Clinical Course Of Disease In Participants With FA-CM

NCT ID: NCT06865482

Last Updated: 2025-12-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

65 participants

Study Classification

OBSERVATIONAL

Study Start Date

2025-09-30

Study Completion Date

2027-09-30

Brief Summary

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

Detailed Description

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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.

Conditions

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Friedreich Ataxia Cardiomyopathy

Keywords

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Friedreich Ataxia FA-CM Cardiomyopathy FA Cardiac Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Cohort 1: Participants ≥16 years of age with FA-CM

Participants ≥16 years of age with FA-CM

No interventions assigned to this group

Cohort 2: Participants 6 to <16 years of age with FA-CM

Participants 6 to \<16 years of age with FA-CM

No interventions assigned to this group

Eligibility Criteria

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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), 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
* 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
Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Lexeo Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Lexeo Clinical Trials

Role: STUDY_DIRECTOR

Lexeo Therapeutics

Locations

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University of California San Diego

La Jolla, California, United States

Site Status RECRUITING

University of South Florida

Tampa, Florida, United States

Site Status RECRUITING

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

Washington University School of Medicine in St. Louis

St Louis, Missouri, United States

Site Status RECRUITING

Cincinnati Children's Hospital

Cincinnati, Ohio, United States

Site Status RECRUITING

Centre Hospitalier de Universite de Montreal (CHUM)

Montreal, Quebec, Canada

Site Status RECRUITING

Countries

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United States Canada

Central Contacts

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Lexeo Clinical Trials

Role: CONTACT

Phone: 212-547-9879

Email: [email protected]

Facility Contacts

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Role: primary

Role: primary

Role: backup

Role: primary

Role: primary

Role: primary

Role: backup

Role: primary

Other Identifiers

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LX2006-02

Identifier Type: -

Identifier Source: org_study_id