One-Time gene shot aims to mend broken hearts

NCT ID NCT07137338

Recruiting now Disease control Sponsor: Rocket Pharmaceuticals Inc. Source: ClinicalTrials.gov ↗

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.

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.

cardiomyopathy cardiovascular disorder Dilatation, Pathologic dilated cardiomyopathy dilated cardiomyopathy 1HH heart disorder heart failure

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DILATED CARDIOMYOPATHY (DCM)

Contacts and locations

Locations

Mayo Clinic
RECRUITING

Rochester, Minnesota, 55905, United States
Medical University of South Carolina
RECRUITING

Charleston, South Carolina, 29425, United States
University of California, San Diego
RECRUITING

San Diego, California, 92037, United States

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