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Gene therapy breakthrough? first human test of TN-201 for heart muscle disease begins

NCT ID NCT05836259

Disease control Sponsor: Tenaya Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a new gene therapy called TN-201 in 30 adults with hypertrophic cardiomyopathy caused by a specific genetic mutation (MYBPC3). The therapy is given as a one-time IV infusion and aims to correct the underlying genetic defect. The main goals are to check safety and find the right dose, while also looking at how it affects heart function and symptoms.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
TN-201 (a gene therapy given as a one-time IV infusion)
What this could lead to
If successful, this could lead to a one-time treatment that addresses the root genetic cause of hypertrophic cardiomyopathy, potentially reducing symptoms and improving heart function.
What could go wrong
This is a very early (first-in-human) trial with only 30 participants, so safety and effectiveness are not yet known. Gene therapies can have serious side effects, and it may not work for everyone.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 30 people

The number the study aims to enrol. It can still change while the study runs.

Started

Aug 2023

Expected to finish

Aug 2032

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

familial hypertrophic cardiomyopathy hypertrophic cardiomyopathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hypertrophic cardiomyopathy

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    5 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Brigham and Women's Hospital

    WITHDRAWN

    Boston, Massachusetts, 02115, United States

  • Cleveland Clinic

    RECRUITING

    Cleveland, Ohio, 44195, United States

  • Emory University

    WITHDRAWN

    Atlanta, Georgia, 30322, United States

  • Hospital of the University of Pennsylvania

    WITHDRAWN

    Philadelphia, Pennsylvania, 19104, United States

  • Houston Methodist Hospital

    RECRUITING

    Houston, Texas, 77030, United States

  • Mayo Clinic

    RECRUITING

    Rochester, Minnesota, 55905, United States

  • Oregon Health & Science University

    WITHDRAWN

    Portland, Oregon, 97239, United States

  • The Christ Hospital Physicians - The Ohio Heart and Vascular Center

    WITHDRAWN

    Cincinnati, Ohio, 45219, United States

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

    RECRUITING

    La Jolla, California, 92093, United States

  • University of California San Francisco

    RECRUITING

    San Francisco, California, 94117, United States

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