Summary

Eligibility
for people ages 18-65 (full criteria)
Location
at La Jolla, California and other locations
Dates
study started
completion around

Description

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 MYBPC3 mutation-associated nonobstructive hypertrophic cardiomyopathy (nHCM).

Official Title

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

Details

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.

Keywords

Hypertrophic Cardiomyopathy, Hypertrophic Cardiomyopathy (HCM), Myosin Binding Protein C3 (MYBPC3), Nonobstructive HCM, Genetic HCM, Familial HCM, Adenoassociated Virus (AAV), Gene Therapy, Cardiomyopathies, Hypertrophy

Eligibility

You can join if…

Open to people ages 18-65

  • 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

You CAN'T join if...

- High AAV9 neutralizing antibody titer

Locations

  • UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research accepting new patients
    La Jolla California 92093 United States
  • University of California San Francisco accepting new patients
    San Francisco California 94117 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Tenaya Therapeutics
ID
NCT05836259
Phase
Phase 1 Cardiomyopathy Research Study
Study Type
Interventional
Participants
Expecting 15 study participants
Last Updated