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    Summary
    EudraCT Number:2019-001829-26
    Sponsor's Protocol Code Number:ECU-NMO-303
    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:2021-01-22
    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 number2019-001829-26
    A.3Full title of the trial
    A Phase 2/3 Open-Label, Single-Arm Trial to Evaluate the Safety and Activity of Eculizumab in Pediatric Patients with Relapsing Neuromyelitis Optica Spectrum Disorder.
    Studio di fase II/III, in aperto e a braccio singolo volto a valutare la sicurezza e l’attività di eculizumab in pazienti pediatrici affetti da disturbo dello spettro della neuromielite ottica recidivante.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Trial to Evaluate the Safety and Activity of Eculizumab in Pediatric Patients with Relapsing Neuromyelitis Optica Spectrum Disorder (NMOSD).
    Studio sulla sicurezza e l’attività di eculizumab in pazienti pediatrici affetti da disturbo dello spettro della neuromielite ottica recidivante.
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    A.4.1Sponsor's protocol code numberECU-NMO-303
    A.5.4Other Identifiers
    Name:NANumber:NA
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/252/2019
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorALEXION PHARMACEUTICALS INCORPORATED
    B.1.3.4CountryUnited States
    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 supportAlexion Pharmaceuticals Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlexion Europe SAS
    B.5.2Functional name of contact pointEuropean Clinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address103-105 rue Anatole France
    B.5.3.2Town/ cityLevallois-Perret
    B.5.3.3Post code92300
    B.5.3.4CountryFrance
    B.5.4Telephone number0033147100615
    B.5.5Fax number0033147100611
    B.5.6E-mailclinicaltrials.eu@alexion.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 Yes
    D.2.1.1.1Trade name SOLIRIS - 300 MG CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO 1 FLACONCINO (VETRO) 30 ML (10 MG/ML)
    D.2.1.1.2Name of the Marketing Authorisation holderALEXION EUROPE S.A.S.
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/13/1185
    D.3 Description of the IMP
    D.3.1Product nameSoliris
    D.3.2Product code [Soliris]
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNECULIZUMAB
    D.3.9.1CAS number 219685-50-4
    D.3.9.2Current sponsor codeEculizumab
    D.3.9.4EV Substance CodeSUB25187
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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 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 Yes
    D.2.1.1.1Trade name MENVEO
    D.2.1.1.2Name of the Marketing Authorisation holderGSK Vaccines S.r.l.
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameMENVEO
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form Powder for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeJ07AH08
    D.3.9.3Other descriptive nameN. MENINGITIDIS GROUP W135 OLIGOSACCHARIDE CONJUGATED CRM197
    D.3.10 Strength
    D.3.10.1Concentration unit U/ml unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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) Yes
    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: 3
    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 Yes
    D.2.1.1.1Trade name Bexsero
    D.2.1.1.2Name of the Marketing Authorisation holderGSK Vaccines S.r.l
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameBexsero
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeBexsero
    D.3.9.3Other descriptive nameRECOMBINANT NEISSERIA MENINGITIDIS GROUP B NHBA FUSION PROTEIN PRODUCED IN E. COLI CELLS BY RECOMBINANT DNA TECHNOLOGY ADSORBED ON ALUMINIUM HYDROXIDE
    D.3.10 Strength
    D.3.10.1Concentration unit U/ml unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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) Yes
    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: 4
    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 Yes
    D.2.1.1.1Trade name Hiberix
    D.2.1.1.2Name of the Marketing Authorisation holderGlaxoSmithKline S.p.A.
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameHiberix
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive nameHAEMOPHILUS TYPE B CONJUGATE VACCINE (TETANUS TOXOID CONJUGATE)
    D.3.10 Strength
    D.3.10.1Concentration unit U/ml unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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) Yes
    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: 5
    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 Yes
    D.2.1.1.1Trade name Prevenar 13
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Europe MA EEIG
    D.2.1.2Country which granted the Marketing AuthorisationBelgium
    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 namePrevenar 13
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive namePNEUMOCOCCAL POLYSACCHARIDE CONJUGATE VACCINE (13-VALENT, ADSORBED) CONJUGATED TO CRM197 CARRIER PROTEIN AND ADSORBED ON ALUMINIUM PHOSPHATE
    D.3.10 Strength
    D.3.10.1Concentration unit U/ml unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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) Yes
    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: 6
    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 Yes
    D.2.1.1.1Trade name PNEUMOVAX
    D.2.1.1.2Name of the Marketing Authorisation holderMSD Italia S.r.l.
    D.2.1.2Country which granted the Marketing AuthorisationBelgium
    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 namePNEUMOVAX
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive namePNEUMOVAX 23 is a vaccine indicated for active immunization for the prevention of pneumococcal disease caused by the 23 serotypes contained in the vaccine (1, 2, 3, 4, 5, 6B, 7F, 8, 9N, 9V, 10A, 11A, 12F, 14, 15B, 17F, 18C, 19F, 19A, 20, 22F, 23F, and 33F).
    D.3.10 Strength
    D.3.10.1Concentration unit U/ml unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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) Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Neuromyelitis Optica Spectrum Disorder (NMOSD)
    Disturbo dello spettro della neuromielite ottica (NMOSD)
    E.1.1.1Medical condition in easily understood language
    Neuromyelitis Optica Spectrum Disorder (NMOSD)
    Disturbo dello spettro della neuromielite ottica (NMOSD)
    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 LLT
    E.1.2Classification code 10077879
    E.1.2Term Neuromyelitis optica spectrum disorder relapse
    E.1.2System Organ Class 100000004852
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • Evaluate the efficacy of eculizumab in relapsing pediatric patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) in the Primary Treatment Period.
    • Valutare l’efficacia di eculizumab in pazienti pediatrici recidivanti affetti da disturbo dello spettro della neuromielite ottica (NMOSD) nel periodo di trattamento primario.
    E.2.2Secondary objectives of the trial
    Primary Treatment Period:
    • Evaluate the safety and tolerability of eculizumab treatment in
    relapsing pediatric patients with NMOSD.
    • Evaluate the efficacy of eculizumab by additional efficacy measures
    including: disease-related disability, quality of life and change in
    ophthalmologic examination findings.
    • Describe the pharmacokinetics (PK) and pharmacodynamics (PD) of
    eculizumab in relapsing pediatric patients with NMOSD.
    Extension Treatment Period:
    • Characterize long-term safety of eculizumab treatment in pediatric
    patients with NMOSD.
    • Characterize long-term efficacy of eculizumab treatment in pediatric
    patients with NMOSD.
    • Valutare la sicurezza e la tollerabilità del trattamento con eculizumab in pazienti pediatrici recidivanti affetti da NMOSD.
    • Valutare l’efficacia di eculizumab tramite misurazioni di efficacia aggiuntive, quali:
    • Disabilità correlata alla malattia
    • Qualità della vita
    • Variazione nei risultati degli esami oftalmologici
    • Descrivere la farmacocinetica (Pharmacokinetics, PK) e la farmacodinamica (Pharmacodynamics, PD) di eculizumab in pazienti pediatrici recidivanti affetti da NMOSD.
    • Caratterizzare l’efficacia a lungo termine del trattamento con eculizumab in pazienti pediatrici affetti da NMOSD.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    assent/consent.
    • Anti-AQP4 Ab-positive and diagnosis of NMOSD as defined by the 2015
    IPND criteria
    • Historical Relapse Rate of at least 2 relapses in the last 2 years, and
    with at least 1 relapse in the year prior to Screening.
    • Expanded Disability Status Scale score = 7
    • Patients who enter the study receiving supportive immunosuppressive
    therapies for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration
    prior to Screening.
    • Vaccinated against N meningitidis within 3 years prior to, or at the
    time of initiating eculizumab. Patients who initiate study drug treatment
    less than 2 weeks after receiving a meningococcal vaccine must receive
    appropriate prophylactic antibiotics until 2 weeks after the vaccination.
    • Documented vaccination against Hib and S pneumoniae infections 2
    weeks prior to dosing as per local and country-specific immunization
    guidelines for the appropriate age group.
    • Willing and capable to give informed assent (if applicable, as
    determined by the central Institutional Review Boards
    (IRBs)/Independent Ethics Committees (IECs) and in accordance with
    local requirements) and whose parent/legal guardian are willing and
    able to give written informed consent, which includes compliance with
    the requirements and restrictions listed in the informed consent form
    (ICF) and in the protocol.
    - Pazienti maschi p femmine di età compresa tra 2 e <18 anni al tempo dell'assenso/consenso
    - Anti-AQP4Ab positivo e diagnosi di NMOSD come definito dai criteri IPND 2015
    - Storia di ricadute, almeno 2 negli ultimi 2 anni, e con almeno 1 ricaduta nell'anno precedente allo screening.
    - Expanded Disability Status Scale score = 7
    - I pazienti che entrano nello studio mentre ricevono terapie di supporto immunosoppressive per la prevenzione delle recidive, sia in combinazione che in monoterapia, devono essere in dosaggio stabile per un'adeguata durata prima dello screening.
    - Vaccinati contro N meningitidis entro i 3 anni prima dello screening o nel momento in cui iniziano il trattament ocon Eculizumab. I pazienti che iniziano il trattamento meno di 2 settimane dopo aver ricevuto il vaccino menigococcico devono ricevere una profilassi antibiotica appropriata fino a 2 settimane dopo la vaccinazione.
    - Vaccinazione documentata contro Hib e S pneumoniae 2 settimane prima del trattamento, secondo la legislazione locale in base all'età del gruppo di trattamento.
    - Volontà e capacità di dare il proprio assenso informato e i cui genitori/tutori siano in grado di fornire il proprio consenso informato per iscritto.
    E.4Principal exclusion criteria
    • Patients known to be human immunodeficiency virus (HIV) positive or
    with congenital immunodeficiency.
    • Unresolved meningococcal or other serious infection.
    • Any unresolved acute or chronic systemic bacterial or other infection
    which is clinically significant in the opinion of the Investigator and has
    not been treated with appropriate antibiotics.
    • Hypersensitivity to murine proteins or to one of the excipients of
    eculizumab.
    • Use of rituximab or other biologicals such as tocilizumab within 6
    months prior to Screening.
    • Use of mitoxantrone within 3 months prior to Screening.
    • Use of intravenous immunoglobulin (IVIg) or plasma exchange (PE)
    within 3 weeks prior to Screening.
    • Use of immunomodulatory therapies for multiple sclerosis within 3
    months prior to Screening.
    - Pazienti con diagnosi di Infezione da HIV o con immunodeficenza congenita
    - Infezione meningococcica p di altro tipo seria e non risolta
    - Qualsiasi infezione batterica acuta o cronica non risolta, che sia clinicamente significativa nell'opinione dello Sperimentatore e che non sia stata trattata con antibiotici appropriati.
    - Ipersensibilità a proteine murine o a qualcun odegli eccipienti di Eculizumab.
    - Uso di Rituximab o altri biologici come Tocilizumab entro i 6 mesi prima dello screening.
    - Uso di mitoxantrone entro i 3 mesi prima dello screening.
    - Uso di Rintravenous immunoglobulin (IVIg) o plasma exchange (PE) entro 3 settimane prima dello screening.
    - Uso di terapie immunomodulatorie pre la sclerosi multipla entro 3 mesi prima dello screening.
    E.5 End points
    E.5.1Primary end point(s)
    • Change from Baseline in the Annualized Relapse Rate (ARR).
    • Time to First Relapse (TFR).
    • Variazione rispetto al basale del tasso di recidive annualizzato (Annualized Relapse Rate, ARR)
    • Tempo alla prima recidiva (Time to First Relapse, TFR).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Primary Treatment Period:
    throughout the primary treatment period
    and
    52/53* Weeks
    * For patients who start in weight cohort 10 to <20 kg, the one-year
    evaluation will occur on Week 53, an odd-numbered week, because
    maintenance dosing, which is administered every 2 weeks, begins on
    Week 1. For all other cohorts the one-year evaluation will occur on Week
    52, because maintenance dosing begins on an even-numbered week.
    Hence, the end of the Primary Treatment Period is designated as Week
    52/53 throughout the study protocol.
    Periodo di trattamento primario:
    durante il periodo di trattamento primario e 52/53 settimane.
    Per pazienti che partono nella coorte 10<20 kg, la valutazione ad 1 anno occorrerrà alla settimana 53, in una settimana dispari, poiché la dose di mantenimento, che viene somministrata ogni 2 settimane, comincia alla settimana 1. Per tutte le latre coorti, la valutazione ad 1 anno occorrerà alla settimana 52, perché la dose di mantenimento conincerà in una settimana pari.
    Quindi, la fine del periodo di trattamento primario è indicata come la settimana 52/53 in accordo con il protocollo
    E.5.2Secondary end point(s)
    Primary Treatment Period:
    Safety:
    • Incidence of treatment-emergent adverse events (TEAEs), serious
    adverse events (SAEs), and adverse events leading to study drug
    • Incidence of antidrug antibodies (ADA).
    • Changes from Baseline in vital signs, electrocardiogram (ECG)
    parameters, and clinical laboratory assessments.
    • Change from Baseline in both weight and height.
    Efficacy:
    Disease-related disability:
    • Change from Baseline in Expanded Disability Status Scale (EDSS) score
    in patients =5 years of age.
    • Change from Baseline in the Hauser Ambulatory Index (HAI) score.
    Quality of life:
    • Change from Baseline in Pediatric Quality of Life Inventory (PedsQL) in
    patients =5 years of age.
    • Change from Baseline in Pediatric Quality of Life Inventory Parent
    Proxy (PedsQL Parent Proxy) in patients < 5 years of age.
    Change in ophthalmologic examination findings:
    • Change from Baseline in Visual Acuity (VA) as measured by the Snellen
    or LEA symbols Eye Chart examination in all patients.
    • Change from Baseline in Confrontational Visual Fields (VF) as
    measured during ophthalmologic examination in all patients.
    • Change from Baseline in color vision as measured during
    ophthalmologic examination in all patients.
    PK/PD:
    • Changes in serum eculizumab concentration over time.
    • Changes in serum free complement protein 5 (C5) concentrations and
    in vitro hemolytic activity over time.
    Extension Treatment Period:
    The safety and efficacy endpoints of the Primary Treatment Period will
    be evaluated during the Extension Treatment Period.
    • Incidenza degli eventi avversi che emergono in corso di trattamento (Treatment-Emergent Adverse Event, TEAE), eventi avversi gravi (Serious Adverse Event, SAE) ed eventi avversi che conducono a interrompere l’assunzione del farmaco sperimentale.
    • Incidenza di anticorpi antifarmaco (Antidrug Antibody, ADA).
    • Variazioni rispetto al basale dei parametri vitali, dei parametri dell’elettrocardiogramma (ECG) e delle valutazioni cliniche di laboratorio.
    • Variazioni rispetto al basale sia di peso che di altezza
    • Variazione rispetto al basale del punteggio della Scala della disabilità espansa (Expanded Disability Status Scale, EDSS) a 52/53 settimane in pazienti =5 anni di età.
    • Variazione rispetto al basale del punteggio dell’indice di deambulazione di Hauser (Hauser Ambulation Index, HAI) a 52/53 settimane.
    • Variazione rispetto al basale dell’inventario della qualità della vita pediatrica (Pediatric Quality of Life Inventory, PedsQL) a 52/53 settimane in pazienti =5 anni di età.
    • Variazione rispetto al basale dell’inventario della qualità della vita pediatrica delegato al genitore (PedsQL Parent Proxy) a 52/53 settimane in pazienti <5 anni di età.
    • Variazione rispetto al basale dell’acuità visiva (AV) misurata tramite l’esame delle tavole ottometriche di Snellen o dei simboli LEA a 52/53 settimane in tutti i pazienti.
    • Variazione rispetto al basale dei campi di visione (Visual Field, VF) confrontazionali misurata nell’esame oftalmologico a 52/53 settimane in tutti i pazienti.
    • Variazione rispetto al basale della visione a colori misurata nell’esame oftalmologico a 52/53 settimane in tutti i pazienti.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Primary Treatment Period:
    throughout the primary treatment period
    and
    At 52/53* weeks
    * For patients who start in weight cohort 10 to <20 kg, the one-year
    evaluation will occur on Week 53, an odd-numbered week, because
    maintenance dosing, which is administered every 2 weeks, begins on
    Week 1. For all other cohorts the one-year evaluation will occur on Week
    52, because maintenance dosing begins on an even-numbered week.
    Hence, the end of the Primary Treatment Period is designated as Week
    52/53 throughout the study protocol.
    Extension Treatment Period:
    104 weeks
    Periodo di trattamento primario:
    durante il periodo di trattamento primario e 52/53 settimane.
    Per pazienti che partono nella coorte 10<20 kg, la valutazione ad 1 anno occorrerrà alla settimana 53, in una settimana dispari, poiché la dose di mantenimento, che viene somministrata ogni 2 settimane, comincia alla settimana 1. Per tutte le latre coorti, la valutazione ad 1 anno occorrerà alla settimana 52, perché la dose di mantenimento conincerà in una settimana pari.
    Quindi, la fine del periodo di trattamento primario è indicata come la settimana 52/53 in accordo con il protocollo.

    Periodo di estensione del trattamento:
    104 settimane
    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
    Tolerability and immunogenicity
    Tollerabilità e immunogenicità
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    In aperto
    Open
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    Canada
    Japan
    Korea, Republic of
    United States
    Germany
    Italy
    Spain
    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
    LPLV
    LPLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days15
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days15
    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: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 5
    F.4.2.2In the whole clinical trial 15
    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)
    The Patients will return to the care of their physician for their standard treatment.
    Il paziente tornerà alla terapia standard sotto la supervisione del medico di famiglia.
    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 Decision2020-06-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-03-02
    P. End of Trial
    P.End of Trial StatusOngoing
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