Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

Phase 1

Recruiting

• Conditions: PKP2-ARVC; Arrhythmogenic Cardiomyopathy; PKP2-ACM
• Interventions: Genetic: LX2020

• Registration Number: NCT06109181
• Lead Sponsor: Lexeo Therapeutics

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-10-25
• Study First Submitted QC: 2023-10-25
• Study First Posted: 2023-10-31
• Study Start: 2024-02-29
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-02
• Last Update Posted: 2025-01-03
• Primary Completion: 2026-02
• Study Completion: 2027-02

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 10

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
LX2020	LX2020	Single ascending dose of LX2020, with a starting dose of 2.0 x10\^13 gc/kg, in multiple cohorts

Primary Outcome Measures

Name	Time	Method
Percentage of subjects who experienced at least 1 treatment emergent adverse event (TEAE) and/or 1 treatment emergent serious adverse event (TESAE).	12 months	Evaluation of Safety and Tolerability of LX2020

Trial Locations

Locations (5)

Johns Hopkins University; 🇺🇸 Baltimore, Maryland, United States

Stanford University; 🇺🇸 Stanford, California, United States

University of Michigan; 🇺🇸 Ann Arbor, Michigan, United States

University of Rochester; 🇺🇸 Rochester, New York, United States

Medical University of South Carolina; 🇺🇸 Charleston, South Carolina, United States