One-Time gene infusion aims to fix inherited heart disease

NCT ID NCT05885412

Recruiting now Disease control Sponsor: Rocket Pharmaceuticals Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial tests a single intravenous dose of a gene therapy called RP-A601 in 9 adults with a genetic heart condition (PKP2-ACM) that causes dangerous heart rhythms and heart failure. The therapy delivers a working copy of the PKP2 gene to heart cells to restore protein function. The main goal is to check safety, but researchers will also measure changes in heart rhythm and protein levels.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for PKP2 ARRHYTHMOGENIC CARDIOMYOPATHY (PKP2-ACM) are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

arrhythmogenic right ventricular cardiomyopathy arrhythmogenic right ventricular dysplasia 9 cardiac arrest Death, Sudden, Cardiac

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PKP2 ARRHYTHMOGENIC CARDIOMYOPATHY (PKP2-ACM)

Contacts and locations

Locations

Children's Hospital of Philadelphia
WITHDRAWN

Philadelphia, Pennsylvania, 19104, United States
Duke University
RECRUITING

Durham, North Carolina, 27710, United States
University of California, San Diego
RECRUITING

La Jolla, California, 92037, United States