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TN-401 Gene Therapy for PKP2-related ARVC

This study is testing a new gene therapy called TN-401 for adults with Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC), a heart condition caused by a specific genetic change (PKP2 mutation). TN-401 is a one-time intravenous (into the vein) dose designed to deliver a healthy copy of the PKP2 gene. The main goals are to see how safe TN-401 is by tracking any side effects over 52 weeks and serious side effects over 5 years. You might be able to join if you are between 18 and 65 years old, have a confirmed PKP2 mutation and ARVC, and meet other heart health criteria, including having a functioning implanted defibrillator. The study plans to enroll up to 15 participants.

Study design
This is an open-label study, meaning you and your doctors will know which treatment you are receiving. It will involve up to 15 participants in two dose groups, each receiving a single intravenous dose of TN-401.
What's involved
You would receive a single intravenous dose of TN-401. The study will track your safety for 52 weeks and serious side effects for 5 years.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for adverse events for 52 weeks and for serious adverse events related to the study drug for 5 years.

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NCT06228924

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

Recruiting
PHASE1Ages 18–65InterventionalTreatment
Tenaya Therapeutics
~15 participants
Updated 2025-02-06 on ClinicalTrials.gov
What's tested:TN-401

At a glance

Recruiting sites
7 of 7 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number and severity of Adverse Events over the course of the study.
Measured over 52 weeks
+1 more outcome measured

Conditions

Arrhythmogenic Right Ventricular Cardiomyopathy

Explore related trials

Arrhythmogenic Right Ventricular DysplasiaPKP2 Gene

Where it's being run

7 sites across 7 states
California1
Colorado1
Maryland1
Massachusetts1
Minnesota1
New York1
Ohio1

Who to contact

Matthew Pollman, M.D.
[email protected]
(650) 416-1186
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Number and severity of Adverse Events over the course of the study.52 weeks
  • Number of Serious Adverse Events related to study drug.5 years