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

Phase 4

Recruiting

• Conditions: Autosomal Dominant Polycystic Kidney
• Interventions: Drug: Empagliflozin 10 MG; Drug: Placebo

• Registration Number: NCT06391450
• Lead Sponsor: Hannover Medical School

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.

Study Timeline

• Study First Submitted: 2024-04-15
• Study First Submitted QC: 2024-04-29
• Study First Posted: 2024-04-30
• Study Start: 2024-06-14
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-02
• Last Update Posted: 2024-10-03
• Primary Completion: 2027-05
• Study Completion: 2027-05

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 44

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)

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Empagliflozin 10 milligram (MG)	Empagliflozin 10 MG	-
Placebo	Placebo	-

Primary Outcome Measures

Name	Time	Method
Total kidney volume (TKV)	18 months	Relative change from baseline TKV (percent/year) to month 18 of treatment.

Secondary Outcome Measures

Name	Time	Method
Estimated glomerular filtration rate (eGFR)	18 months	Change in the eGFR from baseline to month 18 of treatment

Trial Locations

Locations (1)

Medizinische Hochschule Hannover; 🇩🇪 Hannover, Lower Saxony, Germany