[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"dmd-associated-dilated-cardiomyopathy\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:dmd-associated-dilated-cardiomyopathy":56},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,42],{"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":41},"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",1,{"id":43,"slug":44,"hasResults":11,"nctId":45,"briefTitle":46,"officialTitle":47,"acronym":48,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":50,"minAge":19,"maxAge":4,"enrollmentInfo":51,"targetDuration":4,"studyType":23,"phases":52,"briefSummary":54,"conditions":55,"keywords":4,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":58,"lastUpdatePostDateStruct":59,"startDateStruct":61,"completionDateStruct":63,"leadSponsor":65,"locationsCount":68},"100533349","phase-1-modulation-of-serca2a-of-intra-myocytic-calcium-trafficking-in-cardiomyopathy-secondary-to-duchenne-muscular-dystrophy-100533349","NCT06224660","Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy","A Phase 1b, Open-Label, Controlled Trial Evaluating the Safety and Efficacy of SRD-001 (AAV1\u002FSERCA2a) in Subjects With Cardiomyopathy Secondary to Duchenne Muscular Dystrophy","MUSIC-DMD","Inclusion Criteria:\n\n* Diagnosis of DMD with confirmatory genetic testing\n* Cardiomyopathy with left ventricular scar in at least 3 of 16 segments\n* Left ventricular ejection fraction \\\u003C 40%\n* Individualized, optimized cardiac medical therapy and glucocorticoid treatment for at least 12 months prior to enrollment\n* Willing and able to provide informed consent\n\nExclusion Criteria:\n\n* Abnormal blood pressure\n* Non-DMD-related liver function test elevations\n* Cystatin C ≥ 1.2 mg\u002FL\n* Thrombocytopenia\n* Anemia\n* Inadequate pulmonary function","MALE",{"count":21,"type":22},[53],"PHASE1","This research study is testing whether an experimental drug, called SRD-001, is safe and helps the weakened heart of patients with Duchenne muscular dystrophy (DMD) regain its ability to effectively pump blood to the rest of the body. SRD-001 is a form of gene therapy. The goal of SRD-001 gene therapy is to provide the heart muscle cells with extra copies of the SERCA2a gene so that they can produce more SERCA2a protein to help the heart muscle cells squeeze\u002Fcontract better. Researchers will compare SRD-001 treated participants with no-treatment participants; all participants will continue to take their current heart medications. All participants will be followed very closely for 2 years and undergo cardiac magnetic resonance imaging of their heart at baseline, year 1 and year 2 along with assessment of upper limb function and lung function. After the 2 years of close follow-up, all participants will roll over into long-term follow-up where they will be called biannually for information on their current medical status.",[56],"DMD-Associated Dilated Cardiomyopathy","RECRUITING","2025-02-26",{"date":60,"type":33},"2025-02-27",{"date":62,"type":33},"2024-10-02",{"date":64,"type":22},"2030-10",{"name":66,"class":67},"Sardocor Corp.","INDUSTRY",3]