Gene therapy trial aims to fix heart muscle in rare genetic disease
NCT ID NCT07606274
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tests a single dose of a gene therapy called NVC-001 in 21 adults with a genetic form of dilated cardiomyopathy (LMNA mutation). The therapy uses a harmless virus to deliver a modified gene into heart cells, aiming to protect the heart muscle. The main goals are to check safety and see if it can improve heart function.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
NVC-001 (a gene therapy using a harmless virus to deliver a modified gene into heart cells)
What this could lead to
If it works, this could point toward a treatment that slows or stops heart muscle damage in people with a specific genetic form of dilated cardiomyopathy.
What could go wrong
This is a very early, first-in-human trial with only 21 participants. The gene therapy may not improve heart function, and there are risks from the infusion and the virus used to deliver the gene.
Disclaimer
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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.
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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Locations
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Cedars-Sinai Medical Center
Los Angeles, California, 90048, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••