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) (BRAVE)

March 13, 2026 updated by: Biogen

A Phase 3, 2-Part, Randomized, Double-Blind, Placebo-Controlled Study (Part 1) and Open-Label Extension (Part 2) to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich's Ataxia Aged 2 to < 16 Years

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

The primary objective of Part 1 randomized controlled trial (RCT) is to evaluate the efficacy of omaveloxolone at Week 52 and the secondary objectives are to evaluate safety of omaveloxolone through Week 52 and the concentration of omaveloxolone after single and multiple dose administration. The primary objective of Part 2 open-label extension (OLE) trial is to evaluate the safety and tolerability of long-term omaveloxolone use and the secondary objective is to evaluate the efficacy of omaveloxolone following long-term use.

Study Type

Interventional

Enrollment (Estimated)

255

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Australia
    • New South Wales
      • Randwick, New South Wales, Australia, 2031
        • Not yet recruiting
        • Sydney Children's Hospital
    • Victoria
      • Parkville, Victoria, Australia, 3052
        • Recruiting
        • Murdoch Childrens Research Institute (MCRI)
  • Austria
      • Innsbruck, Austria, 6020
        • Recruiting
        • Universitätsklinikum Innsbruck
        • Contact:
        • Principal Investigator:
          • Sylvia M Boesch
  • Brazil
    • Federal District
      • Brasília, Federal District, Brazil, 70200-730
        • Recruiting
        • L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME
        • Contact:
        • Principal Investigator:
          • Ingrid Faber Faber de Vasconcellos
    • São Paulo
      • Campinas, São Paulo, Brazil, 13083-970
        • Not yet recruiting
        • University of Campinas (UNICAMP) School of Medical Sciences
        • Contact:
        • Principal Investigator:
          • Marcondes Franca
      • São Paulo, São Paulo, Brazil, 04024-002
        • Recruiting
        • PSEG Centro de Pesquisa Clínica
        • Principal Investigator:
          • Paulo Victor Sgobbi de Souza
        • Contact:
  • Canada
    • Quebec
      • Montreal, Quebec, Canada, H3H 2R9
        • Recruiting
        • McGill University
        • Contact:
        • Principal Investigator:
          • Maryam Oskoui
      • Québec, Quebec, Canada, G1V 4G2
  • Denmark
      • Copenhagen, Denmark, 2100
        • Not yet recruiting
        • Rigshospitalet - Juliane Marie Centret (JMC) Copenhagen
        • Contact:
        • Principal Investigator:
          • Alfred Peter Born
  • France
      • Paris, France, 75012
        • Recruiting
        • AP-HP - Hôpital Armand Trousseau
        • Principal Investigator:
          • Florence Renaldo
        • Contact:
    • Hérault
      • Montpellier, Hérault, France, 34090
        • Recruiting
        • CHU de Montpellier- Hôpital Gui De Chauliac
        • Contact:
        • Principal Investigator:
          • Agathe Roubertie
  • Germany
      • Giessen, Germany, 35392
        • Recruiting
        • UKGM - Universitätsklinikum Giessen und Marburg GmbH - Standort Gießen
        • Principal Investigator:
          • Andreas Hahn
        • Contact:
      • Hamburg, Germany, 20246
        • Recruiting
        • Universitätsklinikum Hamburg Eppendorf
        • Contact:
        • Principal Investigator:
          • Deike Weiss
    • North Rhine-Westphalia
      • Aachen, North Rhine-Westphalia, Germany, 52074
        • Recruiting
        • Universitätsklinikum Aachen
        • Principal Investigator:
          • Kathrin Reetz
        • Contact:
  • India
    • National Capital Territory of Delhi
      • New Delhi, National Capital Territory of Delhi, India, 110029
        • Not yet recruiting
        • All India Institute of Medical Sciences (AIIMS) - New Delhi
  • Ireland
      • Dublin, Ireland, D01 XD99
        • Recruiting
        • CHI at Temple Street
        • Contact:
        • Principal Investigator:
          • Declan O'Rourke
  • Italy
      • Milan, Italy, 20133
        • Recruiting
        • Fondazione IRCCS Istituto Neurologico Carlo Besta
        • Contact:
        • Principal Investigator:
          • Isabella Moroni
    • Lazio
      • Rome, Lazio, Italy, 165
        • Not yet recruiting
        • Ospedale Pediatrico Bambino Gesù IRCCS
        • Principal Investigator:
          • Gessica Vasco
        • Contact:
    • Veneto
      • Conegliano, Veneto, Italy, 31015
        • Not yet recruiting
        • IRCCS Eugenio Medea - Polo. Scientifico Veneto
        • Principal Investigator:
          • Gabriella Paparella
        • Contact:
  • Netherlands
      • Nijmegen, Netherlands, 6525 GA
        • Not yet recruiting
        • Radboud Universitair Medisch Centrum
        • Contact:
          • Phone Number: 31 243614415
        • Principal Investigator:
          • Nienke van Os
  • Saudi Arabia
    • Ar Riya
      • Riyadh, Ar Riya, Saudi Arabia, 12875
        • Not yet recruiting
        • King Faisal Specialist Hospital & Research Centre
        • Contact:
          • Phone Number: +966 11-4427773
        • Principal Investigator:
          • Amaal AlDakheel
  • Spain
      • Madrid, Spain, 28046
        • Recruiting
        • Hospital Universitario La Paz - PPDS
        • Contact:
        • Principal Investigator:
          • Maria del Mar Garcia Romero
    • Barcelona
      • Espluges de Llobregat, Barcelona, Spain, 8950
        • Recruiting
        • Hospital Sant Joan de Deu - PIN
        • Contact:
        • Principal Investigator:
          • Alejandra Darling
  • Turkey (Türkiye)
      • Istanbul, Turkey (Türkiye), 34093
        • Not yet recruiting
        • Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
        • Contact:
          • Phone Number: 902124142000
        • Principal Investigator:
          • Zuhal Yapici
  • United Kingdom
    • Lincolnshire
      • London, Lincolnshire, United Kingdom, NW1 2BU
        • Recruiting
        • University College Hospital - PPDS
        • Contact:
        • Principal Investigator:
          • Shpresa Pula
    • Oxfordshire
      • Oxford, Oxfordshire, United Kingdom, OX3 9DU
        • Recruiting
        • John Radcliffe Hospital
        • Principal Investigator:
          • Andrea Németh
        • Contact:
          • Phone Number: 44 1865 231556
    • South Yorkshire
      • Sheffield, South Yorkshire, United Kingdom, S10 5DD
        • Recruiting
        • Sheffield Children's Hospital - PPDS
        • Contact:
        • Principal Investigator:
          • Santosh Ravindra Mordekar
  • United States
    • California
      • Los Angeles, California, United States, 90095
        • Not yet recruiting
        • UCLA Neurology Outpatient Clinic at Westwood
        • Contact:
        • Principal Investigator:
          • Susan Perlman
    • Florida
      • Gainesville, Florida, United States, 32610-3010
        • Recruiting
        • Norman Fixel Institute for Neurological Diseases UF Health
        • Contact:
        • Principal Investigator:
          • Sankarsubramoney Subramony
      • Tampa, Florida, United States, 33612
        • Recruiting
        • USF Health Morsani College of Medicine Department of Neurology
        • Principal Investigator:
          • Theresa Zesiewicz
        • Contact:
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - PIN
        • Contact:
        • Principal Investigator:
          • David Robinson Lynch
    • Tennessee
      • Memphis, Tennessee, United States, 38105-3678
        • Recruiting
        • St. Jude Children's Research Hospital - PIN
        • Principal Investigator:
          • Richard Finkel
        • Contact:
    • Virginia
      • Norfolk, Virginia, United States, 23507-1910
        • Recruiting
        • CHKD's Health Center - South Campus - PIN
        • Contact:
        • Principal Investigator:
          • Crystal Proud
    • Washington
      • Seattle, Washington, United States, 98105-3901
        • Recruiting
        • Seattle Children's Hospital
        • Contact:
          • Phone Number: 206-987-2078
        • Principal Investigator:
          • Alicia Henriquez

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Part 1 RCT: Key inclusion criteria:

  • 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

Part 1 RCT: Key exclusion criteria:

  • 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 2 OLE: Eligibility criteria:

  • Participants have completed Part 1 RCT of the study and no discontinuation criteria have been met

  • Safety and tolerability data from Part 1 RCT are supportive of continuation in the judgement of the investigator

    1. If BNP is > 200 pg/mL at the previous visit assessment, Part 2 Day 1 should be delayed until BNP is < 200 pg/mL.
    2. If any other clinically significant laboratory abnormalities are present based on the previous visit assessments, Part 2 Day 1 should be delayed until the abnormalities are resolved.
    3. In the event of intercurrent illness or other change in health status of the participant, additional Part 1 screening assessments may be repeated prior to initiation of Part 2, based on the judgement of the investigator in consultation with the medical monitor.
    4. If dosing has been interrupted at the end of Part 1, Part 2 Day 1 should be delayed until resumption of study drug treatment is appropriate per Section 8.2.

Note: Other protocol-defined Inclusion/Exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Part 1 RCT: 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.
Drug: Omaveloxolone
Administered as specified in the treatment arm.
Other Names:
  • BIIB141, SKYCLARYS, RTA-408
Placebo Comparator: Part 1 RCT: Placebo
Participants will receive placebo, orally, QD for up to 52 weeks in Part 1 of the study.
Drug: Placebo
Administered as specified in the treatment arm.
Experimental: Part 2 OLE: Omaveloxolone
Participants who complete Part 1 of the study and are eligible will receive a single oral dose of omaveloxolone, QD for up to 104 weeks in the Part 2 OLE study.
Drug: Omaveloxolone
Administered as specified in the treatment arm.
Other Names:
  • BIIB141, SKYCLARYS, RTA-408

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Part 1 RCT: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS)
Time Frame: Baseline, 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 2 OLE: Number of Participants With Treatment-Emergent Adverse Event (TEAE) and Treatment-Emergent Serious Adverse Event (TESAE)
Time Frame: From the first dose of the study drug in Part 2 up to the end of follow-up period in Part 2 (up to Week 105)
From the first dose of the study drug in Part 2 up to the end of follow-up period in Part 2 (up to Week 105)
Part 2 OLE: Number of Participants With Change From Baseline in Cardiac Function Assessed by Echocardiogram (ECHO)
Time Frame: Baseline (Week 52) up to Week 104
Baseline (Week 52) up to Week 104
Part 2 OLE: Change From Baseline in Height
Time Frame: Baseline (Week 52) up to Week 104
Baseline (Week 52) up to Week 104
Part 2 OLE: Change From Baseline in Weight
Time Frame: Baseline (Week 52) up to Week 104
Baseline (Week 52) up to Week 104
Part 2 OLE: Change From Baseline in Body Mass Index (BMI)
Time Frame: Baseline (Week 52) up to Week 104
Baseline (Week 52) up to Week 104
Part 2 OLE: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS)
Time Frame: Baseline (Week 52) up to 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) up to Week 104
Part 2 OLE: Percentage of Participants at Each Tanner Stage
Time Frame: Baseline (Week 52) up to 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) up to Week 104
Part 2 OLE: Number of Participants at Each Tanner Stage
Time Frame: Baseline (Week 52) up to 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) up to Week 104

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Part 1 RCT: Change From Baseline in Friedreich's Ataxia-Health Index (FA-HI)
Time Frame: Baseline, Week 52
The FA-HI is a participant reported survey that assesses overall disease burden on a 100-point scale, with 0 representing no disease burden and 100 representing the maximum level of disease burden containing 113 symptoms questions representing 18 symptomatic subscales.
Baseline, Week 52
Part 1 RCT: Change From Baseline in Modified Friedreich's Ataxia Rating Scale (mFARS)
Time Frame: Baseline, 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 1 RCT: Change From Baseline in Patient Global Impressions-Severity (PGI-S)
Time Frame: Baseline, Week 52
PGI-S will be conducted for participants 7 to < 16 years of age. These are clinically meaningful outcome measures that are participant-relevant across all age groups and disease severities for this population. PGI -S is a 1-item questionnaire where the response is recorded on a 4-point scale scored as: 1-normal, 2-mild, 3-moderate, or 4-severe.
Baseline, Week 52
Part 1 RCT: Change From Baseline in Clinical Global Impressions-Severity (CGI-S)
Time Frame: Baseline, Week 52
The CGI-S will be conducted for all enrolled participants, 2 to < 16 years of age. The CGI-S rating evaluates the severity of individual symptoms and treatment response in participants with mental disorders. The CGI-S is a 7-point scale that requires the clinician to rate the severity of the participant's illness at the time of assessment. A rating of 1 is considered normal, or with the least severe symptoms, a rating of 7 is extremely ill, or the worst symptoms.
Baseline, Week 52
Part 1 RCT: Change From Baseline in Friedreich's Ataxia-Activities of Daily Living (FA-ADL)
Time Frame: Baseline, Week 52
Participants will answer the 9 questions of the FA-ADL survey in an interview style conducted by any site staff. The FA-ADL survey assesses 9 concepts: (1) speech; (2) swallowing; (3) cutting food and handling utensils; (4) dressing; (5) personal hygiene; (6) falling; (7) walking; (8) quality of sitting position; and (9) bladder function.
Baseline, Week 52
Part 1 RCT: Number of Participants With Treatment-Emergent Adverse Event (TEAE) and Treatment-Emergent Serious Adverse Event (TESAE)
Time Frame: From first dose of study drug up to end of follow up period in Part 1 RCT (up to Week 54)
From first dose of study drug up to end of follow up period in Part 1 RCT (up to Week 54)
Part 1 RCT: Number of Participants With Change From Baseline in Cardiac Function Assessed by Echocardiogram (ECHO)
Time Frame: Baseline, Weeks 26 and 52
Baseline, Weeks 26 and 52
Part 1 RCT: Change From Baseline in Height
Time Frame: Baseline up to Week 52
Baseline up to Week 52
Part 1 RCT: Change From Baseline in Weight
Time Frame: Baseline up to Week 52
Baseline up to Week 52
Part 1 RCT: Change From Baseline in Body Mass Index (BMI)
Time Frame: Baseline up to Week 52
Baseline up to Week 52
Part 1 RCT: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS)
Time Frame: Baseline up to week 52
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 up to week 52
Part 1 RCT: Percentage of Participants at Each Tanner Stage
Time Frame: Screening and Week 52
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.
Screening and Week 52
Part 1 RCT: Number of Participants at Each Tanner Stage
Time Frame: Screening and Week 52
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.
Screening and Week 52
Part 1 RCT: Plasma Concentrations of Omaveloxolone
Time Frame: Pre-dose and post-dose on Day 1, Weeks 4, 12 and 26
Pre-dose and post-dose on Day 1, Weeks 4, 12 and 26
Part 2 OLE: Change From Baseline in Modified Friedreich's Ataxia Rating Scale (mFARS), Including Upright Stability Score (USS)
Time Frame: Baseline (Week 52) up to Week 104
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) up to Week 104
Part 2 OLE: Change From Baseline in Friedreich's Ataxia-Activities of Daily Living (FA-ADL)
Time Frame: Baseline (Week 52) up to Week 104
Participants will answer the 9 questions of the FA-ADL survey in an interview style conducted by any site staff. The FA-ADL survey assesses 9 concepts: (1) speech; (2) swallowing; (3) cutting food and handling utensils; (4) dressing; (5) personal hygiene; (6) falling; (7) walking; (8) quality of sitting position; and (9) bladder function. Every participant will be instructed to answer the 9 questions. Total scores on FA-ADL will be used as a secondary endpoint to assess efficacy in participants 7 to < 16 years of age.
Baseline (Week 52) up to Week 104
Part 2 OLE: Change From Baseline in Friedreich's Ataxia-Health Index (FA-HI)
Time Frame: Baseline (Week 52) up to Week 104
The FA-HI is a participant reported survey that assesses overall disease burden on a 100-point scale, with 0 representing no disease burden and 100 representing the maximum level of disease burden containing 113 symptoms questions representing 18 symptomatic subscales.
Baseline (Week 52) up to Week 104
Part 2 OLE: Change From Baseline in Patient Global Impressions-Severity (PGI-S)
Time Frame: Baseline (Week 52) up to Week 104
PGI-S will be conducted for participants 7 to < 16 years of age. These are clinically meaningful outcome measures that are participant relevant across all age groups and disease severities for this population. PGI -S is a 1-item questionnaire where the response is recorded on a 4-point scale scored as: 1-normal, 2-mild, 3-moderate, or 4- severe.
Baseline (Week 52) up to Week 104
Part 2 OLE: Change From Baseline in Clinical Global Impressions-Severity (CGI-S)
Time Frame: Baseline (Week 52) up to Week 104
The CGI-S will be conducted for all enrolled participants, 2 to < 16 years of age. The CGI-S rating evaluates the severity of individual symptoms and treatment response in participants with mental disorders. The CGI-S is a 7-point scale that that requires the clinician to rate the severity of the participant's illness at the time of assessment. A rating of 1 is considered normal, or with the least severe symptoms, a rating of 7 is extremely ill, or the worst symptoms.
Baseline (Week 52) up to Week 104

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Biogen

Investigators

  • Study Director: Medical Director, Biogen

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 9, 2025

Primary Completion (Estimated)

November 16, 2027

Study Completion (Estimated)

November 22, 2029

Study Registration Dates

First Submitted

April 11, 2025

First Submitted That Met QC Criteria

April 29, 2025

First Posted (Actual)

May 1, 2025

Study Record Updates

Last Update Posted (Actual)

March 16, 2026

Last Update Submitted That Met QC Criteria

March 13, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 296FA301
  • 2025-520896-13 (Other Identifier: EU CT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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