NCT06976606

Brief Summary

An observational study to assess real-world patient characteristics and clinical course of disease in participants with PKP2-ACM.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
40

participants targeted

Target at P25-P50 for all trials

Timeline
16mo left

Started Jan 2024

Typical duration for all trials

Geographic Reach
1 country

6 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress63%
Jan 2024Sep 2027

Study Start

First participant enrolled

January 23, 2024

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

March 28, 2024

Completed
1.1 years until next milestone

First Posted

Study publicly available on registry

May 16, 2025

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2027

Expected
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2027

Last Updated

May 16, 2025

Status Verified

May 1, 2025

Enrollment Period

3.4 years

First QC Date

March 28, 2024

Last Update Submit

May 13, 2025

Conditions

Arrhythmogenic CardiomyopathyPKP2-ACMPKP2-ARVC

Keywords

Arrhythmogenic CardiomyopathyACMCardiomyopathyARVCArrhythmogenic Right VentricularArrhythmogenic Right Ventricular DysplasiaGenetic cardiomyopathyGene TherapyPKP2 GenePlakophilin-2LX2020SNAPSHOT-PKP2Ventricular ArrhythmiaPVCsSudden Cardiac DeathCardiac Arrest

Outcome Measures

Primary Outcomes (1)

  • Premature ventricular contractions (PVC)

    Change from baseline

    12 months

Secondary Outcomes (7)

  • Ventricular arrhythmias and associated clinical events

    12 months

  • ECG parameters

    12 months

  • Cardiac structure and function

    12 months

  • Patient reported outcomes

    12 months

  • Patient reported outcomes

    12 months

  • +2 more secondary outcomes

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients aged 18-65 years at time of consent with confirmed diagnosis of ACM and documentation of PKP2 variant.

You may qualify if:

  • Adults with a clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
  • Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
  • Frequent premature ventricular contractions (PVCs)
  • Patients must have an ICD placed prior to enrollment
  • Left ventricular ejection fraction (LVEF) ≥ 50% for Part A participants. Left ventricular ejection fraction (LVEF) ≥40% for Part B participants.

You may not qualify if:

  • Evidence of variant(s) in addition to PKP2 that meet standard criteria to be considered pathogenic or likely pathogenic for an arrhythmogenic cardiomyopathy.
  • A history of other cardiac abnormalities as specified in the protocol.
  • New York Heart Association symptoms of heart failure of Class IV at the time of consent.
  • A history of prior gene transfer therapy.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Leland Stanford Junior University

Redwood City, California, 94063-3126, United States

RECRUITING

Johns Hopkins University

Baltimore, Maryland, 21287, United States

RECRUITING

Northshore University Healthsystem Research Institute

Columbia, Maryland, 21044, United States

RECRUITING

University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

Henry Ford Hospital

Detroit, Michigan, 48202, United States

RECRUITING

University of Rochester

Rochester, New York, 14642, United States

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Blood samples will be collected to assess cardiac biomarkers associated with PKP2-ACM and to study existing antibodies to AAVrh.10.

MeSH Terms

Conditions

CardiomyopathiesArrhythmogenic Right Ventricular DysplasiaDeath, Sudden, CardiacHeart Arrest

Condition Hierarchy (Ancestors)

Heart DiseasesCardiovascular DiseasesHeart Defects, CongenitalCardiovascular AbnormalitiesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDeath, SuddenDeathPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Lexeo Clinical Trials

    Lexeo Therapeutics

    STUDY DIRECTOR

Central Study Contacts

Person*: Lexeo Clinical Trials

CONTACT

212-547-9879clinicaltrials@lexeotx.com

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
OTHER
Target Duration
1 Year
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 28, 2024

First Posted

May 16, 2025

Study Start

January 23, 2024

Primary Completion (Estimated)

July 1, 2027

Study Completion (Estimated)

September 1, 2027

Last Updated

May 16, 2025

Record last verified: 2025-05

Data Sharing

IPD Sharing
Will not share

Locations

US(6)