AFTX-201: AFTX-201 is a gene therapy product consisting of the ATC-0187 capsid containing the BCL2-associated Athanogene 3 (BAG3) transgene. It is administered as a single intravenous infusion
Study 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
Eligibility
Sex
ALL
Min age
18 Years
Max age
70 Years
Healthy volunteers
No
Inclusion Criteria:
Male or female, 18 to 70 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
Primary outcome measure(s)
Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) — Baseline through 12 months Safety and tolerability of AFTX-201 over 12 months.
Trial sites (4)
Facility
City
Region
Status
University of Miami Hospital and Clinics
Miami
Florida
Recruiting
St. Lukes Hospital
Kansas City
Missouri
Recruiting
Houston Methodist Hospital
Houston
Texas
Recruiting
Institut de Cardiologie de Montréal / Montreal Heart Institute
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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