Gene therapy for adults with PKP2 mutation-associated heart disease
Official title: First-in-Human, Open-Label, Safety, Tolerability, Dose-Finding, Pharmacodynamic and Cardiac Transgene Expression Study of TN-401, a Recombinant Adeno-associated Virus Serotype 9 (AAV9) Containing Plakophilin-2 (PKP2) Transgene, in Adults With PKP2 Mutation-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
Run by Tenaya Therapeutics · NCT06228924 · Data updated 2026-11-12
This study is testing a new gene therapy called TN-401 to see if it can help adults with heart disease caused by a specific genetic mutation feel better and stay safe.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Enrollment | 15 (estimated) |
| Sponsor | Tenaya Therapeutics Industry-sponsored |
| Locations | 7 sites (San Francisco, California and 6 other locations) |
| Status | Recruiting |
| Started | March 26, 2024 |
| Main results expected | May 1, 2026 |
| Study ends | October 1, 2029 |
| Registry record updated | February 6, 2025 |
| Trial ID | NCT06228924 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates for this study are adults with a confirmed PKP2 mutation and symptomatic ARVC who meet specific cardiac function criteria.
✕Probably not a fit
Patients with advanced heart failure, recent ventricular tachycardia ablation, or significant renal or liver disease may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This open-label, dose escalation study aims to evaluate the safety and preliminary efficacy of TN-401 gene therapy in adults suffering from symptomatic PKP2 mutation-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC). The study will enroll up to 15 patients, who will receive a single intravenous dose of TN-401 in two planned dose cohorts. Following a review by the Data Safety Monitoring Board (DSMB), the next dose cohort will be initiated, allowing for careful monitoring of safety and efficacy as the study progresses.
Why it matters
Potential benefit: If successful, this therapy could provide a novel treatment option for patients with PKP2 mutation-associated ARVC, potentially improving their heart function and quality of life.
How similar studies have gone: While gene therapy approaches are emerging, this specific application for PKP2 mutation-associated ARVC is novel and has not been extensively tested in prior studies.
Who can join: the full rules
Show full inclusion / exclusion criteria
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's running
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University of California San Francisco
San Francisco, California, United States Recruiting -
University of Colorado - Anschutz Medical Campus
Aurora, Colorado, United States Recruiting -
Johns Hopkins University
Baltimore, Maryland, United States Recruiting -
Brigham and Women's Hospital
Boston, Massachusetts, United States Recruiting -
Mayo Clinic
Rochester, Minnesota, United States Recruiting -
NYU Langone Health
New York, New York, United States Recruiting
Show 1 more location
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Cleveland Clinic
Cleveland, Ohio, United States Recruiting
Study contacts
- Study coordinator: Matthew Pollman, M.D.
- Email: mpollman@tenayathera.com
- Phone: (650) 416-1186
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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FAPI PET/CT imaging in arrhythmogenic right ventricular cardiomyopathy
This study will test whether a PET/CT scan using a 68Ga-DOTA-SA-FAPI tracer can detect active scarring in adults with ARVC and provide information beyond standard heart imaging.
-
Observing the prevalence of antibodies in patients with a specific heart condition
This study looks at how common certain antibodies are in people with a heart condition called ARVC who have a specific gene mutation, while they receive regular care and share health information over five years.
-
Targeted therapy for arrhythmogenic cardiomyopathy using tideglusib
This study is testing a new medication called tideglusib to see if it can help people with a specific genetic type of arrhythmogenic cardiomyopathy feel better and reduce their risk of serious heart problems.
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Examining exercise effects on heart activity in ARVC patients
This study tests how different types of exercise affect heart activity in people with Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) to help create safer exercise guidelines for them.
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Registry for patients with cardiomyopathies
This study is collecting health information and samples from people with different types of heart muscle diseases to better understand their causes and effects over time.
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Registry and biobank for arrhythmogenic ventricular cardiomyopathy
This study is trying to gather information and samples from people with arrhythmogenic ventricular cardiomyopathy and their families to better understand the genetic causes of the condition and improve early diagnosis for those at risk.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.