Actively Recruiting
A Phase 3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich's Ataxia Aged 2 to <16 Years
Led by Biogen · Updated on 2026-06-03
255
Participants Needed
34
Research Sites
105 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying omaveloxolone (also known as BIIB141 or SKYCLARYS®) to learn about its effects and safety in children and teens aged 2 to 15 years who have Friedreich's Ataxia (FA). While omaveloxolone is already approved for people 16 and older, this study focuses on younger participants. The study aims to understand how the medicine affects FA symptoms, the number of adverse events, and any changes in overall and heart health. Researchers will use the modified Friedreich's Ataxia Rating Scale (mFARS) and questionnaires to assess nerve function, quality of life, muscle strength, daily task ability, and puberty changes. They will also study how the body processes the drug in this age group. The study has two parts: Part 1 lasts about one year and is randomized and double-blind, where participants take either omaveloxolone or a placebo once daily by mouth. Participants will have up to nine clinic visits and one phone call during this time. Those who finish Part 1 can join Part 2, where everyone receives omaveloxolone openly for about two years with up to eight clinic visits and one phone call, plus a follow-up call after stopping the drug. Overall, participants may be in the study for up to three years, with up to 17 clinic visits and three phone calls. Participants will be assessed through clinic visits and phone calls that include physical exams, safety checks, heart function tests by echocardiogram, growth measurements, and questionnaires about symptoms and quality of life. Researchers will monitor nerve function using the upright stability score (USS) from mFARS and track adverse events throughout the study. Blood samples will be taken to measure omaveloxolone levels in the body. Safety and long-term effects will be evaluated during both study parts and follow-up periods.
CONDITIONS
Brief Title
A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with genetically confirmed Friedreich's Ataxia (FA), including specific genetic mutations
- Showing symptoms of Friedreich's Ataxia confirmed by a clinician
- Children aged 7 to under 16 years must have an upright stability score (USS) between 10 and 34 at baseline
- Completed Part 1 of the study without meeting discontinuation criteria (for Parts 2A and 2B)
- Investigator supports continuation based on safety and tolerability data (for Parts 2A and 2B)
You will not qualify if you...
- Glycosylated hemoglobin A1C (HbA1c) greater than 11%
- B-type natriuretic peptide (BNP) greater than 200 pg/mL at screening
- Ejection fraction less than 40% based on echocardiogram at screening
- Clinically significant cardiac disease except mild to moderate cardiomyopathy
- Did not complete Part 1 or met discontinuation criteria (for Parts 2A and 2B)
- Safety and tolerability concerns preventing continuation (for Parts 2A and 2B)
- Other protocol-defined exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 52 weeks
Participants receive daily oral doses of omaveloxolone or placebo for up to 52 weeks to evaluate efficacy and safety.
Regular visits scheduled throughout treatment period
Duration - Up to 104 weeks
Participants who complete Part 1 may continue receiving daily oral doses of omaveloxolone for up to 104 weeks to further evaluate efficacy and safety.
Regular visits scheduled throughout treatment period
Duration - Up to 104 weeks
Participants are monitored for long-term safety and treatment effects after completion of active treatment.
Visits at Weeks 52 and 104 after baseline of Part 1
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
Actively 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
Patient Navigator
U
US Biogen Clinical Trial Center
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
4
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