Clinical Course Of Disease In Participants With FA-CM

Launched by LEXEO THERAPEUTICS · Mar 4, 2025

Keywords

ClinConnect Summary

This clinical trial, called CLARITY-FA, is looking at how Friedreich Ataxia (FA) affects the heart, particularly in people who have a condition known as cardiomyopathy. Cardiomyopathy means that the heart muscle is not working properly, and in this study, researchers want to understand the characteristics and progression of this disease in people who have both FA and heart issues. The trial is not yet recruiting participants, but it aims to include both boys and girls aged 6 years and older who have been diagnosed with FA and have specific heart conditions, such as thickening of the heart muscle and a certain level of heart function.

To join the study, participants must have been diagnosed with FA at a young age (25 or younger), and they need to show certain heart-related symptoms. However, some people may not qualify if they have other heart conditions, severe heart failure, or recent heart surgeries. Those who meet the criteria can expect to undergo tests, including heart imaging, to help researchers learn more about the disease. This information could potentially lead to better care and treatment for individuals with FA-related heart issues in the future.

Gender

ALL

Eligibility criteria

• 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