SGLT2 inhibitor therapy for Duchenne cardiomyopathy in kids

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 1 pharmacokinetic study evaluating empagliflozin dosing in pediatric Duchenne patients with cardiomyopathy. Researchers will measure how the drug is absorbed, distributed, metabolized, and excreted across different ages and weights to inform dosing. The trial focuses on patients with confirmed Duchenne phenotype and cardiac involvement who have relatively preserved or mildly reduced ejection fraction. Participants will undergo cardiac magnetic resonance imaging and must be able to complete study procedures, including CMR. The goal is to establish PK profiles to guide safe and effective use of SGLT2 inhibitors in this population.

Why it matters

Who can join: the full rules

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

Where it's running

• Vanderbilt University Medical Center
  Nashville, Tennessee, United States Recruiting

Study contacts

• Principal investigator: Larry W. Markham, MD — Vanderbilt University Medical Center
• Study coordinator: Larry W Markham, MD
• Email: larry.w.markham@vumc.org
• Phone: 615 322-7447

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.