Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age

This study aims at exploring the use of empagliflozin in children and adolescents 6-18 years old with Duchenne muscular distrophy (DMD) - associated cardiomyopathy. This molecule is effective in reducing hospitalizations and mortality in adults with heart failure and is used in adolescents with type 2 diabetes mellitus, but little is known on children and adolescents with heart failure. Particularly, the best dose to use in this population is currently unknown. This trial aims to: 1. define a dose rationale for this indication and age group (pharmacokinetic study), 2. assess and monitor safety, 3. assess ease-of-swallow, 4. explore middle-term (3-6 months) efficacy and efficacy markers. Participants will be asked to attend 5 study visits over 6 months, and one end-study visit 2-12 weeks thereafter. Visit 1 will entail an 8h day-hospital stay, while Visits 2, 3, 4 and 5, as well as the end-study visit, will be outpatient clinics (approximately 2h). Participants will be asked to take the studied drug once daily during the 6 months of the study period. No comparison group is foreseen for this study.

NCT Number	NCT06643442
Lead Sponsor	Sebastiano Lava Collaborators: Great Ormond Street Hospital for Children NHS Foundation Trust, University College, London, Centre Hospitalier Universitaire Vaudois
Conditions	DMD-associated Dilated Cardiomyopathy
Enrollment	12 participants
Start Date	2025-10-01
Primary Completion	2027-03-31 (estimated)
Study Completion	2027-03-31 (estimated)
Updated on ClinicalTrials.gov	2025-08-15