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New study tracks heart disease in rare genetic disorder

NCT ID NCT06865482

Knowledge-focused 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 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 03, 2026 · Updated 3 times

Summary

This study follows 65 people with Friedreich ataxia who also have a thickened heart muscle (cardiomyopathy). Researchers will measure changes in heart structure over time using imaging. The goal is to better understand how heart disease progresses in this condition, which could help design future treatments.

Why investors are watching

Lexeo Therapeutics is running CLARITY-FA, a small observational study of 65 people with cardiomyopathy linked to Friedreich ataxia, a rare inherited disease. For a micro-cap company, this readout matters because it could provide early evidence about how the heart condition progresses, which may inform future treatment trials. A clear result could help Lexeo position itself in a niche area where few competitors focus.

If it works: A positive result could show that Lexeo can gather useful data in a rare disease, strengthening its pipeline story and attracting partnership interest. It might also support the design of a larger, later-stage trial for a potential therapy.

If it fails: The study is observational, so it does not test a drug, and a weak or unclear result would not directly advance a product. Trials in rare diseases often face slow enrollment or messy data, and failure or delay could set back Lexeo's timeline and reduce investor confidence.

AI-written from the trial record. Speculative, and not investment advice.

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.

Participants

About 65 people

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

Started

Sep 2025

Expected to finish

Sep 2027

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.

Who is studied

Up to 65 participants with genetically confirmed FA-CM will be enrolled in the study with allocation by age categories and cohorts

Ages

6 years and older

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: * Male or female, ages ≥6 years at the time of signing the informed consent (and assent, if applicable). * Diagnosis of FA, based on clinical phenotype and genotype (GAA expansion on both alleles or compound heterozygous), with onset of FA occurring at ≤25 years of age * Confirmed left ventricular hypertrophy (LVH) * Left ventricular ejection fraction ≥30% Exclusion Criteria: * Presence of other form(s) of CM contributing to heart failure (HF), clinically significant cardiac anatomic abnormality or congenital cardiac malformation, clinically significant coronary artery, uncorrected, hemodynamically significant primary structural valvular disease not due to CM * Currently receiving intermittent or continuous intravenous (IV) inotrope infusion, presence of a ventricular assist device, or history of prior heart transplantation * Contraindication to cMRI, participants \<12 years of age who cannot complete the cMRI without sedation will instead undergo ECHOs and are exempt from this criterion. * Prior organ transplantation * Initiation of cardiac resynchronization therapy (CRT) within 6 months prior to screening. * History of prior gene transfer or cell therapy. * Poorly controlled diabetes (hemoglobin A1c ≥8%) * Active hematologic or solid organ malignancy

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Conditions

The condition(s) this trial relates to.

cardiomyopathy Friedreich ataxia heart disorder

As listed by the trial registrant

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

Cardiomyopathy Friedreich ataxia

How to take part

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

  1. The places running it

    19 sites in 8 countries. The list below names each one and where it is.

  2. 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 ↗

  3. 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

Locations

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • Campinas State University (UNICAMP)

    RECRUITING

    Campinas, São Paulo, 13083-887, Brazil

  • Centre Hospitalier de Universite de Montreal (CHUM)

    RECRUITING

    Montreal, Quebec, H2X 0C1, Canada

  • Cincinnati Children's Hospital

    RECRUITING

    Cincinnati, Ohio, 45229, United States

  • Federico II University

    RECRUITING

    Naples, 80131, Italy

  • Hospital Das Clínicas Da Universidade Federal De Minas Gerais (UFMG)

    RECRUITING

    Belo Horizonte, Minas Gerais, 30130, Brazil

  • Hospital Univeritario Puerta de Hierro de Majadahonda

    RECRUITING

    Majadahonda, Madrid, 28222, Spain

  • Hospital Universitário Walter Cantídio - Universidade Federal do Ceará

    RECRUITING

    Fortaleza, Ceará, 60430, Brazil

  • Indiana University - Riley Children's Health

    RECRUITING

    Indianapolis, Indiana, 46202, United States

  • Institut de Cardiologie Hopital Pitie Salpetriere

    RECRUITING

    Paris, 75013, France

  • Instituto D'or de Pesquisa e Ensino - Hospital São Rafael

    RECRUITING

    Salvador, Estado de Bahia, 41253-190, Brazil

  • Mayo Clinic

    RECRUITING

    Rochester, Minnesota, 55905, United States

  • Motol University Hospital and the Second Faculty of Medicine

    RECRUITING

    Prague, 15006, Czechia

  • Oregon Health & Science University

    RECRUITING

    Portland, Oregon, 97239, United States

  • UPC Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto

    RECRUITING

    Ribeirão Preto, São Paulo, 14051, Brazil

  • University Hospital Tübingen

    RECRUITING

    Tübingen, Baden-Wurttemberg, 72076, Germany

  • University of California San Diego

    RECRUITING

    La Jolla, California, 92037, United States

  • University of South Florida

    RECRUITING

    Tampa, Florida, 33612, United States

  • Washington University School of Medicine in St. Louis

    RECRUITING

    St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.