Tests TN-201 gene therapy in adults with inherited heart thickening disease
Tests TN-201 gene therapy for hypertrophic cardiomyopathy caused by MYBPC3 gene changes in adults.
Plain-language title generated from the registry record
Registry title: Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM
Key facts
- Where
- Atlanta, Boston, Cincinnati, Cleveland, Houston, La Jolla, Philadelphia, Portland, Rochester, and San Francisco, United StatesSee the 10 sites
- Phase
- Phase 1/2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 75 years old?
Source: NCT05836259, who can take part
Where in United States
- Emory UniversityAtlanta, United StatesNo longer taking part
- Brigham and Women's HospitalBoston, United StatesNo longer taking part
- The Christ Hospital Physicians - The Ohio Heart and Vascular CenterCincinnati, United StatesNo longer taking part
- Cleveland ClinicCleveland, United StatesTaking people
- Houston Methodist HospitalHouston, United StatesTaking people
- UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical ResearchLa Jolla, United StatesTaking people
- Hospital of the University of PennsylvaniaPhiladelphia, United StatesNo longer taking part
- Oregon Health Science UniversityPortland, United StatesNo longer taking part
- Mayo ClinicRochester, United StatesTaking people
- University of California San FranciscoSan Francisco, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Tenaya Therapeutics
- Trial code
- NCT05836259
- Record updated
- 19 March 2026
- Original description
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.Read full original descriptionShow less
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.
Inclusion: MYBPC3 mutation; Hypertrophic Cardiomyopathy (obstructive and nonobstructive); Left Ventricular Ejection Fraction ≥45%; NYHA Functional Class II or III symptoms; NT-proBNP ≥160pg/ml.
Exclusion: High AAV9 neutralizing antibody titer.