Actively Recruiting

Phase 1
Phase 2
Age: 18Years - 75Years
All Genders
NCT05836259

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

Led by Tenaya Therapeutics · Updated on 2026-03-19

30

Participants Needed

10

Research Sites

468 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 18Years - 75Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Confirmed MYBPC3 gene mutation
  • Diagnosis of hypertrophic cardiomyopathy (obstructive or nonobstructive)
  • Left ventricular ejection fraction of 45% or higher
  • New York Heart Association Functional Class II or III symptoms
  • NT-proBNP level of 160 pg/ml or higher
Not Eligible

You will not qualify if you...

  • High neutralizing antibody levels against the adeno-associated virus serotype 9 (AAV9)

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 10 locations

1

UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research

La Jolla, California, United States, 92093

Actively Recruiting

2

University of California San Francisco

San Francisco, California, United States, 94117

Actively Recruiting

3

Emory University

Atlanta, Georgia, United States, 30322

Withdrawn

4

Brigham and Women's Hospital

Boston, Massachusetts, United States, 02115

Withdrawn

5

Mayo Clinic

Rochester, Minnesota, United States, 55905

Actively Recruiting

6

The Christ Hospital Physicians - The Ohio Heart and Vascular Center

Cincinnati, Ohio, United States, 45219

Withdrawn

7

Cleveland Clinic

Cleveland, Ohio, United States, 44195

Actively Recruiting

8

Oregon Health & Science University

Portland, Oregon, United States, 97239

Withdrawn

9

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States, 19104

Withdrawn

10

Houston Methodist Hospital

Houston, Texas, United States, 77030

Actively Recruiting

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Research Team

M

Matt Pollman, M.D.

CONTACT

L

LaTanya Tomlinson, RN, MHSA

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

2

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