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Clinical trialNCT06953583RecruitingPhase 3Interventional

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

SponsorBiogenEnrollment255Friedreich Ataxia
View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

In this study, researchers will learn more about omaveloxolone, also known as BIIB141 or SKYCLARYS®. Omaveloxolone is already approved for people with Friedreich's Ataxia (FA) who are 16 years of age or older. However, it is not yet available for younger teens and children. The main goal of this study is to learn how omaveloxolone affects symptoms of FA and its safety in younger participants between the ages of 2 and 15 years old. The main questions researchers want to answer in this study are: * How does omaveloxolone affect the participants' FA symptoms? * How many participants have adverse events during the study? * Are there any changes in the participants' overall health or heart health? Adverse events are health problems that may or may not be caused by the study drug. Researchers will use the modified Friedreich's Ataxia Rating Scale (mFARS) to test nerve function. The mFARS tests movement ability, balance, coordination, speech, and arm and leg functions. They will also use a number of questionnaires to learn more about participants' quality of life, muscle strength, and ability to perform daily tasks. Researchers will also note any changes as participants go through puberty. Finally, researchers will learn more about how the body processes omaveloxolone in children and teenagers. This study will be done in 2 parts as follows: * Participants will be screened for up to 4 weeks to check if they can join the study. * In Part 1, participants will be randomly assigned to take either omaveloxolone or a placebo by mouth once a day for about 1 year. A placebo looks like the study drug but contains no real medicine. * Part 1 will be double blind. This means that the participants, study doctor, and site staff will not know if the participants are receiving omaveloxolone or a placebo. * Including screening, participants will have up to 9 clinic visits and 1 phone call during Part 1. If a participant does not join Part 2, they will have another safety follow-up phone call a month after their last dose of omaveloxolone. * Participants who complete Part 1 will move onto Part 2 where everyone will receive omaveloxolone for about 2 years. * During Part 2, participants will have up to 8 clinic visits and 1 phone call. Participants will also have a follow-up phone call about a month after they stop taking omaveloxolone. * In total, participants will have up to 17 clinic visits and 3 phone calls. Each participant will be in the study for up to 3 years.

Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

34 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

Australia
  • Sydney Children'S Hospital

    Randwick, New South Wales, Australia

    Not, yet, recruiting
  • Murdoch Childrens Research Institute (mcri)

    Parkville, Victoria, Australia

    Recruiting
Austria
  • Universitätsklinikum Innsbruck

    Innsbruck, Austria

    Sylvia M Boesch
    Recruiting
Brazil
  • L2 Ip - Instituto de Pesquisas Clinicas Ltda - Me

    Brasília, Federal District, Brazil

    Ingrid Faber Faber de Vasconcellos
    Recruiting
  • University of Campinas (unicamp) School of Medical Sciences

    Campinas, São Paulo, Brazil

    Marcondes Franca
    Not, yet, recruiting
  • Pseg Centro de Pesquisa Clinica

    São Paulo, São Paulo, Brazil

    Paulo Victor Sgobbi de Souza
    Recruiting
Canada
  • Mcgill University

    Montreal, Quebec, Canada

    Maryam Oskoui
    Recruiting
  • Chu de Quebec -Universite Laval

    Québec, Quebec, Canada

    Nicolas Chrestian
    Recruiting
Denmark
  • Rigshospitalet - Juliane Marie Centret (jmc) Copenhagen

    Copenhagen, Denmark

    Alfred Peter Born
    Not, yet, recruiting
France
  • Chu de Montpellier- Hôpital Gui de Chauliac

    Montpellier, Hérault, France

    Agathe Roubertie
    Recruiting
  • Ap-Hp - Hôpital Armand Trousseau

    Paris, France

    Florence Renaldo
    Recruiting
Germany
  • Universitätsklinikum Aachen

    Aachen, North Rhine-Westphalia, Germany

    Kathrin Reetz
    Recruiting
  • Ukgm - Universitätsklinikum Giessen Und Marburg Gmbh - Standort Gießen

    Giessen, Germany

    Andreas Hahn
    Recruiting
  • Universitätsklinikum Hamburg Eppendorf

    Hamburg, Germany

    Deike Weiss
    Recruiting
India
  • All India Institute of Medical Sciences (aiims) - New Delhi

    New Delhi, National Capital Territory of Delhi, India

    Withdrawn
Ireland
  • Chi at Temple Street

    Dublin, Ireland

    Declan O'Rourke
    Recruiting
Italy
  • Ospedale Pediatrico Bambino Gesù Irccs

    Rome, Lazio, Italy

    Gessica Vasco
    Not, yet, recruiting
  • Irccs Eugenio Medea - Polo. Scientifico Veneto

    Conegliano, Veneto, Italy

    Gabriella Paparella
    Not, yet, recruiting
  • Fondazione Irccs Istituto Neurologico Carlo Besta

    Milan, Italy

    Isabella Moroni
    Recruiting
Netherlands
  • Radboud Universitair Medisch Centrum

    Nijmegen, Netherlands

    Nienke van Os
    Recruiting
Saudi Arabia
  • King Faisal Specialist Hospital & Research Centre

    Riyadh, Ar Riya, Saudi Arabia

    Withdrawn
Spain
  • Hospital Sant Joan de Deu - Pin

    Espluges de Llobregat, Barcelona, Spain

    Alejandra Darling
    Recruiting
  • Hospital Universitario la Paz - Ppds

    Madrid, Spain

    Maria del Mar Garcia Romero
    Recruiting
Turkey (Türkiye)
  • Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi

    Istanbul, Turkey (Türkiye)

    Withdrawn
United Kingdom
  • University College Hospital - Ppds

    London, Lincolnshire, United Kingdom

    Shpresa Pula
    Recruiting
  • John Radcliffe Hospital

    Oxford, Oxfordshire, United Kingdom

    Andrea Németh
    Recruiting
  • Sheffield Children'S Hospital - Ppds

    Sheffield, South Yorkshire, United Kingdom

    Santosh Ravindra Mordekar
    Recruiting
United States
  • Ucla Neurology Outpatient Clinic at Westwood

    Los Angeles, California, United States

    Susan Perlman
    Not, yet, recruiting
  • Norman Fixel Institute for Neurological Diseases Uf Health

    Gainesville, Florida, United States

    Sankarsubramoney Subramony
    Recruiting
  • Usf Health Morsani College of Medicine Department of Neurology

    Tampa, Florida, United States

    Recruiting
  • Children'S Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - Pin

    Philadelphia, Pennsylvania, United States

    David Robinson Lynch
    Recruiting
  • St. Jude Children'S Research Hospital - Pin

    Memphis, Tennessee, United States

    Richard Finkel
    Recruiting
  • Chkd'S Health Center - South Campus - Pin

    Norfolk, Virginia, United States

    Crystal Proud
    Recruiting
  • Seattle Children'S Hospital

    Seattle, Washington, United States

    Alicia Henriquez
    Recruiting

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start
9 Jun 2025
Primary completion
16 Nov 2027
Completion
22 Nov 2029
Last update posted
16 Jun 2026
Registered enrollment
255
Registry id
NCT06953583

Cross-registrations

EU-CTR / CTIS (EudraCT)2025-520896-13

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.