Evidence map›Paper›PMID 42223801›Full record

ArticleCardiology and therapy2026

Repurposing Empagliflozin for Duchenne Muscular Dystrophy-Associated Cardiomyopathy: Protocol for a Pharmacokinetics, Safety and Proof-of-Concept Trial in Children.

Sebastiano A G Lava, Craig Laurence, Janet Mallory, Oscar Della Pasqua, Alessandro Di Deo, Chet Villa, Sean Lang, Michael Quail, Michael Burch

Registry-linked trialAbstract read
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In one paragraph

Article in Cardiology and therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06643442 (Repurposing Empagliflozin for Duchenne Muscular Dystrophy - Associated Cardiomyopathy), which is not on this map. Not yet cited in PubMed.

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1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT06643442 phase2not yet recruitingnot on this map

Repurposing Empagliflozin for Duchenne Muscular Dystrophy - Associated Cardiomyopathy: a Pharmacokinetics, Safety and Proof-of-concept Study Among Children 6-18 Years of Age

TypeinterventionalSponsorSebastiano LavaRan2025 to 2027Enrolled12ConditionsDMD-associated Dilated CardiomyopathyArmsEmpagliflozin Tablets
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Sebastiano A G LavaPaediatric Cardiology Unit, Centre Hospitalier Universitaire Vaudois (CHUV), Lausanne University Hospital, and University of Lausanne, Rue du Bugnon 50, 1011, Lausanne, Switzerland. webmaster@sebastianolava.ch.ORCID http://orcid.org/0000-0002-8391-5462
Craig LaurenceHeart Failure and Transplantation, Department of Pediatric Cardiology, Great Ormond Street Hospital, London, UK.
Janet MalloryHeart Failure and Transplantation, Department of Pediatric Cardiology, Great Ormond Street Hospital, London, UK.
Oscar Della PasquaClinical Pharmacology and Therapeutics Group, University College London, London, UK.
Alessandro Di DeoClinical Pharmacology and Therapeutics Group, University College London, London, UK.
Chet VillaHeart Institute, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Sean LangHeart Institute, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Michael QuailCentre for Cardiovascular Imaging, UCL Institute of Cardiovascular Science, University College London, London, UK.
Michael BurchHeart Failure and Transplantation, Department of Pediatric Cardiology, Great Ormond Street Hospital, London, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionDuchenne muscular dystrophy (DMD) is a life-limiting disease characterized by progressive muscle wasting of skeletal and cardiac myocytes. Nowadays, heart failure is the principal cause of death. Current treatment is still unsatisfactory. Empagliflozin has shown excellent results in adults with heart failure, and is licensed for adolescents > 10 years of age with type 2 diabetes mellitus. In an effort to repurpose empagliflozin for DMD-associated cardiomyopathy, we aim to describe its pharmacokinetic behaviour in this population, assess ease-of-swallow, monitor safety, explore efficacy and screen efficacy markers.

methodsThis is a single-arm, single-centre, open-label phase 2a trial in children and adolescents aged 6 to ≤ 18 years with DMD-associated cardiomyopathy. Participants (n = 12) will take empagliflozin 10 mg once daily for 6 months, with a whole-day stay at visit 1 for the evaluation of pharmacokinetics, and follow-up visits at 1 week, 6 weeks, 3 months and 6 months. On top of acceptability (ease-of-swallow), which will be assessed at visit 1, further secondary endpoints include safety and efficacy clinical (e.g. heart rate, blood pressure), biochemical (e.g. NT-proBNP, haemoglobin, uric acid, electrolytes, renal function), imaging (echocardiography, cardiac magnetic resonance) and bioimpedance (intra- and extracellular fluid volume) parameters. PLANNED OUTCOMES: Characterization of primary and secondary pharmacokinetic parameters will allow one to define the dose range for children and adolescents with DMD-associated cardiomyopathy, informing both current compassionate care and the design of future efficacy trials. Additionally, this trial will enable the identification of efficacy markers to be used as endpoints in future efficacy trials and in clinical practice. TRIAL REGISTRATION NUMBER: NCT06643442, ISRCTN 12497973, IRAS number 1009946.

Indexed as

CardiomyopathyDapagliflozinDilated cardiomyopathyDuchenne muscular dystrophyEmpagliflozinHeart failureMethodologyRepurposingSGLT2 inhibitorsSodium glucose transporter type 2 inhibitors

Identifiers

PMID42223801

What Socratic holds

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.