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)
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
In this study, researchers will learn more about omaveloxolone, also known as BIIB141 or SKYCLARYS®. Omaveloxolone is already approved for people with Friedreich's Ataxia (FA) who are 16 years of age or older. However, it is not yet available for younger teens and children. The main goal of this study is to learn how omaveloxolone affects symptoms of FA and its safety in younger participants between the ages of 2 and 15 years old.
The main questions researchers want to answer in this study are:
- How does omaveloxolone affect the participants' FA symptoms?
- How does the body process omaveloxolone?
- How many participants have adverse events during the study? Adverse events are health problems that may or may not be caused by the study drug.
- Are there any changes in the participants' overall health or heart health? Researchers will use the modified Friedreich's Ataxia Rating Scale (mFARS) to test nerve function. The mFARS tests movement ability, balance, coordination, speech, and arm and leg functions. More specifically, researchers will check participants' balance, coordination, movement, and ability to perform daily tasks using the Upright Stability Score (USS). The USS is part of the mFARS questionnaire.
Researchers will also use other questionnaires to learn more about participants' symptoms, quality of life, ability to perform daily living activities, and how severe participants think their disease is.
Researchers will also note any changes as participants go through puberty. Some participants may also be invited to take part in interviews about their experiences during the study or wear devices that measure movement and physical activity during daily life.
This study will be done in 2 parts as follows:
- Participants will be screened for up to 4 weeks to check if they can join the study.
- In Part 1, participants will be randomly assigned to take either omaveloxolone or a placebo by mouth once a day for about 1 year. A placebo looks like the study drug but contains no real medicine.
- Part 1 will be double blind. This means that the participants, study doctors, and study staff will not know if the participants are receiving omaveloxolone or a placebo.
- Including screening, participants will have up to 9 clinic visits and 1 phone call during Part 1. If a participant does not join Part 2, they will have another safety follow-up phone call a month after their last dose of omaveloxolone.
- Participants who finish Part 1 may move onto Part 2. During Part 2, participants who are at least 7 years old will be randomly assigned to receive either 150 mg or 250 mg of omaveloxolone once a day for about 104 weeks. Participants younger than 7 years old will receive 150 mg of omaveloxolone once a day for about 104 weeks.
- During Part 2, participants will have up to 8 clinic visits and 1 phone call. Participants will also have a follow-up phone call about a month after they stop taking omaveloxolone.
- In total, participants will have up to 17 clinic visits and 3 phone calls. Each participant will be in the study for up to 3 years.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Description détaillée
The primary objective of Part 1 is to evaluate the efficacy of omaveloxolone as measured by upright stability score (USS) and the secondary objectives are to evaluate the efficacy of omaveloxolone as measured by additional secondary efficacy outcomes, safety of omaveloxolone and the plasma concentration of omaveloxolone after single and multiple dose administration.
The primary objective of Part 2A is to evaluate the efficacy of omaveloxolone and the secondary objectives are to characterize the efficacy of omaveloxolone as measured by additional secondary outcomes, evaluate the safety and tolerability of omaveloxolone and plasma concentration of omaveloxolone after single and multiple dose administration.
The primary objective for Part 2B is to evaluate the safety and tolerability of long-term omaveloxolone use and the secondary objective is to evaluate the efficacy of omaveloxolone following long-term use.
Type d'étude
Inscription (Estimé)
Phase
- Phase 3
Contacts et emplacements
Lieux d'étude
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Giessen, Allemagne, 35392
- UKGM - Universitätsklinikum Giessen und Marburg GmbH - Standort Gießen
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Hamburg, Allemagne, 20246
- Universitätsklinikum Hamburg Eppendorf
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North Rhine-Westphalia
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Aachen, North Rhine-Westphalia, Allemagne, 52074
- Universitätsklinikum Aachen
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Ar Riya
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Riyadh, Ar Riya, Arabie Saoudite, 12875
- King Faisal Specialist Hospital & Research Centre
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New South Wales
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Randwick, New South Wales, Australie, 2031
- Sydney Children's Hospital
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Victoria
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Parkville, Victoria, Australie, 3052
- Murdoch Childrens Research Institute (MCRI)
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Federal District
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Brasília, Federal District, Brésil, 70200-730
- L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME
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São Paulo
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Campinas, São Paulo, Brésil, 13083-970
- University of Campinas (UNICAMP) School of Medical Sciences
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São Paulo, São Paulo, Brésil, 04024-002
- PSEG Centro de Pesquisa Clinica
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Quebec
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Montreal, Quebec, Canada, H3H 2R9
- McGill University
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Québec, Quebec, Canada, G1V 4G2
- CHU de Quebec -Universite Laval
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Copenhagen, Danemark, 2100
- Rigshospitalet - Juliane Marie Centret (JMC) Copenhagen
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Madrid, Espagne, 28046
- Hospital Universitario La Paz - PPDS
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Barcelona
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Espluges de Llobregat, Barcelona, Espagne, 8950
- Hospital Sant Joan de Deu - PIN
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Paris, France, 75012
- AP-HP - Hôpital Armand Trousseau
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Hérault
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Montpellier, Hérault, France, 34090
- CHU de Montpellier- Hôpital Gui De Chauliac
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National Capital Territory of Delhi
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New Delhi, National Capital Territory of Delhi, Inde, 110029
- All India Institute of Medical Sciences (AIIMS) - New Delhi
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Dublin, Irlande, D01 XD99
- CHI at Temple Street
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Milan, Italie, 20133
- Fondazione IRCCS Istituto Neurologico Carlo Besta
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Lazio
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Rome, Lazio, Italie, 165
- Ospedale Pediatrico Bambino Gesù IRCCS
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Veneto
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Conegliano, Veneto, Italie, 31015
- IRCCS Eugenio Medea - Polo. Scientifico Veneto
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Innsbruck, L'Autriche, 6020
- Universitätsklinikum Innsbruck
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Nijmegen, Pays-Bas, 6525 GA
- Radboud Universitair Medisch Centrum
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Lincolnshire
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London, Lincolnshire, Royaume-Uni, NW1 2BU
- University College Hospital - PPDS
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Oxfordshire
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Oxford, Oxfordshire, Royaume-Uni, OX3 9DU
- John Radcliffe Hospital
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South Yorkshire
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Sheffield, South Yorkshire, Royaume-Uni, S10 5DD
- Sheffield Children's Hospital - PPDS
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Istanbul, Turquie (Türkiye), 34093
- Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
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California
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Los Angeles, California, États-Unis, 90095
- UCLA Neurology Outpatient Clinic at Westwood
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Florida
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Gainesville, Florida, États-Unis, 32610-3010
- Norman Fixel Institute for Neurological Diseases UF Health
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Tampa, Florida, États-Unis, 33612
- USF Health Morsani College of Medicine Department of Neurology
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Pennsylvania
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Philadelphia, Pennsylvania, États-Unis, 19104
- Children's Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - PIN
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Tennessee
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Memphis, Tennessee, États-Unis, 38105-3678
- St. Jude Children's Research Hospital - PIN
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Virginia
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Norfolk, Virginia, États-Unis, 23507-1910
- CHKD's Health Center - South Campus - PIN
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Washington
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Seattle, Washington, États-Unis, 98105-3901
- Seattle Children's Hospital
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Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
Accepte les volontaires sains
Sexes éligibles pour l'étude
La description
Part 1: Key inclusion criteria:
- Diagnosed with genetically confirmed Friedreich's Ataxia (FA), i.e., homozygous for guanine-adenine-adenine (GAA) repeat expansion in intron-1 of the frataxin gene, or GAA repeat expansion in 1 allele and with point mutations or deletions, or other non-GAA expansion mutations in the other allele.
- Symptomatic for FA as confirmed by clinician assessment. a. Children 7 to < 16 years must also have an upright stability score (USS) score of 10 to ≤ 34 at baseline
Part 1: Key exclusion criteria:
- Glycosylated hemoglobin A1C (HbA1c) > 11%
- B-type natriuretic peptide (BNP) > 200 picograms per milliliter (pg/mL) at screening
- Ejection fraction (EF) < 40% [based on echocardiogram (ECHO) performed at screening visit]
- Clinically significant cardiac disease except mild to moderate cardiomyopathy
Part 2A: Eligibility criteria:
- They have completed Part 1 of the study and no discontinuation criteria have been met.
- Safety and tolerability data from Part 1 are supportive of continuation in the judgement of the investigator.
Part 2B: Eligibility criteria:
- Participants have completed Part 1 of the study and no discontinuation criteria have been met.
- Safety and tolerability data from Part 1 are supportive of continuation in the judgement of the Investigator.
Note: Other protocol-defined Inclusion/Exclusion criteria may apply.
Plan d'étude
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 |
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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. |
Administered as specified in the treatment arm.
Autres noms:
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Comparateur placebo: Part 1: Placebo
Participants will receive placebo, orally, QD for up to 52 weeks in Part 1 of the study. |
Administered as specified in the treatment arm. |
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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. |
Administered as specified in the treatment arm.
Autres noms:
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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. |
Administered as specified in the treatment arm.
Autres noms:
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Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
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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)
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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)
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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
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Baseline (Week 52 of Part 1), Weeks 52 and 104
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Part 2B: Change From Baseline in Height at Weeks 52 and Week 104
Délai: Baseline (Week 52 of Part 1), Weeks 52 and 104
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Baseline (Week 52 of Part 1), Weeks 52 and 104
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Part 2B: Change From Baseline in Weight at Weeks 52 and Week 104
Délai: Baseline (Week 52 of Part 1), Weeks 52 and 104
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Baseline (Week 52 of Part 1), Weeks 52 and 104
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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
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Baseline (Week 52 of Part 1), Weeks 52 and 104
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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
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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
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Part 2A: Change From Baseline in USS Subscale E of mFARS at Week 52
Délai: Baseline (Week 52 of Part 1), Week 52
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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
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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
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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
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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
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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
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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
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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
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Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
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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)
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From first dose of study drug up to end of follow up period in Part 1 (up to Week 52)
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Part 1: Number of Participants With Change From Baseline in Cardiac Function Assessed by ECHO at Week 52
Délai: Baseline, Week 52
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Baseline, Week 52
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Part 1: Change From Baseline in Height at Week 52
Délai: Baseline, Week 52
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Baseline, Week 52
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Part 1: Change From Baseline in Weight at Week 52
Délai: Baseline, Week 52
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Baseline, Week 52
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Part 1: Change From Baseline in Body Mass Index (BMI) at Week 52
Délai: Baseline, Week 52
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Baseline, Week 52
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Part 1: Plasma Concentrations of Omaveloxolone
Délai: Pre-dose and post-dose on Day 1, Weeks 4, 12 and 26
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Pre-dose and post-dose on Day 1, Weeks 4, 12 and 26
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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)
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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)
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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
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Baseline (Week 52 of Part 1), Weeks 52 and 104
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Part 2A: Change From Baseline in Height at Weeks 52 and Week 104
Délai: Baseline (Week 52 of Part 1), Weeks 52 and 104
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Baseline (Week 52 of Part 1), Weeks 52 and 104
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Part 2A: Change From Baseline in Weight at Weeks 52 and Week 104
Délai: Baseline (Week 52 of Part 1), Weeks 52 and 104
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Baseline (Week 52 of Part 1), Weeks 52 and 104
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Part 2A: Change From Baseline in BMI at Weeks 52 and Week 104
Délai: Baseline (Week 52 of Part 1), Weeks 52 and 104
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Baseline (Week 52 of Part 1), Weeks 52 and 104
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Part 2A: Plasma Concentrations of Omaveloxolone
Délai: Pre-dose and post-dose on Day 1, Weeks 4, 12 and 26
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Pre-dose and post-dose on Day 1, Weeks 4, 12 and 26
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Part 1: Change From Baseline in Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52
Délai: Baseline, Week 52
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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
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Part 1: Change From Baseline in Friedreich's Ataxia-Health Index (FA-HI) at Week 52
Délai: Baseline, Week 52
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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
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Part 1: Change From Baseline in Friedreich's Ataxia-Activities of Daily Living (FA-ADL)
Délai: Baseline, Week 52
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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
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Part 1: Change From Baseline in Clinical Global Impressions-Severity (CGI-S) at Week 52
Délai: Baseline, Week 52
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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
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Part 1: Change From Baseline in Patient Global Impressions-Severity (PGI-S) at Week 52
Délai: Baseline, Week 52
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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
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Part 1: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS) at Week 52
Délai: Baseline, Week 52
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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
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Part 1: Percentage of Participants at Each Tanner Stage at Week 52
Délai: Baseline, Week 52
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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
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Part 1: Number of Participants at Each Tanner Stage at Week 52
Délai: Baseline, Week 52
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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
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Part 2A: Change From Baseline in USS Subscale E of mFARS at Week 104
Délai: Baseline (Week 52 of Part 1), Week 104
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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
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Part 2A: Change from baseline in mFARS at Weeks 52 and Week 104
Délai: Baseline (Week 52 of Part 1), Weeks 52 and 104
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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
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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
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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
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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
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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
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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
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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
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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
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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
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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
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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
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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
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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
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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
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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
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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
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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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Collaborateurs et enquêteurs
Parrainer
Les enquêteurs
- Directeur d'études: Medical Director, Biogen
Publications et liens utiles
Liens utiles
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Réel)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
- Maladies du cerveau
- Maladies du système nerveux central
- Maladies du système nerveux
- Maladies génétiques, innées
- Maladies métaboliques
- Maladies neurodégénératives
- Troubles hérédodégénératifs, système nerveux
- Maladies de la moelle épinière
- Maladies mitochondriales
- Maladies cérébelleuses
- Dégénérescences spinocérébelleuses
- Maladies et anomalies congénitales, héréditaires et néonatales
- Maladies nutritionnelles et métaboliques
- Ataxie de Friedreich
- omaveloxolone
Autres numéros d'identification d'étude
- 296FA301
- 2025-520896-13 (Autre identifiant: EU CT Number)
- 2025 (Subvention/contrat des NIH des États-Unis)
Plan pour les données individuelles des participants (IPD)
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Description du régime IPD
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Informations sur les médicaments et les dispositifs, documents d'étude
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