NCT05836259RecruitingPhase 1/2
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)
Source: ClinicalTrials.gov · Last updated 19 Mar 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 30 (estimated)
- First submitted
- 18 Apr 2023
- Start date
- 10 Aug 2023
- Primary completion date
- Aug 2027
- Lead sponsor
- Tenaya Therapeutics
- Diseases
- Hypertrophic cardiomyopathy
- NCT number
- NCT05836259
What you commit to
- Study type: Interventional · Phase 1/2
- Planned participants: 30
- From 10 Aug 2023 to the primary completion date, Aug 2027
- 10 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- High AAV9 neutralizing antibody titer
Who the study is looking for
- MYBPC3 mutation
- Hypertrophic Cardiomyopathy (obstructive and nonobstructive)
- Left Ventricular Ejection Fraction ≥45%
- NYHA Functional Class II or III symptoms
- NT-proBNP ≥160pg/ml
Age in the registry: 18 years to 75 years · Sex: All · Does not accept healthy volunteers
Sites (10)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Emory University Coordinator of this site? Confirm its status | Atlanta | United States | Withdrawn |
| Brigham and Women's Hospital Coordinator of this site? Confirm its status | Boston | United States | Withdrawn |
| The Christ Hospital Physicians - The Ohio Heart and Vascular Center Coordinator of this site? Confirm its status | Cincinnati | United States | Withdrawn |
| Cleveland Clinic Coordinator of this site? Confirm its status | Cleveland | United States | Recruiting |
| Houston Methodist Hospital Coordinator of this site? Confirm its status | Houston | United States | Recruiting |
| UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research Coordinator of this site? Confirm its status | La Jolla | United States | Recruiting |
| Hospital of the University of Pennsylvania Coordinator of this site? Confirm its status | Philadelphia | United States | Withdrawn |
| Oregon Health Science University Coordinator of this site? Confirm its status | Portland | United States | Withdrawn |
| Mayo Clinic Coordinator of this site? Confirm its status | Rochester | United States | Recruiting |
| University of California San Francisco Coordinator of this site? Confirm its status | San Francisco | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.