Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM

Launched by TENAYA THERAPEUTICS · Apr 18, 2023

Keywords

ClinConnect Summary

This clinical trial is looking at a new treatment called TN-201 for adults who have a specific genetic mutation (MYBPC3) that causes a heart condition known as hypertrophic cardiomyopathy (HCM). HCM can lead to symptoms like shortness of breath and fatigue. The study aims to find out how safe this treatment is and how well it works in people who have nonobstructive HCM, meaning their heart has thickened but blood flow isn’t blocked. This is the first time TN-201 is being tested in humans.

To participate in the trial, you need to have the MYBPC3 mutation and be diagnosed with nonobstructive HCM. You should also have a heart function score that shows your heart is working reasonably well and be experiencing moderate symptoms. The study is open to adults aged 65 to 74 or those with other specific health criteria. If you join the study, you’ll be closely monitored for safety and how your body responds to the treatment. It's important to know that this trial is still recruiting participants, so if you're eligible, you might have the opportunity to help researchers learn more about this potential new treatment.

Gender

ALL

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