Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy
ConditionDuchenne Muscular Dystrophy (DMD)
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexMale
Age8-18
SponsorLarry W. Markham
About this trial
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 criteria
Qualifiers
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
Disqualifiers
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
Trial design
Treatments tested in this trial
- SGLT-2 inhibitor
- SGLT2 inhibitor
Treatment groups
10 Participants
are divided into 2 treatment groupsSponsors and collaborators
Larry W. Markham
Lead sponsor
Indiana University
Sponsor institution
National Heart, Lung, and Blood Institute (NHLBI)
Collaborator
Source ClinicalTrials.gov