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Gene therapy trial aims to repair hearts in rare neurological disease

NCT ID NCT05445323

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial tests a gene therapy called LX2006 in 8 people with Friedreich's ataxia who also have heart muscle disease. The therapy delivers a healthy copy of the FXN gene to heart cells using a modified virus. The main goal is to check safety over 5 years, while also looking for signs that the treatment improves heart function.

What this could mean

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

Active substance
LX2006 gene therapy (a modified virus carrying a healthy FXN gene)
What this could lead to
If successful, this could point toward a treatment that slows or stops heart damage in people with Friedreich's ataxia.
What could go wrong
This is a very early, small trial with only 8 participants. It is designed mainly to check safety, not to prove the therapy works. Gene therapies can have unexpected side effects.

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

8 people

The number who actually took part.

Started

Aug 2022

Expected to finish

Sep 2029

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 50 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: * Confirmed genetic diagnosis of FA, with onset being before 25 years of age * Protocol specified ranges for antibodies * Protocol specified measures of FA cardiomyopathy Exclusion Criteria: * Protocol specified ranges for left ventricular ejection fraction (LVEF) as measured by cardiac ECHO * Uncontrolled diabetes * Abnormal liver function * Active infection of any type, including hepatitis virus (A, B or C) or human immunodeficiency virus (HIV-1 and HIV-2) * Contraindication to cardiac MRI * Contraindications to cardiac biopsies * Participants who are receiving systemic corticosteroids or other immunosuppressive medications * History of significant coronary artery disease or any structural heart or vascular disease other than FA cardiomyopathy * Presence of clinically significant, hemodynamically unstable arrhythmias, requiring physician intervention * Presence of clinically significant abnormalities as determined by the investigator, other than ECG abnormalities related to FA * Uncontrolled psychiatric disease Other Inclusion/Exclusion criteria to be applied as per protocol.

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Conditions

The condition(s) this trial relates to.

cardiomyopathy extrinsic cardiomyopathy Friedreich ataxia

As listed by the trial registrant

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

Cardiomyopathy, secondary Friedreich ataxia

Contacts and locations

Locations

  • Ataxia Center and HD Center of Excellence, University of California

    Los Angeles, California, 90095, United States

  • Mayo Clinic

    Rochester, Minnesota, 55905, United States

  • University of South Florida

    Tampa, Florida, 33612, United States

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