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

Sponsor
Sebastiano Lava
Study ID
NCT06643442
Phase
PHASE2
Status
Not Yet Recruiting

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Conditions

  • DMD-associated Dilated Cardiomyopathy

Eligibility Criteria

Sex
ALL
Age
6 Years - 18 Years
Healthy Volunteers
Not accepted

Interventions

Study Details

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.

Key Dates

Start date
Oct 1, 2025
Status verified
Oct 2024
Primary completion
Mar 31, 2027
Completion
Mar 31, 2027

Study Design

Enrollment
12 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Empagliflozin
    All participants will receive the IMP (open-label trial, primary outcome PK)

Primary Outcome Measure

Pharmacokinetics - apparent clearance (CL/F) [ Time Frame: Visit 1 to Visit 3 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks after study start) ]

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