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Clinical Trial Report

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
Recruiting
Enrollment Count 10
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
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAVrh10
Editor Type none
Dose 1 Starting dose: 2.0E13 gc/kg
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2023-10-25
Completion Date 2027-02
Last Update 2025-01-23

Participation Criteria

Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 5
Locations United States

Regulatory Information

Has US IND True
FDA Designations Fast Track, Orphan Drug Designation
Recent Updates Initial clinical data expected late Q1/ early Q2 of 2025

Resources/Links

News and Press Releases
Preclinical Publications