Autosomal Dominant Polycystic KidneyPhase 4EMPA-PKDNCT06391450
Study of Empagliflozin in Patients With Autosomal Dominant Polycystic Kidney Disease (EMPA-PKD)
A Phase 4 study testing Empagliflozin 10 MG in people with Autosomal Dominant Polycystic Kidney, run by Hannover Medical School at 1 site in Germany.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autosomal Dominant Polycystic Kidney
- What is being tested?
- Empagliflozin 10 MG
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 44 participants
- Where
- 1 siteGermany
- Study dates
- 14 Jun 2024to May 2027
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female* patients ≥ 18 of age
- 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
- ADPKD diagnosed by unified criteria (combination of family history, ultrasound, MRI/CT, genotyping as needed)
- Mayo Class I C, D, E
- 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.
- Willing and able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures
- Evidence of signed written informed consent.
Who cannot join
- Kidney or any other solid organ transplant recipient
- Currently receiving SGLT2-inhibitor
- Concomitant treatment with steroids or any other immunosuppressive agent
- Hypersensitivity to the active principle (Empagliflozin) or any of the excipients (e.g. lactose)
- Ketoacidosis (laboratory based) in the past 5 years
- Type 1 diabetes mellitus
Show all 12Show fewer
- Ongoing urinary tract- or genital infections
- Inability to fully understand the possible risks and benefits related to study participation
- Inability to undergo MRI exam (e.g. implanted medical devices)
- Women who are pregnant or breastfeeding
- Unwilling to practice acceptable methods of birth control during study participation
- Participation in another clinical trial (other investigational drugs or devices at the time of enrolment or within 30 days prior to enrolment)
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the 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
- Being tested
- Empagliflozin 10 MG, Placebo
- Medical subject headings
- Polycystic Kidney, Autosomal Dominant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Empagliflozin 10 MGDrug
Oral
PlaceboDrug
Oral
| Study group | Role | What they receive |
|---|---|---|
| Empagliflozin 10 milligram (MG) | Experimental | Not described Receives: Drug: Empagliflozin 10 MG |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total kidney volume (TKV)
Relative change from baseline TKV (percent/year) to month 18 of treatment.
Measured: 18 months
Secondary outcomes (1)
Estimated glomerular filtration rate (eGFR)
Change in the eGFR from baseline to month 18 of treatment
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Apr 2024
- Study start14 Jun 2024
- Primary completionMay 2027 (estimated)
- Study completionMay 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Autosomal Dominant Polycystic KidneyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 44 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hannover Medical School
- Sponsor type
- University, hospital or other
- Collaborators
- Boehringer Ingelheim
- Responsible party
- Sponsor
- Investigators
- Roland SCHMITT, MD, Principal investigator, Hannover Medical School
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06391450
- Sponsor's protocol ID
- 2023-505890-3
- Other IDs
- 2023
- Acronym
- EMPA-PKD
- Status last verified
- Nov 2025
- First submitted
- 15 Apr 2024
- Last updated
- 24 Nov 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 40377984Bahlmann-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.
PubMed 39675824St 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.
PubMed 39356039
Study at a glance
From the registry record, last updated by the sponsor on 24 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Empagliflozin in Patients With Autosomal Dominant Polycystic Kidney Disease (EMPA-PKD)
- ClinicalTrials.gov ID
- NCT06391450
- Acronym / sponsor ID
- EMPA-PKD / 2023-505890-3
- Conditions
- Autosomal Dominant Polycystic Kidney
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 44 participants (estimated)
- Lead sponsor
- Hannover Medical SchoolUniversity, hospital or other
- Collaborators
- Boehringer Ingelheim
- Study start
- 14 Jun 2024 (actual)
- Primary completion
- May 2027 (estimated)
- Study completion
- May 2027 (estimated)
- First posted
- 30 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Empagliflozin 10 milligram (MG) | Experimental | Drug: Empagliflozin 10 MG |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Male and female* patients ≥ 18 of age
- 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
- ADPKD diagnosed by unified criteria (combination of family history, ultrasound, MRI/CT, genotyping as needed)
- Mayo Class I C, D, E
- 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.
Key exclusion criteria (12 listed)
- Kidney or any other solid organ transplant recipient
- Currently receiving SGLT2-inhibitor
- Concomitant treatment with steroids or any other immunosuppressive agent
- Hypersensitivity to the active principle (Empagliflozin) or any of the excipients (e.g. lactose)
- Ketoacidosis (laboratory based) in the past 5 years
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Empagliflozin 10 MGOral | Drug | Empagliflozin 10 milligram (MG) |
| PlaceboOral | Drug | Placebo |
Primary outcome
Total kidney volume (TKV)
Time frame: 18 months
Relative change from baseline TKV (percent/year) to month 18 of treatment.
1 secondary outcome
Estimated glomerular filtration rate (eGFR)
Time frame: 18 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Nov 2025.
- Derived from this study: PubMed 40377984Eswarappa 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.
- Derived from this study: PubMed 39675824Bahlmann-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.
- Derived from this study: PubMed 39356039St 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Roland SCHMITT, MD principal investigator
Hannover Medical School
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Empagliflozin 10 MG in people with Autosomal Dominant Polycystic Kidney, run by Hannover Medical School at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 24 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Total kidney volume (TKV) (time frame: 18 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06391450, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Nov 2025.