Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
Recruiting now · Phase 1
Conditions studied: Arrhythmogenic Right Ventricular Cardiomyopathy
In brief
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.
Key facts
- Study ID
- NCT06228924
- Run by
- Tenaya Therapeutics
- People needed
- 15
- Starts
- 2024-03-26
- Expected to finish
- 2029-10-01
- Last updated by the study team
- 2025-02-06
Who can join
Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.
You may qualify 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
- Frequent premature ventricular contractions (PVCs)
You may not qualify 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
- Clinically significant liver disease
Where it is running
- University of California San Francisco — San Francisco, California, United States (enrolling)
- University of Colorado - Anschutz Medical Campus — Aurora, Colorado, United States (enrolling)
- Johns Hopkins University — Baltimore, Maryland, United States (enrolling)
- Brigham and Women's Hospital — Boston, Massachusetts, United States (enrolling)
- Mayo Clinic — Rochester, Minnesota, United States (enrolling)
- NYU Langone Health — New York, New York, United States (enrolling)
- Cleveland Clinic — Cleveland, Ohio, United States (enrolling)
Full record on ClinicalTrials.gov
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