Friedreich AtaxiaNCT06865482
Clinical Course Of Disease In Participants With FA-CM
An observational study of Friedreich Ataxia and Cardiomyopathy, run by Lexeo Therapeutics at 19 sites in 8 countries.
Trial summary
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA) Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Friedreich Ataxia, Cardiomyopathy
- How many people
- 65 participants
- Where
- 19 sitesin 8 countries
- Study dates
- 30 Sep 2025to Sep 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Up to 65 participants with genetically confirmed FA-CM will be enrolled in the study with allocation by age categories and cohorts
Who may be able to join
- 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%
Who cannot join
- 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
Trial locations and contacts
19 sites in 8 countries. Contact the study team or a site near you to ask about taking part.
19 sites in 8 countries
United States8 sites, 7 recruiting
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02115
Ping.Turongpun@childrens.harvard.edu
- Recruiting
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
Jaynee.Bartsch@cchmc.org
sarah.speed@cchmc.org
- Recruiting
Indiana University - Riley Children's Health
Indianapolis, Indiana, 46202
lkhernan@iu.edu
- Recruiting
University of California San Diego
La Jolla, California, 92037
jiz161@health.ucsd.edu
- Withdrawn
Oregon Health & Science University
Portland, Oregon, 97239
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55905
connors.margaret@mayo.edu
- Recruiting
Washington University School of Medicine in St. Louis
St Louis, Missouri, 63110
aplatts@wustl.edu
- Recruiting
University of South Florida
Tampa, Florida, 33612
lcampbel@usf.edu
anapaulab@usf.edu
Brazil5 sites, 5 recruiting
- Recruiting
Hospital Das Clínicas Da Universidade Federal De Minas Gerais (UFMG)
Belo Horizonte, Minas Gerais, 30130
josyene.lima@ebserh.gov.br
carla-nascimento.cn@ebserh.gov.br
- Recruiting
Campinas State University (UNICAMP)
Campinas, São Paulo, 13083-887
dany.unicamp@gmail.com
- Recruiting
Hospital Universitário Walter Cantídio - Universidade Federal do Ceará
Fortaleza, Ceará, 60430
eduardo.freitas@ebserh.gov.br
solange.alexandre@ebserh.gov.br
- Recruiting
UPC Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto
Ribeirão Preto, São Paulo, 14051
bfsousa@hcrp.usp.br
lssonobe@hcrp.usp.br
- Recruiting
Instituto D'or de Pesquisa e Ensino - Hospital São Rafael
Salvador, Estado de Bahia, 41253-190
hithilla.machado@idor.org
georgia.santos@idor.org
Canada1 site, 1 recruiting
- Recruiting
Centre Hospitalier de Universite de Montreal (CHUM)
Montreal, Quebec, H2X 0C1
celia.parinot.chum@ssss.gouv.qc.ca
Czechia1 site, 1 recruiting
- Recruiting
Motol University Hospital and the Second Faculty of Medicine
Prague, 15006
marcelasedlackova@seznam.cz
bryksiova@gmail.com
France1 site, 1 recruiting
- Recruiting
Institut de Cardiologie Hopital Pitie Salpetriere
Paris, 75013
delphine.brugier-ext@aphp.fr
Germany1 site, 1 recruiting
- Recruiting
University Hospital Tübingen
Tübingen, Baden-Wurttemberg, 72076
kathrin.schweizer@med.uni-tuebingen.de
Italy1 site, 1 recruiting
- Recruiting
Federico II University
Naples, 80131
giorgia.puorro@unina.it
Spain1 site, 1 recruiting
- Recruiting
Hospital Univeritario Puerta de Hierro de Majadahonda
Majadahonda, Madrid, 28222
ariadna.segovia@salud.madrid.org
About this study
Official title: Characteristics And Clinical Course Of Disease In Participants With Cardiomyopathy Associated With Friedreich Ataxia (CLARITY-FA)
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
Read the detailed description
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.
- Conditions
- Friedreich Ataxia, Cardiomyopathy
- Keywords
- Friedreich Ataxia, FA-CM, Cardiomyopathy, FA, Cardiac Disease
- Medical subject headings
- Friedreich Ataxia, Cardiomyopathies, Heart Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: Participants ≥16 years of age with FA-CM | Participants ≥16 years of age with FA-CM | |
| Cohort 2: Participants ≥6 to <16 years of age with FA-CM | Participants ≥6 to <16 years of age with FA-CM |
What is being measured
The primary outcome is the main question the study is designed to answer.
Characterize cardiac disease presentation and progression among participants
Change from baseline in left ventricular mass index (LVMi)
Measured: 52 weeks
Secondary outcomes (10)
Describe progression of maximal wall thickness (MWT) among this population
Change from baseline
Measured: 52 weeks
Describe progression of high sensitivity troponin I among this population
Change from baseline
Measured: 52 weeks
Describe participant perception and clinician assessment of illness in Kansas City Cardiomyopathy Questionnaire-12 (KCCQ-12)
Change from baseline
Measured: 52 weeks
Describe clinician assessment of illness in modified Friedreich Ataxia Rating Scale (mFARS)
Change from baseline
Measured: 52 weeks
Describe participant perception of illness in Patient Global Impression of Severity (PGI-S)
Change from baseline
Measured: 52 weeks
Describe participant perception of illness in Patient Global Impression of Change (PGI-C)
Change from baseline
Measured: 52 weeks
Describe patterns of concomitant medication use among this population
Change from baseline in concomitant medication use, assessed using medication logs, electronic health records and patient self-report.
Measured: 52 weeks
Describe changes to medication use among this population
Change from baseline in medication use, evaluated using prescription records, patient-reported medication, or clinician-reported changes.
Measured: 52 weeks
Evaluate cardiovascular (CV) events
Number of CV events defined as death due to any cause plus adjudicated events of hospitalization for heart failure, non-fatal stroke, non-fatal myocardial infarction, non-fatal life-threatening arrhythmia, heart transplant, implantation of left ventricular assisted device (LVAD)
Measured: 52 weeks
Cumulative measure of CV and non-CV related health care utilization (HRU)
Health care utilization will be assessed through a combination of:
- Data captured via Electronic Data Capture (EDC) system (e.g., hospitalizations, emergency room visits, procedures),
- Patient-reported information collected by the physician during scheduled study visits.
Measured: 52 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2025
- Study start30 Sep 2025
- Primary completionSep 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich AtaxiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 65 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Lexeo Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Lexeo Clinical Trials, Study director, Lexeo Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06865482
- Sponsor's protocol ID
- LX2006-02
- Status last verified
- Aug 2026
- First submitted
- 18 Feb 2025
- Last updated
- 19 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Characteristics And Clinical Course Of Disease In Participants With Cardiomyopathy Associated With Friedreich Ataxia (CLARITY-FA)
- ClinicalTrials.gov ID
- NCT06865482
- Acronym / sponsor ID
- LX2006-02
- Conditions
- Friedreich Ataxia, Cardiomyopathy
- Keywords
- Friedreich Ataxia, FA-CM, Cardiomyopathy, FA, Cardiac Disease
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 65 participants (estimated)
- Lead sponsor
- Lexeo TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 30 Sep 2025 (actual)
- Primary completion
- Sep 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 7 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
Detailed description
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: Participants ≥16 years of age with FA-CM | – | –Participants ≥16 years of age with FA-CM |
| Cohort 2: Participants ≥6 to <16 years of age with FA-CM | – | –Participants ≥6 to <16 years of age with FA-CM |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Up to 65 participants with genetically confirmed FA-CM will be enrolled in the study with allocation by age categories and cohorts
Key inclusion criteria (4 listed)
- 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%
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Characterize cardiac disease presentation and progression among participants
Time frame: 52 weeks
Change from baseline in left ventricular mass index (LVMi)
10 secondary outcomes
Describe progression of maximal wall thickness (MWT) among this population
Time frame: 52 weeks
Describe progression of high sensitivity troponin I among this population
Time frame: 52 weeks
Describe participant perception and clinician assessment of illness in Kansas City Cardiomyopathy Questionnaire-12 (KCCQ-12)
Time frame: 52 weeks
Describe clinician assessment of illness in modified Friedreich Ataxia Rating Scale (mFARS)
Time frame: 52 weeks
Describe participant perception of illness in Patient Global Impression of Severity (PGI-S)
Time frame: 52 weeks
Describe participant perception of illness in Patient Global Impression of Change (PGI-C)
Time frame: 52 weeks
Describe patterns of concomitant medication use among this population
Time frame: 52 weeks
Describe changes to medication use among this population
Time frame: 52 weeks
Evaluate cardiovascular (CV) events
Time frame: 52 weeks
Cumulative measure of CV and non-CV related health care utilization (HRU)
Time frame: 52 Weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Lexeo Clinical Trials study director
Lexeo Therapeutics
19 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Friedreich Ataxia and Cardiomyopathy, run by Lexeo Therapeutics at 19 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 19 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States, Brazil, Canada, Czechia, France and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lexeo Clinical Trials (212-547-9879, clinicaltrials@lexeotx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Characterize cardiac disease presentation and progression among participants (time frame: 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Oct 2026.