Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

NCT06016946 · tracked via the Priya Life Science India tracker

Sponsor

Friedreich's Ataxia Research Alliance

Phase

Observational

Started

2023-06-28

Last updated

2026-09-01

Condition(s) studied

Friedreich Ataxia

Study summary

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.

Eligibility

Sex

ALL

Min age

—

Max age

—

Healthy volunteers

Accepted

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.

Primary outcome measure(s)

Change in modified Friedreich Ataxia Rating Scale (mFARS) Score — Baseline, Year 1-25
The modified Friedreich Ataxia Rating Scale (mFARS) is a disease-specific scale that measures progression of neurological effects of FA. The mFARS is a validated and reliable scale; comprised of the neurologic component of the FARS and evaluates bulbar, upper limb, lower limb, and upright stability/gait function. For each item, responses categorize the corresponding neurological finding, and the findings are assigned a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. The score ranges from 0 to 93. The score will be compared to the previous year annually for up to 25 years.
Change in Scale for the Assessment and Rating of Ataxia (SARA) Score — Baseline, Year 1-25
The Scale for the Assessment and Rating of Ataxia (SARA) is a semi-quantitative assessment of ataxia, measuring ataxia of upper limb, lower limb, gait, balance and speech. It has eight items: gait, stance, sitting, speech disturbance, finger chase, nose-finger test, fast alternating hand movement, and heel-shin slide. The total score ranges from 0 (no ataxia) to 40 (severe ataxia). The score will be compared to the previous year annually for up to 25 years.
Change in FA Activities of Daily Living (ADL) Score — Baseline, Year 1-25
The FARS-ADL is a subsection of the FARS questionnaire that assesses activities of daily living, including speech, personal hygiene, feeding, and mobility. Participants rank each category using a scale of 0 (normal) to 4 (severe disability/ inability to carry out activity independently), with lower scores indicative of "normal" function/activity. The score will be compared to the previous year annually for up to 25 years.
Change in Upright Stability (US) Score — Baseline, Year 1-25
The Upright Stability (US) assessment is part of the neurological examination within the Modified Friedreich Ataxia Rating Scale (mFARS). This component comprises nine items: sitting position, stance with feet apart, stance with feet apart and eyes closed, stance with feet together, stance with feet together and eyes closed, tandem stance, stance with dominant foot, tandem walk, and gait. The score ranges from 0 to 9, with a higher score reflecting poorer upright stability (i.e., greater neurological severity). The score will be compared to the previous year annually for up to 25 years.

Trial sites (34)

Facility	City	Region	Status
UCLA Ataxia Center	Los Angeles	California	Recruiting
University of Colorado	Denver	Colorado	Recruiting
University of Florida - Neurology	Gainesville	Florida	Recruiting
USF Ataxia Research Center	Tampa	Florida	Recruiting
Emory University Hospital - Neurology	Atlanta	Georgia	Recruiting
University of Iowa, Stead Family Children's Hospital	Iowa City	Iowa	Recruiting
Ohio State University - Neurology	Columbus	Ohio	Recruiting
Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	Recruiting
St. Jude Children's Research Hospital	Memphis	Tennessee	Recruiting
Murdoch Childrens Research Institute	Parkville	Victoria	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	Recruiting
CHUM - Hopital Notre-Dame	Montreal	Quebec	Recruiting
McGill University Health Centre - Montreal Neurological Institute	Montreal	Quebec	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	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

More Friedreich's Ataxia Research Alliance trials in India

FA Clinical Outcome Measures Active, not recruiting

Other trials for the same condition

FA Clinical Outcome Measures Active, not recruiting

A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06016946 on ClinicalTrials.gov ↗ ← All trials in India