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一项研究，以了解更多有关BIIB141（Omaveloxolone）对2至15岁弗里德里奇共济失调参与者的影响和长期安全性的研究 (BRAVE)

2026年6月15日 更新者：Biogen

第3阶段，2部分，随机，双盲，安慰剂对照研究（第1部分）和开放标签扩展（第2部分），以评估Omaveloxolone（BiiB141）在弗里德里希（Friedreich

在这项研究中，研究人员将更多地了解BIIB141的效果和安全性，也称为Omaveloxolone或Skyclarys®。 该药物已被批准，或者可以供医生开处方，供弗里德里希共济失调（FA）至少16岁的弗里德里希（Fa）的人开处方。 但是，尚不适用于16岁以下的FA儿童和青少年。 这项研究的主要目的是了解BIIB141在2至15岁的儿童和青少年中如何在体内工作及其安全性。

研究人员希望在这项研究中回答的主要问题是：

  • BIIB141如何影响参与者的FA症状平衡和稳定性？
  • 在研究期间有多少参与者有医疗问题？
  • 在研究期间，参与者的整体健康状况是否有任何变化？
  • 参与者的心脏健康有什么变化吗？
  • 参与者如何进入青春期有什么变化吗？ 青春期是某人一生中的身体从孩子变成成人的时候。

研究人员还将了解更多有关：

- 身体如何处理儿童和青少年的BIIB141

这项研究将如下：

  • 参与者将被筛选以检查他们是否可以加入研究。 筛查期最长为28天，之后，参与者将检查其研究中心。
  • 这项研究有2个部分。 在第1部分期间，参与者每天将服用BIIB141或安慰剂。
  • 在第1部分中，参与者将在第1天将BIIB141或安慰剂在研究中心中服用，然后在第4周，第12周，第26周和第52周进行面对面访问。 在其他所有日子里，他们将在家中使用BiiB141或安慰剂。 第1部分持续长达52周。
  • 在第2部分中，第1部分的参与者将继续服用BiiB141，或者如果他们服用安慰剂，则将开始。 第2部分将持续长达104周。
  • 在第1部分中，参与者将最多10次访问他们的研究研究中心，并在第2周进行电话。在第2部分中，参与者将在第4、812、26周和之后的每26周进行访问，直到他们离开研究，并在第2周离开研究。将在第2周打电话。在他们的最后31天后，将在最后31天内打电话。
  • 每个参与者将在研究中长达3年

研究概览

地位

招聘中

条件

干预/治疗

详细说明

第1部分随机对照试验（RCT）的主要目标是评估第52周Omaveloxolone的功效，次要目标是通过第52周评估Omaveloxolone的安全性，并在单次和多剂量给药后评估Omaveloxolone的浓度。 第2部分开放标签扩展（OLE）试验的主要目标是评估长期使用Omaveloxolone的安全性和耐受性，次要目标是评估长期使用后Omaveloxolone的功效。

研究类型

介入性

注册 (估计的)

255

阶段

  • 第三阶段

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习联系方式

研究联系人备份

学习地点

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

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 孩子

接受健康志愿者

不

描述

第1部分RCT：关键纳入标准：

  • 被诊断为遗传确认的弗里德里希共济失调（FA），即在Frataxin Gene基因内含​​子-1中重复扩展鸟嘌呤 - 腺嘌呤 - 腺嘌呤（GAA），或1等位基因中的GAA重复扩展，以及点突变或其他非GAA膨胀，或其他非GAA膨胀等等。
  • 参与者和/或父/照顾者报告的FA有症状。 7至<16岁的儿童还必须在基线时具有直立的稳定分数（US）分数10至≤34

第1部分RCT：关键排除标准：

  • 糖基化血红蛋白A1C（HBA1C）> 11％
  • 筛查时B型Natriaret肽（BNP）>每毫升200丝（Pg/ml）
  • 射血分数（EF）<40％[基于筛选访问时进行的超声心动图（Echo）]
  • 临床意义的心脏疾病，除了轻度至中度心肌病

第2部分OLE：资格标准：

  • 参与者已经完成了研究的第1部分RCT，尚未满足停止标准

  • 第1部分RCT的安全性和耐受性数据支持调查员的判断中的延续

    1. 如果丙氨酸氨基转移酶（ALT），天冬氨酸氨基转移酶（AST）和/或总胆红素（TBL）在先前的访问评估时> 2倍正常（ULN）的上限> 2×上限，则第2天应延迟第2天，直到ALT和AST为<1.5×ULN，TBL为<2×ULN，
    2. 如果BNP在上一次访问评估中> 200 pg/ml，则应延迟第2部分，直到BNP <200 pg/ml
    3. 如果根据先前的访问评估存在任何其他具有临床意义的实验室异常，则应延迟第2部分，直到解决异常
    4. 如果参与者的健康状况发生互换或其他变化，则基于调查员在与医疗监护仪协商时的判断之前，可以在第2部分启动之前重复其他第1部分筛选评估

注意：其他协议定义的包含/排除标准可能适用。

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：随机化
  • 介入模型：并行分配
  • 屏蔽：四人间

武器和干预

参与者组/臂
干预/治疗
实验性的：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.
药品：奥马维洛酮
按照治疗组中的规定进行给药。
其他名称：
  • BIIB141，Skyclarys，RTA-408
安慰剂比较：Part 1: Placebo
Participants will receive placebo, orally, QD for up to 52 weeks in Part 1 of the study.
药品：安慰剂
按照治疗组的规定给药。
实验性的：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.
药品：奥马维洛酮
按照治疗组中的规定进行给药。
其他名称：
  • BIIB141，Skyclarys，RTA-408
实验性的：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.
药品：奥马维洛酮
按照治疗组中的规定进行给药。
其他名称：
  • BIIB141，Skyclarys，RTA-408

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Part 1: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52
大体时间：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
大体时间：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)
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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

次要结果测量

结果测量
措施说明
大体时间
Part 1: Change From Baseline in Friedreich's Ataxia-Health Index (FA-HI) at Week 52
大体时间：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
大体时间：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
大体时间：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
大体时间：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)
大体时间：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)
大体时间：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
大体时间：Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Height at Week 52
大体时间：Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Weight at Week 52
大体时间：Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Body Mass Index (BMI) at Week 52
大体时间：Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS) at Week 52
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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

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调查人员

  • 研究主任：Medical Director、Biogen

出版物和有用的链接

负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。

有用的网址

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2025年6月9日

初级完成 (估计的)

2027年11月16日

研究完成 (估计的)

2029年11月22日

研究注册日期

首次提交

2025年4月11日

首先提交符合 QC 标准的

2025年4月29日

首次发布 (实际的)

2025年5月1日

研究记录更新

最后更新发布 (实际的)

2026年6月16日

上次提交的符合 QC 标准的更新

2026年6月15日

最后验证

2026年6月1日

更多信息

与本研究相关的术语

其他相关的 MeSH 术语

其他研究编号

  • 296FA301
  • 2025-520896-13 (其他标识符：EU CT Number)

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

是的

IPD 计划说明

根据Biogen在https://www.biogentrialtransparency.com/上的临床试验透明度和数据共享政策

药物和器械信息、研究文件

研究美国 FDA 监管的药品

是的

研究美国 FDA 监管的设备产品

不

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