Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy

NCT07172971 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: 283231R61HL180327-01
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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)

Keywords

Cardiomyopathy; Heart failure; Muscular dystrophy

Outcome Measures

Primary Outcomes (1)

• 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.

  From enrollment to 12 month analysis

Secondary Outcomes (2)

• Medication absorption in blood

  Enrollment to 12 month analysis

• Medication Excretion or Elimination from blood

  Enrollment to 12 months

Study Arms (2)

Dosing

EXPERIMENTAL

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).

Drug: SGLT-2 inhibitor

Pharmacokinetics

EXPERIMENTAL

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.

Drug: SGLT2 inhibitor

Interventions

SGLT2 inhibitor DRUG

SGLT-2 inhibitor will be given once daily by mouth

Pharmacokinetics

Eligibility Criteria

• Age: 8 Years - 18 Years
• Gender Eligibility Details: Male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Larry W. Markham: lead
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (1)

Riley Hospital at Indiana University Health

Indianapolis, Indiana, 46202, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Cardiomyopathies; Heart Failure; Muscular Dystrophies

Interventions

Sodium-Glucose Transporter 2 Inhibitors

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heart Diseases; Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Molecular Mechanisms of Pharmacological Action; Pharmacologic Actions; Chemical Actions and Uses; Hypoglycemic Agents; Physiological Effects of Drugs

Study Officials

• Larry W. Markham, MD

  Riley Children's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Larry W Markham, MD

CONTACT

317 944-8906; lwmarkha@iu.edu

Jennifer Howell, RN

CONTACT

317 944-8906; jemrich@iu.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Pediatrics

Study Record Dates

• First Submitted: August 7, 2025
• First Posted: September 15, 2025
• Study Start: February 1, 2026
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): February 1, 2028
• Last Updated: December 24, 2025

Record last verified: 2025-12

Locations

US (1)