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Gene therapy trial aims to fix heart muscle in rare genetic disease

NCT ID NCT07606274

Disease control Sponsor: Nuevocor Pte. Ltd. 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 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.

Study facts

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

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 21 people

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

Started

Jun 2026

Expected to finish

Mar 2030

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 years and older

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.

Key Inclusion Criteria: 1. Adult ≥18 years old, able to provide informed consent 2. Clinical diagnosis of LMNA-related dilated cardiomyopathy (DCM) with LV systolic dysfunction 3. Pathogenic or likely pathogenic LMNA mutation confirmed by a CLIA-certified/CAP-accredited laboratory 4. NYHA Functional Class I, II, or III 5. LVEF \>25% and ≤45% by echocardiography 6. Implantable cardioverter-defibrillator (ICD) or cardiac resynchronization therapy-defibrillator )CRT-D) implanted \>3 months prior to consent Key Exclusion Criteria: 1. Any chronic medical condition that, in the Investigator's opinion, would compromise participant safety or compliance 2. Hospitalization as an inpatient for the following conditions within 3 months prior to signing the ICF: Heart failure, Persistent/refractory ventricular arrhythmia requiring external defibrillation, or multiple/recurrent cardioversion/defibrillation by ICD Indication for, or history of, LV assist device or cardiac transplantation due to end-stage cardiac dysfunction 3. Current or anticipated need for chronic IV inotropic therapy

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Conditions

The condition(s) this trial relates to.

dilated cardiomyopathy dilated cardiomyopathy 1A

As listed by the trial registrant

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

Lmna-related dilated cardiomyopathy

How to take part

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

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

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

  3. 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 ↗

  4. 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

  • Brigham and Women's Hospital

    NOT_YET_RECRUITING

    Boston, Massachusetts, 02115, United States

    Contact Email: •••••@•••••

  • Cedars-Sinai Medical Center

    RECRUITING

    Los Angeles, California, 90048, United States

    Contact Email: •••••@•••••

  • Houston Methodist Hospital

    RECRUITING

    Houston, Texas, 77030, United States

    Contact Email: •••••@•••••

  • Mayo Clinic

    RECRUITING

    Rochester, Minnesota, 55905, United States

    Contact Email: •••••@•••••

  • The Christ Hospital

    NOT_YET_RECRUITING

    Cincinnati, Ohio, 45219, United States

    Contact Email: •••••@•••••

  • The Ohio State University

    NOT_YET_RECRUITING

    Columbus, Ohio, 43210, United States

    Contact Email: •••••@•••••

  • University of Alabama in Birmingham

    NOT_YET_RECRUITING

    Birmingham, Alabama, 35294, United States

    Contact Email: •••••@•••••

  • University of Michigan

    NOT_YET_RECRUITING

    Ann Arbor, Michigan, 48109, United States

    Contact Email: •••••@•••••