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

NCT06953583 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: 296FA3012025-520896-13
• Study Type: interventional
• Target: 255
• Locations: 15 countries
• Sites: 32

Brief Summary

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.

Conditions

Friedreich Ataxia

Outcome Measures

Primary Outcomes (10)

• Part 1: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52

  The mFARS is a validated and sensitive rating scale that was developed to quantitatively assess the severity of the neurologic features of FA in adults and adolescents. Scores on the mFARS range from 0 to 93, with lower scores indicating better neurological function. The subscales of the mFARS assessment and maximum score for each subscale are: bulbar function (Subscale A; 2 assessments of speech and cough; maximum score = 5), upper limb coordination (Subscale B; 5 assessments of coordination of movement and function in arms and hands with each limb scored individually; maximum score = 36), lower limb coordination (Subscale C; 2 assessments of coordination of movement and function of lower limbs with each limb scored individually; maximum score = 16), and upright stability (USS, Subscale E; 9 assessments of sitting posture, stance, tandem walk, and gait assessments; maximum score = 36).

  Baseline, Week 52

• Part 2A: Change From Baseline in USS Subscale E of mFARS at Week 52

  The mFARS is a validated and sensitive rating scale that was developed to quantitatively assess the severity of the neurologic features of FA in adults and adolescents. Scores on the mFARS range from 0 to 93, with lower scores indicating better neurological function. The subscales of the mFARS assessment and maximum score for each subscale are: bulbar function (Subscale A; 2 assessments of speech and cough; maximum score = 5), upper limb coordination (Subscale B; 5 assessments of coordination of movement and function in arms and hands with each limb scored individually; maximum score = 36), lower limb coordination (Subscale C; 2 assessments of coordination of movement and function of lower limbs with each limb scored individually; maximum score = 16), and upright stability (USS, Subscale E; 9 assessments of sitting posture, stance, tandem walk, and gait assessments; maximum score = 36).

  Baseline (Week 52 of Part 1), Week 52

• Part 2B: Number of Participants With Treatment-Emergent Adverse Event (TEAE) and Treatment-Emergent Serious Adverse Event (TESAE)

  From the first dose of the study drug in Part 2B up to the end of follow-up period in Part 2B (up to Week 104)

• Part 2B: Number of Participants With Change From Baseline in Cardiac Function Assessed by Echocardiogram (ECHO) at Weeks 52 and Week 104

  Baseline (Week 52 of Part 1), Weeks 52 and 104

• Part 2B: Change From Baseline in Height at Weeks 52 and Week 104

  Baseline (Week 52 of Part 1), Weeks 52 and 104

• Part 2B: Change From Baseline in Weight at Weeks 52 and Week 104

  Baseline (Week 52 of Part 1), Weeks 52 and 104

• Part 2B: Change From Baseline in Body Mass Index (BMI) at Weeks 52 and Week 104

  Baseline (Week 52 of Part 1), Weeks 52 and 104

• Part 2B: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS) at Weeks 52 and Week 104

  The C-SSRS is a low-burden measure of the spectrum of suicidal ideation and behavior that was developed to assess severity and track suicidal events through any treatment of individuals ≥ 6 years of age. The C-SSRS is a clinical interview providing a summary of both ideation and behavior that can be administered by the clinician during any evaluation or risk assessment to identify the level and type of suicidality present. The assessment includes "yes" or "no" responses for 5 questions each, related to suicidal ideation (wish to be dead, non-specific active suicidal thoughts, active suicidal ideation with any methods, active suicidal ideation with some intent, active suicidal ideation with specific plan) and suicidal behavior (preparatory acts or behavior, aborted attempt, interrupted attempt, actual attempt, suicide). Numeric ratings are provided for severity of ideation, from 1 to 5, with 5 being the most severe.

  Baseline (Week 52 of Part 1), Weeks 52 and 104

• Part 2B: Percentage of Participants at Each Tanner Stage at Weeks 52 and Week 104

  Assessment of Tanner stages (a scale of physical development) will be performed by a medical doctor experienced with this assessment. Tanner score ranges from Stage 1 (childhood) to Stage 5 (full physical maturity). Information regarding Tanner staging will be collected at baseline for all participants and will be stopped once the participant reaches Tanner Stage 5 in all gender-appropriate scales.

  Baseline (Week 52 of Part 1), Weeks 52 and 104

• Part 2B: Number of Participants at Each Tanner Stage at Weeks 52 and Week 104

  Assessment of Tanner stages (a scale of physical development) will be performed by a medical doctor experienced with this assessment. Tanner score ranges from Stage 1 (childhood) to Stage 5 (full physical maturity). Information regarding Tanner staging will be collected at baseline for all participants and will be stopped once the participant reaches Tanner Stage 5 in all gender-appropriate scales.

  Baseline (Week 52 of Part 1), Weeks 52 and 104

Secondary Outcomes (30)

• Part 1: Change From Baseline in Friedreich's Ataxia-Health Index (FA-HI) at Week 52

  Baseline, Week 52

• Part 1: Change From Baseline in Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52

  Baseline, Week 52

• Part 1: Change From Baseline in Patient Global Impressions-Severity (PGI-S) at Week 52

  Baseline, Week 52

• Part 1: Change From Baseline in Clinical Global Impressions-Severity (CGI-S) at Week 52

  Baseline, Week 52

• Part 1: Change From Baseline in Friedreich's Ataxia-Activities of Daily Living (FA-ADL)

  Baseline, Week 52

Study Arms (4)

Part 1: Omaveloxolone

EXPERIMENTAL

Participants will receive a single oral dose of omaveloxolone once a day (QD) for up to 52 weeks in Part 1 of the study.

Drug: Omaveloxolone

Part 1: Placebo

PLACEBO COMPARATOR

Participants will receive placebo, orally, QD for up to 52 weeks in Part 1 of the study.

Drug: Placebo

Part 2A Continued Efficacy Evaluation: Omaveloxolone

EXPERIMENTAL

Participants will receive a single oral dose of omaveloxolone, QD for up to 104 weeks in Part 2A of the study.

Drug: Omaveloxolone

Part 2B Safety: Omaveloxolone

EXPERIMENTAL

Participants will receive a single oral dose of open-label omaveloxolone, QD for up to 104 weeks in Part 2B of the study.

Drug: Omaveloxolone

Interventions

Omaveloxolone DRUG

Administered as specified in the treatment arm.

Also known as: BIIB141, SKYCLARYS, RTA-408

Part 1: Omaveloxolone; Part 2A Continued Efficacy Evaluation: Omaveloxolone; Part 2B Safety: Omaveloxolone

Placebo DRUG

Administered as specified in the treatment arm.

Part 1: Placebo

Eligibility Criteria

• Age: 2 Years - 15 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Diagnosed with genetically confirmed Friedreich's Ataxia (FA), i.e., homozygous for guanine-adenine-adenine (GAA) repeat expansion in intron-1 of the frataxin gene, or GAA repeat expansion in 1 allele and with point mutations or deletions, or other non-GAA expansion mutations in the other allele.
• Symptomatic for FA as confirmed by clinician assessment. a. Children 7 to \< 16 years must also have an upright stability score (USS) score of 10 to ≤ 34 at baseline

You may not qualify if:

• Glycosylated hemoglobin A1C (HbA1c) \> 11%
• B-type natriuretic peptide (BNP) \> 200 picograms per milliliter (pg/mL) at screening
• Ejection fraction (EF) \< 40% \[based on echocardiogram (ECHO) performed at screening visit\]
• Clinically significant cardiac disease except mild to moderate cardiomyopathy
• Part 2A: Eligibility criteria:
• They have completed Part 1 of the study and no discontinuation criteria have been met.
• Safety and tolerability data from Part 1 are supportive of continuation in the judgement of the investigator.
• Part 2B: Eligibility criteria:
• Participants have completed Part 1 of the study and no discontinuation criteria have been met.
• Safety and tolerability data from Part 1 are supportive of continuation in the judgement of the Investigator.

Sponsors & Collaborators

• Biogen: lead

Study Sites (34)

UCLA Neurology Outpatient Clinic at Westwood

Los Angeles, California, 90095, United States

NOT YET RECRUITING

Norman Fixel Institute for Neurological Diseases UF Health

Gainesville, Florida, 32610-3010, United States

RECRUITING

USF Health Morsani College of Medicine Department of Neurology

Tampa, Florida, 33612, United States

RECRUITING

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

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

St. Jude Children's Research Hospital - PIN

Memphis, Tennessee, 38105-3678, United States

RECRUITING

CHKD's Health Center - South Campus - PIN

Norfolk, Virginia, 23507-1910, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105-3901, United States

RECRUITING

Sydney Children's Hospital

Randwick, New South Wales, 2031, Australia

NOT YET RECRUITING

Murdoch Childrens Research Institute (MCRI)

Parkville, Victoria, 3052, Australia

RECRUITING

Universitätsklinikum Innsbruck

Innsbruck, 6020, Austria

RECRUITING

L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME

Brasília, Federal District, 70200-730, Brazil

RECRUITING

University of Campinas (UNICAMP) School of Medical Sciences

Campinas, São Paulo, 13083-970, Brazil

NOT YET RECRUITING

PSEG Centro de Pesquisa Clinica

São Paulo, São Paulo, 04024-002, Brazil

RECRUITING

McGill University

Montreal, Quebec, H3H 2R9, Canada

RECRUITING

CHU de Quebec -Universite Laval

Québec, Quebec, G1V 4G2, Canada

RECRUITING

Rigshospitalet - Juliane Marie Centret (JMC) Copenhagen

Copenhagen, 2100, Denmark

NOT YET RECRUITING

CHU de Montpellier- Hôpital Gui De Chauliac

Montpellier, Hérault, 34090, France

RECRUITING

AP-HP - Hôpital Armand Trousseau

Paris, 75012, France

RECRUITING

Universitätsklinikum Aachen

Aachen, North Rhine-Westphalia, 52074, Germany

RECRUITING

UKGM - Universitätsklinikum Giessen und Marburg GmbH - Standort Gießen

Giessen, 35392, Germany

RECRUITING

Universitätsklinikum Hamburg Eppendorf

Hamburg, 20246, Germany

RECRUITING

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

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

WITHDRAWN

CHI at Temple Street

Dublin, D01 XD99, Ireland

RECRUITING

Ospedale Pediatrico Bambino Gesù IRCCS

Rome, Lazio, 165, Italy

NOT YET RECRUITING

IRCCS Eugenio Medea - Polo. Scientifico Veneto

Conegliano, Veneto, 31015, Italy

NOT YET RECRUITING

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, 20133, Italy

RECRUITING

Radboud Universitair Medisch Centrum

Nijmegen, 6525 GA, Netherlands

RECRUITING

King Faisal Specialist Hospital & Research Centre

Riyadh, Ar Riya, 12875, Saudi Arabia

WITHDRAWN

Hospital Sant Joan de Deu - PIN

Espluges de Llobregat, Barcelona, 8950, Spain

RECRUITING

Hospital Universitario La Paz - PPDS

Madrid, 28046, Spain

RECRUITING

Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi

Istanbul, 34093, Turkey (Türkiye)

WITHDRAWN

University College Hospital - PPDS

London, Lincolnshire, NW1 2BU, United Kingdom

RECRUITING

John Radcliffe Hospital

Oxford, Oxfordshire, OX3 9DU, United Kingdom

RECRUITING

Sheffield Children's Hospital - PPDS

Sheffield, South Yorkshire, S10 5DD, United Kingdom

RECRUITING

Related Links

• Related Info

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

omaveloxolone

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Medical Director

  Biogen

  STUDY DIRECTOR

Central Study Contacts

Patient Navigator

CONTACT

1-877-223-3576; biogenBRAVE_patientnavigator@thermofisher.com

US Biogen Clinical Trial Center

CONTACT

866-633-4636; clinicaltrials@biogen.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 11, 2025
• First Posted: May 1, 2025
• Study Start: June 9, 2025
• Primary Completion (Estimated): November 16, 2027
• Study Completion (Estimated): November 22, 2029
• Last Updated: June 16, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will share

Locations

IT (3); FR (2); ES (2); CA (2); TU (1); IN (1); NL (1); AU (1); DK (1); DE (3); BR (3); US (7); IE (1); GB (3); SA (1)