SGLT2 Inhibitor Therapy in Cystic Fibrosis-related Diabetes

Empagliflozin in CFRD: Efficacy and Safety of SGLT2 Inhibitor Therapy in Overweight and Obese Subjects With Cystic Fibrosis-related Diabetes: a Pilot Study

This will be a placebo-controlled, randomized, double blind, crossover study with a 4 week wash out period.

Collect pilot data on the safety, tolerability, and feasibility of empagliflozin therapy in overweight/obese patients with CFRD to support a future larger randomized controlled trial.

Study Overview

• Status: Active, not recruiting
• Conditions: Cystic Fibrosis; Cystic Fibrosis-related Diabetes
• Intervention / Treatment: Other: Placebo Control; Drug: Empagliflozin

Detailed Description

The overall goal of this proposal is to collect pilot data for safety and feasibility metrics to support a future larger randomized controlled trial. In this study, we will examine the safety and tolerability of Sodium glucose co-transporter 2 inhibitor (SGLT2i) empagliflozin as an add-on therapy to insulin for overweight/obese adult patients with CFRD. We hypothesize that empagliflozin will be safe, well tolerated and will improve glycemic control in overweight/obese adult patients with CFRD.

Specific Aim 1: Collect pilot data on the safety, tolerability, and feasibility of empagliflozin therapy in overweight/obese patients with CFRD to support a future larger randomized controlled trial.

Hypothesis 1: Empagliflozin will be safe and well tolerated, with no evidence for electrolyte abnormalities, metabolic acidosis, ketosis or increased risk of severe hypoglycemia in overweight/obese adults people with CFRD.

Specific Aim 2: Collect preliminary data to evaluate the effect of empagliflozin on glycemic outcomes.

Hypothesis 2: Treatment with empagliflozin will improve glycemic control as indicated by glucose levels as measured by oral glucose tolerance test (OGTT), and on glycemic outcomes as measured by continuous glucose monitoring (CGM).

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• United States
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Adult subjects 18 years or older with CFRD and on insulin treatment
• BMI >25 kg/m2
• A participant who is capable of becoming pregnant must agree to take precautions that are effective in preventing pregnancy throughout this study. These methods could include one of the following 1. Complete abstinence from sexual intercourse; 2. Tubal ligation; 3. Use combination of two forms of contraception during the study including, oral, injectable, or implanted hormonal contraceptives, intrauterine device or barrier method.

Exclusion Criteria:

• acute pulmonary exacerbation requiring IV antibiotics or systemic glucocorticoids within 4 weeks prior to baseline study procedures
• less than 12 weeks since start of a new CFTR corrector/modulator therapy
• type 1 diabetes
• Positive GAD 65 antibody or low fasting C-peptide (below the normal reference range of the lab)
• A history of diabetic ketoacidosis
• history of recurrent genital or urinary tract infections
• pregnancy or lactation
• prior solid organ transplant
• Chronic liver disease (based on problem list in medical records or alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 times elevated above the upper limit of normal.
• chronic kidney disease (eGFR < 60mL/min/1.73 m2)
• Hypersensitivity to empagliflozin or any excipients of Jardiance
• History of eating disorder
• Non-English speakers and those unable to read in English • Diminished capacity to consent (study staff will utilize the MacCAT-CR for assessment)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: empagliflozin; randomized to study medication (empagliflozin) 10 mg taken by mouth daily for 4 weeks	Drug: Empagliflozin; Sodium glucose co-transporter 2 inhibitor; Other Names: Jardiance; SGLT2 inhibitor
Placebo Comparator: placebo control; randomized to placebo control taken by mouth daily for 4 weeks	Other: Placebo Control; Placebo Control

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Feasibility, safety, tolerability	The primary outcomes are related to feasibility, including safety and treatment tolerability. Safety will be evaluated as the proportion (in %) of participants who experience a serious adverse event during the study. Tolerability will be evaluated as the proportion (in %) of participants who discontinue their assigned treatment due to side effects.	Duration of anticipated participation for an individual participant's would be around 15 weeks.Duration anticipated to complete all study procedures, including any long-term follow-up, and data analysis is 24 months.

Collaborators and Investigators

• Sponsor: University of Minnesota

Study record dates

Study Major Dates

• Study Start (Actual): January 8, 2024
• Primary Completion (Actual): June 3, 2025
• Study Completion (Estimated): December 31, 2026

Study Registration Dates

• First Submitted: November 20, 2023
• First Submitted That Met QC Criteria: November 20, 2023
• First Posted (Actual): November 29, 2023

Study Record Updates

• Last Update Posted (Actual): January 20, 2026
• Last Update Submitted That Met QC Criteria: January 16, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Keywords: cystic fibrosis; cystic fibrosis related diabetes; CFRD; empagliflozin
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Respiratory Tract Diseases; Digestive System Diseases; Lung Diseases; Infant, Newborn, Diseases; Pancreatic Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cystic Fibrosis; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Hypoglycemic Agents; Pharmacologic Actions; Chemical Actions and Uses; Sodium-Glucose Transporter 2 Inhibitors; empagliflozin
• Other Study ID Numbers: MED-2023-31645

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No