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

Plain-language summary

This study evaluates the safety and tolerability of TN-201, a gene therapy, in adults with hypertrophic cardiomyopathy caused by a mutation in the MYBPC3 gene. Adults with a MYBPC3 mutation, symptoms (NYHA Class II or III), and a certain level of preserved heart function may take part. The study is in Phase 1/Phase 2 and is currently enrolling participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Recruiting

Sponsor

Tenaya Therapeutics

Intervention

TN-201

Start date

8/10/2023

Estimated completion

8/1/2032

Countries

United States

Related genes

Locations

• La Jolla, United States
• San Francisco, United States
• Atlanta, United States
• Boston, United States
• Rochester, United States
• Cincinnati, United States
• Cleveland, United States
• Portland, United States
• Philadelphia, United States
• Houston, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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