IRB Study Number 23-471
Status Recruiting
Location Cleveland Clinic Main Campus
Institute Heart and Vascular Institute
Description
This first-in-human (FIH) study will evaluate the safety, tolerability, pharmacodynamics (PD), and efficacy of a single IV administration of TN-201 in adult patients (>18 years of age) with MYBPC3 mutation-associated non-obstructive HCM. TN-201 uses the adeno-associated virus 9 (AAV-9) vector to deliver a wild-type copy of the MYBPC3 gene that is expressed specifically in cardiac myocytes. The study will consist of 2 sequential dose level cohorts.
Inclusion Criteria
>18 years of age
Positive for MYBPC3 genetic mutation and hypertrophic cardiomyopathy
Has an implantable cardioverter-defibrillator
Exclusion Criteria
Clinically significant liver disease
History of HCV, HBV, or HIV
Previously dosed with AAV-based gene therapy
