Actively Recruiting

Phase 3
Age: 2Years - 15Years
All Genders
NCT06953583

A Study to Learn More About the Effects and Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old (BRAVE)

Led by Biogen · Updated on 2026-03-16

255

Participants Needed

34

Research Sites

232 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

In this study, researchers will learn more about the effects and safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedreich's Ataxia (FA) who are at least 16 years old. But, it is not yet available for children and teens with FA who are younger than 16 years old. The main objective of this study is to learn how BIIB141 works in the body and about its safety in children and teens who are 2 to 15 years old. The main questions researchers want to answer in this study are: * How does BIIB141 affect the participants' FA symptoms balance and stability? * How many participants have medical problems during the study? * Are there any changes in the participants' overall health during the study? * Are there any changes in the participants' heart health? * Are there any changes in how the participants move through puberty? Puberty is the time in someone's life when their body changes from a child to an adult. Researchers will also learn more about: \- How the body processes BIIB141 in children and teens This study will be done as follows: * Participants will be screened to check if they can join the study. The screening period will be up to 28 days, after which participants will check into their study research center. * There are 2 parts in this study. During Part 1, participants will take either BIIB141 or a placebo once a day. * In Part 1, participants will take BIIB141 or the placebo in a study research center on Day 1, and then at in-person visits at Week 4, Week 12, Week 26, and Week 52. On all other days, they will take BIIB141 or the placebo at home. Part 1 lasts up to 52 weeks. * During Part 2, participants from Part 1 will either continue taking BIIB141 or start it if they were taking the placebo. Part 2 will last up to 104 weeks. * In Part 1, participants will have up to 10 visits to their study research center and a phone call at Week 2. In Part 2, participants will have visits at Weeks 4, 8,12, 26, and every 26 weeks after that until they leave the study, and a phone call at Week 2. There will be a final phone call to check on the participants' health 31 days after their last dose. * Each participant will be in the study for up to about 3 years

CONDITIONS

Official Title

A Study to Learn More About the Effects and Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old (BRAVE)

Who Can Participate

Age: 2Years - 15Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosed with genetically confirmed Friedreich's Ataxia
  • Showing symptoms of Friedreich's Ataxia
  • Children aged 7 to under 16 years must have an upright stability score of 10 to 34 at baseline
  • Participants have completed Part 1 RCT without meeting discontinuation criteria to join Part 2
  • Safety and tolerability data from Part 1 support continuation in investigator's judgment
Not Eligible

You will not qualify if you...

  • Glycosylated hemoglobin A1C (HbA1c) greater than 11%
  • B-type natriuretic peptide (BNP) over 200 pg/mL at screening
  • Ejection fraction less than 40% based on echocardiogram at screening
  • Clinically significant cardiac disease except mild to moderate cardiomyopathy
  • For Part 2, delays in starting if BNP is above 200 pg/mL or other lab abnormalities are unresolved
  • Any intercurrent illness or health changes requiring repeat screening before Part 2
  • Dosing interruptions at end of Part 1 must be resolved before Part 2 start

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 34 locations

1

UCLA Neurology Outpatient Clinic at Westwood

Los Angeles, California, United States, 90095

Not Yet Recruiting

2

Norman Fixel Institute for Neurological Diseases UF Health

Gainesville, Florida, United States, 32610-3010

Actively Recruiting

3

USF Health Morsani College of Medicine Department of Neurology

Tampa, Florida, United States, 33612

Actively Recruiting

4

Children's Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - PIN

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

5

St. Jude Children's Research Hospital - PIN

Memphis, Tennessee, United States, 38105-3678

Actively Recruiting

6

CHKD's Health Center - South Campus - PIN

Norfolk, Virginia, United States, 23507-1910

Actively Recruiting

7

Seattle Children's Hospital

Seattle, Washington, United States, 98105-3901

Actively Recruiting

8

Sydney Children's Hospital

Randwick, New South Wales, Australia, 2031

Not Yet Recruiting

9

Murdoch Childrens Research Institute (MCRI)

Parkville, Victoria, Australia, 3052

Actively Recruiting

10

Universitätsklinikum Innsbruck

Innsbruck, Austria, 6020

Actively Recruiting

11

L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME

Brasília, Federal District, Brazil, 70200-730

Actively Recruiting

12

University of Campinas (UNICAMP) School of Medical Sciences

Campinas, São Paulo, Brazil, 13083-970

Not Yet Recruiting

13

PSEG Centro de Pesquisa Clinica

São Paulo, São Paulo, Brazil, 04024-002

Actively Recruiting

14

McGill University

Montreal, Quebec, Canada, H3H 2R9

Actively Recruiting

15

CHU de Quebec -Universite Laval

Québec, Quebec, Canada, G1V 4G2

Actively Recruiting

16

Rigshospitalet - Juliane Marie Centret (JMC) Copenhagen

Copenhagen, Denmark, 2100

Not Yet Recruiting

17

CHU de Montpellier- Hôpital Gui De Chauliac

Montpellier, Hérault, France, 34090

Actively Recruiting

18

AP-HP - Hôpital Armand Trousseau

Paris, France, 75012

Actively Recruiting

19

Universitätsklinikum Aachen

Aachen, North Rhine-Westphalia, Germany, 52074

Actively Recruiting

20

UKGM - Universitätsklinikum Giessen und Marburg GmbH - Standort Gießen

Giessen, Germany, 35392

Actively Recruiting

21

Universitätsklinikum Hamburg Eppendorf

Hamburg, Germany, 20246

Actively Recruiting

22

All India Institute of Medical Sciences (AIIMS) - New Delhi

New Delhi, National Capital Territory of Delhi, India, 110029

Not Yet Recruiting

23

CHI at Temple Street

Dublin, Ireland, D01 XD99

Actively Recruiting

24

Ospedale Pediatrico Bambino Gesù IRCCS

Rome, Lazio, Italy, 165

Not Yet Recruiting

25

IRCCS Eugenio Medea - Polo. Scientifico Veneto

Conegliano, Veneto, Italy, 31015

Not Yet Recruiting

26

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, Italy, 20133

Actively Recruiting

27

Radboud Universitair Medisch Centrum

Nijmegen, Netherlands, 6525 GA

Not Yet Recruiting

28

King Faisal Specialist Hospital & Research Centre

Riyadh, Ar Riya, Saudi Arabia, 12875

Not Yet Recruiting

29

Hospital Sant Joan de Deu - PIN

Espluges de Llobregat, Barcelona, Spain, 8950

Actively Recruiting

30

Hospital Universitario La Paz - PPDS

Madrid, Spain, 28046

Actively Recruiting

31

Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi

Istanbul, Turkey (Türkiye), 34093

Not Yet Recruiting

32

University College Hospital - PPDS

London, Lincolnshire, United Kingdom, NW1 2BU

Actively Recruiting

33

John Radcliffe Hospital

Oxford, Oxfordshire, United Kingdom, OX3 9DU

Actively Recruiting

34

Sheffield Children's Hospital - PPDS

Sheffield, South Yorkshire, United Kingdom, S10 5DD

Actively Recruiting

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Research Team

P

Patient Navigator

CONTACT

U

US Biogen Clinical Trial Center

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

QUADRUPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

3

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