Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia (NCT06560346) | Clinical Trial Compass
WithdrawnNot Applicable
Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia
Stopped: Lack of Funding
United States, Australia, Canada0Started 2025-05-01
Plain-language summary
Multicenter, prospective, observational natural history and outcome measure study of children and young adults with Friedreich ataxia.
Who can participate
Age range
4 Years – 21 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Genetic diagnosis of Friedreich Ataxia
. Ages 4-21 years at enrollment
. Enrollment in the UNIFAI study and ability to have simultaneous visits for both UNIFAI and EARLY-FA
. Informed consent must be obtained for all participants:
. For underage participants, they and the parent/ legally authorized representative have to sign the informed consent form, child assent (if applicable)
. Persons who are not legally competent require the informed consent of their legally authorized representative
. Ages 4-21 years at enrollment
. Matching criteria to an enrolled participant with FA (age, sex and educational status)
Exclusion criteria
. Diagnosis of non-FA medical or other condition that in the opinion of the investigator would interfere with the conduct and assessments of the study or be confounding and contraindication to participation.
. Pregnant female participants
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Correlation between growth (height in z-score) and disease severity in FRDA (mFARS score)