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Étude de phase 3 sur l'efficacité et l'innocuité de BG00012 chez des sujets pédiatriques atteints de sclérose en plaques récurrente-rémittente (SEP-RR) (CONNECT)

16 mai 2026 mis à jour par: Biogen

Étude ouverte, randomisée, multicentrique, à doses multiples, à contrôle actif, en groupes parallèles, d'efficacité et d'innocuité de BG00012 chez les enfants de 10 à moins de 18 ans atteints de sclérose en plaques récurrente-rémittente, avec extension ouverte facultative

Les principaux objectifs de la partie 1 sont les suivants : évaluer l'innocuité, la tolérabilité et l'efficacité de BG00012 chez des sujets pédiatriques atteints de SEP-RR, par rapport à un traitement modificateur de la maladie, et évaluer les résultats pour la santé et l'évolution du handicap. L'objectif principal de la partie 2 est d'évaluer l'innocuité à long terme de BG00012 chez les sujets qui ont terminé la semaine 96 de la partie 1 de l'étude 109MS306. L'objectif secondaire de la partie 2 est de décrire les résultats à long terme de la SEP de BG00012 chez les sujets qui ont terminé la semaine 96 de la partie 1 de l'étude 109MS306.

Aperçu de l'étude

Statut

Complété

Les conditions

Intervention / Traitement

Type d'étude

Interventionnel

Inscription (Réel)

156

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.

Lieux d'étude

  • Allemagne
    • Bavaria
      • Augsburg, Bavaria, Allemagne, 86156
        • Universitaetsklinikum Augsburg
      • Munich, Bavaria, Allemagne, 80337
        • Klinikum der Universitaet Muenchen
    • North Rhine-Westphalia
      • Bochum, North Rhine-Westphalia, Allemagne, 44791
        • Katholisches Klinikum Bochum gGmbH
  • Belgique
      • Brussels, Belgique, 1020
        • Universitair Kinderziekenhuis Koningin Fabiola
      • Ghent, Belgique, 9000
        • Universitair Ziekenhuis Ghent
  • Bulgarie
      • Sofia, Bulgarie, 1113
        • MHATNP 'Sv.Naum', EAD
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T3B 6A8
        • University of Calgary - Alberta Children's Hospital
  • Danemark
      • Aarhus N, Danemark, 8200
        • Århus Universitetshospital
      • Copenhagen, Danemark, 2100
        • Rigshospitalet
      • Odense, Danemark, 5000
        • Odense Universitetshospital
  • Espagne
      • Seville, Espagne, 41009
        • Hospital Universitario Virgen Macarena
    • Barcelona
      • Esplugues de Llobregat, Barcelona, Espagne, 8950
        • Hospital Sant Joan de Déu
    • Córdoba
      • Córdoba, Córdoba, Espagne, 14011
        • Hospital Universitario Reina Sofia
    • Madrid
      • Torrejón de Ardoz, Madrid, Espagne, 28850
        • Hospital Universitario De Torrejon
  • France
    • Bas Rhin
      • Strasbourg, Bas Rhin, France, 67098
        • CHU Strasbourg - Hôpital Hautepierre
    • Bouches-du-Rhône
      • Marseille, Bouches-du-Rhône, France, 13385
        • Hôpital de la Timone
    • Côte-d'Or
      • Dijon, Côte-d'Or, France, 21079
        • CHU Dijon -BOCAGE CENTRAL
    • Herault
      • Montpellier, Herault, France, 34295
        • Hôpital Gui de Chauliac
    • Ille Et Vilaine
      • Rennes, Ille Et Vilaine, France, 35033
        • CHU Rennes - Hôpital Pontchaillou
    • Meurthe Et Moselle
      • Vandœuvre-lès-Nancy, Meurthe Et Moselle, France, 54500
        • Hôpital de Brabois Enfants
    • Nord
      • Lille, Nord, France, 59037
        • Hopital Roger Salengro - CHU Lille
    • Puy De Dome
      • Clermont-Ferrand, Puy De Dome, France, 63003
        • CHU Clermont Ferrand - Hopital d'Estaing
    • Rhone
      • Bron, Rhone, France, 69677
        • Hopital Neurologique Pierre Wertheimer
    • Somme
      • Amiens, Somme, France, 80054
        • CHU Amiens - Hopital Sud
    • Val De Marne
      • Le Kremlin-Bicêtre, Val De Marne, France, 94275
        • Hôpital Bicêtre
  • Hongrie
      • Budapest, Hongrie, 1083
        • Semmelweis Egyetem
      • Budapest, Hongrie, 1089
        • Heim Pal Orszagos Gyermekgyogyaszati Intezet
  • Israël
      • Jerusalem, Israël, 91120
        • Hadassah University Hospital - Ein Kerem
      • Petah Tikva, Israël, 4920235
        • Schneider Children's Medical Center
      • Ramat Gan, Israël, 5265601
        • Chaim Sheba Medical Center
  • Italie
      • Bari, Italie, 70124
        • Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
      • Genova, Italie, 16132
        • IRCCS Ospedale Policlinico San Martino
      • Milan, Italie, 20132
        • Ospedale San Raffaele
      • Naples, Italie, 80131
        • Azienda Ospedaliera Universitaria 'Federico II'
      • Padova, Italie, 35128
        • Azienda Ospedale-Università di Padova
      • Palermo, Italie, 90127
        • Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone
      • Roma, Italie, 00189
        • Azienda Ospedaliera Sant'Andrea-Università di Roma La Sapienza
      • Roma, Italie, 00165
        • Ospedale Pediatrico Bambino Gesu
    • Varese
      • Gallarate, Varese, Italie, 21013
        • Azienda Socio Sanitaria Territoriale della Valle Olona (presidio di Gallarate)
  • Koweit
      • Ash Shuwaykh, Koweit, 12345
        • Ibn Sina Hospital
  • Pologne
      • Bialystok, Pologne, 15-274
        • SPZOZ Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa
      • Gdansk, Pologne, 80-211
        • Uniwersyteckie Centrum Kliniczne
      • Lodz, Pologne, 93-338
        • Instytut Centrum Zdrowia Matki Polki
      • Poznan, Pologne, 60-355
        • Szpital Kliniczny im.Heliodora Swiecickiego Uniwersytetu Medycznego im.K. Marcinkowskiego w Poznaniu
      • Warsaw, Pologne, 04-730
        • Instytut 'Pomnik - Centrum Zdrowia Dziecka'
  • Royaume-Uni
    • Greater London
      • London, Greater London, Royaume-Uni, WC1N 3BG
        • The National Hospital for Neurology & Neurosurgery
      • London, Greater London, Royaume-Uni, WC1N 3JH
        • Great Ormond Street Hospital for Children
      • London, Greater London, Royaume-Uni, SE1 7EH
        • Evelina London Children's Hospital
    • West Midlands
      • Birmingham, West Midlands, Royaume-Uni, B4 6NH
        • Birmingham Children's Hospital
  • Serbie
      • Belgrade, Serbie, 11000
        • Clinic of Neurology and Psychiatry for Children and Youth
      • Belgrade, Serbie, 11000
        • Mother and Child Health Care Institute of Serbia ,,Dr Vukan Cupic''
      • Kragujevac, Serbie, 34000
        • University Clinical Center Kragujevac
  • Suède
      • Gothenburg, Suède, 41345
        • Sahlgrenska Sjukhuset
      • Stockholm, Suède, SE-113 61
        • Karolinska
  • Tchéquie
      • Brno, Tchéquie, 656 91
        • Fakultni nemocnice u sv. Anny v Brne
      • Hradec Králové, Tchéquie, 500 05
        • Fakultni nemocnice Hradec Kralove
      • Jihlava, Tchéquie, 58633
        • Nemocnice Jihlava p.o.
      • Ostrava, Tchéquie, 708 52
        • Fakultni Nemocnice Ostrava
  • Turquie (Türkiye)
      • Ankara, Turquie (Türkiye), 06100
        • Hacettepe University Medical Faculty
      • Antalya, Turquie (Türkiye), 07070
        • Akdeniz University Faculty of Medicine
  • États-Unis
    • Massachusetts
      • Boston, Massachusetts, États-Unis, 2115
        • Boston Children's Hospital
    • Virginia
      • Charlottesville, Virginia, États-Unis, 22903
        • The Rector and Visitors of the University of Virginia

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

10 ans à 17 ans (Enfant)

Accepte les volontaires sains

Non

La description

Critères d'inclusion clés :

  • Doit avoir un poids corporel ≥ 30 kg.
  • Doit avoir un diagnostic de SEP-RR (définition consensuelle de la SEP-RR pédiatrique [Krupp 2007]).
  • Doit être ambulatoire avec un score EDSS de base compris entre 0 et 5,5 inclus.
  • Doit avoir subi au moins 1 des 3 conditions suivantes : a) au moins 1 rechute au cours des 12 derniers mois précédant le jour 1 avec une IRM cérébrale antérieure démontrant des lésions compatibles avec la SEP ; b) au moins 2 rechutes au cours des 24 derniers mois précédant le jour 1, avec une IRM cérébrale antérieure démontrant des lésions compatibles avec la SEP ; c) preuve de lésions cérébrales rehaussées par le Gd sur une IRM réalisée dans les 6 semaines précédant le jour 1.
  • Doit être neurologiquement stable, sans signe de rechute dans les 50 jours précédant le jour 1 et aucun signe de traitement aux corticostéroïdes dans les 30 jours précédant le jour 1.
  • - Les sujets en âge de procréer qui sont sexuellement actifs doivent être disposés à pratiquer une contraception efficace pendant l'étude et être disposés et capables de continuer la contraception pendant au moins 30 jours après leur dernière dose de traitement à l'étude.

Critères d'exclusion clés :

  • SEP primaire progressive, secondaire progressive ou progressive récurrente (telle que définie par [Lublin et Reingold 1996]). Ces conditions nécessitent la présence d'une maladie clinique continue s'aggravant sur une période d'au moins 3 mois. Les sujets atteints de ces affections peuvent également avoir des rechutes superposées mais se distinguent des sujets récurrents-rémittents par l'absence de périodes cliniquement stables ou d'amélioration clinique.
  • Troubles imitant la SEP, tels que d'autres troubles démyélinisants (par exemple, l'encéphalomyélite aiguë disséminée), les troubles auto-immuns systémiques (par exemple, la maladie de Sjögren, le lupus érythémateux), les troubles métaboliques (par exemple, les dystrophies) et les troubles infectieux.
  • Antécédents de maladie précancéreuse ou maligne. Les sujets atteints d'un carcinome basocellulaire qui a été complètement excisé avant le dépistage resteront éligibles.
  • Antécédents de réactions allergiques ou anaphylactiques graves, ou hypersensibilité médicamenteuse connue au DMF, aux esters d'acide fumarique ou à l'interféron bêta-1a (IFN bêta-1a).
  • Antécédents de résultats de laboratoire anormaux indiquant toute maladie endocrinologique, hématologique, hépatique, immunologique, métabolique, urologique, rénale et/ou toute autre maladie majeure qui empêcherait la participation à une étude clinique.

  • Antécédents de maladies cardiovasculaires, pulmonaires, gastro-intestinales, dermatologiques, de croissance, de développement, psychiatriques (y compris la dépression), neurologiques (autres que la SEP) et/ou d'autres maladies majeures cliniquement significatives qui empêcheraient la participation à une étude clinique.

    -.Histoire du virus de l'immunodéficience humaine.

  • Une rechute de SEP qui s'est produite dans les 50 jours précédant le Jour 1 ET/OU le sujet ne s'est pas stabilisé depuis une rechute précédente avant le Jour 1.
  • Autres raisons non précisées qui, de l'avis de l'investigateur ou de Biogen Idec, rendent le sujet impropre à l'inscription.
  • Pour le sous-ensemble de sujets PD/PK : sujets incapables d'avaler la capsule BG00012 entière.

Clé Historique du traitement

  • Tout traitement antérieur avec Fumaderm (esters d'acide fumarique) ou BG00012.
  • Traitement antérieur par l'un des éléments suivants : irradiation lymphoïde totale, cladribine, vaccination contre les lymphocytes T ou les récepteurs des lymphocytes T, tout anticorps monoclonal thérapeutique, à l'exception du rituximab ou du natalizumab.
  • Traitement antérieur avec l'un des médicaments suivants dans les 12 mois précédant le jour 1 : mitoxantrone, cyclophosphamide, rituximab.
  • Traitement antérieur avec l'un des médicaments ou procédures suivants dans les 6 mois précédant le jour 1 : fingolimod ; tériflunomide; le natalizumab ; ciclosporine; l'azathioprine; méthotrexate; mycophénolate mofétil; laquinimod; immunoglobuline intraveineuse (IV); plasmaphérèse ou cytaphérèse
  • Traitement avec l'un des médicaments suivants dans les 30 jours précédant le jour 1 : stéroïdes (corticothérapie par voie intraveineuse ou orale, y compris les agents qui peuvent ne pas agir par la voie des corticostéroïdes [par exemple, naltrexone à faible dose]), 4-aminopyridine ou produits apparentés (à l'exception des sujets sous dose stable de fampridine à libération contrôlée pendant 3 mois)

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: Aucun (étiquette ouverte)

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: BG00012
Les participants recevront la dose recommandée de 240 mg par voie orale, deux fois par jour
Médicament: fumarate de diméthyle
administré par voie orale
Autres noms:
  • BG00012
  • Tecfidera
Comparateur actif: IFN β-1a (Avonex)
Les participants recevront la dose recommandée de 30 μg (hebdomadaire)
Médicament: Interféron β-1a
administré par injection intramusculaire
Autres noms:
  • Avonex

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Part 1: Proportion of Participants Free of New or Newly Enlarging T2 Hyperintense Lesions on Brain Magnetic Resonance Imaging (MRI) Scans
Délai: At Week 96
Participants who were free of new or newly enlarging T2 hyperintense lesions were assessed on Brain MRI scans.
At Week 96
Part 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)
Délai: From Week 96 up to last follow-up visit (up to Week 340)
An adverse event (AE) was any unfavorable and unintended sign (including an abnormal assessment such as an abnormal laboratory value), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE was any untoward medical occurrence that at any dose resulted in death, in the view of the Investigator, placed the participant at immediate risk of death, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in a birth defect. TEAEs were defined as AEs occurring or worsening after beginning study treatment (after the first dose).
From Week 96 up to last follow-up visit (up to Week 340)
Part 2: Number of Participants Who Discontinued Study Treatment Due to an AE
Délai: From Week 96 up to last follow-up visit (up to Week 340)
An AE was any unfavorable and unintended sign (including an abnormal assessment such as an abnormal laboratory value), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
From Week 96 up to last follow-up visit (up to Week 340)

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Part 1: Number of New or Newly Enlarged T2 Hyperintense Lesions on Brain MRI Scans
Délai: At Weeks 24 and Week 96
The number of new or newly enlarging T2 hyperintense lesions that developed in each participant was assessed on Brain MRI scans.
At Weeks 24 and Week 96
Part 1: Proportion of Participants Free of New or Newly Enlarging T2 Hyperintense Lesions on Brain MRI Scans
Délai: At Weeks 24 and 48
Participants who were free of new or newly enlarging T2 hyperintense lesions were assessed on Brain MRI scans.
At Weeks 24 and 48
Part 1: Proportion of Participants Free of New MRI Activity as Measured by Brain MRI Scans
Délai: At Weeks 24, 48, and 96
Participants free of Gd-enhancing MRI lesions and new or newly enlarging T2 MRI lesions were assessed on Brain MRI Scans.
At Weeks 24, 48, and 96
Part 1: Time to First Relapse
Délai: Up to Week 96
Relapses are defined as new or recurrent neurologic symptoms not associated with fever or infection, lasting at least 24 hours, and accompanied by new objective neurological findings upon examination by the examining neurologist. The time to first relapse was defined as the time from the first dose in Part 1 up to the first relapse. Time to First Relapse was estimated by Kaplan-Meier method.
Up to Week 96
Part 1: Proportion of Relapse-Free Participants
Délai: Up to Week 96
Relapses are defined as new or recurrent neurologic symptoms not associated with fever or infection, lasting at least 24 hours, and accompanied by new objective neurological findings upon examination by the examining neurologist. The proportion of relapse-free participants was estimated using the Kaplan-Meier method.
Up to Week 96
Part 1: Annualized Relapse Rate (ARR)
Délai: At Weeks 48 and 96
ARR is calculated as the total number of relapses that occurred during the study divided by the total number of participant-years. ARR was analyzed using negative binomial regression model.
At Weeks 48 and 96
Part 1: Number of Participants With TEAEs and TESAEs
Délai: From Day 1 up to Week 96
An AE was any unfavorable and unintended sign (including an abnormal assessment such as an abnormal laboratory value), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE was any untoward medical occurrence that at any dose resulted in death, in the view of the Investigator, placed the participant at immediate risk of death, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in a birth defect. TEAEs were defined as AEs occurring or worsening after beginning study treatment (after the first dose).
From Day 1 up to Week 96
Part 1: Change From Baseline in Vital Signs (Temperature)
Délai: Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84 and 96
Negative change from baseline indicated reduction in temperature.
Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84 and 96
Part 1: Change From Baseline in Vital Signs (Pulse Rate)
Délai: Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84 and 96
Negative change from baseline indicated reduction in pulse rate.
Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84 and 96
Part 1: Change From Baseline in Vital Signs (Blood Pressure)
Délai: Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84 and 96
Negative change from baseline indicated reduction in blood pressure.
Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84 and 96
Part 1: Change From Baseline in Vital Signs (Respiratory Rate)
Délai: Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84 and 96
Negative change from baseline indicated reduction in respiratory rate.
Baseline, Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84 and 96
Part 1: Number of Participants With Shifts From Baseline in Electrocardiograms (ECG) Abnormalities
Délai: Up to Week 96
Clinical significance of abnormalities in ECG was determined based on the investigator's discretion. Shift to abnormal indicated values that were normal or unknown at baseline and shifted to abnormal values post-baseline.
Up to Week 96
Part 1: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Hematology Parameters)
Délai: Baseline up to Week 96
Hematology parameters included leukocytes, erythrocytes, hemoglobin, hematocrit, platelets, eosinophils, lymphocytes, monocytes and neutrophils. These parameters were flagged as low, normal, or high relative to parameter's normal range or as unknown if no result was available. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicated values that were normal, low or unknown at baseline and shifted to high postbaseline values. The categories with at least one participant with shift from baseline in these parameters are reported.
Baseline up to Week 96
Part 1: Change From Baseline in Coagulation Parameters [Activated Partial Thromboplastin Time (aPTT)]
Délai: Baseline, Weeks 24, 48 and 96
A negative change from baseline indicated a reduction in aPTT.
Baseline, Weeks 24, 48 and 96
Part 1: Change From Baseline in Coagulation Parameters [Prothrombin Time (PT)]
Délai: Baseline, Weeks 24, 48 and 96
A negative change from baseline indicated a reduction in PT.
Baseline, Weeks 24, 48 and 96
Part 1: Change From Baseline in Coagulation Parameters [International Normalized Ratio (INR)]
Délai: Baseline, Weeks 24, 48 and 96
A negative change from baseline indicated a reduction in clotting time.
Baseline, Weeks 24, 48 and 96
Part 1: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Blood Chemistry Parameters)
Délai: Baseline up to Week 96
Parameters included sodium, potassium, chloride, bilirubin, alkaline phosphatase, aspartate aminotransferase, alanine aminotransferase, gamma glutamyl transferase, urea nitrogen, creatinine, bicarbonate, calcium, magnesium, phosphate, urate and glucose. These parameters were flagged as low, normal or high relative to parameter's normal range or as unknown if no result was available, by Investigator. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicated values that were normal, low or unknown at baseline and shifted to high values postbaseline. Categories with at least one participant with shift from baseline in these parameters are reported.
Baseline up to Week 96
Part 1: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Urinalysis Parameters)
Délai: Baseline up to Week 96
Urinalysis included assessments of glucose, ketones, occult blood, protein, specific gravity. These parameters were flagged as low, normal, or high relative to parameter's normal range or as unknown if no result was available, by the Investigator. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicated values that were normal, low or unknown at baseline and shifted to high postbaseline. The categories with at least one participant with shift from baseline in these parameters are reported.
Baseline up to Week 96
Part 1: Fatigue Score Measured by the Pediatric Quality of Life Inventory (PedsQL) Multidimensional Fatigue Scale
Délai: Up to Week 96
The PedsQL multidimensional fatigue questionnaire was answered by the participant (self-assessment) and parent. The fatigue scale contains 18 questions in 3 fatigue dimensions: general fatigue, sleep/rest fatigue and cognitive fatigue. Each item was scored on 5-point Likert scale (0=never to 4=almost always). Each individual score was then reversed (subtracted from 4) and linearly transformed as follows: 0=100, 1=75, 2=50, 3=25, 4=0. For each dimension, total score was calculated as the sum of all the items/number of items answered. A higher total score indicated fewer problems.
Up to Week 96
Part 1: Quality of Life (QOL) as Measured by the PedsQL
Délai: Up to Week 96
The PedsQL QOL questionnaire was answered by the participant (self-assessment) and parent. The QoL scale contains 23 questions in 4 dimensions: Physical Functioning, Emotional Fatigue, Social Fatigue and School Fatigue. Each item was scored on 5-point Likert scale (0=never to 4=almost always). Each individual score was then reversed (subtracted from 4) and linearly transformed as follows: 0=100, 1=75, 2=50, 3=25, 4=0. For each dimension, total score was calculated as the sum of all the items/number of items answered. A higher total score indicated better quality of life.
Up to Week 96
Part 1: Change From Baseline in the Expanded Disability Status Scale (EDSS) Score
Délai: Baseline, Week 96
The EDSS measures the disability status of participants with multiple sclerosis on a scale that ranges from 0 to 10. The first levels 1.0 to 4.5 refer to people with a high degree of ambulatory ability and the subsequent levels 5.0 to 9.5 refer to the loss of ambulatory ability. The range of main categories include (0) = normal neurologic exam; to (5) = ambulatory without aid or rest for 200 meters; disability severe enough to impair full daily activities; to (10) = death due to MS with higher scores indicating more disability. A negative change from baseline indicated an improvement in the disability.
Baseline, Week 96
Part 2: Annualized Relapse Rate (ARR)
Délai: From Baseline (Week 96) up to Week 336
ARR is calculated as the total number of relapses that occurred during the study divided by the total number of participant-years. ARR was analyzed using negative binomial regression model.
From Baseline (Week 96) up to Week 336
Part 2: Change From Baseline in the Expanded Disability Status Scale (EDSS) Score
Délai: Baseline (Week 96), Week 336
The EDSS measures the disability status of participants with multiple sclerosis on a scale that ranges from 0 to 10. The first levels 1.0 to 4.5 refer to people with a high degree of ambulatory ability and the subsequent levels 5.0 to 9.5 refer to the loss of ambulatory ability. The range of main categories include (0) = normal neurologic exam; to (5) = ambulatory without aid or rest for 200 meters; disability severe enough to impair full daily activities; to (10) = death due to MS. A negative change from baseline indicated an improvement in the disability.
Baseline (Week 96), Week 336
Part 2: Change From Baseline in Brief Visuospatial Memory Test - Revised (BVMT-R) Score
Délai: Baseline (Week 96), Weeks 144,192, 240, 288 and 336
BVMT-R is used to assess learning/memory. In this test, six abstract designs are presented for 10 sec. The display is removed from view, and participants render the stimuli via pencil on paper, manual responses. Each design receives from 0 to 2 points, representing accuracy and location. There are three Learning Trials, and the score is reported as the total number of points earned over the trials. Thus, scores range from 0 to 12 per trial; total score range is 0 to 36 for all three trials. Scores were converted to standardized scores using normative data, ranging from 2-86. The lower the score, the more severe the cognitive impairment while higher scores reflect better visuospatial memory.
Baseline (Week 96), Weeks 144,192, 240, 288 and 336
Part 2: Change From Baseline in Symbol Digit Modalities Test (SDMT) Score
Délai: Baseline (Week 96), Weeks 144,192, 240, 288 and 336
SDMT is used to assess processing speed. It consists of 9 abstract symbols. Each symbol is paired with a single digit. The participant is provided with a key, showing each symbol digit pair. In addition, the participants are shown several rows of the 9 symbols, which are arranged pseudo-randomly, without the digit. Participants are asked to voice the digit associated with each symbol as rapidly as possible for 90 seconds. The SDMT score ranges from 0 to 110, where higher scores indicate improvement in cognitive functioning and lower scores indicate worsening.
Baseline (Week 96), Weeks 144,192, 240, 288 and 336
Part 2: Number of Participants With or Without School Progression
Délai: At Weeks 144, 192, 240, 288 and 336
Participants or caregivers were posed the following question: "During the past year, did [you/the participant] progress from one [class/grade-level] to the next in school?" Responses were recorded as Yes (participant advanced to the next grade) or No (participant did not advance).
At Weeks 144, 192, 240, 288 and 336
Part 2: Change From Baseline in Vital Signs (Temperature)
Délai: Baseline (Week 96), Weeks 120,144, 168, 192, 216, 240, 264, 288, 312, 336, and 340
Negative change from baseline indicated reduction in temperature.
Baseline (Week 96), Weeks 120,144, 168, 192, 216, 240, 264, 288, 312, 336, and 340
Part 2: Change From Baseline in Vital Signs (Pulse Rate)
Délai: Baseline (Week 96), Weeks 120,144, 168, 192, 216, 240, 264, 288, 312, 336, and 340
Negative change from baseline indicated reduction in pulse rate.
Baseline (Week 96), Weeks 120,144, 168, 192, 216, 240, 264, 288, 312, 336, and 340
Part 2: Change From Baseline in Vital Signs (Blood Pressure)
Délai: Baseline (Week 96), Weeks 120,144, 168, 192, 216, 240, 264, 288, 312, 336, and 340
Negative change from baseline indicated reduction in blood pressure.
Baseline (Week 96), Weeks 120,144, 168, 192, 216, 240, 264, 288, 312, 336, and 340
Part 2: Change From Baseline in Vital Signs (Respiratory Rate)
Délai: Baseline (Week 96), Weeks 120,144, 168, 192, 216, 240, 264, 288, 312, 336, and 340
Negative change from baseline indicated reduction in respiratory rate.
Baseline (Week 96), Weeks 120,144, 168, 192, 216, 240, 264, 288, 312, 336, and 340
Part 2: Number of Participants With Shifts From Baseline in ECG Abnormalities
Délai: From Baseline (Week 96) to Week 336
Clinical significance of abnormalities in ECG was determined based on the investigator's discretion. Shift to abnormal indicated values that were normal or unknown at baseline and shifted to abnormal values post-baseline.
From Baseline (Week 96) to Week 336
Part 2: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Hematology Parameters)
Délai: Baseline (Week 96) up to Week 340
Hematology parameters included leukocytes, erythrocytes, hemoglobin, hematocrit, platelets, eosinophils, lymphocytes, monocytes and neutrophils. These parameters were flagged as low, normal, or high relative to parameter's normal range or as unknown if no result was available. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicated values that were normal, low or unknown at baseline and shifted to high postbaseline values. The categories with at least one participant with shift from baseline in these parameters are reported.
Baseline (Week 96) up to Week 340
Part 2: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Blood Chemistry Parameters)
Délai: Baseline (Week 96) up to Week 340
Parameters included potassium, bilirubin, alkaline phosphatase, alanine aminotransferase, aspartate aminotransferase, gamma glutamyl transferase, urea nitrogen, creatinine, bicarbonate, calcium, magnesium, phosphate, urate and glucose. These parameters were flagged as low, normal or high relative to parameter's normal range or as unknown if no result was available, by Investigator. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicated values that were normal, low or unknown at baseline and shifted to high values postbaseline. Categories with at least one participant with shift from baseline in these parameters are reported.
Baseline (Week 96) up to Week 340
Part 2: Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Urinalysis Parameters)
Délai: Baseline up to Week 340
Urinalysis included assessments of erythrocytes, leukocytes and specific gravity. These parameters were flagged as low, normal, or high relative to parameter's normal range or as unknown if no result was available, by the Investigator. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicated values that were normal, low or unknown at baseline and shifted to high postbaseline. The categories with at least one participant with shift from baseline in these parameters are reported.
Baseline up to Week 340
Part 2: Change From Baseline in Height
Délai: Baseline (Week 96), Weeks 120,144, 168, 192, 216, 240, 264, 288, 312, 336, and 340
Baseline (Week 96), Weeks 120,144, 168, 192, 216, 240, 264, 288, 312, 336, and 340
Part 2: Change From Baseline in Weight
Délai: Baseline (Week 96), Weeks 120,144, 168, 192, 216, 240, 264, 288, 312, 336, and 340
Baseline (Week 96), Weeks 120,144, 168, 192, 216, 240, 264, 288, 312, 336, and 340
Part 2: Change From Baseline in Bone Age
Délai: Baseline (Week 96), Weeks 144, 192 and 240
Bone age was tested until the participant reached a bone age of 16 years.
Baseline (Week 96), Weeks 144, 192 and 240
Part 2: Number of Male Participants by Tanner Stage Assessment
Délai: At Weeks 96, 144, 192, 240, 288 and 336
Tanner pubertal staging in males was assessed for testes and scrotum development and pubic hair growth, progressing from stage 1 (prepubertal) to stage 5 (adult). Assessments ended when bone age reached ≥16 years. Testes and scrotum stages were: 1-prepubertal; 2-enlargement of testes, scrotum reddens, texture change; 3-Enlargement of penis, further growth of testes; 4-Increased size of penis with growth in breadth and development of glans; testes and scrotum larger, scrotum skin darker; 5-adult genitalia. Pubic hair stages were: 1-prepubertal; 2-Sparse growth of long, slightly pigmented hair, straight or curled; 3-Darker, coarser and more curled hair, spreading sparsely over junction of pubes; 4-Hair adult in type, but covering smaller area than in adult; no spread to medial surface of thighs; 5-Adult in type and quantity, with horizontal distribution.
At Weeks 96, 144, 192, 240, 288 and 336
Part 2: Number of Female Participants by Tanner Stage Assessment
Délai: At Weeks 96, 144, 192, 240, 288 and 336
Tanner pubertal staging in females was assessed for breast development and pubic hair growth, progressing from stage 1 (prepubertal) to stage 5 (adult). Assessments ended when bone age reached ≥16 years or the participant was post-menarche. Breast development stages: 1-prepubertal; 2-breast bud stage with elevation of breast and papilla; enlargement of areola, 3-further enlargement of breast and areola; no separation of their contour; 4-areola and papilla form secondary mound above level of breast; 5-mature stage: projection of papilla only, related to recession of areola. Pubic hair stages: 1-prepubertal (can see velus hair similar to abdominal wall); 2-sparse growth of long, slightly pigmented hair, straight or curled, mainly on labia; 3-Darker, coarser and more curled hair, spreading sparsely over junction of pubes; 4-hair adult in type, but covering smaller area than in adult; no spread to medial surface of thighs; 5-adult in type and quantity, with horizontal distribution.
At Weeks 96, 144, 192, 240, 288 and 336

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  • Directeur d'études: Medical Director, Biogen

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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)

28 août 2014

Achèvement primaire (Réel)

8 juillet 2025

Achèvement de l'étude (Réel)

8 juillet 2025

Dates d'inscription aux études

Première soumission

3 novembre 2014

Première soumission répondant aux critères de contrôle qualité

4 novembre 2014

Première publication (Estimé)

5 novembre 2014

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

19 mai 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

16 mai 2026

Dernière vérification

1 mai 2026

Plus d'information

Termes liés à cette étude

Mots clés

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • 109MS306
  • 2013-002318-11 (Numéro EudraCT)

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