Clinical Research Alert: Natural History Study of Individuals with Cardiomyopathy Associated with FRDA

Researchers at Lexeo Therapeutics are seeking individuals with cardiomyopathy associated with Friedreich’s Ataxia (FRDA) to participate in an observational study (CLARITY-FA). The assessments and questionnaires from this study will help to provide valuable data about heart disease in FRDA and advance Lexeo’s gene therapy research.

The study

This is an observational study, which means that participants will not receive an investigational study drug. The study will include two age groups: (1) Individuals who are at least 16 years of age and (2) individuals who are between 6 and under 16 years of age. After an initial screening visit, eligible participants will be required to attend five doctor visits over a period of one year. The study doctor will collect medical information and perform routine study assessments, including laboratory tests (e.g. blood and urine), imaging, questionnaires, and safety assessments.

Study criteria

To be eligible for the research study, individuals must meet the following inclusion criteria:

• Male or female, ages ≥6 years at the time of signing the informed consent (and assent, if applicable).
• Diagnosis of FRDA, based on clinical phenotype and genotype (GAA expansion on both alleles), with onset of FRDA occurring at ≤25 years of age
• Confirmed left ventricular hypertrophy (LVH)
• Left ventricular ejection fraction ≥40%

Individuals may not be eligible to participate if they are affected by another condition or receiving another treatment that might interfere with the ability to undergo safe testing. Eligibility criteria will be evaluated by the clinical study doctor.

Interested in participating?

To learn more about the study or inquire about participation, visit the study website through this link or email clincaltrials@lexeotx.com.