Testing a gene therapy to help hearts with a specific genetic condition

Plain-English translation of NCT05885412 on ClinicalTrials.gov ↗ · Source last updated April 2026 · Translation generated August 2026 · How we translate trials

Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.

What is this trial?

This is a Phase 1 trial testing a new treatment called RP-A601, which uses gene therapy to help repair hearts damaged by a specific genetic mutation. The trial is designed for adults with arrhythmogenic cardiomyopathy caused by PKP2 gene variants—a rare condition that makes the heart prone to dangerous, irregular rhythms. Researchers will give participants a single infusion of this medication and carefully monitor them to see if it's safe and whether it helps reduce heart rhythm problems.

People with this type of inherited heart disease currently have limited treatment options beyond devices like defibrillators to prevent life-threatening rhythms. This trial is exploring whether gene therapy—which aims to fix the underlying genetic problem rather than just manage symptoms—could offer a new way to treat the root cause.

Do you qualify?

You likely qualify if…

you are 18 years or older
you have been diagnosed with arrhythmogenic cardiomyopathy caused by a PKP2 gene mutation
you had an implantable defibrillator placed at least 6 months ago
you have experienced frequent irregular heartbeats (at least 500 per day)
your heart's main pumping chamber is functioning reasonably well (ejection fraction of 50% or higher)

You likely don't qualify if…

you have existing antibodies against this type of viral vector that would prevent the treatment from working
your heart problems are caused by a different genetic condition or non-genetic cause
you have severe weakness of the right side of your heart
you have advanced heart failure (New York Heart Association Class IV)
you have previously participated in other gene therapy or gene editing studies

What participation looks like

As a participant, you would receive a single intravenous infusion of the medication in a clinical setting. The trial is designed to test the safety of different dose levels, so you'll be monitored closely with heart tests, blood work, and rhythm monitoring both during and after your infusion. Researchers will follow you for a period of time to watch for any side effects and to see whether the treatment helps reduce your heart rhythm problems.

AI-generated summary from trial data · Aug 24, 2026 · Not medical advice

Trial locations(3 sites)

United States

University of California, San Diego, La Jolla, California

Duke University, Durham, North Carolina

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania