NCT07426419 · Affinia Therapeutics
An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
(UPBEAT)
What this study is about
This is a Phase 1/2, where both patients and doctors know the treatment given, dose-exploration and dose-expansion, clinical trial evaluating the safety, tolerability, how the drug affects the body, and preliminary effectiveness of a single given through a vein (IV) infusion of AFTX-201 in adults with dilated cardiomyopathy caused by a BAG3 gene mutation
View original scientific 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 mutation
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Male or female, 18 to 55 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
Where
- Miami, Florida
- Kansas City, Missouri
- Houston, Texas
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 9, 2026 · Source of record for eligibility and locations