An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
Condition(s) studied
Investigational drug(s) / intervention(s)
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
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 (5)
| Facility | City | Region | Status |
|---|---|---|---|
| University of Miami Hospital and Clinics | Miami | Florida | Recruiting |
| St. Lukes Hospital | Kansas City | Missouri | Recruiting |
| NYU Langone | New York | New York | Recruiting |
| Houston Methodist Hospital | Houston | Texas | Recruiting |
| Institut de Cardiologie de Montréal / Montreal Heart Institute | Montreal | Quebec | Recruiting |
Other trials for the same condition
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.
View NCT07426419 on ClinicalTrials.gov ↗ ← All trials in the USA