Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

Lexeo Therapeutics

Status and phase: Active, not recruiting; Phase 2; Phase 1

Conditions: Arrhythmogenic Cardiomyopathy; PKP2-ACM; PKP2-ARVC

Treatments: Genetic: LX2020

Study type: Interventional

Funder types: Industry

Identifiers

NCT06109181

LX2020-01

Details and patient eligibility

About

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

Enrollment: 10 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Selected 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 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

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10 participants in 1 patient group

LX2020

Experimental group

Description:

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

Treatment:

Genetic: LX2020