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

NCT ID: NCT05836259

Last Updated: 2025-07-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-08-10

Study Completion Date

2032-08-31

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Hypertrophic Cardiomyopathy

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

This is a first-in-human, non-randomized, open label Phase 1b/2 study. The study will consist of 2 escalating dose cohorts. The study will enroll at least 6 and as many as 30 patients. All patients will receive active drug.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

Dose for Cohort 1 will be 3E13 vg/kg

Group Type EXPERIMENTAL

TN-201

Intervention Type GENETIC

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.

Cohort 2

Dose for Cohort 2 will be 6E13 vg/kg

Group Type EXPERIMENTAL

TN-201

Intervention Type GENETIC

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.

Interventions

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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.

Intervention Type GENETIC

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Tenaya Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research

La Jolla, California, United States

Site Status RECRUITING

University of California San Francisco

San Francisco, California, United States

Site Status RECRUITING

Emory University

Atlanta, Georgia, United States

Site Status WITHDRAWN

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status WITHDRAWN

Mayo Clinic

Rochester, Minnesota, United States

Site Status RECRUITING

The Christ Hospital Physicians - The Ohio Heart and Vascular Center

Cincinnati, Ohio, United States

Site Status WITHDRAWN

Cleveland Clinic

Cleveland, Ohio, United States

Site Status RECRUITING

Oregon Health & Science University

Portland, Oregon, United States

Site Status WITHDRAWN

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status WITHDRAWN

Houston Methodist Hospital

Houston, Texas, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Matt Pollman, M.D.

Role: CONTACT

[email protected]
650-209-8092

LaTanya Tomlinson, RN, MHSA

Role: CONTACT

[email protected]
650-825-6990

Facility Contacts

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Connor Davies

Role: primary

[email protected]
858-534-3798

Emma Reagan

Role: primary

[email protected]
415-514-7280

Sierra Slade

Role: primary

[email protected]
507-422-5433

Danielle Kellner

Role: primary

[email protected]
216-445-6321

Jennifer Garrett

Role: primary

[email protected]
281-222-9983

References

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Grisorio L, Bongianino R, Gianeselli M, Priori SG. Gene therapy for cardiac diseases: methods, challenges, and future directions. Cardiovasc Res. 2024 Nov 25;120(14):1664-1682. doi: 10.1093/cvr/cvae207.

Reference Type DERIVED
PMID: 39302117 (View on PubMed)

Other Identifiers

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TN-201-0009

Identifier Type: -

Identifier Source: org_study_id

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