Feasibility of Study of Empagliflozin in Patients With Autosomal Dominant Polycystic Kidney Disease

NCT05510115 | Active Not Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 22-06161R01DK131755-01A1
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

The investigator proposes a pilot randomized clinical trial to determine the safety and tolerability of empagliflozin in ADPKD patients. To achieve this, the investigator will conduct a 12-month parallel-group, randomized, double-blind, placebo-controlled trial in 50 ADPKD patients with an eGFR 30-90 mL/min/1.73m2.

Conditions

Polycystic Kidney, Autosomal Dominant

Outcome Measures

Primary Outcomes (2)

• Safety of the intervention

  Safety will be assessed by laboratory testing and recording of adverse events.

  Baseline, check-in visits (every 2 weeks the 1st month, monthly on month 2 and 3 and then every 3 months until the end of the study), post-testing (12-months).

• Tolerability and adherence of the intervention

  Tolerability will be defined by the percentage of participants who are taking 25mg/day of study drug and who report affirmatively to the question "Can you tolerate this dose of the study drug/placebo for the rest of your life?

  Baseline, check-in visits (every 2 weeks the 1st month, monthly on month 2 and 3 and then every 3 months until the end of the study), post-testing (12-months).

Secondary Outcomes (5)

• Height-Adjusted Total kidney volume

  Baseline, check-in visit (3 months), post-testing (12-months)

• Estimated glomerular filtration rate

  Baseline, 2, 4, 8 weeks, check-in visit (3 months), 6, 9, post-testing (12-months).

• Aortic Pulse Wave Velocity (aPWV)

  Baseline, check-in visit (3 months), post-testing (12 months).

• Mechanistic Biomarkers

  Baseline, check-in visit (3 months), post-testing (12 months).

• Patient Related Outcomes

  Baseline, check-in visit (3 months), post-testing (12 months)

Study Arms (2)

Experimental

EXPERIMENTAL

Empagliflozin

Drug: Empagliflozin

Placebo comparator

PLACEBO COMPARATOR

Placebo

Drug: Placebo

Interventions

Empagliflozin DRUG

Empagliflozin: The chemical name of empagliflozin is D-Glucitol,1,5-anhydro-1-C-\[4-chloro-3-\[\[4-\[\[(3S)-tetrahydro-3furanyl\]oxy\]phenyl\]methyl\]phenyl\]-, (1S). Empagliflozin is a white to yellowish, non-hygroscopic powder. It is very slightly soluble in water, sparingly soluble in methanol, slightly soluble in ethanol and acetonitrile; soluble in 50% acetonitrile/water; and practically insoluble in toluene. Empagliflozin power will be added in white and bovine origin gelatin capsules. Each capsule of empagliflozin will contain 10 mg or 25 mg of empagliflozin (free base) and the following inactive ingredients: microcrystalline cellulose magnesium, stearate, dicalcium phosphate, and silicone dioxide.

Experimental

Placebo DRUG

Placebo capsules will be matched in size and color to empagliflozin capsules. Each placebo capsule will contain the following inactive ingredients: microcrystalline cellulose magnesium, stearate, dicalcium phosphate, and silicone dioxide.

Placebo comparator

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Autosomal Dominant Polycystic Kidney Disease (ADPKD) as defined by modified Pei-Ravine Criteria;
• Age 18-55 yrs;
• Estimated Glomerular Filtration Rate (eGFR) 30-90 ml/min/1.73m2;
• Mayo imaging-based risk classification 1C, 1D, or 1E;
• Stable renal function over prior 3 months.

You may not qualify if:

• Known diabetes mellitus;
• Fasting Glucose \>120 mg/dL;
• HbA1C≥6.5%;
• Seated systolic blood pressure \<100 mm Hg;
• Seated systolic blood pressure \>160 mm Hg;
• Known heart failure with reduced ejection fraction (HFrEF);
• Current use of loop diuretic;
• Current use of tolvaptan or other V2 receptor antagonist;
• Current urinary tract or urogenital infection;
• Pregnant or lactating;
• Vascular claudication, lower extremity skin infection or ulcers;
• Contraindication to magnetic resonance imaging (e.g., severe claustrophobia, implanted ferromagnetic device).

Sponsors & Collaborators

• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): collaborator
• University of Colorado, Denver: lead
• University of Maryland: collaborator

Study Sites (1)

University of Coloardo Anschutz Medical Campus

Aurora, Colorado, 80045, United States

Location

Related Publications (3)

• 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.

  PMID: 40377984 DERIVED

• St 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.

  PMID: 39356039 DERIVED

• Hogan MC, Simmons K, Ullman L Jr, Gondal M, Dahl NK. Beyond Loss of Kidney Function: Patient Care in Autosomal Dominant Polycystic Kidney Disease. Kidney360. 2023 Dec 1;4(12):1806-1815. doi: 10.34067/KID.0000000000000296. Epub 2023 Nov 27.

  PMID: 38010035 DERIVED

MeSH Terms

Conditions

Polycystic Kidney, Autosomal Dominant

Interventions

empagliflozin

Condition Hierarchy (Ancestors)

Polycystic Kidney Diseases; Kidney Diseases, Cystic; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ciliopathies; Genetic Diseases, Inborn

Study Officials

• Michel B Chonchol, MD

  University of Colorado, Denver

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 11, 2022
• First Posted: August 22, 2022
• Study Start: November 18, 2022
• Primary Completion: March 18, 2026
• Study Completion (Estimated): August 1, 2026
• Last Updated: May 1, 2026

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: NIH applicable guidelines regarding data sharing will be followed.
• Access Criteria: NIH applicable guidelines regarding data sharing will be followed.

Locations

US (1)