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One-Time gene infusion aims to fix heart muscle in rare genetic disease

NCT ID NCT05885412

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

First seen Apr 18, 2026 · Last updated May 08, 2026 · Updated 4 times

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. The goal is to see if the treatment is safe and can restore a missing protein in heart cells to reduce arrhythmias. Participants must have an implanted defibrillator and frequent extra heartbeats.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Children's Hospital of Philadelphia

    WITHDRAWN

    Philadelphia, Pennsylvania, 19104, United States

  • Duke University

    RECRUITING

    Durham, North Carolina, 27710, United States

    Contact

    Contact Email: •••••@•••••

  • University of California, San Diego

    RECRUITING

    La Jolla, California, 92037, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.

PKP2 ARRHYTHMOGENIC CARDIOMYOPATHY (PKP2-ACM)