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

NCT06643442 · Status: NOT_YET_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2025-08-15

No results posted yet for this study

Summary

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.

Conditions

  • DMD-associated Dilated Cardiomyopathy

Interventions

DRUG

Empagliflozin Tablets

Empagliflozin 10mg p.o. once daily (commercially available tablet)

Sponsors & Collaborators

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    collaborator OTHER

  • University College, London

    collaborator OTHER

  • Centre Hospitalier Universitaire Vaudois

    collaborator OTHER

  • Sebastiano Lava

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-10-01
Primary Completion
2027-03-31
Completion
2027-03-31

Countries

  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06643442 on ClinicalTrials.gov