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Can a 25-Year global study unlock the secrets of friedreich ataxia?

NCT ID NCT06016946

Knowledge-focused Sponsor: Friedreich's Ataxia Research Alliance Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Sep 01, 2026 · Last updated Sep 02, 2026 · Updated 1 time

Summary

This study follows 3,000 people with Friedreich ataxia, a rare inherited disease that affects movement and coordination, to map how the condition changes over time. Researchers will measure symptoms yearly using standard rating scales for up to 25 years. The goal is to build a detailed picture of disease progression that can help design and evaluate future treatments.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

What this could lead to
If this study succeeds, it could provide the detailed understanding of Friedreich ataxia progression needed to design and evaluate new treatments more effectively.
What could go wrong
This is an observational study, not a treatment trial, so it offers no direct benefit to participants. The long follow-up may face dropouts, and results depend on consistent data collection across many sites.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 3,000 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2023

Expected to finish

Jan 2048

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

The study will include current participants from the existing FA-COMS and EFACTS natural history studies as well as enroll new individuals with Friedreich ataxia (FA) and healthy controls.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

Ataxia Friedreich ataxia hereditary disease neurodegenerative disease Neurologic Manifestations Rare Diseases

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Friedreich ataxia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    34 sites in 17 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • All India Institute of Medical Sciences (AIIMS)

    RECRUITING

    New Delhi, National Capital Territory of Delhi, 110020, India

  • Auckland City Hospital

    RECRUITING

    Auckland, 1023, New Zealand

  • Bambino Gesù Children's Hospital, Department of Neurosciences

    NOT_YET_RECRUITING

    Roma, 00146, Italy

  • CHUM - Hopital Notre-Dame

    RECRUITING

    Montreal, Quebec, h2x0a9, Canada

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Deutsches Zentrum Für Neurodegenerative Erkrankungen

    NOT_YET_RECRUITING

    Bonn, 53105, Germany

  • Emory University Hospital - Neurology

    RECRUITING

    Atlanta, Georgia, 30329, United States

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    RECRUITING

    Milan, 20133, Italy

  • Hospital Sant Joan de Déu, Servicio de Neurología

    NOT_YET_RECRUITING

    Barcelona, 08950, Spain

  • Hospital Universitario La Paz, Servicio de Neurologia

    NOT_YET_RECRUITING

    Madrid, 28046, Spain

  • Hôpital de Hautepierre, Service de Neurologie

    NOT_YET_RECRUITING

    Strasbourg, 67098, France

  • McGill University Health Centre - Montreal Neurological Institute

    RECRUITING

    Montreal, Quebec, H9R 2Y2, Canada

  • Medical University Innsbruck, Department of Neurology

    RECRUITING

    Innsbruck, 6020, Austria

  • Motol University Hospital, Centre for Hereditary Ataxias

    RECRUITING

    Prague, 150 06, Czechia

  • Murdoch Childrens Research Institute

    RECRUITING

    Parkville, Victoria, 3052, Australia

  • National and Kapodistrian University of Athens, Neurogenetics Unit

    RECRUITING

    Athens, 11528, Greece

  • Ohio State University - Neurology

    RECRUITING

    Columbus, Ohio, 43221, United States

  • Paris Brain Institute

    RECRUITING

    Paris, 75013, France

  • Referente Clinico-Scientifico di Polo IRCCS "E. Medea"

    RECRUITING

    Conegliano, 21015, Italy

  • St. Jude Children's Research Hospital

    RECRUITING

    Memphis, Tennessee, 38105, United States

  • Stichting Radboud Universitair Medisch Centrum

    RECRUITING

    Nijmegen, 6525, Netherlands

  • Tallaght University Hospital, Department of Neurology

    RECRUITING

    Dublin, D24 TP66, Ireland

  • The Hospital for Sick Children

    RECRUITING

    Toronto, Ontario, M5G 1X8, Canada

  • UCLA Ataxia Center

    RECRUITING

    Los Angeles, California, 90095, United States

  • USF Ataxia Research Center

    RECRUITING

    Tampa, Florida, 33612, United States

  • University College of London, Ataxia Centre, National Hospital for Neurology and Neurosurgery

    NOT_YET_RECRUITING

    London, WC1N 3 BG, United Kingdom

  • University Hospital Aachen, Dept. of Neurology

    RECRUITING

    Aachen, 52074, Germany

  • University of Campinas

    NOT_YET_RECRUITING

    Campinas, Brazil

  • University of Colorado

    RECRUITING

    Denver, Colorado, 80045, United States

  • University of Florida - Neurology

    RECRUITING

    Gainesville, Florida, 32610, United States

  • University of Iowa, Stead Family Children's Hospital

    RECRUITING

    Iowa City, Iowa, 52242, United States

  • University of Munich, Dept. of Neurology, Friedrich-Baur-Institut

    RECRUITING

    Munich, 80336, Germany

  • University of Tübingen, Dept. of Neurodegenerative Diseases, Hertie-Institute for Clinical Brain Research

    RECRUITING

    Tübingen, 72076, Germany

  • Université Libre de Bruxelles, Hôpital Erasme, Dpt of Neurology

    RECRUITING

    Brussels, 1070, Belgium

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