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Long-term effects and safety of omaveloxolone (SKYCLARYS) for children 2–15 with Friedreich's ataxia (BRAVE)

A Phase 3, 2-Part, Randomized, Double-Blind, Placebo-Controlled Study (Part 1) and Open-Label Extension (Part 2) to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich's Ataxia Aged 2 to < 16 Years

Phase 3 Interventional Biogen · NCT06953583

This trial will test whether omaveloxolone (SKYCLARYS) is safe and helps balance, heart health, and overall wellbeing in children and teens aged 2 to 15 with Friedreich's ataxia.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment255 (estimated)
Ages2 Years to 15 Years
SexAll
SponsorBiogen Industry-sponsored
Locations34 sites (Los Angeles, California and 33 other locations)
Trial IDNCT06953583 on ClinicalTrials.gov

What this trial studies

Part 1 is a randomized, placebo-controlled 52-week trial that compares omaveloxolone to placebo in children and adolescents with genetically confirmed Friedreich's ataxia, measuring effects on balance and stability and monitoring safety and drug concentrations. Key safety assessments include overall adverse events, laboratory tests, cardiac monitoring (including BNP and ejection fraction), and tracking pubertal development. Part 2 is an open-label extension that continues to follow participants for longer-term safety, tolerability, and sustained effects of omaveloxolone. The trial enrolls symptomatic participants meeting defined genetic and functional criteria and excludes those with significant cardiac dysfunction or very high HbA1c levels.

Who should consider this trial

Good fit: Children aged 2 to 15 with genetically confirmed Friedreich's ataxia who are symptomatic (and for ages 7–15 have an upright stability score of 10 to ≤34) are the intended participants.

Not a fit: Patients with significant cardiac disease (ejection fraction <40%), BNP >200 pg/mL, HbA1c >11%, or those outside the specified genetic or age criteria are unlikely to qualify or benefit from this trial.

Why it matters

Potential benefit: If successful, this could provide a safe, approved treatment option for children under 16 with Friedreich's ataxia and potentially improve balance and disease-related outcomes.

How similar studies have performed: Omaveloxolone has shown benefit in older adolescents and adults with Friedreich's ataxia leading to regulatory availability for people 16 and older, but pediatric efficacy and long-term safety data are limited.

Eligibility criteria

Show full inclusion / exclusion criteria 
Part 1 RCT: 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 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% \[based on echocardiogram (ECHO) performed at screening visit\]
* 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 BNP is \> 200 pg/mL at the previous visit assessment, Part 2 Day 1 should be delayed until BNP is \< 200 pg/mL.
  2. 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.
  3. 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.
  4. If dosing has been interrupted at the end of Part 1, Part 2 Day 1 should be delayed until resumption of study drug treatment is appropriate per Section 8.2.

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

Where this trial is running

Los Angeles, California and 33 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Friedreich Ataxia
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.