Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM (MyPEAK-1)

Tenaya Therapeutics

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Hypertrophic Cardiomyopathy

Treatments: Genetic: TN-201

Study type: Interventional

Funder types: Industry

Identifiers

NCT05836259

TN-201-0009

Details and patient eligibility

About

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.

Full 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.

Enrollment: 30 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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30 participants in 2 patient groups

Cohort 1

Experimental group

Description:

Dose for Cohort 1 will be 3E13 vg/kg

Treatment:

Genetic: TN-201

Cohort 2

Experimental group

Description:

Dose for Cohort 2 will be 6E13 vg/kg

Treatment:

Genetic: TN-201