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

T

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

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

Trial contacts and locations

0

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Central trial contact

Laura Robertson, M.D.; LaTanya Tomlinson, RN, MHSA

Data sourced from clinicaltrials.gov

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