CLARITY-FA
Characteristics and clinical course of disease in participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
The goal of the Lexeo sponsored CLARITY-FA natural history study is to learn about how heart disease develops and worsens in individuals with FA. This is important because even though approximately 80% percent of people with FA develop heart muscle disease, researchers currently do not know much about how the heart is impacted in FA. Information gained from participation in this study will provide valuable data to learn more about heart disease in FA. Unfortunately, there currently are no approved treatments for heart muscle disease in FA. Lexeo is researching an investigational gene therapy, called LX2006, to address this unmet need. The information from CLARITY-FA will also help advance our gene therapy research.
Link to Enroll: https://trials.lexeotx.com
Disclaimer: This research opportunity is not affiliated with NAF. We are sharing this information as a resource for the Ataxia community. Sharing this information does not imply endorsement by NAF.
About the Study
Eligible Ataxia Types
Friedreich Ataxia
Type of Study
Observational Clinical Trial
Clinical Trial Phase
N/A
Age Range
Ages 6+
Study Start Date
06/17/2025
Estimated Completion Date
N/A
IRB Approval #
Pro00084390
Location(s)
Washington University School of Medicine
St. Louis, MO
University of South Florida
Tampa, FL
University of California, San Diego
San Diego, CA
Cincinnati Children’s Hospital
Cincinnati, OH
Boston Children’s Hospital
Boston, MA
Riley Children’s Health (Indiana University)
Indianapolis, IN
Centre hospitalier de l’Universite de Montréal
Montréal, Canada
Motol University Hospital and the Second Faculty of Medicine, Charles University
Prague, Czech Republic
L’Institut de Cardiologie de la Pitié-Salpêtrière
Paris, France
Hospital Universitarlo Puerta de Hierro de Majadahondal
Madrid, Spain
Contact Information
What does participation in the study entail?
Participants do not receive any investigational study drug. After screening, there will be 5 visits over a period of 1 year. Your study doctor will learn about your heart disease through collecting medical information and performing routine study assessments, including lab tests (including blood and urine), imaging, questionnaires, and safety assessments.
What are the potential benefits for participants?
Information gained from participation in this study will provide valuable data to learn more about heart disease in FA and help advance our gene therapy research. Additionally, data collected from CLARITY-FA will be used to compare the results collected in the new gene therapy interventional study.
Participation in the CLARITY-FA study is voluntary. You may withdraw from the study at any time for any reason. If you refuse to participate or decide to withdraw, you will not suffer any penalty, loss of rights, or loss of benefits to which you are entitled.
What are the potential risks for participants?
As with any clinical study or trial, there may be risks involved with participation. We encourage any individual considering participation in a clinical study or trial to consult with your physician or medical team.
Is there financial compensation?
No.
Is there travel reimbursement?
Yes. Participants and their caregiver will be reimbursed for reasonable study-related expenses for travel (e.g., transportation, meals, and hotel, as appropriate).
Who is eligible?
The study includes two age groups: Individuals who are at least 16 years of age, and individuals who are between 6 and under 16 years of age. The study is open to individuals with genetically confirmed FA diagnosis and evidence of cardiomyopathy based on specific criteria. Full eligibility criteria will be evaluated by the clinical study doctor.
Participants taking Skyclarys® should be on a stable dose for at least 12 weeks prior to the Baseline visit and remain at a stable dose throughout the study.
Additional Information or Resources
Link to Enroll: https://trials.lexeotx.com
ClinicalTrials.gov Identifier# NCT06865482
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