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    EudraCT Number:2019-000969-21
    Sponsor's Protocol Code Number:MG0004
    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:2021-01-27
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    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
    Studio di estensione, randomizzato, in aperto volto a valutare la sicurezza, la tollerabilità e l’efficacia a lungo termine di rozanolixizumab in pazienti adulti con miastenia gravis generalizzata
    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
    Studio di estensione di Fase 3 volto a valutare la sicurezza, la tollerabilità e l’efficacia di rozanolixizumab in pazienti adulti con miastenia gravis generalizzata
    A.3.2Name or abbreviated title of the trial where available
    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 SRL
    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 SRL
    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.5.4Telephone number000000
    B.5.5Fax number000000
    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.3.10 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. cell therapy medicinal product Information not present in EudraCT
    D. therapy medical product Information not present in EudraCT
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. 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.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 generalizzata
    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 (MG) è una malattia autoimmune che causa debolezza muscolare, è causata da problemi di comunicazione tra nervi e muscoli.
    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)
    Valutare la sicurezza a lungo termine e la tollerabilità di rozanolixizumab nei partecipanti allo studio con MG generalizzata
    E.2.2Secondary objectives of the trial
    Evaluate the long-term efficacy of rozanolixizumab in study participants with generalized myasthenia gravis (MG)
    Valutare l’efficacia a lungo termine di rozanolixizumab nei partecipanti allo studio con MG generalizzata
    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] or MGC003 at the time of enrollment into either study and the participant either completed
    the Observation Period of MG0003 or MGC003 or required rescue therapy during the Observation Period of the lead-in studies
    - 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
    - I partecipanti devono avere un'età >=18 anni al momento della firma consenso informato
    - Il partecipante era elegibile per MG0003 [NCT03971422] o MGC003 al momento dell’ arruolamento in uno degli studi e ha completato il periodo di osservazione di MG0003 o MGC003 o la terapia di salvataggio richiesta durante il periodo di osservazione degli studi iniziali.
    - Peso corporeo >o uguale a 35 kg alla visita 1
    - I partecipanti allo studio possono essere maschi o femmine
    - Un partecipante maschile allo studio deve accettare di usare la contraccezione
    - Le partecipanti femminili allo studio in età fertile devono accettare di utilizzare un metodo di controllo delle nascite altamente efficace
    - Una donna partecipante ha diritto a partecipare se non è incinta, non allatta al seno e si applica almeno una delle seguenti condizioni:
    a) una donna non in età fertile (WOCBP) OR
    b) Un WOCBP che accetta di seguire la guida contraccettiva
    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 Plus 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 glomerular filtration rate (GFR) less than 60 ml/min/1.73 m^2
    - Study participant has >3x 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%).
    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 MGC003 or discontinued study medication in either study, 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 Columbia Suicide
    Severity Rating Scale (C-SSRS)
    - Evidenza di tubercolosi attiva o latente (TB) come documentato da storia medica ed esami, se applicabile, radiografia del torace (posteriore anteriore e laterale) e test TB tramite test QuantiFERON®-TB Gold plus positivo (non indeterminato)
    - Il partecipante ha ricevuto una vaccinazione viva entro 8 settimane prima della visita basale; o intende sottoporsi a una vaccinazione viva nel corso dello studio o entro 8 settimane dalla dose finale del farmaco in studio
    - I partecipanti allo studio hanno manifestato una reazione di ipersensibilità dopo esposizione ad altri farmaci antineonatali del recettore Fc (FcRn)
    - Partecipante allo studio con grave debolezza (definita come Grado 3 sulla attività quotidiane in presenza di MG (scala MG-ADL)) che colpisce muscoli orofaringei o respiratori o con crisi miastenica o crisi imminente
    - Il partecipante ha una conta assoluta dei neutrofili <1500 cellule / mm3
    - Il partecipante presenta anomalie di laboratorio che, a giudizio dello sperimentatore, è clinicamente significativo, non si è risolto alla randomizzazione e potrebbe compromettere la capicità del soggetto di partecipare a questo studio
    - Il partecipante ha un elettrocardiogramma a 12 derivazioni (ECG) con risultati clinicamente significativi al momento della revisione medica. Il significato clinico dei risultati deve essere valutato dallo sperimentatore che ne deve determinare l'idoneità e qualsiasi domanda relativa alla prosecuzione dello studio da parte del soggetto, deve essere indirizzata al Medical Monitor
    - Il partecipante allo studio ha insufficienza renale, definita come velocità di filtrazione glomerulare ( VFR) minore di 60 ml/min/1.73 m^2
    - Il partecipante allo studio ha il limite superiore normale (ULN) > 3x per uno dei seguenti parametri alla visita 1: alanina aminotransferasi (ALT), aspartato aminotransferasi (AST), fosfatasi alcalina (ALP) o bilirubina > 1,5xULN (bilirubina isolata > 1,5xULN è accettabile se la bilirubina è bilirubina frazionata e diretta <35%).
    - Il partecipante allo studio ha un test per anticorpo del virus dell'immunodeficienza umana positivo
    - Il partecipante allo studio ha i requisiti di ritiro obbligatorio dallo studio o criteri di sospensione del farmaco MG0003 [NCT03971422] o MGC003 o ha interrotto il trattamento in entrambe gli studi, ad eccezione della sospensione dovuta a una necessità di cure di salvataggio
    - Il partecipante allo studio non è considerato in grado di aderire al programma di visita del protocollo o assumere di farmaci secondo il giudizio dello sperimentatore
    - Il partecipante allo studio ha una storia di tentativi di suicidio nel corso della vita (incluso un tentativo attivo, tentativo interrotto o abortito) o ha ideazione di suicidio dall'ultima visita dello studio MG0003 come indicato da un risposta positiva (Sì) alla domanda 4 o alla domanda 5 delal scala della Columbia University per la valutazione della gravità del rischio di suicidio (C-SSRS) .
    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. Percentuale di partefcipanti con evento avverso emergente dal trattamento (TEAEs)
    2. Percentuale di partefcipanti con evento avverso emergente dal trattamento (TEAEs) che conducono alla interruzione permanente del farmaco in studio
    E.5.1.1Timepoint(s) of evaluation of this end point
    1.+2.: From Baseline until End of Study (up to Week 60)
    1.+2.: Dal basale sino alla Fine dello Studio ( sino alla settimana 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 -Composite (MG-C) 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
    4. Percentage of participants using rescue medication (intravenous infusion of immunoglobulin G (IVIg) or plasma exchange (PEX)) during
    the study
    1. Valore e variazione rispetto al basale del punteggio MG-ADL a ogni valutazione programmata durante i periodi di trattamento e osservazione
    2. Valore e variazione rispetto al basale del punteggio composito della MG ( MG-C) a ogni valutazione programmata durante i periodi di trattamento e osservazione
    3. Valore e variazione rispetto al basale del punteggio QMG a ogni valutazione programmata durante i periodi di trattamento e osservazione
    4. Percentuale dei partecipanti che usano il farmaco di soccorso (infusione endovenosa di immunoglobuline G (IVIg) o scambio di plasma (PEX) durante lo studio.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1.-4.: From Baseline during Treatment and Observation Periods (up to Week 60)
    1.-4.: Dal basale durante i Periodi di Trattamento e di Osservazione ( sino alla settimana 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
    Tollerabilità, 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. 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 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) Yes
    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 concerned8
    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 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
    Russian Federation
    United States
    United Kingdom
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months7
    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 months5
    E.8.9.2In all countries concerned by the trial days24
    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: 248
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 28
    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 state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 110
    F.4.2.2In the whole clinical trial 276
    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
    Ai pazienti ideoni sarà consentito di ricevere il trattamento per mezzo di programmi di accesso allargato.
    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 Decision2020-01-15
    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 StatusOngoing
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