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

Phase 1

Recruiting

• Conditions: Hypertrophic Cardiomyopathy
• Interventions: Genetic: TN-201

• Registration Number: NCT05836259
• Lead Sponsor: Tenaya Therapeutics

Brief 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 hypertrophic cardiomyopathy (HCM) caused by mutations in the MYBPC3 gene.

Detailed Description

The study will consist of 2 escalating dose cohorts (groups). The study will enroll at least 6 and as many as 30 patients. All patients will receive active drug (TN-201 Gene Therapy). The study will follow patients for 5 years following a single dose of TN-201.

Study Timeline

• Study First Submitted: 2023-04-18
• Study First Submitted QC: 2023-04-18
• Study First Posted: 2023-05-01
• Study Start: 2023-08-10
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-12
• Last Update Posted: 2024-11-15
• Primary Completion: 2027-08
• Study Completion: 2032-08

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 30

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

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Exclusion Criteria

• High AAV9 neutralizing antibody titer

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 2	TN-201	Dose for Cohort 2 will be 6E13 vg/kg
Cohort 1	TN-201	Dose for Cohort 1 will be 3E13 vg/kg

Primary Outcome Measures

Name	Time	Method
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

Secondary Outcome Measures

Name	Time	Method
Change from baseline to Week 52 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS).	52 Weeks	The score ranges from 0-100, with zero being a worse outcome

Trial Locations

Locations (10)

The Christ Hospital Physicians - The Ohio Heart and Vascular Center; 🇺🇸 Cincinnati, Ohio, United States

UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research; 🇺🇸 La Jolla, California, United States

University of California San Francisco; 🇺🇸 San Francisco, California, United States

Cleveland Clinic; 🇺🇸 Cleveland, Ohio, United States

Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States

Hospital of the University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States

Houston Methodist Hospital; 🇺🇸 Houston, Texas, United States

Emory University; 🇺🇸 Atlanta, Georgia, United States

Brigham and Women's Hospital; 🇺🇸 Boston, Massachusetts, United States

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States