark SGLT2i trials. In an investigator-initiated, double-blind, mono-center, placebo-controlled, randomized clinical trial the EMPA-PKD study is assessing the safety of empagliflozin in patients with rapid progressive ADPKD with and without concomitant tolvaptan use by monitoring kidney growth and the rate of loss of kidney function.

44 participants will be randomly allocated (1:1) to receive a daily dose of either empagliflozin (10 mg/day) or placebo for 18 months. Patients will be stratified according to concomitant tolvaptan use. The primary endpoint is progression of cystic kidney growth by monitoring MRI-based changes in total kidney volume and the secondary endpoint is exploring changes in glomerular filtration rate. Additional endpoints include adverse events and changes in copeptin levels, albuminuria and blood pressure.

• Study Type: Interventional
• Enrollment (Estimated): 44
• Phase: Phase 4

Contacts and Locations

Study Locations

• Germany
  • Lower Saxony
    • Hanover, Lower Saxony, Germany, 30625
      • Medizinische Hochschule Hannover

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Male and female* patients ≥ 18 of age
2. Screening eGFR ≥ 25 and ≤ 90 mL/min/1.73 m2 if age ≥ 18 and ≤50 years or Screening eGFR ≥ 25 and ≤ 65 mL/min/1.73 m2 if age > 50 years
3. ADPKD diagnosed by unified criteria (combination of family history, ultrasound, MRI/CT, genotyping as needed)
4. Mayo Class I C, D, E
5. Patients with and without tolvaptan use will be included. Patients with tolvaptan use will be included if tolvaptan has been taken for ≥ 3 months at study entry.
6. Willing and able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures
7. Evidence of signed written informed consent.

Exclusion criteria:

1. Kidney or any other solid organ transplant recipient
2. Currently receiving SGLT2-inhibitor
3. Concomitant treatment with steroids or any other immunosuppressive agent
4. Hypersensitivity to the active principle (Empagliflozin) or any of the excipients (e.g. lactose)
5. Ketoacidosis (laboratory based) in the past 5 years
6. Type 1 diabetes mellitus
7. Ongoing urinary tract- or genital infections
8. Inability to fully understand the possible risks and benefits related to study participation
9. Inability to undergo MRI exam (e.g. implanted medical devices)
10. Women who are pregnant or breastfeeding
11. Unwilling to practice acceptable methods of birth control during study participation
12. Participation in another clinical trial (other investigational drugs or devices at the time of enrolment or within 30 days prior to enrolment)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Oral
Experimental: Empagliflozin 10 milligram (MG)	Drug: Empagliflozin 10 MG; Oral

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Total kidney volume (TKV)	Relative change from baseline TKV (percent/year) to month 18 of treatment.	18 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Estimated glomerular filtration rate (eGFR)	Change in the eGFR from baseline to month 18 of treatment	18 months

Collaborators and Investigators

• Sponsor: Hannover Medical School
• Collaborators: Boehringer Ingelheim
• Investigators: Principal Investigator: Roland SCHMITT, MD, Hannover Medical School

Publications and helpful links

General Publications

• St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3.
• Eswarappa M, Madden E, Shlipak MG, Cui X, Mrug M, Estrella MM, Park M. Sodium-Glucose Cotransporter-2 Inhibitor Therapy and Longitudinal Changes in Kidney Function among Veterans with Autosomal Dominant Polycystic Kidney Disease. Clin J Am Soc Nephrol. 2025 May 16;20(7):940-949. doi: 10.2215/CJN.0000000725.
• Bahlmann-Kroll E, Hackl S, Kramer S, Wulfmeyer VC, Glandorf J, Kaufeld J, Koch A, Hartung D, Schmidt BMW, Schmidt-Ott K, Schmitt R. Empagliflozin in patients with autosomal dominant polycystic kidney disease (EMPA-PKD): study protocol for a randomised controlled trial. BMJ Open. 2024 Dec 15;14(12):e088317. doi: 10.1136/bmjopen-2024-088317.

Study record dates

Study Major Dates

• Study Start (Actual): June 14, 2024
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): May 1, 2027

Study Registration Dates

• First Submitted: April 15, 2024
• First Submitted That Met QC Criteria: April 29, 2024
• First Posted (Actual): April 30, 2024

Study Record Updates

• Last Update Posted (Actual): November 24, 2025
• Last Update Submitted That Met QC Criteria: November 19, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Ciliopathies; Urogenital Diseases; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Genetic Diseases, Inborn; Congenital Abnormalities; Abnormalities, Multiple; Kidney Diseases, Cystic; Polycystic Kidney Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Polycystic Kidney, Autosomal Dominant; Hypoglycemic Agents; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Sodium-Glucose Transporter 2 Inhibitors; empagliflozin
• Other Study ID Numbers: 2023-505890-3

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No