Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age6-18
SponsorSebastiano Lava
About this trial
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.
Eligibility criteria
Qualifiers
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.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- Empagliflozin Tablets
Treatment groups
12 Participants
are divided into 1 treatment groupSponsors and collaborators
Sebastiano Lava
Lead sponsor
Centre Hospitalier Universitaire Vaudois
Sponsor institution
Great Ormond Street Hospital for Children NHS Foundation Trust
Collaborator
University College, London
Collaborator
Centre Hospitalier Universitaire Vaudois
Collaborator
Source ClinicalTrials.gov