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

NCT05836259 · tracked via the Priya Life Science USA tracker

Sponsor

Tenaya Therapeutics

Phase

Phase 1/2

Started

2023-08-10

Last updated

2026-03-19

Condition(s) studied

Hypertrophic Cardiomyopathy

Investigational drug(s) / intervention(s)

TN-201

TN-201: TN-201 is a recombinant adeno-associated virus serotype 9 (AAV9) containing Myosin Binding Protein C (MYBPC3) transgene. It is a single (one-time) intravenous dose.

Study 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.

Eligibility

Sex

ALL

Min age

18 Years

Max age

75 Years

Healthy volunteers

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

Primary outcome measure(s)

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

Trial sites (10)

Facility	City	Region	Status
UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research	La Jolla	California	Recruiting
University of California San Francisco	San Francisco	California	Recruiting
Emory University	Atlanta	Georgia	Withdrawn
Brigham and Women's Hospital	Boston	Massachusetts	Withdrawn
Mayo Clinic	Rochester	Minnesota	Recruiting
The Christ Hospital Physicians - The Ohio Heart and Vascular Center	Cincinnati	Ohio	Withdrawn
Cleveland Clinic	Cleveland	Ohio	Recruiting
Oregon Health & Science University	Portland	Oregon	Withdrawn
Hospital of the University of Pennsylvania	Philadelphia	Pennsylvania	Withdrawn
Houston Methodist Hospital	Houston	Texas	Recruiting

More Tenaya Therapeutics trials in the USA

Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC Recruiting

Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy Active, not recruiting

Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) Recruiting

Other trials for the same condition

Mavacamten Pregnancy Surveillance Program Recruiting

A Study of EDG-7500 in Adults With Hypertrophic Cardiomyopathy (CIRRUS-HCM) Active, not recruiting

VIZ ACCESS HCM - Multi-Site Registry Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05836259 on ClinicalTrials.gov ↗ ← All trials in the USA