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    Summary
    EudraCT Number:2019-003352-37
    Sponsor's Protocol Code Number:ALXN1210-NMO-307
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-01-14
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2019-003352-37
    A.3Full title of the trial
    A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD)
    Estudio multicéntrico, abierto, controlado con placebo externo y de fase III para evaluar la eficacia y la seguridad de ravulizumab en pacientes adultos
    con trastorno del espectro de neuromielitis óptica (NMOSD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical study testing the efficacy and safety of Ravulizumab in adults with NMOSD.
    Estudio clínico para probar la eficacia y la seguridad de ravulizumab en adultos con NMOSD
    A.3.2Name or abbreviated title of the trial where available
    A Phase 3 Efficacy and Safety Study of Ravulizumab in Adult Patients with NMOSD
    Estudio de eficacia y seguridad de fase III de ravulizumab en pacientes adultos con NMOSD
    A.4.1Sponsor's protocol code numberALXN1210-NMO-307
    A.5.4Other Identifiers
    Name:INDNumber:144,187
    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 Pharma Spain
    B.5.2Functional name of contact pointRosa Enrique
    B.5.3 Address:
    B.5.3.1Street AddressPº de Gracia,85
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08008
    B.5.3.4CountrySpain
    B.5.4Telephone number+34932723019
    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 Ultomiris
    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.3.1Product nameravulizumab
    D.3.2Product code ALXN1210
    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 INNRAVULIZUMAB
    D.3.9.1CAS number 1803171-55-2
    D.3.9.2Current sponsor codeALXN1210
    D.3.9.4EV Substance CodeSUB192773
    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) 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.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
    Trastorno del espectro de neuromielitis óptica
    E.1.1.1Medical condition in easily understood language
    NMOSD
    NMOSD
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10077875
    E.1.2Term Neuromyelitis optica spectrum disorder
    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
    To evaluate the effect of ravulizumab on adjudicated On-Trial Relapses in adult patients with NMOSD
    Evaluar el efecto de ravulizumab en recaídas durante el ensayo adjudicadas en pacientes adultos con NMOSD
    E.2.2Secondary objectives of the trial
    To evaluate the safety of ravulizumab in adult patients with NMOSD

    To evaluate the effect of ravulizumab on adjudicated annualized response rate (ARR) in adult patients with NMOSD

    To characterize the PK of ravulizumab in adult patients with NMOSD

    To characterize the pharmacodynamics (PD) of ravulizumab in adult patients with NMOSD
    Evaluar la seguridad de ravulizumab en pacientes adultos con NMOSD

    Evaluar el efecto de ravulizumab en la tasa de recaídas anualizada (TRA) adjudicada en pacientes adultos con NMOSD

    Caracterizar la farmacocinética (FC) de ravulizumab en pacientes adultos con NMOSD

    Caracterizar la farmacodinámica (FD) de ravulizumab en pacientes adultos con NMOSD
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patient must be 18 years of age or older, at the time of signing the informed consent. Anti-AQP4 Ab-positive and a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria (Wingerchuk, 2015).
    2. At least 1 attack or relapse in the last 12 months prior to the Screening Period NOTE: Patients with a single life-time attack will be considered to satisfy inclusion criterion #3 if the attack occurred in the last 12 months.
    3. Expanded Disability Status Scale (EDSS) score ≤ 7
    4. Patients who enter the trial receiving supportive IST (eg, corticosteroids, azathioprine [AZA], mycophenolate mofetil [MMF], methotrexate [MTX], and tacrolimus [TAC]) for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration prior to Screening with no plan to change the dose during the study period as follows:
    a. If patients who enter the study are receiving AZA, they must have been on AZA for ≥ 6 months and have been on a stable dose for ≥ 2 months prior to Screening.
    b. If patients who enter the study are receiving other ISTs (eg, MMF, MTX, or TAC), they must have been on the IST for ≥ 3 months and have been on a stable dose for ≥ 4 weeks prior to Screening.
    c. If patients who enter the study are receiving oral corticosteroids, they must have been on a stable dose for ≥ 4 weeks prior to Screening.
    d. If a patient enters the trial receiving oral corticosteroid(s) with or without other IST(s), the daily corticosteroid dose must be no more than prednisone 20 mg/day (or equivalent) prior to Screening.
    5. Vaccinated against N. meningitidis within 3 years prior to, or at the time of, initiating ravulizumab. 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.
    Please see the detailed list of inclusion criteria in the protocol.
    1. El paciente debe tener, al menos, 18 años en el momento de firmar el consentimiento informado. Ser positivo a anticuerpos y anti-AQP4 y tener un diagnóstico de NMOSD, según lo definido por los criterios de diagnóstico de consenso internacional de 2015 (Wingerchuk, 2015).
    2. Haber tenido al menos una crisis o recaída en los últimos 12 meses antes del período de selección. NOTA: Los pacientes con una sola crisis en toda su vida serán considerados para el cumplimiento del criterio de inclusión n.º 3 si dicha crisis tuvo lugar en los últimos 12 meses.
    3. Puntuación ≤ 7 en la Escala del estado de discapacidad ampliada (EDSS)
    4. Los pacientes que entren al ensayo mientras reciben TIS de apoyo (p. ej., corticosteroides, azatioprina [AZA], micofenolato mofetilo [MMF], metotrexato [MTX] y tacrolimús [TAC]) para la prevención de recaídas, ya sea en combinación o en monoterapia, deben estar recibiendo una pauta posológica estable de una duración adecuada antes de la selección sin tener previsto cambiar la dosis durante el período del estudio, del siguiente modo:
    a. Si los pacientes que ingresen en el estudio están recibiendo AZA, tienen que haberla recibido durante ≥ 6 meses y estar con una pauta posológica estable durante ≥ 2 meses antes de la selección.
    b. Si los pacientes que ingresen en el estudio están recibiendo otros TIS (p. ej., MMF, MTX o TAC), tienen que haberlos recibido durante ≥ 3 meses y estar recibiendo una dosis estable durante ≥ 4 semanas antes de la selección.
    c. Si los pacientes que ingresen en el estudio están recibiendo corticoesteroides por vía oral, tienen que estar recibiendo una dosis estable durante ≥ 4 semanas antes de la selección.
    d. Si un paciente ingresa en el estudio mientras recibe corticosteroides por vía oral con o sin TIS, la dosis diaria de corticosteroides debe ser inferior a 20 mg/día (o equivalente) de prednisona antes de la selección.
    5. Debe de estar vacunado contra la N. meningitidis en los 3 años anteriores o en el momento de empezar a recibir ravulizumab. Los pacientes que comiencen el tratamiento con el fármaco del estudio menos de dos semanas después de recibir una vacuna meningocócica deben recibir también antibióticos profilácticos adecuados hasta 2 semanas después de vacunarse
    Consulte la lista detallada de criterios de inclusión en el protocolo
    E.4Principal exclusion criteria
    1. History of N. meningitidis infection.
    2. Human immunodeficiency virus (HIV) infection (evidenced by HIV-1 or HIV-2 antibody titer)
    3. History of unexplained infections
    4. Active systemic bacterial, viral, or fungal infection within 14 days prior to study drug administration on Day 1
    5. Previously or currently treated with a complement inhibitor.
    6. Use of rituximab within 3 months prior to Screening
    7. Use of mitoxantrone within 3 months prior to Screening
    8. Use of Intravenous Immunoglobulin (IVIg) within 3 weeks prior to Screening
    9. Participation in any other investigational drug study or exposure to an investigational drug or device within 30 days of Screening or 5 half-lives of the study drug, whichever is greater
    10. Pregnant, breastfeeding, or intending to conceive during the course of the study
    Please see the detailed list of exclusion criteria in the protocol.
    1. Antecedentes de infección por N. meningitidis.
    2. Infección por el virus de inmunodeficiencia humana (VIH) (que se pone de manifiesto mediante valoración de anticuerpos contra el VIH-1 o VIH-2).
    3. Antecedentes de infecciones sin explicar.
    4. Infección bacteriana, vírica o fúngica sistémica y activa en los 14 días previos a la administración del fármaco del estudio el día 1.
    5. Haber estado o estar actualmente sometido a tratamiento con un inhibidor del complemento.
    6. Uso de rituximab en los 3 meses anteriores a la selección.
    7. Uso de mitoxantrona en los 3 meses anteriores a la selección.
    8. Uso de inmunoglobulina intravenosa (Ig i.v.) en las 3 semanas anteriores a la selección.
    9. Participación en cualquier otro estudio con un fármaco en investigación o exposición a un fármaco o dispositivo en investigación en los 30 días anteriores a la selección o 5 semividas del fármaco del estudio, lo que dure más.
    10. Estar embarazada, amamantando o intentando quedarse embarazada durante el transcurso del estudio
    Consulte la lista detallada de criterios de exclusion en el protocolo
    E.5 End points
    E.5.1Primary end point(s)
    Time to first adjudicated On-Trial Relapse and relapse risk reduction
    Tiempo hasta la primera recaída durante el ensayo adjudicada y reducción del riesgo de recaída
    E.5.1.1Timepoint(s) of evaluation of this end point
    On-Trial Relapses will be monitored throughout the study. The Investigator or a qualified designee will review the signs and symptoms of a potential relapse with the patient in detail at each visit.
    Las recaídas en el ensayo se controlarán durante todo el estudio. El investigador o un designado calificado revisará los signos y síntomas de una posible recaída con el paciente en detalle en cada visita.
    E.5.2Secondary end point(s)
    1. Adjudicated On Trial ARR
    2. Clinically important worsening in expanded disability status scale (EDSS)
    3. Change from baseline in EuroQoL-5D (EQ-5D)
    4. Clinically important change in Hauser ambulation index (HAI)
    5. Change in serum ravulizumab concentration over the study duration
    6. Change in serum free C5 concentration over the study duration
    7. Presence and titer of ADAs over the study duration
    1. TRA durante el ensayo adjudicada
    2. Empeoramiento clínicamente importante en la escala ampliada del estado de discapacidad (EDSS)
    3. Cambio respecto al inicio en EuroQoL-5D (EQ-5D)
    4. Cambio clínicamente importante en el índice de deambulación de Hauser (HAI)
    5. Cambio en la concentración sérica de ravulizumab durante el estudio
    6. Cambio en la concentración de C5 libre de suero durante el estudio
    7. Presencia y valor de anticuerpos antifármaco (AAF) durante el estudio
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Time of on-trial relapse
    2. Throughout the study
    3. Throughout the study
    4. Throughout the study
    5. Throughout the study
    6. Throughout the study
    7. Throughout the study
    1. En el momento de recaída durante el ensayo
    2. Durante todo el estudio
    3. Durante todo el estudio
    4. Durante todo el estudio
    5. Durante todo el estudio
    6. Durante todo el estudio
    7. Durante todo el estudio
    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
    Immunogenicity Assessments
    Evaluaciones de inmunogenicidad
    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 No
    E.8.1.2Open Yes
    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
    Placebo externo
    External Placebo
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Placebo externo
    External Placebo
    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 concerned9
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA35
    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
    Australia
    Austria
    Canada
    Denmark
    France
    Germany
    Italy
    Japan
    Korea, Republic of
    Netherlands
    Russian Federation
    Spain
    Turkey
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    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
    Date of the last visit of the last patient in the trial globally.
    Fecha de la última visita del último paciente en el ensayo a nivel global.
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    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: 50
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 18
    F.4.2.2In the whole clinical trial 55
    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)
    Ravulizumab will not be provided to the patients after the last scheduled dosing. All patients will be followed for safety for an additional 8 weeks after the last dose of study drug or early discontinuation.
    Ravulizumab no se proporcionará a los pacientes después de la última dosis programada. Todos los pacientes serán seguidos por seguridad durante 8 semanas adicionales después de la última dosis del fármaco del estudio o la interrupción temprana.
    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 Decision2020-02-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-02-18
    P. End of Trial
    P.End of Trial StatusOngoing
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