An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
Sponsored by Affinia Therapeutics
About this trial
Last updated 3 months ago
Study ID
AFTX-201-001
Status
Not yet recruiting
Type
Interventional
Phase
Phase 1
Placebo
No
Accepting
18 to 55 Years
All Sexes
Trial Timing
Started 3 months ago
What is this trial about?
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
What are the participation requirements?
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