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Studio di fase 3 sull'efficacia e la sicurezza di BG00012 in soggetti pediatrici con sclerosi multipla recidivante-remittente (SMRR) (CONNECT)

16 maggio 2026 aggiornato da: Biogen

Studio in aperto, randomizzato, multicentrico, a dosi multiple, con controllo attivo, a gruppi paralleli, di efficacia e sicurezza di BG00012 nei bambini di età compresa tra 10 e meno di 18 anni con sclerosi multipla recidivante-remittente, con estensione opzionale in aperto

Gli obiettivi principali della Parte 1 sono i seguenti: Valutare la sicurezza, la tollerabilità e l'efficacia di BG00012 in soggetti pediatrici con SMRR, rispetto a un trattamento modificante la malattia e valutare gli esiti di salute e l'evoluzione della disabilità. L'obiettivo principale della Parte 2 è valutare la sicurezza a lungo termine di BG00012 nei soggetti che hanno completato la Settimana 96 nella Parte 1 dello Studio 109MS306. L'obiettivo secondario della Parte 2 è descrivere gli esiti a lungo termine sulla SM di BG00012 nei soggetti che hanno completato la Settimana 96 nella Parte 1 dello Studio 109MS306.

Panoramica dello studio

Stato

Completato

Condizioni

Intervento / Trattamento

Tipo di studio

Interventistico

Iscrizione (Effettivo)

156

Fase

  • Fase 3

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Luoghi di studio

  • Belgio
      • Brussels, Belgio, 1020
        • Universitair Kinderziekenhuis Koningin Fabiola
      • Ghent, Belgio, 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
  • Cechia
      • Brno, Cechia, 656 91
        • Fakultni nemocnice u sv. Anny v Brne
      • Hradec Králové, Cechia, 500 05
        • Fakultni nemocnice Hradec Kralove
      • Jihlava, Cechia, 58633
        • Nemocnice Jihlava p.o.
      • Ostrava, Cechia, 708 52
        • Fakultni nemocnice Ostrava
  • Danimarca
      • Aarhus N, Danimarca, 8200
        • Århus Universitetshospital
      • Copenhagen, Danimarca, 2100
        • Rigshospitalet
      • Odense, Danimarca, 5000
        • Odense Universitetshospital
  • 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 - Hopital 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
        • Hôpital 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
  • Germania
    • Bavaria
      • Augsburg, Bavaria, Germania, 86156
        • Universitaetsklinikum Augsburg
      • Munich, Bavaria, Germania, 80337
        • Klinikum der Universitaet Muenchen
    • North Rhine-Westphalia
      • Bochum, North Rhine-Westphalia, Germania, 44791
        • Katholisches Klinikum Bochum gGmbH
  • Israele
      • Jerusalem, Israele, 91120
        • Hadassah University Hospital - Ein Kerem
      • Petah Tikva, Israele, 4920235
        • Schneider Children's Medical Center
      • Ramat Gan, Israele, 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 Gesù
    • 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'
  • Regno Unito
    • Greater London
      • London, Greater London, Regno Unito, WC1N 3BG
        • The National Hospital for Neurology & Neurosurgery
      • London, Greater London, Regno Unito, WC1N 3JH
        • Great Ormond Street Hospital For Children
      • London, Greater London, Regno Unito, SE1 7EH
        • Evelina London Children's Hospital
    • West Midlands
      • Birmingham, West Midlands, Regno Unito, 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
  • Spagna
      • Seville, Spagna, 41009
        • Hospital Universitario Virgen Macarena
    • Barcelona
      • Esplugues de Llobregat, Barcelona, Spagna, 8950
        • Hospital Sant Joan de Déu
    • Córdoba
      • Córdoba, Córdoba, Spagna, 14011
        • Hospital Universitario Reina Sofía
    • Madrid
      • Torrejón de Ardoz, Madrid, Spagna, 28850
        • Hospital Universitario De Torrejon
  • Stati Uniti
    • Massachusetts
      • Boston, Massachusetts, Stati Uniti, 2115
        • Boston Children's Hospital
    • Virginia
      • Charlottesville, Virginia, Stati Uniti, 22903
        • The Rector and Visitors of the University of Virginia
  • Svezia
      • Gothenburg, Svezia, 41345
        • Sahlgrenska Sjukhuset
      • Stockholm, Svezia, SE-113 61
        • Karolinska
  • Turchia (Türkiye)
      • Ankara, Turchia (Türkiye), 06100
        • Hacettepe University Medical Faculty
      • Antalya, Turchia (Türkiye), 07070
        • Akdeniz University Faculty of Medicine
  • Ungheria
      • Budapest, Ungheria, 1083
        • Semmelweis Egyetem
      • Budapest, Ungheria, 1089
        • Heim Pal Orszagos Gyermekgyogyaszati Intezet

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

Da 10 anni a 17 anni (Bambino)

Accetta volontari sani

No

Descrizione

Criteri chiave di inclusione:

  • Deve avere un peso corporeo ≥30 kg.
  • Deve avere una diagnosi di SMRR (definizione di consenso per SMRR pediatrica [Krupp 2007]).
  • Deve essere deambulante con un punteggio EDSS al basale compreso tra 0 e 5,5 inclusi.
  • Deve aver sperimentato almeno 1 delle seguenti 3 condizioni: a) almeno 1 ricaduta negli ultimi 12 mesi prima del Giorno 1 con una precedente risonanza magnetica cerebrale che dimostri lesioni coerenti con la SM; b) almeno 2 recidive negli ultimi 24 mesi prima del giorno 1, con una precedente risonanza magnetica cerebrale che dimostri lesioni coerenti con la SM; c) evidenza di lesioni cerebrali potenzianti il ​​Gd su una risonanza magnetica eseguita entro le 6 settimane precedenti il ​​giorno 1.
  • Deve essere neurologicamente stabile, senza evidenza di recidiva entro 50 giorni prima del Giorno 1 e nessuna evidenza di trattamento con corticosteroidi entro 30 giorni prima del Giorno 1.
  • I soggetti in età fertile che sono sessualmente attivi devono essere disposti a praticare una contraccezione efficace durante lo studio ed essere disposti e in grado di continuare la contraccezione per almeno 30 giorni dopo la dose finale del trattamento in studio.

Criteri chiave di esclusione:

  • SM primaria progressiva, secondaria progressiva o progressiva recidivante (come definita da [Lublin e Reingold 1996]). Queste condizioni richiedono la presenza di un continuo peggioramento clinico della malattia per un periodo di almeno 3 mesi. I soggetti con queste condizioni possono anche avere recidive sovrapposte ma si distinguono dai soggetti recidivanti-remittente per la mancanza di periodi clinicamente stabili o di miglioramento clinico.
  • Disturbi che mimano la SM, come altri disturbi demielinizzanti (per es., encefalomielite acuta disseminata), disturbi autoimmuni sistemici (per es., malattia di Sjögren, lupus eritematoso), disturbi metabolici (per es., distrofie) e malattie infettive.
  • Storia di malattia precancerosa o maligna. I soggetti con carcinoma basocellulare che è stato completamente asportato prima dello screening rimarranno idonei.
  • Anamnesi di gravi reazioni allergiche o anafilattiche o nota ipersensibilità al farmaco al DMF, agli esteri dell'acido fumarico o all'interferone beta-1a (IFN Beta-1a).
  • Anamnesi di risultati di laboratorio anormali indicativi di qualsiasi malattia endocrinologica, ematologica, epatica, immunologica, metabolica, urologica, renale e/o qualsiasi altra malattia importante che precluderebbe la partecipazione a uno studio clinico.

  • - Storia di malattie cardiovascolari, polmonari, gastrointestinali, dermatologiche, della crescita, dello sviluppo, psichiatriche (inclusa la depressione), neurologiche (diverse dalla SM) e/o altre malattie clinicamente significative che precluderebbero la partecipazione a uno studio clinico.

    -.Storia del virus dell'immunodeficienza umana.

  • Una recidiva di SM che si è verificata entro 50 giorni prima del Giorno 1 E/O il soggetto non si è stabilizzato da una precedente ricaduta prima del Giorno 1.
  • Altri motivi non specificati che, a giudizio dello Sperimentatore o di Biogen Idec, rendono il soggetto non idoneo all'arruolamento.
  • Per il sottogruppo di soggetti PD/PK: soggetti incapaci di deglutire la capsula BG00012 intera.

Cronologia del trattamento chiave

  • Qualsiasi precedente trattamento con Fumaderm (esteri dell'acido fumarico) o BG00012.
  • Trattamento precedente con uno qualsiasi dei seguenti: irradiazione linfoide totale, cladribina, vaccinazione contro i linfociti T o il recettore dei linfociti T, qualsiasi anticorpo monoclonale terapeutico, ad eccezione di rituximab o natalizumab.
  • Precedente trattamento con uno qualsiasi dei seguenti farmaci nei 12 mesi precedenti il ​​Giorno 1: mitoxantrone, ciclofosfamide, rituximab.
  • Precedente trattamento con uno qualsiasi dei seguenti farmaci o procedure entro 6 mesi prima del giorno 1: fingolimod; teriflunomide; natalizumab; ciclosporina; azatioprina; metotrexato; micofenolato mofetile; laquinimod; immunoglobulina endovenosa (IV); plasmaferesi o citaferesi
  • Trattamento con uno qualsiasi dei seguenti farmaci entro 30 giorni prima del giorno 1: steroidi (trattamento con corticosteroidi EV o orale, inclusi agenti che potrebbero non agire attraverso la via dei corticosteroidi [ad es. naltrexone a basso dosaggio]), 4-aminopiridina o prodotti correlati (ad eccezione con una dose stabile di fampridina a rilascio controllato per 3 mesi)

NOTA: potrebbero essere applicati altri criteri di inclusione/esclusione definiti dal protocollo

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: Randomizzato
  • Modello interventistico: Assegnazione parallela
  • Mascheramento: Nessuno (etichetta aperta)

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: BG00012
I partecipanti riceveranno la dose raccomandata di 240 mg per via orale, due volte al giorno
Droga: dimetilfumarato
somministrato per via orale
Altri nomi:
  • BG00012
  • Tecfidera
Comparatore attivo: IFNβ-1a (Avonex)
I partecipanti riceveranno la dose raccomandata di 30 μg (settimanale)
Droga: Interferone β-1a
somministrato per iniezione intramuscolare
Altri nomi:
  • Avonex

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Part 1: Proportion of Participants Free of New or Newly Enlarging T2 Hyperintense Lesions on Brain Magnetic Resonance Imaging (MRI) Scans
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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)

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Part 1: Number of New or Newly Enlarged T2 Hyperintense Lesions on Brain MRI Scans
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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)]
Lasso di tempo: 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)]
Lasso di tempo: 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)]
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Biogen

Investigatori

  • Direttore dello studio: Medical Director, Biogen

Pubblicazioni e link utili

La persona responsabile dell'inserimento delle informazioni sullo studio fornisce volontariamente queste pubblicazioni. Questi possono riguardare qualsiasi cosa relativa allo studio.

Pubblicazioni generali

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Effettivo)

28 agosto 2014

Completamento primario (Effettivo)

8 luglio 2025

Completamento dello studio (Effettivo)

8 luglio 2025

Date di iscrizione allo studio

Primo inviato

3 novembre 2014

Primo inviato che soddisfa i criteri di controllo qualità

4 novembre 2014

Primo Inserito (Stimato)

5 novembre 2014

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

19 maggio 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

16 maggio 2026

Ultimo verificato

1 maggio 2026

Maggiori informazioni

Termini relativi a questo studio

Parole chiave

Termini MeSH pertinenti aggiuntivi

Altri numeri di identificazione dello studio

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

Piano per i dati dei singoli partecipanti (IPD)

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SÌ

Descrizione del piano IPD

In conformità con la Politica sulla trasparenza delle sperimentazioni cliniche e sulla condivisione dei dati di Biogen disponibile su https://www.biogentrialtransparency.com/.

Informazioni su farmaci e dispositivi, documenti di studio

prodotto fabbricato ed esportato dagli Stati Uniti

No

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

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