Evaluating the safety of TN-201 in adults with a specific heart condition
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 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 |
| Enrollment | 30 (estimated) |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Sponsor | Tenaya Therapeutics Industry-sponsored |
| Locations | 10 sites (La Jolla, California and 9 other locations) |
| Trial ID | NCT05836259 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are adults with symptomatic hypertrophic cardiomyopathy caused by MYBPC3 mutations.
Not a fit: Patients without MYBPC3 mutations or those with high AAV9 neutralizing antibody titers may not benefit from this study.
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 performed: While this approach is novel, similar gene therapies have shown promise in other conditions, indicating potential for success.
Eligibility criteria
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 this trial is running
La Jolla, California and 9 other locations
- 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)
- Emory University — Atlanta, Georgia, United States (Withdrawn)
- Brigham and Women's Hospital — Boston, Massachusetts, United States (Withdrawn)
- Mayo Clinic — Rochester, Minnesota, United States (Recruiting)
- The Christ Hospital Physicians - The Ohio Heart and Vascular Center — Cincinnati, Ohio, United States (Withdrawn)
- Cleveland Clinic — Cleveland, Ohio, United States (Recruiting)
- Oregon Health & Science University — Portland, Oregon, United States (Withdrawn)
- Hospital of the University of Pennsylvania — Philadelphia, Pennsylvania, United States (Withdrawn)
- Houston Methodist Hospital — Houston, Texas, United States (Recruiting)
Study contacts
- Study coordinator: Matt Pollman, M.D.
- Email: mpollman@tenayathera.com
- Phone: 650-209-8092
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.