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

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)

Status

Recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05836259

Acronym

MyPEAK-1

Enrollment

Registered

2023-05-01

Start date

2023-08-10

Completion date

2032-08-01

Last updated

2026-03-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hypertrophic Cardiomyopathy

Keywords

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

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.

Detailed description

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.

Interventions

GENETICTN-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 design

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

NONE

Intervention model description

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.

Eligibility

Sex/Gender

ALL

Age

18 Years to 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

Design outcomes

Primary

Measure	Time frame
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

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

Countries

United States

Contacts

CONTACTLaTanya Tomlinson, RN, MHSA

clinical.trials@tenayathera.com650-825-6990

CONTACTMatt Pollman, M.D.

mpollman@tenayathera.com650-209-8092

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Mar 20, 2026