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NCT06865482Recruiting

Clinical Course Of Disease In Participants With FA-CM

Lexeo Therapeutics

Start Date

9/30/2025

Completion Date

9/1/2027

Summary

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-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.

Eligibility Criteria

Age Range: 6 years to No maximum

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

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Conditions

Friedreich AtaxiaCardiomyopathy

Locations

University of California San Diego

La Jolla, California 92037

United States

University of South Florida

Tampa, Florida 33612

United States

Indiana University - Riley Children's Health

Indianapolis, Indiana 46202

United States

Boston Children's Hospital

Boston, Massachusetts 02115

United States

Mayo Clinic

Rochester, Minnesota 55905

United States

Washington University School of Medicine in St. Louis

St Louis, Missouri 63110

United States

Cincinnati Children's Hospital

Cincinnati, Ohio 45229

United States

Oregon Health & Science University

Portland, Oregon 97239

United States

Hospital Universitário Walter Cantídio - Universidade Federal do Ceará

Fortaleza, Ceará 60430

Brazil

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

Salvador, Estado de Bahia 41253-190

Brazil

Hospital Das Clínicas Da Universidade Federal De Minas Gerais (UFMG)

Belo Horizonte, Minas Gerais 30130

Brazil

Campinas State University (UNICAMP)

Campinas, São Paulo 13083-887

Brazil

UPC Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto

Ribeirão Preto, São Paulo 14051

Brazil

Centre Hospitalier de Universite de Montreal (CHUM)

Montreal, Quebec H2X 0C1

Canada

Motol University Hospital and the Second Faculty of Medicine

Prague, 15006

Czechia

Institut de Cardiologie Hopital Pitie Salpetriere

Paris, 75013

France

University Hospital Tübingen

Tübingen, Baden-Wurttemberg 72076

Germany

Federico II University

Naples, 80131

Italy

Hospital Univeritario Puerta de Hierro de Majadahonda

Majadahonda, Madrid 28222

Spain