Duchenne Muscular Dystrophy (DMD) / SGLT-2 inhibitor / SGLT2 inhibitor · Phase 1 Trial

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

ClinicalTrials.gov · August 20, 2026

Preliminary Why this matters to you Expert discussion

Early or partial results. Treat as a signal, not a conclusion.

AI Summary

This is a registered Phase 1 pharmacokinetic study of empagliflozin in pediatric Duchenne muscular dystrophy patients, currently recruiting. The study aims to characterize drug absorption, distribution, metabolism, and excretion to inform dosing; no efficacy or safety results are yet available in this registry record.

What was studied

Phase 1, Interventional, Randomized, Parallel, Open label, Other purpose. Duchenne Muscular Dystrophy (DMD); Male; age from 8 Years; to 18 Years. Intervention: Dosing; Pharmacokinetics. n = 10. 1 site: United States.

Key findings

Safety

This is a registry record with no results posted; no efficacy, safety, or pharmacokinetic data are reported. Primary outcome is dosing characterization, not clinical efficacy or mortality benefit.

Who this may help

Clinicians should not draw conclusions about empagliflozin efficacy or safety in DMD cardiomyopathy from this record. Results, when available, will inform optimal pediatric dosing of off-label SGLT2 inhibitor use in this population.

Plain-language summary

This is an early-phase, uncontrolled pharmacokinetic study in a small pediatric population designed to characterize drug dosing rather than efficacy, with no results yet reported.

Study design

As stated by the source record.

MethodsPrimary outcome measure: Medication dose (assessed at From enrollment to 12 month analysis)

DesignPhase 1, Interventional, Randomized, Parallel, Open label, Other purpose

PopulationDuchenne Muscular Dystrophy (DMD); Male; age from 8 Years; to 18 Years

Sample size10

InterventionDosing; Pharmacokinetics

Setting1 site: United States

Reported figures

Quoted from the source exactly as published.

Planned sample size10

PhasePhase 1

Primary outcome measureMedication dose

Follow-up duration12 months

Clinical bottom line

Caveats

This is a registry record with no results posted; no efficacy, safety, or pharmacokinetic data are reported.
Study is in Phase 1 with very small planned enrollment (n=10); findings will require confirmation in larger studies.
No comparator or control arm described.
Primary outcome is dosing characterization, not clinical efficacy or mortality benefit.

Evidence assessment

Graded across the dimensions that decide whether you should act, each from what the source actually supports. There is no single score, and where a dimension was not assessed it says so.

Study design

limited

Phase 1, uncontrolled PK study with no comparator arm reported.

Directness

limited

Primary outcome is medication dose characterization, not clinical efficacy or safety.

Consistency

insufficient

Registry record; no results reported for comparison to other studies.

Risk of bias

insufficient

Registry record does not describe blinding, allocation concealment, or analysis methods.

Sample adequacy

limited

Estimated enrollment 10 participants; very small for PK inference across ages and weights.

What is missing. This record has no key findings. That is a gap in the analysis, not a judgement about the study.

Source abstract

Registry record from ClinicalTrials.gov (NCT07172971). This is a study registration, not published results. Lead sponsor: Vanderbilt University Medical Center. Recruitment status: RECRUITING. Phase: PHASE1. Study type: INTERVENTIONAL. Enrollment: 10 participants (ESTIMATED). Conditions: Duchenne Muscular Dystrophy (DMD). Interventions: DRUG: SGLT-2 inhibitor; DRUG: SGLT2 inhibitor. Primary outcome measures: Medication dose , From enrollment to 12 month analysis. 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.

Follow from this

Taken from the source record, never inferred. Follow any of these and new work involving them reaches your briefing.

Duchenne Muscular Dystrophy (DMD)NCT07172971SGLT-2 inhibitorVanderbilt University Medical CenterSGLT2 inhibitor