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 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 MYBPC3 mutation-associated nonobstructive hypertrophic cardiomyopathy (nHCM).

Full 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.

Enrollment: 15 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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15 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