Gene therapy trial aims to fix heart muscle in rare genetic disease
NCT ID NCT07606274
First seen Jun 25, 2026 · Last updated Aug 07, 2026 · Updated 4 times
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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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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Brigham and Women's Hospital
NOT_YET_RECRUITINGBoston, Massachusetts, 02115, United States
Contact Email: •••••@•••••
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Cedars-Sinai Medical Center
RECRUITINGLos Angeles, California, 90048, United States
Contact Email: •••••@•••••
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Houston Methodist Hospital
RECRUITINGHouston, Texas, 77030, United States
Contact Email: •••••@•••••
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Mayo Clinic
RECRUITINGRochester, Minnesota, 55905, United States
Contact Email: •••••@•••••
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The Christ Hospital
NOT_YET_RECRUITINGCincinnati, Ohio, 45219, United States
Contact Email: •••••@•••••
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The Ohio State University
NOT_YET_RECRUITINGColumbus, Ohio, 43210, United States
Contact Email: •••••@•••••
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University of Alabama in Birmingham
NOT_YET_RECRUITINGBirmingham, Alabama, 35294, United States
Contact Email: •••••@•••••
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University of Michigan
NOT_YET_RECRUITINGAnn Arbor, Michigan, 48109, United States
Contact Email: •••••@•••••