Clinical Course Of Disease In Participants With FA-CM

NCT06865482 | Recruiting

• 1 other identifier: LX2006-02
• Study Type: observational
• Target: 65
• Locations: 7 countries
• Sites: 14

Brief Summary

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

Conditions

Friedreich Ataxia; Cardiomyopathy

Keywords

Friedreich Ataxia; FA-CM; Cardiomyopathy; FA; Cardiac Disease

Outcome Measures

Primary Outcomes (1)

• Characterize cardiac disease presentation and progression among participants

  Change from baseline in left ventricular mass index (LVMi)

  26 weeks

Secondary Outcomes (15)

• Describe progression of left ventricular wall thickness (LVWT) among this population

  52 weeks

• Describe progression of relative wall thickness (RWT) among this population

  52 weeks

• Describe progression of relative wall mass (RWM) among this population

  52 weeks

• Describe progression of left ventricular ejection fraction (LVEF) among this population

  52 weeks

• Describe progression of left ventricular mass index (LVMi) among this population

  52 weeks

Study Arms (2)

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

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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%

You may not qualify if:

• 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

Sponsors & Collaborators

• Lexeo Therapeutics: lead

Study Sites (14)

University of California San Diego

La Jolla, California, 92037, United States

RECRUITING

University of South Florida

Tampa, Florida, 33612, United States

RECRUITING

Indiana University - Riley Children's Health

Indianapolis, Indiana, 46202, United States

RECRUITING

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, 55905, United States

RECRUITING

Washington University School of Medicine in St. Louis

St Louis, Missouri, 63110, United States

RECRUITING

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

RECRUITING

Instituto D'or de Pesquisa e Ensino - Hospital São Rafael

Salvador, Estado de Bahia, 41253-190, Brazil

RECRUITING

Campinas State University (UNICAMP)

Campinas, São Paulo, 13083-887, Brazil

RECRUITING

Centre Hospitalier de Universite de Montreal (CHUM)

Montreal, Quebec, H2X 0C1, Canada

RECRUITING

Motol University Hospital and the Second Faculty of Medicine

Prague, 15006, Czechia

RECRUITING

Institut de Cardiologie Hopital Pitie Salpetriere

Paris, 75013, France

RECRUITING

University Hospital Tübingen

Tübingen, Baden-Wurttemberg, 72076, Germany

RECRUITING

Hospital Univeritario Puerta de Hierro de Majadahonda

Majadahonda, Madrid, 28222, Spain

RECRUITING

MeSH Terms

Conditions

Friedreich Ataxia; Cardiomyopathies; Heart Diseases

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Cardiovascular Diseases

Study Officials

• Lexeo Clinical Trials

  Lexeo Therapeutics

  STUDY DIRECTOR

Central Study Contacts

Lexeo Clinical Trials

CONTACT

212-547-9879; clinicaltrials@lexeotx.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 18, 2025
• First Posted: March 7, 2025
• Study Start: September 30, 2025
• Primary Completion (Estimated): September 1, 2027
• Study Completion (Estimated): September 1, 2027
• Last Updated: April 15, 2026

Record last verified: 2026-04

Locations

ES (1); US (7); CA (1); FR (1); CZ (1); DE (1); BR (2)