duCHennE caRdiomyopathy mItigation Sglt2 inHibitor
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
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.
Eligibility
Participation Requirements
Sex: Male
Minimum Age: 8
Maximum Age: 18
Healthy Volunteers: f
View:
• 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
Locations
United States
Tennessee
Vanderbilt University Medical Center
RECRUITING
Nashville
Contact Information
Primary
Larry W Markham, MD
larry.w.markham@vumc.org
615 322-7447
Backup
Jennifer B Nicotera, RN
janet.b.nicotera@vumc.org
615 322-7447
Time Frame
Start Date: 2026-07-01
Estimated Completion Date: 2028-02-01
Participants
Target number of participants: 10
Treatments
Experimental: Dosing
At the initial stage, investigators will allocate 3 subjects to 5 mg and 3 subjects 10 mg depending on their body weight (i.e., 5 mg for children with weight les than 40 kg, 10 mg for those with greater than 40 kg).
Experimental: Pharmacokinetics
In the second stage, based on the Pharmacokinetics (PK) analysis results from the initial 6 subjects divided in 5 mg and 10 mg dose groups, the next dose will be determined, for which the remaining 4 subjects will be allocated. The next dose decision will be made based on the target drug concentration levels along with the estimated PK parameters (e.g., the area under the drug concentration time curve and the maximum concentration), which correspond to adults PK and drug levels.
Related Therapeutic Areas
Sponsors
Leads: Vanderbilt University Medical Center
Collaborators: National Heart, Lung, and Blood Institute (NHLBI)