Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM (MyPEAK-1)

January 19, 2024 updated by: Tenaya Therapeutics

First-in-Human, Open-Label, Safety, Tolerability, Dose-Finding, Pharmacodynamic and Cardiac Transgene Expression Study of TN-201, a Recombinant Adenoassociated Virus Serotype 9 (AAV9) Containing Myosin Binding Protein C Transgene, in Adults With MYBPC3 Mutation-Associated Hypertrophic Cardiomyopathy (HCM)

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 MYBPC3 mutation-associated nonobstructive hypertrophic cardiomyopathy (nHCM).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

The study will consist of 2 escalating dose cohorts. The study will enroll at least 6 and as many as 15 patients. All patients will receive active drug. The study will follow patients for 5 years following a single dose of TN-201.

Study Type

Interventional

Enrollment (Estimated)

15

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • California
      • La Jolla, California, United States, 92093
        • Not yet recruiting
        • UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research
      • San Francisco, California, United States, 94117
        • Recruiting
        • University of California San Francisco
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Not yet recruiting
        • Emory University
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Not yet recruiting
        • Brigham and Women's Hospital
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Mayo Clinic
    • Ohio
      • Cincinnati, Ohio, United States, 45219
        • Not yet recruiting
        • University of Cincinnati Health Physicians - Clifton
      • Cleveland, Ohio, United States, 44195
        • Recruiting
        • Cleveland Clinic
    • Oregon
      • Portland, Oregon, United States, 97239
        • Not yet recruiting
        • Oregon Health & Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Not yet recruiting
        • Hospital of the University of Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • Not yet recruiting
        • University of Pittsburgh Medical Center
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • Houston Methodist Hospital
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • Not yet recruiting
        • University Of Utah

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.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • MYBPC3 mutation
  • Nonobstructive Hypertrophic Cardiomyopathy
  • Left Ventricular Ejection Fraction ≥50%
  • Functioning Implantable Cardiac Defibrillator
  • NYHA Functional Class II or III symptoms
  • NT-proBNP ≥300pg/ml

Exclusion Criteria:

  • High AAV9 neutralizing antibody titer

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1
Dose for Cohort 1 will be 3E13 vg/kg
Genetic: 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.
Experimental: Cohort 2
Dose for Cohort 2 will be 6E13 vg/kg
Genetic: 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.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline to Week 52 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS).
Time Frame: 52 Weeks
The score ranges from 0-100, with zero being a worse outcome
52 Weeks

Other Outcome Measures

Outcome Measure
Time Frame
Expression levels of vector genomes and transgene messenger ribonucleic acid (mRNA) and MyBP-C protein in right ventricular (RV) septal biopsy samples at Week 8 and Week 52.
Time Frame: 52 Weeks
52 Weeks
Change from Pre-dose in N-terminal pro B-type natriuretic peptide (NTproBNP), and high-sensitivity cardiac troponin I (hs-cTnI) levels.
Time Frame: 5 Years
5 Years
Change from baseline peak exercise capacity (pVO2).
Time Frame: 5 Years
5 Years
Percentage of patients who had a change in New York Heart Association (NYHA) Functional Class from baseline.
Time Frame: 5 Years
5 Years
Change from baseline in echocardiography measurement of left ventricular mass index (g/m^2).
Time Frame: 5 Years
5 Years
Change from baseline in echocardiography measurement of e/e' ratio.
Time Frame: 5 Years
5 Years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Tenaya Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 10, 2023

Primary Completion (Estimated)

December 1, 2025

Study Completion (Estimated)

December 1, 2029

Study Registration Dates

First Submitted

April 18, 2023

First Submitted That Met QC Criteria

April 18, 2023

First Posted (Actual)

May 1, 2023

Study Record Updates

Last Update Posted (Estimated)

January 23, 2024

Last Update Submitted That Met QC Criteria

January 19, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TN-201-0009

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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