Clinical trial NCT06391450: Study of Empagliflozin in Patients with Autosomal Dominant Polycystic Kidney Disease (EMPA-PKD)
Official title: Study of Empagliflozin in Patients with Autosomal Dominant Polycystic Kidney Disease (EMPA-PKD)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Autosomal Dominant Polycystic Kidney
Lead sponsor: Hannover Medical School
Sponsor class: OTHER
Collaborators: name: Boehringer Ingelheim
First posted: 2024-04-30
Enrollment: 44
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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)

Primary outcomes:
measure: Total kidney volume (TKV)
timeFrame: 18 months
Secondary outcomes:
measure: Estimated glomerular filtration rate (eGFR)
timeFrame: 18 months
Other outcomes:
Locations:
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
state: Lower Saxony
Study officials:
affiliation: Hannover Medical School
name: Roland SCHMITT, MD
role: PRINCIPAL_INVESTIGATOR
References: