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)
Tenaya Therapeutics
30 participants
Aug 10, 2023
INTERVENTIONAL
Conditions
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.
Eligibility
Inclusion Criteria5
- MYBPC3 mutation
- Hypertrophic Cardiomyopathy (obstructive and nonobstructive)
- Left Ventricular Ejection Fraction ≥45%
- NYHA Functional Class II or III symptoms
- NT-proBNP ≥160pg/ml
Exclusion Criteria1
- High AAV9 neutralizing antibody titer
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Interventions
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)
View Full Details on ClinicalTrials.gov
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NCT05836259