RecruitingPhase 3

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

United States, Australia, Austria255 participantsStarted 2025-06-09

Plain-language 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.

Who can participate

Age range

2 Years – 15 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Part 1: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52

Timeframe: Baseline, Week 52

Part 2A: Change From Baseline in USS Subscale E of mFARS at Week 52

Timeframe: 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)

Timeframe: 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

Timeframe: Baseline (Week 52 of Part 1), Weeks 52 and 104

Part 2B: Change From Baseline in Height at Weeks 52 and Week 104

Timeframe: Baseline (Week 52 of Part 1), Weeks 52 and 104

Part 2B: Change From Baseline in Weight at Weeks 52 and Week 104

Timeframe: Baseline (Week 52 of Part 1), Weeks 52 and 104

Trial details

NCT IDNCT06953583

SponsorBiogen

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-11-16

Contact for this trial

Patient Navigator

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

View on ClinicalTrials.gov More Friedreich Ataxia trials

Plain-language summary

Who can participate

Age range

2 Years – 15 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Part 1: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52

Timeframe: Baseline, Week 52

Part 2A: Change From Baseline in USS Subscale E of mFARS at Week 52

Timeframe: 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)

Timeframe: 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

Timeframe: Baseline (Week 52 of Part 1), Weeks 52 and 104

Part 2B: Change From Baseline in Height at Weeks 52 and Week 104

Timeframe: Baseline (Week 52 of Part 1), Weeks 52 and 104

Part 2B: Change From Baseline in Weight at Weeks 52 and Week 104

Timeframe: Baseline (Week 52 of Part 1), Weeks 52 and 104