Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age
NCT ID: NCT06643442
Last Updated: 2025-08-15
Study Results
Results pending
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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Recruitment Status
NOT_YET_RECRUITING
Clinical Phase
PHASE2
Total Enrollment
12 participants
Study Classification
INTERVENTIONAL
Study Start Date
2025-10-01
Study Completion Date
2027-03-31
Brief Summary
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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.
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.
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Detailed Description
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Cardiac disease represents the main life-limiting condition in Duchenne muscular dystrophy (DMD). It is important to recognize and address this early in the disease course. Because of lack of DMD specific drugs, present attitudes for established DMD-related cardiomyopathy ground on current treatment for heart failure. Unfortunately, however, current heart failure therapy in Paediatrics is still unsatisfactory, with high in-hospital (7-26%), and 5-year mortality (30%-50%). Furthermore, mortality rate for DMD cardiomyopathy is worse than similarly aged idiopathic dilated cardiomyopathy (DCM) patients.
Among the recent improvements in adult heart failure management, the sodium glucose transporter type 2 inhibitors (SGLT2i) dapagliflozin and empagliflozin were found to reduce cardiovascular death or worsening heart failure by 25% on top of optimal medical therapy. Indeed, since 2021, they have been recommended as part of standard heart failure therapy.
In the past, paediatric heart failure trials often failed, mainly because of suboptimal dose or inappropriate formulations and endpoints.
This phase II.a, open-label trial is designed to characterize pharmacokinetics (primary outcome), ease-of-swallow, safety and explore potential efficacy markers (secondary outcomes) of empagliflozin in 12 children and adolescents with DMD-related cardiomyopathy, so to inform the design and performance of subsequent, state-of-the-art, high-quality efficacy trials.
Participants will receive empagliflozin during 6 months. They will have 5 visits, one end-study visit and 7 to 8 pharmacokinetic samples. The timing of these samples will be optimized exploiting contemporary modeling and simulation techniques.
Safety evaluation will occur throughout the study, while ease-of-swallow will be evaluated at Visit 1, and efficacy markers at Visits 1, 4 and 5.
Pharmacokinetic modeling will characterize primary and secondary pharmacokinetic parameters and allow to define the optimal paediatric dose, informing both current compassionate-care use and the design of future efficacy trials.
Among the recent improvements in adult heart failure management, the sodium glucose transporter type 2 inhibitors (SGLT2i) dapagliflozin and empagliflozin were found to reduce cardiovascular death or worsening heart failure by 25% on top of optimal medical therapy. Indeed, since 2021, they have been recommended as part of standard heart failure therapy.
In the past, paediatric heart failure trials often failed, mainly because of suboptimal dose or inappropriate formulations and endpoints.
This phase II.a, open-label trial is designed to characterize pharmacokinetics (primary outcome), ease-of-swallow, safety and explore potential efficacy markers (secondary outcomes) of empagliflozin in 12 children and adolescents with DMD-related cardiomyopathy, so to inform the design and performance of subsequent, state-of-the-art, high-quality efficacy trials.
Participants will receive empagliflozin during 6 months. They will have 5 visits, one end-study visit and 7 to 8 pharmacokinetic samples. The timing of these samples will be optimized exploiting contemporary modeling and simulation techniques.
Safety evaluation will occur throughout the study, while ease-of-swallow will be evaluated at Visit 1, and efficacy markers at Visits 1, 4 and 5.
Pharmacokinetic modeling will characterize primary and secondary pharmacokinetic parameters and allow to define the optimal paediatric dose, informing both current compassionate-care use and the design of future efficacy trials.
Conditions
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DMD-associated Dilated Cardiomyopathy
Study Design
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Allocation Method
NA
Intervention Model
SINGLE_GROUP
Primary Study Purpose
TREATMENT
Blinding Strategy
NONE
Study Groups
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Empagliflozin
All participants will receive the IMP (open-label trial, primary outcome PK)
Group Type
EXPERIMENTAL
Empagliflozin Tablets
Intervention Type
DRUG
Empagliflozin 10mg p.o. once daily (commercially available tablet)
Interventions
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Empagliflozin Tablets
Empagliflozin 10mg p.o. once daily (commercially available tablet)
Intervention Type
DRUG
Eligibility Criteria
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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.
* 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 formula or Filler formula).
* K+ \>6.5mmol/L.
* Blood glucose \<4mmol/L.
* Sustained or symptomatic arrhythmia insufficiently controlled with drug and/or device therapy.
* 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).
* 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.
* Known lactose intolerance, galactose intolerance, total lactase deficiency, or glucose-galactose malabsorption.
* Known allergies to active ingredients or excipients of commercially available empagliflozin tablets.
* Significant medical history of active severe medical disease.
* Significant liver disease, Child Pugh Class C, or significant laboratory abnormalities at enrolment.
* Significant gastroenterological or hepatic disease that could significantly impair absorption or metabolism of orally administered drugs.
* 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.
* 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.
* 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 formula or Filler formula).
* K+ \>6.5mmol/L.
* Blood glucose \<4mmol/L.
* Sustained or symptomatic arrhythmia insufficiently controlled with drug and/or device therapy.
* 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).
* 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.
* Known lactose intolerance, galactose intolerance, total lactase deficiency, or glucose-galactose malabsorption.
* Known allergies to active ingredients or excipients of commercially available empagliflozin tablets.
* Significant medical history of active severe medical disease.
* Significant liver disease, Child Pugh Class C, or significant laboratory abnormalities at enrolment.
* Significant gastroenterological or hepatic disease that could significantly impair absorption or metabolism of orally administered drugs.
* 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.
* 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.
Minimum Eligible Age
6 Years
Maximum Eligible Age
18 Years
Eligible Sex
ALL
Accepts Healthy Volunteers
No
Sponsors
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Great Ormond Street Hospital for Children NHS Foundation Trust
OTHER
Sponsor Role
collaborator
University College, London
OTHER
Sponsor Role
collaborator
Centre Hospitalier Universitaire Vaudois
OTHER
Sponsor Role
collaborator
Sebastiano Lava
OTHER
Sponsor Role
lead
Responsible Party
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Sebastiano Lava
Chief Investigator
Responsibility Role
SPONSOR_INVESTIGATOR
Locations
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Great Ormond Street Hospital NHS Foundation Trust
London, Greater London, United Kingdom
Site Status
Countries
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United Kingdom
References
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Adorisio R, Mencarelli E, Cantarutti N, Calvieri C, Amato L, Cicenia M, Silvetti M, D'Amico A, Grandinetti M, Drago F, Amodeo A. Duchenne Dilated Cardiomyopathy: Cardiac Management from Prevention to Advanced Cardiovascular Therapies. J Clin Med. 2020 Oct 1;9(10):3186. doi: 10.3390/jcm9103186.
Reference Type
BACKGROUND
Das BB. Current State of Pediatric Heart Failure. Children (Basel). 2018 Jun 28;5(7):88. doi: 10.3390/children5070088.
Reference Type
BACKGROUND
Rossano JW, Kim JJ, Decker JA, Price JF, Zafar F, Graves DE, Morales DL, Heinle JS, Bozkurt B, Towbin JA, Denfield SW, Dreyer WJ, Jefferies JL. Prevalence, morbidity, and mortality of heart failure-related hospitalizations in children in the United States: a population-based study. J Card Fail. 2012 Jun;18(6):459-70. doi: 10.1016/j.cardfail.2012.03.001. Epub 2012 Apr 10.
Reference Type
BACKGROUND
Andrews RE, Fenton MJ, Dominguez T, Burch M; British Congenital Cardiac Association. Heart failure from heart muscle disease in childhood: a 5-10 year follow-up study in the UK and Ireland. ESC Heart Fail. 2016 Jun;3(2):107-114. doi: 10.1002/ehf2.12082. Epub 2016 Jan 24.
Reference Type
BACKGROUND
Towbin JA, Lowe AM, Colan SD, Sleeper LA, Orav EJ, Clunie S, Messere J, Cox GF, Lurie PR, Hsu D, Canter C, Wilkinson JD, Lipshultz SE. Incidence, causes, and outcomes of dilated cardiomyopathy in children. JAMA. 2006 Oct 18;296(15):1867-76. doi: 10.1001/jama.296.15.1867.
Reference Type
BACKGROUND
Packer M, Anker SD, Butler J, Filippatos G, Pocock SJ, Carson P, Januzzi J, Verma S, Tsutsui H, Brueckmann M, Jamal W, Kimura K, Schnee J, Zeller C, Cotton D, Bocchi E, Bohm M, Choi DJ, Chopra V, Chuquiure E, Giannetti N, Janssens S, Zhang J, Gonzalez Juanatey JR, Kaul S, Brunner-La Rocca HP, Merkely B, Nicholls SJ, Perrone S, Pina I, Ponikowski P, Sattar N, Senni M, Seronde MF, Spinar J, Squire I, Taddei S, Wanner C, Zannad F; EMPEROR-Reduced Trial Investigators. Cardiovascular and Renal Outcomes with Empagliflozin in Heart Failure. N Engl J Med. 2020 Oct 8;383(15):1413-1424. doi: 10.1056/NEJMoa2022190. Epub 2020 Aug 28.
Reference Type
BACKGROUND
Bourke JP, Bueser T, Quinlivan R. Interventions for preventing and treating cardiac complications in Duchenne and Becker muscular dystrophy and X-linked dilated cardiomyopathy. Cochrane Database Syst Rev. 2018 Oct 16;10(10):CD009068. doi: 10.1002/14651858.CD009068.pub3.
Reference Type
BACKGROUND
Muntoni F. Cardiomyopathy in muscular dystrophies. Curr Opin Neurol. 2003 Oct;16(5):577-83. doi: 10.1097/01.wco.0000093100.34793.81.
Reference Type
BACKGROUND
Bourke J, Turner C, Bradlow W, Chikermane A, Coats C, Fenton M, Ilina M, Johnson A, Kapetanakis S, Kuhwald L, Morley-Davies A, Quinlivan R, Savvatis K, Schiava M, Yousef Z, Guglieri M. Cardiac care of children with dystrophinopathy and females carrying DMD-gene variations. Open Heart. 2022 Oct;9(2):e001977. doi: 10.1136/openhrt-2022-001977.
Reference Type
BACKGROUND
Authors/Task Force Members:; McDonagh TA, Metra M, Adamo M, Gardner RS, Baumbach A, Bohm M, Burri H, Butler J, Celutkiene J, Chioncel O, Cleland JGF, Coats AJS, Crespo-Leiro MG, Farmakis D, Gilard M, Heymans S, Hoes AW, Jaarsma T, Jankowska EA, Lainscak M, Lam CSP, Lyon AR, McMurray JJV, Mebazaa A, Mindham R, Muneretto C, Francesco Piepoli M, Price S, Rosano GMC, Ruschitzka F, Kathrine Skibelund A; ESC Scientific Document Group. 2021 ESC Guidelines for the diagnosis and treatment of acute and chronic heart failure: Developed by the Task Force for the diagnosis and treatment of acute and chronic heart failure of the European Society of Cardiology (ESC). With the special contribution of the Heart Failure Association (HFA) of the ESC. Eur J Heart Fail. 2022 Jan;24(1):4-131. doi: 10.1002/ejhf.2333.
Reference Type
BACKGROUND
Braunwald E. Gliflozins in the Management of Cardiovascular Disease. N Engl J Med. 2022 May 26;386(21):2024-2034. doi: 10.1056/NEJMra2115011. No abstract available.
Reference Type
BACKGROUND
Lava SAG, Laurence C, Di Deo A, Sekarski N, Burch M, Della Pasqua O. Dapagliflozin and Empagliflozin in Paediatric Indications: A Systematic Review. Paediatr Drugs. 2024 May;26(3):229-243. doi: 10.1007/s40272-024-00623-z. Epub 2024 Apr 18.
Reference Type
BACKGROUND
Verhaert D, Richards K, Rafael-Fortney JA, Raman SV. Cardiac involvement in patients with muscular dystrophies: magnetic resonance imaging phenotype and genotypic considerations. Circ Cardiovasc Imaging. 2011 Jan;4(1):67-76. doi: 10.1161/CIRCIMAGING.110.960740. No abstract available.
Reference Type
BACKGROUND
Bellanti F, Della Pasqua O. Modelling and simulation as research tools in paediatric drug development. Eur J Clin Pharmacol. 2011 May;67 Suppl 1(Suppl 1):75-86. doi: 10.1007/s00228-010-0974-3. Epub 2011 Jan 19.
Reference Type
BACKGROUND
Bellanti F, Di Iorio VL, Danhof M, Della Pasqua O. Sampling Optimization in Pharmacokinetic Bridging Studies: Example of the Use of Deferiprone in Children With beta-Thalassemia. J Clin Pharmacol. 2016 Sep;56(9):1094-103. doi: 10.1002/jcph.708. Epub 2016 Apr 1.
Reference Type
BACKGROUND
Loss KL, Shaddy RE, Kantor PF. Recent and Upcoming Drug Therapies for Pediatric Heart Failure. Front Pediatr. 2021 Nov 11;9:681224. doi: 10.3389/fped.2021.681224. eCollection 2021.
Reference Type
BACKGROUND
Lava SA, Simonetti GD, Bianchetti AA, Ferrarini A, Bianchetti MG. Prevention of vitamin D insufficiency in Switzerland: a never-ending story. Int J Pharm. 2013 Nov 30;457(1):353-6. doi: 10.1016/j.ijpharm.2013.08.068. No abstract available.
Reference Type
BACKGROUND
Cella M, Knibbe C, Danhof M, Della Pasqua O. What is the right dose for children? Br J Clin Pharmacol. 2010 Oct;70(4):597-603. doi: 10.1111/j.1365-2125.2009.03591.x. No abstract available.
Reference Type
BACKGROUND
Other Identifiers
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2024-000201-33
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
24HL14
Identifier Type: -
Identifier Source: org_study_id
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