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Empagliflozin in ESKD - A Feasibility Study

Phase 1
Recruiting
Conditions
Heart Failure
Kidney Failure, Chronic
Interventions
Registration Number
NCT05687058
Lead Sponsor
University of Mississippi Medical Center
Brief Summary

The aim of this study is to learn about the safety of empagliflozin in dialysis patients as a preparation for a future large clinical trial. Empagliflozin has been approved by the Food and Drug Administration for the treatment of either type 2 diabetes, heart failure, or chronic kidney disease among patients not on dialysis. The use of empagliflozin has not been studied or approved among patients on dialysis for kidney failure because empagliflozin acts on the kidneys. However, recent experimental studies have indicated that empagliflozin may provide direct heart benefits. Some dialysis patients have substantial residual kidney function, which may be protected by empagliflozin.

Participants will be given empagliflozin for three (3) months on top of the standard of care (usual medical care for participants' condition) and will be followed up until one (1) month after the last dose. The investigators will collect information about participants' general health, obtain blood, urine, and imaging studies, check home blood pressure, monitor home blood sugar levels, and ask health-related questions to assess the safety and potential benefits of empagliflozin over four (4) months, including one month before the three (3)-month empagliflozin treatment.

Detailed Description

The incidence of end-stage kidney disease (ESKD) in the US ranks among the highest in the world. ESKD is the last phase of chronic kidney disease when the kidneys are functioning below 10-15% of normal capacity, and the patient is on dialysis. According to the US Renal Data System (USRDS), 120,834 individuals started dialysis and nearly 524,000 people were living on dialysis in 2017.1 Although advancement in technology and general medical care has led to a modest decrease in mortality among dialysis patients, their mortality rate remains extremely high at approximately 16.5 per 100 patient-years. The leading cause of death among dialysis patients is cardiovascular disease (CVD), accounting for almost 45% of deaths. Unfortunately, established therapies to prevent incident CVD in the general population, such as renin-angiotensin system inhibitors or statins, have not been shown to be effective in the dialysis population.

Sodium-glucose transporter type 2 (SGLT2) inhibitors are originally approved by FDA for the treatment for type 2 diabetes. SGLT2 is localized to the brush border of the early proximal tubule, and hence, SGLT2inhibitors induce osmotic diuresis and natriuresis but do not activate the systemic renin-angiotensin-aldosterone system.2 Recent clinical trials have consistently shown their potent renal and cardiovascular benefits in both diabetic and non-diabetic patients, which cannot be explained only by their glucose-lowering and diuretic properties. In fact, diuretics have not been shown to reduce cardiovascular mortality and such benefits of SGLT2 inhibitors are clear even among non-diabetic populations.3-5 Their renoprotective effect potentially extends to the dialysis population where residual kidney function (RKF) still plays a major role in solute clearance and volume control and has a strong association with patient outcomes.6 Patients who retain greater RKF can consume a more liberal diet and have better nutritional status, less pill burden, better blood pressure, and less interdialytic fluid gain with less frequent intradialytic hypotension, as well as greater quality of life and better survival.6 The pathophysiology underlying the cardiovascular benefits of SGLT2 inhibitors are yet to be fully elucidated, but a recent in-vitro studies indicate its direct effects on cardiomyocytes. Therefore, the investigators hypothesize that dialysis patients also benefit from SGLT2 inhibitors even if they do not have any RKF.

Efficacy and safety studies with SGLT2 inhibitors did not enroll end-stage kidney disease (ESKD) patients on dialysis. Empagliflozin, canagliflozin, and dapagliflozin can be started if the glomerular filtration rate is more than 20-25 mL/min per 1.73 m2 and can be continued until dialysis initiation or kidney transplant. From a pharmacokinetics standpoint, those SGLT2 inhibitors are extensively metabolized by glucuronidation into inactive metabolites, and are not likely to cause dose-dependent toxicity even in ESKD. Nevertheless, extra caution is necessary for their use in the setting of ESKD because SGLT2 inhibitors are not well dialyzable due to large distribution volumes and high protein binding rates.

Our overall goal is to conduct a non-randomized feasibility clinical trial of empagliflozin in the dialysis population to obtain data that will help plan future larger, sufficiently powered efficacy clinical trials. The investigators plan to enroll a total of 24 dialysis patients (18 patients on hemodialysis and 6 patients on peritoneal dialysis). After one month of the run-in period, participants will take oral empagliflozin for 3 months.

\*Hemodialysis is a form of renal replacement therapy that utilizes an external filter (dialyzer) to remove wastes from the bloodstream. Peritoneal dialysis utilizes the peritoneum as a filter to remove wastes.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
24
Inclusion Criteria
  1. age ≥18 years;
  2. diagnosis of end-stage kidney disease requiring dialysis, and
  3. ability to provide informed consent.
Exclusion Criteria
  1. systolic blood pressure <100 mm Hg (pre-dialysis for HD patients)
  2. two or more episodes of urinary tract infection within the last 12 months
  3. history of urinary retention or urinary tract obstruction
  4. liver cirrhosis
  5. advanced heart failure requiring heart assist device or inotropic support
  6. heart or liver transplant recipient
  7. major surgery performed within the last 3 months ("major" per the investigator's assessment)
  8. major surgery scheduled within 3 months after screening ("major" per the investigator's assessment)
  9. active cancer
  10. pregnant or lactating women
  11. known allergy or hypersensitivity to any SGLT2 inhibitors
  12. history of ketoacidosis during the last 12 months
  13. any other medical condition considered unappropriated by their nephrologists or a study physician (i.e., cachexia, short life expectancy, or uncontrolled personality/phycological disorder).

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Empagliflozin 25 mg thrice-weekly post-hemodialysis dosingEmpagliflozin 25 mg thrice-weekly post-hemodialysis dosingAll participants undergoing thrice-weekly hemodialysis (HD) on the Monday-Wednesday-Friday (MWF) schedule will be assigned to the empagliflozin 25 mg thrice-weekly post-hemodialysis dosing arm (Group I).
Empagliflozin 10 mg daily dosingEmpagliflozin 10 mg daily dosingPatients undergoing thrice-weekly hemodialysis (HD) on the Tuesday-Thursday-Saturday (TTS) schedule, patients on twice-weekly HD, or patients on peritoneal dialysis will receive empagliflozin 10 mg daily (Group II).
Primary Outcome Measures
NameTimeMethod
Success rate of obtaining consent from those eligible patientsDuring the enrollment process
Proportion of missing doses3 months

The investigators will do pill count using medication bottles and calculate the proportion of missing doses from each patient.

Proportion of empagliflozin discontinuation3 months

Proportion of participants who discontinue empagliflozin for any reason

Blood empagliflozin concentrations after the first dose among patients on peritoneal dialysisAt 24 hours of the first dose

The 10th blood draw for the pharmacokinetic study among patients on peritoneal dialysis

Random blood empagliflozin levelAt Month 3

Time since the last dose will be recorded.

Proportion of eligible patients out of screened patientsDuring the screening process
Dropout rate3 months

Proportion of participants who dropped out from the study for any reason

Length of time on continuous glucose monitoring3 months

Continuous glucose monitoring will be done for up to 14 days.

Completion rate of timed urine collection3 months
Peritoneal dialysis clearance of empagliflozinAt Month 3

Peritoneal dialysis fluid will be collected for 24 hours.

Secondary Outcome Measures
NameTimeMethod
Cardiovascular mortality3 months
All-cause mortality3 months
Time in very high range (%)At Month 2

Percent time for plasma glucose \>250 mg/dL

Changes from baseline to Month 3 in home diastolic blood pressure3 months
Glucose variabilityAt Month 2

Per CGM report

Number of Participants with Hepatic injury3 months

defined by an elevation of AST and/or ALT \>3-fold upper limit of normal (ULN) combined with an elevation of total bilirubin \>2-fold ULN measured, and/or marked peak aminotransferase (ALT and/or AST) elevations ≥5-fold ULN

Average glucoseAt Month 2

Per CGM report

Number of Participants with Skin and soft tissue infection3 months

per patient report

% Time of active CGMAt Month 2

Per CGM report

Time in high range (%)At Month 2

Percent time for plasma glucose \>180 to 250 mg/dL

Number of Participants with Symptomatic urinary tract infection3 months

defined by symptoms consistent with urinary tract infection plus pyuria and bacteriuria - urine culture sample has to be taken and sent to central lab for confirmation of the diagnosis

Number of Participants with Nausea3 months

per patient report

Number of Participants with Vomiting3 months

per patient report

Changes from baseline to Month 3 in left ventricular end-diastolic volume3 months

To be evaluated by transthoracic echocardiogram (optional)

Number of Participants with Ketoacidosis3 months

defined by elevated serum beta hydroxybutyrate ≥3.0 mmol/L

Number of Participants with Lower limb amputation3 months

defined by any non-trauma-related event leading to a lower limb procedure of amputation, auto-amputation or disarticulation

Number of Participants with genital infection3 months

per patient report

Glucose management indicator (estimated A1C level based on the average glucose level from CGM readings for 14 or more days)At Month 2

Per CGM report

Time in low range (%)At Month 2

Percent time for plasma glucose \>54 to 70 mg/dL

Number of Participants with Prolonged hypoglycemiaAt Month 2

Plasma glucose \<54 mg/dL for ≥2.0 hours

Changes from baseline to Month 3 in left ventricular end-systolic volume3 months

To be evaluated by transthoracic echocardiogram (optional)

Changes from baseline to Month 3 in left ventricular diastolic function3 months

To be evaluated by transthoracic echocardiogram (optional)

Number of Participants with Tinea cruris3 months

per patient report

Days on continuous glucose monitoring (CGM)At Month 2

Per CGM report

Time in target range (%)At Month 2

Percent time for plasma glucose \>70 to 180 mg/dL

Time in very low range (%)At Month 2

Percent time for plasma glucose 54 mg/dL or lower

Number of Participants with Hypoglycemia levels 2At Month 2

Plasma glucose \<54 mg/dL for ≥15 minutes

Number of Participants with Hypoglycemia levels 1At Month 2

Plasma glucose \<70 mg/dL for ≥15 minutes

Changes from baseline to Month 3 in left ventricular mass index3 months

To be evaluated by transthoracic echocardiogram (optional)

Changes from baseline to Month 3 in left ventricular ejection fraction3 months

To be evaluated by transthoracic echocardiogram (optional)

Changes from baseline to Month 3 in longitudinal global strain3 months

To be evaluated by transthoracic echocardiogram (optional)

Changes from baseline to Month 3 in circumferential global strain3 months

To be evaluated by transthoracic echocardiogram (optional)

Changes from baseline to Month 3 in Kidney Disease Quality of Life (KDQOL)-36 questionnaire3 months

Details are available at the URL below. https://www.rand.org/content/dam/rand/www/external/health/surveys_tools/kdqol/kdqol36.pdf

Changes from baseline to Month 3 in erythropoiesis stimulating drug dose3 months
Changes from baseline to Month 3 in radial global strain3 months

To be evaluated by transthoracic echocardiogram (optional)

Changes from baseline to Month 3 in home systolic blood pressure3 months
Changes from baseline to Month 3 in residual kidney function3 months

Renal urea clearance

Changes from baseline to Month 3 in hemoglobin3 months
Hospitalization/Emergency room visit rate for heart failure3 months
Changes Estimated glomerular filtration rate (GFR) from baseline to Month 33 months

Estimated by Cystatin C and beta-2 macroglobulin

Trial Locations

Locations (2)

University of Mississippi Medical Center

🇺🇸

Jackson, Mississippi, United States

Jackson Medicall Mall Dialysis Clinic

🇺🇸

Jackson, Mississippi, United States

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