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Phase-3-Studie zur Wirksamkeit und Sicherheit von BG00012 bei pädiatrischen Patienten mit schubförmig remittierender Multipler Sklerose (RRMS) (CONNECT)

16. Mai 2026 aktualisiert von: Biogen

Offene, randomisierte, multizentrische, mehrfach dosierte, aktiv kontrollierte Parallelgruppen-Wirksamkeits- und Sicherheitsstudie von BG00012 bei Kindern im Alter von 10 bis unter 18 Jahren mit schubförmig remittierender Multipler Sklerose, mit optionaler offener Verlängerung

Die Hauptziele von Teil 1 sind wie folgt: Bewertung der Sicherheit, Verträglichkeit und Wirksamkeit von BG00012 bei pädiatrischen Patienten mit RRMS im Vergleich zu einer krankheitsmodifizierenden Behandlung und Bewertung der gesundheitlichen Folgen und der Entwicklung der Behinderung. Das Hauptziel von Teil 2 ist die Bewertung der Langzeitsicherheit von BG00012 bei Patienten, die Woche 96 in Teil 1 der Studie 109MS306 abgeschlossen haben. Das sekundäre Ziel von Teil 2 ist die Beschreibung der langfristigen MS-Ergebnisse von BG00012 bei Patienten, die Woche 96 in Teil 1 der Studie 109MS306 abgeschlossen haben.

Studienübersicht

Status

Abgeschlossen

Bedingungen

Intervention / Behandlung

Studientyp

Interventionell

Einschreibung (Tatsächlich)

156

Phase

  • Phase 3

Kontakte und Standorte

Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.

Studienorte

  • Belgien
      • Brussels, Belgien, 1020
        • Universitair Kinderziekenhuis Koningin Fabiola
      • Ghent, Belgien, 9000
        • Universitair Ziekenhuis Ghent
  • Bulgarien
      • Sofia, Bulgarien, 1113
        • MHATNP 'Sv.Naum', EAD
  • Deutschland
    • Bavaria
      • Augsburg, Bavaria, Deutschland, 86156
        • Universitaetsklinikum Augsburg
      • Munich, Bavaria, Deutschland, 80337
        • Klinikum der Universitaet Muenchen
    • North Rhine-Westphalia
      • Bochum, North Rhine-Westphalia, Deutschland, 44791
        • Katholisches Klinikum Bochum gGmbH
  • Dänemark
      • Aarhus N, Dänemark, 8200
        • Århus Universitetshospital
      • Copenhagen, Dänemark, 2100
        • Rigshospitalet
      • Odense, Dänemark, 5000
        • Odense Universitetshospital
  • Frankreich
    • Bas Rhin
      • Strasbourg, Bas Rhin, Frankreich, 67098
        • CHU Strasbourg - Hôpital Hautepierre
    • Bouches-du-Rhône
      • Marseille, Bouches-du-Rhône, Frankreich, 13385
        • Hôpital de la Timone
    • Côte-d'Or
      • Dijon, Côte-d'Or, Frankreich, 21079
        • CHU Dijon -BOCAGE CENTRAL
    • Herault
      • Montpellier, Herault, Frankreich, 34295
        • Hôpital Gui de Chauliac
    • Ille Et Vilaine
      • Rennes, Ille Et Vilaine, Frankreich, 35033
        • CHU Rennes - Hôpital Pontchaillou
    • Meurthe Et Moselle
      • Vandœuvre-lès-Nancy, Meurthe Et Moselle, Frankreich, 54500
        • Hôpital de Brabois Enfants
    • Nord
      • Lille, Nord, Frankreich, 59037
        • Hopital Roger Salengro - CHU Lille
    • Puy De Dome
      • Clermont-Ferrand, Puy De Dome, Frankreich, 63003
        • CHU Clermont Ferrand - Hopital d'Estaing
    • Rhone
      • Bron, Rhone, Frankreich, 69677
        • Hopital Neurologique Pierre Wertheimer
    • Somme
      • Amiens, Somme, Frankreich, 80054
        • CHU Amiens - Hopital Sud
    • Val De Marne
      • Le Kremlin-Bicêtre, Val De Marne, Frankreich, 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
  • Italien
      • Bari, Italien, 70124
        • Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
      • Genova, Italien, 16132
        • IRCCS Ospedale Policlinico San Martino
      • Milan, Italien, 20132
        • Ospedale San Raffaele
      • Naples, Italien, 80131
        • Azienda Ospedaliera Universitaria 'Federico II'
      • Padova, Italien, 35128
        • Azienda Ospedale-Università di Padova
      • Palermo, Italien, 90127
        • Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone
      • Roma, Italien, 00189
        • Azienda Ospedaliera Sant'Andrea-Università di Roma La Sapienza
      • Roma, Italien, 00165
        • Ospedale Pediatrico Bambino Gesu
    • Varese
      • Gallarate, Varese, Italien, 21013
        • Azienda Socio Sanitaria Territoriale della Valle Olona (presidio di Gallarate)
  • Kanada
    • Alberta
      • Calgary, Alberta, Kanada, T3B 6A8
        • University of Calgary - Alberta Children's Hospital
  • 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'
  • Schweden
      • Gothenburg, Schweden, 41345
        • Sahlgrenska Sjukhuset
      • Stockholm, Schweden, SE-113 61
        • Karolinska
  • Serbien
      • Belgrade, Serbien, 11000
        • Clinic of Neurology and Psychiatry for Children and Youth
      • Belgrade, Serbien, 11000
        • Mother and Child Health Care Institute of Serbia ,,Dr Vukan Cupic''
      • Kragujevac, Serbien, 34000
        • University Clinical Center Kragujevac
  • Spanien
      • Seville, Spanien, 41009
        • Hospital Universitario Virgen Macarena
    • Barcelona
      • Esplugues de Llobregat, Barcelona, Spanien, 8950
        • Hospital Sant Joan de Déu
    • Córdoba
      • Córdoba, Córdoba, Spanien, 14011
        • Hospital Universitario Reina Sofia
    • Madrid
      • Torrejón de Ardoz, Madrid, Spanien, 28850
        • Hospital Universitario De Torrejon
  • Tschechien
      • Brno, Tschechien, 656 91
        • Fakultni nemocnice u sv. Anny v Brne
      • Hradec Králové, Tschechien, 500 05
        • Fakultni nemocnice Hradec Kralove
      • Jihlava, Tschechien, 58633
        • Nemocnice Jihlava p.o.
      • Ostrava, Tschechien, 708 52
        • Fakultni Nemocnice Ostrava
  • Türkei (türkiye)
      • Ankara, Türkei (türkiye), 06100
        • Hacettepe University Medical Faculty
      • Antalya, Türkei (türkiye), 07070
        • Akdeniz University Faculty of Medicine
  • Ungarn
      • Budapest, Ungarn, 1083
        • Semmelweis Egyetem
      • Budapest, Ungarn, 1089
        • Heim Pal Orszagos Gyermekgyogyaszati Intezet
  • Vereinigte Staaten
    • Massachusetts
      • Boston, Massachusetts, Vereinigte Staaten, 2115
        • Boston Children's Hospital
    • Virginia
      • Charlottesville, Virginia, Vereinigte Staaten, 22903
        • The Rector and Visitors of the University of Virginia
  • Vereinigtes Königreich
    • Greater London
      • London, Greater London, Vereinigtes Königreich, WC1N 3BG
        • The National Hospital for Neurology & Neurosurgery
      • London, Greater London, Vereinigtes Königreich, WC1N 3JH
        • Great Ormond Street Hospital for Children
      • London, Greater London, Vereinigtes Königreich, SE1 7EH
        • Evelina London Children's Hospital
    • West Midlands
      • Birmingham, West Midlands, Vereinigtes Königreich, B4 6NH
        • Birmingham Children's Hospital

Teilnahmekriterien

Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.

Zulassungskriterien

Studienberechtigtes Alter

10 Jahre bis 17 Jahre (Kind)

Akzeptiert gesunde Freiwillige

Nein

Beschreibung

Wichtige Einschlusskriterien:

  • Muss ein Körpergewicht von ≥ 30 kg haben.
  • Muss eine Diagnose von RRMS haben (Konsensdefinition für pädiatrische RRMS [Krupp 2007]).
  • Muss gehfähig sein mit einem EDSS-Basiswert zwischen 0 und 5,5, einschließlich.
  • Muss mindestens 1 der folgenden 3 Zustände erlebt haben: a) mindestens 1 Rückfall innerhalb der letzten 12 Monate vor Tag 1 mit einem vorherigen MRT des Gehirns, das Läsionen zeigt, die mit MS übereinstimmen; b) mindestens 2 Schübe innerhalb der letzten 24 Monate vor Tag 1, wobei ein vorheriges MRT des Gehirns Läsionen zeigt, die mit MS übereinstimmen; c) Nachweis von Gd-anreichernden Läsionen des Gehirns in einer MRT, die innerhalb von 6 Wochen vor Tag 1 durchgeführt wurde.
  • Muss neurologisch stabil sein, ohne Anzeichen eines Rückfalls innerhalb von 50 Tagen vor Tag 1 und ohne Anzeichen einer Kortikosteroidbehandlung innerhalb von 30 Tagen vor Tag 1.
  • Probanden im gebärfähigen Alter, die sexuell aktiv sind, müssen bereit sein, während der Studie eine wirksame Empfängnisverhütung anzuwenden, und bereit und in der Lage sein, die Empfängnisverhütung mindestens 30 Tage nach ihrer letzten Dosis der Studienbehandlung fortzusetzen.

Wichtige Ausschlusskriterien:

  • Primär progrediente, sekundär progrediente oder progrediente schubförmige MS (wie von [Lublin und Reingold 1996] definiert). Diese Erkrankungen erfordern das Vorliegen einer kontinuierlichen klinischen Verschlechterung der Erkrankung über einen Zeitraum von mindestens 3 Monaten. Personen mit diesen Zuständen können auch überlagerte Schübe haben, unterscheiden sich jedoch von Personen mit schubförmiger Remission durch das Fehlen klinisch stabiler Perioden oder klinischer Besserung.
  • Erkrankungen, die MS nachahmen, wie andere demyelinisierende Erkrankungen (z. B. akute disseminierte Enzephalomyelitis), systemische Autoimmunerkrankungen (z. B. Sjögren-Krankheit, Lupus erythematodes), Stoffwechselerkrankungen (z. B. Dystrophien) und Infektionserkrankungen.
  • Anamnese einer prämalignen oder bösartigen Erkrankung. Probanden mit Basalzellkarzinom, das vor dem Screening vollständig entfernt wurde, bleiben teilnahmeberechtigt.
  • Vorgeschichte schwerer allergischer oder anaphylaktischer Reaktionen oder bekannter Arzneimittelüberempfindlichkeit gegen DMF, Fumarsäureester oder Interferon beta-1a (IFN Beta-1a).
  • Vorgeschichte abnormaler Laborergebnisse, die auf eine signifikante endokrinologische, hämatologische, hepatische, immunologische, metabolische, urologische, renale und/oder andere schwere Krankheit hinweisen, die die Teilnahme an einer klinischen Studie ausschließen würde.

  • Vorgeschichte klinisch signifikanter kardiovaskulärer, pulmonaler, GI-, dermatologischer, Wachstums-, Entwicklungs-, psychiatrischer (einschließlich Depressionen), neurologischer (außer MS) und/oder anderer schwerer Erkrankungen, die die Teilnahme an einer klinischen Studie ausschließen würden.

    -.Geschichte des menschlichen Immunschwächevirus.

  • Ein MS-Schub, der innerhalb von 50 Tagen vor Tag 1 aufgetreten ist UND/ODER sich der Patient gegenüber einem früheren Schub vor Tag 1 nicht stabilisiert hat.
  • Andere nicht näher bezeichnete Gründe, die nach Meinung des Prüfarztes oder von Biogen Idec den Probanden für die Einschreibung ungeeignet machen.
  • Für die PD/PK-Untergruppe von Probanden: Probanden sind nicht in der Lage, die BG00012-Kapsel als Ganzes zu schlucken.

Schlüssel Behandlungsgeschichte

  • Jede vorherige Behandlung mit Fumaderm (Fumarsäureester) oder BG00012.
  • Vorherige Behandlung mit einem der folgenden: Gesamtlymphoidbestrahlung, Cladribin, T-Zell- oder T-Zell-Rezeptor-Impfung, alle therapeutischen monoklonalen Antikörper, mit Ausnahme von Rituximab oder Natalizumab.
  • Vorherige Behandlung mit einem der folgenden Medikamente innerhalb der 12 Monate vor Tag 1: Mitoxantron, Cyclophosphamid, Rituximab.
  • Vorherige Behandlung mit einem der folgenden Medikamente oder Verfahren innerhalb von 6 Monaten vor Tag 1: Fingolimod; Teriflunomid; Natalizumab; Cyclosporin; Azathioprin; Methotrexat; Mycophenolatmofetil; laquinimod; intravenöses (IV) Immunglobulin; Plasmapherese oder Cytapherese
  • Behandlung mit einem der folgenden Medikamente innerhalb von 30 Tagen vor Tag 1: Steroide (IV oder orale Kortikosteroidbehandlung, einschließlich Wirkstoffe, die möglicherweise nicht über den Kortikosteroidweg wirken [z. B. niedrig dosiertes Naltrexon]), 4-Aminopyridin oder verwandte Produkte (außer Probanden auf einer stabilen Dosis von Fampridin mit kontrollierter Freisetzung für 3 Monate)

HINWEIS: Andere protokolldefinierte Einschluss-/Ausschlusskriterien können gelten

Studienplan

Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.

Wie ist die Studie aufgebaut?

Designdetails

  • Hauptzweck: Behandlung
  • Zuteilung: Zufällig
  • Interventionsmodell: Parallele Zuordnung
  • Maskierung: Keine (Offenes Etikett)

Waffen und Interventionen

Teilnehmergruppe / Arm
Intervention / Behandlung
Experimental: BG00012
Die Teilnehmer erhalten zweimal täglich die empfohlene Dosis von 240 mg oral
Arzneimittel: Dimethylfumarat
oral verabreicht
Andere Namen:
  • BG00012
  • Tecfidera
Aktiver Komparator: IFN-β-1a (Avonex)
Die Teilnehmer erhalten die empfohlene Dosis von 30 μg (wöchentlich)
Arzneimittel: Interferon β-1a
durch intramuskuläre Injektion verabreicht
Andere Namen:
  • Avonex

Was misst die Studie?

Primäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Part 1: Proportion of Participants Free of New or Newly Enlarging T2 Hyperintense Lesions on Brain Magnetic Resonance Imaging (MRI) Scans
Zeitfenster: 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)
Zeitfenster: 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
Zeitfenster: 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)

Sekundäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Part 1: Number of New or Newly Enlarged T2 Hyperintense Lesions on Brain MRI Scans
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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)]
Zeitfenster: 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)]
Zeitfenster: 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)]
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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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Studienaufzeichnungsdaten

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Haupttermine studieren

Studienbeginn (Tatsächlich)

28. August 2014

Primärer Abschluss (Tatsächlich)

8. Juli 2025

Studienabschluss (Tatsächlich)

8. Juli 2025

Studienanmeldedaten

Zuerst eingereicht

3. November 2014

Zuerst eingereicht, das die QC-Kriterien erfüllt hat

4. November 2014

Zuerst gepostet (Geschätzt)

5. November 2014

Studienaufzeichnungsaktualisierungen

Letztes Update gepostet (Tatsächlich)

19. Mai 2026

Letztes eingereichtes Update, das die QC-Kriterien erfüllt

16. Mai 2026

Zuletzt verifiziert

1. Mai 2026

Mehr Informationen

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Schlüsselwörter

Zusätzliche relevante MeSH-Bedingungen

Andere Studien-ID-Nummern

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

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