A Study to Learn More About the Effects and Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old

Last updated: June 18, 2025
Sponsor: Biogen
Overall Status: Active - Recruiting

Phase

3

Condition

Spinocerebellar Disorders

Dyskinesias

Friedreich's Ataxia

Treatment

Placebo

Omaveloxolone

Clinical Study ID

NCT06953583
296FA301
2025-520896-13
  • Ages 2-15
  • All Genders

Study Summary

In this study, researchers will learn more about the effects and safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedreich's Ataxia (FA) who are at least 16 years old. But, it is not yet available for children and teens with FA who are younger than 16 years old. The main objective of this study is to learn how BIIB141 works in the body and about its safety in children and teens who are 2 to 15 years old.

The main questions researchers want to answer in this study are:

  • How does BIIB141 affect the participants' FA symptoms balance and stability?

  • How many participants have medical problems during the study?

  • Are there any changes in the participants' overall health during the study?

  • Are there any changes in the participants' heart health?

  • Are there any changes in how the participants move through puberty? Puberty is the time in someone's life when their body changes from a child to an adult.

Researchers will also learn more about:

  • How the body processes BIIB141 in children and teens

This study will be done as follows:

  • Participants will be screened to check if they can join the study. The screening period will be up to 28 days, after which participants will check into their study research center.

  • There are 2 parts in this study. During Part 1, participants will take either BIIB141 or a placebo once a day.

  • In Part 1, participants will take BIIB141 or the placebo in a study research center on Day 1, and then at in-person visits at Week 4, Week 12, Week 26, and Week 52. On all other days, they will take BIIB141 or the placebo at home. Part 1 lasts up to 52 weeks.

  • During Part 2, participants from Part 1 will either continue taking BIIB141 or start it if they were taking the placebo. Part 2 will last up to 104 weeks.

  • In Part 1, participants will have up to 10 visits to their study research center and a phone call at Week 2. In Part 2, participants will have visits at Weeks 4, 8,12, 26, and every 26 weeks after that until they leave the study, and a phone call at Week 2. There will be a final phone call to check on the participants' health 31 days after their last dose.

  • Each participant will be in the study for up to about 3 years

Eligibility Criteria

Inclusion

Part 1 RCT: Key inclusion criteria:

  • Diagnosed with genetically confirmed Friedreich's Ataxia (FA), i.e., homozygous forguanine-adenine-adenine (GAA) repeat expansion in intron-1 of the frataxin gene, orGAA repeat expansion in 1 allele and with point mutations or deletions, or othernon-GAA expansion mutations in the other allele.

  • Symptomatic for FA as reported by the participant and/or the parent/caregiver a.Children 7 to < 16 years must also have an upright stability score (USS) score of 10to ≤ 34 at baseline

Exclusion

Part 1 RCT: 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 screeningvisit]

  • Clinically significant cardiac disease except mild to moderate cardiomyopathy

Part 2 OLE: Eligibility criteria:

  • Participants have completed Part 1 RCT of the study and no discontinuation criteriahave been met

  • Safety and tolerability data from Part 1 RCT are supportive of continuation in thejudgement of the investigator

  1. If alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/ortotal bilirubin (TBL) are > 2× upper limit of normal (ULN) at the previousvisit assessment, Part 2 Day 1 should be delayed until ALT and AST are < 1.5×ULN and TBL is < 2× ULN

  2. If BNP is > 200 pg/mL at the previous visit assessment, Part 2 Day 1 should bedelayed until BNP is < 200 pg/mL

  3. If any other clinically significant laboratory abnormalities are present basedon the previous visit assessments, Part 2 Day 1 should be delayed until theabnormalities are resolved

  4. In the event of intercurrent illness or other change in health status of theparticipant, additional Part 1 screening assessments may be repeated prior toinitiation of Part 2, based on the judgement of the investigator inconsultation with the medical monitor

Note: Other protocol-defined Inclusion/Exclusion criteria may apply.

Study Design

Total Participants: 255
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 3
Study Start date:
June 09, 2025
Estimated Completion Date:
November 22, 2029

Study Description

The primary objective of Part 1 randomized controlled trial (RCT) is to evaluate the efficacy of omaveloxolone at Week 52 and the secondary objectives are to evaluate safety of omaveloxolone through Week 52 and the concentration of omaveloxolone after single and multiple dose administration. The primary objective of Part 2 open-label extension (OLE) trial is to evaluate the safety and tolerability of long-term omaveloxolone use and the secondary objective is to evaluate the efficacy of omaveloxolone following long-term use.

Connect with a study center

  • Murdoch Childrens Research Institute (MCRI)

    Parkville, Victoria 3052
    Australia

    Site Not Available

  • Universitätsklinikum Innsbruck

    Innsbruck, 6020
    Austria

    Site Not Available

  • L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME

    Brasília, Distrito Federal 70200-730
    Brazil

    Site Not Available

  • University of Campinas (UNICAMP) School of Medical Sciences

    Campinas, Sao Paulo 13083-970
    Brazil

    Site Not Available

  • PSEG Centro de Pesquisa Clinica

    Sao Paulo, 04038-002
    Brazil

    Site Not Available

  • McGill University

    Montreal, Quebec H3H 2R9
    Canada

    Site Not Available

  • Rigshospitalet - Juliane Marie Centret (JMC) Copenhagen

    Copenhagen, 2100
    Denmark

    Site Not Available

  • CHU de Montpellier - Hôpital Arnaud de Villeneuve

    Montpellier, Hérault 34090
    France

    Site Not Available

  • AP-HP - Hôpital Armand Trousseau

    Paris, 75012
    France

    Site Not Available

  • Universitätsklinikum Aachen

    Aachen, 52074
    Germany

    Site Not Available

  • UKGM - Universitätsklinikum Giessen und Marburg GmbH - Standort Gießen

    Giessen, 35392
    Germany

    Site Not Available

  • UKE Hamburg

    Hamburg, 20251
    Germany

    Site Not Available

  • CHI at Temple Street

    Dublin, D01 XD99
    Ireland

    Site Not Available

  • Ospedale Pediatrico Bambino Gesù IRCCS

    Roma, Lazio 165
    Italy

    Site Not Available

  • IRCCS Eugenio Medea - Polo. Scientifico Veneto

    Conegliano, Veneto 31015
    Italy

    Site Not Available

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milano, 20133
    Italy

    Site Not Available

  • Radboud Universitair Medisch Centrum

    Nijmegen, 6525 GA
    Netherlands

    Site Not Available

  • Hospital Sant Joan de Deu

    Espluges De Llobregat, 8950
    Spain

    Site Not Available

  • Hospital Universitario La Paz - PPDS

    Madrid, 28046
    Spain

    Site Not Available

  • Sheffield Children's Hospital

    Sheffield, South Yorkshire S10 2TH
    United Kingdom

    Site Not Available

  • University College London, Institue of Neurology

    London, WC1N 3BG
    United Kingdom

    Site Not Available

  • UCLA Neurology Outpatient Clinic at Westwood

    Los Angeles, California 90095
    United States

    Site Not Available

  • Norman Fixel Institute for Neurological Diseases UF Health

    Gainesville, Florida 32608-3928
    United States

    Site Not Available

  • USF Health Morsani College of Medicine Department of Neurology

    Tampa, Florida 33612
    United States

    Site Not Available

  • Children's Hospital of Philadelphia Buerger Center

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • St Jude Children's Research Hospital

    Memphis, Tennessee 38105-3678
    United States

    Site Not Available

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia 23507-1910
    United States

    Active - Recruiting

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