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Une étude pour en savoir plus sur les effets et la sécurité à long terme de BIIB141 (Omaveloxolone) chez les participants atteints d'ataxie de Friedreich âgés de 2 à 15 ans (BRAVE)

15 juin 2026 mis à jour par: Biogen

Une phase 3, en 2 parties, randomisées, en double aveugle, étude contrôlée par placebo (partie 1) et extension ouverte (partie 2) pour évaluer l'efficacité, la sécurité, la pharmacocinétique et la pharmacodynamique de l'omaveloxolone (BIIB141) chez les participants avec l'ataxie de Friedreich 2 à <16 ans

Dans cette étude, les chercheurs en apprendront plus sur les effets et la sécurité de BIIB141, également connus sous le nom d'omaveloxolone ou Skyclarys®. Ce médicament a été approuvé ou mis à la disposition des médecins, pour les personnes atteintes d'ataxie de Friedreich (FA) qui ont au moins 16 ans. Mais, il n'est pas encore disponible pour les enfants et les adolescents avec FA qui ont moins de 16 ans. L'objectif principal de cette étude est d'apprendre comment BIIB141 fonctionne dans le corps et sur sa sécurité chez les enfants et les adolescents qui ont 2 à 15 ans.

Les principales questions que les chercheurs souhaitent répondre dans cette étude sont:

  • Comment le BIIB141 affecte-t-il l'équilibre et la stabilité des symptômes de l'AF des participants?
  • Combien de participants ont des problèmes médicaux pendant l'étude?
  • Y a-t-il des changements dans la santé globale des participants au cours de l'étude?
  • Y a-t-il des changements dans la santé cardiaque des participants?
  • Y a-t-il des changements dans la façon dont les participants se déplacent dans la puberté? La puberté est le moment de la vie de quelqu'un où son corps passe d'un enfant à un adulte.

Les chercheurs en apprendront également plus sur:

- Comment le corps traite BIIB141 chez les enfants et les adolescents

Cette étude sera réalisée comme suit:

  • Les participants seront examinés pour vérifier s'ils peuvent rejoindre l'étude. La période de dépistage s'élèvera à 28 jours, après quoi les participants vérifieront leur centre de recherche d'étude.
  • Il y a 2 parties dans cette étude. Pendant la partie 1, les participants prendront soit BIIB141 ou un placebo une fois par jour.
  • Dans la partie 1, les participants prendront le BIIB141 ou le placebo dans un centre de recherche d'étude le jour 1, puis lors de visites en personne à la semaine 4, la semaine 12, la semaine 26 et la semaine 52. Tous les autres jours, ils prendront BIIB141 ou le placebo à la maison. La partie 1 dure jusqu'à 52 semaines.
  • Pendant la partie 2, les participants de la partie 1 continueront de prendre BIIB141 ou de le démarrer s'ils prenaient le lieubo. La partie 2 durera jusqu'à 104 semaines.
  • Dans la partie 1, les participants auront jusqu'à 10 visites dans leur centre de recherche d'étude et un appel téléphonique à la semaine 2. Dans la partie 2, les participants auront des visites aux semaines 4, 8,12, 26 et toutes les 26 semaines après leur départ de l'étude, et un appel téléphonique à la semaine 2. Il y aura un dernier appel téléphonique pour vérifier la santé des participants 31 jours après leur dernière dose.
  • Chaque participant sera dans l'étude jusqu'à environ 3 ans

Aperçu de l'étude

Statut

Recrutement

Les conditions

Intervention / Traitement

Description détaillée

L'objectif principal de la partie 1 essai contrôlé randomisé (ECR) est d'évaluer l'efficacité de l'omaveloxolone à la semaine 52 et les objectifs secondaires sont d'évaluer la sécurité de l'omaveloxolone pendant la semaine 52 et la concentration de l'omaveloxolone après l'administration de dose unique et multiple. L'objectif principal de l'essai d'extension ouverte de la partie 2 (OLE) est d'évaluer l'innocuité et la tolérabilité de l'utilisation à long terme de l'omaveloxolone et l'objectif secondaire est d'évaluer l'efficacité de l'omaveloxolone après une utilisation à long terme.

Type d'étude

Interventionnel

Inscription (Estimé)

255

Phase

  • Phase 3

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Coordonnées de l'étude

Sauvegarde des contacts de l'étude

Lieux d'étude

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

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

  • Enfant

Accepte les volontaires sains

Non

La description

Partie 1 RCT: Critères d'inclusion clés:

  • Diagnostiqué avec une ataxie (FA) de Friedreich génétiquement confirmée, c'est-à-dire homozygote pour la guanine-adénine-adénine (GAA), l'expansion de l'intron-1 du gène de la frataxine, ou une expansion de répétition GAA dans 1 allèle et avec des mutations ponctuelles ou des suppressions, ou d'autres muties d'expansion non GAA dans l'autre alle.
  • Symptomatique pour l'AF, comme indiqué par le participant et / ou le parent / soignant a. Les enfants de 7 à <16 ans doivent également avoir un score de score de stabilité vertical (USS) de 10 à ≤ 34 au départ

Partie 1 RCT: Critères d'exclusion clés:

  • Hémoglobine glycosylée A1C (HbA1c)> 11%
  • Peptide natriurétique de type B (BNP)> 200 picogrammes par millilitre (pg / ml) au dépistage
  • Fraction d'éjection (EF) <40% [sur la base de l'échocardiogramme (écho) effectué lors de la visite de dépistage]
  • Maladie cardiaque cliniquement significative sauf cardiomyopathie légère à modérée

Partie 2 OLE: Critères d'éligibilité:

  • Les participants ont terminé la partie 1 de l'étude et aucun critère d'arrêt n'a été respecté

  • Les données sur la sécurité et la tolérabilité de la partie 1 RCT soutiennent la poursuite dans le jugement de l'enquêteur

    1. Si l'alanine aminotransférase (ALT), l'aspartate aminotransférase (AST) et / ou la bilirubine totale (TBL) sont> 2 × la limite supérieure de la normale (ULN) lors de l'évaluation de la visite précédente, la partie 2 jour 1 doit être retardée jusqu'à
    2. Si le BNP est> 200 pg / ml lors de l'évaluation de la visite précédente, la partie 2 jour 1 doit être retardée jusqu'à ce que le BNP soit <200 pg / ml
    3. Si d'autres anomalies de laboratoire cliniquement significatives sont présentes sur la base des évaluations des visites précédentes, la partie 2 jour 1 devrait être retardée jusqu'à ce que les anomalies soient résolues
    4. En cas de maladie intercurrente ou d'autres changements de l'état de santé du participant, des évaluations de dépistage supplémentaires de la partie 1 peuvent être répétées avant le début de la partie 2, sur la base du jugement de l'enquêteur en consultation avec le moniteur médical

Remarque: D'autres critères d'inclusion / exclusion définis par le protocole peuvent s'appliquer.

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: Randomisé
  • Modèle interventionnel: Affectation parallèle
  • Masquage: Quadruple

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: 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.
Médicament: Omaveloxolone
Administré comme spécifié dans le bras de traitement.
Autres noms:
  • BIIB141, Skyclarys, RTA-408
Comparateur placebo: Part 1: Placebo
Participants will receive placebo, orally, QD for up to 52 weeks in Part 1 of the study.
Médicament: Placebo
Administré comme spécifié dans le bras de traitement.
Expérimental: 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.
Médicament: Omaveloxolone
Administré comme spécifié dans le bras de traitement.
Autres noms:
  • BIIB141, Skyclarys, RTA-408
Expérimental: 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.
Médicament: Omaveloxolone
Administré comme spécifié dans le bras de traitement.
Autres noms:
  • BIIB141, Skyclarys, RTA-408

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Part 1: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52
Délai: 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
Délai: 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)
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Part 1: Change From Baseline in Friedreich's Ataxia-Health Index (FA-HI) at Week 52
Délai: 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
Délai: 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
Délai: 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
Délai: 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)
Délai: 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)
Délai: 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
Délai: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Height at Week 52
Délai: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Weight at Week 52
Délai: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Body Mass Index (BMI) at Week 52
Délai: Baseline, Week 52
Baseline, Week 52
Part 1: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS) at Week 52
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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

Collaborateurs et enquêteurs

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Biogen

Les enquêteurs

  • Directeur d'études: Medical Director, Biogen

Publications et liens utiles

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Liens utiles

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

9 juin 2025

Achèvement primaire (Estimé)

16 novembre 2027

Achèvement de l'étude (Estimé)

22 novembre 2029

Dates d'inscription aux études

Première soumission

11 avril 2025

Première soumission répondant aux critères de contrôle qualité

29 avril 2025

Première publication (Réel)

1 mai 2025

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

16 juin 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

15 juin 2026

Dernière vérification

1 juin 2026

Plus d'information

Termes liés à cette étude

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • 296FA301
  • 2025-520896-13 (Autre identifiant: EU CT Number)

Plan pour les données individuelles des participants (IPD)

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OUI

Description du régime IPD

Conformément à la politique de transparence et de partage des données de Biogen sur les essais cliniques sur https://www.biogenentrialtransparency.com/

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Oui

Étudie un produit d'appareil réglementé par la FDA américaine

Non

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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