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Estudio de eficacia y seguridad de fase 3 de BG00012 en sujetos pediátricos con esclerosis múltiple remitente recurrente (EMRR) (CONNECT)

16 de mayo de 2026 actualizado por: Biogen

Estudio abierto, aleatorizado, multicéntrico, de dosis múltiples, con control activo, de grupos paralelos, de eficacia y seguridad de BG00012 en niños de 10 a menos de 18 años con esclerosis múltiple remitente-recurrente, con extensión abierta opcional

Los objetivos principales de la Parte 1 son los siguientes: Evaluar la seguridad, la tolerabilidad y la eficacia de BG00012 en sujetos pediátricos con EMRR, en comparación con un tratamiento modificador de la enfermedad y evaluar los resultados de salud y la evolución de la discapacidad. El objetivo principal de la Parte 2 es evaluar la seguridad a largo plazo de BG00012 en sujetos que completaron la Semana 96 en la Parte 1 del Estudio 109MS306. El objetivo secundario de la Parte 2 es describir los resultados de EM a largo plazo de BG00012 en sujetos que completaron la Semana 96 en la Parte 1 del Estudio 109MS306.

Descripción general del estudio

Estado

Terminado

Condiciones

Intervención / Tratamiento

Tipo de estudio

Intervencionista

Inscripción (Actual)

156

Fase

  • Fase 3

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

  • Alemania
    • Bavaria
      • Augsburg, Bavaria, Alemania, 86156
        • Universitaetsklinikum Augsburg
      • Munich, Bavaria, Alemania, 80337
        • Klinikum der Universitaet Muenchen
    • North Rhine-Westphalia
      • Bochum, North Rhine-Westphalia, Alemania, 44791
        • Katholisches Klinikum Bochum gGmbH
  • Bulgaria
      • Sofia, Bulgaria, 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
  • Chequia
      • Brno, Chequia, 656 91
        • Fakultni nemocnice u sv. Anny v Brne
      • Hradec Králové, Chequia, 500 05
        • Fakultni nemocnice Hradec Kralove
      • Jihlava, Chequia, 58633
        • Nemocnice Jihlava p.o.
      • Ostrava, Chequia, 708 52
        • Fakultni Nemocnice Ostrava
  • Dinamarca
      • Aarhus N, Dinamarca, 8200
        • Århus Universitetshospital
      • Copenhagen, Dinamarca, 2100
        • Rigshospitalet
      • Odense, Dinamarca, 5000
        • Odense Universitetshospital
  • España
      • Seville, España, 41009
        • Hospital Universitario Virgen Macarena
    • Barcelona
      • Esplugues de Llobregat, Barcelona, España, 8950
        • Hospital Sant Joan de Déu
    • Córdoba
      • Córdoba, Córdoba, España, 14011
        • Hospital Universitario Reina Sofia
    • Madrid
      • Torrejón de Ardoz, Madrid, España, 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
  • Francia
    • Bas Rhin
      • Strasbourg, Bas Rhin, Francia, 67098
        • CHU Strasbourg - Hôpital Hautepierre
    • Bouches-du-Rhône
      • Marseille, Bouches-du-Rhône, Francia, 13385
        • Hôpital de la Timone
    • Côte-d'Or
      • Dijon, Côte-d'Or, Francia, 21079
        • CHU Dijon -BOCAGE CENTRAL
    • Herault
      • Montpellier, Herault, Francia, 34295
        • Hôpital Gui de Chauliac
    • Ille Et Vilaine
      • Rennes, Ille Et Vilaine, Francia, 35033
        • CHU Rennes - Hôpital Pontchaillou
    • Meurthe Et Moselle
      • Vandœuvre-lès-Nancy, Meurthe Et Moselle, Francia, 54500
        • Hôpital de Brabois Enfants
    • Nord
      • Lille, Nord, Francia, 59037
        • Hopital Roger Salengro - CHU Lille
    • Puy De Dome
      • Clermont-Ferrand, Puy De Dome, Francia, 63003
        • CHU Clermont Ferrand - Hopital d'Estaing
    • Rhone
      • Bron, Rhone, Francia, 69677
        • Hopital Neurologique Pierre Wertheimer
    • Somme
      • Amiens, Somme, Francia, 80054
        • CHU Amiens - Hopital Sud
    • Val De Marne
      • Le Kremlin-Bicêtre, Val De Marne, Francia, 94275
        • Hôpital Bicêtre
  • Hungría
      • Budapest, Hungría, 1083
        • Semmelweis Egyetem
      • Budapest, Hungría, 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
  • Italia
      • Bari, Italia, 70124
        • Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
      • Genova, Italia, 16132
        • IRCCS Ospedale Policlinico San Martino
      • Milan, Italia, 20132
        • Ospedale San Raffaele
      • Naples, Italia, 80131
        • Azienda Ospedaliera Universitaria 'Federico II'
      • Padova, Italia, 35128
        • Azienda Ospedale-Università di Padova
      • Palermo, Italia, 90127
        • Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone
      • Roma, Italia, 00189
        • Azienda Ospedaliera Sant'Andrea-Università di Roma La Sapienza
      • Roma, Italia, 00165
        • Ospedale Pediatrico Bambino Gesu
    • Varese
      • Gallarate, Varese, Italia, 21013
        • Azienda Socio Sanitaria Territoriale della Valle Olona (presidio di Gallarate)
  • Kuwait
      • Ash Shuwaykh, Kuwait, 12345
        • Ibn Sina Hospital
  • Polonia
      • Bialystok, Polonia, 15-274
        • SPZOZ Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa
      • Gdansk, Polonia, 80-211
        • Uniwersyteckie Centrum Kliniczne
      • Lodz, Polonia, 93-338
        • Instytut Centrum Zdrowia Matki Polki
      • Poznan, Polonia, 60-355
        • Szpital Kliniczny im.Heliodora Swiecickiego Uniwersytetu Medycznego im.K. Marcinkowskiego w Poznaniu
      • Warsaw, Polonia, 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
  • Serbia
      • Belgrade, Serbia, 11000
        • Clinic of Neurology and Psychiatry for Children and Youth
      • Belgrade, Serbia, 11000
        • Mother and Child Health Care Institute of Serbia ,,Dr Vukan Cupic''
      • Kragujevac, Serbia, 34000
        • University Clinical Center Kragujevac
  • Suecia
      • Gothenburg, Suecia, 41345
        • Sahlgrenska Sjukhuset
      • Stockholm, Suecia, SE-113 61
        • Karolinska
  • Turquía (Türkiye)
      • Ankara, Turquía (Türkiye), 06100
        • Hacettepe University Medical Faculty
      • Antalya, Turquía (Türkiye), 07070
        • Akdeniz University Faculty of Medicine

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

10 años a 17 años (Niño)

Acepta Voluntarios Saludables

No

Descripción

Criterios clave de inclusión:

  • Debe tener un peso corporal de ≥30 kg.
  • Debe tener un diagnóstico de RRMS (definición de consenso para RRMS pediátrica [Krupp 2007]).
  • Debe ser ambulatorio con una puntuación EDSS inicial entre 0 y 5,5, inclusive.
  • Debe haber experimentado al menos 1 de las siguientes 3 condiciones: a) al menos 1 recaída en los últimos 12 meses antes del Día 1 con una resonancia magnética cerebral previa que demuestre lesiones compatibles con EM; b) al menos 2 recaídas en los últimos 24 meses antes del día 1, con una resonancia magnética cerebral previa que demuestre lesiones compatibles con EM; c) evidencia de lesiones del cerebro realzadas con Gd en una resonancia magnética realizada dentro de las 6 semanas anteriores al día 1.
  • Debe estar neurológicamente estable, sin evidencia de recaída dentro de los 50 días anteriores al Día 1 y sin evidencia de tratamiento con corticosteroides dentro de los 30 días anteriores al Día 1.
  • Los sujetos en edad fértil que son sexualmente activos deben estar dispuestos a practicar métodos anticonceptivos efectivos durante el estudio y estar dispuestos y ser capaces de continuar con la anticoncepción durante al menos 30 días después de la dosis final del tratamiento del estudio.

Criterios clave de exclusión:

  • EM primaria progresiva, secundaria progresiva o progresiva recidivante (según la definición de [Lublin y Reingold 1996]). Estas condiciones requieren la presencia de un empeoramiento clínico continuo de la enfermedad durante un período de al menos 3 meses. Los sujetos con estas condiciones también pueden tener recaídas superpuestas, pero se distinguen de los sujetos con recaídas y remisiones por la falta de períodos clínicamente estables o de mejoría clínica.
  • Trastornos que simulan la EM, como otros trastornos desmielinizantes (p. ej., encefalomielitis diseminada aguda), trastornos autoinmunes sistémicos (p. ej., enfermedad de Sjögren, lupus eritematoso), trastornos metabólicos (p. ej., distrofias) y trastornos infecciosos.
  • Antecedentes de enfermedad premaligna o maligna. Los sujetos con carcinoma de células basales que hayan sido completamente extirpados antes de la selección seguirán siendo elegibles.
  • Antecedentes de reacciones alérgicas o anafilácticas graves, o hipersensibilidad conocida al fármaco a DMF, ésteres de ácido fumárico o interferón beta-1a (IFN Beta-1a).
  • Historial de resultados de laboratorio anormales indicativos de cualquier enfermedad endocrinológica, hematológica, hepática, inmunológica, metabólica, urológica, renal y/o cualquier otra enfermedad importante que impida la participación en un estudio clínico.

  • Antecedentes de enfermedades cardiovasculares, pulmonares, gastrointestinales, dermatológicas, del crecimiento, del desarrollo, psiquiátricas (incluida la depresión), neurológicas (distintas de la EM) y/u otras enfermedades clínicamente significativas que impedirían la participación en un estudio clínico.

    -.Historia del virus de la inmunodeficiencia humana.

  • Una recaída de EM que haya ocurrido dentro de los 50 días anteriores al Día 1 Y/O el sujeto no se haya estabilizado de una recaída anterior al Día 1.
  • Otras razones no especificadas que, en opinión del Investigador o de Biogen Idec, hacen que el sujeto no sea apto para la inscripción.
  • Para el subconjunto de sujetos PD/PK: sujetos incapaces de tragar la cápsula BG00012 entera.

Historial de tratamiento clave

  • Cualquier tratamiento previo con Fumaderm (ésteres de ácido fumárico) o BG00012.
  • Tratamiento previo con cualquiera de los siguientes: irradiación linfoide total, cladribina, vacunación contra células T o receptores de células T, cualquier anticuerpo monoclonal terapéutico, con la excepción de rituximab o natalizumab.
  • Tratamiento previo con cualquiera de los siguientes medicamentos dentro de los 12 meses anteriores al Día 1: mitoxantrona, ciclofosfamida, rituximab.
  • Tratamiento previo con cualquiera de los siguientes medicamentos o procedimientos dentro de los 6 meses anteriores al Día 1: fingolimod; teriflunomida; natalizumab; ciclosporina; azatioprina; metotrexato; micofenolato mofetilo; laquinimod; inmunoglobulina intravenosa (IV); plasmaféresis o citaféresis
  • Tratamiento con cualquiera de los siguientes medicamentos dentro de los 30 días anteriores al Día 1: esteroides (tratamiento con corticosteroides intravenosos u orales, incluidos los agentes que pueden no actuar a través de la vía de los corticosteroides [p. ej., dosis baja de naltrexona]), 4-aminopiridina o productos relacionados (excepto sujetos con una dosis estable de fampridina de liberación controlada durante 3 meses)

NOTA: Es posible que se apliquen otros criterios de inclusión/exclusión definidos en el protocolo

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: Aleatorizado
  • Modelo Intervencionista: Asignación paralela
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: BG00012
Los participantes recibirán la dosis recomendada de 240 mg por vía oral, dos veces al día.
Droga: fumarato de dimetilo
administrado por vía oral
Otros nombres:
  • BG00012
  • Tecfidera
Comparador activo: IFN β-1a (Avonex)
Los participantes recibirán la dosis recomendada de 30 μg (semanalmente)
Droga: Interferón β-1a
administrado por inyección intramuscular
Otros nombres:
  • Avonex

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Part 1: Proportion of Participants Free of New or Newly Enlarging T2 Hyperintense Lesions on Brain Magnetic Resonance Imaging (MRI) Scans
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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 resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Part 1: Number of New or Newly Enlarged T2 Hyperintense Lesions on Brain MRI Scans
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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)]
Periodo de tiempo: 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)]
Periodo de tiempo: 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)]
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Biogen

Investigadores

  • Director de estudio: Medical Director, Biogen

Publicaciones y enlaces útiles

La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.

Publicaciones Generales

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

28 de agosto de 2014

Finalización primaria (Actual)

8 de julio de 2025

Finalización del estudio (Actual)

8 de julio de 2025

Fechas de registro del estudio

Enviado por primera vez

3 de noviembre de 2014

Primero enviado que cumplió con los criterios de control de calidad

4 de noviembre de 2014

Publicado por primera vez (Estimado)

5 de noviembre de 2014

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

19 de mayo de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

16 de mayo de 2026

Última verificación

1 de mayo de 2026

Más información

Términos relacionados con este estudio

Palabras clave

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

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

Plan de datos de participantes individuales (IPD)

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SÍ

Descripción del plan IPD

De acuerdo con la Política de intercambio de datos y transparencia de ensayos clínicos de Biogen en https://www.biogentrialtransparency.com/.

Información sobre medicamentos y dispositivos, documentos del estudio

producto fabricado y exportado desde los EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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