Recruiting
Phase 1
Phase 2

TN-201

Sponsor:

Tenaya Therapeutics

Code:

NCT05836259

Conditions

Hypertrophic Cardiomyopathy

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

TN-201

Study Details

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.

Conditions

Hypertrophic Cardiomyopathy

Study ID

NCT05836259

Start date

Aug 10, 2023

Status verified date

Mar, 2026

Completion date

Aug, 2032

Anticipated

Primary completion date

Aug, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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 Design

Enrollment

30 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort 1

Dose for Cohort 1 will be 3E13 vg/kg

experimental: Cohort 2

Dose for Cohort 2 will be 6E13 vg/kg

Interventions

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.

Primary outcome measure

  • Number and severity of Adverse Events over the course of the study. [ Time Frame: 5 Years ]
  • Number of Serious Adverse Events related to study drug. [ Time Frame: 5 Years ]

Central Contacts and Locations

Central contacts

Matt Pollman, M.D.

650-209-8092mpollman@tenayathera.com

LaTanya Tomlinson, RN, MHSA

650-825-6990clinical.trials@tenayathera.com

Locations

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

Recruiting

La Jolla, California, United States, 92093

Contacts

Matt Fahey

858-534-3798mjfahey@health.ucsd.edu

University of California San Francisco

Recruiting

San Francisco, California, United States, 94117

Contacts

Emma Reagan

415-514-7280Emma.reagan@ucsf.edu

Mayo Clinic

Recruiting

Rochester, Minnesota, United States, 55905

Contacts

Sierra Slade

507-422-5433Slade.Sierra@mayo.edu

Cleveland Clinic

Recruiting

Cleveland, Ohio, United States, 44195

Contacts

Rachael Landis

216-444-3650LANDISR@ccf.org

Houston Methodist Hospital

Recruiting

Houston, Texas, United States, 77030

Contacts

Jennifer Garrett

281-222-9983jmgarrett@houstonmethodist.org

More Information

Sponsor

Tenaya Therapeutics

Last update posted

Mar 19, 2026

Last verified

Mar, 2026

Keywords

  • Hypertrophic Cardiomyopathy (HCM)
  • Myosin Binding Protein C3 (MYBPC3)
  • Nonobstructive HCM
  • Genetic HCM
  • Familial HCM
  • Adenoassociated Virus (AAV)
  • Gene Therapy
  • Obstructive HCM
  • nHCM
  • oHCM

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Tenaya Therapeutics on 2026-03-19.