NCT05836259

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at P25-P50 for phase_1

Timeline
76mo left

Started Aug 2023

Longer than P75 for phase_1

Geographic Reach
1 country

10 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress31%
Aug 2023Aug 2032

First Submitted

Initial submission to the registry

April 18, 2023

Completed
13 days until next milestone

First Posted

Study publicly available on registry

May 1, 2023

Completed
3 months until next milestone

Study Start

First participant enrolled

August 10, 2023

Completed
4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2027

Expected
5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2032

Last Updated

March 19, 2026

Status Verified

March 1, 2026

Enrollment Period

4 years

First QC Date

April 18, 2023

Last Update Submit

March 18, 2026

Conditions

Hypertrophic Cardiomyopathy

Keywords

Hypertrophic Cardiomyopathy (HCM)Myosin Binding Protein C3 (MYBPC3)Nonobstructive HCMGenetic HCMFamilial HCMAdenoassociated Virus (AAV)Gene TherapyObstructive HCMnHCMoHCM

Outcome Measures

Primary Outcomes (2)

  • 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 Outcomes (1)

  • Change from baseline to Week 52 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS).

    52 Weeks

Other Outcomes (9)

  • Expression levels of vector genomes and transgene messenger ribonucleic acid (mRNA) and MyBP-C protein in right ventricular (RV) septal biopsy samples at Pre-Treatment, Week 26, and Week 52.

    52 Weeks

  • Change from Pre-Treatment period in N-terminal pro B-type natriuretic peptide (NTproBNP), and high-sensitivity cardiac troponin I (hs-cTnI) levels.

    5 Years

  • Percentage of patients who had a change in New York Heart Association (NYHA) Functional Class from baseline.

    5 Years

  • +6 more other outcomes

Study Arms (2)

Cohort 1

EXPERIMENTAL

Dose for Cohort 1 will be 3E13 vg/kg

Genetic: TN-201

Cohort 2

EXPERIMENTAL

Dose for Cohort 2 will be 6E13 vg/kg

Genetic: TN-201

Interventions

TN-201GENETIC

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.

Cohort 1Cohort 2

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

  • High AAV9 neutralizing antibody titer

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

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

La Jolla, California, 92093, United States

RECRUITING

University of California San Francisco

San Francisco, California, 94117, United States

RECRUITING

Emory University

Atlanta, Georgia, 30322, United States

WITHDRAWN

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

WITHDRAWN

Mayo Clinic

Rochester, Minnesota, 55905, United States

RECRUITING

The Christ Hospital Physicians - The Ohio Heart and Vascular Center

Cincinnati, Ohio, 45219, United States

WITHDRAWN

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

Oregon Health & Science University

Portland, Oregon, 97239, United States

WITHDRAWN

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

WITHDRAWN

Houston Methodist Hospital

Houston, Texas, 77030, United States

RECRUITING

Related Publications (1)

  • Grisorio L, Bongianino R, Gianeselli M, Priori SG. Gene therapy for cardiac diseases: methods, challenges, and future directions. Cardiovasc Res. 2024 Nov 25;120(14):1664-1682. doi: 10.1093/cvr/cvae207.

    PMID: 39302117DERIVED

MeSH Terms

Conditions

Cardiomyopathy, HypertrophicCardiomyopathy, Hypertrophic, Familial

Condition Hierarchy (Ancestors)

CardiomyopathiesHeart DiseasesCardiovascular DiseasesAortic Stenosis, SubvalvularAortic Valve StenosisAortic Valve DiseaseHeart Valve DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Matt Pollman, M.D.

CONTACT

650-209-8092mpollman@tenayathera.com

LaTanya Tomlinson, RN, MHSA

CONTACT

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

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Model Details: This is a first-in-human, non-randomized, open label Phase 1b/2 study. The study will consist of 2 escalating dose cohorts. The study will enroll at least 6 and as many as 30 patients. All patients will receive active drug.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 18, 2023

First Posted

May 1, 2023

Study Start

August 10, 2023

Primary Completion (Estimated)

August 1, 2027

Study Completion (Estimated)

August 1, 2032

Last Updated

March 19, 2026

Record last verified: 2026-03

Locations

US(10)