What side effects are associated with this type of intervention?

Common treatments for Polycystic Kidney Disease (PKD) include Metformin, Salsalate, and dietary interventions like caloric restriction and intermittent fasting. Metformin is generally well-tolerated but can cause gastrointestinal issues such as nausea, diarrhea, and abdominal discomfort. Salsalate, an AMPK activator, may cause gastrointestinal side effects and tinnitus. Dietary interventions are generally safe but may lead to nutritional deficiencies if not properly managed. Empagliflozin, an SGLT2 inhibitor being studied for PKD, is known to cause side effects such as urinary tract infections, genital infections, and dehydration.

What prior FDA approvals exist?

Currently, Tolvaptan is the only FDA-approved drug specifically for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD). Tolvaptan slows the progression of kidney function decline in ADPKD patients. While Empagliflozin, an SGLT2 inhibitor, is being studied for its potential benefits in ADPKD, it has not yet received FDA approval for this indication. SGLT2 inhibitors like Empagliflozin are primarily used to reduce glucose reabsorption in the kidneys and have shown benefits in diabetic nephropathy, but their efficacy and safety in ADPKD are still under investigation.

What other types of research exist?

Promising alternatives to empagliflozin for Polycystic Kidney Disease patients include metformin, which activates AMPK and downregulates mTOR and CFTR pathways, and salsalate, a direct AMPK activator. Both have shown efficacy in slowing cyst growth in animal models. Additionally, tolvaptan, a vasopressin V2 receptor antagonist, has shown benefits in reducing the rate of eGFR decline in high-risk patients. Dietary interventions like caloric restriction and intermittent fasting may also be beneficial by activating AMPK signaling.

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Sodium-glucose cotransporter 2 (SGLT2) inhibitor

Empagliflozin for Polycystic Kidney Disease

Phase 2

Recruiting

Led By Michel B Chonchol, MD

Research Sponsored by University of Colorado, Denver

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Autosomal Dominant Polycystic Kidney Disease (ADPKD) as defined by modified Pei-Ravine Criteria

Age 18-50 yrs

Must not have

Current use of tolvaptan or other V2 receptor antagonist

Current use of loop diuretic

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, check-in visit (3 months), post-testing (12 months)

Summary

This trial tests empagliflozin, a drug that helps remove excess sugar through urine, in patients with ADPKD who suffer from kidney cysts and eventual kidney failure. The drug may help reduce kidney damage and improve heart health by blocking a protein that reabsorbs sugar. Empagliflozin has been shown to slow the progression of diabetic kidney disease.

Who is the study for?

This trial is for adults aged 18-50 with Autosomal Dominant Polycystic Kidney Disease (ADPKD) and moderately reduced kidney function, but not diabetes or heart failure. Participants should have stable kidney health without recent infections and must not be pregnant, using certain diuretics, or have contraindications to MRI scans.

What is being tested?

The study tests the safety of Empagliflozin in ADPKD patients over a year. It's a double-blind trial where half the participants will receive Empagliflozin and the other half a placebo, neither group knowing which they're getting to ensure unbiased results.

What are the potential side effects?

Potential side effects of Empagliflozin may include urinary tract infections, dehydration leading to low blood pressure, ketoacidosis (a serious condition related to diabetes), genital yeast infections, increased cholesterol levels, and allergic reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with ADPKD according to specific criteria.

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I am between 18 and 50 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently taking tolvaptan or a similar medication.

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I am currently taking medication to help remove excess fluid from my body.

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I currently have a urinary tract or genital infection.

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I have heart failure with a low heart pumping rate.

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I have leg pain when walking, skin infections, or ulcers on my legs.

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I have been diagnosed with diabetes.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, check-in visit (3 months), post-testing (12 months)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, check-in visit (3 months), post-testing (12 months)

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, check-in visit (3 months), post-testing (12 months) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Adverse Events

Returned Number of Tablets

Subject Drop Out Rate

Secondary study objectives

Aortic Pulse Wave Velocity (aPWV)

Height-Adjusted Total kidney volume

Tumor Markers

+1 more

Side effects data

From 2021 Phase 3 trial • 5988 Patients • NCT03057951

15%

Cardiac failure

Hypertension

Hypotension

Urinary tract infection

Renal impairment

Hyperkalaemia

Fall

Atrial fibrillation

Diabetes mellitus

Hyperuricaemia

Anaemia

Acute kidney injury

Pneumonia

COVID-19

Death

Acute myocardial infarction

Cardiac failure congestive

Cardiac failure chronic

Cellulitis

Syncope

Chronic kidney disease

COVID-19 pneumonia

Ischaemic stroke

Coronary artery disease

Basal cell carcinoma

Chronic obstructive pulmonary disease

Myocardial infarction

Sepsis

Transient ischaemic attack

Angina unstable

Cerebrovascular accident

Angina pectoris

Ventricular tachycardia

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

10 mg Empagliflozin

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: ExperimentalExperimental Treatment1 Intervention

Empagliflozin

Group II: Placebo comparatorPlacebo Group1 Intervention

Placebo

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Empagliflozin

2017

Completed Phase 4

~181750

0 / 8Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Polycystic Kidney Disease (PKD) include AMPK activators like metformin and salsalate, which downregulate mTOR and CFTR pathways to slow cyst growth. SGLT2 inhibitors, such as empagliflozin, reduce glucose reabsorption in the kidneys, potentially benefiting kidney function and slowing disease progression. These mechanisms are crucial for PKD patients as they offer strategies to manage and slow the progression of the disease.

Sodium-glucose cotransporter 2 inhibition suppresses HIF-1α-mediated metabolic switch from lipid oxidation to glycolysis in kidney tubule cells of diabetic mice.Renal Effects of Incretin-Based Diabetes Therapies: Pre-clinical Predictions and Clinical Trial Outcomes.