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

Study acronym: BRAVE
StatusActive, Not Recruiting
PhasePhase 3
SponsorBiogen
Started
View on ClinicalTrials.gov ↗

Record History

  1. Notable Primary endpoints: details revised at 1 of 10 entries Enrollment closed, study ongoing · Eligibility criteria shortened, about 19 words · Secondary endpoints: details revised at 17 of 30 entries · 1 more Show 4 more changesShow less
    • Enrollment closed, study ongoing
    • Eligibility criteria shortened, about 19 words
    • Secondary endpoints: details revised at 17 of 30 entries
    • Site details revised at 34 of 34 sites
  2. Minor Site details revised at 3 of 34 sitesv17
  3. Notable Primary endpoint(s) modified Trial arms changed: 3 → 4 · Site details revised at 1 of 34 sitesv16 Show 2 more changesShow less
    • Trial arms changed: 3 → 4
    • Site details revised at 1 of 34 sites

    See this version in full on ClinicalTrials.gov ↗

  4. Notable Primary endpoints: 9 to 10 Eligibility criteria shortened, about 91 words · Secondary endpoints: 19 to 30 · Study description expanded, about 53 words · 2 morev15 Show 5 more changesShow less
    • Eligibility criteria shortened, about 91 words
    • Secondary endpoints: 19 to 30
    • Study description expanded, about 53 words
    • Study arms: 3 to 4
    • Study title shortened, about 5 words

    See this version in full on ClinicalTrials.gov ↗

  5. Minor Study sites revised (34)v14 See this version in full on ClinicalTrials.gov ↗
Show 12 earlier versionsHide 12 earlier versions
  1. Minor Study sites revised (34)v13 See this version in full on ClinicalTrials.gov ↗
  2. Minor Study sites: 28 to 34v12
  3. Minor Study sites revised (28)v11 See this version in full on ClinicalTrials.gov ↗
  4. Minor Eligibility criteria shortened, about 19 words Study title expanded, about 1 wordv10 Show 1 more changeShow less
    • Study title expanded, about 1 word

    See this version in full on ClinicalTrials.gov ↗

  5. Minor Study sites revised (28)v9 See this version in full on ClinicalTrials.gov ↗
  6. Minor Study sites: 27 to 28v8
  7. Minor Study Status, Contacts/Locations, Referencesv7
  8. Minor Site details revised at 1 of 27 sitesv6
  9. Minor Re-verified, no change to tracked fieldsv5
  10. Minor Site contacts updated at 1 of 27 sitesv4
  11. Minor Start date changed from estimate of Jul 3, 2025 to Jun 9, 2025v3
  12. Notable Trial status: Not Yet Recruiting to Recruiting Site details revised at 27 of 27 sitesv2 Show 1 more changeShow less
    • Site details revised at 27 of 27 sites
  1. Minor Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
92 trials monitored
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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 does the body process omaveloxolone? * How many participants have adverse events during the study? Adverse events are health problems that may or may not be caused by the study drug. * Are there any changes in the participants' overall health or heart health? 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. More specifically, researchers will check participants' balance, coordination, movement, and ability to perform daily tasks using the Upright Stability Score (USS). The USS is part of the mFARS questionnaire. Researchers will also use other questionnaires to learn more about participants' symptoms, quality of life, ability to perform daily living activities, and how severe participants think their disease is. Researchers will also note any changes as participants go through puberty. Some participants may also be invited to take part in interviews about their experiences during the study or wear devices that measure movement and physical activity during daily life. 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 doctors, and study 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 finish Part 1 may move onto Part 2. During Part 2, participants who are at least 7 years old will be randomly assigned to receive either 150 mg or 250 mg of omaveloxolone once a day for about 104 weeks. Participants younger than 7 years old will receive 150 mg of omaveloxolone once a day for about 104 weeks. * 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.
Trial Details
NCT Number NCT06953583
Lead Sponsor Biogen
Conditions Friedreich Ataxia
Enrollment 255 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov