duCHennE caRdiomyopathy mItigation Sglt2 inHibitor
Larry W. Markham
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.
Description
Duchenne muscular dystrophy (DMD) is an X-linked skeletal and cardiac myopathy resulting from a defect in the gene coding for dystrophin. DMD myopathy leads to loss of ambulation, respiratory failure, cardiomyopathy (CM), and premature death. There is no cure, and while gene therapies now have approval, their cardiac effects are largely unstudied. Supportive care advances have decreased death from respiratory failure but have simultaneously unmasked the fully penetrant CM phenotype, which is now the leading cause of death in DMD. Investigators have documented that DMD CM progresses from norma…
Eligibility
- Age range
- 8–18 years
- Sex
- Male
- Healthy volunteers
- No
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…
Interventions
- DrugSGLT-2 inhibitor
SGLT-2 inhibitor will be given once daily by mouth
- DrugSGLT2 inhibitor
SGLT-2 inhibitor will be given once daily by mouth
Location
- Riley Hospital at Indiana University HealthIndianapolis, Indiana