Clinical trial

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)

Name

TN-201-0009

Description

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).

Trial arms

Trial start

2023-08-10

Estimated PCD

2025-12-01

Trial end

2029-12-01

Status

Recruiting

Phase

Early phase I

Treatment

TN-201

TN-201 is a recombinant adeno-associated virus serotype 9 (AAV9) containing Myosin Binding Protein C (MYBPC3) transgene. It is a single (one-time) intravenous dose.

Arms:

Cohort 1, Cohort 2

Size

Primary endpoint

Number and severity of Adverse Events over the course of the study.

5 Years

Number of Serious Adverse Events related to study drug.

5 Years

Eligibility criteria

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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'This is a first-in-human, non-randomized, open label Phase 1b study. 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.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 15, 'type': 'ESTIMATED'}}

Updated at

2024-01-23

1 organization

1 product

1 indication

Product

TN-201

Indication

Hypertrophic Cardiomyopathy

Organization

Tenaya Therapeutics