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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.
The study will consist of 2 escalating dose cohorts (groups). The study will enroll at least 6 and as many as 30 patients. All patients will receive active drug (TN-201 Gene Therapy). The study will follow patients for 5 years following a single dose of TN-201.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Experimental | Dose for Cohort 1 will be 3E13 vg/kg |
|
| Cohort 2 | Experimental | Dose for Cohort 2 will be 6E13 vg/kg |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TN-201 | Genetic | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Number and severity of Adverse Events over the course of the study. | 5 Years | |
| Number of Serious Adverse Events related to study drug. | 5 Years |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline to Week 52 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS). | The score ranges from 0-100, with zero being a worse outcome | 52 Weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Expression levels of vector genomes and transgene messenger ribonucleic acid (mRNA) and MyBP-C protein in right ventricular (RV) septal biopsy samples at Pre-Treatment, Week 26, and Week 52. | 52 Weeks | |
| Change from Pre-Treatment period in N-terminal pro B-type natriuretic peptide (NTproBNP), and high-sensitivity cardiac troponin I (hs-cTnI) levels. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Matt Pollman, M.D. | Contact | 650-209-8092 | mpollman@tenayathera.com | |
| LaTanya Tomlinson, RN, MHSA | Contact | 650-825-6990 | clinical.trials@tenayathera.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research | Recruiting | La Jolla | California | 92093 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39302117 | Derived | Grisorio L, Bongianino R, Gianeselli M, Priori SG. Gene therapy for cardiac diseases: methods, challenges, and future directions. Cardiovasc Res. 2024 Nov 25;120(14):1664-1682. doi: 10.1093/cvr/cvae207. |
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This is a first-in-human, non-randomized, open label Phase 1b/2 study. The study will consist of 2 escalating dose cohorts. The study will enroll at least 6 and as many as 30 patients. All patients will receive active drug.
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| 5 Years |
| Percentage of patients who had a change in New York Heart Association (NYHA) Functional Class from baseline. | 5 Years |
| Change from baseline in echocardiography measurements of indexed left atrial and left ventricular (LV) volumes. | 5 Years |
| Change from baseline in echocardiography measurements of LV mass. | 5 Years |
| Change from baseline in echocardiography measurements of maximal LV wall thickness. | 5 Years |
| Change from baseline in echocardiography measurements of global longitudinal strain. | 5 Years |
| Change from baseline in 6MWT distance. | 5 Years |
| Change from baseline peak exercise capacity (pVO2). | 5 Years |
| University of California San Francisco | Recruiting | San Francisco | California | 94117 | United States |
|
| Emory University | Withdrawn | Atlanta | Georgia | 30322 | United States |
| Brigham and Women's Hospital | Withdrawn | Boston | Massachusetts | 02115 | United States |
| Mayo Clinic | Recruiting | Rochester | Minnesota | 55905 | United States |
|
| The Christ Hospital Physicians - The Ohio Heart and Vascular Center | Withdrawn | Cincinnati | Ohio | 45219 | United States |
| Cleveland Clinic | Recruiting | Cleveland | Ohio | 44195 | United States |
|
| Oregon Health & Science University | Withdrawn | Portland | Oregon | 97239 | United States |
| Hospital of the University of Pennsylvania | Withdrawn | Philadelphia | Pennsylvania | 19104 | United States |
| Houston Methodist Hospital | Recruiting | Houston | Texas | 77030 | United States |
|
| ID | Term |
|---|---|
| D002312 | Cardiomyopathy, Hypertrophic |
| D024741 | Cardiomyopathy, Hypertrophic, Familial |
| ID | Term |
|---|---|
| D009202 | Cardiomyopathies |
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
| D001020 | Aortic Stenosis, Subvalvular |
| D001024 | Aortic Valve Stenosis |
| D000082862 | Aortic Valve Disease |
| D006349 | Heart Valve Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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