Duchenne Cardiomyopathy Mitigation Sglt2 Inhibitor (Cherish) Pharmacokinetic Study

Investigating Investigational Medication Dosing in Pediatric Duchenne Muscular Dystrophy

L
Larry Markham

Primary Investigator

Enrolling By Invitation
8 years - 18 years
All
Phase N/A
5 participants needed
1 Location

Brief description of study

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. We 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.

THIS STUDY IS ENROLLING BY INVITATION ONLY - Potential participants will be identified via Muscular Dystrophy Clinic patient list and clinic schedule review by a study team member who will screen patient for inclusion/exclusion criteria. Eligible patients will be approached by telephone or in private clinic room about participation in the study.

Detailed description of study

Define the SGLT2i pharmacokinetic (PK) profile in pediatric DMD patients

ExperimentalExperimentalDosing

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).
 
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.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Duchenne Muscular Dystrophy (DMD), Riley
  • Age: 8 years - 18 years
  • Gender: All

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

This study investigates the use of empagliflozin in children with Duchenne muscular dystrophy, a condition that affects muscles and can lead to heart problems. The purpose of this study is to learn how the body absorbs and processes empagliflozin, which is currently used to help with heart issues in adults, to see if it can be used for children with this condition.

Participants will receive different doses of the investigational medication based on their weight to understand how it works in their bodies. The study will check how the medicine is absorbed, distributed, and removed from the body, which helps in deciding the right amount to give to children with Duchenne muscular dystrophy.

  • Who can participate: Children aged 8 to 18 years with Duchenne muscular dystrophy confirmed by muscle biopsy or genetic test can participate. They must have certain heart imaging results and normal or mildly depressed heart function. Children with Type 1 diabetes, heart failure, or certain infections cannot participate.
  • Study details: Participants will receive either 5 mg or 10 mg of the investigational medication based on their weight. The study will observe how the medication moves through their body to determine the best dose for treating Duchenne cardiomyopathy.
Updated on 24 Feb 2026. Study ID: PCRD-IIR-CHERISH, 28323
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Interested in the study?

This study is accepting only persons who receive care at a certain clinic or doctor or who are part of an invited group. Questions about this study can be directed to the study team listed in the description or contact your doctor to see if you are eligible.

Accepting Referrals by Invitation Only