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
NCT ID: NCT06953583
Last Updated: 2025-11-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
255 participants
INTERVENTIONAL
2025-06-09
2029-11-22
Brief Summary
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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
Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Part 1 RCT: Omaveloxolone
Participants will receive a single oral dose of omaveloxolone once a day (QD) for up to 52 weeks in Part 1 of the study.
Omaveloxolone
Administered as specified in the treatment arm.
Part 1 RCT: Placebo
Participants will receive placebo, orally, QD for up to 52 weeks in Part 1 of the study.
Placebo
Administered as specified in the treatment arm.
Part 2 OLE: Omaveloxolone
Participants who complete Part 1 of the study and are eligible will receive a single oral dose of omaveloxolone, QD for up to 104 weeks in the Part 2 OLE study.
Omaveloxolone
Administered as specified in the treatment arm.
Interventions
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Omaveloxolone
Administered as specified in the treatment arm.
Placebo
Administered as specified in the treatment arm.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 10 to ≤ 34 at baseline
Exclusion Criteria
* 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 2 OLE: Eligibility criteria:
* Participants have completed Part 1 RCT of the study and no discontinuation criteria have been met
* Safety and tolerability data from Part 1 RCT are supportive of continuation in the judgement of the investigator
1. If alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or total bilirubin (TBL) are \> 2× upper limit of normal (ULN) at the previous visit 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 be delayed until BNP is \< 200 pg/mL
3. If any other clinically significant laboratory abnormalities are present based on the previous visit assessments, Part 2 Day 1 should be delayed until the abnormalities are resolved
4. In the event of intercurrent illness or other change in health status of the participant, additional Part 1 screening assessments may be repeated prior to initiation of Part 2, based on the judgement of the investigator in consultation with the medical monitor
2 Years
15 Years
ALL
No
Sponsors
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Biogen
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Biogen
Locations
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UCLA Neurology Outpatient Clinic at Westwood
Los Angeles, California, United States
Norman Fixel Institute for Neurological Diseases UF Health
Gainesville, Florida, United States
USF Health Morsani College of Medicine Department of Neurology
Tampa, Florida, United States
Children's Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - PIN
Philadelphia, Pennsylvania, United States
St Jude Children's Research Hospital
Memphis, Tennessee, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States
Murdoch Childrens Research Institute (MCRI)
Parkville, Victoria, Australia
Universitätsklinikum Innsbruck
Innsbruck, , Austria
L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME
Brasília, Federal District, Brazil
University of Campinas (UNICAMP) School of Medical Sciences
Campinas, São Paulo, Brazil
PSEG Centro de Pesquisa Clinica
São Paulo, , Brazil
McGill University
Montreal, Quebec, Canada
CHU de Quebec -Universite Laval
Québec, , Canada
Rigshospitalet - Juliane Marie Centret (JMC) Copenhagen
Copenhagen, , Denmark
CHU de Montpellier - Hôpital Arnaud de Villeneuve
Montpellier, Hérault, France
AP-HP - Hôpital Armand Trousseau
Paris, , France
Universitätsklinikum Aachen
Aachen, , Germany
UKGM - Universitätsklinikum Giessen und Marburg GmbH - Standort Gießen
Giessen, , Germany
UKE Hamburg
Hamburg, , Germany
CHI at Temple Street
Dublin, , Ireland
Ospedale Pediatrico Bambino Gesù IRCCS
Rome, Lazio, Italy
IRCCS Eugenio Medea - Polo. Scientifico Veneto
Conegliano, Veneto, Italy
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, , Italy
Radboud Universitair Medisch Centrum
Nijmegen, , Netherlands
Hospital Sant Joan de Deu
Espluges de Llobregat, , Spain
Hospital Universitario La Paz - PPDS
Madrid, , Spain
Sheffield Children's Hospital
Sheffield, South Yorkshire, United Kingdom
University College London, Institue of Neurology
London, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Related Links
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Related Info
Other Identifiers
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2025-520896-13
Identifier Type: OTHER
Identifier Source: secondary_id
296FA301
Identifier Type: -
Identifier Source: org_study_id