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Fase 3 Effekt- og sikkerhetsstudie av BG00012 hos pediatriske personer med residiverende-remitterende multippel sklerose (RRMS) (CONNECT)

16. mai 2026 oppdatert av: Biogen

Åpen, randomisert, multisenter-, multippeldose-, aktiv-kontrollert, parallellgruppe-, effekt- og sikkerhetsstudie av BG00012 hos barn fra 10 til under 18 år med residiverende-remitterende multippel sklerose, med valgfri åpen utvidelse

Hovedmålene for del 1 er som følger: Å evaluere sikkerheten, tolerabiliteten og effekten av BG00012 hos pediatriske personer med RRMS, sammenlignet med en sykdomsmodifiserende behandling og å vurdere helseutfall og utvikling av funksjonshemming. Hovedmålet med del 2 er å evaluere den langsiktige sikkerheten til BG00012 hos personer som fullførte uke 96 i del 1 av studie 109MS306. Det sekundære målet med del 2 er å beskrive de langsiktige MS-resultatene av BG00012 i fag som fullførte uke 96 i del 1 av studie 109MS306.

Studieoversikt

Status

Fullført

Forhold

Intervensjon / Behandling

Studietype

Intervensjonell

Registrering (Faktiske)

156

Fase

  • Fase 3

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

  • Belgia
      • Brussels, Belgia, 1020
        • Universitair Kinderziekenhuis Koningin Fabiola
      • Ghent, Belgia, 9000
        • Universitair Ziekenhuis Ghent
  • Bulgaria
      • Sofia, Bulgaria, 1113
        • MHATNP 'Sv.Naum', EAD
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T3B 6A8
        • University of Calgary - Alberta Children's Hospital
  • Danmark
      • Aarhus N, Danmark, 8200
        • Århus Universitetshospital
      • Copenhagen, Danmark, 2100
        • Rigshospitalet
      • Odense, Danmark, 5000
        • Odense Universitetshospital
  • Forente stater
    • Massachusetts
      • Boston, Massachusetts, Forente stater, 2115
        • Boston Children's Hospital
    • Virginia
      • Charlottesville, Virginia, Forente stater, 22903
        • The Rector and Visitors of the University of Virginia
  • Frankrike
    • Bas Rhin
      • Strasbourg, Bas Rhin, Frankrike, 67098
        • CHU Strasbourg - Hôpital Hautepierre
    • Bouches-du-Rhône
      • Marseille, Bouches-du-Rhône, Frankrike, 13385
        • Hôpital de la Timone
    • Côte-d'Or
      • Dijon, Côte-d'Or, Frankrike, 21079
        • CHU Dijon -BOCAGE CENTRAL
    • Herault
      • Montpellier, Herault, Frankrike, 34295
        • Hôpital Gui de Chauliac
    • Ille Et Vilaine
      • Rennes, Ille Et Vilaine, Frankrike, 35033
        • CHU Rennes - Hôpital Pontchaillou
    • Meurthe Et Moselle
      • Vandœuvre-lès-Nancy, Meurthe Et Moselle, Frankrike, 54500
        • Hôpital de Brabois Enfants
    • Nord
      • Lille, Nord, Frankrike, 59037
        • Hopital Roger Salengro - CHU Lille
    • Puy De Dome
      • Clermont-Ferrand, Puy De Dome, Frankrike, 63003
        • CHU Clermont Ferrand - Hopital d'Estaing
    • Rhone
      • Bron, Rhone, Frankrike, 69677
        • Hopital Neurologique Pierre Wertheimer
    • Somme
      • Amiens, Somme, Frankrike, 80054
        • CHU Amiens - Hopital Sud
    • Val De Marne
      • Le Kremlin-Bicêtre, Val De Marne, Frankrike, 94275
        • Hôpital Bicêtre
  • 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
  • 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'
  • 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
  • Spania
      • Seville, Spania, 41009
        • Hospital Universitario Virgen Macarena
    • Barcelona
      • Esplugues de Llobregat, Barcelona, Spania, 8950
        • Hospital Sant Joan de Déu
    • Córdoba
      • Córdoba, Córdoba, Spania, 14011
        • Hospital Universitario Reina Sofia
    • Madrid
      • Torrejón de Ardoz, Madrid, Spania, 28850
        • Hospital Universitario De Torrejon
  • Storbritannia
    • Greater London
      • London, Greater London, Storbritannia, WC1N 3BG
        • The National Hospital for Neurology & Neurosurgery
      • London, Greater London, Storbritannia, WC1N 3JH
        • Great Ormond Street Hospital for Children
      • London, Greater London, Storbritannia, SE1 7EH
        • Evelina London Children's Hospital
    • West Midlands
      • Birmingham, West Midlands, Storbritannia, B4 6NH
        • Birmingham Children's Hospital
  • Sverige
      • Gothenburg, Sverige, 41345
        • Sahlgrenska Sjukhuset
      • Stockholm, Sverige, SE-113 61
        • Karolinska
  • Tsjekkia
      • Brno, Tsjekkia, 656 91
        • Fakultni nemocnice u sv. Anny v Brne
      • Hradec Králové, Tsjekkia, 500 05
        • Fakultni nemocnice Hradec Kralove
      • Jihlava, Tsjekkia, 58633
        • Nemocnice Jihlava p.o.
      • Ostrava, Tsjekkia, 708 52
        • Fakultni Nemocnice Ostrava
  • Tyrkia (Türkiye)
      • Ankara, Tyrkia (Türkiye), 06100
        • Hacettepe University Medical Faculty
      • Antalya, Tyrkia (Türkiye), 07070
        • Akdeniz University Faculty of Medicine
  • Tyskland
    • Bavaria
      • Augsburg, Bavaria, Tyskland, 86156
        • Universitaetsklinikum Augsburg
      • Munich, Bavaria, Tyskland, 80337
        • Klinikum der Universitaet Muenchen
    • North Rhine-Westphalia
      • Bochum, North Rhine-Westphalia, Tyskland, 44791
        • Katholisches Klinikum Bochum gGmbH
  • Ungarn
      • Budapest, Ungarn, 1083
        • Semmelweis Egyetem
      • Budapest, Ungarn, 1089
        • Heim Pal Orszagos Gyermekgyogyaszati Intezet

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

10 år til 17 år (Barn)

Tar imot friske frivillige

Nei

Beskrivelse

Viktige inkluderingskriterier:

  • Må ha en kroppsvekt på ≥30 kg.
  • Må ha en diagnose RRMS (konsensusdefinisjon for pediatrisk RRMS [Krupp 2007]).
  • Må være ambulerende med en baseline EDSS-score mellom 0 og 5,5, inklusive.
  • Må ha opplevd minst 1 av følgende 3 tilstander: a) minst 1 tilbakefall i løpet av de siste 12 månedene før dag 1 med en tidligere hjerne-MR som viser lesjoner i samsvar med MS; b) minst 2 tilbakefall i løpet av de siste 24 månedene før dag 1, med en tidligere MR av hjernen som viser lesjoner i samsvar med MS; c) bevis på Gd-forsterkende lesjoner i hjernen på en MR utført innen 6 uker før dag 1.
  • Må være nevrologisk stabil, uten tegn på tilbakefall innen 50 dager før dag 1 og ingen tegn på kortikosteroidbehandling innen 30 dager før dag 1.
  • Fertile personer som er seksuelt aktive må være villige til å praktisere effektiv prevensjon under studien og være villige og i stand til å fortsette prevensjon i minst 30 dager etter sin siste dose av studiebehandlingen.

Nøkkelekskluderingskriterier:

  • Primær progressiv, sekundær progressiv eller progressiv residiverende MS (som definert av [Lublin og Reingold 1996]). Disse tilstandene krever tilstedeværelse av kontinuerlig klinisk sykdom som forverres over en periode på minst 3 måneder. Pasienter med disse tilstandene kan også ha overliggende tilbakefall, men skiller seg fra pasienter med tilbakefall-remitterende ved mangel på klinisk stabile perioder eller klinisk bedring.
  • Lidelser som etterligner MS, slik som andre demyeliniserende lidelser (f.eks. akutt disseminert encefalomyelitt), systemiske autoimmune lidelser (f.eks. Sjögrens sykdom, lupus erythematosus), metabolske forstyrrelser (f.eks. dystrofier) ​​og infeksjonssykdommer.
  • Anamnese med premalign eller ondartet sykdom. Personer med basalcellekarsinom som er fullstendig fjernet før screening vil fortsatt være kvalifisert.
  • Anamnese med alvorlige allergiske eller anafylaktiske reaksjoner, eller kjent medikamentoverfølsomhet overfor DMF, fumarsyreestere eller interferon beta-1a (IFN Beta-1a).
  • Historikk med unormale laboratorieresultater som indikerer enhver signifikant endokrinologisk, hematologisk, hepatisk, immunologisk, metabolsk, urologisk, nyre- og/eller annen alvorlig sykdom som ville utelukke deltakelse i en klinisk studie.

  • Anamnese med klinisk signifikant kardiovaskulær, pulmonal, GI, dermatologisk, vekst, utviklingsmessig, psykiatrisk (inkludert depresjon), nevrologisk (annet enn MS) og/eller annen alvorlig sykdom som vil utelukke deltakelse i en klinisk studie.

    -.Historie om humant immunsviktvirus.

  • Et MS-tilbakefall som har skjedd innen 50 dager før dag 1 OG/ELLER forsøkspersonen har ikke stabilisert seg fra et tidligere tilbakefall før dag 1.
  • Andre uspesifiserte grunner som etter Utforskerens eller Biogen Idecs oppfatning gjør faget uegnet for påmelding.
  • For PD/PK-undergruppen av forsøkspersoner: forsøkspersoner som ikke kan svelge BG00012-kapselen hel.

Nøkkel behandlingshistorie

  • Eventuell tidligere behandling med Fumaderm (fumarsyreestere) eller BG00012.
  • Tidligere behandling med noen av følgende: total lymfoid bestråling, kladribin, T-celle- eller T-cellereseptorvaksinasjon, ethvert terapeutisk monoklonalt antistoff, med unntak av rituximab eller natalizumab.
  • Tidligere behandling med noen av følgende medisiner innen 12 måneder før dag 1: mitoksantron, cyklofosfamid, rituximab.
  • Tidligere behandling med noen av følgende medisiner eller prosedyrer innen 6 måneder før dag 1: fingolimod; teriflunomid; natalizumab; cyklosporin; azatioprin; metotreksat; mykofenolatmofetil; laquinimod; intravenøst ​​(IV) immunglobulin; plasmaferese eller cytaferese
  • Behandling med noen av følgende medisiner innen 30 dager før dag 1: steroider (IV eller oral kortikosteroidbehandling, inkludert midler som kanskje ikke virker gjennom kortikosteroidveien [f.eks. lavdose naltrekson]), 4-aminopyridin eller relaterte produkter (unntatt forsøkspersoner) på en stabil dose fampridin med kontrollert frigjøring i 3 måneder)

MERK: Andre protokolldefinerte inkluderings-/ekskluderingskriterier kan gjelde

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: BG00012
Deltakerne vil motta den anbefalte dosen på 240 mg oralt, to ganger daglig
Legemiddel: dimetylfumarat
administrert oralt
Andre navn:
  • BG00012
  • Tecfidera
Aktiv komparator: IFN β-1a (Avonex)
Deltakerne vil motta den anbefalte dosen på 30 μg (ukentlig)
Legemiddel: Interferon β-la
administreres ved intramuskulær injeksjon
Andre navn:
  • Avonex

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Part 1: Proportion of Participants Free of New or Newly Enlarging T2 Hyperintense Lesions on Brain Magnetic Resonance Imaging (MRI) Scans
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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)

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Part 1: Number of New or Newly Enlarged T2 Hyperintense Lesions on Brain MRI Scans
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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)]
Tidsramme: 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)]
Tidsramme: 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)]
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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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Sponsor

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Etterforskere

  • Studieleder: Medical Director, Biogen

Publikasjoner og nyttige lenker

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Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

28. august 2014

Primær fullføring (Faktiske)

8. juli 2025

Studiet fullført (Faktiske)

8. juli 2025

Datoer for studieregistrering

Først innsendt

3. november 2014

Først innsendt som oppfylte QC-kriteriene

4. november 2014

Først lagt ut (Antatt)

5. november 2014

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

19. mai 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

16. mai 2026

Sist bekreftet

1. mai 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

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

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