Gene therapy AFTX-201 for dilated cardiomyopathy caused by a BAG3 gene change
Tests AFTX-201, given once by infusion, in adults with BAG3-related dilated cardiomyopathy.
Key facts
- Who runs it
- Affinia Therapeutics
- Where
- Houston, Kansas City, Miami, and New York, United StatesSee the 4 sitesAlso in 1 other country, 1 site
- Trial code
- Registry title
- An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
- Status
- Recruiting
- Phase
- Phase 1/2
- People taking part
- 22 people, planned
- Starts
- Dec 2026
- Expected to end
- Dec 2032
- Type of study
- Interventional
- Treatments tested
- AFTX-201
- Record updated
- 3 November 2026
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with dilated cardiomyopathy, a weak and enlarged heart muscle, caused by a change in the BAG3 gene. Everyone gets one infusion into a vein of the gene therapy AFTX-201.
- Age
- 18 to 70 years
- Sex
- Men and women
- Disease stage
- Dilated cardiomyopathy with left ventricular ejection fraction (LVEF) < 50% on echocardiogram or cardiac MRI. Heart failure symptoms of NYHA Class II or III. Body weight ≤ 120 kg (265 lb).
- Earlier treatment
- No prior heart transplant. No IV therapy with positive inotropes, vasodilators or diuretics for heart failure within 30 days before enrollment.
- Biomarker or test result
- A truncating mutation in BAG3. People with positive neutralizing antibodies to ATC-0187 cannot join.
Main reasons for not taking part
- Past heart attack, when the doctor judges that coronary artery disease causes the heart weakness
- Prior heart transplant
- Having or needing a left ventricular assist device (LVAD)
- IV heart failure drugs (inotropes, vasodilators, diuretics) within 30 days before enrollment
- Positive neutralizing antibodies to ATC-0187
The registry criteria, word for word
Show all 12 criteriaShow less
Who can take part
- 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
Who cannot take part
- 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)
Check if you might fit
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?
Where in United States
Also in 1 other country, 1 site
- 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
About the trial
What the trial wants to find out
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
The group in the trial
- AFTX-201Treatment being tested
Participants will receive a single intravenous dose of AFTX-201
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 doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- 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
- Plain-language title
- Gene therapy AFTX-201 for dilated cardiomyopathy caused by a BAG3 gene changeGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 14 November 2026.
- Fit questions
- Built from NCT07426419, who can take part
- Record updated
- 3 November 2026