RecruitingOBSERVATIONAL

Clinical Course Of Disease In Participants With FA-CM

This research study, called Clinical Course Of Disease In Participants With FA-CM, is exploring how a specific heart condition, called cardiomyopathy, develops and changes in people who also have Friedreich Ataxia (FA). Researchers want to understand the nature and speed at which this heart problem progresses. By carefully observing participants over time, they hope to gather important information that could pave the way for future treatments, including gene therapy. After being in this study for at least 26 weeks, some participants who meet certain requirements might have an opportunity to join another study offering a gene therapy treatment.

At a glance

Status

Recruiting

Sponsor

Lexeo Therapeutics

Enrolment target

Start

30 Sep 2025

Estimated completion

01 Sep 2027

Friedreich Ataxia Cardiomyopathy

What is this study about?

This study is called 'Clinical Course Of Disease In Participants With FA-CM' and it's looking into a heart problem known as cardiomyopathy. This heart condition sometimes affects people who have Friedreich Ataxia (FA), a genetic condition that causes damage to the nervous system. The main goal of this study is to understand how this heart problem changes and develops over time.

Researchers want to see how quickly the heart condition progresses and what effects it has on people's health. By carefully watching participants and collecting information, the study aims to build a clear picture of how FA-related cardiomyopathy behaves. This knowledge is really important because it could help doctors and scientists develop better ways to treat or manage the condition in the future.

This study will carefully observe participants for a period. For those who meet specific criteria and have been in this study for at least 26 weeks, there might be a chance to join another study that explores a new gene therapy treatment. This means the information gathered from this observation study could help identify people who might benefit from cutting-edge treatments down the line.

Key takeaways

This study observes how a heart condition linked to Friedreich Ataxia changes over time.
It aims to inform future treatments, potentially including gene therapy.
Participation involves regular check-ups and heart scans.
You must be at least 6 years old with a confirmed FA diagnosis and specific heart changes.
It's an observational study, not a treatment study (initially).
You might get to join a gene therapy study after 26 weeks if you meet extra criteria.

Who may be eligible?

To join this study, people need to be at least 6 years old. They must have a confirmed diagnosis of Friedreich Ataxia, meaning their doctors have found the genetic cause. Their Friedreich Ataxia symptoms must have started before they turned 26. They also need to have a specific heart condition called left ventricular hypertrophy (LVH) and their heart's pumping ability (called left ventricular ejection fraction) must be at least 40%.

However, some people cannot join this study. This includes those whose heart problems are caused by other conditions or birth defects, or if they have serious heart valve issues. People who are currently receiving certain heart medications through a drip, have a heart assist device, or have had a heart transplant are also not able to participate. If they have had other organ transplants or certain gene/cell therapies in the past, they won't be eligible.

Other reasons someone might not be able to join include having very high blood sugar levels (poorly controlled diabetes) or if they have an active cancer. Also, if they've recently had a specific heart treatment called cardiac resynchronization therapy within the last six months, they won't be able to take part.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you at least 6 years old?
Do you have a confirmed diagnosis of Friedreich Ataxia?
Did your FA symptoms start before you were 26?
Do you have a specific heart thickening called left ventricular hypertrophy (LVH)?
Is your heart's pumping ability (ejection fraction) at least 40%?
Are you generally free from other serious heart conditions or recent heart treatments?

Answer every question to see your result.

What does participation involve?

This study involves careful observation over time. It is considered a "low-intervention" study, meaning it won't involve taking new experimental medications directly within this specific phase. You would likely have regular check-ups, which might include heart scans like an MRI or ECHO, to help researchers understand how your heart condition is progressing. The exact number of visits or tests will be explained by the study team.

Participants will be in the study for at least 26 weeks. After this initial period, if you meet certain additional health requirements, you may then be given the option to take part in a separate study that actually tests a new gene therapy treatment.

Potential risks and benefits

Potential benefits of taking part include contributing to a better understanding of FA-related cardiomyopathy, which could help develop future treatments. For those who meet the criteria after 26 weeks, there might be an opportunity to participate in a gene therapy study. Potential risks are generally low in an observational study but could include discomfort from tests like MRI scans, and the time commitment involved. You always have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (19)

University of California San Diego
Verified postcode
La Jolla, United States· Recruiting
University of South Florida
Verified postcode
Tampa, United States· Recruiting
Indiana University - Riley Children's Health
Verified postcode
Indianapolis, United States· Recruiting
Boston Children's Hospital
Verified postcode
Boston, United States· Recruiting
Mayo Clinic
Verified postcode
Rochester, United States· Recruiting
Washington University School of Medicine in St. Louis
Verified postcode
St Louis, United States· Recruiting
Cincinnati Children's Hospital
Verified postcode
Cincinnati, United States· Recruiting
Oregon Health & Science University
Verified postcode
Portland, United States· Recruiting
Hospital Universitário Walter Cantídio - Universidade Federal do Ceará
Verified postcode
Fortaleza, Brazil· Recruiting
Instituto D'or de Pesquisa e Ensino - Hospital São Rafael
Verified postcode
Salvador, Brazil· Recruiting
Hospital Das Clínicas Da Universidade Federal De Minas Gerais (UFMG)
Verified postcode
Belo Horizonte, Brazil· Recruiting
Campinas State University (UNICAMP)
Verified postcode
Campinas, Brazil· Recruiting

Common questions

What is Friedreich Ataxia cardiomyopathy?

It's a heart condition that sometimes affects people with Friedreich Ataxia, where the heart muscle thickens and can struggle to pump blood effectively.

What does 'low-intervention' mean?

It means this study mainly involves observation and tests, not giving new medicines directly within this part of the study.

Why is this study important?

It helps researchers understand how the heart condition changes over time in FA, which is key for developing new treatments.

Can children join this study?

Yes, people aged 6 years and older can take part if they meet the other requirements.

Will I receive new treatment in this study?

Not directly in this part of the study. After 26 weeks, you *might* be offered a chance to join another study that offers gene therapy, if you're eligible.

How to find out more

Lexeo Clinical Trials

clinicaltrials@lexeotx.com 212-547-9879 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.