Tests empagliflozin for heart weakness in children with Duchenne muscular dystrophy

Trial details

Who runs it

Sebastiano Lava

Trial code

NCT06643442

Record updated

15 August 2025

Original description

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:Read full original descriptionShow less

Repurposing Empagliflozin for Duchenne Muscular Dystrophy - Associated Cardiomyopathy: a Pharmacokinetics, Safety and Proof-of-concept Study Among 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.

Inclusion: 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: Inability to receive medications per os or through a nasogastric tube; Any known illicit drug abuse; Any major surgery within 4 weeks of first dose administration; Cardio-surgical procedure within the 2 months prior to Visit 1, or interventional cardiac catheterization within 2 weeks prior to Visit 1, or the patient is planned to undergo cardiac surgery or an interventional cardiac catheterization during the study period (i.e. in the 6 months following Visit 1); Active urinary tract infection (being treated with antibiotics at the moment of Visit 1) or other relevant bacterial infection, as judged by the treating clinician and/or the study investigator; The patient is currently participating in another interventional clinical trial or has participated in such a trial during the <14 days before Visit 1 (or if enrolment in this study is incompatible with the protocol of that preceding trial), or the duration of five half-lives of the IMP, whichever is longer; Type 1 or Type 2 Diabetes mellitus or any underlying metabolic disease associated with hypoglycaemias; Active chronic HBV, HCV or HIV; Sustained or symptomatic arrhythmia insufficiently controlled with drug and/or device therapy; Known allergies to active ingredients or excipients of commercially available empagliflozin tablets; Significant medical history of active severe medical disease; Significant gastroenterological or hepatic disease that could significantly impair absorption or metabolism of orally administered drugs; eGFR <45mL/min/1.73m2 (simplified Schwartz formula or Filler formula); Blood glucose <4mmol/L; Known lactose intolerance, galactose intolerance, total lactase deficiency, or glucose-galactose malabsorption; Significant liver disease, Child Pugh Class C, or significant laboratory abnormalities at enrolment; Inability to understand and go through the informed consent procedure; Body weight <15kg; Current smokers (defined as >1 cigarette/week); Use of any other nicotine-delivering product (e.g. nicotine patches); Blood transfusion recipient within 4 weeks of dose administration; K+ >6.5mmol/L; There are no blood pressure exclusion criteria foreseen, but participants need to be haemodynamically stable, as assessed by the local investigator; Post-menarchal female patients of childbearing potential cannot be included. Participants who begin menstruating during the trial will be discontinued from the IMP. However, their monitoring will continue up to 6 months after their first dose of IMP; Any medical co-morbidity, which is deemed incompatible (or only with relevant risk) with study participation by the treating clinician and/or the study investigator.

ClinicalTrials.gov NCT06643442