An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM) (NCT07426419) | Clinical Trial Compass
Not Yet RecruitingPhase 1/2
An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
United States22 participantsStarted 2026-06
Plain-language 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
Who can participate
Age range
18 Years – 55 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this is a Phase 1/2 trial of a brand-new AAV gene therapy called AFTX-201, and the main thing being measured is how many people experience serious side effects, what does that tell us about how much is still unknown about its safety at this stage?
2This trial specifically targets people with a BAG3 gene mutation causing their DCM — has my genetic testing confirmed I actually have a BAG3 mutation, and if not, would that make me ineligible?
3The trial is listed as 'not yet recruiting,' so it may not even be open yet — realistically, how long might it be before enrollment starts, and is waiting for it a reasonable option given where my heart function is right now?
4AAV-based gene therapies typically can only be given once because the immune system builds antibodies against the viral vector — is that the case here with AFTX-201, and what would that mean if the first dose doesn't work or if better treatments come along later?
5Compared to the standard medical treatments already available for DCM, what are the potential advantages and risks of pursuing an experimental gene therapy at this early phase, and would it make more sense to try optimizing my current medications first?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Timeframe: Baseline through 12 months
Trial details
NCT IDNCT07426419
SponsorAffinia Therapeutics
Sponsor typeINDUSTRY
Study typeINTERVENTIONAL
Primary completion2028-12
Contact for this trial
Jennifer (Garrett) Medley Clinical Trials Information