Health Trials

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

ConditionHypertrophic Cardiomyopathy
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-75
SponsorTenaya Therapeutics

About this trial

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.

Eligibility criteria

Qualifiers

MYBPC3 mutation

Hypertrophic Cardiomyopathy (obstructive and nonobstructive)

Left Ventricular Ejection Fraction ≥45%

NYHA Functional Class II or III symptoms

Disqualifiers

High AAV9 neutralizing antibody titer

Trial design

Treatments tested in this trial

  • TN-201

Treatment groups

30 Participants
are divided into 2 treatment groups

Locations

10
California
UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research92093, La Jolla United States
University of California San Francisco94117, San Francisco United States
Georgia
Emory University30322, Atlanta United States
Massachusetts
Brigham and Women's Hospital02115, Boston United States

Sponsors and collaborators

Tenaya Therapeutics

Lead sponsor

Source ClinicalTrials.gov