Not Yet RecruitingPhase 1

Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy

United States10 participantsStarted 2026-02-01

Plain-language summary

This is a pharmacokinetic study (PK Study) to better understand empagliflozin dosing in pediatric Duchenne muscular dystrophy patients. Empagliflozin is currently used off-label in this population due to the mortality benefits seen in adult cardiomyopathy and heart failure. Investigators will perform PK studies in DMD patients of various ages and weights to better understand the PK profile (absorption, distribution, metabolism, excretion) and dosing to better treat Duchenne cardiomyopathy.

Who can participate

Age range8 Years – 18 Years

SexMALE

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Clinical phenotype of DMD confirmed with muscle biopsy or genotype * Presence of late gadolinium enhancement (LGE) imaging by CMR * Either normal or mildly depressed systolic function (LVEF\>40%) * ≥8 years old and ≤18 years old Exclusion Criteria: * Current investigational therapy that may affect cardiovascular function * Additional genetic or congenital abnormality that may affect cardiovascular function or progression * Contraindication to or inability to undergo CMR * Symptomatic heart failure * History of ketoacidosis or hypersensitivity to SGLT2i therapy * Type 1 diabetes * Renal disease or history of frequent urinary tract infections or genitourinary skin infections

What they're measuring

Medication dose

Timeframe: From enrollment to 12 month analysis

Trial details

NCT IDNCT07172971

SponsorLarry W. Markham

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-08-01

Contact for this trial

Larry W Markham, MD

317 944-8906 lwmarkha@iu.edu

View on ClinicalTrials.gov More Duchenne Muscular Dystrophy (DMD) trials