NCT07426419PHASE1, PHASE2Recruiting
An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
Affinia Therapeutics
Start Date
6/1/2026
Completion Date
12/1/2032
Summary
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
Detailed Description
Participants will receive a single intravenous infusion of AFTX-201 and will be followed for safety and clinical assessments. The study includes short-term safety monitoring and long-term follow-up for up to 60 months
Eligibility Criteria
Age Range: 18 years to 55 years
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
Interventions
GENETIC
AFTX-201
Conditions
Dilated Cardiomyopathy (DCM)BAG3 Mutation Associated Dilated Cardiomyopathy
Locations
University of Miami Hospital and Clinics
Miami, Florida 33136
United States
St. Lukes Hospital
Kansas City, Missouri 64111
United States
Houston Methodist Hospital
Houston, Texas 77030
United States