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.
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
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
Parallel
SGLT-2 inhibitor
DrugSGLT-2 inhibitor will be given once daily by mouth
SGLT2 inhibitor
DrugSGLT-2 inhibitor will be given once daily by mouth
Medication dose
DMD patients will be given different doses of medication to better understand the most appropriate dosing of medication in this rare disease population. Because age and weight can impact how a DMD patient absorbs and metabolizes medication, the study will include DMD patients of various ages and weights. This includes giving a dose of medication and measuring drug levels in blood over a 24 hour period of time.
Medication absorption in blood
DMD patients will be given different doses of medication to better understand the most appropriate dosing of medication in this rare disease population. Because age and weight can impact how a DMD patient absorbs medication, the study will include DMD patients of various ages and weights. This includes giving a dose of medication and measuring drug levels in blood over a 24 hour period of time.
Medication Excretion or Elimination from blood
DMD patients will be given different doses of medication to better understand the most appropriate dosing of medication in this rare disease population. Because age and weight can impact how a DMD patient absorbs and excretes or eliminates a medication, the study will include DMD patients of various ages and weights. This includes giving a dose of medication and measuring drug levels in blood over a 24 hour period of time.
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Vanderbilt University Medical Center
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Collaborator
