Actively Recruiting
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
Eligibility Criteria
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
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
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
Research Team
P
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CONTACT
U
US Biogen Clinical Trial Center
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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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