This is a Phase 1, open-label, dose-escalation trial to characterize the safety, tolerability, and preliminary efficacy of RP-A701 following a single IV administration in high-risk adult patients with BAG3-DCM.
Male or female between 18 and 65 years of age at the time of signing the informed consent
Capable of and willing to provide signed informed consent
Mild to moderate systolic dysfunction (LVEF ≥ 25% and ≤ 45%) by echocardiography or CMR performed within 3 months of enrollment.
Absence of severe coronary artery disease (>70% stenosis) or active myocardial ischemia as the etiology of LV systolic dysfunction
CV disease that may be related to a genetic etiology other than a BAG3 pathogenic or likely pathogenic variant.
Previous participation in a study of gene transfer or gene editing.
I.V. inotropic, vasodilator, or diuretic therapy ≤ 30 days prior to enrollment.
History of intracardiac thrombosis or arterial thromboembolic events
Rocket Pharmaceuticals Inc.
Lead sponsor