Hypertrophic Cardiomyopathy Clinical Trial
Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM
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 MYBPC3 mutation-associated nonobstructive hypertrophic cardiomyopathy (nHCM).
Full Description
The study will consist of 2 escalating dose cohorts. The study will enroll at least 6 and as many as 15 patients. All patients will receive active drug. The study will follow patients for 5 years following a single dose of TN-201.
Eligibility Criteria
Inclusion Criteria:
MYBPC3 mutation
Nonobstructive Hypertrophic Cardiomyopathy
Left Ventricular Ejection Fraction ≥50%
Functioning Implantable Cardiac Defibrillator
NYHA Functional Class II or III symptoms
NT-proBNP ≥300pg/ml
Exclusion Criteria:
High AAV9 neutralizing antibody titer
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There are 12 Locations for this study
La Jolla California, 92093, United States
San Francisco California, 94117, United States
Atlanta Georgia, 30322, United States
Boston Massachusetts, 02115, United States
Rochester Minnesota, 55905, United States
Cincinnati Ohio, 45219, United States
Cleveland Ohio, 44195, United States
Portland Oregon, 97239, United States
Philadelphia Pennsylvania, 19104, United States
Pittsburgh Pennsylvania, 15213, United States
Houston Texas, 77030, United States
Salt Lake City Utah, 84132, United States
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