Recruiting

Observational StudyFriedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

Sponsor:

Friedreich's Ataxia Research Alliance

Code:

NCT06016946

Conditions

Friedreich Ataxia

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Accepted

Study Details

Brief 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.

Conditions

Friedreich Ataxia

Study ID

NCT06016946

Start date

Jun 28, 2023

Status verified date

Aug, 2026

Completion date

Jan 28, 2048

Anticipated

Primary completion date

Jan 28, 2048

Anticipated

Eligibility Criteria

Sex: All

Age: 0+

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.

Study Design

Enrollment

3000 participants

Anticipated

Interventions and Outcome Measures

Primary outcome measure

Change in modified Friedreich Ataxia Rating Scale (mFARS) Score [ Time Frame: Baseline, Year 1-25 ]
Change in Scale for the Assessment and Rating of Ataxia (SARA) Score [ Time Frame: Baseline, Year 1-25 ]
Change in FA Activities of Daily Living (ADL) Score [ Time Frame: Baseline, Year 1-25 ]
Change in Upright Stability (US) Score [ Time Frame: Baseline, Year 1-25 ]

Central Contacts and Locations

Central contacts

Mackenzi Coker

+14848796160 fagcc-clinops@curefa.org

Jennifer Farmer

14848796160 jen.farmer@cureFA.org

Locations

UCLA Ataxia Center

Recruiting

Los Angeles, California, United States, 90095

Contacts

Westley Ulit

1-310-206-8153 WUlit@mednet.ucla.edu

Principal Investigator:

Susan Perlman, MD

University of Colorado

Recruiting

Denver, Colorado, United States, 80045

Contacts

Dillan Patel

303-724-2642 DILLAN.PATEL@CUANSCHUTZ.EDU

Principal Investigator:

Trevor Hawkins, MD

University of Florida - Neurology

Recruiting

Gainesville, Florida, United States, 32610

Contacts

Mackenzi Coker

1-352-294-8754 mcoker@peds.ufl.edu

general clinic line

1-352-294-5757

Principal Investigator:

SH Subramony, MD

USF Ataxia Research Center

Recruiting

Tampa, Florida, United States, 33612

Contacts

FA Study

1-813-974-5909 lcampbel@usf.edu

Principal Investigator:

Theresa Zesiewicz, MD

Emory University Hospital - Neurology

Recruiting

Atlanta, Georgia, United States, 30329

Contacts

UNIFAI Study

404-727-1509

Principal Investigator:

George Wilmot, MD

University of Iowa, Stead Family Children's Hospital

Recruiting

Iowa City, Iowa, United States, 52242

Contacts

Corey McDaniel

1-319-335-7498 corey-mcdaniel@uiowa.edu

Principal Investigator:

Katherine Mathews, MD

Ohio State University - Neurology

Recruiting

Columbus, Ohio, United States, 43221

Contacts

Marco Tellez

614-688-7837 marco.tellez@osumc.edu

Alison .Arter

1-614-685-8661 Alison.Arter@osumc.edu

Principal Investigator:

Chad Hoyle, MD

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

FA Program

267-426-7538 FAPROGRAM@chop.edu

Principal Investigator:

David Lynch, MD PhD

St. Jude Children's Research Hospital

Recruiting

Memphis, Tennessee, United States, 38105

Contacts

Jean Laboe

1-901-595-1693 jean.laboe@stjude.org

Principal Investigator:

Richard Finkel, MD

The Hospital for Sick Children

Recruiting

Toronto, Ontario, Canada, M5G 1X8

Contacts

Adrien D'Alonzo

adrien.dalonzo@sickkids.ca

Principal Investigator:

Grace Yoon, MD

CHUM - Hopital Notre-Dame

Recruiting

Montreal, Quebec, Canada, h2x0a9

Contacts

Martine Comeau

514-890-8000 martine.comeau.chum@ssss.gouv.qc.ca

Principal Investigator:

Antoine Duquette, MD

McGill University Health Centre - Montreal Neurological Institute

Recruiting

Montreal, Quebec, Canada, H9R 2Y2

Contacts

Christian Rampal

1-416-659-0750 christian.rampal@mcgill.ca

general clinic line

1-514-398-5500

Principal Investigator:

Massimo Pandolfo, MD

More Information

Sponsor

Friedreich's Ataxia Research Alliance

Last update posted

Sep 1, 2026

Last verified

Aug, 2026

Keywords

Rare Disease
Neuro-degenerative disease
Ataxia
Neurodegenerative Diseases
Neurologic Manifestations
Genetic Diseases, Inborn

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-09. This information was provided to ClinicalTrials.gov by Friedreich's Ataxia Research Alliance on 2026-09-01. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.