Recruiting
Phase 3

BIIB141

Sponsor:

Biogen

Code:

NCT06953583

Conditions

Friedreich Ataxia

Eligibility Criteria

Sex: All

Age: 2 - 15

Healthy Volunteers: Not accepted

Interventions

Omaveloxolone

Placebo

Study Details

Brief summary:

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.

Conditions

Friedreich Ataxia

Study ID

NCT06953583

Start date

Jun 9, 2025

Status verified date

Jun, 2026

Completion date

Nov 22, 2029

Anticipated

Primary completion date

Nov 16, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 15

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

255 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Part 1: 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.

placebo comparator: Part 1: Placebo

Participants will receive placebo, orally, QD for up to 52 weeks in Part 1 of the study.

experimental: Part 2A Continued Efficacy Evaluation: Omaveloxolone

Participants will receive a single oral dose of omaveloxolone, QD for up to 104 weeks in Part 2A of the study.

experimental: Part 2B Safety: Omaveloxolone

Participants will receive a single oral dose of open-label omaveloxolone, QD for up to 104 weeks in Part 2B of the study.

Interventions

Omaveloxolone

Administered as specified in the treatment arm.

Placebo

Administered as specified in the treatment arm.

Primary outcome measure

  • Part 1: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52 [ Time Frame: Baseline, Week 52 ]
  • Part 2A: Change From Baseline in USS Subscale E of mFARS at Week 52 [ Time Frame: Baseline (Week 52 of Part 1), Week 52 ]
  • Part 2B: Number of Participants With Treatment-Emergent Adverse Event (TEAE) and Treatment-Emergent Serious Adverse Event (TESAE) [ Time Frame: From the first dose of the study drug in Part 2B up to the end of follow-up period in Part 2B (up to Week 104) ]
  • Part 2B: Number of Participants With Change From Baseline in Cardiac Function Assessed by Echocardiogram (ECHO) at Weeks 52 and Week 104 [ Time Frame: Baseline (Week 52 of Part 1), Weeks 52 and 104 ]
  • Part 2B: Change From Baseline in Height at Weeks 52 and Week 104 [ Time Frame: Baseline (Week 52 of Part 1), Weeks 52 and 104 ]
  • Part 2B: Change From Baseline in Weight at Weeks 52 and Week 104 [ Time Frame: Baseline (Week 52 of Part 1), Weeks 52 and 104 ]
  • Part 2B: Change From Baseline in Body Mass Index (BMI) at Weeks 52 and Week 104 [ Time Frame: Baseline (Week 52 of Part 1), Weeks 52 and 104 ]
  • Part 2B: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS) at Weeks 52 and Week 104 [ Time Frame: Baseline (Week 52 of Part 1), Weeks 52 and 104 ]
  • Part 2B: Percentage of Participants at Each Tanner Stage at Weeks 52 and Week 104 [ Time Frame: Baseline (Week 52 of Part 1), Weeks 52 and 104 ]
  • Part 2B: Number of Participants at Each Tanner Stage at Weeks 52 and Week 104 [ Time Frame: Baseline (Week 52 of Part 1), Weeks 52 and 104 ]

Central Contacts and Locations

Central contacts

Patient Navigator

1-877-223-3576biogenBRAVE_patientnavigator@thermofisher.com

US Biogen Clinical Trial Center

866-633-4636clinicaltrials@biogen.com

Locations

Norman Fixel Institute for Neurological Diseases UF Health

Recruiting

Gainesville, Florida, United States, 32610-3010

Contacts

352-733-3032s.subramony@neurology.ufl.edu

Principal Investigator:

Sankarsubramoney Subramony

USF Health Morsani College of Medicine Department of Neurology

Recruiting

Tampa, Florida, United States, 33612

Contacts

813-974-5909tzesiewi@hsc.usf.edu

Principal Investigator:

Theresa Zesiewicz

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

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

215-590-2242lynchd@pennmedicine.upenn.edu

Principal Investigator:

David Robinson Lynch

St. Jude Children's Research Hospital - PIN

Recruiting

Memphis, Tennessee, United States, 38105-3678

Contacts

407-650-7250richard.finkel@stjude.org

Principal Investigator:

Richard Finkel

CHKD's Health Center - South Campus - PIN

Recruiting

Norfolk, Virginia, United States, 23507-1910

Contacts

757-668-6981Proud.research@chkd.org

Principal Investigator:

Crystal Proud

Seattle Children's Hospital

Recruiting

Seattle, Washington, United States, 98105-3901

Contacts

206-987-2078

Principal Investigator:

Alicia Henriquez

McGill University

Recruiting

Montreal, Quebec, Canada, H3H 2R9

Contacts

514-412-4466maryam.oskoui@mcgill.ca

Principal Investigator:

Maryam Oskoui

CHU de Quebec -Universite Laval

Recruiting

Québec, Quebec, Canada, G1V 4G2

Contacts

418-525-4444nicolas.chrestian.med@ssss.gouv.qc.ca

Principal Investigator:

Nicolas Chrestian

More Information

Sponsor

Biogen

Last update posted

Jun 16, 2026

Last verified

Jun, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Biogen on 2026-06-16.