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En undersøgelse for at lære mere om virkningerne og langvarig sikkerhed for BIIB141 (Omaveloxolon) hos deltagere med Friedreichs ataksi i alderen 2 til 15 år gammel (BRAVE)

2. juni 2026 opdateret af: Biogen

En fase 3, 2-delt, randomiseret, dobbeltblind, placebokontrolleret undersøgelse (del 1) og open-label-udvidelse (del 2) for at evaluere effektiviteten, sikkerhed, farmakokinetik og farmakodynamik af omaveloxolon (BIIB141) i deltagere med Friedreichs ataksi i alderen 2 til <16 år

I denne undersøgelse vil forskere lære mere om virkningerne og sikkerheden af ​​BIIB141, også kendt som omaveloxolon eller Skyclarys®. Dette stof er godkendt eller stillet til rådighed for læger at ordinere for mennesker med Friedreichs ataksi (FA), der er mindst 16 år gamle. Men det er endnu ikke tilgængeligt for børn og teenagere med FA, der er yngre end 16 år gamle. Hovedmålet med denne undersøgelse er at lære, hvordan BIIB141 fungerer i kroppen og om dens sikkerhed hos børn og teenagere, der er 2 til 15 år gamle.

De vigtigste spørgsmål, som forskere ønsker at besvare i denne undersøgelse, er:

  • Hvordan påvirker BIIB141 deltagernes FA -symptomer balance og stabilitet?
  • Hvor mange deltagere har medicinske problemer under undersøgelsen?
  • Er der nogen ændringer i deltagernes generelle helbred under undersøgelsen?
  • Er der nogen ændringer i deltagernes hjertesundhed?
  • Er der nogen ændringer i, hvordan deltagerne bevæger sig gennem puberteten? Puberteten er tiden i nogens liv, når deres krop skifter fra et barn til en voksen.

Forskere vil også lære mere om:

- Hvordan kroppen behandler BIIB141 hos børn og teenagere

Denne undersøgelse vil blive udført som følger:

  • Deltagerne vil blive screenet for at kontrollere, om de kan deltage i undersøgelsen. Screeningsperioden vil være op til 28 dage, hvorefter deltagerne tjekker ind i deres studieforskningscenter.
  • Der er 2 dele i denne undersøgelse. I løbet af del 1 vil deltagerne tage enten BIIB141 eller en placebo en gang om dagen.
  • I del 1 vil deltagerne tage BIIB141 eller placebo i et studieforskningscenter på dag 1 og derefter ved personlige besøg i uge 4, uge ​​12, uge ​​26 og uge 52. På alle andre dage tager de BIIB141 eller placebo derhjemme. Del 1 varer op til 52 uger.
  • I løbet af del 2 vil deltagere fra del 1 enten fortsætte med at tage BIIB141 eller starte det, hvis de tog placebo. Del 2 varer op til 104 uger.
  • I del 1 vil deltagerne have op til 10 besøg på deres studieforskningscenter og et telefonopkald i uge 2.. I del 2 vil deltagerne have besøg i uger 4, 8,12, 26, og hver 26. uge efter det, indtil de forlader undersøgelsen, og et telefonopkald i uge 2. vil der være et sidste telefonopkald for at kontrollere deltagernes sundhed 31 dage efter deres sidste dosis.
  • Hver deltager vil være i undersøgelsen i op til ca. 3 år

Studieoversigt

Status

Rekruttering

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

Det primære mål med del 1 randomiseret kontrolleret forsøg (RCT) er at evaluere effektiviteten af ​​omaveloxolon i uge 52, og de sekundære mål er at evaluere sikkerhed for omaveloxolon gennem uge 52 og koncentrationen af ​​omaveloxolon efter enkelt- og multiple dosisadministration. Det primære mål med del 2 open-label-udvidelse (OLE) -forsøg er at evaluere sikkerheden og tolerabiliteten af ​​langvarig omaveloxolonbrug, og det sekundære mål er at evaluere effektiviteten af ​​omaveloxolon efter langvarig anvendelse.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

255

Fase

  • Fase 3

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Undersøgelse Kontakt Backup

Studiesteder

  • Australien
    • New South Wales
      • Randwick, New South Wales, Australien, 2031
        • Ikke rekrutterer endnu
        • Sydney Children's Hospital
    • Victoria
      • Parkville, Victoria, Australien, 3052
        • Rekruttering
        • Murdoch Childrens Research Institute (MCRI)
  • Brasilien
    • Federal District
      • Brasília, Federal District, Brasilien, 70200-730
        • Rekruttering
        • L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME
        • Kontakt:
        • Ledende efterforsker:
          • Ingrid Faber Faber de Vasconcellos
    • São Paulo
      • Campinas, São Paulo, Brasilien, 13083-970
        • Ikke rekrutterer endnu
        • University of Campinas (UNICAMP) School of Medical Sciences
        • Kontakt:
        • Ledende efterforsker:
          • Marcondes Franca
      • São Paulo, São Paulo, Brasilien, 04024-002
        • Rekruttering
        • PSEG Centro de Pesquisa Clinica
        • Ledende efterforsker:
          • Paulo Victor Sgobbi de Souza
        • Kontakt:
  • Canada
    • Quebec
      • Montreal, Quebec, Canada, H3H 2R9
        • Rekruttering
        • McGill University
        • Kontakt:
        • Ledende efterforsker:
          • Maryam Oskoui
      • Québec, Quebec, Canada, G1V 4G2
  • Danmark
      • Copenhagen, Danmark, 2100
        • Ikke rekrutterer endnu
        • Rigshospitalet - Juliane Marie Centret (JMC) Copenhagen
        • Kontakt:
        • Ledende efterforsker:
          • Alfred Peter Born
  • Det Forenede Kongerige
    • Lincolnshire
      • London, Lincolnshire, Det Forenede Kongerige, NW1 2BU
        • Rekruttering
        • University College Hospital - PPDS
        • Kontakt:
        • Ledende efterforsker:
          • Shpresa Pula
    • Oxfordshire
      • Oxford, Oxfordshire, Det Forenede Kongerige, OX3 9DU
        • Rekruttering
        • John Radcliffe Hospital
        • Ledende efterforsker:
          • Andrea Németh
        • Kontakt:
          • Telefonnummer: 44 1865 231556
    • South Yorkshire
      • Sheffield, South Yorkshire, Det Forenede Kongerige, S10 5DD
        • Rekruttering
        • Sheffield Children's Hospital - PPDS
        • Kontakt:
        • Ledende efterforsker:
          • Santosh Ravindra Mordekar
  • Forenede Stater
    • California
      • Los Angeles, California, Forenede Stater, 90095
        • Ikke rekrutterer endnu
        • UCLA Neurology Outpatient Clinic at Westwood
        • Kontakt:
        • Ledende efterforsker:
          • Susan Perlman
    • Florida
      • Gainesville, Florida, Forenede Stater, 32610-3010
        • Rekruttering
        • Norman Fixel Institute for Neurological Diseases UF Health
        • Kontakt:
        • Ledende efterforsker:
          • Sankarsubramoney Subramony
      • Tampa, Florida, Forenede Stater, 33612
        • Rekruttering
        • USF Health Morsani College of Medicine Department of Neurology
        • Ledende efterforsker:
          • Theresa Zesiewicz
        • Kontakt:
    • Pennsylvania
      • Philadelphia, Pennsylvania, Forenede Stater, 19104
        • Rekruttering
        • Children's Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - PIN
        • Kontakt:
        • Ledende efterforsker:
          • David Robinson Lynch
    • Tennessee
      • Memphis, Tennessee, Forenede Stater, 38105-3678
        • Rekruttering
        • St. Jude Children's Research Hospital - PIN
        • Ledende efterforsker:
          • Richard Finkel
        • Kontakt:
    • Virginia
      • Norfolk, Virginia, Forenede Stater, 23507-1910
        • Rekruttering
        • CHKD's Health Center - South Campus - PIN
        • Kontakt:
        • Ledende efterforsker:
          • Crystal Proud
    • Washington
      • Seattle, Washington, Forenede Stater, 98105-3901
        • Rekruttering
        • Seattle Children's Hospital
        • Kontakt:
          • Telefonnummer: 206-987-2078
        • Ledende efterforsker:
          • Alicia Henriquez
  • Frankrig
      • Paris, Frankrig, 75012
        • Rekruttering
        • AP-HP - Hôpital Armand Trousseau
        • Ledende efterforsker:
          • Florence Renaldo
        • Kontakt:
    • Hérault
      • Montpellier, Hérault, Frankrig, 34090
        • Rekruttering
        • CHU de Montpellier- Hôpital Gui De Chauliac
        • Kontakt:
        • Ledende efterforsker:
          • Agathe Roubertie
  • Holland
      • Nijmegen, Holland, 6525 GA
        • Rekruttering
        • Radboud Universitair Medisch Centrum
        • Kontakt:
          • Telefonnummer: 31 243614415
        • Ledende efterforsker:
          • Nienke van Os
  • Indien
    • National Capital Territory of Delhi
      • New Delhi, National Capital Territory of Delhi, Indien, 110029
        • Ikke rekrutterer endnu
        • All India Institute of Medical Sciences (AIIMS) - New Delhi
  • Irland
      • Dublin, Irland, D01 XD99
        • Rekruttering
        • CHI at Temple Street
        • Kontakt:
        • Ledende efterforsker:
          • Declan O'Rourke
  • Italien
      • Milan, Italien, 20133
        • Rekruttering
        • Fondazione IRCCS Istituto Neurologico Carlo Besta
        • Kontakt:
        • Ledende efterforsker:
          • Isabella Moroni
    • Lazio
      • Rome, Lazio, Italien, 165
        • Ikke rekrutterer endnu
        • Ospedale Pediatrico Bambino Gesù IRCCS
        • Ledende efterforsker:
          • Gessica Vasco
        • Kontakt:
    • Veneto
      • Conegliano, Veneto, Italien, 31015
        • Ikke rekrutterer endnu
        • IRCCS Eugenio Medea - Polo. Scientifico Veneto
        • Ledende efterforsker:
          • Gabriella Paparella
        • Kontakt:
  • Saudi Arabien
    • Ar Riya
      • Riyadh, Ar Riya, Saudi Arabien, 12875
        • Ikke rekrutterer endnu
        • King Faisal Specialist Hospital & Research Centre
        • Kontakt:
          • Telefonnummer: +966 11-4427773
        • Ledende efterforsker:
          • Amaal AlDakheel
  • Spanien
      • Madrid, Spanien, 28046
        • Rekruttering
        • Hospital Universitario La Paz - PPDS
        • Kontakt:
        • Ledende efterforsker:
          • Maria del Mar Garcia Romero
    • Barcelona
      • Espluges de Llobregat, Barcelona, Spanien, 8950
        • Rekruttering
        • Hospital Sant Joan de Deu - PIN
        • Kontakt:
        • Ledende efterforsker:
          • Alejandra Darling
  • Tyrkiet (Türkiye)
      • Istanbul, Tyrkiet (Türkiye), 34093
        • Ikke rekrutterer endnu
        • Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
        • Kontakt:
          • Telefonnummer: 902124142000
        • Ledende efterforsker:
          • Zuhal Yapici
  • Tyskland
      • Giessen, Tyskland, 35392
        • Rekruttering
        • UKGM - Universitätsklinikum Giessen und Marburg GmbH - Standort Gießen
        • Ledende efterforsker:
          • Andreas Hahn
        • Kontakt:
      • Hamburg, Tyskland, 20246
        • Rekruttering
        • Universitatsklinikum Hamburg Eppendorf
        • Kontakt:
        • Ledende efterforsker:
          • Deike Weiss
    • North Rhine-Westphalia
      • Aachen, North Rhine-Westphalia, Tyskland, 52074
        • Rekruttering
        • Universitätsklinikum Aachen
        • Ledende efterforsker:
          • Kathrin Reetz
        • Kontakt:
  • Østrig
      • Innsbruck, Østrig, 6020
        • Rekruttering
        • Universitätsklinikum Innsbruck
        • Kontakt:
        • Ledende efterforsker:
          • Sylvia M Boesch

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn

Tager imod sunde frivillige

Ingen

Beskrivelse

Del 1 RCT: Nøgleindeslutningskriterier:

  • Diagnosticeret med genetisk bekræftet Friedreichs ataksi (FA), dvs. homozygot for guanine-adenine-adenine (GAA) gentagelsesekspansion i intron-1 af frataxingenet, eller GAA-gentagelsesekspansion i 1 allele og med punktmutationer eller deletioner eller andre ikke-GAA-ekspansionsmutationer i det andet allele.
  • Symptomatisk for FA som rapporteret af deltageren og/eller forælderen/plejeren a. Børn 7 til <16 år skal også have en lodret stabilitetsscore (USS) score på 10 til ≤ 34 ved baseline

Del 1 RCT: Nøgleekskluderingskriterier:

  • Glycosyleret hæmoglobin A1C (HBA1C)> 11%
  • Natriuretisk peptid af B-type (BNP)> 200 picograms pr. Ml (PG/ml) ved screening
  • Ejekterfraktion (EF) <40% [baseret på ekkokardiogram (ECHO) udført ved screeningsbesøg]
  • Klinisk signifikant hjertesygdom undtagen mild til moderat kardiomyopati

Del 2 ole: Kriterier for støtteberettigelse:

  • Deltagerne har afsluttet del 1 RCT i undersøgelsen, og der er ikke opfyldt seponeringskriterier

  • Sikkerheds- og tolerabilitetsdata fra del 1 RCT støtter fortsættelsen i efterforskerens dom

    1. Hvis alaninaminotransferase (ALT), aspartataminotransferase (AST) og/eller total bilirubin (TBL) er> 2 × øvre grænse for normal (ULN) ved den forrige besøgsvurdering, skal del 2 dag 1 blive forsinket, indtil alt og ast er <1,5 × uln og tbl er <2 × ULN ULN ULN
    2. Hvis BNP er> 200 pg/ml ved den forrige besøgsvurdering, skal del 2 dag 1 blive forsinket, indtil BNP er <200 pg/ml
    3. Hvis der er andre klinisk signifikante laboratorie abnormiteter til stede baseret på de forrige besøgsvurderinger, skal del 2 dag 1 forsinkes, indtil abnormiteterne er løst
    4. I tilfælde af intercurrent sygdom eller anden ændring i deltagerens sundhedsstatus, kan yderligere del 1 -screeningsvurderinger gentages inden påbegyndelse af del 2, baseret på efterforskerens dom i samråd med den medicinske monitor

Bemærk: Andre protokoldefinerede inklusions-/ekskluderingskriterier kan gælde.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Firedobbelt

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: 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.
Medicin: Omaveloxolone
Indgives som specificeret i behandlingsarmen.
Andre navne:
  • 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.
Medicin: Placebo
Indgives som specificeret i behandlingsarmen.
Eksperimentel: 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.
Medicin: Omaveloxolone
Indgives som specificeret i behandlingsarmen.
Andre navne:
  • Biib141, Skyclarys, RTA-408
Eksperimentel: 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.
Medicin: Omaveloxolone
Indgives som specificeret i behandlingsarmen.
Andre navne:
  • Biib141, Skyclarys, RTA-408

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
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
Foranstaltningsbeskrivelse
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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Biogen

Efterforskere

  • Studieleder: Medical Director, Biogen

Publikationer og nyttige links

Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.

Hjælpsomme links

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

9. juni 2025

Primær færdiggørelse (Anslået)

16. november 2027

Studieafslutning (Anslået)

22. november 2029

Datoer for studieregistrering

Først indsendt

11. april 2025

Først indsendt, der opfyldte QC-kriterier

29. april 2025

Først opslået (Faktiske)

1. maj 2025

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

3. juni 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

2. juni 2026

Sidst verificeret

1. juni 2026

Mere information

Begreber relateret til denne undersøgelse

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

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

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-planbeskrivelse

I overensstemmelse med Biogens kliniske forsøgs gennemsigtighed og datadelingspolitik på https://www.biogentrialtransparency.com/

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

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