REDMeD
Research type
Research Study
Full title
Repurposing Empagliflozin for Duchenne muscular dystrophy-associated cardiomyopathy in children: a pharmacokinetics, safety and proof-of-concept study among children 6-18 years of age.
IRAS ID
1009946
Contact name
Michael Burch
Contact email
Sponsor organisation
Great Ormond Street Hospital/Institute of Child Health
Eudract number
2024-000201-33
ISRCTN Number
ISRCTN12497973
Clinicaltrials.gov Identifier
Research summary
Heart disease is a major cause of death in Duchenne muscular dystrophy (DMD). It is important to recognize and address this during childhood/adolescence. Sadly, current heart failure therapy in Paediatrics is unsatisfactory, with high in-hospital (10-25%), and 5-year mortality (30%-50%). Fortunately, however, some advances are in the pipeline. Indeed, we aim at repurposing empagliflozin for DMD-associated cardiomyopathy, a novel molecule that demonstrated impressive benefits in adults. In adolescents 10-18 years, it is approved for diabetes mellitus. Also, it has been used in children with a metabolic disease called glycogen storage disease type Ib, chronic kidney disease, and heart failure, with positive clinical results. Safety studies in children have been reassuring. However, little is known on their use in children or adolescents with DMD-associated cardiomyopathy.
Trials in pediatric heart failure have hitherto often failed, among others because of suboptimal dose and inadequate endpoints. Learning from the past, this time, founded by Duchenne UK, we will leverage prior knowledge from available studies and developmental pharmacology, so to define the dose rationale (“pharmacokinetic analysis”), assess safety and ease-of-swallow, and explore efficacy of empagliflozin among 12 children/adolescents with DMD-associated cardiomyopathy 6-18 years of age, followed at GOSH, London.
Participants will receive empagliflozin during 6 months and will have 5 study visits (Visit 1; follow-up visits at 1 week, 6 weeks, 3 and 6 months), and one end-study visit. Safety evaluation will occur throughout the study, ease-of-swallow will be evaluated at Visit 1, and efficacy markers (clinical examination, bloods, echocardiography, cardiac magnetic resonance) at Visits 1 & 5. Advanced calculations (pharmacokinetic modeling) will allow to define the optimal paediatric dose, informing both current compassionate-care clinical use and the design of subsequent efficacy trials.REC name
London - Hampstead Research Ethics Committee
REC reference
25/LO/0361
Date of REC Opinion
27 Jun 2025
REC opinion
Further Information Favourable Opinion