MedPath

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

Phase 1
Recruiting
Conditions
Hypertrophic Cardiomyopathy
Interventions
Genetic: TN-201
Registration Number
NCT05836259
Lead Sponsor
Tenaya Therapeutics
Brief Summary

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.

Detailed 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.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
30
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
Read More
Exclusion Criteria
  • High AAV9 neutralizing antibody titer
Read More

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Cohort 2TN-201Dose for Cohort 2 will be 6E13 vg/kg
Cohort 1TN-201Dose for Cohort 1 will be 3E13 vg/kg
Primary Outcome Measures
NameTimeMethod
Number and severity of Adverse Events over the course of the study.5 Years
Number of Serious Adverse Events related to study drug.5 Years
Secondary Outcome Measures
NameTimeMethod
Change from baseline to Week 52 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS).52 Weeks

The score ranges from 0-100, with zero being a worse outcome

Trial Locations

Locations (10)

The Christ Hospital Physicians - The Ohio Heart and Vascular Center

🇺🇸

Cincinnati, Ohio, United States

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

🇺🇸

La Jolla, California, United States

University of California San Francisco

🇺🇸

San Francisco, California, United States

Cleveland Clinic

🇺🇸

Cleveland, Ohio, United States

Oregon Health & Science University

🇺🇸

Portland, Oregon, United States

Hospital of the University of Pennsylvania

🇺🇸

Philadelphia, Pennsylvania, United States

Houston Methodist Hospital

🇺🇸

Houston, Texas, United States

Emory University

🇺🇸

Atlanta, Georgia, United States

Brigham and Women's Hospital

🇺🇸

Boston, Massachusetts, United States

Mayo Clinic

🇺🇸

Rochester, Minnesota, United States

© Copyright 2025. All Rights Reserved by MedPath