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Un estudio para obtener más información sobre los efectos y la seguridad a largo plazo de BiIB141 (omeloxolona) en participantes con Ataxia de Friedreich de 2 a 15 años. (BRAVE)

15 de junio de 2026 actualizado por: Biogen

Un estudio de fase 3, 2 partes, aleatorizado, doble ciego, controlado con placebo (Parte 1) y extensión abierta (Parte 2) para evaluar la eficacia, la seguridad, la farmacocinética y la farmacodinámica de la omeloxolona (BiIB141) en participantes con friedreich de 2 a <16 años de 16 años <16 años de 16 años.

En este estudio, los investigadores aprenderán más sobre los efectos y la seguridad de BiIB141, también conocido como omeloxolona o SkyClarys®. Este medicamento ha sido aprobado o puesto a disposición para que los médicos receten, para que las personas con Ataxia (FA) de Friedreich (FA) tengan al menos 16 años. Pero aún no está disponible para niños y adolescentes con FA que tienen más de 16 años. El objetivo principal de este estudio es aprender cómo funciona BIIB141 en el cuerpo y sobre su seguridad en niños y adolescentes de 2 a 15 años.

Las principales preguntas que los investigadores quieren responder en este estudio son:

  • ¿Cómo afecta BIIB141 el equilibrio y la estabilidad del equilibrio y la estabilidad de los síntomas de FA de los participantes?
  • ¿Cuántos participantes tienen problemas médicos durante el estudio?
  • ¿Hay algún cambio en la salud general de los participantes durante el estudio?
  • ¿Hay algún cambio en la salud del corazón de los participantes?
  • ¿Hay algún cambio en cómo los participantes se mueven a través de la pubertad? La pubertad es el momento de la vida de alguien cuando su cuerpo cambia de un niño a un adulto.

Los investigadores también aprenderán más sobre:

- Cómo el cuerpo procesa BIIB141 en niños y adolescentes

Este estudio se realizará de la siguiente manera:

  • Los participantes serán seleccionados para verificar si pueden unirse al estudio. El período de detección será de hasta 28 días, después de lo cual los participantes se registrarán en su centro de investigación de estudio.
  • Hay 2 partes en este estudio. Durante la Parte 1, los participantes tomarán BIIB141 o un placebo una vez al día.
  • En la Parte 1, los participantes tomarán BIIB141 o el placebo en un centro de investigación de estudio el día 1, y luego en las visitas en persona en la semana 4, la semana 12, la semana 26 y la semana 52. En todos los demás días, tomarán BiIB141 o el placebo en casa. La Parte 1 dura hasta 52 semanas.
  • Durante la Parte 2, los participantes de la Parte 1 continuarán tomando BIIB141 o comenzarán si estaban tomando el placebo. La Parte 2 durará hasta 104 semanas.
  • En la Parte 1, los participantes tendrán hasta 10 visitas a su centro de investigación de estudio y una llamada telefónica en la Semana 2. En la Parte 2, los participantes tendrán visitas en las semanas 4, 8,12, 26, y cada 26 semanas después de eso hasta que abandonen el estudio, y una llamada telefónica en la semana 2. Habrá una llamada telefónica final para verificar la salud de los participantes 31 días después de su última dosis.
  • Cada participante estará en el estudio por hasta unos 3 años.

Descripción general del estudio

Estado

Reclutamiento

Condiciones

Intervención / Tratamiento

Descripción detallada

El objetivo principal del ensayo controlado aleatorizado (ECA) de la Parte 1 es evaluar la eficacia de la omeloxolona en la semana 52 y los objetivos secundarios son evaluar la seguridad de la omeloxolona hasta la semana 52 y la concentración de omeloxolona después de la administración de dosis única y múltiple. El objetivo principal del ensayo de extensión abierta (OLE) de la Parte 2 es evaluar la seguridad y la tolerabilidad del uso de omeloxolona a largo plazo y el objetivo secundario es evaluar la eficacia de la omeloxolona después del uso a largo plazo.

Tipo de estudio

Intervencionista

Inscripción (Estimado)

255

Fase

  • Fase 3

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

Copia de seguridad de contactos de estudio

Ubicaciones de estudio

  • Alemania
      • Giessen, Alemania, 35392
        • Reclutamiento
        • UKGM - Universitätsklinikum Giessen und Marburg GmbH - Standort Gießen
        • Investigador principal:
          • Andreas Hahn
        • Contacto:
      • Hamburg, Alemania, 20246
        • Reclutamiento
        • Universitätsklinikum Hamburg Eppendorf
        • Contacto:
          • Número de teléfono: +49 40 7410 56126
          • Correo electrónico: d.weiss@uke.de
        • Investigador principal:
          • Deike Weiss
    • North Rhine-Westphalia
      • Aachen, North Rhine-Westphalia, Alemania, 52074
        • Reclutamiento
        • Universitätsklinikum Aachen
        • Investigador principal:
          • Kathrin Reetz
        • Contacto:
  • Arabia Saudita
    • Ar Riya
      • Riyadh, Ar Riya, Arabia Saudita, 12875
        • Retirado
        • King Faisal Specialist Hospital & Research Centre
  • Australia
    • New South Wales
      • Randwick, New South Wales, Australia, 2031
        • Aún no reclutando
        • Sydney Children's Hospital
    • Victoria
      • Parkville, Victoria, Australia, 3052
        • Reclutamiento
        • Murdoch Childrens Research Institute (MCRI)
  • Austria
      • Innsbruck, Austria, 6020
        • Reclutamiento
        • Universitätsklinikum Innsbruck
        • Contacto:
        • Investigador principal:
          • Sylvia M Boesch
  • Brasil
    • Federal District
      • Brasília, Federal District, Brasil, 70200-730
        • Reclutamiento
        • L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME
        • Contacto:
        • Investigador principal:
          • Ingrid Faber Faber de Vasconcellos
    • São Paulo
      • Campinas, São Paulo, Brasil, 13083-970
        • Aún no reclutando
        • University of Campinas (UNICAMP) School of Medical Sciences
        • Contacto:
        • Investigador principal:
          • Marcondes Franca
      • São Paulo, São Paulo, Brasil, 04024-002
        • Reclutamiento
        • Pseg Centro de Pesquisa Clinica
        • Investigador principal:
          • Paulo Victor Sgobbi de Souza
        • Contacto:
  • Canadá
    • Quebec
      • Montreal, Quebec, Canadá, H3H 2R9
        • Reclutamiento
        • McGill University
        • Contacto:
        • Investigador principal:
          • Maryam Oskoui
      • Québec, Quebec, Canadá, G1V 4G2
        • Reclutamiento
        • CHU de Quebec -Universite Laval
        • Contacto:
        • Investigador principal:
          • Nicolas Chrestian
  • Dinamarca
      • Copenhagen, Dinamarca, 2100
        • Aún no reclutando
        • Rigshospitalet - Juliane Marie Centret (JMC) Copenhagen
        • Contacto:
        • Investigador principal:
          • Alfred Peter Born
  • España
      • Madrid, España, 28046
        • Reclutamiento
        • Hospital Universitario La Paz - PPDS
        • Contacto:
        • Investigador principal:
          • Maria del Mar Garcia Romero
    • Barcelona
      • Espluges de Llobregat, Barcelona, España, 8950
        • Reclutamiento
        • Hospital Sant Joan de Deu - PIN
        • Contacto:
        • Investigador principal:
          • Alejandra Darling
  • Estados Unidos
    • California
      • Los Angeles, California, Estados Unidos, 90095
        • Aún no reclutando
        • UCLA Neurology Outpatient Clinic at Westwood
        • Contacto:
        • Investigador principal:
          • Susan Perlman
    • Florida
      • Gainesville, Florida, Estados Unidos, 32610-3010
        • Reclutamiento
        • Norman Fixel Institute for Neurological Diseases UF Health
        • Contacto:
        • Investigador principal:
          • Sankarsubramoney Subramony
      • Tampa, Florida, Estados Unidos, 33612
        • Reclutamiento
        • USF Health Morsani College of Medicine Department of Neurology
        • Investigador principal:
          • Theresa Zesiewicz
        • Contacto:
    • Pennsylvania
      • Philadelphia, Pennsylvania, Estados Unidos, 19104
        • Reclutamiento
        • Children's Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - PIN
        • Contacto:
        • Investigador principal:
          • David Robinson Lynch
    • Tennessee
      • Memphis, Tennessee, Estados Unidos, 38105-3678
        • Reclutamiento
        • St. Jude Children's Research Hospital - PIN
        • Investigador principal:
          • Richard Finkel
        • Contacto:
    • Virginia
      • Norfolk, Virginia, Estados Unidos, 23507-1910
        • Reclutamiento
        • CHKD's Health Center - South Campus - PIN
        • Contacto:
        • Investigador principal:
          • Crystal Proud
    • Washington
      • Seattle, Washington, Estados Unidos, 98105-3901
        • Reclutamiento
        • Seattle Children's Hospital
        • Contacto:
          • Número de teléfono: 206-987-2078
        • Investigador principal:
          • Alicia Henriquez
  • Francia
      • Paris, Francia, 75012
        • Reclutamiento
        • AP-HP - Hôpital Armand Trousseau
        • Investigador principal:
          • Florence Renaldo
        • Contacto:
    • Hérault
      • Montpellier, Hérault, Francia, 34090
        • Reclutamiento
        • CHU de Montpellier- Hôpital Gui De Chauliac
        • Contacto:
        • Investigador principal:
          • Agathe Roubertie
  • India
    • National Capital Territory of Delhi
      • New Delhi, National Capital Territory of Delhi, India, 110029
        • Retirado
        • All India Institute of Medical Sciences (AIIMS) - New Delhi
  • Irlanda
      • Dublin, Irlanda, D01 XD99
        • Reclutamiento
        • CHI at Temple Street
        • Contacto:
        • Investigador principal:
          • Declan O'Rourke
  • Italia
      • Milan, Italia, 20133
        • Reclutamiento
        • Fondazione IRCCS Istituto Neurologico Carlo Besta
        • Contacto:
        • Investigador principal:
          • Isabella Moroni
    • Lazio
      • Rome, Lazio, Italia, 165
        • Aún no reclutando
        • Ospedale Pediatrico Bambino Gesù IRCCS
        • Investigador principal:
          • Gessica Vasco
        • Contacto:
    • Veneto
      • Conegliano, Veneto, Italia, 31015
        • Aún no reclutando
        • IRCCS Eugenio Medea - Polo. Scientifico Veneto
        • Investigador principal:
          • Gabriella Paparella
        • Contacto:
  • Países Bajos
      • Nijmegen, Países Bajos, 6525 GA
        • Reclutamiento
        • Radboud Universitair Medisch Centrum
        • Contacto:
          • Número de teléfono: 31 243614415
        • Investigador principal:
          • Nienke van Os
  • Reino Unido
    • Lincolnshire
      • London, Lincolnshire, Reino Unido, NW1 2BU
        • Reclutamiento
        • University College Hospital - PPDS
        • Contacto:
        • Investigador principal:
          • Shpresa Pula
    • Oxfordshire
      • Oxford, Oxfordshire, Reino Unido, OX3 9DU
        • Reclutamiento
        • John Radcliffe Hospital
        • Investigador principal:
          • Andrea Németh
        • Contacto:
          • Número de teléfono: 44 1865 231556
    • South Yorkshire
      • Sheffield, South Yorkshire, Reino Unido, S10 5DD
        • Reclutamiento
        • Sheffield Children's Hospital - PPDS
        • Contacto:
        • Investigador principal:
          • Santosh Ravindra Mordekar
  • Turquía (Türkiye)
      • Istanbul, Turquía (Türkiye), 34093
        • Retirado
        • Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

  • Niño

Acepta Voluntarios Saludables

No

Descripción

Parte 1 RCT: Criterios de inclusión clave:

  • Diagnosticado con la ataxia (FA) de Friedreich genéticamente confirmada, es decir, homocigoto para la expansión repetida de guanina-adenina-adenina (GAA) en intron-1 del gen frataxina, o expansión repetida GAA en alelo 1 y con mutaciones o deleciones puntuales, u otras mutaciones de expansión de no GAA en las otras alóse.
  • Sintomático para FA según lo informado por el participante y/o el padre/cuidador a. Los niños de 7 a <16 años también deben tener un puntaje de estabilidad vertical (USS) de 10 a ≤ 34 al inicio

Parte 1 RCT: Criterios de exclusión clave:

  • Hemoglobina A1C glicosilada (HBA1C)> 11%
  • Péptido natriurético de tipo B (BNP)> 200 Picogramas por mililitro (PG/ml) en la detección
  • Fracción de eyección (EF) <40% [basada en ecocardiograma (ECHO) realizada en la visita de detección]
  • Enfermedad cardíaca clínicamente significativa, excepto la miocardiopatía leve a moderada

Parte 2 OLE: Criterios de elegibilidad:

  • Los participantes han completado el ECA de la Parte 1 del estudio y no se han cumplido criterios de interrupción

  • Los datos de seguridad y tolerabilidad de la Parte 1 ECA apoyan la continuación en el juicio del investigador

    1. Si la alanina aminotransferasa (ALT), la aspartato aminotransferasa (AST) y/o la bilirrubina total (TBL) son> 2 × límite superior de lo normal (ULN) en la evaluación de la visita anterior, la Parte 2 Día 1 debe retrasarse hasta que ALT y AST sean <1.5 × ULN y TBL es <2 × ULNN.
    2. Si BNP es> 200 pg/ml en la evaluación de la visita anterior, la parte 2 del día 1 debe retrasarse hasta que BNP sea <200 pg/ml
    3. Si alguna otra anormalidades de laboratorio clínicamente significativas está presente en función de las evaluaciones de la visita anterior, la parte 2 del día 1 debe retrasarse hasta que se resuelvan las anormalidades
    4. En caso de enfermedad intercurrente u otro cambio en el estado de salud del participante, las evaluaciones de detección adicionales de la Parte 1 pueden repetirse antes del inicio de la Parte 2, en función del juicio del investigador en consulta con el Monitor Médico

Nota: Se pueden aplicar otros criterios de inclusión/exclusión definidos por el protocolo.

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: Aleatorizado
  • Modelo Intervencionista: Asignación paralela
  • Enmascaramiento: Cuadruplicar

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: 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.
Droga: Omaveloxolona
Administrado según lo especificado en el grupo de tratamiento.
Otros nombres:
  • Biib141, SkyClarys, RTA-408
Comparador de placebos: Part 1: Placebo
Participants will receive placebo, orally, QD for up to 52 weeks in Part 1 of the study.
Droga: Placebo
Administrado como se especifica en el brazo de tratamiento.
Experimental: 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.
Droga: Omaveloxolona
Administrado según lo especificado en el grupo de tratamiento.
Otros nombres:
  • Biib141, SkyClarys, RTA-408
Experimental: 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.
Droga: Omaveloxolona
Administrado según lo especificado en el grupo de tratamiento.
Otros nombres:
  • Biib141, SkyClarys, RTA-408

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Part 1: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Part 1: Change From Baseline in Friedreich's Ataxia-Health Index (FA-HI) at Week 52
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Height at Week 52
Periodo de tiempo: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Weight at Week 52
Periodo de tiempo: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Body Mass Index (BMI) at Week 52
Periodo de tiempo: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS) at Week 52
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Biogen

Investigadores

  • Director de estudio: Medical Director, Biogen

Publicaciones y enlaces útiles

La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.

Enlaces Útiles

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

9 de junio de 2025

Finalización primaria (Estimado)

16 de noviembre de 2027

Finalización del estudio (Estimado)

22 de noviembre de 2029

Fechas de registro del estudio

Enviado por primera vez

11 de abril de 2025

Primero enviado que cumplió con los criterios de control de calidad

29 de abril de 2025

Publicado por primera vez (Actual)

1 de mayo de 2025

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

16 de junio de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

15 de junio de 2026

Última verificación

1 de junio de 2026

Más información

Términos relacionados con este estudio

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • 296FA301
  • 2025-520896-13 (Otro identificador: EU CT Number)

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

SÍ

Descripción del plan IPD

De acuerdo con la política de transparencia y intercambio de datos de ensayos clínicos de Biogen en https://www.biogentrialtransparency.com/

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

Sí

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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