AAV Gene Therapy for BAG3-Related Dilated Cardiomyopathy in Adults

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a phase 1/2 open-label study that explores dose safety, tolerability, pharmacodynamics, and preliminary efficacy of a single intravenous infusion of AFTX-201 in adults with BAG3 mutation–associated dilated cardiomyopathy. Participants are followed for short-term safety and long-term outcomes for up to 60 months. The trial includes dose-exploration and dose-expansion components to identify an appropriate dose range. Primary goals focus on safety and pharmacodynamic signals, with secondary observations of clinical effect. Recruitment involves specific genetic and cardiac function criteria, and enrollment is limited to adults aged 18–70 with BAG3 truncating mutations and reduced left ventricular function.

Why it matters

Who can join: the full rules

Inclusion Criteria:

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 Criteria:

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

Presence or 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 it's running

• University of Miami Hospital and Clinics
  Miami, Florida, United States Recruiting
• St. Lukes Hospital
  Kansas City, Missouri, United States Recruiting
• NYU Langone
  New York, New York, United States Recruiting
• Houston Methodist Hospital
  Houston, Texas, United States Recruiting
• Institut de Cardiologie de Montréal / Montreal Heart Institute
  Montreal, Quebec, Canada Recruiting

Study contacts

• Study coordinator: Clinical Trials Information
• Email: clinicaltrials@affiniatx.com
• Phone: (617) 430-7817

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.