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    Summary
    EudraCT Number:2013-001150-10
    Sponsor's Protocol Code Number:ECU-NMO-301
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-08-08
    Trial results View 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 ConcernedSpain - AEMPS
    A.2EudraCT number2013-001150-10
    A.3Full title of the trial
    A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTI-CENTER TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF ECULIZUMAB IN PATIENTS WITH RELAPSING NEUROMYELITIS OPTICA (NMO)
    Ensayo multicéntrico, aleatorizado, a doble ciego y controlado con placebo para evaluar la seguridad y la eficacia de eculizumab en pacientes con neuromielitis óptica (NMO) recidivante
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    STUDY OF ECULIZUMAB IN RELAPSING NEUROMYELITIS OPTICA SUBJECTS
    A.4.1Sponsor's protocol code numberECU-NMO-301
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAlexion Pharmaceuticals, Inc.
    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 Address25 boulevard de l'Amiral Bruix
    B.5.3.2Town/ cityParis
    B.5.3.3Post code75016
    B.5.3.4CountryFrance
    B.5.4Telephone number+33153 643941
    B.5.5Fax number+33153 643820
    B.5.6E-mailclinicaltrials.eu@alxn.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
    D.2.1.1.2Name of the Marketing Authorisation holderALEXION EUROPE SAS
    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.3.4Pharmaceutical 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 codeh5G1.1-mAb
    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 No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    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 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.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboConcentrate for solution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Relapsing neuromyelitis optica
    Neuromielitis óptica recidivante
    E.1.1.1Medical condition in easily understood language
    Neuromyelitis optica
    Neuromielitis óptica
    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 16.0
    E.1.2Level PT
    E.1.2Classification code 10029322
    E.1.2Term Neuromyelitis optica
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Efficacy of eculizumab treatment as compared with placebo in relapsing NMO patients based on the time to first relapse and relapse risk reduction.
    Evaluar la eficacia del tratamiento con eculizumab en comparación con un placebo en pacientes con NMO recidivante en función del tiempo transcurrido hasta la primera recidiva y la reducción del riesgo de recidivas.
    E.2.2Secondary objectives of the trial
    - Safety and tolerability of eculizumab compared with placebo
    - Efficacy of eculizumab compared with placebo
    - Describe the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab
    - Evaluar la seguridad y la tolerabilidad generales de eculizumab en comparación con placebo en pacientes con NMO recidivante
    - Evaluar la eficacia de eculizumab en comparación con placebo
    - Describir la farmacocinética (FC) y la farmacodinámica (FD) de eculizumab
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male or female patient greater than or equal to 18 years old
    - Diagnosis of NMO or NMO spectrum disorder
    - NMO-IgG seropositive at screening visit
    - Historical Relapse of at least 2 in last 6 months or 3 in the last 12 months with the most recent attack occurring ?30 days and less than or equal to 90 days prior to Randomization
    - EDSS score less than or equal to 7
    - If patients enter the trial receiving an IST, they must be on the IST on which they had the most recent relapse and must remain at the same dose thoughout the trial
    - Patients must be willing and able to give written informed consent
    - Pacientes varones o mujeres maior ou igual que 18 años de edad
    - Diagnóstico de NMO o trastorno del espectro seropositivo
    - Seropositividad para la IgG de NMO en la visita de selección
    - Recidivas históricas (según la definición de este protocolo) de al menos 2 en los últimos 6 meses o de 3 en los últimos 12 meses, con la crisis más reciente producida maior ou igual que 30 días y menor ou igual que 90 días antes de la aleatorización
    - Puntuación en la EDSS menor ou igual que 7
    - Si los pacientes ingresan en el ensayo recibiendo un TID, deberán seguir con el TID con el que hayan tenido la recidiva más reciente, y la pauta posológica del TID mencionado deberá ser la misma que en el momento de la recidiva más reciente, sin que se permita la adición de ningún nuevo TID tras la recidiva
    - Los pacientes deben otorgar su consentimiento informado por escrito
    E.4Principal exclusion criteria
    - Use of rituximab or mitoxantrone within 3 months prior to randomization
    - Use of IVIg within 3 weeks prior to screening
    - Has received treatment with eculizumab at any time prior to enrolling in this study
    - Consumo de rituximab en los 3 meses previos a la aleatorización
    - Consumo de IgIV en las 3 semanas anteriores a la selección
    - Haber recibido tratamiento previo con eculizumab
    E.5 End points
    E.5.1Primary end point(s)
    Time to first relapse
    tiempo transcurrido hasta la primera recidiva
    E.5.1.1Timepoint(s) of evaluation of this end point
    Relapse evaluation visits:
    - Within 24/48 hours after relapse
    - Week +1 after relapse
    - Week +4 after relapse
    - Week +6 after relapse

    Completion of the trial after 29 relapses in 29 distinct patients.
    E.5.2Secondary end point(s)
    - Change from baseline in EDSS score at the End of Study Period
    - Annualized relapse rate
    - Change from baseline in EQ-5D at the End of the Study Period
    - Change from baseline in modified Rankin Scale (mRS) score at the End of the Study Period
    - Change from baseline in ambulatory function as measured by Hauser Ambulation Index at the End of the Study Period.
    - Cambio con respecto a la línea basal en la puntuación de la EDSS al final del periodo del estudio
    - Tasa anualizada de recidivas
    - Cambio con respecto a la línea basal en la puntuación de EQ-5D (Cuestionario europeo de calidad de vida de 5 dimensiones) al final del periodo del estudio
    - Cambio con respecto a la línea basal en la puntuación de la escala de Rankin modificada (mRS) al final del periodo del estudio
    - Cambio con respecto a la línea basal en la actividad ambulatoria medida por el índice ambulatorio de Hauser al final del periodo del estudio.
    E.5.2.1Timepoint(s) of evaluation of this end point
    evaluation at:
    - Screening visit
    - Weeks 1, 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, 104
    - Relapse evaluation visits
    - End of trial visit
    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 No
    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 No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA25
    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 No
    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
    Austria
    Belgium
    Brazil
    Canada
    Chile
    Finland
    France
    Germany
    Hong Kong
    Italy
    Japan
    Korea, Republic of
    Peru
    Russian Federation
    Saudi Arabia
    Singapore
    Spain
    Sweden
    Switzerland
    Taiwan
    Turkey
    United Arab Emirates
    United Kingdom
    United States
    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
    Last patient last visit, expected to occur when one of the following conditions is met, whichever comes first:
    - 20 relapse in 20 distinct patients are observed,
    - The maximum number of relapses (29 relapses) is reached
    última visita al último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 84
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 9
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 21
    F.4.2.2In the whole clinical trial 93
    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)
    Subjects completing the study with no major protocol violations will be offered the opportunity to participate in an extension trial (separate protocol) to receive eculizumab after completion of the End of Study Period Visit. Subjects who prematurely discontinue will return to the care of their physician.
    A los pacientes se les ofrecerá la oportunidad de participar en un ensayo de extensión (protocolo independiente) para recibir eculizumab una vez finalizada la visita de final del periodo del estudio. Los pacientes que abandonen prematuramente el estudio volverán al cuidado de su médico habitual.
    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 Decision2013-10-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-09-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-07-17
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