This Phase 1 dose escalation trial will assess the safety and preliminary efficacy of a single dose intravenous infusion of RP-A601 in high-risk adult patients with PKP2-ACM.
Male or female ≥18 years at the time of signing the informed consent
Capable and willing to provide signed informed consent
Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
Anti-AAVrh.74 capsid neutralizing antibody titer of >1:40
Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant
Previous participation in a study of gene transfer or gene editing
Severe Right Ventricular (RV) dysfunction
Sequential
RP-A601
GeneticRP-A601 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, human plakophilin 2 (PKP2), transcript variant 2a (PKP2a)
Evaluation of safety associated with RP-A601
Incidence of treatment emergent adverse events (TEAEs), incidence of Serious Adverse Events (SAEs), and identification of Dose Limiting Toxicities (DLTs)
Preliminary efficacy of RP-A601 - Myocardial PKP2 protein expression
Assessment of changes in myocardial PKP2 protein expression
Preliminary efficacy of RP-A601 - Ventricular ectopy and arrhythmia
Assessment of changes in levels of ventricular ectopy and arrhythmia on cardiac rhythm monitoring
Preliminary efficacy of RP-A601 - Cardiac biomarkers
Assessment of changes in circulating levels of cardiac biomarkers of injury and stress
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