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.
MYBPC3 mutation
Hypertrophic Cardiomyopathy (obstructive and nonobstructive)
Left Ventricular Ejection Fraction ≥45%
NYHA Functional Class II or III symptoms
High AAV9 neutralizing antibody titer
Sequential
TN-201
GeneticTN-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.
Number and severity of Adverse Events over the course of the study.
Number of Serious Adverse Events related to study drug.
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
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.
Change from Pre-Treatment period in N-terminal pro B-type natriuretic peptide (NTproBNP), and high-sensitivity cardiac troponin I (hs-cTnI) levels.
Percentage of patients who had a change in New York Heart Association (NYHA) Functional Class from baseline.
Change from baseline in echocardiography measurements of indexed left atrial and left ventricular (LV) volumes.
Click on the lead sponsor to view all of their trials.
