Gene therapy trial hopes to fix heart muscle in rare genetic disease
NCT ID NCT06109181
First seen Nov 12, 2025 · Last updated May 12, 2026 · Updated 21 times
Summary
This early-stage trial tests a gene therapy called LX2020 in 10 adults with a rare, inherited heart condition (PKP2-ACM) that can cause dangerous heart rhythms and heart failure. The therapy is given once through a vein and aims to deliver a working copy of the PKP2 gene to heart cells. The main goal is to check safety, but researchers will also look for signs that the treatment improves heart function.
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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.
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
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Locations
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Johns Hopkins University
Baltimore, Maryland, 21287, United States
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Medical University of South Carolina
Charleston, South Carolina, 29425, United States
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Stanford University
Stanford, California, 94305, United States
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University of Michigan
Ann Arbor, Michigan, 48109, United States
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University of Rochester
Rochester, New York, 14642, United States
Conditions
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