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)
Status: Recruiting
Location: See all (7) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1
SUMMARY
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 65
Healthy Volunteers: f
View:
• 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)
Locations
United States
California
University of California San Francisco
RECRUITING
San Francisco
Colorado
University of Colorado - Anschutz Medical Campus
RECRUITING
Aurora
Massachusetts
Brigham and Women's Hospital
RECRUITING
Boston
Maryland
Johns Hopkins University
RECRUITING
Baltimore
Minnesota
Mayo Clinic
RECRUITING
Rochester
New York
NYU Langone Health
RECRUITING
New York
Ohio
Cleveland Clinic
RECRUITING
Cleveland
Contact Information
Primary
Matthew Pollman, M.D.
mpollman@tenayathera.com
(650) 416-1186
Backup
Niha Kamat
clinical.trials@tenayathera.com
Time Frame
Start Date: 2024-03-26
Estimated Completion Date: 2029-10-01
Participants
Target number of participants: 15
Treatments
Experimental: Cohort 1
Dose for Cohort 1 will be 3E13 vg/kg
Experimental: Cohort 2
Dose for Cohort 2 will be 6E13 vg/kg
Related Therapeutic Areas
Sponsors
Leads: Tenaya Therapeutics