Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: TN-201.
- Who it may be relevant to
- Registry conditions: Hypertrophic Cardiomyopathy. Basic parameters: 18 years — 75 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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)
Overview
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.
Detailed description
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.
Interventions
- Genetic TN-201
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.
Primary outcome measures
- Number and severity of Adverse Events over the course of the study. [Time frame: 5 Years]
- Number of Serious Adverse Events related to study drug. [Time frame: 5 Years]
Secondary outcome measures (1)
- Change from baseline to Week 52 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS). [Time frame: 52 Weeks]
Eligibility 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 10 centers
- UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Re — La Jolla
- University of California San Francisco — San Francisco
- Emory University — Atlanta
- Brigham and Women's Hospital — Boston
- Mayo Clinic — Rochester
- The Christ Hospital Physicians - The Ohio Heart and Vascular Center — Cincinnati
- Cleveland Clinic — Cleveland
- Oregon Health & Science University — Portland
- … and 2 more centers
Publications
- 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. PMID 39302117
Identifiers
NCT: NCT05836259 · TN-201-0009