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

Conditions

• Friedreich Ataxia

Eligibility Criteria

Sex

ALL

Age

2 Years - 15 Years

Healthy Volunteers

Not accepted

Interventions

• Omaveloxolone — DRUG
  Administered as specified in the treatment arm.
• Placebo — DRUG
  Administered as specified in the treatment arm.

Study Details

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.

Key Dates

Start date

Jun 9, 2025

Status verified

Jun 2026

Primary completion

Nov 16, 2027

Completion

Nov 22, 2029

Study Design

Enrollment

255 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

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.

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 ]

Central Contacts

• Patient Navigator
  1-877-223-3576
  [email protected]
• US Biogen Clinical Trial Center
  866-633-4636
  [email protected]

Locations (7)

Find similar trials in Los Angeles, CA

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