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Gene Therapy Trial Report

Summary

Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC


NCTID NCT06228924 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Arrhythmogenic Right Ventricular Cardiomyopathy
Disease Ontology Term DOID:0050431
Drug Product Name TN-401
Sponsor Tenaya Therapeutics
Funder Type Industry
Recruitment Status
Enrollment Count 15 (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 AAV9
Dose 1 3E13 vg/kg
Dose 2 6E13 vg/kg

Study Record Dates


Current Phase Phase1
Submit Date 2024-01-18
Completion Date 2029-10-01
Last Update 2025-02-06

Participation Criteria


Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * PKP2 mutation (pathogenic or likely pathogenic) * Arrhythmogenic Right Ventricular Cardiomyopathy as defined by the 2010 revised Task Force Criteria * Left Ventricular Ejection Fraction ≥50% * Functioning Implantable Cardiac Defibrillator with remote integration capabilities at least 9 months prior to Screening * NYHA Functional Class I, II, or III * Frequent premature ventricular contractions (PVCs) Exclusion Criteria: * Ventricular tachycardia (VT) ablation within 6 months of Screening or planned VT ablation within 6 months after Screening * High AAV9 neutralizing antibody titer * Prior myocardial infarction * Right Ventricular Heart Failure * Class IV Heart Failure * Clinically significant renal disease * Clinically significant liver disease
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 7
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates First patient dosed November 2024, initial data announced December 2025

Resources/Links