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Um estudo para saber mais sobre os efeitos e a segurança a longo prazo do BIIB141 (olaveroxolona) em participantes com ataxia de Friedreich com idades entre 2 e 15 anos (BRAVE)

2 de junho de 2026 atualizado por: Biogen

Um estudo de fase 3, 2 partes, randomizado, duplo-cego e controlado por placebo (Parte 1) e extensão de etiqueta aberta (Parte 2) para avaliar a eficácia, segurança, farmacocinética e farmacodinâmica de olavelxolona (BIIB141) em participantes da Ataxia de Friedreich, 2 a <16 anos

Neste estudo, os pesquisadores aprenderão mais sobre os efeitos e a segurança do BIIB141, também conhecidos como olaveloxolona ou Skyclarys®. Este medicamento foi aprovado ou disponibilizado para os médicos prescrever, para pessoas com ataxia (FA) de Friedreich, com pelo menos 16 anos de idade. Mas ainda não está disponível para crianças e adolescentes com FA com menos de 16 anos de idade. O principal objetivo deste estudo é aprender como o BIIB141 funciona no corpo e sobre sua segurança em crianças e adolescentes com 2 a 15 anos de idade.

As principais perguntas que os pesquisadores desejam responder neste estudo são:

  • Como o BIIB141 afeta o equilíbrio e a estabilidade dos sintomas da FA dos participantes?
  • Quantos participantes têm problemas médicos durante o estudo?
  • Existem mudanças na saúde geral dos participantes durante o estudo?
  • Existem mudanças na saúde do coração dos participantes?
  • Existem mudanças na maneira como os participantes se movem pela puberdade? A puberdade é o momento da vida de alguém em que seu corpo muda de uma criança para um adulto.

Os pesquisadores também aprenderão mais sobre:

- Como o corpo processa BIIB141 em crianças e adolescentes

Este estudo será feito da seguinte forma:

  • Os participantes serão selecionados para verificar se podem participar do estudo. O período de triagem será de até 28 dias, após o que os participantes entrarão em seu centro de pesquisa de estudo.
  • Existem 2 partes neste estudo. Durante a Parte 1, os participantes tomarão o BIIB141 ou um placebo uma vez por dia.
  • Na Parte 1, os participantes levarão o BIIB141 ou o placebo em um centro de pesquisa de estudo no dia 1 e depois em visitas pessoais na semana 4, semana 12, semana 26 e semana 52. Em todos os outros dias, eles levarão o BIIB141 ou o placebo em casa. A parte 1 dura até 52 semanas.
  • Durante a Parte 2, os participantes da Parte 1 continuarão tomando BIIB141 ou o iniciarão se estivessem tomando o placebo. A parte 2 durará até 104 semanas.
  • Na Parte 1, os participantes terão até 10 visitas ao seu centro de pesquisa de estudo e um telefonema na semana 2. Na Parte 2, os participantes terão visitas às semanas 4, 8,12, 26, e a cada 26 semanas depois que eles deixarem o estudo e um telefonema na semana 2.
  • Cada participante estará no estudo por até cerca de 3 anos

Visão geral do estudo

Status

Recrutamento

Condições

Intervenção / Tratamento

Descrição detalhada

O objetivo principal do estudo controlado randomizado da Parte 1 (ECR) é avaliar a eficácia da olaveloxolona na semana 52 e os objetivos secundários são avaliar a segurança da olaveloxolona até a semana 52 e a concentração de olaveloxolona após administração de dose única e múltipla. O objetivo primário do estudo de extensão de marcha aberta (OLE) da Parte 2 é avaliar a segurança e a tolerabilidade do uso de olaveloxolona a longo prazo e o objetivo secundário é avaliar a eficácia da olaveloxolona após o uso a longo prazo.

Tipo de estudo

Intervencional

Inscrição (Estimado)

255

Estágio

  • Fase 3

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Contato de estudo

Estude backup de contato

Locais de estudo

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

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

  • Filho

Aceita Voluntários Saudáveis

Não

Descrição

Parte 1 RCT: Critérios de inclusão de chaves:

  • Diagnosticado com a ataxia de Friedreich confirmada geneticamente (FA), isto é, homozigoto para a expansão repetida de guanina-adenina-adenina (GAA) no íntron-1 do gene da frataxina, ou expansão repetida no GAA em 1 alelo e em mutações ou delineações ou outras exagero não-GA.
  • Sintomático para FA, conforme relatado pelo participante e/ou pelos pais/cuidadores a. Crianças de 7 a <16 anos também devem ter uma pontuação na estabilidade vertical (USS) de 10 a ≤ 34 na linha de base

Parte 1 RCT: Critérios de exclusão -chave:

  • Hemoglobina A1C glicosilada (HbA1c)> 11%
  • Peptídeo natriurético do tipo B (BNP)> 200 picogramas por mililitro (PG/ml) na triagem
  • Fração de ejeção (EF) <40% [com base no ecocardiograma (ECHO) realizado na visita de triagem]
  • Doença cardíaca clinicamente significativa, exceto cardiomiopatia leve a moderada

Parte 2 OLE: Critérios de elegibilidade:

  • Os participantes concluíram a Parte 1 do RCT do estudo e nenhum critério de descontinuação foi atendido

  • Os dados de segurança e tolerabilidade da Parte 1 RCT são apoiados à continuação no julgamento do investigador

    1. Se alanina aminotransferase (ALT), aspartato aminotransferase (AST) e/ou bilirrubina total (TBL) são> 2 × limite superior do normal (ULN) na avaliação da visita anterior, a parte 2 do dia 1 deve ser atrasada até que ALT e AST sejam <1,5 × ULN e TBL IS <2 °
    2. Se o BNP for> 200 pg/ml na avaliação da visita anterior, a parte 2 do dia 1 deve ser adiada até que o BNP seja <200 pg/ml
    3. Se qualquer outra anormalidade laboratorial clinicamente significativa estiver presente com base nas avaliações de visita anterior, a Parte 2 do dia 1 deve ser adiada até que as anormalidades sejam resolvidas
    4. No caso de doença intercurrent ou outra mudança no estado de saúde do participante, as avaliações adicionais de triagem da Parte 1 podem ser repetidas antes do início da Parte 2, com base no julgamento do investigador em consulta com o monitor médico

Nota: Outros critérios de inclusão/exclusão definidos por protocolo podem ser aplicados.

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: Randomizado
  • Modelo Intervencional: Atribuição Paralela
  • Mascaramento: Quadruplicar

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
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.
Medicamento: Omaveloxolona
Administrado conforme especificado no braço de tratamento.
Outros nomes:
  • BIIB141, Skyclarys, RTA-408
Comparador de Placebo: Part 1: Placebo
Participants will receive placebo, orally, QD for up to 52 weeks in Part 1 of the study.
Medicamento: Placebo
Administrado conforme especificado no braço de tratamento.
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.
Medicamento: Omaveloxolona
Administrado conforme especificado no braço de tratamento.
Outros nomes:
  • 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.
Medicamento: Omaveloxolona
Administrado conforme especificado no braço de tratamento.
Outros nomes:
  • BIIB141, Skyclarys, RTA-408

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Part 1: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52
Prazo: 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
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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 resultados secundários

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

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Biogen

Investigadores

  • Diretor de estudo: Medical Director, Biogen

Publicações e links úteis

A pessoa responsável por inserir informações sobre o estudo fornece voluntariamente essas publicações. Estes podem ser sobre qualquer coisa relacionada ao estudo.

Links úteis

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Real)

9 de junho de 2025

Conclusão Primária (Estimado)

16 de novembro de 2027

Conclusão do estudo (Estimado)

22 de novembro de 2029

Datas de inscrição no estudo

Enviado pela primeira vez

11 de abril de 2025

Enviado pela primeira vez que atendeu aos critérios de CQ

29 de abril de 2025

Primeira postagem (Real)

1 de maio de 2025

Atualizações de registro de estudo

Última Atualização Postada (Real)

3 de junho de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

2 de junho de 2026

Última verificação

1 de junho de 2026

Mais Informações

Termos relacionados a este estudo

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

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

Plano para dados de participantes individuais (IPD)

Planeja compartilhar dados de participantes individuais (IPD)?

SIM

Descrição do plano IPD

De acordo com a política de transparência e compartilhamento de dados do ensaio clínico da Biogen em https://www.biogentrialtransparency.com/

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Sim

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .

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