Arrhythmogenic Right Ventricular Cardiomyopathy study: TN-401
Official title Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
ClinicalTrials.gov ID: NCT06228924
What this study is testing
What is TN-401?
TN-401 is an investigational single‑dose intravenous gene therapy being studied for Arrhythmogenic Right Ventricular Cardiomyopathy. is designed to deliver a functional copy of the PKP2 gene to heart cells using an AAV9 viral vector.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study aims to see how safe TN-401 is and whether it shows early signs of working in these patients.
- Who it's for
- Adults who have symptomatic arrhythmogenic right ventricular cardiomyopathy caused by a PKP2 mutation.
- What's involved
- Participants will receive the TN‑401 gene therapy and attend scheduled visits for monitoring and tests.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 65
You may be able to join if
- 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
You likely can't join if
- 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
See the full eligibility 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)
- 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
The study team makes the final eligibility decision.
Where it is enrolling
- San Francisco, California, United States
- Aurora, Colorado, United States
- Baltimore, Maryland, United States
- Boston, Massachusetts, United States
- Rochester, Minnesota, United States
- New York, New York, United States
- Cleveland, Ohio, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at San Francisco, California, United States; Aurora, Colorado, United States; Baltimore, Maryland, United States; Boston, Massachusetts, United States; Rochester, Minnesota, United States; New York, New York, United States and 1 more location. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.