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Gene Therapy Trial Report

Summary

Gene Therapy for ACM Due to a PKP2 Pathogenic Variant


NCTID NCT06109181 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Arrhythmogenic Right Ventricular Cardiomyopathy
Disease Ontology Term DOID:0110077
Compound Name LX2020
Sponsor Lexeo Therapeutics
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 10 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant PKP2
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAVrh10
Editor Type none
Dose 1 2.0E13 gc/kg
Dose 2 6E10 vg/kg

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2023-10-25
Completion Date 2027-03
Last Update 2025-11-12

Participation Criteria


Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility 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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 5
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation
Recent Updates Completed enrollment of LX2020 Phase 1/2 trial, additional clinical data expected January 2026

Resources/Links