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An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
A Phase 1/2 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
Who can join
Ages 18 to 70 · All sexes
Full eligibility criteria
Inclusion Criteria: Male or female, 18 to 70 years of age Truncating mutation in BAG3 Dilated cardiomyopathy with left ventricular ejection fraction (LVEF) \< 45% NYHA Class II or III heart failure symptoms NT-proBNP ≥ 300 pg/mL for subjects in sinus rhythm or paced rhythm, or ≥ 600 pg/mL for subjects in atrial fibrillation Willing and able to sign informed consent and comply with study procedures Exclusion Criteria: Prior myocardial infarction, heart transplant, or presence/requirement of a left ventricular assist device (LVAD) IV therapy with positive inotropes, vasodilators, or diuretics for heart failure within 30 days prior to enrollment Positive neutralizing antibodies to ATC-0187 Any condition that, in the investigator's opinion, would place the subject at undue risk or interfere with study participation or interpretation of results
About the study
This is a Phase 1/2, 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
What is being tested
- AFTX-201 (genetic)
Sponsor: Affinia Therapeutics · Participants: 22 · Started: 2026-09
Contact the study team
- Clinical Trials Information · Phone: (617) 430-7817
Official record on ClinicalTrials.gov — NCT07426419
Locations in the U.S.
| Florida | University of Miami Hospital and Clinics, Miami |
| Missouri | St. Lukes Hospital, Kansas City |
| Texas | Houston Methodist Hospital, Houston |
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.