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En studie for å lære mer om effektene og langsiktig sikkerhet for BIIB141 (omaveloxolone) hos deltakere med Friedreichs ataksi i alderen 2 til 15 år gammel (BRAVE)

15. juni 2026 oppdatert av: Biogen

En fase 3, 2-delt, randomisert, dobbeltblind, placebokontrollert studie (del 1) og åpen etikettforlengelse (del 2) for å evaluere effektiviteten, sikkerhet, farmakokinetikk og farmakodynamikk av omaveloxolone (Biib141) i deltakere med Friedreichs ataxia i alderen til <16 år

I denne studien vil forskere lære mer om effektene og sikkerheten til BIIB141, også kjent som omaveloxolone eller SkyClarys®. Dette stoffet er godkjent, eller gjort tilgjengelig for leger å foreskrive, for personer med Friedreichs ataksi (FA) som er minst 16 år gamle. Men det er ennå ikke tilgjengelig for barn og tenåringer med FA som er yngre enn 16 år. Hovedmålet med denne studien er å lære hvordan BIIB141 fungerer i kroppen og om dens sikkerhet hos barn og tenåringer som er 2 til 15 år gamle.

De viktigste spørsmålene forskere ønsker å svare på i denne studien er:

  • Hvordan påvirker BIIB141 deltakernes FA -symptomer balanse og stabilitet?
  • Hvor mange deltakere har medisinske problemer i løpet av studien?
  • Er det noen endringer i deltakernes samlede helse under studien?
  • Er det noen endringer i deltakernes hjertehelse?
  • Er det noen endringer i hvordan deltakerne beveger seg gjennom puberteten? Pubertet er tiden i noens liv når kroppen deres endrer seg fra et barn til en voksen.

Forskere vil også lære mer om:

- Hvordan kroppen behandler biib141 hos barn og tenåringer

Denne studien vil bli gjort som følger:

  • Deltakerne vil bli vist for å sjekke om de kan bli med på studien. Screeningsperioden vil være opptil 28 dager, hvoretter deltakerne vil sjekke inn på studieforskningssenteret.
  • Det er to deler i denne studien. I løpet av del 1 vil deltakerne ta enten BIIB141 eller en placebo en gang om dagen.
  • I del 1 vil deltakerne ta BIIB141 eller placebo i et studieforskningssenter på dag 1, og deretter på personlige besøk på uke 4, uke 12, uke 26 og uke 52. På alle andre dager vil de ta BIIB141 eller placebo hjemme. Del 1 varer opptil 52 uker.
  • Under del 2 vil deltakere fra del 1 enten fortsette å ta BIIB141 eller starte den hvis de tok placebo. Del 2 vil vare opptil 104 uker.
  • I del 1 vil deltakerne ha opptil 10 besøk i studieforskningssenteret og en telefonsamtale i uke 2. I del 2 vil deltakerne ha besøk på uke 4, 8,12, 26, og hver 26. uke etter det til de forlater studien, og en telefonsamtale i uke 2.. Det vil være en siste telefonsamtale for å sjekke deltakernes helse 31 dager etter deres siste dose.
  • Hver deltaker vil være i studien i opptil 3 år

Studieoversikt

Status

Rekruttering

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

Det primære målet med del 1 randomisert kontrollert studie (RCT) er å evaluere effekten av omaveloxolon i uke 52, og de sekundære målene er å evaluere sikkerheten til omaveloxolon gjennom uke 52 og konsentrasjonen av omaveloxolon etter administrering av enkelt og flere dose. Det primære målet med del 2 åpen e-lettingsutvidelse (OLE) er å evaluere sikkerheten og toleransen av langvarig omaveloxolonbruk, og det sekundære målet er å evaluere effekten av omaveloxolon etter langvarig bruk.

Studietype

Intervensjonell

Registrering (Antatt)

255

Fase

  • Fase 3

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Studer Kontakt Backup

Studiesteder

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

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Barn

Tar imot friske frivillige

Nei

Beskrivelse

Del 1 RCT: Kriterier for nøkkel inkludering:

  • Diagnostisert med genetisk bekreftet Friedreichs ataksi (FA), dvs. homozygot for guanin-adenin-adenin (GAA) gjentatt ekspansjon i intron-1 av frelingenet, eller GAA gjentatt ekspansjon i 1 allel og med punktmutasjoner eller delioner, eller andre ikke-gaaaa-ekspansjoner i de andre mutasjonene.
  • Symptomatisk for FA som rapportert av deltakeren og/eller foreldrene/omsorgspersonen a. Barn 7 til <16 år må også ha en oppreist stabilitetspoeng (USS) på 10 til ≤ 34 ved baseline

Del 1 RCT: Key -eksklusjonskriterier:

  • Glykosylert hemoglobin A1c (HbA1c)> 11%
  • B-type natriuretisk peptid (BNP)> 200 picogrammer per milliliter (PG/ml) ved screening
  • Utkastingsfraksjon (EF) <40% [Basert på ekkokardiogram (Echo) utført ved screeningbesøk]
  • Klinisk signifikant hjertesykdom bortsett fra mild til moderat kardiomyopati

Del 2 ole: Kvalifiseringskriterier:

  • Deltakerne har fullført del 1 RCT i studien, og ingen seponeringskriterier er oppfylt

  • Sikkerhets- og tolerabilitetsdata fra del 1 RCT støtter fortsettelsen av etterforskerens dom i etterforskeren

    1. Hvis alaninaminotransferase (ALT), aspartataminotransferase (AST) og/eller total bilirubin (TBL) er> 2 × øvre grense for normal (ULN) ved forrige besøksvurdering, bør del 2 dag 1 forsinket til ALT og AST er <1,5 × ULN og TBL er <2 x uln
    2. Hvis BNP er> 200 pg/ml ved forrige besøksvurdering, bør del 2 dag 1 bli forsinket til BNP er <200 pg/ml
    3. Hvis noen andre klinisk signifikante laboratorieavvik er til stede basert på de tidligere besøksvurderingene, bør del 2 dag 1 forsinket til abnormitetene er løst
    4. I tilfelle interstrøm sykdom eller annen endring i deltakerens helsestatus, kan ytterligere del 1 -screeningvurderinger gjentas før initiering av del 2, basert på etterforskerens dom i samråd med den medisinske skjermen

Merk: Andre protokolldefinerte inkluderings-/eksklusjonskriterier kan gjelde.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Firemannsrom

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: 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.
Legemiddel: Omaveloxolone
Administreres som spesifisert i behandlingsarmen.
Andre navn:
  • BIIB141, SkyClarys, RTA-408
Placebo komparator: Part 1: Placebo
Participants will receive placebo, orally, QD for up to 52 weeks in Part 1 of the study.
Legemiddel: Placebo
Administreres som spesifisert i behandlingsarmen.
Eksperimentell: 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.
Legemiddel: Omaveloxolone
Administreres som spesifisert i behandlingsarmen.
Andre navn:
  • BIIB141, SkyClarys, RTA-408
Eksperimentell: 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.
Legemiddel: Omaveloxolone
Administreres som spesifisert i behandlingsarmen.
Andre navn:
  • BIIB141, SkyClarys, RTA-408

Hva måler studien?

Primære resultatmål

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

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Part 1: Change From Baseline in Friedreich's Ataxia-Health Index (FA-HI) at Week 52
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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
Tidsramme: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Height at Week 52
Tidsramme: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Weight at Week 52
Tidsramme: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Body Mass Index (BMI) at Week 52
Tidsramme: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS) at Week 52
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Biogen

Etterforskere

  • Studieleder: Medical Director, Biogen

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Hjelpsomme linker

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

9. juni 2025

Primær fullføring (Antatt)

16. november 2027

Studiet fullført (Antatt)

22. november 2029

Datoer for studieregistrering

Først innsendt

11. april 2025

Først innsendt som oppfylte QC-kriteriene

29. april 2025

Først lagt ut (Faktiske)

1. mai 2025

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

16. juni 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

15. juni 2026

Sist bekreftet

1. juni 2026

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • 296FA301
  • 2025-520896-13 (Annen identifikator: EU CT Number)

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

JA

IPD-planbeskrivelse

I samsvar med Biogens kliniske studiepolitikk og datadelingspolitikk på https://www.biogentrialtransparency.com/

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

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