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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
Drug Product 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
Therapeutic Modality Gene transfer
Therapy Route in vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Gene Delivery System Type Viral transduction
Vector Type 🔄 Adeno-associated virus
Viral Vector Subtype AAVrh10
Dose 1 2.0E13 gc/kg
Dose 2 6E10 vg/kg

Study Record Dates


Current Phase 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