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Gene therapy for rare heart disease passes early safety check

NCT ID NCT07050160

Disease control Sponsor: Lexeo Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study follows 10 people who already received LX2020 gene therapy for arrhythmogenic cardiomyopathy caused by a PKP2 gene mutation. Researchers will monitor them for years to see if the treatment remains safe and continues to help control the disease. The goal is to understand long-term effects, not to test if it works as a cure.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
LX2020 gene therapy
What this could lead to
If successful, this could show that a single dose of gene therapy safely controls arrhythmogenic cardiomyopathy long-term, reducing the need for other treatments.
What could go wrong
This is a very small, early-stage follow-up study with only 10 participants. It is not designed to prove the therapy works, and long-term risks or loss of effect are possible.

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.

Participants

About 10 people

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

Started

Aug 2025

Expected to finish

Aug 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.

Who is studied

Participants who received LX2020 in Study LX2020-01 will roll over into this study.

Ages

18 to 67 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: * Participants who received LX2020 in study LX2020-01 Exclusion Criteria: * Concurrent enrollment in any other clinical investigation involving use of an investigational agent.

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Conditions

The condition(s) this trial relates to.

arrhythmogenic right ventricular cardiomyopathy arrhythmogenic right ventricular dysplasia 9 cardiac arrest cardiomyopathy Death, Sudden, Cardiac

As listed by the trial registrant

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

Arrhythmogenic cardiomyopathy PKP2-acm PKP2-arvc

How to take part

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

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    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

  • University of Michigan

    Ann Arbor, Michigan, 48109, United States

More trials for these conditions

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