Gene Therapy for ACM Due to a PKP2 Pathogenic Variant
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
1 other identifier
interventional
10
1 country
5
Brief 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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Feb 2024
Typical duration for phase_1
5 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 25, 2023
CompletedFirst Posted
Study publicly available on registry
October 31, 2023
CompletedStudy Start
First participant enrolled
February 29, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
March 1, 2027
November 12, 2025
November 1, 2025
2.8 years
October 25, 2023
November 7, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (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
12 months
Other Outcomes (1)
Selected Exploratory
12 months
Study Arms (1)
LX2020
EXPERIMENTALSingle ascending dose of LX2020, with a starting dose of 2.0 x10\^13 gc/kg, in multiple cohorts
Interventions
LX2020 is an adeno-associated viral vector encoding the human Plakophilin-2 (PKP2) gene (AAVrh.10hPKP2)
Eligibility Criteria
You may qualify if:
- 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%
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (5)
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
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
LEXEO Clinical Trials
Lexeo Therapeutics
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 25, 2023
First Posted
October 31, 2023
Study Start
February 29, 2024
Primary Completion (Estimated)
December 1, 2026
Study Completion (Estimated)
March 1, 2027
Last Updated
November 12, 2025
Record last verified: 2025-11