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

Description

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

Hypertrophic Cardiomyopathy

Study Overview

Study Details

Study 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.

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)

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

Condition
Hypertrophic Cardiomyopathy
Intervention / Treatment

-

Contacts and Locations

La Jolla

UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research, La Jolla, California, United States, 92093

San Francisco

University of California San Francisco, San Francisco, California, United States, 94117

Atlanta

Emory University, Atlanta, Georgia, United States, 30322

Boston

Brigham and Women's Hospital, Boston, Massachusetts, United States, 02115

Rochester

Mayo Clinic, Rochester, Minnesota, United States, 55905

Cincinnati

The Christ Hospital Physicians - The Ohio Heart and Vascular Center, Cincinnati, Ohio, United States, 45219

Cleveland

Cleveland Clinic, Cleveland, Ohio, United States, 44195

Portland

Oregon Health & Science University, Portland, Oregon, United States, 97239

Philadelphia

Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, United States, 19104

Houston

Houston Methodist Hospital, Houston, Texas, United States, 77030

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * MYBPC3 mutation
  • * Hypertrophic Cardiomyopathy (obstructive and nonobstructive)
  • * Left Ventricular Ejection Fraction ≥45%
  • * NYHA Functional Class II or III symptoms
  • * NT-proBNP ≥160pg/ml
  • * High AAV9 neutralizing antibody titer

Ages Eligible for Study

18 Years to 75 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Tenaya Therapeutics,

Study Record Dates

2032-08