Evaluating the safety of TN-201 in adults with a specific heart condition
Official title: 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)
Run by Tenaya Therapeutics · NCT05836259 · Data updated 2026-11-12
This study is testing a new gene therapy called TN-201 to see if it is safe for adults with a specific heart condition related to MYBPC3 mutations.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Enrollment | 30 (estimated) |
| Sponsor | Tenaya Therapeutics Industry-sponsored |
| Locations | 10 sites (La Jolla, California and 9 other locations) |
| Status | Recruiting |
| Started | August 10, 2023 |
| Main results expected | August 2027 |
| Study ends | August 2032 |
| Registry record updated | March 19, 2026 |
| Trial ID | NCT05836259 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are adults with symptomatic hypertrophic cardiomyopathy caused by MYBPC3 mutations.
✕Probably not a fit
Patients without MYBPC3 mutations or those with high AAV9 neutralizing antibody titers may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This study is designed to assess the safety and tolerability of TN-201, a gene therapy, in adults suffering from symptomatic hypertrophic cardiomyopathy (HCM) linked to MYBPC3 mutations. It involves a single-ascending dose approach where participants will receive the active drug and be monitored for effects over a five-year period. The study will include two escalating dose cohorts and aims to enroll between 6 to 30 patients. The focus is on understanding the pharmacodynamics of TN-201 in this specific patient population.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with MYBPC3 mutation-associated HCM.
How similar studies have gone: While this approach is novel, similar gene therapies have shown promise in other conditions, indicating potential for success.
Who can join: the full rules
Show full inclusion / exclusion 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
Where it's running
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UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research
La Jolla, California, United States Recruiting -
University of California San Francisco
San Francisco, California, United States Recruiting -
Mayo Clinic
Rochester, Minnesota, United States Recruiting -
Cleveland Clinic
Cleveland, Ohio, United States Recruiting -
Houston Methodist Hospital
Houston, Texas, United States Recruiting -
Emory University
Atlanta, Georgia, United States Withdrawn
Show 4 more locations
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Brigham and Women's Hospital
Boston, Massachusetts, United States Withdrawn -
The Christ Hospital Physicians - The Ohio Heart and Vascular Center
Cincinnati, Ohio, United States Withdrawn -
Oregon Health & Science University
Portland, Oregon, United States Withdrawn -
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States Withdrawn
Study contacts
- Study coordinator: Matt Pollman, M.D.
- Email: mpollman@tenayathera.com
- Phone: 650-209-8092
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Mavacamten for symptomatic obstructive hypertrophic cardiomyopathy in Chinese adults
This project will test how well mavacamten works for Chinese adults with symptomatic obstructive hypertrophic cardiomyopathy when used in everyday clinical care.
-
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This study tests the long-term safety of the oral drug HRS-1893 in people with hypertrophic cardiomyopathy who previously received HRS-1893.
-
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Comparing inspiratory muscle training and Baduanjin exercises for heart health
This study tests whether breathing exercises or a special exercise program can help people with hypertrophic cardiomyopathy improve their lung function, exercise ability, and overall quality of life.
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Using advanced imaging to evaluate heart muscle scarring in various heart conditions
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.