CLARITY-FA
Research type
Research Study
Full title
Characteristics and Clinical Course of Disease in Participants with Cardiomyopathy Associated with Friedreich Ataxia
IRAS ID
362019
Contact name
Greg Aubert
Contact email
Sponsor organisation
Lexeo Therapeutics, Inc.
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 2 months, 1 days
Research summary
Friedreich ataxia (FA) is a rare inherited condition that affects many parts of the body, especially the nervous system and heart. It is the most common type of inherited ataxia (a group of disorders that cause problems with coordination and balance). People with FA often begin to show symptoms in childhood or adolescence. These may include trouble with balance, frequent falls, tiredness, and increasing difficulty walking. Over time, other symptoms such as slurred speech, foot abnormalities, and curvature of the spine (scoliosis) may develop.
A serious complication of FA is heart disease, particularly a form called cardiomyopathy (FA-CM). This condition affects the heart muscle, making it thicker and stiffer, which can lead to heart problems. About 81% of people with FA develop cardiomyopathy, and it is the main cause of death in this group.
The goal of Study LX2006-02 is to better understand how heart disease develops and changes over time in people with FA-CM. The study will collect information using heart scans, blood tests, patient and doctor reports, and healthcare usage data. This will help researchers understand how the disease progresses with current treatments and identify areas where new treatments are needed. Participants will continue with their usual medical care while researchers collect information to help improve future treatment and care. The study does not involve any new or experimental treatments.
The company running the study, Lexeo, is also planning a future trial to test LX2006, a gene therapy being developed for FA-CM. Information from study LX2006-02 may help create a comparison group for that trial. People in the current study who qualify may be able to join the gene therapy study when it becomes available.REC name
South East Scotland REC 02
REC reference
26/SS/0018
Date of REC Opinion
4 Mar 2026
REC opinion
Further Information Unfavourable Opinion