ClinicalTrials.gov record NCT06865482

Clinical Course Of Disease In Participants With FA-CM

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 7, 2026

Characteristics And Clinical Course Of Disease In Participants With Cardiomyopathy Associated With Friedreich Ataxia (CLARITY-FA)

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
Age6 years old and older
SexAll
LocationBrazil, Canada, Czechia, France, Germany, Italy, Spain, United States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This prospective observational study follows people with genetically confirmed Friedreich ataxia who have cardiomyopathy (age 6 and older). It collects heart imaging, blood markers, symptom questionnaires, and medical-record data over time to describe heart disease presentation and progression.

What it is studying. The study seeks to describe the nature and rate of cardiac disease progression in people with FA-CM by tracking heart structure and function, biomarkers, symptoms, medication use, and health-care events over time.

What the researchers hope to learn. To characterize cardiac disease presentation and progression in Friedreich Ataxia with cardiomyopathy, including changes in left ventricular mass and function, biomarkers (such as troponin I), patient- and clinician-reported measures, medication use, mortality, major adverse cardiovascular events, and health-care utilization.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

Study LX2006-02 is a prospective, longitudinal, low-intervention, multicenter, global study aimed at characterizing the nature and rate of cardiac disease progression in participants with genetically confirmed FA.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is an observational, prospective study with scheduled visits and follow-up for up to 12 months. Participants will have heart imaging (cardiac MRI when possible; echocardiography if MRI is contraindicated or for young children who cannot complete MRI without sedation), blood tests including measures such as high-sensitivity troponin I, and complete questionnaires about symptoms and function (KCCQ-12, PGI-S/PGI-C); clinicians assess the mFARS. Study staff will also record medications and health-care use (hospitalizations, procedures).

How the study is structured. A prospective, longitudinal observational cohort study. Participants are followed over time with regular heart imaging (cardiac MRI when possible; echocardiogram for some children or when MRI is not possible), blood tests, symptom and function questionnaires, and review of medical records. The study does not assign treatments.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in Brazil, Canada, Czechia, France, Germany, Italy, Spain, United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Friedreich ataxia

Clinical trials26 trials listed for Friedreich ataxiaEvery trial the registry lists for Friedreich ataxia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Friedreich ataxia researchLatest monthly update: September 2026. Includes: Heart change stages. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Sep 30, 2025
Primary completion
September 2027 (estimated)
Study completion
September 2027 (estimated)
Target enrollment
65 participants (estimated)
Last updated on registry
Sep 2, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Lexeo Therapeutics

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (19)

Terms used on this page · explanations by MedicalUpdates.ai

informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
assent
A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
Contraindication
A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
prior to screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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