Clinical Trials:
PKP2-ACM
RP-A601 is being developed for plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM), a devastating, inherited heart disease caused by mutations in the PKP2 gene and associated with life-threatening arrhythmias, cardiac structural abnormalities, and sudden cardiac death.
The Phase 1 pivotal trial for RP-A601 is currently recruiting patients. The trial is evaluating the safety and preliminary efficacy of RP-A601 in at least six adult PKP2-ACM patients with implantable cardioverter defibrillators (ICDs) and overall high risk for severe arrhythmias. The study assesses the impact of RP-A601 on PKP2 myocardial protein expression, cardiac biomarkers, and clinical predictors of life-threatening ventricular arrhythmias and sudden cardiac death.
Trial Sites
For more details about the trial, visit clinicaltrials.gov.
For more information about PKP2-ACM, visit our Patients and Community Page
To read our Expanded Access statement, click here.