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One-Time gene shot aims to mend broken hearts

NCT ID NCT07137338

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 8 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Jun 2026

An estimate. Start dates often move.

Expected to finish

Jun 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: Subjects are eligible for inclusion into the study only if all the following criteria apply: 1. Male or female between 18 and 65 years of age at the time of signing the informed consent 2. Capable of and willing to provide signed informed consent 3. Clinical diagnosis of DCM defined as and requiring each of the following: 1. Mild to moderate systolic dysfunction (LVEF ≥ 25% and ≤ 45%) by echocardiography or CMR performed within 3 months of enrollment. 2. Absence of severe coronary artery disease (\>70% stenosis) or active myocardial ischemia as the etiology of LV systolic dysfunction 3. Absence of uncontrolled hypertension, significant cardiac valve disease (i.e., greater than moderate in severity), infiltrative disorder, or systemic disease known to cause cardiomyopathy. 4. Documentation of a pathogenic or likely pathogenic variant in BAG3 5. History of ICD implantation ≥ 3 months prior to enrollment 6. NYHA Class II or III HF symptoms with stable HF therapeutic guideline-directed medical regimen for 30 days prior to enrollment Exclusion Criteria: 1. CV disease that may be related to a genetic etiology other than a BAG3 pathogenic or likely pathogenic variant. 2. Previous participation in a study of gene transfer or gene editing. 3. I.V. inotropic, vasodilator, or diuretic therapy ≤ 30 days prior to enrollment. 4. History of intracardiac thrombosis or arterial thromboembolic events 5. Severe RV dysfunction assessed by echocardiogram or CMR ≤ 12 months prior to screening 6. LVEF \< 25% by echocardiogram or CMR at ≤ 3 months prior to screening 7. NYHA Class I or IV HF

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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)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    3 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

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

More trials for these conditions

Other studies related to the condition(s) this trial covers.