NCT06016946 · Friedreich's Ataxia Research Alliance
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
(UNIFAI)
What this study is about
This project is a global, conducted at multiple hospitals, forward-looking, longitudinal, observational natural history study that can be used to understand the disease progression and support the development of safe and effective drugs and biological products for Friedreich ataxia.
View original scientific description
This project is a global, multicenter, prospective, longitudinal, observational natural history study that can be used to understand the disease progression and support the development of safe and effective drugs and biological products for Friedreich ataxia.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Both males and females of any age
- Individuals with Friedreich ataxia (FA): Participants that meet genetically confirmed diagnosis of Friedreich ataxia
- Written informed consent provided
- 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
Exclusion criteria
- Unable or unwilling to provide informed consent
- Acute or ongoing medical or other conditions that would interfere with the conduct and assessments of the study
- For any reason in the opinion of the investigator, participant would be unlikely or unable to comply with study protocol requirements.
Where
- Los Angeles, California
- Denver, Colorado
- Gainesville, Florida
- Tampa, Florida
- Atlanta, Georgia
- Iowa City, Iowa
- Columbus, Ohio
- Philadelphia, Pennsylvania
- Memphis, Tennessee
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 17, 2025 · Source of record for eligibility and locations