ACTIVE_NOT_RECRUITING

Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

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Conditions

Arrhythmogenic CardiomyopathyPKP2-ACMPKP2-ARVCACMCardiomyopathyARVCArrhythmogenic Right VentricularArrhythmogenic Right Ventricular DysplasiaGenetic cardiomyopathyGene TherapyPKP2 GenePlakophilin-2LX2020HEROIC-PKP2Ventricular ArrhythmiaPVCsSudden Cardiac DeathCardiac Arrest

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

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

Quick Facts

Study Start:2024-02-29
Study Completion:2027-03
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT06109181

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 65 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Adults with a clinical diagnosis of ACM meeting the 2010 revised Task Force Criteria (TFC)
  2. * Genetic testing documenting a pathogenic or likely pathogenic variant in PKP2
  3. * Frequent premature ventricular complexes (PVCs)
  4. * Implantable cardioverter-defibrillator (ICD) implantation ≥ 12 weeks prior to the pre-screening MRI
  5. * Left ventricular ejection fraction ≥ 40%
  1. * Evidence of variant(s) in addition to PKP2 that meets the standard criteria to be considered pathogenic or likely pathogenic for ACM
  2. * Other cardiac abnormalities as specified in the protocol
  3. * New York Heart Association Functional Class IV at the time of consent
  4. * History of prior gene transfer therapy

Contacts and Locations

Principal Investigator

LEXEO Clinical Trials
STUDY_DIRECTOR
Lexeo Therapeutics

Study Locations (Sites)

Stanford University
Stanford, California, 94305
United States
Johns Hopkins University
Baltimore, Maryland, 21287
United States
University of Michigan
Ann Arbor, Michigan, 48109
United States
University of Rochester
Rochester, New York, 14642
United States
Medical University of South Carolina
Charleston, South Carolina, 29425
United States

Collaborators and Investigators

Sponsor: Lexeo Therapeutics

  • LEXEO Clinical Trials, STUDY_DIRECTOR, Lexeo Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-02-29
Study Completion Date2027-03

Study Record Updates

Study Start Date2024-02-29
Study Completion Date2027-03

Terms related to this study

Keywords Provided by Researchers

  • 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

Additional Relevant MeSH Terms

  • Arrhythmogenic Cardiomyopathy
  • PKP2-ACM
  • PKP2-ARVC