Ignite Creation Date:
2025-12-24 @ 2:25 PM
Ignite Modification Date:
2025-12-26 @ 1:08 AM
Study NCT ID:
NCT05836259
Status:
RECRUITING
Last Update Posted:
2025-07-15
First Post:
2023-04-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D002312', 'term': 'Cardiomyopathy, Hypertrophic'}, {'id': 'D024741', 'term': 'Cardiomyopathy, Hypertrophic, Familial'}], 'ancestors': [{'id': 'D009202', 'term': 'Cardiomyopathies'}, {'id': 'D006331', 'term': 'Heart Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D001020', 'term': 'Aortic Stenosis, Subvalvular'}, {'id': 'D001024', 'term': 'Aortic Valve Stenosis'}, {'id': 'D000082862', 'term': 'Aortic Valve Disease'}, {'id': 'D006349', 'term': 'Heart Valve Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': '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.'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 30}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2023-08-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-07', 'completionDateStruct': {'date': '2032-08', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-07-10', 'studyFirstSubmitDate': '2023-04-18', 'studyFirstSubmitQcDate': '2023-04-18', 'lastUpdatePostDateStruct': {'date': '2025-07-15', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-05-01', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-08', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': '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.', 'timeFrame': '52 Weeks'}, {'measure': 'Change from Pre-Treatment period in N-terminal pro B-type natriuretic peptide (NTproBNP), and high-sensitivity cardiac troponin I (hs-cTnI) levels.', 'timeFrame': '5 Years'}, {'measure': 'Percentage of patients who had a change in New York Heart Association (NYHA) Functional Class from baseline.', 'timeFrame': '5 Years'}, {'measure': 'Change from baseline in echocardiography measurements of indexed left atrial and left ventricular (LV) volumes.', 'timeFrame': '5 Years'}, {'measure': 'Change from baseline in echocardiography measurements of LV mass.', 'timeFrame': '5 Years'}, {'measure': 'Change from baseline in echocardiography measurements of maximal LV wall thickness.', 'timeFrame': '5 Years'}, {'measure': 'Change from baseline in echocardiography measurements of global longitudinal strain.', 'timeFrame': '5 Years'}, {'measure': 'Change from baseline in 6MWT distance.', 'timeFrame': '5 Years'}, {'measure': 'Change from baseline peak exercise capacity (pVO2).', 'timeFrame': '5 Years'}], 'primaryOutcomes': [{'measure': 'Number and severity of Adverse Events over the course of the study.', 'timeFrame': '5 Years'}, {'measure': 'Number of Serious Adverse Events related to study drug.', 'timeFrame': '5 Years'}], 'secondaryOutcomes': [{'measure': 'Change from baseline to Week 52 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS).', 'timeFrame': '52 Weeks', 'description': 'The score ranges from 0-100, with zero being a worse outcome'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Hypertrophic Cardiomyopathy (HCM)', 'Myosin Binding Protein C3 (MYBPC3)', 'Nonobstructive HCM', 'Genetic HCM', 'Familial HCM', 'Adenoassociated Virus (AAV)', 'Gene Therapy', 'Obstructive HCM', 'nHCM', 'oHCM'], 'conditions': ['Hypertrophic Cardiomyopathy']}, 'referencesModule': {'references': [{'pmid': '39302117', 'type': 'DERIVED', 'citation': '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.'}]}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* MYBPC3 mutation\n* Hypertrophic Cardiomyopathy (obstructive and nonobstructive)\n* Left Ventricular Ejection Fraction ≥45%\n* NYHA Functional Class II or III symptoms\n* NT-proBNP ≥160pg/ml\n\nExclusion Criteria:\n\n* High AAV9 neutralizing antibody titer'}, 'identificationModule': {'nctId': 'NCT05836259', 'acronym': 'MyPEAK-1', 'briefTitle': 'Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM', 'organization': {'class': 'INDUSTRY', 'fullName': 'Tenaya Therapeutics'}, 'officialTitle': '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)', 'orgStudyIdInfo': {'id': 'TN-201-0009'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1', 'description': 'Dose for Cohort 1 will be 3E13 vg/kg', 'interventionNames': ['Genetic: TN-201']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2', 'description': 'Dose for Cohort 2 will be 6E13 vg/kg', 'interventionNames': ['Genetic: TN-201']}], 'interventions': [{'name': 'TN-201', 'type': 'GENETIC', 'description': '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.', 'armGroupLabels': ['Cohort 1', 'Cohort 2']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92093', 'city': 'La Jolla', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Connor Davies', 'role': 'CONTACT', 'email': 'crdavies@health.ucsd.edu', 'phone': '858-534-3798'}], 'facility': 'UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research', 'geoPoint': {'lat': 32.84727, 'lon': -117.2742}}, {'zip': '94117', 'city': 'San Francisco', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Emma Reagan', 'role': 'CONTACT', 'email': 'Emma.reagan@ucsf.edu', 'phone': '415-514-7280'}], 'facility': 'University of California San Francisco', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'status': 'WITHDRAWN', 'country': 'United States', 'facility': 'Emory University', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'status': 'WITHDRAWN', 'country': 'United States', 'facility': "Brigham and Women's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55905', 'city': 'Rochester', 'state': 'Minnesota', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Sierra Slade', 'role': 'CONTACT', 'email': 'Slade.Sierra@mayo.edu', 'phone': '507-422-5433'}], 'facility': 'Mayo Clinic', 'geoPoint': {'lat': 44.02163, 'lon': -92.4699}}, {'zip': '45219', 'city': 'Cincinnati', 'state': 'Ohio', 'status': 'WITHDRAWN', 'country': 'United States', 'facility': 'The Christ Hospital Physicians - The Ohio Heart and Vascular Center', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '44195', 'city': 'Cleveland', 'state': 'Ohio', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Danielle Kellner', 'role': 'CONTACT', 'email': 'Kellned@ccf.org', 'phone': '216-445-6321'}], 'facility': 'Cleveland Clinic', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '97239', 'city': 'Portland', 'state': 'Oregon', 'status': 'WITHDRAWN', 'country': 'United States', 'facility': 'Oregon Health & Science University', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'status': 'WITHDRAWN', 'country': 'United States', 'facility': 'Hospital of the University of Pennsylvania', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Jennifer Garrett', 'role': 'CONTACT', 'email': 'jmgarrett@houstonmethodist.org', 'phone': '281-222-9983'}], 'facility': 'Houston Methodist Hospital', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}], 'centralContacts': [{'name': 'Matt Pollman, M.D.', 'role': 'CONTACT', 'email': 'mpollman@tenayathera.com', 'phone': '650-209-8092'}, {'name': 'LaTanya Tomlinson, RN, MHSA', 'role': 'CONTACT', 'email': 'clinical.trials@tenayathera.com', 'phone': '650-825-6990'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Tenaya Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}