Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
Recruiting · NCT06228924 · Interventional (participants receive a specific treatment) · Lead sponsor: Tenaya Therapeutics
View the official record on ClinicalTrials.gov →Interventions studied
TN-401
What this trial is about
This first-in-human study is designed to evaluate the safety, and preliminary efficacy (PD) of TN-401 gene therapy in adult patients with symptomatic PKP2 mutation-associated ARVC.
Who can take part
Age range
18 Years to 65 Years
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 1
Study type
Interventional (participants receive a specific treatment)
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
Where it is running
7 locations listed across 7 US states.
- University of California San Francisco - San Francisco, California, United States
- University of Colorado - Anschutz Medical Campus - Aurora, Colorado, United States
- Johns Hopkins University - Baltimore, Maryland, United States
- Brigham and Women's Hospital - Boston, Massachusetts, United States
- Mayo Clinic - Rochester, Minnesota, United States
- NYU Langone Health - New York, New York, United States
- Cleveland Clinic - Cleveland, Ohio, United States