NCT06228924RecruitingPhase 1
Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
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)
Source: ClinicalTrials.gov · Last updated 6 Feb 2025 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 15 (estimated)
- First submitted
- 18 Jan 2024
- Start date
- 26 Mar 2024
- Primary completion date
- 1 May 2026
- Lead sponsor
- Tenaya Therapeutics
- Diseases
- Heart failure
- NCT number
- NCT06228924
What you commit to
- Study type: Interventional · Phase 1
- Planned participants: 15
- From 26 Mar 2024 to the primary completion date, 1 May 2026
- 7 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- High AAV9 neutralizing antibody titer
- Prior myocardial infarction
Other illness
- Right Ventricular Heart Failure
- Class IV Heart Failure
- Clinically significant renal disease
- Clinically significant liver disease
Other
- Ventricular tachycardia (VT) ablation within 6 months of Screening or planned VT ablation within 6 months after Screening
Who the study is looking for
- 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)
Age in the registry: 18 years to 65 years · Sex: All · Does not accept healthy volunteers
Sites (7)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University of Colorado Anschutz Medical Campus Coordinator of this site? Confirm its status | Aurora | United States | Recruiting |
| Johns Hopkins University Coordinator of this site? Confirm its status | Baltimore | United States | Recruiting |
| Brigham and Women's Hospital Coordinator of this site? Confirm its status | Boston | United States | Recruiting |
| Cleveland Clinic Coordinator of this site? Confirm its status | Cleveland | United States | Recruiting |
| NYU Langone Health Coordinator of this site? Confirm its status | New York | United States | Recruiting |
| Mayo Clinic Coordinator of this site? Confirm its status | Rochester | United States | Recruiting |
| University of California San Francisco Coordinator of this site? Confirm its status | San Francisco | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.