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New gene therapy trial hopes to strengthen hearts in rare genetic condition

NCT ID NCT07218887

Disease control Sponsor: Alexion 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 28, 2026 · Updated 3 times

Summary

This early-stage study tests a one-time gene therapy called ALXN2350 for adults with a specific genetic heart condition (BAG3-related dilated cardiomyopathy). The heart muscle becomes weak and enlarged, making it hard to pump blood. The treatment aims to fix the faulty gene to improve heart function. About 6 participants will receive the therapy intravenously, and researchers will monitor safety and heart health over time.

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 6 people

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

Started

Oct 2025

Expected to finish

Jan 2032

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 70 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: * Pathogenic or likely pathogenic mutation in BAG3 * Medical history of diagnosis of DCM * Stable combination of HF SoC medications * Adequate acoustic windows for echocardiography * LVEF \> 15% and ≤ 45% at Screening by echocardiography * Diagnosis of chronic HF for at least 3 months * NYHA Class I/II/III at Screening Exclusion Criteria: * Presence of antibodies to AAV9 * Presence of a pathogenic or likely pathogenic variant in another gene where that other gene is authoritatively recognized as causal for DCM * Decompensated HF * Cardiac ventricular arrhythmia that requires treatment * History of untreated clinically significant valve disease or a Screening confirmation of severe aortic stenosis, severe mitral stenosis, moderate or severe aortic insufficiency or severe mitral insufficiency * NYHA Class IV * History of substance abuse that could contribute to cardiac dysfunction Note: Other inclusion and exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

dilated cardiomyopathy 1HH

As listed by the trial registrant

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

BAG3 mutation associated 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

    11 sites in 4 countries. 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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Research Site

    RECRUITING

    Birmingham, Alabama, 35233, United States

  • Research Site

    RECRUITING

    Palo Alto, California, 94304, United States

  • Research Site

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • Research Site

    RECRUITING

    Cincinnati, Ohio, 45219, United States

  • Research Site

    RECRUITING

    Portland, Oregon, 97239-3098, United States

  • Research Site

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Research Site

    RECRUITING

    Houston, Texas, 77030, United States

  • Research Site

    RECRUITING

    Massa, 54100, Italy

  • Research Site

    RECRUITING

    Barcelona, 08035, Spain

  • Research Site

    RECRUITING

    Majadahonda, 28222, Spain

  • Research Site

    RECRUITING

    London, SW3 6NP, United Kingdom

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