One-Time gene shot aims to mend broken hearts
NCT ID NCT07137338
First seen Jun 27, 2026 · Last updated Jul 01, 2026 · Updated 2 times
Summary
This early-stage trial tests a one-time gene therapy called RP-A701 in 8 adults with dilated cardiomyopathy caused by a faulty BAG3 gene. The therapy uses a harmless virus to deliver a working copy of the gene, aiming to improve heart function. The main goal is to check safety, but researchers will also look for signs that the heart is pumping better.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
RP-A701 (a gene therapy using a harmless virus to deliver a working BAG3 gene)
What this could lead to
If successful, this could point toward a one-time treatment that improves heart function in people with this inherited form of heart failure.
What could go wrong
This is a very early, small trial (8 people) focused on safety, not proof of effectiveness. Gene therapies can cause immune reactions or other side effects.
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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Mayo Clinic
RECRUITINGRochester, Minnesota, 55905, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Medical University of South Carolina
RECRUITINGCharleston, South Carolina, 29425, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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University of California, San Diego
RECRUITINGSan Diego, California, 92037, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••