Recruiting

Observational StudyClinical Course Of Disease In Participants With FA-CM

Sponsor:

Lexeo Therapeutics

Code:

NCT06865482

Conditions

Friedreich Ataxia

Cardiomyopathy

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

Study Details

Brief summary:

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

Conditions

Friedreich Ataxia

Cardiomyopathy

Study ID

NCT06865482

Start date

Sep 30, 2025

Status verified date

Aug, 2026

Completion date

Sep, 2027

Anticipated

Primary completion date

Sep, 2027

Anticipated

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

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 Design

Enrollment

65 participants

Anticipated

Interventions and Outcome Measures

Arms

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

Primary outcome measure

Characterize cardiac disease presentation and progression among participants [ Time Frame: 52 weeks ]

Central Contacts and Locations

Central contacts

Lexeo Clinical Trials

212-547-9879 clinicaltrials@lexeotx.com

Locations

University of California San Diego

Recruiting

La Jolla, California, United States, 92037

Contacts

jiz161@health.ucsd.edu

University of South Florida

Recruiting

Tampa, Florida, United States, 33612

Contacts

lcampbel@usf.edu

anapaulab@usf.edu

Indiana University - Riley Children's Health

Recruiting

Indianapolis, Indiana, United States, 46202

Contacts

lkhernan@iu.edu

Boston Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

Contacts

Ping.Turongpun@childrens.harvard.edu

Mayo Clinic

Recruiting

Rochester, Minnesota, United States, 55905

Contacts

connors.margaret@mayo.edu

Washington University School of Medicine in St. Louis

Recruiting

St Louis, Missouri, United States, 63110

Contacts

aplatts@wustl.edu

Cincinnati Children's Hospital

Recruiting

Cincinnati, Ohio, United States, 45229

Contacts

Jaynee.Bartsch@cchmc.org

sarah.speed@cchmc.org

Oregon Health & Science University

Recruiting

Portland, Oregon, United States, 97239

Contacts

volkh@ohsu.edu

Centre Hospitalier de Universite de Montreal (CHUM)

Recruiting

Montreal, Quebec, Canada, H2X 0C1

Contacts

celia.parinot.chum@ssss.gouv.qc.ca

More Information

Sponsor

Lexeo Therapeutics

Last update posted

Sep 2, 2026

Last verified

Aug, 2026

Keywords

Friedreich Ataxia
FA-CM
Cardiomyopathy
FA
Cardiac Disease

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Lexeo Therapeutics on 2026-09-02.