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

NCT ID: NCT06391450

Last Updated: 2025-11-24

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE4
• Total Enrollment: 44 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-06-14
• Study Completion Date: 2027-05-31

Brief Summary

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.

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.

Conditions

Autosomal Dominant Polycystic Kidney

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Empagliflozin 10 milligram (MG)

Group Type: EXPERIMENTAL

Empagliflozin 10 MG

Intervention Type: DRUG

Oral

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Oral

Interventions

Empagliflozin 10 MG

Oral

Intervention Type: DRUG

Placebo

Oral

Intervention Type: DRUG

Eligibility Criteria

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)

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Boehringer Ingelheim

INDUSTRY

Sponsor Role: collaborator

Hannover Medical School

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Roland SCHMITT, MD

Role: PRINCIPAL_INVESTIGATOR

Hannover Medical School

Locations

Medizinische Hochschule Hannover

Hanover, Lower Saxony, Germany

Countries

Germany

References

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.

Reference Type: DERIVED

PMID: 40377984 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 39675824 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 39356039 (View on PubMed)

Other Identifiers

2023-505890-3

Identifier Type: -

Identifier Source: org_study_id