One-Time gene infusion aims to fix inherited heart disease
NCT ID NCT05885412
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.
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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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital of Philadelphia
WITHDRAWNPhiladelphia, Pennsylvania, 19104, United States
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Duke University
RECRUITINGDurham, North Carolina, 27710, United States
Contact
Contact Email: •••••@•••••
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University of California, San Diego
RECRUITINGLa Jolla, California, 92037, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••