Clinical Course Of Disease In Participants With FA-CM

June 4, 2026 updated by: Lexeo Therapeutics

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)

Study Overview

Status

Recruiting

Conditions

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-CM. After completing at least 26 weeks in Study LX2006-02, participants who meet the eligibility criteria may have the opportunity to participate in an LX2006 interventional study and receive gene therapy.

Study Type

Observational

Enrollment (Estimated)

65

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

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

Description

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 ≥40%

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
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 the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Characterize cardiac disease presentation and progression among participants
Time Frame: 26 weeks
Change from baseline in left ventricular mass index (LVMi)
26 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Describe progression of left ventricular wall thickness (LVWT) among this population
Time Frame: 52 weeks
Change from baseline
52 weeks
Describe progression of relative wall thickness (RWT) among this population
Time Frame: 52 weeks
Change from baseline
52 weeks
Describe progression of relative wall mass (RWM) among this population
Time Frame: 52 weeks
Change from baseline
52 weeks
Describe progression of high sensitivity troponin I among this population
Time Frame: 52 weeks
Change from baseline
52 weeks
Describe participant perception of illness in Patient Global Impression of Severity (PGI-S)
Time Frame: 52 weeks
Change from baseline
52 weeks
Describe participant perception of illness in Patient Global Impression of Change (PGI-C)
Time Frame: 52 weeks
Change from baseline
52 weeks
Describe patterns of concomitant medication use among this population
Time Frame: 12 months
Change from baseline in concomitant medication use, assessed using medication logs, electronic health records and patient self-report.
12 months
Describe changes to medication use among this population
Time Frame: 12 months
Change from baseline in medication use, evaluated using prescription records, patient-reported medication, or clinician-reported changes.
12 months
Evaluate all-cause mortality
Time Frame: 12 months
Time from baseline to the first occurrence of any event of death due to any cause
12 months
Evaluate major adverse cardiovascular events (MACE)
Time Frame: 12 months
Time from baseline to the first occurrence of any MACE, defined as cardiovascular hospitalization/ambulatory visit, non-fatal stroke, non-fatal life-threatening arrhythmia, heart transplant, implantation of left ventricular assisted device (LVAD)
12 months
Cumulative measure of CV and non-CV related health care utilization (HRU)
Time Frame: 12 months

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.
12 months
Describe progression of left ventricular ejection fraction (LVEF) among this population
Time Frame: 52 weeks
Change from baseline
52 weeks
Describe progression of left ventricular mass index (LVMi) among this population
Time Frame: 52 weeks
Change from baseline
52 weeks
Describe participant perception and clinician assessment of illness in Kansas City Cardiomyopathy Questionnaire-12 (KCCQ-12)
Time Frame: 52 weeks
Change from baseline
52 weeks
Describe clinician assessment of illness in modified Friedreich Ataxia Rating Scale (mFARS)
Time Frame: 52 weeks
Change from baseline
52 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Lexeo Therapeutics

Investigators

  • Study Director: Lexeo Clinical Trials, Lexeo Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 30, 2025

Primary Completion (Estimated)

September 1, 2027

Study Completion (Estimated)

September 1, 2027

Study Registration Dates

First Submitted

February 18, 2025

First Submitted That Met QC Criteria

March 4, 2025

First Posted (Actual)

March 7, 2025

Study Record Updates

Last Update Posted (Actual)

June 8, 2026

Last Update Submitted That Met QC Criteria

June 4, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • LX2006-02

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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