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    Summary
    EudraCT Number:2019-000969-21
    Sponsor's Protocol Code Number:MG0004
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Restarted
    Date on which this record was first entered in the EudraCT database:2019-11-11
    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 number2019-000969-21
    A.3Full title of the trial
    A Randomized, Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
    Estudio de extensión, aleatorizado y abierto, para investigar la seguridad, tolerabilidad y eficacia a largo plazo de rozanolixizumab en pacientes adultos con miastenia gravis generalizada
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate the long-term safety, tolerability, and efficacy of rozanolixizumab in adult patients with generalized myasthenia gravis
    Estudio para investigar la seguridad, tolerabilidad y eficacia a largo plazo de rozanolixizumab en pacientes adultos con miastenia gravis generalizada.
    A.3.2Name or abbreviated title of the trial where available
    MycarinGstudy
    A.4.1Sponsor's protocol code numberMG0004
    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 SponsorUCB Biopharma SPRL
    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 supportUCB Biopharma SPRL
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUCB BIOSCIENCES GmbH
    B.5.2Functional name of contact pointClin Trial Reg & Results Disclosure
    B.5.3 Address:
    B.5.3.1Street AddressAlfred-Nobel-Strasse 10
    B.5.3.2Town/ cityMonheim
    B.5.3.3Post code40789
    B.5.3.4CountryGermany
    B.5.6E-mailclinicaltrials@ucb.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 nameRozanolixizumab
    D.3.2Product code UCB7665
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNrozanolixizumab
    D.3.9.1CAS number 1584645-37-3
    D.3.9.2Current sponsor codeUCB7665
    D.3.9.4EV Substance CodeSUB166282
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number140
    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
    Generalized myasthenia gravis
    Miastenia gravis generalizada
    E.1.1.1Medical condition in easily understood language
    Myasthenia gravis is an autoimmune disease that causes weakness in your muscles; it is caused by a communication problem between nerves and muscles.
    La Miastenia Gravis es una enfermedad autoimmune que causa debilidad en los músculos; esta causada por problemas de comunicación entre nervios y músculos.
    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 10028417
    E.1.2Term Myasthenia gravis
    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
    Evaluate the long-term safety and tolerability of rozanolixizumab in study participants with generalized myasthenia gravis (MG)
    Evaluar la seguridad y tolerabilidad a largo plazo de rozanolixizumab en sujetos con miastenia gravis generalizada (MG)
    E.2.2Secondary objectives of the trial
    Evaluate the long-term efficacy of rozanolixizumab in study participants with generalized myasthenia gravis (MG)
    Evaluar la eficacia a largo plazo de rozanolixizumab en sujetos con miastenia gravis generalizada (MG)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Participant must be >= 18 years of age at the time of signing the informed consent
    - Participant was eligible for MG0003 [NCT03971422] at the time of enrollment into MG0003 and the participant either completed the Observation Period of MG0003 or required rescue therapy during the Observation Period of MG0003
    - Body weight >35 kg at Visit 1
    - Study participants may be male or female
    - A male study participant must agree to use contraception
    - Female study participants of childbearing potential must agree to use a highly effective method of birth control
    - A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:
    a) Not a woman of childbearing potential (WOCBP) OR
    b) A WOCBP who agrees to follow the contraceptive guidance
    - El participante debe tener 18 años en el momento de firmar el consentimiento informado
    - El participante era elegible para el estudio MG0003 [NCT03971422] en el momento que fue reclutado en el MG0003 y, o bien completó el período de observación del MG0003, o bien requirió terapia de rescate durante el período de observación del MG0003
    - Peso corporal >35 kg en La Visita 1
    - Los participantes del estudio pueden ser hombres o mujeres
    - Los varones participantes de estudio deben aceptar usar métodos anticonceptivos
    - Las mujeres participantes de estudio en edad fértil deben estar de acuerdo en utilizar un método anticonceptivo altamente eficaz
    - Una mujer participante es elegible para participar si no está embarazada, no está amamantando, y se da al menos una de las siguientes condiciones:
    a) No es una mujer en edad fértil ó
    b) Es una mujer en edad fértil que acepta seguir la guía anticonceptiva
    E.4Principal exclusion criteria
    - Evidence of active or latent tuberculosis (TB) as documented by medical history and examination, if applicable, chest X-rays (posterior anterior and lateral), and TB testing by a positive (not indeterminate) QuantiFERON®-TB Gold test
    - Participant has received a live vaccination within 8 weeks prior to the Baseline visit; or intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of study medication
    - Study participant has experienced hypersensitivity reaction after exposure to other antineonatal Fc receptor (FcRn) drugs
    - Study participant with severe (defined as Grade 3 on the myasthenia gravis-activates of daily living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis
    - Participant has absolute neutrophil count <1500 cells/mm3
    - Participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at randomization, and could jeopardize or compromise the study participant’s ability to participate in this study
    - Participant has 12-lead electrocardiogram (ECG) with findings considered to be clinically significant upon medical review. The clinical significance of the findings needs to be assessed by the Investigator to determine eligibility, and any queries regarding continuation of the study participant must be addressed with the Medical Monitor
    - Study participant has renal impairment, defined as serum creatinine level of ≥1.4 mg/dL for females and ≥1.5mg/dL for males
    - Study participant has >2x upper limit of normal (ULN) of any of the following at Visit 1: alanine aminotransferase (ALT), aspartate aminotransferase (AST), alkaline phosphatase (ALP), or bilirubin >1.5xULN (isolated bilirubin >1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin <35%). If study participant has elevations only in total bilirubin that are >ULN and <1.5xULN, fractionate bilirubin to identify possible undiagnosed Gilbert’s syndrome (ie, direct
    bilirubin <35%). For randomized study participants with a baseline result >ULN for ALT, AST, ALP, or total bilirubin but <1.5xULN, a baseline diagnosis and/or the cause of any clinically meaningful elevation must be understood and recorded in the electronic Case Report form (eCRF). Tests that result in ALT, AST, or ALP up to 25% above the exclusion limit (>2xULN) may be repeated once for confirmation
    - Study participant has positive human immunodeficiency virus antibody test
    - Study participant met any mandatory withdrawal or mandatory study drug discontinuation criteria MG0003 [NCT03971422] or discontinued study medication in MG0003, with the exception of discontinuation due to a need for rescue treatment
    - Study participant is not considered capable of adhering to the protocol visit schedule, or medication intake according to the judgment of the Investigator
    - Study participant has a lifetime history of suicide attempt (including an active attempt, interrupted attempt, or aborted attempt), or had suicidal ideation since the last visit in MG0003 as indicated by a positive response (Yes) to either Question 4 or Question 5 of the ColumbiaSuicide Severity Rating Scale (C-SSRS) at Screening
    - Evidencia de tuberculosis (TB) activa o latente documentada mediante historia clínica y examen, si procede, radiografías de tórax (anteroposterior y lateral) y pruebas de TB mediante una prueba de QuantiFERON®-TB Gold positiva (no indeterminada).
    - El participante ha recibido una vacuna viva atenuada en las 8 semanas previas a la visita basal; o tiene la intención de recibir una vacuna viva atenuada en el trascurso del estudio o en las 8 semanas siguientes a la dosis final de la medicación del estudio.
    - Participante del estudio que haya experimentado reacción de hipersensibilidad después de la exposición a otras drogas contra el receptor Fc neonatal (FcRn).
    - Participante con debilidad grave (definida como grado 3 en la escala de actividades de la vida diaria en la miastenia gravis (AVD-MG)) que afecte a los músculos orofaríngeos o respiratorios, o con crisis miasténica o crisis inminente.
    - El participante tiene un recuento absoluto de neutrófilos <1500 células/mm3
    - El participante tiene cualquier anomalía de laboratorio que, en opinión del Investigador, sea clínicamente significativa, no se haya resuelto en la aleatorización, y pueda poner en peligro o comprometer la capacidad para participar en este estudio
    - El participante tiene un electrocardiograma (ECG) de 12 derivaciones con hallazgos considerados clínicamente significativos tras la revisión médica. La importancia clínica de los hallazgos debe ser evaluada por el Investigador para determinar la elegibilidad, y cualquier pregunta con respecto a la continuación del participante del estudio debe ser abordada con el Monitor Médico
    - El participante del estudio tiene insuficiencia renal, definida como un nivel sérico de creatinina >= 1,4 mg/dL para las mujeres y >=1,5 mg/dL para los hombres.
    - El participante del estudio tiene >2x límite superior normal (ULN) de cualquiera de los siguientes en la Visita 1: alanina aminotransferasa (ALT), aspartato aminotransferasa (AST), fosfatasa alcalina (ALP), o bilirrubina >1.5xULN (se acepta un aumento puntual >1.5xULN si la bilirrubina está fraccionada y la bilirrubina directa <35%).
    Si el participante del estudio tiene elevaciones sólo en la bilirrubina total que es >ULN y <1.5xULN, la bilirrubina fraccionada puede identificar un posible síndrome de Gilbert no diagnosticado (es decir, bilirrubina directa <35%).
    Para los participantes del estudio aleatorizado con un resultado basal >ULN para ALT, AST, ALP, o bilirrubina total, pero <1.5xULN, un diagnóstico basal y / o la causa de cualquier elevación clínicamente significativa debe ser entendido y registrado en el cuaderno electrónico de recogida de datos (eCRF). Las pruebas que den como resultado ALT, AST o ALP hasta un 25% por encima del límite de exclusión (>2xULN) pueden repetirse una vez para su confirmación.
    - El participante del estudio tiene una prueba positiva de anticuerpos del virus de la inmunodeficiencia humana.
    - El participante en el estudio cumplió cualquier criterio de retirada o interrupción del medicamento del estudio MG0003 [NCT03971422] o discontinuó la medicación del estudio MG0003, a excepción de la interrupción por necesitar tratamiento de rescate.
    - El participante del estudio no es considerado capaz de adherirse al calendario de visitas del protocolo, o tomar la medicación a juicio del Investigador.
    - El participante del estudio tiene historial de intento de suicidio (incluyendo un intento activo, intento interrumpido o intento abortado), o tuvo ideas suicidas desde la última visita en el MG0003 como se indica en una respuesta positiva (Sí) a la pregunta 4 o a la pregunta 5 de la Escala Columbia para Evaluar el Riesgo de Suicidio (C-SSRS) durante la selección.
    E.5 End points
    E.5.1Primary end point(s)
    1. Percentage of participants with treatment-emergent adverse events (TEAEs)
    2. Percentage of participants with treatment-emergent adverse events (TEAEs) leading to permanent withdrawal of study medication
    1. Porcentaje de participantes con acontecimientos adversos surgidos durante el tratamiento (AAST).
    2. Porcentaje de participantes con acontecimientos adversos surgidos durante el tratamiento (AAST) que provocan la retirada de la medicacion de estudio
    E.5.1.1Timepoint(s) of evaluation of this end point
    1.+2.: From Baseline until Final Visit (up to Week 60)
    1.+2.: Desde visita basal hasta visita final (hasta semana 60)
    E.5.2Secondary end point(s)
    1. Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at each scheduled assessment during Treatment and Observation Periods.
    2. Change from Baseline in Myasthenia Gravis (MG)-Composite score at each scheduled assessment during Treatment and Observation Periods.
    3. Change from Baseline in Quantitative Myasthenia Gravis (QMG) score at each scheduled assessment during Treatment and Observation Periods.
    4. Percentage of participants using rescue medication (intravenous infusion of immunoglobulin G (IVIg) or plasma exchange (PEX)) during the study.
    1. Cambio desde la visita basal en la puntuación de la escala de actividades de la vida diaria en la miastenia gravis (AVD-MG), en cada evaluación durante los periodos de tratamiento y observación.
    2. Cambio desde la visita basal en la puntuación compuesta de miastenia gravis, en cada evaluación durante los periodos de tratamiento y observación.
    3. Cambio desde la visita basal en la puntuación cuantitativa de la miastenia gravis (QMG) en cada evaluación durante los periodos de tratamiento y observación.
    4. Porcentaje de pacientes que utilizan medicación de rescate (perfusión intravenosa de Inmunoglobulina G o plasmaféresis) durante el estudio.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1.-4.: From Baseline at each scheduled assessment during Treatment and Observation Periods (up to Week 60)
    1.-4.: Desde la visita basal en cada evaluación durante los periodos de tratamiento y observación (hasta la semana 60)
    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, Immunogenicity
    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 No
    E.8.1.1Randomised Yes
    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 No
    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 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA55
    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
    Belgium
    Canada
    Czech Republic
    Denmark
    France
    Germany
    Hungary
    Italy
    Poland
    Russian Federation
    Spain
    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
    LVLS
    Última visita del 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 months6
    E.8.9.1In the Member State concerned days1
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days1
    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: 216
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 24
    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 state16
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 110
    F.4.2.2In the whole clinical trial 240
    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)
    Eligible participants will be allowed receiving expanded access.
    Los participantes elegibles podrán recibir un uso compasivo
    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 Decision2020-02-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-11-13
    P. End of Trial
    P.End of Trial StatusRestarted
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