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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)
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.
The study will consist of 2 escalating dose cohorts (groups). The study will enroll at least 6 and as many as 30 patients. All patients will receive active drug (TN-201 Gene Therapy). The study will follow patients for 5 years following a single dose of TN-201.
Age
18 - 75 years
Sex
ALL
Healthy Volunteers
No
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
Start Date
August 10, 2023
Primary Completion Date
August 1, 2027
Completion Date
August 1, 2032
Last Updated
March 19, 2026
30
ESTIMATED participants
TN-201
GENETIC
Lead Sponsor
Tenaya Therapeutics
Data Source & Attribution
This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.
Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.
View ClinicalTrials.gov Terms and ConditionsNCT07382128