[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07426419":3,"trial-entities:NCT07426419":127,"trial-summary:NCT07426419":131},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":21,"study_type":29,"primary_purpose":30,"phases":31,"enrollment_info":34,"interventions":37,"primary_outcomes":42,"secondary_outcomes":47,"sex":66,"minimum_age":67,"maximum_age":68,"healthy_volunteers":69,"eligibility_criteria":70,"std_ages":74,"locations":76,"central_contacts":118,"overall_officials":124,"references":125,"see_also_links":126},"NCT07426419","AFTX-201-001","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","RECRUITING","2032-12","2026-07","2026-07-09","2026-06","Affinia Therapeutics","INDUSTRY",true,"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","Participants will receive a single intravenous infusion of AFTX-201 and will be followed for safety and clinical assessments. The study includes short-term safety monitoring and long-term follow-up for up to 60 months",[19,20],"Dilated Cardiomyopathy (DCM)","BAG3 Mutation Associated Dilated Cardiomyopathy",[22,23,24,25,26,27,28],"AFTX-201","Gene Therapy","AAV","BAG3","Dilated Cardiomyopathy","DCM","ATC-0187","INTERVENTIONAL","TREATMENT",[32,33],"PHASE1","PHASE2",{"count":35,"type":36},22,"ESTIMATED",[38],{"type":39,"name":22,"description":40,"armGroupLabels":41},"GENETIC","AFTX-201 is a gene therapy product consisting of the ATC-0187 capsid containing the BCL2-associated Athanogene 3 (BAG3) transgene. It is administered as a single intravenous infusion",[22],[43],{"measure":44,"description":45,"timeFrame":46},"Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)","Safety and tolerability of AFTX-201 over 12 months.","Baseline through 12 months",[48,51,54,56,59,62],{"measure":49,"description":50,"timeFrame":46},"Change from baseline in pharmacodynamic biomarkers","Assessment of the extent of AFTX-201 transduction and myocardial BAG3 protein expression after a single IV dose",{"measure":52,"description":53,"timeFrame":46},"Change from baseline in measurement of cardiac function including left ventricular ejection fraction (LVEF)","Assessment of the impact of a single IV dose of AFTX-201 on measurement of cardiovascular function",{"measure":55,"description":53,"timeFrame":46},"Change from baseline in measurement of cardiac function including peak oxygen consumption (pVO2) by cardiopulmonary exercise test (CPET)",{"measure":57,"description":58,"timeFrame":46},"Change from baseline in New York Heart Association (NYHA) functional class","Assessment of the impact of a single IV dose of AFTX-201 on features of heart failure (HF) including functional classification by New York Heart Association (NYHA) class",{"measure":60,"description":61,"timeFrame":46},"Change from baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)","Assessment of the impact of a single IV dose of AFTX-201 on quality of life",{"measure":63,"description":64,"timeFrame":65},"Number of participants with adverse events during long-term follow-up","Safety and tolerability of AFTX-201 over 60 months","Baseline through 60 months","ALL","18 Years","55 Years",false,{"inclusion":71,"exclusion":72,"raw_text":73},[],[],"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",[75],"ADULT",[77,92,105],{"facility":78,"status":8,"city":79,"state":80,"zip":81,"country":82,"contacts":83,"geoPoint":89},"University of Miami Hospital and Clinics","Miami","Florida","33136","United States",[84],{"name":85,"role":86,"phone":87,"email":88},"Lina Carceres","CONTACT","954 249 3587","caceresl@miami.edu",{"lat":90,"lon":91},25.77427,-80.19366,{"facility":93,"status":8,"city":94,"state":95,"zip":96,"country":82,"contacts":97,"geoPoint":102},"St. Lukes Hospital","Kansas City","Missouri","64111",[98],{"name":99,"role":86,"phone":100,"email":101},"Amanda Huffman, RN, BSN","816-932-0391","amanda.huffman@bjc.org",{"lat":103,"lon":104},39.09973,-94.57857,{"facility":106,"status":8,"city":107,"state":108,"zip":109,"country":82,"contacts":110,"geoPoint":115},"Houston Methodist Hospital","Houston","Texas","77030",[111],{"name":112,"role":86,"phone":113,"email":114},"Jennifer (Garrett) Medley Sr. Clinical Trials Manager","346-238-4516","jmgarrett@houstonmethodist.org",{"lat":116,"lon":117},29.76328,-95.36327,[119],{"name":120,"role":86,"phone":121,"phoneExt":122,"email":123},"Clinical Trials Information","(617) 430-7817","2","clinicaltrials@affiniatx.com",[],[],[],{"nct_id":4,"conditions":128,"biomarkers":129},[26],[130],"BAG3 Gene",{"nct_id":4,"found":69,"summary":132,"prompt_version":132},null]