Actively Recruiting
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
Led by Friedreich's Ataxia Research Alliance · Updated on 2025-06-17
3000
Participants Needed
34
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a global, multicenter, prospective, longitudinal, observational natural history study to better understand Friedreich ataxia, a rare inherited neurological and cardiac condition caused by mutations in the FXN gene. This study aims to support the development of safe and effective drugs and biological treatments by tracking the disease progression in diagnosed individuals worldwide over time. Participants will be assessed annually for up to 25 years through yearly study visits. Data collection includes medical records, neurological exams, timed walking tests, upper limb function measures, and patient-reported quality of life assessments. Additional optional assessments at some sites may cover speech, vision, fatigue, balance, and cognition. This study combines previous natural history studies to strengthen data and expand research networks globally. During the study, participants will undergo evaluations such as neurological and functional assessments, cardiac examinations, laboratory tests, and health questionnaires. Researchers will monitor changes in clinical outcomes like disease progression, symptom severity, and quality of life using measures including the modified Friedreich Ataxia Rating Scale and the Scale for the Assessment and Rating of Ataxia. This long-term observation aims to identify disease milestones, track treatment impacts, and inform future clinical trials and outcome measures.
CONDITIONS
Brief Title
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Both males and females of any age
- Genetically confirmed diagnosis of Friedreich ataxia
- Written informed consent provided by participant or legally authorized representative
- For minors, assent from the child and consent from parent or guardian must be obtained
You will not qualify if you...
- Unable or unwilling to provide informed consent
- Acute or ongoing medical or other conditions interfering with study participation
- Unlikely or unable to comply with study protocol requirements as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 25 years
Participants attend yearly study visits where data on disease progression, symptoms, and quality of life are collected through neurological exams, functional assessments, and questionnaires.
Annual visits
Trial Site Locations
Total: 34 locations
1
UCLA Ataxia Center
Los Angeles, California, United States, 90095
Actively Recruiting
2
University of Colorado
Denver, Colorado, United States, 80045
Actively Recruiting
3
University of Florida - Neurology
Gainesville, Florida, United States, 32610
Actively Recruiting
4
USF Ataxia Research Center
Tampa, Florida, United States, 33612
Actively Recruiting
5
Emory University Hospital - Neurology
Atlanta, Georgia, United States, 30329
Actively Recruiting
6
University of Iowa, Stead Family Children's Hospital
Iowa City, Iowa, United States, 52242
Actively Recruiting
7
Ohio State University - Neurology
Columbus, Ohio, United States, 43221
Actively Recruiting
8
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
9
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
10
Murdoch Childrens Research Institute
Parkville, Victoria, Australia, 3052
Actively Recruiting
11
Medical University Innsbruck, Department of Neurology
Innsbruck, Austria, 6020
Actively Recruiting
12
Université Libre de Bruxelles, Hôpital Erasme, Dpt of Neurology
Brussels, Belgium, 1070
Actively Recruiting
13
University of Campinas
Campinas, Brazil
Not Yet Recruiting
14
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Actively Recruiting
15
CHUM - Hopital Notre-Dame
Montreal, Quebec, Canada, h2x0a9
Actively Recruiting
16
McGill University Health Centre - Montreal Neurological Institute
Montreal, Quebec, Canada, H9R 2Y2
Actively Recruiting
17
Motol University Hospital, Centre for Hereditary Ataxias
Prague, Czechia, 150 06
Actively Recruiting
18
Paris Brain Institute
Paris, France, 75013
Actively Recruiting
19
Hôpital de Hautepierre, Service de Neurologie
Strasbourg, France, 67098
Not Yet Recruiting
20
University Hospital Aachen, Dept. of Neurology
Aachen, Germany, 52074
Actively Recruiting
21
Deutsches Zentrum Für Neurodegenerative Erkrankungen
Bonn, Germany, 53105
Not Yet Recruiting
22
University of Munich, Dept. of Neurology, Friedrich-Baur-Institut
Munich, Germany, 80336
Actively Recruiting
23
University of Tübingen, Dept. of Neurodegenerative Diseases, Hertie-Institute for Clinical Brain Research
Tübingen, Germany, 72076
Actively Recruiting
24
National and Kapodistrian University of Athens, Neurogenetics Unit
Athens, Greece, 11528
Actively Recruiting
25
All India Institute of Medical Sciences (AIIMS)
New Delhi, National Capital Territory of Delhi, India, 110020
Actively Recruiting
26
Tallaght University Hospital, Department of Neurology
Dublin, Ireland, D24 TP66
Actively Recruiting
27
Referente Clinico-Scientifico di Polo IRCCS "E. Medea"
Conegliano, Italy, 21015
Actively Recruiting
28
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Italy, 20133
Actively Recruiting
29
Bambino Gesù Children's Hospital, Department of Neurosciences
Roma, Italy, 00146
Not Yet Recruiting
30
Stichting Radboud Universitair Medisch Centrum
Nijmegen, Netherlands, 6525
Actively Recruiting
31
Auckland City Hospital
Auckland, New Zealand, 1023
Actively Recruiting
32
Hospital Sant Joan de Déu, Servicio de Neurología
Barcelona, Spain, 08950
Not Yet Recruiting
33
Hospital Universitario La Paz, Servicio de Neurologia
Madrid, Spain, 28046
Not Yet Recruiting
34
University College of London, Ataxia Centre, National Hospital for Neurology and Neurosurgery
London, United Kingdom, WC1N 3 BG
Not Yet Recruiting
Research Team
C
Cait Monette
J
Jennifer Farmer
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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