Phase 1 RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
This study is testing a new treatment called RP-A601 for adults with a heart condition called PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM). RP-A601 is a genetic therapy that uses a harmless virus to deliver a specific protein (PKP2) to the heart. The main goal of this Phase 1 study is to see if RP-A601 is safe. Researchers will also look for early signs of how well it works. You might be able to join if you are an adult (18 or older) with a diagnosis of ACM and a specific genetic change in your PKP2 gene. The study plans to enroll 9 participants, but the current recruitment status is unclear.
- Study design
- This is a Phase 1 study designed to test increasing doses of RP-A601 in a small group of participants (9 planned).
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for safety for 12 months after receiving the treatment.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
At a glance
Conditions
Where it's being run
3 sites across 3 statesStudy leadership
- Barry Greenberg, MD · PRINCIPAL_INVESTIGATOR · University of California, San Diego
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- Evaluation of safety associated with RP-A60112 months post-infusion
Incidence of treatment emergent adverse events (TEAEs), incidence of Serious Adverse Events (SAEs), and identification of Dose Limiting Toxicities (DLTs)