An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
Key facts
- Where
- Houston, Kansas City, Miami, and New York, United StatesSee the 4 sites
- Phase
- Phase 1/2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 70 years old?
Source: NCT07426419, who can take part
Where in United States
- Houston Methodist HospitalHouston, United StatesContact: Jennifer (Garrett) Medley Sr. Clinical Trials ManagerTaking people
- St Lukes HospitalKansas City, United StatesContact: Amanda Huffman, RN, BSNTaking people
- University of Miami Hospital and ClinicsMiami, United StatesContact: Lina CarceresTaking people
- NYU LangoneNew York, United StatesContact: Loriya ThaoTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Affinia Therapeutics
- Trial code
- NCT07426419
- Record updated
- 3 November 2026
- Original description
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 mutationRead full original descriptionShow less
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
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
Inclusion: Male or female, 18 to 70 years of age; Truncating mutation in BAG3; Dilated cardiomyopathy with left ventricular ejection fraction (LVEF) < 50% by echocardiogram or cardiac MRI; NYHA Class II or III heart failure symptoms; Body weight ≤ 120 kg; Willing and able to sign informed consent and comply with study procedures.
Exclusion: Prior myocardial infarction when, in the investigator's judgment, the totality of clinical and imaging evidence supports that left ventricular dysfunction is due to coronary artery disease rather than BAG3-associated dilated cardiomyopathy; Prior heart transplant; 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; Presence or requirement of a left ventricular assist device (LVAD).