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

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

Recruiting
18-75 years
All
Phase 1/2
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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.

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.

Eligibility

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

Study details
    Hypertrophic Cardiomyopathy

NCT05836259

Tenaya Therapeutics

15 May 2026

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