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Hope for kids with Friedreich's ataxia: drug already approved for teens now tested in younger children

NCT ID NCT06953583

Disease control Sponsor: Biogen 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests whether omaveloxolone, a drug already approved for people 16 and older with Friedreich's ataxia, can safely help children aged 2 to 15. About 255 participants will first receive either the drug or a placebo for a year, then all will receive the drug for two more years. Researchers will measure changes in movement, balance, and daily function, and monitor side effects and overall health.

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.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 255 people

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

Started

Jun 2025

Expected to finish

Nov 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

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

Ages

2 to 15 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

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.

Part 1: Key inclusion criteria: * Diagnosed with genetically confirmed Friedreich's Ataxia (FA), i.e., homozygous for guanine-adenine-adenine (GAA) repeat expansion in intron-1 of the frataxin gene, or GAA repeat expansion in 1 allele and with point mutations or deletions, or other non-GAA expansion mutations in the other allele. * Symptomatic for FA as confirmed by clinician assessment. a. Children 7 to \< 16 years must also have an upright stability score (USS) score of 10 to ≤ 34 at baseline Part 1: Key exclusion criteria: * Glycosylated hemoglobin A1C (HbA1c) \> 11% * B-type natriuretic peptide (BNP) \> 200 picograms per milliliter (pg/mL) at screening * Ejection fraction (EF) \< 40% \[based on echocardiogram (ECHO) performed at screening visit\] * Clinically significant cardiac disease except mild to moderate cardiomyopathy Part 2A: Eligibility criteria: * They have completed Part 1 of the study and no discontinuation criteria have been met. * Safety and tolerability data from Part 1 are supportive of continuation in the judgement of the investigator. Part 2B: Eligibility criteria: * Participants have completed Part 1 of the study and no discontinuation criteria have been met. * Safety and tolerability data from Part 1 are supportive of continuation in the judgement of the Investigator. Note: Other protocol-defined Inclusion/Exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

Friedreich ataxia

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

    31 sites in 13 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

  • AP-HP - Hôpital Armand Trousseau

    RECRUITING

    Paris, 75012, France

  • All India Institute of Medical Sciences (AIIMS) - New Delhi

    WITHDRAWN

    New Delhi, National Capital Territory of Delhi, 110029, India

  • CHI at Temple Street

    RECRUITING

    Dublin, D01 XD99, Ireland

  • CHKD's Health Center - South Campus - PIN

    RECRUITING

    Norfolk, Virginia, 23507-1910, United States

  • CHU de Montpellier- Hôpital Gui De Chauliac

    RECRUITING

    Montpellier, Hérault, 34090, France

  • CHU de Quebec -Universite Laval

    RECRUITING

    Québec, Quebec, G1V 4G2, Canada

  • Children's Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - PIN

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    RECRUITING

    Milan, 20133, Italy

  • Hospital Sant Joan de Deu - PIN

    RECRUITING

    Espluges de Llobregat, Barcelona, 8950, Spain

  • Hospital Universitario La Paz - PPDS

    RECRUITING

    Madrid, 28046, Spain

  • IRCCS Eugenio Medea - Polo. Scientifico Veneto

    NOT_YET_RECRUITING

    Conegliano, Veneto, 31015, Italy

  • Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi

    WITHDRAWN

    Istanbul, 34093, Turkey (Türkiye)

  • John Radcliffe Hospital

    RECRUITING

    Oxford, Oxfordshire, OX3 9DU, United Kingdom

  • King Faisal Specialist Hospital & Research Centre

    WITHDRAWN

    Riyadh, Ar Riya, 12875, Saudi Arabia

  • L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME

    RECRUITING

    Brasília, Federal District, 70200-730, Brazil

  • McGill University

    RECRUITING

    Montreal, Quebec, H3H 2R9, Canada

  • Murdoch Childrens Research Institute (MCRI)

    RECRUITING

    Parkville, Victoria, 3052, Australia

  • Norman Fixel Institute for Neurological Diseases UF Health

    RECRUITING

    Gainesville, Florida, 32610-3010, United States

  • Ospedale Pediatrico Bambino Gesù IRCCS

    NOT_YET_RECRUITING

    Rome, Lazio, 165, Italy

  • PSEG Centro de Pesquisa Clinica

    RECRUITING

    São Paulo, São Paulo, 04024-002, Brazil

  • Radboud Universitair Medisch Centrum

    RECRUITING

    Nijmegen, 6525 GA, Netherlands

  • Rigshospitalet - Juliane Marie Centret (JMC) Copenhagen

    NOT_YET_RECRUITING

    Copenhagen, 2100, Denmark

  • Seattle Children's Hospital

    RECRUITING

    Seattle, Washington, 98105-3901, United States

  • Sheffield Children's Hospital - PPDS

    RECRUITING

    Sheffield, South Yorkshire, S10 5DD, United Kingdom

  • St. Jude Children's Research Hospital - PIN

    RECRUITING

    Memphis, Tennessee, 38105-3678, United States

  • Sydney Children's Hospital

    NOT_YET_RECRUITING

    Randwick, New South Wales, 2031, Australia

  • UCLA Neurology Outpatient Clinic at Westwood

    NOT_YET_RECRUITING

    Los Angeles, California, 90095, United States

  • UKGM - Universitätsklinikum Giessen und Marburg GmbH - Standort Gießen

    RECRUITING

    Giessen, 35392, Germany

  • USF Health Morsani College of Medicine Department of Neurology

    RECRUITING

    Tampa, Florida, 33612, United States

  • University College Hospital - PPDS

    RECRUITING

    London, Lincolnshire, NW1 2BU, United Kingdom

  • University of Campinas (UNICAMP) School of Medical Sciences

    NOT_YET_RECRUITING

    Campinas, São Paulo, 13083-970, Brazil

  • Universitätsklinikum Aachen

    RECRUITING

    Aachen, North Rhine-Westphalia, 52074, Germany

  • Universitätsklinikum Hamburg Eppendorf

    RECRUITING

    Hamburg, 20246, Germany

  • Universitätsklinikum Innsbruck

    RECRUITING

    Innsbruck, 6020, Austria

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