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Gene therapy trial targets rare heart disease in 10 patients

NCT ID NCT06109181

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
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026

Summary

This early-stage trial tests a gene therapy called LX2020 for people with a heart condition called arrhythmogenic cardiomyopathy, caused by a change in the PKP2 gene. The study involves 10 adults who already have a defibrillator implanted. The main goal is to see if the treatment is safe and tolerable.

Why investors are watching

Lexeo Therapeutics is testing LX2020, a gene therapy for a rare heart condition called arrhythmogenic cardiomyopathy caused by PKP2 gene mutations. This is a first-in-human trial with only 10 patients, so the main goal is to see if the treatment is safe and tolerable. For a micro-cap company, this early data will be a key signal of whether its lead program has a future.

If it works: If the trial shows LX2020 is safe and well-tolerated, Lexeo could advance the drug to later-stage testing. That would validate its gene therapy platform and potentially attract more attention from partners or investors.

If it fails: The trial could fail to show safety, or the company could face delays in enrolling or dosing patients. Early-stage trials often fail, and a setback here would be a major blow for a company of this size, since LX2020 appears to be its main focus.

AI-written from the trial record. Speculative, and not investment advice.

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 10 people

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

Started

Feb 2024

Expected to finish

Mar 2027

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.

Selected Inclusion Criteria: * Adults with a clinical diagnosis of ACM meeting the 2010 revised Task Force Criteria (TFC) * Genetic testing documenting a pathogenic or likely pathogenic variant in PKP2 * Frequent premature ventricular complexes (PVCs) * Implantable cardioverter-defibrillator (ICD) implantation ≥ 12 weeks prior to the pre-screening MRI * Left ventricular ejection fraction ≥ 40% Selected Exclusion Criteria: * Evidence of variant(s) in addition to PKP2 that meets the standard criteria to be considered pathogenic or likely pathogenic for ACM * Other cardiac abnormalities as specified in the protocol * New York Heart Association Functional Class IV at the time of consent * History of prior gene transfer therapy

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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

Contacts and locations

Locations

  • Johns Hopkins University

    Baltimore, Maryland, 21287, United States

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Stanford University

    Stanford, California, 94305, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109, United States

  • University of Rochester

    Rochester, New York, 14642, United States

More trials for these conditions

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