Testing Omaveloxolone against a placebo for friedreich ataxia
Official title: A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia
A Phase 3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich's Ataxia Aged 2 to < 16 Years
- Phase 3
- 4 groups
- Sites in Montreal and Québec
- Recruiting
Interventions
- Medication
Omaveloxolone
Administered as specified in the treatment arm.
- Medication
Placebo
Administered as specified in the treatment arm.
Canadian Sites (2)
2 of 2 recruiting
- Recruiting
McGill University
Montreal, Quebec
- Recruiting
CHU de Quebec -Universite Laval
Québec, Quebec
Eligibility Criteria
See who this study is looking for14 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +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 baselineBaselineYour starting measurements, taken before treatment begins.Read more →
- +Part 1: Key
Exclusion
- −Glycosylated hemoglobin A1C (HbA1c) \> 11%
- −B-type natriuretic peptide (BNP) \> 200 picograms per milliliter (pg/mL) at screeningScreeningThe checks done before joining, to see whether a study fits.Read more →
- −Ejection fraction (EF) \< 40% \[based on echocardiogram (ECHO) performed at screening visit\]
- −Clinically significant cardiac disease except mild to moderate cardiomyopathy
- −Part 2A: Eligibility criteriaEligibility criteriaThe full list of requirements for taking part in a study.Read more →:
- −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 investigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →.
- −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 protocolProtocolThe detailed plan a study must follow.Read more →-defined InclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more →/Exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → may apply.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 3Phase 3A large study comparing a treatment against the current standard.Read more → — a large study comparing this against the current standard, usually across many hospitals.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 4 groups in this study.
Groups A, C and D receive Omaveloxolone.
Registry label: A: Part 1: Omaveloxolone · C: Part 2A Continued Efficacy Evaluation: Omaveloxolone · D: Part 2B Safety: Omaveloxolone
Group B, the placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group, receives Placebo.
Registry label: B: Part 1: Placebo
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
One group receives a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → — a dummy treatment with no active medicine in it.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists an age range of 2 years to 15 years.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 255 people.
The study is currently expected to finish around November 2029.
The main measurement is taken over: BaselineBaselineYour starting measurements, taken before treatment begins.Read more →, Week 52.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Part 1: Change From BaselineBaselineYour starting measurements, taken before treatment begins.Read more → in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52 — measured over Baseline, Week 52.
Part 2A: Change From Baseline in USS Subscale E of mFARS at Week 52 — measured over Baseline (Week 52 of Part 1), Week 52.
Part 2B: Number of Participants With Treatment-Emergent Adverse EventAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more → (TEAE) and Treatment-Emergent Serious Adverse EventSerious adverse eventAn adverse event meeting a formal severity threshold, such as requiring hospital admission.Read more → (TESAE) — measured over From the first dose of the study drug in Part 2B up to the end of follow-up periodFollow-upContinued check-ins after the treatment part is finished.Read more → 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 — measured over Baseline (Week 52 of Part 1), Weeks 52 and 104.
The study lists 6 further main measurements.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT06953583 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About OmaveloxoloneDrug
Administered as specified in the treatment arm.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About PlaceboDrug
Administered as specified in the treatment arm.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 2 years to 15 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
Yes. This study includes a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group — a dummy treatment with no active medicine in it.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 2 years to 15 years.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: BaselineBaselineYour starting measurements, taken before treatment begins.Read more →, Week 52.
The study as a whole is currently expected to finish around 2029-11-22.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 255 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 3 locations, including: Montreal, Quebec, Canada; Québec, Quebec, Canada; Seattle, Washington, United States.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
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.
Other Sites (1)
Seattle Children's Hospital
Seattle, Washington, United States
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.