Recruiting
Phase 1

Empagliflozin

Sponsor:

Vanderbilt University Medical Center

Code:

NCT07172971

Conditions

Duchenne Muscular Dystrophy (DMD)

Eligibility Criteria

Sex: Male

Age: 8 - 18

Healthy Volunteers: Not accepted

Interventions

SGLT-2 inhibitor

SGLT2 inhibitor

Study Details

Brief 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.

Conditions

Duchenne Muscular Dystrophy (DMD)

Study ID

NCT07172971

Start date

Jul 1, 2026

Status verified date

Aug, 2026

Completion date

Feb 1, 2028

Anticipated

Primary completion date

Aug 1, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 8 - 18

Healthy Volunteers: Not accepted

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 Design

Enrollment

10 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Other

Interventions and Outcome Measures

Arms

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.

Interventions

SGLT-2 inhibitor

SGLT-2 inhibitor will be given once daily by mouth

SGLT2 inhibitor

SGLT-2 inhibitor will be given once daily by mouth

Primary outcome measure

  • Medication dose [ Time Frame: From enrollment to 12 month analysis ]

Central Contacts and Locations

Central contacts

Larry W Markham, MD

615 322-7447larry.w.markham@vumc.org

Jennifer B Nicotera, RN

615 322-7447janet.b.nicotera@vumc.org

Locations

Vanderbilt University Medical Center

Recruiting

Nashville, Tennessee, United States, 37232

More Information

Sponsor

Vanderbilt University Medical Center

Last update posted

Aug 24, 2026

Last verified

Aug, 2026

Keywords

  • Cardiomyopathy
  • Heart failure
  • Muscular dystrophy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-05. This information was provided to ClinicalTrials.gov by Vanderbilt University Medical Center on 2026-08-24.