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En studie för att lära sig mer om effekterna och långsiktiga säkerheten i BIIB141 (Omaveloxolone) hos deltagare med Friedreichs ataxi i åldern 2 till 15 år gammal (BRAVE)

15 juni 2026 uppdaterad av: Biogen

En fas 3, 2-delad, randomiserad, dubbelblind, placebokontrollerad studie (del 1) och öppen etikettförlängning (del 2) för att utvärdera effektiviteten, säkerheten, farmakokinetiken och farmakodynamiken för omaveloxolon (BIIB141) hos deltagare med Friedreichs ataxi Aged 2 till <16 år

I denna studie kommer forskare att lära sig mer om effekterna och säkerheten i BIIB141, även känd som Omaveloxolone eller Skyclarys®. Detta läkemedel har godkänts eller gjorts tillgängligt för läkare att förskriva för personer med Friedreichs ataxi (FA) som är minst 16 år gamla. Men det är ännu inte tillgängligt för barn och tonåringar med FA som är yngre än 16 år gamla. Huvudmålet med denna studie är att lära sig hur BIIB141 fungerar i kroppen och om dess säkerhet hos barn och tonåringar som är 2 till 15 år gamla.

De viktigaste frågorna som forskare vill svara i denna studie är:

  • Hur påverkar BIIB141 deltagarnas FA -symtom balans och stabilitet?
  • Hur många deltagare har medicinska problem under studien?
  • Finns det några förändringar i deltagarnas allmänna hälsa under studien?
  • Finns det några förändringar i deltagarnas hjärthälsa?
  • Finns det några förändringar i hur deltagarna rör sig genom puberteten? Puberteten är tiden i någons liv när deras kropp förändras från ett barn till en vuxen.

Forskare kommer också att lära sig mer om:

- Hur kroppen bearbetar BIIB141 hos barn och tonåringar

Denna studie kommer att göras enligt följande:

  • Deltagarna kommer att screenas för att kontrollera om de kan gå med i studien. Screeningperioden kommer att vara upp till 28 dagar, varefter deltagarna kommer att kolla in sitt studieforskningscenter.
  • Det finns två delar i denna studie. Under del 1 kommer deltagarna att ta antingen BIIB141 eller en placebo en gång om dagen.
  • I del 1 kommer deltagarna att ta BIIB141 eller placebo i ett studieforskningscenter på dag 1 och sedan vid personbesök vid vecka 4, vecka 12, vecka 26 och vecka 52. På alla andra dagar kommer de att ta BIIB141 eller placebo hemma. Del 1 varar upp till 52 veckor.
  • Under del 2 kommer deltagarna från del 1 antingen att fortsätta ta BIIB141 eller starta det om de tog placebo. Del 2 kommer att pågå i upp till 104 veckor.
  • I del 1 kommer deltagarna att ha upp till tio besök i sitt studieforskningscenter och ett telefonsamtal vid vecka 2. I del 2 kommer deltagarna att ha besök vid veckor 4, 8,12, 26 och var 26 veckor efter det tills de lämnar studien och ett telefonsamtal vid vecka 2. Det kommer att finnas ett slutligt telefonsamtal för att kontrollera deltagarna 31 dagar efter deras sista dos.
  • Varje deltagare kommer att vara i studien i upp till cirka 3 år

Studieöversikt

Status

Rekrytering

Betingelser

Intervention / Behandling

Detaljerad beskrivning

Det primära målet med del 1 randomiserad kontrollerad studie (RCT) är att utvärdera effektiviteten av omaveloxolon vid vecka 52 och de sekundära målen är att utvärdera säkerheten för omaveloxolon genom vecka 52 och koncentrationen av omaveloxolon efter enstaka och multipel dosadministrering. Det primära målet med del 2 Open-Label Extension (OLE) -studie är att utvärdera säkerheten och tolerabiliteten för långvarig omaveloxolonanvändning och det sekundära målet är att utvärdera effektiviteten av omaveloxolon efter långvarig användning.

Studietyp

Interventionell

Inskrivning (Beräknad)

255

Fas

  • Fas 3

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

Studera Kontakt Backup

Studieorter

  • Australien
    • New South Wales
      • Randwick, New South Wales, Australien, 2031
        • Har inte rekryterat ännu
        • Sydney Children's Hospital
    • Victoria
      • Parkville, Victoria, Australien, 3052
        • Rekrytering
        • Murdoch Childrens Research Institute (MCRI)
  • Brasilien
    • Federal District
      • Brasília, Federal District, Brasilien, 70200-730
        • Rekrytering
        • L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME
        • Kontakt:
        • Huvudutredare:
          • Ingrid Faber Faber de Vasconcellos
    • São Paulo
      • Campinas, São Paulo, Brasilien, 13083-970
        • Har inte rekryterat ännu
        • University of Campinas (UNICAMP) School of Medical Sciences
        • Kontakt:
        • Huvudutredare:
          • Marcondes França
      • São Paulo, São Paulo, Brasilien, 04024-002
        • Rekrytering
        • PSEG Centro de Pesquisa Clinica
        • Huvudutredare:
          • Paulo Victor Sgobbi de Souza
        • Kontakt:
  • Danmark
      • Copenhagen, Danmark, 2100
        • Har inte rekryterat ännu
        • Rigshospitalet - Juliane Marie Centret (JMC) Copenhagen
        • Kontakt:
        • Huvudutredare:
          • Alfred Peter Born
  • Frankrike
      • Paris, Frankrike, 75012
        • Rekrytering
        • AP-HP - Hôpital Armand Trousseau
        • Huvudutredare:
          • Florence Renaldo
        • Kontakt:
    • Hérault
      • Montpellier, Hérault, Frankrike, 34090
        • Rekrytering
        • CHU de Montpellier- Hôpital Gui De Chauliac
        • Kontakt:
        • Huvudutredare:
          • Agathe Roubertie
  • Förenta staterna
    • California
      • Los Angeles, California, Förenta staterna, 90095
        • Har inte rekryterat ännu
        • UCLA Neurology Outpatient Clinic at Westwood
        • Kontakt:
        • Huvudutredare:
          • Susan Perlman
    • Florida
      • Gainesville, Florida, Förenta staterna, 32610-3010
        • Rekrytering
        • Norman Fixel Institute for Neurological Diseases UF Health
        • Kontakt:
        • Huvudutredare:
          • Sankarsubramoney Subramony
      • Tampa, Florida, Förenta staterna, 33612
        • Rekrytering
        • USF Health Morsani College of Medicine Department of Neurology
        • Huvudutredare:
          • Theresa Zesiewicz
        • Kontakt:
    • Pennsylvania
      • Philadelphia, Pennsylvania, Förenta staterna, 19104
        • Rekrytering
        • Children's Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - PIN
        • Kontakt:
        • Huvudutredare:
          • David Robinson Lynch
    • Tennessee
      • Memphis, Tennessee, Förenta staterna, 38105-3678
        • Rekrytering
        • St. Jude Children's Research Hospital - PIN
        • Huvudutredare:
          • Richard Finkel
        • Kontakt:
    • Virginia
      • Norfolk, Virginia, Förenta staterna, 23507-1910
        • Rekrytering
        • CHKD's Health Center - South Campus - PIN
        • Kontakt:
        • Huvudutredare:
          • Crystal Proud
    • Washington
      • Seattle, Washington, Förenta staterna, 98105-3901
        • Rekrytering
        • Seattle Children's Hospital
        • Kontakt:
          • Telefonnummer: 206-987-2078
        • Huvudutredare:
          • Alicia Henriquez
  • Indien
    • National Capital Territory of Delhi
      • New Delhi, National Capital Territory of Delhi, Indien, 110029
        • Indragen
        • All India Institute of Medical Sciences (AIIMS) - New Delhi
  • Irland
      • Dublin, Irland, D01 XD99
        • Rekrytering
        • CHI at Temple Street
        • Kontakt:
        • Huvudutredare:
          • Declan O'Rourke
  • Italien
      • Milan, Italien, 20133
        • Rekrytering
        • Fondazione IRCCS Istituto Neurologico Carlo Besta
        • Kontakt:
        • Huvudutredare:
          • Isabella Moroni
    • Lazio
      • Rome, Lazio, Italien, 165
        • Har inte rekryterat ännu
        • Ospedale Pediatrico Bambino Gesu Irccs
        • Huvudutredare:
          • Gessica Vasco
        • Kontakt:
    • Veneto
      • Conegliano, Veneto, Italien, 31015
        • Har inte rekryterat ännu
        • IRCCS Eugenio Medea - Polo. Scientifico Veneto
        • Huvudutredare:
          • Gabriella Paparella
        • Kontakt:
  • Kanada
    • Quebec
      • Montreal, Quebec, Kanada, H3H 2R9
        • Rekrytering
        • McGill University
        • Kontakt:
        • Huvudutredare:
          • Maryam Oskoui
      • Québec, Quebec, Kanada, G1V 4G2
  • Nederländerna
      • Nijmegen, Nederländerna, 6525 GA
        • Rekrytering
        • Radboud Universitair Medisch Centrum
        • Kontakt:
          • Telefonnummer: 31 243614415
        • Huvudutredare:
          • Nienke van Os
  • Saudiarabien
    • Ar Riya
      • Riyadh, Ar Riya, Saudiarabien, 12875
        • Indragen
        • King Faisal Specialist Hospital & Research Centre
  • Spanien
      • Madrid, Spanien, 28046
        • Rekrytering
        • Hospital Universitario La Paz - PPDS
        • Kontakt:
        • Huvudutredare:
          • Maria del Mar Garcia Romero
    • Barcelona
      • Espluges de Llobregat, Barcelona, Spanien, 8950
        • Rekrytering
        • Hospital Sant Joan de Deu - PIN
        • Kontakt:
        • Huvudutredare:
          • Alejandra Darling
  • Storbritannien
    • Lincolnshire
      • London, Lincolnshire, Storbritannien, NW1 2BU
        • Rekrytering
        • University College Hospital - PPDS
        • Kontakt:
        • Huvudutredare:
          • Shpresa Pula
    • Oxfordshire
      • Oxford, Oxfordshire, Storbritannien, OX3 9DU
        • Rekrytering
        • John Radcliffe Hospital
        • Huvudutredare:
          • Andrea Németh
        • Kontakt:
          • Telefonnummer: 44 1865 231556
    • South Yorkshire
      • Sheffield, South Yorkshire, Storbritannien, S10 5DD
        • Rekrytering
        • Sheffield Children's Hospital - PPDS
        • Kontakt:
        • Huvudutredare:
          • Santosh Ravindra Mordekar
  • Turkiet (Türkiye)
      • Istanbul, Turkiet (Türkiye), 34093
        • Indragen
        • Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
  • Tyskland
      • Giessen, Tyskland, 35392
        • Rekrytering
        • UKGM - Universitätsklinikum Giessen und Marburg GmbH - Standort Gießen
        • Huvudutredare:
          • Andreas Hahn
        • Kontakt:
      • Hamburg, Tyskland, 20246
        • Rekrytering
        • Universitatsklinikum Hamburg Eppendorf
        • Kontakt:
        • Huvudutredare:
          • Deike Weiss
    • North Rhine-Westphalia
      • Aachen, North Rhine-Westphalia, Tyskland, 52074
        • Rekrytering
        • Universitätsklinikum Aachen
        • Huvudutredare:
          • Kathrin Reetz
        • Kontakt:
  • Österrike
      • Innsbruck, Österrike, 6020
        • Rekrytering
        • Universitätsklinikum Innsbruck
        • Kontakt:
        • Huvudutredare:
          • Sylvia M Boesch

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

  • Barn

Tar emot friska volontärer

Nej

Beskrivning

DEL 1 RCT: Key Inclusion Criteria:

  • Diagnostiserad med genetiskt bekräftade Friedreichs ataxi (FA), dvs homozygot för guanin-adenin-adenin (GAA) upprepade expansion i intron-1 av frataxingenen, eller GAA-upprepning av expansion i 1 allel och med punktmutationer eller deletioner, eller andra icke-GAA-expansionsmutationer i den andra allelen.
  • Symtomatisk för FA som rapporterats av deltagaren och/eller föräldern/vårdgivaren a. Barn 7 till <16 år måste också ha en Upright Stability Score (USS) poäng på 10 till ≤ 34 vid baslinjen

Del 1 RCT: Knappsuteslutningskriterier:

  • Glykosylerat hemoglobin A1C (HbA1c)> 11%
  • B-typ natriuretisk peptid (BNP)> 200 picogram per milliliter (PG/ml) vid screening
  • Ejektionsfraktion (EF) <40% [baserat på ekokardiogram (ECHO) som utförs vid screeningbesök]
  • Kliniskt signifikant hjärtsjukdom utom mild till måttlig kardiomyopati

Del 2 OLE: Behörighetskriterier:

  • Deltagarna har slutfört del 1 RCT i studien och inga avbrottskriterier har uppfyllts

  • Säkerhets- och tolerabilitetsdata från del 1 RCT stöder fortsättningen i utredarens bedömning

    1. Om alaninaminotransferas (ALT), aspartataminotransferas (AST) och/eller totalt bilirubin (TBL) är> 2 × övre gräns för normal (Uln) vid föregående besöksbedömning, del 2 dag 1 bör försenas tills ALT och AST är <1,5 × uln och TBL är <2 × Uln
    2. Om BNP är> 200 pg/ml vid föregående besöksbedömning, bör del 2 dag 1 försenas tills BNP är <200 pg/ml
    3. Om någon annan kliniskt signifikant laboratorieavvikelser finns baserat på de tidigare besöksbedömningarna, bör del 2 dag 1 försenas tills avvikelserna har lösts
    4. I händelse av samtidig sjukdom eller annan förändring i deltagarens hälsostatus kan ytterligare del 1 -screeningbedömningar upprepas före inledningen av del 2, baserat på utredarens bedömning i samråd med Medical Monitor

Obs: Andra protokolldefinierade inkluderings-/uteslutningskriterier kan gälla.

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: Randomiserad
  • Interventionsmodell: Parallellt uppdrag
  • Maskning: Fyrdubbla

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: Part 1: 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.
Läkemedel: Omaveloxolon
Administreras enligt beskrivningen i behandlingsarmen.
Andra namn:
  • BIIB141, Skyclarys, RTA-408
Placebo-jämförare: Part 1: Placebo
Participants will receive placebo, orally, QD for up to 52 weeks in Part 1 of the study.
Läkemedel: Placebo
Administreras enligt beskrivningen i behandlingsarmen.
Experimentell: Part 2A Continued Efficacy Evaluation: Omaveloxolone
Participants will receive a single oral dose of omaveloxolone, QD for up to 104 weeks in Part 2A of the study.
Läkemedel: Omaveloxolon
Administreras enligt beskrivningen i behandlingsarmen.
Andra namn:
  • BIIB141, Skyclarys, RTA-408
Experimentell: Part 2B Safety: Omaveloxolone
Participants will receive a single oral dose of open-label omaveloxolone, QD for up to 104 weeks in Part 2B of the study.
Läkemedel: Omaveloxolon
Administreras enligt beskrivningen i behandlingsarmen.
Andra namn:
  • BIIB141, Skyclarys, RTA-408

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Part 1: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52
Tidsram: 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 2A: Change From Baseline in USS Subscale E of mFARS at Week 52
Tidsram: Baseline (Week 52 of Part 1), 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)
Tidsram: 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)
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
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 104
Baseline (Week 52 of Part 1), Weeks 52 and 104
Part 2B: Change From Baseline in Height at Weeks 52 and Week 104
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 104
Baseline (Week 52 of Part 1), Weeks 52 and 104
Part 2B: Change From Baseline in Weight at Weeks 52 and Week 104
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 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
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 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
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 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
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 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
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 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

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Part 1: Change From Baseline in Friedreich's Ataxia-Health Index (FA-HI) at Week 52
Tidsram: 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: Change From Baseline in Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52
Tidsram: 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: Change From Baseline in Patient Global Impressions-Severity (PGI-S) at Week 52
Tidsram: 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: Change From Baseline in Clinical Global Impressions-Severity (CGI-S) at Week 52
Tidsram: 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: Change From Baseline in Friedreich's Ataxia-Activities of Daily Living (FA-ADL)
Tidsram: 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: Number of Participants With Treatment-Emergent Adverse Event (TEAE) and Treatment-Emergent Serious Adverse Event (TESAE)
Tidsram: From first dose of study drug up to end of follow up period in Part 1 (up to Week 52)
From first dose of study drug up to end of follow up period in Part 1 (up to Week 52)
Part 1: Number of Participants With Change From Baseline in Cardiac Function Assessed by ECHO at Week 52
Tidsram: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Height at Week 52
Tidsram: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Weight at Week 52
Tidsram: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Body Mass Index (BMI) at Week 52
Tidsram: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS) at Week 52
Tidsram: Baseline, 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, Week 52
Part 1: Percentage of Participants at Each Tanner Stage at Week 52
Tidsram: Baseline, 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.
Baseline, Week 52
Part 1: Number of Participants at Each Tanner Stage at Week 52
Tidsram: Baseline, 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.
Baseline, Week 52
Part 1: Plasma Concentrations of Omaveloxolone
Tidsram: 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 2A: Change From Baseline in USS Subscale E of mFARS at Week 104
Tidsram: Baseline (Week 52 of Part 1), 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 of Part 1), Week 104
Part 2A: Change from baseline in mFARS at Weeks 52 and Week 104
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 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 of Part 1), Weeks 52 and 104
Part 2A: Number of Participants With TEAE and TESAE
Tidsram: From the first dose of the study drug in Part 2A up to the end of follow-up period in Part 2A (up to Week 104)
From the first dose of the study drug in Part 2A up to the end of follow-up period in Part 2A (up to Week 104)
Part 2A: Number of Participants With Change From Baseline in Cardiac Function Assessed by ECHO at Weeks 52 and Week 104
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 104
Baseline (Week 52 of Part 1), Weeks 52 and 104
Part 2A: Change From Baseline in Height at Weeks 52 and Week 104
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 104
Baseline (Week 52 of Part 1), Weeks 52 and 104
Part 2A: Change From Baseline in Weight at Weeks 52 and Week 104
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 104
Baseline (Week 52 of Part 1), Weeks 52 and 104
Part 2A: Change From Baseline in BMI at Weeks 52 and Week 104
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 104
Baseline (Week 52 of Part 1), Weeks 52 and 104
Part 2A: Change From Baseline in C-SSRS at Weeks 52 and Week 104
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 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 2A: Percentage of Participants at Each Tanner Stage at Weeks 52 and Week 104
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 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 2A: Number of Participants at Each Tanner Stage at Weeks 52 and Week 104
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 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 2A: Plasma Concentrations of Omaveloxolone
Tidsram: 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 2B: Change From Baseline in mFARS Including USS at Weeks 52 and Week 104
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 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 of Part 1), Weeks 52 and 104
Parts 2A and 2B: Change From Baseline in FA-HI at Weeks 52 and Week 104
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 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 of Part 1), Weeks 52 and 104
Parts 2A and 2B: Change From Baseline in PGI-S at Weeks 52 and Week 104
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 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 of Part 1), Weeks 52 and 104
Parts 2A and 2B: Change From Baseline in CGI-S at Weeks 52 and Week 104
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 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 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 of Part 1), Weeks 52 and 104
Parts 2A and 2B: Change from baseline in FA-ADL at Part 2A Weeks 52 and Week 104
Tidsram: Baseline (Week 52 of Part 1), Weeks 52 and 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.
Baseline (Week 52 of Part 1), Weeks 52 and 104

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Biogen

Utredare

  • Studierektor: Medical Director, Biogen

Publikationer och användbara länkar

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Användbara länkar

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

9 juni 2025

Primärt slutförande (Beräknad)

16 november 2027

Avslutad studie (Beräknad)

22 november 2029

Studieregistreringsdatum

Först inskickad

11 april 2025

Först inskickad som uppfyllde QC-kriterierna

29 april 2025

Första postat (Faktisk)

1 maj 2025

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

16 juni 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

15 juni 2026

Senast verifierad

1 juni 2026

Mer information

Termer relaterade till denna studie

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • 296FA301
  • 2025-520896-13 (Annan identifierare: EU CT Number)

Plan för individuella deltagardata (IPD)

Planerar du att dela individuella deltagardata (IPD)?

JA

IPD-planbeskrivning

I enlighet med BioGen's Clinical Trial Transparency and Data Sharing Policy på https://www.biogentrialtransparency.com/

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Ja

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

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