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Arrhythmogenic CardiomyopathyPhase 1/2NCT06109181

Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

A Phase 1/2 study testing LX2020 in people with Arrhythmogenic Cardiomyopathy and PKP2-ACM, run by Lexeo Therapeutics at 5 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This is a Phase 1/2, first-in-human, open-label, intravenous, dose-escalating, multicenter trial that is designed to assess the safety and tolerability of LX2020 in adult patients with PKP2-ACM Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC
What is being tested?
LX2020
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
5 sitesUnited States
Study dates
29 Feb 2024to Mar 2027
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

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

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

United States5 sites

  • University of Michigan

    Ann Arbor, Michigan, 48109

  • Johns Hopkins University

    Baltimore, Maryland, 21287

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

  • University of Rochester

    Rochester, New York, 14642

  • Stanford University

    Stanford, California, 94305

Other trials in United States

About this study

Official title: A Phase 1/2 Study of the Safety and Efficacy of LX2020 Gene Therapy in Patients With Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant

This is a Phase 1/2, first-in-human, open-label, intravenous, dose-escalating, multicenter trial that is designed to assess the safety and tolerability of LX2020 in adult patients with PKP2-ACM

Being tested
LX2020
Keywords
Arrhythmogenic Cardiomyopathy, ACM, Cardiomyopathy, ARVC, Arrhythmogenic Right Ventricular, Arrhythmogenic Right Ventricular Dysplasia, Genetic cardiomyopathy, Gene Therapy, PKP2 Gene, Plakophilin-2, LX2020, HEROIC-PKP2, Ventricular Arrhythmia, PVCs, Sudden Cardiac Death, Cardiac Arrest
Medical subject headings
Cardiomyopathies, Arrhythmogenic Right Ventricular Dysplasia, Death, Sudden, Cardiac, Heart Arrest

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LX2020Genetic

    LX2020 is an adeno-associated viral vector encoding the human Plakophilin-2 (PKP2) gene (AAVrh.10hPKP2)

Study groups in NCT06109181
Study groupRoleWhat they receive
LX2020ExperimentalSingle ascending dose of LX2020, with a starting dose of 2.0 x10^13 gc/kg, in multiple cohorts
Receives: Genetic: LX2020

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of subjects who experienced at least 1 treatment emergent adverse event (TEAE) and/or 1 treatment emergent serious adverse event (TESAE).

    Evaluation of Safety and Tolerability of LX2020

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Oct 2023
  2. Study start29 Feb 2024
  3. Primary completionDec 2026 (estimated)
  4. Study completionMar 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Masking
None (open label)
Enrolment
10 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06109181
Sponsor's protocol ID
LX2020-01
Status last verified
Nov 2025
First submitted
25 Oct 2023
Last updated
12 Nov 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing LX2020 in people with Arrhythmogenic Cardiomyopathy and PKP2-ACM, run by Lexeo Therapeutics at 5 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 12 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of subjects who experienced at least 1 treatment emergent adverse event (TEAE) and/or 1 treatment emergent serious adverse event (TESAE). (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06109181.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Nov 2025.

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