[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Sebastiano Lava\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":41},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":5},"100565535","phase-2-repurposing-empagliflozin-for-dmd-associated-cardiomyopathy-in-children-6-18-years-of-age-100565535",false,"NCT06643442","Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age","Repurposing Empagliflozin for Duchenne Muscular Dystrophy - Associated Cardiomyopathy: a Pharmacokinetics, Safety and Proof-of-concept Study Among Children 6-18 Years of Age","REDMeD","Inclusion Criteria:\n\n* Children or adolescents 6 to 18 years of age with DMD-associated cardiomyopathy, followed either as in- or outpatients, will be eligible for inclusion.\n* Currently on heart failure medication (any drug or any combination).\n* Patients should potentially benefit from adding a SGLT2i (as judged by the treating physician and the PI or Co-PI).\n* 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.\n* Adolescents, respectively parents or caregivers of children, capable of giving informed consent.\n* Ability to tolerate a cardiac MRI investigation without the need of general anaesthesia.\n\nExclusion Criteria:\n\n* Inability to understand and go through the informed consent procedure.\n* Inability to receive medications per os or through a nasogastric tube.\n* Type 1 or Type 2 Diabetes mellitus or any underlying metabolic disease associated with hypoglycaemias.\n* Body weight \\\u003C15kg.\n* Current smokers (defined as \\>1 cigarette\u002Fweek).\n* Use of any other nicotine-delivering product (e.g. nicotine patches).\n* Any known illicit drug abuse.\n* Active chronic HBV, HCV or HIV.\n* Any major surgery within 4 weeks of first dose administration.\n* Blood transfusion recipient within 4 weeks of dose administration.\n* eGFR \\\u003C45mL\u002Fmin\u002F1.73m2 (simplified Schwartz formula or Filler formula).\n* K+ \\>6.5mmol\u002FL.\n* Blood glucose \\\u003C4mmol\u002FL.\n* There are no blood pressure exclusion criteria foreseen, but participants need to be haemodynamically stable, as assessed by the local investigator.\n* Sustained or symptomatic arrhythmia insufficiently controlled with drug and\u002For device therapy.\n* 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).\n* 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.\n* Known lactose intolerance, galactose intolerance, total lactase deficiency, or glucose-galactose malabsorption.\n* Known allergies to active ingredients or excipients of commercially available empagliflozin tablets.\n* Significant medical history of active severe medical disease.\n* Significant liver disease, Child Pugh Class C, or significant laboratory abnormalities at enrolment.\n* Significant gastroenterological or hepatic disease that could significantly impair absorption or metabolism of orally administered drugs.\n* Any medical co-morbidity, which is deemed incompatible (or only with relevant risk) with study participation by the treating clinician and\u002For the study investigator.\n* 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\u002For the study investigator.\n* The patient is currently participating in another interventional clinical trial or has participated in such a trial during the \\\u003C14 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.","ALL","6 Years","18 Years",{"count":21,"type":22},12,"ESTIMATED","INTERVENTIONAL",[25],"PHASE2","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:\n\n1. define a dose rationale for this indication and age group (pharmacokinetic study),\n2. assess and monitor safety,\n3. assess ease-of-swallow,\n4. explore middle-term (3-6 months) efficacy and efficacy markers.\n\nParticipants 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.\n\nNo comparison group is foreseen for this study.",[28],"DMD-associated Dilated Cardiomyopathy","NOT_YET_RECRUITING","2025-08-12",{"date":32,"type":33},"2025-08-15","ACTUAL",{"date":35,"type":22},"2025-10-01",{"date":37,"type":22},"2027-03-31",{"name":39,"class":40},"Sebastiano Lava","OTHER",""]