Lexeo Therapeutics, Inc. (Lexeo) is conducting a natural history study called CLARITY-FA that is looking for individuals who have cardiomyopathy (heart muscle disease) associated with Friedreich ataxia (FA). The goal of this study is to learn about how heart disease develops and worsens in individuals with FA. Information gained from participation in this study will provide valuable data to learn more about heart disease in FA and help advance Lexeo’s gene therapy research.
It is important to note that participants in natural history studies do not receive any investigational study drug as the goal is to learn about the natural course of that disease.
Anyone considering participating in a clinical trial should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.
Study Details:
Inclusion Criteria:
-Male or female, ages ≥6 years at the time of signing the informed consent (and assent, if applicable)
-Diagnosis of FA confirmed by genetic testing (GAA expansion on both alleles) with onset of FA occurring before 25 years of age
-Confirmed left ventricular hypertrophy (LVH)
-Left ventricular ejection fraction ≥40%
Exclusion Criteria:
-Presence of other forms of cardiomyopathy contributing to heart failure or other clinically significant cardiac issues including anatomic or congenital abnormality, coronary artery disease, or valvular disease
-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
Other inclusion/exclusion criteria may apply.