Gene therapy for a rare genetic heart weakening condition

Plain-English translation of NCT07137338 on ClinicalTrials.gov ↗ · Source last updated June 2026 · Translation generated July 2026 · How we translate trials

Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.

What is this trial?

This trial is testing a new gene therapy called RP-A701 for a rare inherited condition called BAG3-related dilated cardiomyopathy, which causes the heart muscle to weaken and enlarge. The treatment works by delivering a corrected copy of the BAG3 gene directly into your heart through a single intravenous infusion. Researchers want to see if this approach is safe and whether it can help improve heart function in patients with this genetic condition.

Currently, there is no cure for BAG3-related heart weakness—patients can only manage symptoms with medications and devices like implantable defibrillators. This trial exists because gene therapy offers a potential way to address the root cause by providing the missing or faulty gene, which could slow or even reverse the heart damage.

Do you qualify?

You likely qualify if…

you are between 18 and 65 years old
you have been diagnosed with dilated cardiomyopathy caused by a pathogenic BAG3 gene variant
your heart's pumping function is mildly to moderately reduced (ejection fraction between 25% and 45%)
you have had an implantable cardioverter-defibrillator (ICD) implanted at least 3 months ago
you have mild to moderate heart failure symptoms and have been on a stable heart failure medication regimen for at least 30 days

You likely don't qualify if…

you have severe damage to the right side of your heart
your heart's pumping function is very severely weakened (ejection fraction below 25%)
you have severe heart failure symptoms (NYHA Class IV) or no heart failure symptoms at all (NYHA Class I)
you have previously participated in a gene therapy or gene editing study
you have a history of blood clots in your heart or major arterial blood clots

What participation looks like

You would receive a single intravenous infusion of the medication on Day 0 of the trial. After that, you would be closely monitored with follow-up visits, heart function tests, and blood work over a two-year period to track your safety and how well the treatment works. The study involves up to 8 participants total, with doses starting low and carefully increasing to find the right treatment level.

AI-generated summary from trial data · Jul 1, 2026 · Not medical advice

Trial locations(3 sites)

United States

University of California, San Diego, San Diego, California

Mayo Clinic, Rochester, Minnesota

Medical University of South Carolina, Charleston, South Carolina