SGLT2 Inhibitors as a Novel Treatment for Pediatric Non-Alcoholic Fatty Liver Disease

NCT03867487 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: IRB 2023-6034
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a randomized, double-blind, placebo-controlled trial specifically designed to evaluate the preliminary feasibility, initial efficacy and safety of SGLT2 inhibitors for treating NAFLD in adolescents with obesity.

Conditions

Non-Alcoholic Fatty Liver Disease; NAFLD; Pediatric NAFLD

Outcome Measures

Primary Outcomes (1)

• Efficacy as Measured by Change in Hepatic Fat Fraction (HFF)

  HFF is measured by MRI via 1H- magnetic resonance spectroscopy (MRS). HFF will be measured with single-voxel 1H-MRS on a 3.0 T Trio whole body MRI scanner, using the software package provided by the vendor. The MR elastography measurement will consist of a phase-contrast 2D GRE scan (TR/TE = 50/25 ms, matrix 256 x 90, GRAPPA R=3, slice thickness 7mm) with motion encoding in the z-direction, and acoustic excitation at 60 Hz. Four axial slices will be acquired, each with a single breath-hold. Manual ROIs covering the liver will be drawn on the stiffness maps (in kPa units) generated by the system software. Fibrosis staging will be determined following previously published guidelines.

  Baseline to 26 weeks

Secondary Outcomes (9)

• Change in Body Measurements: Body mass index (BMI)

  Baseline to 26 weeks

• Change in Body Measurements: Body Fat %

  Baseline to 26 weeks

• Change in Body Measurements: Visceral Fat %

  Baseline to 26 weeks

• Change in Biomarkers of NAFLD: Alanine transaminase (ALT)

  Baseline to 26 weeks

• Change in Biomarkers of NAFLD: Cytokeratin (CK)-18

  Baseline to 26 weeks

Study Arms (2)

Study intervention

EXPERIMENTAL

Empagliflozin 10 mg will be taken daily

Drug: Empagliflozin 10 MG

Control arm

PLACEBO COMPARATOR

Placebo oral tablet will be taken daily

Drug: Placebo Oral Tablet

Interventions

Empagliflozin 10 MG DRUG

Participants will take a 10 mg oral tablet of empagliflozin, an orally-active inhibitor of sodium-glucose co-transporter 2 (SGLT2)

Also known as: Jardiance

Study intervention

Placebo Oral Tablet DRUG

Participants will take an identical appearing oral tablet with zero active ingredient.

Also known as: Control group

Control arm

Eligibility Criteria

• Age: 12 Years - 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• For clinical referral to screening visit:
• Age: 12 to \<20 years old
• Diagnosis of Obesity: BMI-percentile \>95th (using age- and sex- based Center for Disease Control definitions) or BMI ≥30 kg/m2
• Elevated alanine aminotransferase (ALT) more than twice the upper limit of normal by gender (≥44 U/L for girls, ≥50 U/L for boys) within 3 months prior to screening (used for historic ALT value) OR diagnosis of NAFLD from ultrasound, MRI, or participants with biopsy-proven NASH within 12 moths of screening
• History of lifestyle modification to treat obesity or NAFLD
• To be obtained at screening visit:
• Confirmation of Obesity
• Tanner stage 2,3,4 or 5;
• Normal fasting glucose tolerance (fasting blood glucose \<100 mg/dL)
• An ultrasound will be done to diagnose NAFLD if the diagnosis has not previously been made by ultrasound, MRI or biopsy
• A MRI-derived HFF ≥ 5.5%
• Willingness to adhere to lifestyle considerations throughout the study

You may not qualify if:

• ALT \> 250U/L at screening
• History of significant alcohol intake or current use
• Impaired fasting glucose (\>100 mg/dL)
• Diabetes (type 1 or 2)
• Current or recent (\<6 months prior to enrollment) use of weight loss medication(s)
• Vitamin E supplementation
• Previous bariatric surgery
• Use of metformin
• Prior use of empagliflozin
• Lower limb infection/ulceration within 3 months of screening
• Metal or magnetic implants, devices or objects inside of or on the body, which are not MRI compatible
• Structural and functional urogenital abnormalities, that predispose for urogenital infections
• Recent initiation (\<3 months prior to enrollment) of anti-hypertensive or lipid medication(s)
• Major psychiatric disorder
• Known hypothalamic or pituitary dysfunction

Sponsors & Collaborators

• Justin Ryder: lead

Study Sites (1)

Ann & Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, 60601, United States

Location

MeSH Terms

Conditions

Non-alcoholic Fatty Liver Disease

Interventions

empagliflozin; Control Groups

Condition Hierarchy (Ancestors)

Fatty Liver; Liver Diseases; Digestive System Diseases

Intervention Hierarchy (Ancestors)

Epidemiologic Research Design; Epidemiologic Methods; Investigative Techniques; Research Design; Methods

Study Officials

• Justin Ryder, PhD

  Ann & Robert H Lurie Children's Hospital of Chicago

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Farah Salim, M.S.

CONTACT

773-550-0749; SHIELD@luriechildrens.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: This is a double blind clinical trial in which the study team and the participants are blinded to whether the subject received placebo or study drug.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Vice Chair of Research for the Department of Surgery

Study Record Dates

• First Submitted: February 28, 2019
• First Posted: March 8, 2019
• Study Start (Estimated): May 1, 2027
• Primary Completion (Estimated): January 1, 2028
• Study Completion (Estimated): February 1, 2028
• Last Updated: January 20, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)