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RecruitingPhase 1

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

duCHennE caRdiomyopathy mItigation Sglt2 inHibitor

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
8 Years to 18 Years
Sex
Male only
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study looks at how the body takes in, uses, and removes empagliflozin to better understand dosing. It is for children and teens ages 8 to 18 with Duchenne muscular dystrophy (DMD) and signs of heart muscle damage.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Have signs of DMD confirmed by a muscle tissue test or genetic test.
  • Have signs of heart muscle damage on a heart MRI scan.
  • Have normal or mildly reduced heart pumping strength, with a left ventricular ejection fraction (LVEF) above 40%.
  • Be 8 to 18 years old.

Who can't join

  • Currently receive an experimental treatment that may affect the heart or blood vessels.
  • Have another genetic condition or a condition present at birth that may affect the heart or blood vessels or how the disease worsens.
  • Cannot safely have a heart MRI scan or are unable to have one.
  • Have heart failure that causes symptoms.
  • Have had ketoacidosis, a dangerous buildup of acids in the blood, or an allergic reaction to SGLT2 inhibitor drugs.
  • Have type 1 diabetes.
  • Have kidney disease, frequent urinary tract infections, or frequent skin infections around the genitals or urinary organs.

Simplified for readability. The official criteria below are what the study team uses.

Official 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

Study sites (1)

TN