Global study on the natural history of Friedreich Ataxia
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
This study looks at how Friedreich Ataxia changes over time in people with the condition to better understand its progression and improve future treatments.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 3000 (estimated) |
| Sex | All |
| Sponsor | Friedreich's Ataxia Research Alliance Academic / other |
| Locations | 34 sites (Los Angeles, California and 33 other locations) |
| Trial ID | NCT06016946 on ClinicalTrials.gov |
What this trial studies
The UNIFAI study is a global, multicenter, prospective, longitudinal observational study aimed at understanding the natural progression of Friedreich Ataxia, a rare genetic disorder. Participants with genetically confirmed Friedreich Ataxia will be assessed annually for up to 25 years, with data collected from medical records, clinical evaluations, and patient-reported outcomes. This study seeks to harmonize existing natural history studies to enhance the understanding of clinical measures that can quantitatively assess the disease.
Who should consider this trial
Good fit: Ideal candidates include individuals of any age with a genetically confirmed diagnosis of Friedreich Ataxia.
Not a fit: Patients who are unable or unwilling to provide informed consent or have acute medical conditions that interfere with study assessments may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide critical insights that support the development of effective treatments for Friedreich Ataxia.
How similar studies have performed: Previous natural history studies in Friedreich Ataxia have laid the groundwork for this research, indicating a potential for success in understanding the disease's progression.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Both males and females of any age 2. Individuals with Friedreich ataxia (FA): Participants that meet genetically confirmed diagnosis of Friedreich ataxia 3. Written informed consent provided 1. Informed consent must be obtained for all participants 2. For underage participants, they and the parent/ legally authorized representative have to sign the informed consent form, child assent (if applicable) 3. Persons who are not legally competent require the informed consent of their legally authorized representative Exclusion Criteria: 4. Unable or unwilling to provide informed consent 5. Acute or ongoing medical or other conditions that would interfere with the conduct and assessments of the study 6. For any reason in the opinion of the investigator, participant would be unlikely or unable to comply with study protocol requirements.
Where this trial is running
Los Angeles, California and 33 other locations
- UCLA Ataxia Center — Los Angeles, California, United States (Recruiting)
- University of Colorado — Denver, Colorado, United States (Recruiting)
- University of Florida - Neurology — Gainesville, Florida, United States (Recruiting)
- USF Ataxia Research Center — Tampa, Florida, United States (Recruiting)
- Emory University Hospital - Neurology — Atlanta, Georgia, United States (Recruiting)
- University of Iowa, Stead Family Children's Hospital — Iowa City, Iowa, United States (Recruiting)
- Ohio State University - Neurology — Columbus, Ohio, United States (Recruiting)
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (Recruiting)
- St. Jude Children's Research Hospital — Memphis, Tennessee, United States (Recruiting)
- Murdoch Childrens Research Institute — Parkville, Victoria, Australia (Recruiting)
- Medical University Innsbruck, Department of Neurology — Innsbruck, Austria (Recruiting)
- Université Libre de Bruxelles, Hôpital Erasme, Dpt of Neurology — Brussels, Belgium (Recruiting)
- University of Campinas — Campinas, Brazil (Not_yet_recruiting)
- The Hospital for Sick Children — Toronto, Ontario, Canada (Recruiting)
- CHUM - Hopital Notre-Dame — Montreal, Quebec, Canada (Recruiting)
- McGill University Health Centre - Montreal Neurological Institute — Montreal, Quebec, Canada (Recruiting)
- Motol University Hospital, Centre for Hereditary Ataxias — Prague, Czechia (Recruiting)
- Paris Brain Institute — Paris, France (Recruiting)
- Hôpital de Hautepierre, Service de Neurologie — Strasbourg, France (Not_yet_recruiting)
- University Hospital Aachen, Dept. of Neurology — Aachen, Germany (Recruiting)
- Deutsches Zentrum Für Neurodegenerative Erkrankungen — Bonn, Germany (Not_yet_recruiting)
- University of Munich, Dept. of Neurology, Friedrich-Baur-Institut — Munich, Germany (Recruiting)
- University of Tübingen, Dept. of Neurodegenerative Diseases, Hertie-Institute for Clinical Brain Research — Tübingen, Germany (Recruiting)
- National and Kapodistrian University of Athens, Neurogenetics Unit — Athens, Greece (Recruiting)
- All India Institute of Medical Sciences (AIIMS) — New Delhi, National Capital Territory of Delhi, India (Recruiting)
- Tallaght University Hospital, Department of Neurology — Dublin, Ireland (Recruiting)
- Referente Clinico-Scientifico di Polo IRCCS "E. Medea" — Conegliano, Italy (Recruiting)
- Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan, Italy (Recruiting)
- Bambino Gesù Children's Hospital, Department of Neurosciences — Roma, Italy (Not_yet_recruiting)
- Stichting Radboud Universitair Medisch Centrum — Nijmegen, Netherlands (Recruiting)
- Auckland City Hospital — Auckland, New Zealand (Recruiting)
- Hospital Sant Joan de Déu, Servicio de Neurología — Barcelona, Spain (Not_yet_recruiting)
- Hospital Universitario La Paz, Servicio de Neurologia — Madrid, Spain (Not_yet_recruiting)
- University College of London, Ataxia Centre, National Hospital for Neurology and Neurosurgery — London, United Kingdom (Not_yet_recruiting)
Study contacts
- Principal investigator: David Lynch, MD — Children's Hospital of Philadelphia
- Study coordinator: Cait Monette
- Email: cait.monette@cureFA.org
- Phone: 16513291892
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.