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
Sequential
RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3)
GeneticOne-time treatment with a single ascending dose
Incidence of Treatment-emergent Adverse Events (TEAE)
Number of participants with Adverse Events following a single IV dose of RP-A701
Incidence of Treatment-emergent Serious Adverse Events (SAE).
Number of participants with Serious Adverse Events (SAE) following a single IV dose of RP-A701
Incidence of Dose Limiting Toxicities (DLT).
Number of participants with Dose Limiting Toxicities (DLT) following a single IV dose of RP-A701
To assess the impact of RP-A701 on features of cardiovascular function.
Change in measures of LV systolic function, including LV ejection fraction (LVEF), LV global longitudinal strain.
To assess the impact of RP-A701 on features of cardiovascular function.
Change in peak oxygen consumption (VO2) or ventilatory efficiency (VE/VCO2 slope) by cardiopulmonary exercise test (CPET)
To assess the impact of RP-A701 on features of cardiovascular function.
Change in 6-minute walk distance.
To assess the extent of RP-A701 transduction and protein expression.
Change in BAG3 myocardial protein expression
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