Actively Recruiting
Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
Led by Tenaya Therapeutics · Updated on 2025-02-06
15
Participants Needed
7
Research Sites
287 weeks
Total Duration
On this page
AI-Summary
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.
CONDITIONS
Official Title
Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed pathogenic or likely pathogenic PKP2 mutation
- Diagnosis of Arrhythmogenic Right Ventricular Cardiomyopathy based on 2010 revised Task Force Criteria
- Left Ventricular Ejection Fraction of 50% or higher
- Functioning implantable cardiac defibrillator with remote integration capabilities for at least 9 months before screening
- New York Heart Association Functional Class I, II, or III
- Frequent premature ventricular contractions (PVCs)
You will not qualify if you...
- Ventricular tachycardia ablation within 6 months before screening or planned within 6 months after screening
- High neutralizing antibody titer against AAV9
- Previous myocardial infarction
- Right ventricular heart failure
- Class IV heart failure
- Clinically significant kidney disease
- Clinically significant liver disease
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 7 locations
1
University of California San Francisco
San Francisco, California, United States, 94143
Actively Recruiting
2
University of Colorado - Anschutz Medical Campus
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Johns Hopkins University
Baltimore, Maryland, United States, 21287
Actively Recruiting
4
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
5
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
6
NYU Langone Health
New York, New York, United States, 10016
Actively Recruiting
7
Cleveland Clinic
Cleveland, Ohio, United States, 44195
Actively Recruiting
Research Team
M
Matthew Pollman, M.D.
CONTACT
N
Niha Kamat
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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