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% of people with FA develop heart muscle disease, researchers currently do not know much about how the heart is impacted in FA.1
The information from CLARITY-FA will also help advance our gene therapy research.
What is a Natural History Study and Why Is It Important?
In order to develop treatments for a disease, researchers must first learn more about it. A natural history study is a type of research that tracks how a disease develops and changes over time in individuals who have it.
Researchers observe symptoms, changes and patterns to better learn what to expect from the disease, find ways to diagnose it earlier and how to manage the disease better.
Information from natural history studies provides important knowledge that can help develop better or new treatments for a disease.
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 the disease.
Patient Eligibility*
Condition
The study is open to individuals with genetically confirmed FA diagnosis and evidence of cardiomyopathy based on specific criteria.
Population
The study includes two age groups: Individuals who are at least 16 years of age and individuals who are between 6 and 16 years of age.
Duration
If you participate in this study, you will be asked to visit the study site 3 to 4 times over a 52-week period.
*Full eligibility criteria will be evaluated by the clinical trial doctor to determine if an individual is the right candidate for this trial.
Important Information
• This study is sponsored by Lexeo Therapeutics, Inc.
• All study-related assessments will be provided at no cost.
• You will receive no direct payment for taking part in the study.
Participants and their caregiver will be reimbursed for reasonable study-related expenses for travel (e.g., transportation, meals and hotel, as appropriate).
As with any clinical study or trial, there may be risks involved with participation. We encourage any individual considering participation to consult with your physicians or medical team.
Study Logistics
Timing:
Currently enrollingMain Intervention:
Approximately 52 weeksLocations:
Location details forthcoming
References
1. Payne RM. Cardiovascular Research in Friedreich Ataxia. JACC: Basic to Translational Science. 2022;7(12):1267-1283. doi:10.1016/j.jacbts.2022.04.005