Clinical Trials:
Plakophilin-2 Related Arrhythmogenic Cardiomyopathy (PKP2-ACM)
RP-A601 is being developed for plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM), also known as ARVC and ARVD, 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.
Interested in learning more?
Visit our Patients & Caregivers page or email us at clinicaltrials@rocketpharma.com.
For more details about the trial, visit clinicaltrials.gov.