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NCT Number: NCT05836259

Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated 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 hypertrophic cardiomyopathy (HCM) caused by mutations in the MYBPC3 gene.

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Key information

Conditions

Hypertrophic Cardiomyopathy Aortic Stenosis, Subvalvular Aortic Valve Disease Aortic Valve Stenosis Cardiomyopathies Cardiomyopathy, Hypertrophic Cardiomyopathy, Hypertrophic, Familial Cardiovascular Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heart Diseases Heart Valve Diseases

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research, La Jolla, California, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

TN-201

Genetic

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 outcomes

  1. Number and severity of Adverse Events over the course of the study.

    Time frame: 5 Years

  2. Number of Serious Adverse Events related to study drug.

    Time frame: 5 Years

Secondary outcomes

  1. 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

Other outcomes

  1. 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.

    Time frame: 52 Weeks

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

    Time frame: 5 Years

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

    Time frame: 5 Years

  4. Change from baseline in echocardiography measurements of indexed left atrial and left ventricular (LV) volumes.

    Time frame: 5 Years

  5. Change from baseline in echocardiography measurements of LV mass.

    Time frame: 5 Years

  6. Change from baseline in echocardiography measurements of maximal LV wall thickness.

    Time frame: 5 Years

  7. Change from baseline in echocardiography measurements of global longitudinal strain.

    Time frame: 5 Years

  8. Change from baseline in 6MWT distance.

    Time frame: 5 Years

  9. Change from baseline peak exercise capacity (pVO2).

    Time frame: 5 Years

Study contacts

Contact information is provided by the study sponsor or research team.

LaTanya Tomlinson, RN, MHSA

CONTACT

[email protected]

650-825-6990

Matt Pollman, M.D.

CONTACT

[email protected]

650-209-8092

Sponsors and collaborators

Lead sponsor

Tenaya Therapeutics

Industry

Registry information

Official study title

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

Acronym: MyPEAK-1

Important dates

Study start
2023
Primary completion
2027
Study completion
2032
First posted
May 1, 2023
Registry last updated
Mar 19, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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