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

RecruitingPhase 1

Sponsor: Vanderbilt University Medical Center

Conditions: Duchenne Muscular Dystrophy (DMD)

Interventions: SGLT-2 inhibitor, SGLT2 inhibitor

Countries: United States

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.

Eligibility overview

Sex: MALE

Age: 8 Years to 18 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria

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

Locations (1)

Nashville, Tennessee, United States