Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age (NCT06643442) | Clinical Trial Compass
Not Yet RecruitingPhase 2
Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age
United Kingdom12 participantsStarted 2025-10-01
Plain-language 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.
Who can participate
Age range
6 Years – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Children or adolescents 6 to 18 years of age with DMD-associated cardiomyopathy, followed either as in- or outpatients, will be eligible for inclusion.
* Currently on heart failure medication (any drug or any combination).
* Patients should potentially benefit from adding a SGLT2i (as judged by the treating physician and the PI or Co-PI).
* Patients need to be on stable medical treatment, defined as no new heart failure drug started over the preceding 2 weeks and no major drug dose modification (apart minor adaptations, like weight adaptations, rounding or formulation changes) during the 2 weeks prior to enrolment.
* Adolescents, respectively parents or caregivers of children, capable of giving informed consent.
* Ability to tolerate a cardiac MRI investigation without the need of general anaesthesia.
Exclusion Criteria:
* Inability to understand and go through the informed consent procedure.
* Inability to receive medications per os or through a nasogastric tube.
* Type 1 or Type 2 Diabetes mellitus or any underlying metabolic disease associated with hypoglycaemias.
* Body weight \<15kg.
* Current smokers (defined as \>1 cigarette/week).
* Use of any other nicotine-delivering product (e.g. nicotine patches).
* Any known illicit drug abuse.
* Active chronic HBV, HCV or HIV.
* Any major surgery within 4 weeks of first dose administration.
* Blood transfusion recipient within 4 weeks of dose administration.
* eGFR \<45mL/min/1.73m2 (simplified Schwartz …
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Pharmacokinetics - apparent clearance (CL/F)
Timeframe: Visit 1 to Visit 3 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks after study start)
2
Pharmacokinetics - apparent (central) volume of distribution (Vd/F)
Timeframe: Visit 1 to Visit 3 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks after study start)
3
Pharmacokinetics - half-life
Timeframe: Visit 1 to Visit 3 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks after study start)
4
Pharmacokinetics - AUC
Timeframe: Visit 1 to Visit 3 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks after study start)
5
Pharmacokinetics - maximal concentration (Cmax)
Timeframe: Time Frame: Visit 1 to Visit 3 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks after study start)