Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43871   clinical trials with a EudraCT protocol, of which   7290   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2020-004431-24
    Sponsor's Protocol Code Number:67896153MSC3001
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-09-14
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2020-004431-24
    A.3Full title of the trial
    Multi-center, Randomized, Double-blind, Parallel-group, Double-dummy, Active-controlled, Comparative Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Ponesimod Versus Fingolimod During 108 Weeks of Treatment in Pediatric Participants, 10 to <18 Years Old, with Relapsing-remitting Multiple Sclerosis
    Studio comparativo, multicentrico, randomizzato, in doppio cieco, a gruppi paralleli, a doppia simulazione, con controllo attivo, volto a valutare l’efficacia, la sicurezza, la farmacocinetica e la farmacodinamica di ponesimod rispetto a fingolimod nel corso di 108 settimane di trattamento in partecipanti pediatrici, di età compresa tra 10 e <18 anni, con sclerosi multipla recidivante-remittente
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Clinical Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Ponesimod Versus Fingolimod During 108 Weeks of Treatment in Pediatric Participants, 10 to <18 Years Old, with Relapsing-remitting Multiple Sclerosis
    Uno studio clinico per valutare l'efficacia, la sicurezza, la farmacocinetica e la farmacodinamica di Ponesimod rispetto a Fingolimod nel corso di 108 settimane di trattamento in partecipanti pediatrici, di età compresa tra 10 e <18 anni, con sclerosi multipla recidivante-remittente
    A.3.2Name or abbreviated title of the trial where available
    PIONEER
    PIONEER
    A.4.1Sponsor's protocol code number67896153MSC3001
    A.5.4Other Identifiers
    Name:Investigational New Drug (IND)Number:101722
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/066/2021
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorJANSSEN CILAG INTERNATIONAL NV
    B.1.3.4CountryBelgium
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportJanssen Research & Development, LLC
    B.4.2CountryUnited States
    B.4.1Name of organisation providing supportJANSSEN CILAG SPA
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationJanssen-Cilag International NV
    B.5.2Functional name of contact pointClinical Registry Group
    B.5.3 Address:
    B.5.3.1Street AddressArchimedesweg 29
    B.5.3.2Town/ cityLeiden
    B.5.3.3Post code2333CM
    B.5.3.4CountryNetherlands
    B.5.6E-mailClinicalTrialsEU@its.jnj.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namePonesimod
    D.3.2Product code [JNJ-67896153/ACT-128800]
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPonesimod
    D.3.9.1CAS number 854107-55-4
    D.3.9.2Current sponsor codeJNJ-67896153
    D.3.9.3Other descriptive nameACT-128800
    D.3.9.4EV Substance CodeSUB182605
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Gilenya hard capsules
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameFingolimod
    D.3.2Product code [.]
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNFINGOLIMOD
    D.3.9.1CAS number 162359-55-9
    D.3.9.2Current sponsor code.
    D.3.9.4EV Substance CodeSUB31908
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number0.5
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Relapsing-remitting Multiple Sclerosis
    Sclerosi multipla recidivante-remittente
    E.1.1.1Medical condition in easily understood language
    Chronic inflammatory disease attacking the CNS
    Malattia infiammatoria cronica che attacca il Sistema Nervoso Centrale
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10063399
    E.1.2Term Relapsing-remitting multiple sclerosis
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to demonstrate that ponesimod is not less effective than fingolimod in reducing relapses in pediatric participants with relapsing remitting multiple sclerosis (RRMS) if treatment is taken as directed through Week 108
    Dimostrare che ponesimod non è meno efficace di fingolimod nel ridurre le recidive nei partecipanti pediatrici con sclerosi multipla recidivante-remittente (SMRR) se il trattamento viene assunto come indicato fino alla Settimana 108
    E.2.2Secondary objectives of the trial
    The secondary objectives are:
    To assess the effect of ponesimod vs fingolimod on the annualized relapse rate (ARR) in pediatric participants with RRMS, as measured by the rate ratio of ponesimod to fingolimod
    To assess the effect of ponesimod on MS disability accumulation and magnetic resonance imaging (MRI) lesion accrual in comparison with fingolimod in pediatric participants with RRMS
    To evaluate the safety, tolerability, and pharmacodynamics (PD) of ponesimod in comparison with fingolimod in pediatric participants with RRMS
    To evaluate the pharmacokinetics(PK) of ponesimod in pediatric participants with RRMS
    To study the PK/PD relationship for key efficacy and safety outcomes in pediatric participants with RRMS treated with ponesimod
    Valutare l’effetto di ponesimod rispetto a fingolimod sull’ARR nei partecipanti pediatrici con SMRR, come misurato dal rapporto tra tassi di ponesimod rispetto a fingolimod
    Valutare l’effetto di ponesimod sull’accumulo di disabilità da SM e di lesioni visibili dalla risonanza magnetica (RMI) rispetto a fingolimod nei partecipanti pediatrici con SMRR
    Valutare la sicurezza, la tollerabilità e la farmacodinamica (PD) di ponesimod rispetto a fingolimod nei partecipanti pediatrici con SMRR
    Valutare la farmacocinetica (PK) di ponesimod nei partecipanti pediatrici con SMRR
    Studiare la relazione PK/PD per i risultati chiave di efficacia e sicurezza nei partecipanti pediatrici con SMRR trattati con ponesimod
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives

    Other types of substudies
    Specify title, date and version of each substudy with relative objectives: Open-Label PK sub-study will be conducted in parallel to the main study
    Objective: To determine if a maintenance dose of < 20 mg ponesimod in pediatric population with body weight <= 40 kg results in PK exposure and PD effects comparable to those achieved in adult participants with RMS

    Altre tipologie di sottostudi
    specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Il sotto-studio PK in aperto sarà condotto in parallelo allo studio principale.
    Obiettivo: Determinare se una dose di mantenimento di < 20 mg di ponesimod nella popolazione pediatrica con peso corporeo <= 40 kg determina un'esposizione PK ed effetti PDparagonabili a quelli ottenuti in partecipanti adulti con RMS
    E.3Principal inclusion criteria
    - A diagnosis of relapsing-remitting multiple sclerosis (RRMS) as defined by the 2017 revisions of the McDonald criteria. Central review of the diagnosis of pediatric multiple sclerosis (MS) will be required for all participants prior to Visit 3 (Day 1, randomization).

    - Expanded Disability Status Scale (EDSS) score between 0 and 5.5 (inclusive).

    - At least 1 MS relapse during the previous year or 2 MS relapses in the previous 2 years or evidence of gadolinium-enhancing (Gd+) lesions on magnetic resonance imaging (MRI) 6 months prior to Visit 3 (Day 1, Randomization), including MRI at Visit 2 (Baseline).

    - Must be otherwise healthy per investigator clinical judgement on the basis of physical examination, medical history, vital signs, laboratory tests, and electrocardiogram (ECGs) at Visit 1 (Screening). Any documented abnormalities must be consistent with pediatric MS. This determination must be recorded in participant’s source documents and initiated by Principal investigator (PI).

    - Participants with body weight or body surface area between 5th and 95th percentile for age and sex. Overweight participants greater than 95th percentile and underweight participants less than 5th percentile may participate following medical clearance.

    Refer protocol for all inclusion criteria.
    Per essere arruolato nello studio, ogni potenziale partecipante deve soddisfare tutti i criteri seguenti.
    Età
    1. Età compresa tra 10 e <18 anni alla Visita 3 (Giorno 1, Randomizzazione; o per il sottostudio di PK - inizio del periodo di trattamento).
    Tipo di partecipante e caratteristiche della malattia
    2. Una diagnosi di RRMS definita secondo le revisioni dei criteri di McDonald del 2017 (Thompson 2018). Sarà necessaria una revisione a livello centrale della diagnosi di SM pediatrica per tutti i partecipanti prima della Visita 3 (Giorno 1, randomizzazione).
    3. Punteggio della Scala estesa dello stato di disabilità (EDSS) compreso tra 0 e 5,5 (compreso).
    4. Almeno 1 recidiva di SM durante l’anno precedente o 2 recidive di SM nei 2 anni precedenti o evidenza di lesioni Gd+ alla RMI 6 mesi prima della Visita 3 (Giorno 1, Randomizzazione), compresa la RMI alla Visita 2 (basale).
    5. Deve essere altrimenti sano secondo il parere clinico dello sperimentatore in base all’esame obiettivo, all’anamnesi medica, ai segni vitali, ai test di laboratorio e all’ECG eseguiti alla Visita 1 (Screening). Eventuali anomalie documentate devono essere coerenti con la SM pediatrica. Questa determinazione deve essere registrata nei documenti originali del partecipante e avviata dallo sperimentatore principale.
    Peso
    6. Partecipanti con peso corporeo o superficie corporea compresa tra il 5° e il 95° percentile per età e sesso. I partecipanti con sovrappeso superiore al 95° percentile e i partecipanti con sottopeso inferiore al 5° percentile possono partecipare previa autorizzazione medica.
    Requisiti relativi a sesso e contraccettivi
    7. Sesso maschile o femminile (in base agli organi riproduttivi e alle funzioni assegnate dal complemento cromosomico).
    8. Deve soddisfare lo stadio di Tanner per la rispettiva fascia di età.
    9. Una donna in età fertile deve presentare un risultato negativo del test sul siero altamente sensibile betagonadotropina corionica umana [beta-hCG]) alla Visita 1 (Screening) e un test di gravidanza sulle urine negativo alla Visita 2 (Basale).
    10. Una donna deve essere:
    a. non in età fertile;
    b. in età fertile e non eterosessualmente attiva, oppure
    c. in età fertile, eterosessualmente attiva e deve adottare un metodo contraccettivo altamente efficace, preferibilmente indipendente dall’utilizzatore (tasso di fallimento <1% all’anno se utilizzato in modo costante e corretto, in linea con le normative locali). I partecipanti devono accettare di continuare a utilizzare un metodo altamente efficace durante il trattamento dello studio e fino a 60 giorni dopo l’ultima dose - la fine dell’esposizione sistemica pertinente. Definizioni ed esempi di metodi contraccettivi altamente efficaci si trovano nella sezione 10.5, Appendice 5: Guida alla contraccezione.
    Nota: Se, dopo l’inizio dello studio, il potenziale di fertilità varia (ad es., una partecipante di sesso femminile in fase premenarcale ha il menarca) o varia il rischio di gravidanza (ad es., una partecipante di sesso femminile non eterosessualmente attiva diventa attiva), la partecipante deve discuterne con lo sperimentatore, che deve stabilire se una partecipante di sesso femminile deve iniziare a utilizzare un metodo contraccettivo altamente efficace. Se lo stato riproduttivo è dubbio, occorre considerare ulteriori valutazioni.

    Per l'elenco completo dei criteri di inclusione si prega di fare riferimento al Protocollo di Studio
    E.4Principal exclusion criteria
    - Presenting with a diagnosis of MS with progressive course from onset (that is, primary progressive MS or progressive relapsing MS).

    - Widespread and symmetric white matter alterations in the baseline MRI suggestive of other demyelinating disorders (example, metabolic disorders, mitochondrial disorders).

    - Tested positive for aquaporin 4 (AQP4) or anti-myelin oligodendrocyte glycoprotein (MOG) antibodies at Visit 1 (Screening).

    - Pregnant (defined as the state of a female after conception and until the termination of gestation, confirmed by a positive Human Chorionic Gonadotropin laboratory test), or breastfeeding, or planning to become pregnant while enrolled in this study.

    - Any condition for which, in the opinion of the investigator, participation would not be in the best interest of the participant (example, compromise the well-being) or that could prevent, limit, or confound the protocol-specified assessments.

    Refer protocol for all exclusion criteria.
    Qualsiasi potenziale partecipante che soddisfi uno qualunque dei seguenti criteri sarà escluso dalla partecipazione allo studio:
    Criteri correlati alla SM
    1. Presentare una diagnosi di SM con decorso progressivo dall’insorgenza (ovvero, SM progressiva primaria o SM progressiva recidivante).
    2. Alterazioni della sostanza bianca diffuse e simmetriche nella RM al basale, suggestive di altri disturbi demielinizzanti (ad es., disturbi metabolici, disturbi mitocondriali).
    3. Risultato positivo per gli anticorpi anti-acquaporina 4 (AQP4) o anti-glicoproteina oligodendrocitaria mielinica (MOG) alla Visita 1 (Screening).
    Terapia precedente/concomitante
    4. Trattato con:
    • Interferone (IFN) ¿-1a, IFN ¿-1b, peg-IFN ß-1a o glatiramer acetato nei 30 giorni precedenti la randomizzazione,
    • Corticosteroidi sistemici o ormone adrenocorticotropo (ACTH) 30 giorni prima della RM al basale,
    • Dimetilfumarato, diroximel fumurato o monometilfumarato nei 30 giorni precedenti la randomizzazione,
    • plasmaferesi, citaferesi nei 90 giorni precedenti la randomizzazione;
    • Immunoglobulina ad alto dosaggio 90 giorni prima della randomizzazione,
    • Natalizumab nei 90 giorni o teriflunomide nei 106 giorni precedenti la randomizzazione (a meno che non sia stata eseguita una procedura di eliminazione accelerata per teriflunomide, abbreviando il periodo di washout a 14 giorni),
    • Farmaci immunosoppressori/immunomodulatori, ad es. azatioprina, metotrexato, laquinimod, rituximab, ofatumumab, ocrelizumab nei 6 mesi precedenti la randomizzazione,
    • Alemtuzumab, cladribina, ciclofosfamide o mitoxantrone in qualsiasi momento,
    • Fingolimod o altri modulatori del recettore S1P in qualsiasi momento,
    • Antiaritmici di classe Ia (ad es. chinidina, disopiramide) o classe III (ad es. amiodarone, sotalolo) alla Visita 1 (Screening),
    • Trattamento concomitante con farmaci che abbassano la frequenza cardiaca (HR) nei 15 giorni precedenti la randomizzazione (ad es.,beta-bloccanti, calcio-antagonisti [ad es., verapamil, diltiazem o ivabradina], digossina, agenti anticolinesterasici, pilocarpina [vedere elenco non esaustivo dei farmaci fornito nella Tabella 2 nella Sezione 10.9, Appendice 9: antiaritmici proibiti]. È necessario consultare un cardiologo pediatrico riguardo al passaggio a farmaci non antiaritmici
    • Trapianto di cellule staminali in qualsiasi momento;
    • Un trattamento sperimentale (compresi i vaccini sperimentali) o utilizzo di un dispositivo medico sperimentale invasivo nei 180 giorni precedenti la randomizzazione o 5 emivite prima della randomizzazione, a seconda di quale periodo sia più lungo.

    Per l'elenco completo dei criteri di esclusione si prega di fare riferimento al Protocollo di Studio
    E.5 End points
    E.5.1Primary end point(s)
    Time from randomization to first confirmed relapse
    Tempo dalla randomizzazione alla prima recidiva confermata
    E.5.1.1Timepoint(s) of evaluation of this end point
    From Randomization to Week 108
    Dalla randomizzazione alla settimana 108
    E.5.2Secondary end point(s)
    a. ARR, defined as the number of confirmed relapses per participant year
    b. Time to 12- and 24-week confirmed disability accumulation as measured by Expanded Disability Status Scale (EDSS) score
    c. Annualized rate of new/newly enlarging T2 lesions
    d. Adverse events (AEs), including AEs of special interest (AESI)
    e. Electrocardiogram (ECG) parameters, including heart rate (HR), intervals for PR, QRS, QT, and QTcF
    f. Pulmonary function test (PFT), including forced expiratory volume in 1 second (FEV1), and forced vital capacity (FVC)
    g. Blood pressure
    h. Laboratory values, including alanine aminotransferase (ALT), aspartate aminotransferase (AST), and bilirubin
    i. Change in body weight/height
    j. Suicidality assessment using the Columbia-Suicide Severity Rating Scale (C-SSRS)
    k. Peripheral blood lymphocyte counts
    l. Population PK parameters
    ARR, definito come il numero di recidive confermate per partecipante all’anno
    Tempo dalla randomizzazione alla prima recidiva confermata
    RMI:
    • Tasso annualizzato di lesioni nuove/ingrandite in T2
    • Variazione del numero di lesioni in T1 captanti gadolinio (Gd+) dal basale alla settimana 24, 48 e 108
    • Variazione del volume delle lesioni in T1 Gd+ dal basale alla Settimana 24, 48 e 108
    • Percentuale di partecipanti senza lesioni in T1 Gd+ fino alla Settimana 108
    • Numero cumulativo di lesioni attive uniche combinate (CUAL) dal basale alla Settimana 108
    • Proporzione di partecipanti privi di CUAL alla fine del trattamento (EOT) e per visita (ossia, Settimana 24, 48 e 108)
    • Eventi avversi (AE), compresi gli AE di interesse speciale (AESI)
    • Parametri dell’elettrocardiogramma (ECG), tra cui frequenza cardiaca (HR), intervalli PR, QRS, QT e QTcF
    • Test di funzionalità polmonare (PFT), compreso il volume espiratorio forzato in 1 secondo (FEV1) e la capacità vitale forzata (FVC)
    • Pressione arteriosa
    • Valori di laboratorio, tra cui alanina aminotransferasi (ALT), aspartato aminotransferasi (AST) e bilirubina
    • Variazione nel peso corporeo/altezza dal baseline fino alla fine dello studio (EOS)
    • Valutazione del rischio di suicidio mediante la scala C-SSRS (Columbia-Suicide Severity Rating Scale)
    • Conte linfocitarie del sangue periferico il Giorno 1 e alle Settimane 4 e 12
    • Sottostudio PK: parametri PK di popolazione (AUC, Cmax, Tmax, t1/2)
    • Studio principale: parametri PK di popolazione, come AUC e Cmax (se i dati lo consentono)
    E.5.2.1Timepoint(s) of evaluation of this end point
    At timepoints as specified within the protocol
    Ai timepoint specificati nel protocollo
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Biomarker evaluation, Tolerability, analysis of MRI parameters, cognitive function, fatigue and Health-related quality of life
    Valutazione dei biomarcatori, tollerabilità, analisi dei parametri di risonanza magnetica, funzione cognitiva, fatica e qualità di vita.
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Double-dummy
    Double-dummy
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned8
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA58
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Argentina
    Australia
    Brazil
    Canada
    Israel
    Peru
    Puerto Rico
    Taiwan
    United States
    Austria
    Estonia
    Finland
    France
    Poland
    Sweden
    Bulgaria
    Spain
    Switzerland
    Czechia
    Germany
    Greece
    Italy
    Belgium
    Croatia
    Hungary
    Portugal
    Slovakia
    Turkey
    United Kingdom
    Serbia
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days2
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 22
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 190
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Pediatric population
    Popolazione pediatrica
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state17
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 92
    F.4.2.2In the whole clinical trial 212
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    A long-term extension study may be offered to participants who complete the 108-week double blind treatment (or the 108-week open-label treatment for PK sub-study participants)
    I soggetti che completano il periodo di trattamento di 108 settimane potranno scegliere di partecipare alla fase di estensione in aperto dello studio che sarà descritta successivamente in un protocollo separato.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-12-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-01-11
    P. End of Trial
    P.End of Trial StatusOngoing
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sat May 04 21:01:32 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA