Gene Therapy for ACM Due to a PKP2 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
Conditions:
🦠 Arrhythmogenic Cardiomyopathy 🦠 PKP2-ACM 🦠 PKP2-ARVC
🗓️ Study Start (Actual) 29 February 2024
🗓️ Primary Completion (Estimated) February 2026
✅ Study Completion (Estimated) February 2027
👥 Enrollment (Estimated) 10
🔬 Study Type INTERVENTIONAL
📊 Phase PHASE1
Locations:
📍 Ann Arbor, Michigan, United States
📍 Charleston, South Carolina, United States

📋 Eligibility Criteria

Description

    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 consent
    • * Left ventricular ejection fraction ≥ 50%

    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 III and IV at the time of consent
    • * History of prior gene transfer therapy
Ages Eligible for Study: 18 Years to 65 Years (ADULT, OLDER_ADULT)
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No

🗓️ 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

  • First Submitted 25 October 2023
  • First Submitted that Met QC Criteria 25 October 2023
  • First Posted 31 October 2023

Study Record Updates

  • Last Update Submitted that Met QC Criteria 23 July 2024
  • Last Update Posted 24 July 2024
  • Last Verified July 2024