Actively Recruiting
First-in-Human Study of TN-401 Gene Therapy for Adults With PKP2 Mutation-Associated Arrhythmogenic Right Ventricular Cardiomyopathy Evaluating Safety and Preliminary Effects
Led by Tenaya Therapeutics · Updated on 2025-02-06
15
Participants Needed
7
Research Sites
178 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and early effects of TN-401 gene therapy in adults with symptomatic arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by PKP2 gene mutations. This first-in-human study focuses on how well this gene therapy is tolerated and its preliminary impact on the condition. The study is in its initial phase and aims to understand both safety and gene expression in the heart. Participants are divided into two groups receiving different doses of TN-401, a one-time intravenous gene therapy using a recombinant adeno-associated virus containing the PKP2 gene. The first group receives a dose of 3 x 10^13 vector genomes per kilogram, and the second group receives 6 x 10^13 vector genomes per kilogram. After safety reviews by an independent board, dosing for each group may be expanded. During the study, participants will be monitored for side effects and serious adverse events over one to five years. Researchers will track daily heart rhythm irregularities, frequency of implantable defibrillator therapies, and sustained ventricular tachycardia episodes over 52 weeks. The study involves regular assessments to check heart function, device performance, and gene therapy effects, ensuring close safety and treatment monitoring throughout the participation period.
CONDITIONS
Brief 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...
- Documented PKP2 mutation classified as pathogenic or likely pathogenic
- Diagnosis of Arrhythmogenic Right Ventricular Cardiomyopathy meeting the 2010 revised Task Force Criteria
- Left Ventricular Ejection Fraction of 50% or higher
- Functioning implantable cardiac defibrillator with remote monitoring for at least 9 months before screening
- New York Heart Association (NYHA) Functional Class I, II, or III
- Presence of frequent premature ventricular contractions (PVCs)
You will not qualify if you...
- Ventricular tachycardia (VT) ablation performed within 6 months before screening or planned within 6 months after screening
- High neutralizing antibody levels against AAV9
- History of prior myocardial infarction
- Diagnosis of right ventricular heart failure
- New York Heart Association (NYHA) 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
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 1 day
Participants receive a single intravenous dose of TN-401 gene therapy to assess safety and preliminary efficacy.
1 dosing visit (in-person)
Duration - Up to 5 years
Participants are monitored for safety, adverse events, and preliminary treatment effects over an extended period.
Regular visits during first year with additional long-term safety monitoring visits
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.
N
Niha Kamat
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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