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DMD-associated Dilated CardiomyopathyPhase 2REDMeDNCT06643442

Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age

A Phase 2 study testing Empagliflozin Tablets in people with DMD-associated Dilated Cardiomyopathy, run by Sebastiano Lava at 1 site in United Kingdom.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
Search all trials

Trial summary

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… Read more about this study

Who is it for?
Ages 6 to 18 years
Conditions the study is for
DMD-associated Dilated Cardiomyopathy
What is being tested?
Empagliflozin Tablets
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
1 siteUnited Kingdom
Study dates
1 Oct 2025to 31 Mar 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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.

Who cannot join

  • 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).
Show all 25Show fewer
  • 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.
  • There are no blood pressure exclusion criteria foreseen, but participants need to be haemodynamically stable, as assessed by the local investigator.
  • 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.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

United Kingdom1 site

  • Great Ormond Street Hospital NHS Foundation Trust

    London, Greater London, WC1N 3JH

Other trials in United Kingdom

About this study

Official title: 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.
Read more

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.

Read the detailed description

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.

Being tested
Empagliflozin Tablets
Medical subject headings
Dmd-Associated Dilated Cardiomyopathy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Empagliflozin TabletsDrug

    Empagliflozin 10mg p.o. once daily (commercially available tablet)

Study groups in NCT06643442
Study groupRoleWhat they receive
EmpagliflozinExperimentalAll participants will receive the IMP (open-label trial, primary outcome PK)
Receives: Drug: Empagliflozin Tablets

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Pharmacokinetics - apparent clearance (CL/F)

    Empagliflozin concentrations at the different time-points (6 samples at Visit 1, 1 opportunistic sample at Visits 2 and 3; the timing of the samples will be optimized with modeling \& simulation techniques).

    Basing on these concentrations, non-compartmental pharmacokinetic calculations will be performed, allowing to determine pharmacokinetic parameters (including CL/F).

    Measured: Visit 1 to Visit 3 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks after study start)

  2. Pharmacokinetics - apparent (central) volume of distribution (Vd/F)

    Empagliflozin concentrations at the different time-points (6 samples at Visit 1, 1 opportunistic sample at Visits 2 and 3; the timing of the samples will be optimized with modeling \& simulation techniques).

    Basing on these concentrations, non-compartmental pharmacokinetic calculations will be performed, allowing to determine pharmacokinetic parameters (including Vd/F).

    Measured: Visit 1 to Visit 3 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks after study start)

  3. Pharmacokinetics - half-life

    Empagliflozin concentrations at the different time-points (6 samples at Visit 1, 1 opportunistic sample at Visits 2 and 3; the timing of the samples will be optimized with modeling \& simulation techniques).

    Basing on these concentrations, non-compartmental pharmacokinetic calculations will be performed, allowing to determine pharmacokinetic parameters (including t1/2).

    Measured: Visit 1 to Visit 3 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks after study start)

  4. Pharmacokinetics - AUC

    Empagliflozin concentrations at the different time-points (6 samples at Visit 1, 1 opportunistic sample at Visits 2 and 3; the timing of the samples will be optimized with modeling \& simulation techniques).

    Basing on these concentrations, non-compartmental pharmacokinetic calculations will be performed, allowing to determine pharmacokinetic parameters (including AUC).

    Measured: Visit 1 to Visit 3 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks after study start)

  5. Pharmacokinetics - maximal concentration (Cmax)

    Empagliflozin concentrations at the different time-points (6 samples at Visit 1, 1 opportunistic sample at Visits 2 and 3; the timing of the samples will be optimized with modeling \& simulation techniques).

    Basing on these concentrations, non-compartmental pharmacokinetic calculations will be performed, allowing to determine pharmacokinetic parameters (including Cmax).

    Measured: Time Frame: Visit 1 to Visit 3 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks after study start)

Secondary outcomes (16)

  1. Safety 1 - eGFR

    Creatinine, respectively Cystatin C, will be collected in order to calculate eGFR (bedside Schwartz formula, respectively Filler equation).

    Measured: Visit 1 to Visit 5 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks, Visit 4 = 3 months, Visit 5 = 6 months after enrolment)

  2. Safety 2 - Occurrence of hypoglycemia

    Blood glucose will be checked three times at Visit 1 (baseline, at approximately 2-3h post-intake, and before discharge at 8h post-intake), as well as once at Visits 2 to 5. Outcome measure: number of patients experiencing hypoglycemia.

    Measured: Visit 1 to Visit 5 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks, Visit 4 = 3 months, Visit 5 = 6 months after enrolment)

  3. Safety 3 - Occurrence of ketoacidosis

    The outcome is presence (or absence) of ketoacidosis. This will be assessed at Visits 1, 2, 3, 4 and 5. Outcome measure: number of patients experiencing ketoacidosis.

    Measured: Visit 1 to Visit 5 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks, Visit 4 = 3 months, Visit 5 = 6 months after enrolment)

  4. Safety 4 - Occurrence of UTI

    The outcome is presence (or absence) of UTI diagnosis. This will be assessed at Visits 1, 2, 3, 4 and 5. Outcome measure: number of patients experiencing UTI between Visit 2 and Visit 5.

    Measured: Visit 1 to Visit 5 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks, Visit 4 = 3 months, Visit 5 = 6 months after enrolment)

  5. Ease of swallow

    Ease of swallow will be assessed by means of facial hedonic scales, according to our standard procedure, at Visit 1. (Scale 1 to 4, 1 being the worse and 4 the best score: very difficult - difficult - possible - easy to swallow.)

    Measured: Visit 1 (Visit 1 = day 1)

  6. Efficacy and efficacy markers (exploratory) 1 - Heart failure severity class

    Symptoms, clinical signs, NYHA (if > or =8 years of age) / Ross (if <8 years of age) class assignment. NYHA and Ross heart failure classes share the same scale of I (no limitation of physical activity) to IV (symptoms at rest). Analysis will be performed at Visit 1, Visit 4 and Visit 5. Outcome: change between Visit 1 and Visit 5.

    Measured: Visit 1 to Visit 5 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks, Visit 4 = 3 months, Visit 5 = 6 months after enrolment)

  7. Efficacy and efficacy markers (exploratory) 2 - NT-proBNP level

    Analysis will be performed at Visits 1, 3, 4 and 5. Outcome: change between Visit 1 and Visit 5.

    Measured: Visit 1 to Visit 5 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks, Visit 4 = 3 months, Visit 5 = 6 months after enrolment)

  8. Efficacy and efficacy markers (exploratory) 3 - Echocardiography 1: Left-ventricular end-diastolic diameter (LVEDd)

    LVEDd (z-score) will be measured at Visits 1, 4 and 5. Outcome: change between Visit 1 and Visit 5.

    Measured: Visits 1, 4 and 5 (Visit 1 = day 1, Visit 4 = 3 months, Visit 5 = 6 months after enrolment)

  9. Efficacy and efficacy markers (exploratory) 4 - Echocardiography 2: Left-ventricular end-systolic diameter (LVESd)

    LVESd (z-score) will be measured at Visits 1, 4 and 5. Outcome: change between Visit 1 and Visit 5.

    Measured: Visits 1, 4 and 5 (Visit 1 = day 1, Visit 4 = 3 months, Visit 5 = 6 months after enrolment)

  10. Efficacy and efficacy markers (exploratory) 5 - Echocardiography 3: Fractional shortening (FS)

    FS (%) will be measured at Visits 1, 4 and 5. Outcome: change between Visit 1 and Visit 5.

    Measured: Visits 1, 4 and 5 (Visit 1 = day 1, Visit 4 = 3 months, Visit 5 = 6 months after enrolment)

  11. Efficacy and efficacy markers (exploratory) 6 - Echocardiography 4: Left ventricular ejection fraction (LV-EF)

    LV-EF (%) will be measured at Visits 1, 4 and 5. Outcome: change between Visit 1 and Visit 5.

    Measured: Visits 1, 4 and 5 (Visit 1 = day 1, Visit 4 = 3 months, Visit 5 = 6 months after enrolment)

  12. Efficacy and efficacy markers (exploratory) 7 - cMRI 1: Left ventricular end-diastolic volume

    LV end-diastolic volume will be measured at Visits 1 and 5. Outcome: change between Visit 1 and Visit 5.

    Measured: Visit 1, Visit 5 (Visit 1 = day 1, Visit 5 = 6 months after enrolment)

  13. Efficacy and efficacy markers (exploratory) 8 - cMRI 2: Left ventricular end-systolic volume

    LV end-systolic volume will be measured at Visits 1 and 5. Outcome: change between Visit 1 and Visit 5.

    Measured: Visit 1, Visit 5 (Visit 1 = day 1, Visit 5 = 6 months after enrolment)

  14. Efficacy and efficacy markers (exploratory) 9 - cMRI 3: Left ventricular ejection fraction

    LV end-systolic ejection fraction will be measured/calculated at Visits 1 and 5. Outcome: change between Visit 1 and Visit 5.

    Measured: Visit 1, Visit 5 (Visit 1 = day 1, Visit 5 = 6 months after enrolment

  15. Efficacy and efficacy markers (exploratory) 10 - cMRI 4: Presence of late gadolinium enhancement

    Presence (y) or absence (n) of late gadolinium enhancement in each of the 17 AHA segments will be measured at Visits 1 and 5. Outcome: change in number of LGE positive segments between Visit 1 and Visit 5.

    Measured: Visit 1, Visit 5 (Visit 1 = day 1, Visit 5 = 6 months after enrolment)

  16. Efficacy and efficacy markers (exploratory) 11 - cMRI 5: Extracellular volume (ECV)

    Extracellular volume (ECV) will be measured/calculated at Visits 1 and 5. Outcome: change between Visit 1 and Visit 5.

    Measured: Visit 1, Visit 5 (Visit 1 = day 1, Visit 5 = 6 months after enrolment)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Oct 2024
  2. Study start1 Oct 2025 (estimated)
  3. Primary completion31 Mar 2027 (estimated)
  4. Study completion31 Mar 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06643442
Sponsor's protocol ID
24HL14
Other IDs
2024-000201-33
Acronym
REDMeD
Status last verified
Oct 2024
First submitted
3 Jun 2024
Last updated
15 Aug 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • 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.

    PubMed 33019553

  • Das BB. Current State of Pediatric Heart Failure. Children (Basel). 2018 Jun 28;5(7):88. doi: 10.3390/children5070088.

    PubMed 29958420

  • 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.

    PubMed 22633303

  • 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.

    PubMed 27812385

  • 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.

    PubMed 17047217

  • 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.

    PubMed 32865377

  • 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.

    PubMed 30326162

  • Muntoni F. Cardiomyopathy in muscular dystrophies. Curr Opin Neurol. 2003 Oct;16(5):577-83. doi: 10.1097/01.wco.0000093100.34793.81.

    PubMed 14501841

  • 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.

    PubMed 36252992

  • 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.

    PubMed 35083827

  • 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.

    PubMed 35613023

  • 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.

    PubMed 38635113

  • 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.

    PubMed 21245364

  • 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.

    PubMed 21246352

  • 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.

    PubMed 26785826

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Empagliflozin Tablets in people with DMD-associated Dilated Cardiomyopathy, run by Sebastiano Lava at 1 site in United Kingdom.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 15 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Pharmacokinetics - apparent clearance (CL/F) (time frame: Visit 1 to Visit 3 (Visit 1 = day 1, Visit 2 = 1 week, Visit 3 = 5-6 weeks after study start)). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06643442, which also lists 19 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Aug 2025.

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