[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"bag3-mutation-associated-dilated-cardiomyopathy\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:bag3-mutation-associated-dilated-cardiomyopathy":75},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,50],{"id":9,"slug":4,"hasResults":10,"nctId":11,"briefTitle":12,"officialTitle":13,"acronym":14,"eligibilityCriteria":15,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":30,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":39,"lastUpdatePostDateStruct":40,"startDateStruct":43,"completionDateStruct":45,"leadSponsor":47,"locationsCount":5},"100625729",false,"NCT07426419","An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)","A Phase 1\u002F2 Multicenter, Open-Label, Dose-Escalation and Dose-Expansion Trial to Assess the Safety, Tolerability, Pharmacodynamics, and Preliminary Efficacy of AFTX-201 Administered to Adult Participants With BCL2-Associated Athanogene 3 (BAG3) Mutation-Associated Dilated Cardiomyopathy: UPBEAT Trial","UPBEAT","Inclusion Criteria:\n\nMale or female, 18 to 55 years of age\n\nTruncating mutation in BAG3\n\nDilated cardiomyopathy with left ventricular ejection fraction (LVEF) \\\u003C 45%\n\nNYHA Class II or III heart failure symptoms\n\nNT-proBNP ≥ 300 pg\u002FmL for subjects in sinus rhythm or paced rhythm, or ≥ 600 pg\u002FmL for subjects in atrial fibrillation\n\nWilling and able to sign informed consent and comply with study procedures\n\nExclusion Criteria:\n\nPrior myocardial infarction, heart transplant, or presence\u002Frequirement of a left ventricular assist device (LVAD)\n\nIV therapy with positive inotropes, vasodilators, or diuretics for heart failure within 30 days prior to enrollment\n\nPositive neutralizing antibodies to ATC-0187\n\nAny condition that, in the investigator's opinion, would place the subject at undue risk or interfere with study participation or interpretation of results","ALL","18 Years","55 Years",{"count":20,"type":21},22,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","This is a Phase 1\u002F2, open-label, dose-exploration and dose-expansion, clinical trial evaluating the safety, tolerability, pharmacodynamics, and preliminary efficacy of a single intravenous infusion of AFTX-201 in adults with dilated cardiomyopathy caused by a BAG3 gene mutation",[28,29],"Dilated Cardiomyopathy (DCM)","BAG3 Mutation Associated Dilated Cardiomyopathy",[31,32,33,34,35,36,37],"AFTX-201","Gene Therapy","AAV","BAG3","Dilated Cardiomyopathy","DCM","ATC-0187","RECRUITING","2026-06-30",{"date":41,"type":42},"2026-07-02","ACTUAL",{"date":44,"type":21},"2026-06",{"date":46,"type":21},"2032-12",{"name":48,"class":49},"Affinia Therapeutics","INDUSTRY",{"id":51,"slug":4,"hasResults":10,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":55,"eligibilityCriteria":56,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":57,"enrollmentInfo":58,"targetDuration":4,"studyType":22,"phases":60,"briefSummary":61,"conditions":62,"keywords":63,"overallStatus":38,"whyStopped":4,"lastUpdateSubmitDate":65,"lastUpdatePostDateStruct":66,"startDateStruct":68,"completionDateStruct":70,"leadSponsor":72,"locationsCount":74},"100609769","NCT07218887","ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy","A Phase 1\u002F2, Open-Label, Multicenter, Dose Finding and Dose Expansion Study to Investigate the Safety, Tolerability, and Efficacy of ALXN2350 Gene Therapy in Adult Participants With BAG3 Mutation Associated Dilated Cardiomyopathy","DCMRestore","Inclusion Criteria:\n\n* Pathogenic or likely pathogenic mutation in BAG3\n* Medical history of diagnosis of DCM\n* Stable combination of HF SoC medications\n* Adequate acoustic windows for echocardiography\n\nExclusion Criteria:\n\n* Presence of antibodies to AAV9\n* Presence of a pathogenic or likely pathogenic variant in another gene where that other gene is authoritatively recognized as causal for DCM.\n* Decompensated HF\n\nNote: Other inclusion and exclusion criteria may apply.","70 Years",{"count":59,"type":21},6,[24,25],"This Phase 1\u002F2 study is an open-label, dose finding and dose expansion study investigating the safety, tolerability, and efficacy of a single IV infusion of ALXN2350 in adult participants with BAG3 associated DCM.",[29],[64,32,29],"ALXN2350","2026-06-12",{"date":67,"type":42},"2026-06-15",{"date":69,"type":42},"2025-10-24",{"date":71,"type":21},"2032-01-27",{"name":73,"class":49},"Alexion Pharmaceuticals, Inc.",10,""]