Clinical Research Directory
Browse clinical research sites, groups, and studies.
Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM
Sponsor: Tenaya Therapeutics
Summary
This is a first-in-human, non-randomized, open-label study designed to evaluate the safety, tolerability, and pharmacodynamics (PD) of TN-201 in adult patients with symptomatic hypertrophic cardiomyopathy (HCM) caused by mutations in the MYBPC3 gene.
Official title: First-in-Human, Open-Label, Safety, Tolerability, Dose-Finding, Pharmacodynamic and Cardiac Transgene Expression Study of TN-201, a Recombinant Adeno-associated Virus Serotype 9 (AAV9) Containing Myosin Binding Protein C Transgene, in Adults With MYBPC3 Mutation-associated Hypertrophic Cardiomyopathy (HCM)
Key Details
Gender
All
Age Range
18 Years - 75 Years
Study Type
INTERVENTIONAL
Enrollment
30
Start Date
2023-08-10
Completion Date
2032-08
Last Updated
2026-03-19
Healthy Volunteers
No
Conditions
Interventions
TN-201
TN-201 is a recombinant adeno-associated virus serotype 9 (AAV9) containing Myosin Binding Protein C (MYBPC3) transgene. It is a single (one-time) intravenous dose.
Locations (10)
UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research
La Jolla, California, United States
University of California San Francisco
San Francisco, California, United States
Emory University
Atlanta, Georgia, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
Mayo Clinic
Rochester, Minnesota, United States
The Christ Hospital Physicians - The Ohio Heart and Vascular Center
Cincinnati, Ohio, United States
Cleveland Clinic
Cleveland, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States
Houston Methodist Hospital
Houston, Texas, United States