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Fase 3-onderzoek naar werkzaamheid en veiligheid van BG00012 bij pediatrische proefpersonen met relapsing-remitting multiple sclerose (RRMS) (CONNECT)

16 mei 2026 bijgewerkt door: Biogen

Open-label, gerandomiseerd, multicenter, actief gecontroleerd, werkzaamheids- en veiligheidsonderzoek met meerdere doses van BG00012 bij kinderen van 10 tot jonger dan 18 jaar met relapsing-remitting multiple sclerose, met optionele open-label extensie

De belangrijkste doelstellingen van deel 1 zijn als volgt: evalueren van de veiligheid, verdraagbaarheid en werkzaamheid van BG00012 bij pediatrische proefpersonen met RRMS, in vergelijking met een ziektemodificerende behandeling en het beoordelen van gezondheidsresultaten en de ontwikkeling van invaliditeit. Het primaire doel van deel 2 is het evalueren van de veiligheid op lange termijn van BG00012 bij proefpersonen die week 96 in deel 1 van onderzoek 109MS306 hebben voltooid. Het secundaire doel van deel 2 is het beschrijven van de MS-uitkomsten op lange termijn van BG00012 bij proefpersonen die week 96 voltooiden in deel 1 van onderzoek 109MS306.

Studie Overzicht

Toestand

Voltooid

Conditie

Interventie / Behandeling

Studietype

Ingrijpend

Inschrijving (Werkelijk)

156

Fase

  • Fase 3

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

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

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

10 jaar tot 17 jaar (Kind)

Accepteert gezonde vrijwilligers

Nee

Beschrijving

Belangrijkste opnamecriteria:

  • Moet een lichaamsgewicht hebben van ≥30 kg.
  • Moet een diagnose van RRMS hebben (consensusdefinitie voor pediatrische RRMS [Krupp 2007]).
  • Moet ambulant zijn met een baseline EDSS-score tussen 0 en 5,5, inclusief.
  • Moet ten minste 1 van de volgende 3 aandoeningen hebben ervaren: a) ten minste 1 terugval in de laatste 12 maanden voorafgaand aan dag 1 met een eerdere hersen-MRI die laesies aantoont die consistent zijn met MS; b) ten minste 2 recidieven in de laatste 24 maanden voorafgaand aan dag 1, met een eerdere hersen-MRI die laesies aantoont die consistent zijn met MS; c) bewijs van Gd-aankleurende laesies van de hersenen op een MRI uitgevoerd binnen de 6 weken voorafgaand aan Dag 1.
  • Moet neurologisch stabiel zijn, zonder bewijs van terugval binnen 50 dagen voorafgaand aan dag 1 en geen bewijs van behandeling met corticosteroïden binnen 30 dagen voorafgaand aan dag 1.
  • Proefpersonen in de vruchtbare leeftijd die seksueel actief zijn, moeten bereid zijn om effectieve anticonceptie toe te passen tijdens het onderzoek en bereid en in staat zijn om anticonceptie voort te zetten gedurende ten minste 30 dagen na hun laatste dosis van de onderzoeksbehandeling.

Belangrijkste uitsluitingscriteria:

  • Primair progressieve, secundair progressieve of progressieve recidiverende MS (zoals gedefinieerd door [Lublin en Reingold 1996]). Deze aandoeningen vereisen de aanwezigheid van een continue verslechtering van de klinische ziekte gedurende een periode van ten minste 3 maanden. Proefpersonen met deze aandoeningen kunnen ook gesuperponeerde terugvallen hebben, maar onderscheiden zich van relapsing-remitting proefpersonen door het ontbreken van klinisch stabiele perioden of klinische verbetering.
  • Aandoeningen die lijken op MS, zoals andere demyeliniserende aandoeningen (bijv. acute gedissemineerde encefalomyelitis), systemische auto-immuunziekten (bijv. de ziekte van Sjögren, lupus erythematosus), stofwisselingsstoornissen (bijv. dystrofieën) en infectieziekten.
  • Geschiedenis van premaligne of kwaadaardige ziekte. Proefpersonen met basaalcelcarcinoom die voorafgaand aan de screening volledig zijn weggesneden, blijven in aanmerking komen.
  • Geschiedenis van ernstige allergische of anafylactische reacties, of bekende overgevoeligheid voor geneesmiddelen voor DMF, fumaarzuuresters of interferon beta-1a (IFN Beta-1a).
  • Geschiedenis van abnormale laboratoriumresultaten die wijzen op een significante endocrinologische, hematologische, hepatische, immunologische, metabolische, urologische, nierziekte en/of enige andere ernstige ziekte die deelname aan een klinische studie zou uitsluiten.

  • Geschiedenis van klinisch significante cardiovasculaire, pulmonaire, GI, dermatologische, groei-, ontwikkelings-, psychiatrische (inclusief depressie), neurologische (anders dan MS) en/of andere ernstige ziekten die deelname aan een klinische studie zouden uitsluiten.

    -.Geschiedenis van het humaan immunodeficiëntievirus.

  • Een MS-terugval die is opgetreden binnen 50 dagen voorafgaand aan dag 1 EN/OF de proefpersoon is niet gestabiliseerd na een eerdere terugval voorafgaand aan dag 1.
  • Andere niet-gespecificeerde redenen die, naar de mening van de Onderzoeker of Biogen Idec, de proefpersoon ongeschikt maken voor inschrijving.
  • Voor de PD/PK-subgroep van proefpersonen: proefpersonen die de BG00012-capsule niet in hun geheel kunnen doorslikken.

Belangrijkste behandelingsgeschiedenis

  • Elke eerdere behandeling met Fumaderm (fumaarzuuresters) of BG00012.
  • Voorafgaande behandeling met een van de volgende: totale lymfoïde bestraling, cladribine, T-cel- of T-celreceptorvaccinatie, elk therapeutisch monoklonaal antilichaam, met uitzondering van rituximab of natalizumab.
  • Voorafgaande behandeling met een van de volgende medicijnen binnen de 12 maanden voorafgaand aan dag 1: mitoxantron, cyclofosfamide, rituximab.
  • Voorafgaande behandeling met een van de volgende medicijnen of procedures binnen 6 maanden voorafgaand aan Dag 1: fingolimod; teriflunomide; natalizumab; ciclosporine; azathioprine; methotrexaat; mycofenolaatmofetil; laquinimod; intraveneus (IV) immunoglobuline; plasmaferese of cytaferese
  • Behandeling met een van de volgende medicijnen binnen 30 dagen voorafgaand aan Dag 1: steroïden (IV of orale behandeling met corticosteroïden, inclusief middelen die mogelijk niet werken via de corticosteroïdroute [bijv. Lage dosis naltrexon]), 4-aminopyridine of verwante producten (behalve proefpersonen op een stabiele dosis fampridine met gereguleerde afgifte gedurende 3 maanden)

OPMERKING: Er kunnen andere door het protocol gedefinieerde opname-/uitsluitingscriteria van toepassing zijn

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: BG00012
Deelnemers krijgen de aanbevolen dosis van 240 mg oraal, tweemaal daags
Geneesmiddel: dimethylfumaraat
oraal toegediend
Andere namen:
  • BG00012
  • Tecfidera
Actieve vergelijker: IFN β-1a (Avonex)
Deelnemers krijgen de aanbevolen dosis van 30 μg (wekelijks)
Geneesmiddel: Interferon β-1a
toegediend via intramusculaire injectie
Andere namen:
  • Avonex

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Part 1: Proportion of Participants Free of New or Newly Enlarging T2 Hyperintense Lesions on Brain Magnetic Resonance Imaging (MRI) Scans
Tijdsspanne: 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)
Tijdsspanne: 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
Tijdsspanne: 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)

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Part 1: Number of New or Newly Enlarged T2 Hyperintense Lesions on Brain MRI Scans
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: 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)]
Tijdsspanne: 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)]
Tijdsspanne: 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)]
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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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Onderzoekers

  • Studie directeur: Medical Director, Biogen

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Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

28 augustus 2014

Primaire voltooiing (Werkelijk)

8 juli 2025

Studie voltooiing (Werkelijk)

8 juli 2025

Studieregistratiedata

Eerst ingediend

3 november 2014

Eerst ingediend dat voldeed aan de QC-criteria

4 november 2014

Eerst geplaatst (Geschat)

5 november 2014

Updates van studierecords

Laatste update geplaatst (Werkelijk)

19 mei 2026

Laatste update ingediend die voldeed aan QC-criteria

16 mei 2026

Laatst geverifieerd

1 mei 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

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

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