Home › Hypertrophic Cardiomyopathy › NCT05836259
Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM
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)
Who can join
Ages 18 to 75 · All sexes
Full eligibility criteria
Inclusion Criteria: * MYBPC3 mutation * Hypertrophic Cardiomyopathy (obstructive and nonobstructive) * Left Ventricular Ejection Fraction ≥45% * NYHA Functional Class II or III symptoms * NT-proBNP ≥160pg/ml Exclusion Criteria: * High AAV9 neutralizing antibody titer
About the study
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.
What is being tested
- TN-201 (genetic)
Sponsor: Tenaya Therapeutics · Participants: 30 · Started: Aug 10, 2023
Contact the study team
- Matt Pollman, M.D. · Phone: 650-209-8092
- LaTanya Tomlinson, RN, MHSA · Phone: 650-825-6990
Official record on ClinicalTrials.gov — NCT05836259
Locations in the U.S.
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.