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    Summary
    EudraCT Number:2019-001564-30
    Sponsor's Protocol Code Number:RA101495-02.301(RAISE)
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-01-07
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2019-001564-30
    A.3Full title of the trial
    A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects with Generalized Myasthenia Gravis
    Studio di fase 3, multicentrico, randomizzato, in doppio cieco, controllato con placebo, volto a confermare la sicurezza, la tollerabilità e l’efficacia di zilucoplan in soggetti affetti da miastenia grave generalizzata
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Patients with Generalized Myasthenia Gravis
    Studio volto a confermare la sicurezza, la tollerabilità e l’efficacia di zilucoplan in soggetti affetti da miastenia grave generalizzata
    A.3.2Name or abbreviated title of the trial where available
    RAISE
    RAISE
    A.4.1Sponsor's protocol code numberRA101495-02.301(RAISE)
    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 SponsorRa 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 supportRa Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRa Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address87 Cambridge Park Drive
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA, 02140
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16174014060
    B.5.5Fax number000000
    B.5.6E-mailtrials@rapharma.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 nameZilucoplan
    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 IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNZilucoplan
    D.3.9.1CAS number 1841136-73-9
    D.3.9.2Current sponsor codeRA101495
    D.3.9.4EV Substance CodeSUB194709
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 Yes
    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 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    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 grave generalizzata
    E.1.1.1Medical condition in easily understood language
    Myasthenia Gravis
    miastenia grave
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10028415
    E.1.2Term Myasthenia
    E.1.2System Organ Class 100000004859
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    •To confirm the efficacy of zilucoplan in subjects with gMG
    •To confirm the safety and tolerability of zilucoplan in subjects with gMG
    • Confermare l’efficacia di zilucoplan in soggetti con gMG
    • Confermare la sicurezza e la tollerabilità di zilucoplan in soggetti con gMG
    E.2.2Secondary objectives of the trial
    na
    na
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IV] at Screening
    2.Positive serology for acetylcholine receptor (AChR) binding autoantibodies
    3.MG-ADL Score of =6 at Screening and Baseline
    4.QMG Score of =12 at Screening and Baseline (off acetylcholinesterase inhibitor therapy for at least 10 hours)
    5.No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the 12-week treatment period
    6.No change in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the 12-week treatment period
    1. Diagnosi di gMG (classe II-IV secondo la classificazione della Myasthenia Gravis Foundation of America [MGFA] [Fondazione americana per la miastenia grave]) allo Screening
    2. Sierologia positiva per autoanticorpi anti-recettore dell’acetilcolina (AChR)
    3. Punteggio MG-ADL =6 allo Screening e al Basale
    4. Punteggio QMG =12 allo Screening e al Basale (dopo sospensione della terapia con inibitore dell’acetilcolinesterasi per almeno 10 ore)
    5. Nessuna variazione nella dose dei corticosteroidi per almeno 30 giorni prima del Basale o prevista durante il Periodo di trattamento di 12 settimane
    6. Nessuna variazione nella terapia immunosoppressiva, inclusa la dose, per almeno 30 giorni prima del Basale o prevista durante il Periodo di trattamento di 12 settimane
    E.4Principal exclusion criteria
    1.Thymectomy within 12 months prior to baseline or scheduled to occur during the 12-week study
    2.History of meningococcal disease
    3.Current or recent systemic infection within 2 weeks prior to Baseline or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Baseline
    1. Timectomia nei 12 mesi precedenti il Basale o in programma durante lo studio di 12 settimane
    2. Anamnesi di malattia meningococcica
    3. Infezione sistemica attuale o recente, entro 2 settimane prima del Basale, o infezione con necessità di terapia antibiotica endovenosa (EV) entro 4 settimane prima del Basale
    E.5 End points
    E.5.1Primary end point(s)
    •Change from Baseline (CFB) to Week 12 in the MG-ADL Score
    • Variazione dal Basale (CFB) alla Settimana 12 nel punteggio MG-ADL
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 12
    settimana 12
    E.5.2Secondary end point(s)
    •CFB to Week 12 in the QMG Score
    •CFB to Week 12 in the MG Composite (MGC)
    •CFB to Week 12 in the MG-QOL15r Survey
    • CFB alla Settimana 12 nel punteggio QMG
    • CFB alla Settimana 12 nel punteggio composito della MG (MGC)
    • CFB alla Settimana 12 nel questionario MG-QOL15r
    E.5.2.1Timepoint(s) of evaluation of this end point
    week 12
    settimana 12
    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 No
    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 Yes
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    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
    United States
    France
    Germany
    Italy
    Norway
    Spain
    United Kingdom
    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
    LVLS
    LVLS
    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 months2
    E.8.9.1In the Member State concerned days21
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months10
    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: 100
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 30
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 28
    F.4.2.2In the whole clinical trial 130
    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)
    At the end of the Treatment Period, all subjects will have the option to receive zilucoplan in a separate Extension Study, provided they meet the Extension Study inclusion criteria
    Alla fine del periodo di trattamento, tutti i soggetti avranno la possibilità di ricevere zilucoplan in uno studio di estensione separato, a condizione che presentino i criteri di inclusione dello studio di estensione
    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 Decision2020-01-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-10-16
    P. End of Trial
    P.End of Trial StatusCompleted
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