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Estudo de eficácia e segurança de fase 3 de BG00012 em pacientes pediátricos com esclerose múltipla recorrente-remitente (EMRR) (CONNECT)

16 de maio de 2026 atualizado por: Biogen

Estudo Aberto, Randomizado, Multicêntrico, Dose Múltipla, Controle Ativo, Grupo Paralelo, Eficácia e Segurança do BG00012 em Crianças de 10 a Menos de 18 Anos de Idade com Esclerose Múltipla Recorrente-Remitente, com Extensão Aberta Opcional

Os principais objetivos da Parte 1 são os seguintes: Avaliar a segurança, tolerabilidade e eficácia do BG00012 em pacientes pediátricos com EMRR, em comparação com um tratamento modificador da doença e avaliar os resultados de saúde e a evolução da incapacidade. O objetivo principal da Parte 2 é avaliar a segurança a longo prazo de BG00012 em indivíduos que completaram a Semana 96 na Parte 1 do Estudo 109MS306. O objetivo secundário da Parte 2 é descrever os resultados de MS a longo prazo de BG00012 em indivíduos que completaram a Semana 96 na Parte 1 do Estudo 109MS306.

Visão geral do estudo

Status

Concluído

Condições

Intervenção / Tratamento

Tipo de estudo

Intervencional

Inscrição (Real)

156

Estágio

  • Fase 3

Contactos e Locais

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

Locais de estudo

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

Critérios de participação

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

Critérios de elegibilidade

Idades elegíveis para estudo

10 anos a 17 anos (Filho)

Aceita Voluntários Saudáveis

Não

Descrição

Principais Critérios de Inclusão:

  • Deve ter um peso corporal ≥30 kg.
  • Deve ter um diagnóstico de EMRR (definição de consenso para EMRR pediátrica [Krupp 2007]).
  • Deve ser ambulatorial com uma pontuação EDSS de linha de base entre 0 e 5,5, inclusive.
  • Deve ter experimentado pelo menos 1 das 3 condições a seguir: a) pelo menos 1 recaída nos últimos 12 meses antes do Dia 1 com uma ressonância magnética cerebral prévia demonstrando lesões consistentes com EM; b) pelo menos 2 recidivas nos últimos 24 meses antes do Dia 1, com uma ressonância magnética cerebral anterior demonstrando lesões consistentes com EM; c) evidência de lesões cerebrais intensificadas por Gd em uma ressonância magnética realizada nas 6 semanas anteriores ao Dia 1.
  • Deve estar neurologicamente estável, sem evidência de recidiva nos 50 dias anteriores ao Dia 1 e nenhuma evidência de tratamento com corticosteroides nos 30 dias anteriores ao Dia 1.
  • Indivíduos com potencial para engravidar que são sexualmente ativos devem estar dispostos a praticar contracepção eficaz durante o estudo e estar dispostos e aptos a continuar a contracepção por pelo menos 30 dias após a dose final do tratamento do estudo.

Principais Critérios de Exclusão:

  • EM primária progressiva, secundária progressiva ou recidivante progressiva (conforme definido por [Lublin e Reingold 1996]). Essas condições requerem a presença de piora clínica contínua da doença por um período de pelo menos 3 meses. Indivíduos com essas condições também podem ter recaídas sobrepostas, mas se distinguem de indivíduos remitentes-recorrentes pela falta de períodos clinicamente estáveis ​​ou melhora clínica.
  • Distúrbios que mimetizam a EM, como outros distúrbios desmielinizantes (por exemplo, encefalomielite disseminada aguda), distúrbios autoimunes sistêmicos (por exemplo, doença de Sjögren, lúpus eritematoso), distúrbios metabólicos (por exemplo, distrofias) e distúrbios infecciosos.
  • História de doença pré-maligna ou maligna. Indivíduos com carcinoma basocelular que foi completamente extirpado antes da triagem permanecerão elegíveis.
  • História de reações alérgicas ou anafiláticas graves, ou hipersensibilidade conhecida a medicamentos a DMF, ésteres de ácido fumárico ou interferon beta-1a (IFN Beta-1a).
  • Histórico de resultados laboratoriais anormais indicativos de qualquer doença endocrinológica, hematológica, hepática, imunológica, metabólica, urológica, renal e/ou qualquer outra doença importante que impeça a participação em um estudo clínico.

  • Histórico de doenças cardiovasculares, pulmonares, gastrointestinais, dermatológicas, de crescimento, de desenvolvimento, psiquiátricas (incluindo depressão), neurológicas (exceto EM) e/ou outras doenças graves clinicamente significativas que impeçam a participação em um estudo clínico.

    -.História do vírus da imunodeficiência humana.

  • Uma recaída de EM que ocorreu dentro de 50 dias antes do Dia 1 E/OU o sujeito não estabilizou de uma recaída anterior ao Dia 1.
  • Outros motivos não especificados que, na opinião do Investigador ou da Biogen Idec, tornem o sujeito inadequado para inscrição.
  • Para o subconjunto de indivíduos PD/PK: indivíduos incapazes de engolir a cápsula BG00012 inteira.

Histórico de tratamento chave

  • Qualquer tratamento anterior com Fumaderm (ésteres de ácido fumárico) ou BG00012.
  • Tratamento prévio com qualquer um dos seguintes: irradiação linfoide total, cladribina, vacinação com células T ou receptores de células T, qualquer anticorpo monoclonal terapêutico, com exceção de rituximabe ou natalizumabe.
  • Tratamento prévio com qualquer um dos seguintes medicamentos nos 12 meses anteriores ao Dia 1: mitoxantrona, ciclofosfamida, rituximab.
  • Tratamento anterior com qualquer um dos seguintes medicamentos ou procedimentos dentro de 6 meses antes do Dia 1: fingolimod; teriflunomida; natalizumabe; ciclosporina; azatioprina; metotrexato; micofenolato de mofetil; laquinimod; imunoglobulina intravenosa (IV); plasmaferese ou citaferese
  • Tratamento com qualquer um dos seguintes medicamentos dentro de 30 dias antes do Dia 1: esteroides (tratamento intravenoso ou oral com corticosteroides, incluindo agentes que podem não agir pela via dos corticosteroides [por exemplo, naltrexona em dose baixa]), 4-aminopiridina ou produtos relacionados (exceto indivíduos em uma dose estável de fampridina de liberação controlada por 3 meses)

NOTA: Outros critérios de inclusão/exclusão definidos pelo protocolo podem ser aplicados

Plano de estudo

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

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: Randomizado
  • Modelo Intervencional: Atribuição Paralela
  • Mascaramento: Nenhum (rótulo aberto)

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Experimental: BG00012
Os participantes receberão a dose recomendada de 240 mg por via oral, duas vezes ao dia
Medicamento: fumarato de dimetilo
administrado por via oral
Outros nomes:
  • BG00012
  • Tecfidera
Comparador Ativo: IFN β-1a (Avonex)
Os participantes receberão a dose recomendada de 30 μg (semanal)
Medicamento: Interferon β-1a
administrado por injeção intramuscular
Outros nomes:
  • Avonex

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Part 1: Proportion of Participants Free of New or Newly Enlarging T2 Hyperintense Lesions on Brain Magnetic Resonance Imaging (MRI) Scans
Prazo: 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)
Prazo: 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
Prazo: 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)

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Part 1: Number of New or Newly Enlarged T2 Hyperintense Lesions on Brain MRI Scans
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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)
Prazo: 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
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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
Prazo: 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)
Prazo: 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)]
Prazo: 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)]
Prazo: 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)]
Prazo: 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)
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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

Colaboradores e Investigadores

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

Patrocinador

Biogen

Investigadores

  • Diretor de estudo: Medical Director, Biogen

Publicações e links úteis

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Publicações Gerais

Datas de registro do estudo

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

Datas Principais do Estudo

Início do estudo (Real)

28 de agosto de 2014

Conclusão Primária (Real)

8 de julho de 2025

Conclusão do estudo (Real)

8 de julho de 2025

Datas de inscrição no estudo

Enviado pela primeira vez

3 de novembro de 2014

Enviado pela primeira vez que atendeu aos critérios de CQ

4 de novembro de 2014

Primeira postagem (Estimado)

5 de novembro de 2014

Atualizações de registro de estudo

Última Atualização Postada (Real)

19 de maio de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

16 de maio de 2026

Última verificação

1 de maio de 2026

Mais Informações

Termos relacionados a este estudo

Palavras-chave

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

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

Plano para dados de participantes individuais (IPD)

Planeja compartilhar dados de participantes individuais (IPD)?

SIM

Descrição do plano IPD

De acordo com a Política de Transparência de Ensaios Clínicos e Compartilhamento de Dados da Biogen em https://www.biogentrialtransparency.com/.

Informações sobre medicamentos e dispositivos, documentos de estudo

produto fabricado e exportado dos EUA

Não

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